11th Annual Sickle Cell Disease and Thalassaemia Conference (ASCAT) 2017
This event has now taken place.
11-13 October 2017
The 11th Annual Sickle Cell and Thalassaemia Conference will be held at:
etc. Venues Prospero House
241 Borough High Street
Pictures of this year’s event can be found below:
The 2017 Annual Scientific Conference on Sickle Cell and Thalassaemia is one of the must attend events of the year for consultants and specialist psychologists, nurses, scientists and all relevant experts. This is the 11th consecutive opportunity to see the latest advances in diagnosis, treatment and emerging fields in haemoglobinopathies. It is an opportunity to interact on the latest advances in clinical care, transition services and emerging new therapies including updates for curative treatment options. Furthermore, you will have the opportunity to show case your work through abstracts (oral and posters) and to network with leading and cutting edge practitioners.
This year’s theme is ‘Patient choice in a changing landscape of treatment and cure for sickle cell and thalassaemia’. This will include case scenarios, expert and patient opinion, and educational sessions. During the three days there will be sessions on genetics and genomic progress, curative therapies and emerging services.
The abstract and poster presentations will take place during the three days. We are supported by a record number of sponsors with exhibitions taking place throughout the event. Among the highlights we will have a book launch for ‘Sickle Cell Disease – Pain and Common Chronic Complications’ and a review of the first ten years of the Annual Scientific Conference on Sickle Cell and Thalassaemia.
The Conference is hosted by Evelina London Children’s Hospital, Guy’s and St Thomas’ NHS Foundation Trust.
Who should attend?
This three day conference is aimed at all those with a common interest in sickle cell and thalassaemia, including:
- Scientists and clinicians in all areas of haemoglobinopathies including
- Consultant haematologists / Paediatricians / Psychologists/Scientists
- Fellows and trainees in all related fields
- There will be patient led sessions and opportunity for interactions
20 CPD/CME points have been accredited from the Royal College of Paediatrics and Child Health and the Royal College Pathologists.
Day 1 – 11 October 2017
09.00-11.00 Session 1 – Overview of sickle cell disease (Illuminate Room)
09.00-09.05 – Welcome Address, Baba Inusa, Paediatric Haematology, Evelina London Children’s Hospital, Guy and St Thomas’ NHS Foundation Trust
09.05-10.00 Chair: Dr Mbololwa Mbikusita-Lewanika BSc, MSc, PhD, AKC, Health Adviser.
Introduction- report of the first 10 years – Baba Inusa, Paediatric Haematology, Evelina London Children’s Hospital, Guy and St Thomas’ NHS Foundation Trust
– Dr. Matt Heeney, Associate Chief, Hematology, Director, Sickle Cell Program, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center
– Dr. Mona Hamdy, Professor of Pediatrics, Cairo University and Consultant of Hematology at Ministry of Health, Egypt
- Dr. Jo Howard, Consultant Haematologist and Clinical Lead/ /Honorary Reader in Haemoglobinopathies, Guy’s and St Thomas’ NHS Foundation Trust
Book launch- Review of Digital chapter- Dr Stephan Lobitz, Paediatric Haematologist, Amsterdam Street Children’s Hospital, Cologne
10.00-10.35 Dr. Winfred Wang, Member, St. Jude Children’s Research Hospital
Sickle Cell Disease: An overview
10.35-11.00 Dr Victoria Rathbone, Pathology, Sebastian Lucas, Morbid Anatomist, Guy’s and St Thomas’ NHS Foundation Trust Pathological changes in SCD
11.00-11.30 Refreshment Break
11.30-13.35 Session 2 – Plenary session – ASH 2016 Highlights – Innovation in Sickle Cell Disease (Illuminate Room)
11.30-11.40 Chair: Dr Alexis Thompson, Hematology Section Head, Ann & Robert H. Lurie Children’s Hospital of Chicago
Welcome and introduction
11.40-11.55 Dr Russell Ware, Director, Division of Hematology, Institute Co-Director, Cancer and Blood Diseases Institute, Associate Director, Global Health Center, Marjory J. Johnson Chair of Hematology Translational Research
Using hydroxyurea in Low Resource Settings
11.55-12.25 Dr Miguel Abboud, Professor and Chairman, Department of Pediatrics and Adolescent Medicine, American University of Beirut – Medical Center, Children’s Cancer Center of Lebanon, Beirut, Lebanon
New therapies for Sickle Cell Disease
12.25-12.55 Dr Deepa Manwani, Associate Professor, Hematology Section Head, Children’s Hospital at Montefiore, Albert Einstein College of Medicine, Bronx, NY
Role of Neutrophil Mac-1 activation in SCD Pathophysiology and as a Potential Therapeutic Target
12.55-13.35 Dr Kenneth Ataga, Professor of Medicine and Director, University of North Carolina Comprehensive Sickle Cell Program
SUSTAIN: A Multicenter, Randomized, Placebo-Controlled, Double-Blind, 12
Month Study to Assess Safety and Efficacy of SelG1 with or without Hydroxyurea
Therapy in Sickle Cell Disease Patients with Sickle Cell-Related Pain Crises
13.35-14.30 Lunch, Exhibition and Networking
13.50-14.20 Sponsored Session: Dr. Andrew Dunham, Hemanext (Illuminate Room)
Control of Oxidative damage in RBC transfusion
14.30-15.45 Session 3 – Parallel sessions
A – Complex Issues in Adults with Sickle Cell Disease (Illuminate Room)
14.30-14.35 Chair: Dr Biree Andemariam, Associate Professor, Hematology/Oncology, Director, New England Sickle Cell Institute, Director, Connecticut Bleeding Disorders Center, University of Connecticut, US
Welcome and introduction
14.35-15.00 Dr. Kathryn Hassell, Director of Colorado Sickle Cell Treatment and Research Centre, University of Colorado
15.00-15.25 Dr. Jo Howard, Consultant Haematologist and Clinical Lead, Honorary Reader in Haemoglobinopathies, Guy’s and St Thomas’ NHS Foundation Trust
The older patient with multi-organ dysfunction
15.25-15.45 Dr. Claire Sharpe, King’s College Hospital
B – Complex Issues in Adults with Thalassaemia (Motivate Room)
14.30-14.35 Chair: Dr Giovanna Russo: Head of the Pediatric Hematology Oncology of the University of Catania
14.35-15.10 Dr. Adriana Ceci, former Professor of Paediatrics at the University of Bari, President of the Gianni Benzi Pharmacological Research Foundation
Deferiprone in children: novel safety data and comparison with adults
15.10-15.45 Dr. Gian Luca Forni, Università di Cagliari, Italy
Efficacy and safety of sildenafil in the treatment of severe pulmonary hypertension in patients with haemoglobinopathies
15.45-16.00 Refreshment Break
16.00-17.15 Session 4 – Parallel sessions continued
A – Complex Issues in Adults with Sickle Cell Disease (Illuminate Room)
16.00-16.25 Dr Praveen Anand, Professor of Clinical Neurology and Head, Centre for Clinical Translation, at Hammersmith Hospital, Imperial College London
Chronic Pain: A diagnostic algorithm of Neuropathic syndromes
16.25-16.50 Dr Deepika Darbari, Attending Physician, Division of Hematology, Center for Cancer and Blood Disorders, Children’s National Medical Center,
Associate Professor of Pediatrics, George Washington University School of Medicine and Health Sciences, Washington, D.C.
Thinking beyond sickling for effective management of pain in SCD
16.50-17.15 Dr Caterina Minniti, Professor of Clinical Medicine and Pediatrics,
Einstein College of Medicine
B – Effective Partnership with service users for advocacy (Motivate Room)
16.00-16.05 Chair: Ms Joy Enahoro and Mr George Constantinou, Assistant Secretary on the Board of Directors of the Federation, Secretary of the UK Thalassaemia Society
16.05-16.20 Prof. Ambroise Wonkam, Specialist Medical Geneticist, Division of Human Genetics, Faculty of Health Sciences, University of Cape Town, South Africa
Abstract – Clinical and genetic factors are associated with pain and hospitalization rates in sickle cell anaemia in Cameroon
16.20-16.35 Mrs Shifneez Shakir, Patient Advocate for Thalassaemia & Sickle Cell
A patient experience with hydroxyurea
16.35-17.00 Mr George Constantinou, Assistant Secretary on the Board of Directors of the Federation, Secretary of the UK Thalassaemia Society and Ms Roanna Maharaj
Iron Chelation therapy in thalassaemia – A Patient’s Journey
17.00 -17.15 Cathy Coppinger, NHS Sickle Cell and Thalassaemia Screening Programme Manager, PHE Screening – Learning from Parents’ Stories
17.15 Conference close
Day 2 – 12 October 2017
09.00-11.15 Session 1 – Plenary session
A – Education – Emerging themes in Sickle Cell Disease (Illuminate Room)
09.00-09.15 Chair: Dr Beatrice Gulbis
Welcome and Introduction
09.15-09.45 Dr Jacques Elion
Chasing Sickle cell disease globally: Capacity Building
09.45-10.15 Dr Yvonne Daniel, Specialist Lead Scientist, Haematological Sciences and Special Haematology Lead at Viapath, Guy’s & St Thomas’ Hospital London
Newborn screening for sickle cell disease through use of tandem mass spectrometry
10.15-10.45 Dr Mariane de Montalembert, Necker Hospital
First consultations after neonatal screening, what must be said and when?
10.45- 11.15 Dr Kalpna Gupta, University of Minnesota Medical School
Pros and cons of chronic opioid use
B – Education session on Thalassaemia (Motivate Room)
09.00-09.40 Chair: Dr Antonio Piga, Professor of Pediatrics at Turin University
Welcome and introduction
A genetic score for the prediction of beta-thalassemia severity
09.40-10.15 Dr Renu Saxena
Thalassemia Intermedia in India: genotypic phenotypic correlations
10.15-10.45 Dr Stephan Menzel
Control of Fetal Haemoglobin: genomic and clinical implications in thalassaemia and Sickle cell disease
10.45-11.15 Dr Paul Telfer, Haematologist, Barts Health NHS Trust
Survival of patients with thalassaemia over the last 20 years
11.15-11.30 Refreshment Break
11.30-13.35 –Session 2 – Parallel sessions
A – Approaches to Monitoring End Organ Damage within Pediatric SCD Patients (Illuminate Room)
11.30-11.35 Chair: Dr Andrew Campbell, Haematology, Director, Comprehensive Pediatric Sickle Cell Disease Program, Children’s National Medical Center, George Washington University School of Medicine, Washington, DC
Welcome and introduction
11.35-12.00 Dr Raffaella Colombatti, Clinic of Pediatric Hematology Oncology, Azienda Ospedaliera – University of Padova
Coagulation activation and cerebral vasculopathy in SCD
12.00-12.25 Dr Maria Pelidis, Consultant Haematologist and Oncologist, Evelina London Children’s Hospital
Enuresis in SCD: A consensus for management
12.25-12.50 Dr Andrew Campbell, Haematology, Director, Comprehensive Pediatric Sickle Cell Disease Program, Children’s National Medical Center, George Washington University School of Medicine, Washington, DC
End organ monitoring in paediatric patients-renal and growth
12.50-13.10 Dr Corinna McMahon
Acute Chest syndrome in children: A management protocol
13.10-13.35 Dr Mark Velangi, Consultant Paediatric Haematologist, Birmingham Children’s Hospital
Hyperhaemolysis -consensus on diagnosis/management
B – Thalassaemia – symposium (Motivate Room)
11.30-12.10 Dr Mona Hamdy, Professor of Pediatrics, Cairo University, Cairo
Prenatal diagnosis for thalassaemia in Egypt: What changed parents’ attitude?
12.10-12.50 Prof Antonis Kattamis
Pregnancy in patients with thalassemia major: a cohort study and conclusions for an adequate care management approach
12.50-13.35 Dr Antonio Piga, Professor of Pediatrics at Turin University
Ageing in thalassaemia
13.35-14.35 Lunch, Exhibition and Networking
14.00 – 14.30 Sponsored Session – Dr Paul Telfer (Illuminate Room)
Implementation of automated red blood cell exchange in a new center to improve patient care
Sponsored by: Terumo BCT
14.35-17.20 Session 3 – Parallel sessions – Abstracts, SCD and thalassaemia
A – Sleep disorders and cardiopulmonary complications in SCD (Illuminate Room)
14.35-14.40 Chair: Dr Fenella Kirkham, University College London
Welcome and introduction
14.40-14.55 Dr Michele Ariglianai, Pediatrician, University Hospital of Udine, Italy
Abstract – Respiratory impairment in children with sickle cell anaemia
14.55-15.25 Dr Anne Greenough, Professor of Clinical Respiratory Physiology, King’s College London
Do patients with sickle cell disease have asthma?
B – Challenges to care in Sickle Cell and Thalassemia: Assessment and intervention (Motivate Room)
14.35-14.45 Chair: Dr Marsha Treadwell, Clinical Scientist, UCSF Benioff Children’s Hospital Oakland and Co-Principal Investigator, Pacific Sickle Cell Regional Collaborative
Welcome and introduction
14.45-15.00 Karen Smith Wong
Abstract – Experienced RN Coordination of Pediatric Sickle Cell Patients in the Office and Outpatient Clinic Setting: A Comprehensive, Pro-Active Nursing Model
15.00-15.40 – Dr Anne Gordon, Newcomen Centre, GSTT, London and Vijeya Ganesan
Guidelines for stroke management – Acute care and rehabilitation for Sickle Cell Stroke
15.40-15.50 Refreshment Break
15.50-17.20 Session 3 – Parallel sessions continued
A – Sleep disorders and cardiopulmonary complications in SCD (Illuminate Room)
15.50-16.20 Dr Elizabeth Klings, Boston University School of Medicine
Pulmonary hypertension, obstructive sleep apnoea and the endothelium in sickle cell disease
16.20-16.50 Dr Julie Kanter, Director of Sickle Cell Disease Research, The Medical University of South Carolina
Sleep disruption in sickle cell disease
16.50-17.20 Dr Fenella Kirkham, University College London
Sleep, cognition and brain structure
B – Challenges to care in Sickle Cell and Thalassemia: Assessment and intervention (Motivate Room)
15.50-16.20 Dr Kimberley Wesley, Paediatric Psycologist, Children’s Hospital, Philadelphia
Caregiver Perspectives of Stigma Associated With Sickle Cell Disease in Adolescents
16.20-16.50 Dr Heather Rawle, Consultant Clinical and Health Psychologist at Guys and St Thomas’ Hospital Foundation NHS Trust (GSTT)
Enhancing adherence – latest research and practical tips
16.50-17.20 Dr Lewis Hsu, Professor of Pediatrics and Director of Pediatric Sickle Cell at the University of Illinois at Chicago
Community Health Worker Support; Health Maintenance
17.20 – Conference Close
17.20-18.30 – Poster Walk (Main Reception)
19.30-23.00 Conference dinner (by invitation only)
Day 3 – 13 October 2017
08.30-11.30 Session 1 – Parallel sessions – Training and Nursing sessions
A – Haemoglobinopathy morphology and clinical case scenarios (Illuminate Room)
08.30-8.35 Chair: Dr Rachel Kesse-Adu, Consultant Haematologist, Guy’s and St Thomas’ NHS Foundation Trust
Welcome and introduction
08.35-08.50 Yvonne Brew
Abstract – Prevelance of Haemoglobin S and C Among babies according to parental ethnic groups in Ghana
08.50-09.15 Dr Vishal Jayakar, Consultant Haematologist, Kingston NHS Foundation Trust and Honorary Senior Lecturer, Imperial College London
Playing Sherlock Holmes!
09.15-09.40 Dr Chris Lambert, Service Delivery Manager – Haematology, Haematology Department, King’s College Hospital
Laboratory diagnosis of Haemoglobinopathies (with HPLC cases)
09.40-10.05 Dr Emma Drasar, Consultant Haematologist, Whittington Hospital NHS
Thalassaemia case scenarios
10.05-10.30 Dr Rachel Kesse-Adu, Consultant Haematologist, Guy’s and St Thomas’ NHS Foundation Trust
Sickle Case Studies
10.30-11.00 Dr Banu Kaya, The Royal London and St Bart’s NHS Trust, London
Paediatric haemoglobinopathy case studies
B – Nursing (Motivate Room)
08.30-09.05 Chair: Mrs Nkechi Anyanwu, Clinical Nurse Manager (Haemoglobinopathies), Sickle Cell & Thalassaemia Community Services
Welcome and introduction/ Role of the community nurse
09.05-09.20 Samuel Mbunya
Abstract – Outreach programs and advocacy of Sickle Cell Disease Care in Kenya
09.20-09.50 Miss Sharon Ndoro, Senior Research Nurse, Guy’s and St Thomas’ NHS Foundation Trust and Bessie Crone, Senior Paediatric Research Nurse, Bart’s Health NHS Trust
Research Expertise for Nurses- Case Studies
09.50-10.20 Mr Luhanga Musumadi, Advanced Nurse Practitioner Haemoglobinopathies & Lead nurse for Adolescent Transition, Guy’s and St Thomas’ NHS Foundation Trust
Dr Hatel Bhatt, Clinical Specialist, Counselling Psychologist, St Thomas Hospital
Assessing readiness for transition from paediatric to adult centred care using patient passport
10.20-11.00 Mrs Olukemi Ajamufua, Guys and St Thomas’ Hospital, Sickle Cell and Thalassaemia Centre
Antenatal Counselling for haemoglobinopathies – Case Studies
11.00 – 11.30 Refreshment Break
11.30-13.30 Session 2 – Parallel Session
A – Global Perspectives (Illuminate Room)
11.30-11.55 Dr Alexis Thompson, Hematology Section Head, Ann & Robert H. Lurie Children’s Hospital of Chicago
Sickle cell Action- ASH global Agenda
11.55-12.20 Dr Ambroise Wonkam, Specialist medical geneticist, in the Division of Human Genetics, Faculty of Health Sciences, University of Cape Town, South Africa
ONTOLOGY in Sickle Cell Disease
12.20-12.40 Dr Kwaku-Ohene-Frempong, Children’s Hospital, Philadelphia
Sickle Cell Africa
12.40-13.00 Regine Grosse
How to measure iron overload beyond liver and heart
B – Educational session (Motivate Room)
11.30-12.00 Dr Barnaby Clark, Consultant Clinical Scientist, King’s College Hospital and Honorary Senior Lecturer, King’s College London University
Application of Next Generation Sequencing to Haemoglobinopathy Diagnosis Genome editing landscape- contemporary laboratory applications and therapeutic potential
12.00-12.15 Dr Marina Kleanthous, Professor, The Cyprus School of Molecular Medicine. Head, Molecular Genetics Thalassaemia Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus
Gene editing: Mechanisms of gene expression during blood cell development
12.15-12.30 Dr Carsten W. Lederer, Researcher, Department of Molecular Genetics Thalassaemia, Cyprus Institute of Neurology and Genetics (CING)
Genome editing for the therapy of beta-haemoglobinopathies
12.30-13.00 Dr John Porter, Professor of Haematology and Consultant Haematologist, University College Hospital
Optimizing Iron chelation in haemoglobinopathies
13.00-14.00 Lunch, Exhibition and Networking
14.00-15.20 Session 3 – Patient Perspective – Plenary Session (Illuminate Room)
14.00- 14.05 – Chairs: Dr. Moji Awogbade, Haematologist, King’s College Hospital, London and Dr. Mariane de Montalembert, Associate Professor of Pediatrics, Necker Hospital.
14.05- 14.20 Yaa Kufuor – A mother’s perspectives of the bone marrow transplant experience
14.20-17.00- Debate: A cure for Sickle Cell disease or Thalassaemia Is the Priority over comprehensive care! : Yes and NO!
With increasing competing demand between comprehensive care sickle cell disease and thalassaemia considering emerging prospects of a cure what are the choices for service users, providers and commissioners? In view of these competing priorities we seize this opportunity to bring leading experts to lead the DEBATE:
Yes Search for cure should be the priority!
- Dr Marsha Treadwell, Clinical Psychologist and Clinical and Health Services Researcher, UCSF Benioff Children’s Hospital Oakland and
- Dr Marina Cavazanna, Director of Hematology, Head of Biotherapy Department, Necker Hospital
No, Cure cannot be the prority!
- Dr Jerlym Porter, Assistant Member, Department of Psychology, St. Jude Children’s Research Hospital and
- Dr Russell Ware, Director, Division of Hematology, Institute Co-Director, Cancer and Blood Diseases Institute, Associate Director, Global Health Center, Marjory J. Johnson Chair of Hematology Translational Research
15.20 – 15.35 Refreshment Break
15.35 – 17.00 Session 4 – Patient Perspective – Plenary Session (cont.) (Illuminate Room)
Debate (cont.): A cure for Sickle Cell disease or Thalassaemia Is the Priority over comprehensive care! : Yes and NO!
17.00 Conferences close
Sessions and times are subject to change.
The full programme will also be available at www.scorecharity.com.
Dr. Baba Inusa (chair), Paediatric Haematology, Evelina London Children’s Hospital, Guy’s and St Thomas’ NHS Foundation Trust
Dr. Biree Andemariam, Associate Professor, Hematology/Oncology, Director, New England Sickle Cell Institute, Director, Connecticut Bleeding Disorders Center, University of Connecticut, USA
Dr. Andrew Campbell, Haematology, Director, Comprehensive Pediatric Sickle Cell Disease Program, Children’s National Medical Center, George Washington University School of Medicine, Washington, DC
Dr. Raffaella Colombatti, Azienda Ospedaliera – University of Padova, Italy
Dr. Rachel Kesse-Adu, Haematology, Guy’s and St Thomas NHS Trust
Dr. Marsha Treadwell, Northern California Network of Care for Sickle Cell Disease, UCSF Benio Children’s Hospital Oakland
Dr. Barnaby Clark
Dr Barnaby Clark (PhD) is currently a Consultant Clinical Scientist at King’s College Hospital and Honorary Senior Lecturer at King’s College London University.
He joined the prenatal diagnosis unit at Kings College Hospital in 2002 after completing a post-doctoral research study examining genetic factors that influence Haemoglobin F levels. Within Kings he has developed numerous molecular assays to enhance the diagnosis of haemoglobinopathies. In 2008, he was made head of the laboratory and his lab has just released a next generation sequencing gene panel for the diagnosis of rare anaemia and iron overload. He has been a member of the laboratory subcommittee for the National Programme for Sickle Cell and Thalassaemia for four years providing advice on issues affecting prenatal testing. He is also committed to teaching the laboratory diagnosis of Haemoglobinopathies, being part of the PEGASUS counsellor training course and the STSN junior doctor training programme.
Professor and Chairman, Department of Pediatrics and Adolescent Medicine, American University of Beirut – Medical Center, Children’s Cancer Center of Lebanon, Beirut, Lebanon.
In August 2002, he became the first Medical Director of the Children’s Cancer Center of Lebanon (CCCL) at the American University of Beirut Medical Center in Lebanon as well as and Professor of Pediatrics at AUB. During his tenure, he established highly visible and successful bone marrow transplant, limb salvage surgery, and sickle cell disease programs as well as an acute lymphoblastic leukemia research protocol in collaboration with the Leukemia/Lymphoma Division at St. Jude Children’s Research Hospital in Memphis, TN, USA. The sickle cell program at AUBMC has grown and now provides comprehensive service to over 300 children and adolescents with this disease. The program research focuses now on pulmonary hypertension and the vasculopathy in sickle cell disease. In February 2011, he was appointed as Chairman of the Department of Pediatrics and Adolescent Medicine at AUBMC.
Kemi currently work as a Community Sickle cell & Thalassaemia Specialist Nurse at Guys and St Thomas’ Hospital NHS Foundation Trust, which includes the Evelina Children’s Hospital, London.
Kemi has gathered over a decade’s experience in working and supporting clients with haemoglobinopathies as well as their families / carers. Kemi’s role involves counselling pregnant women and their partners within the antenatal period and carrying out new- birth visits for families affected with haemoglobinopathy.Kemi also provides screening and counselling for individuals who would like to know their genotype and she is involved in raising awareness about sickle cell and thalassaemia within the wider community and “at risk” population.
Prior to her current role, Kemi previously worked as a High Risk Midwife with a special interest in caring for women with haemoglobinopathies.
Kemi is currently the Deputy Treasurer for the Sickle cell and Thalassaemia association for Nurses, Midwives and Allied Professional (STANMAP).
Dr. Praveen Anand
Prof. Anand is Professor of Clinical Neurology and Head, Centre for Clinical Translation, at Hammersmith Hospital, Imperial College London. His medical education was at the Universities of Oxford and Cambridge, and post-graduate training at the Hammersmith Hospital and the National Hospital for Neurology and Neurosurgery, Queen Square, London. His research focuses on pathophysiological and molecular mechanisms in human sensory neuropathies and chronic pain syndromes. Collaborations with colleagues and pharmaceutical companies are directed to projects which bridge the gap between pre-clinical developments and their successful clinical applications. The translational approach has guided the recent success of 4 novel drugs from the laboratory to Phase II trials for chronic neuropathic pain and itch. He has over 200 peer-reviewed publications in journals including Nature, Nature Medicine, Nature Genetics, Science and The Lancet.
Dr. Biree Andemariam
Associate Professor, Hematology/Oncology, Director, New England Sickle Cell Institute, Director, Connecticut Bleeding Disorders Center, University of Connecticut, U.S.A.
Biree Andemariam, M.D. is founding director of the New England Sickle Cell Institute (NESCI) and directs the Connecticut Bleeding Disorders Center. She received her undergraduate degree in molecular biology and African studies from Princeton University, her medical degree with research honors from Tufts University School of Medicine, and her medical training at Cornell University. She is Chief Medical Officer of the Sickle Cell Disease Association of America.
Dr. Kofi Anie
Consultant Psychologist & Honorary Clinical Senior Lecturer London North West Healthcare NHS Trust & Imperial College London
Dr Kofi Anie is the Psychology Service Lead for children and adults with sickle cell disease at London North West Healthcare NHS Trust. He trained at the University of Surrey and University of London, and began his professional career at King’s College Hospital, London where he pioneered psychological interventions for children with sickle cell disease. He was later a Research Fellow at St George’s University of London, and was appointed to his current position in 1998. He is a Fellow of the Royal Society of Medicine and Associate Fellow of the British Psychological Society; his other professional affiliations include the British Association of Behavioural and Cognitive Psychotherapies, and American Psychological Association. Dr Anie’s research interests include Sickle Cell Pain, Quality of Life, Adherence to Treatment, and Mobile Technology in Patient Self-Management. He is currently a collaborator on several international initiatives including the USA, Ghana, Nigeria, and South Africa.
I am the Service Manager and Clinical Lead for South East London Community Sickle & Thalassaemia services, for Guy’s and St Thomas’ NHS Trust covering the boroughs of Lambeth, Southwark, Lewisham and Greenwich & Bexley.
I am currently the Vice chair for Sickle Cell and Thalassaemia Association of Nurses, Midwives and Allied Professionals ( STANMAP); and the co-lead for Nursing in the South Thames Sickle Cell & Thalassaemia Network ( STSTN).
I have worked with clients, families and carers with Sickle Cell and Thalassaemia, since 1998 as the senior haemoglobinopathy nurse counsellor at Whittington Health Trust and have a wide range of experiences in the field.
My role includes providing management, professional and clinical leadership to a team of skilled specialist haemoglobinopathy nurses, in the delivery of high-quality integrated community based, nursing service. We increase patients coping strategies in the community which helps to reduce their hospital admission rate. I advocate for clients with major haemoglobinopathies and strive to raise awareness amongst the local population.
Michele was born in Benevento, Italy, in 1985. He graduated in Medicine in 2010 at University Campus Biomedico of Rome and com pleted his training in general pediatrics in 2016 at the University Hospital of Udine, Italy, where he is currently employed in the Division of Pediatrics. During his training he had two short-term research fellowships at Evelina London Children’s Hospital and at the Great Ormond Street Hospital. He is also a PhD student at the University of Udine, with a project on the use of the Multiple Breath Washout for lung function assessment of preterm born children at schoolage. His research interests are mainly focused on respiratory physiology and respiratory impairment in sickle cell disease. He co-authored 7 papers published in international, peer-reviewed journals and more than 20 abstracts at international meetings, most of them as first author.
Dr. Kenneth Ataga
Dr. Kenneth Ataga is an adult hematologist with a clinical and research interest in sickle cell disease. He attended medical school at the University of Benin, Benin City, Nigeria, followed by a residency in Internal Medicine at Upstate Medical University (formerly State University of New York Health Sciences Center) at Syracuse, NY and a fellowship in Hematology-Oncology at the University of North Carolina at Chapel Hill.
His clinical research is focused on the development of new therapies and the vasculopathy of sickle cell disease, with an emphasis on renal complications, pulmonary hypertension and coagulation activation. He is presently a Professor of Medicine and Director of the Comprehensive Sickle Cell Program at the University of North Carolina at Chapel Hill, North Carolina, USA.
Dr. Moji Awogbade
Dr. Awogbade is a haematologist with an interest in sickle cell disease. Her specific interests include the evolving understanding of the pathophysiology of sickle cell disease and how this translates into effective therapies.
She has been a consultant haematologist at King’s College Hospital, London, UK, since 2003. She earned her medical degree from the University of Lagos Nigeria, and earlier obtained an Intercalated Bachelors degree in clinical pathology. She completed general medical and specialist training in haematology in the UK. She is a Fellow of the Royal College of Pathologists and Fellow of the Royal College of Physicians. She was a contributing author to the Standards for the Clinical Care of Adults with Sickle Cell Disease in the UK.
Hatel Bhatt is a Counselling Psychologist in the Paediatric Sickle Cell team at the Evelina Children’s Hospital. She is passionate about supporting children and young people who have a diagnosis of Sickle Cell. Within her psychology role, she offers neuropsychology assessments and individual and family therapy sessions. In addition to this, she also facilitates liaison and teaching to schools and other professionals. Hatel has a special interest in working with schools and other professionals supporting children in the community, with the aim to help provide holistic care for patients and raise the awareness of sickle cell and Thalassemia. She is also keen to explore the cultural differences in families’ understanding of their child’s diagnosis, and experiences of using psychological therapies to help with the management of pain. Prior to this role, for the past 6 years, Hatel has worked in a number of child and adolescent mental health services (CAMHS) across London.
Dr. Marina Cavazzana
Marina Cavazzana is a paediatrician, Professor of Haematology since 2000, Director of the Department of Biotherapy at Hospital Necker, University Paris Descartes. She is the Director of the Inserm / Assistance Publique – Hôpitaux de Paris GHU Ouest Biotherapy Clinical Investigation Center and leads a research Laboratory at Imagine Institute. She studied medicine in Padua, Italy and received the degree of Doctor of Medicine in 1983, her certification in Paediatrics in 1987 and a PhD in Life Sciences in 1993 (University Paris VII).
Dr. Adrianna Ceci
Adriana Ceci, former Professor of Paediatrics at the University of Bari, is President of the Gianni Benzi Pharmacological Research Foundation, a registered “not-for-profit” scientific research organisation. She has been member of the European Medicines Agency’s Paediatric Committee – PDCO (2008-2014). She is member of many scientific and management Bodies in many EC- funded projects: CloSed (Clonidine for Sedation of Paediatric Patients in the Intensive Care Unit, 602453-FP7-HEALTH-2013-INNOVATION-1); InNerMeD-I-Network (Inherited NeuroMetabolic Diseases Information Network, 2012 12 12, Second Health Programme); GRiP (Global Research in Paediatrics, 261060-FP7- HEALTH-F5-2010); DEEP (DEferiprone Evaluation in Paediatrics, 261483-FP7-HEALTH-2010); GAPP (GAbapentin in Paediatric Pain, 602962 – FP7-HEALTH-2013-INNOVATION-1), TEDDY Network of Excellence (Task-Force in Europe for Drug Development for the Young, FP6, LSHB-CT-2005-005216).
Dr. Raffaella Colombatti
Dr.Raffaella Colombatti is pediatric hematologist oncologist at the Pediatric Hematology-Oncology Unit of the Azienda Ospedaliera-Università di Padova, in Padova, Italy. Her main field of interest are Red Cell Disorders, especially Sickle Cell Disease (SCD). She has contributed in creating the Sickle Cell Veneto Region Reference Center in Padova and is clinical coordinator of the local Universal Newborn Screening Program; she is in charge of the General Hematology-Anemias clinic. In the past years she has been involved in clinical research on SCD vasculopathy, cerebral and neurocognitive abnormalities and brain function.
Dr.Colombatti is also interested in Child Health in Africa and is leading several projects in Guinea Bissau, West Africa, in the field of Hematology and Infectious Disorders.
Programme Manager, NHS Sickle Cell & Thalassaemia screening programme, Public Health England.
The NHS Sickle Cell and Thalassaemia Screening Programme screens pregnant women to find out if they’re at risk of having a baby with sickle cell disease or thalassaemia major.
Pregnant women and couples at risk of having a baby with one of these conditions can face some very difficult decisions. These include whether to have prenatal diagnosis and if the baby is affected to decide whether to continue or to end the pregnancy.
Early access to screening and the early offer of prenatal diagnosis is important in giving women and couples time to consider their options.
In 2016, national data revealed that many trusts were failing to meet the standards for early offer of screening and prenatal diagnosis.
In order to tackle this issue, a multi-disciplinary sub-group of the Sickle Cell and Thalassaemia Advisory Group set about looking at the whole system to determine why women are tested late.
This work included user interviews which were carried out by the Sickle Cell and UK Thalassaemia Societies and published in ‘Parent’s Stories’. Cathy will present the findings from this work and how they have been used to influence and improve the screening pathway.
Bessie has been a Paediatric nurse for 16 years working with children and their families. Her clinical experience lies within General Medicine, Paediatric Accident & Emergency and Trauma & Orthopaedics which included the rehabilitation of children following acute head injury and major trauma. She has 6 years’ experience in clinical research and currently works as a Senior Paediatric Research Nurse at Barts Health NHS Trust to manage a portfolio of National Institute of Health Research (NIHR) studies and care pathways for patients and families on clinical studies.
She is currently undertaking a Master of Research in Clinical Research (MRes(Clin)) at St.George’s University, London
Dr. Yvonne Daniel – PhD, CSci-FIBMS, MSc
Yvonne Daniel is Specialist Lead Scientist, Haematological Sciences and Special Haematology Lead at Viapath, Guy’s & St Thomas’ Hospital London. She has a specific interest in the screening and diagnosis of haemoglobinopathies and has been working in this area for over 20 years. Her experience encompasses both protein and molecular based techniques.
Yvonne completed her initial training in New Zealand before moving to London where she completed her MSc in Biomedical Sciences at University of Westminster in 1997. Her PhD at Kings College, London was completed in 2010 and developed a highly sensitive and specific method of screening for haemoglobinopathies using mass spectrometry-mass spectrometry.
In addition to her laboratory based role Yvonne is seconded as a scientific advisor to the NHS Sickle Cell & Thalassaemia Screening Programme. This role includes drafting policy, review of procedures and protocols and advising on incidents. She is co-author of the Laboratory Handbooks. Yvonne has also acted as a scientific advisor to projects implementing screening in Nigeria and Tanzania. Here she has led on technical implementation, policy formation for interpretative and reporting algorithms, quality process and training. She is actively involved in lecturing, training and education.
Dr. Deepika Darbari
Attending Physician, Division of Hematology, Center for Cancer and Blood Disorders, Children’s National Medical Center, Associate Professor of Pediatrics, George Washington University School of Medicine and Health Sciences, Washington, D.C.
Dr. Darbari is a Pediatric Hematologist-Oncologist at the Children’s National Medical Center and Associate Professor of Pediatrics at the George Washington University in Washington DC. Sickle cell team at Children’s National provides comprehensive care to one of the largest populations of children and adolescents with sickle cell disease in the United States. Dr. Darbari studies complications of sickle cell disease with emphasis on pain. She has been conducting clinical and translational studies directed to better understanding of sickle cell pain and its management. She has published many peer reviewed papers on the subject. Her work was one of the first in the field showing altered brain connectivity in patients who were frequently hospitalized for pain suggesting possible role of central mechanisms of pain in sickle cell disease.
Emma trained at St Barts and the Royal London School of Medicine, University of London and qualified in 2001. She has recently been appointed Locum Haematology Consultant at The Whittington Hospital. She was Clinical Lecturer in Sickle Cell Disease at King’s College Hospital from 2009 to 2013 and during that time was awarded her PhD based on research into markers of severity and predictors of organ dysfunction in sickle cell disease. In 2013 she was awarded the Early Stage Investigator prize from the British Society of Haematology.
Emma is currently a locum consultant haematologist based at the Whittington hospital where she is the clinical lead for Thalassaemia, Transfusion at the Whittington Hospital and also provides haemoglobinopathy cover for The Royal Free Hospital.
Dr. Andrew Dunham
Dr. Dunham serves as Vice President of Research and Clinical Affairs of Hemanext since January 2015. Prior to joining Hemanext, Dr. Dunham held researcher and senior positions at both specialty chemical and healthcare companies including 14 years at Baxter Healthcare. His experience at Baxter included leading the corporate R&D group comprising the global centers for Sterility Assurance, Particle & Imaging, Human Factors & Industrial Design, Pre-clinical, Toxicology, and Histology, Biochemistry and Cell Biology, Organic Synthesis and Analytical Chemistry, Advanced Engineering and Prototyping, and Stability Operations teams. Dr. Dunham has extensive experience applying this broad range of technologies across a diverse range of pharmaceutical and medical device products from development through product support. He is a graduate of the Northern Illinois University, the University of Colorado at Boulder, and had post-doctoral research experience at the University of Colorado Health Sciences Center School of Pharmacy.
Dr. Jaques Elion
Dr. Elion is Professor of Molecular Genetics, Paris Diderot Medical School – Sorbonne Paris Cité University and Visiting Professor, University of São Paulo. He received his training at the Universities Paris Descartes (MD) and Paris Diderot (PhD). He was a Research Assistant at the Mayo Clinic and a Fogarty Scientist at the NIH (Bethesda, USA). Dr Elion is the former Director of the Department of Medical Genetics at the Robert Debré University Hospital. His research is focused on the pathophysiology and innovative treatments of sickle cell disease. It is conducted at the French National Institute of Health and Medical Research (Inserm) in Paris (National Institute of Blood Transfusion) and in Guadeloupe (French West Indies). His group is part of the French Laboratory of Excellence on the Red Cell (GR-Ex). Dr Elion has organized several international meetings, including the scientific session at the inaugural ceremony for the 1st World SCD Day, June 19, 2009, UN Headquarters, NYC
Joy Enahoro is a specialist adviser with the Citizens Advice Bureau Southwark with an undergraduate degree in law from the University of East London and a mother to a 12 yr old boy living with Sicklecell Disease who has transitioned from many years on blood transfusion to hydroxyurea.
Dr. Gina Luca Forni
Starting from 1977 responsible of Hematology- Microcitemia and Congenital Anemia and Iron Dismetabolism Centre, Galliera Hospital, Genoa, Italy, regional reference and ERN EuroBlood Center. Mainly training in the field of not oncologic hematology to include in particular congenital blood disorders, the iron metabolism alterations, the overall approach to chronic diseases with high clinical complexity, rare diseases both adult and pediatric. Auditor of several international scientific journals, and the Ministry of Education. Principal investigator of national and international multicenter clinical trials fase1-2-3 particularly dedicated to the development of orphan drugs. Consultant for the “Italian Institute of Nuclear Physics (INFN)” for the development of “Magnetic Iron Detector” (MID), a new room temperature biosusceptometer for the non invasive measurement of iron liver overload. Data and Safety Monitoring Board of FAIR-PARK 2 project (Horizon 20-20). Consultant of Italian Minister for Reasearch (MIUR). Consultant of Italian Agency for Drugs (AIFA). Reviewer for French National Research Agency (ANR). Starting from October 2014 President of “Società Italiana Talassemie ed Emoglobinopatie” (SITE)
Dr. Anne Gordon
Dr. Anne Gordon is Senior Consultant Allied Health Professional & Strategic Lead for Therapies, Evelina London Children’s Hospital, Guy’s & St Thomas’ Hospital NHS Foundation Trust.
Anne holds a senior leadership role including clinical and research responsibilities and a pan-Therapies strategic position. She runs a unique clinical service in collaboration with the charitable sector for children who have had an acquired brain injury and their families. Anne has led a number of service-user engagement projects that have resulted in service improvements and clinical standards initiatives. This has included leading the national scoping project of children’s rehabilitation for NHS England in 2015, and chair of the rehabilitation and long-term outcomes section of the National Childhood Stroke Guidelines (Royal College of Paediatrics and Child Health, published 2017). Combining stakeholder data and that of best known practice she has prepared national recommendations to improve equity of access to care for children and young people.
Dr. Anne Greenough
Anne Greenough is Professor of Neonatology and Clinical Respiratory Physiology, Director of Education and Training at King’s Heath Partners Academic Health Science Centre and Board Member of the Higher Education Funding Council for England. She was Chair of the National Institute for Health Research (NIHR) Paediatrics (non medicines) Specialty Group and is now Vice President Science and Research, Royal College of Paediatrics and Child Health.
Pr. Beatrice Gulbis
Pr. Béatrice Gulbis is a physician specialized in Laboratory Medicine who has developed a reference laboratory centre for the diagnosis, prevention and follow-up of patients with hereditary red blood cells disorders and in particular, haemoglobinopathies and membrane red blood cells disorders. She has joined the Academic Hospital Erasme in Brussels, Belgium. She has contributed to the implementation of a national network of health professionals on non-oncological red blood cells disorders through the Belgian Hematology Society (www.bhs.be) and is working for more than 10 years for a European network on rare anaemias (www.enerca.org), and since March 2017 is the co-coordinator of the ERN EuroBloodNet (www.eurobloodnet.eu).
Dr. Kaplna Gupta
Kaplna Gupta is Professor of Medicine, Department of Medicine, and Co-leader, Molecular and Cellular Engineering Program at The Institute for Engineering in Medicine University of Minnesota medical School.
Dr Kalpna Gupta has led pioneering work in understanding the mechanisms of pain in sickle cell disease. These insights will help us treat both pain and the underlying disease process causing pain in the first place. Her laboratory has identified several new targets at the intersection of the sickle disease process and pain, including cannabinoid receptors, mast cells, and the nociceptin receptor, in addition to integrative approaches including diet modification, acupuncture and perception modulation to relieve pain. Dr Gupta is also a recipient of the Excellence in Hemoglobinopathies Research Award from NHLBI to examine the potential of cannabinoids to treat pain and develop methods to quantify pain objectively.
Dr. Mona Hamdy
Mona Hamdy, MD is a Professor of Pediatrics, Cairo University and Consultant of Hematology at Ministry of Health, Egypt. She is a winner of the State Award in Medical Sciences and holder of a Master degree in Clinical Research Administration, Walden University, USA. She is one of the founders of the clinical research center at Faculty of Medicine of Cairo University with 15 years of experience as a co-investigator and a principle investigator in multicenter clinical trials. She has multiple publications in peer review journals and is an editor and reviewer in several peer review journals and a member of the Egyptian Research Network.
Dr. Kathryn Hassell
Dr. Kathryn Hassell is a Professor of Medicine in the Division of Hematology at the University of Colorado Denver, in Aurora, CO. As an active, board certified academic hematologist, she has provided care for adults living with sickle cell disease (SCD) since 1991 at the Colorado Sickle Cell Treatment and Research Center at the University of Colorado Denver (UCD), which she directs. She also is an expert in thrombotic disorders and anticoagulation, with a large academic consultative practice, and serves as medical director of large inpatient and outpatient anticoagulation services at the University of Colorado Hospital. She has conducted observational studies, translational research, phase II and III interventional trials, and health services research in hemoglobinopathies and thrombosis.
Dr. Jo Howard
Consultant Haematologist and Clinical Lead/ /Honorary Reader in Haemoglobinopathies, Guy’s and St Thomas’ NHS Foundation Trust
Dr Howard is the chair of the UK Forum for Haemoglobin Disorders is Co-Chair of the adult peer review of haemoglobinopathy services and is on the Steering Committee for the UK Haemoglobinopathy Registry. She leads the Editorial board for the UK Standards for the Care of Adults with Sickle Cell Disease and she is an advisor on the UK NEQAS Steering Committee for General Haematology, and is a reviewer for the Cochrane Collaboration. She has published articles on the management of sickle cell disease. Her current interests are in the improvement of care for adults with Sickle Cell Disease, both acute pain management and in the management of its long term complications.
Dr. Lewis Hsu
MD, PhD, is Professor of Pediatrics and Director of Pediatric Sickle Cell at the University of Illinois at Chicago.
Dr. Hsu’s dedication to finding more cures for sickle cell disease, and improving treatment and education until more cures can be found, began during his MD/PhD (Biophysics) at University of Rochester. He did pediatric residency at Yale and pediatric hematology-oncology fellowship training at Children’s Hospital of Philadelphia. His clinical and translational research in sickle cell disease includes the landmark STOP study of transfusion to reduce stroke risk in sickle cell, bone marrow transplantation to cure sickle cell disease, clinical research on management of sickle cell pain, and inflammatory vasculopathy due to hemolysis in mouse models. He volunteers as Vice Chief Medical Officer of the Sickle Cell Disease Association of America. His current projects examine care transitions, social determinants of health, and patient-centered outcomes. Dr. Lewis Hsu’s recent White Papers are among the first to highlight the utility for sickle cell disease of Community Health Workers and of Implementation Science. His global experience in sickle cell disease includes collaboration in Nigeria and Brazil.
Dr. Vishal Jayakar
Dr V Jayakar runs the popular FRCPath Revision Courses at Kingston for exam –going haematology trainees which are widely subscribed from UK, Ireland, UAE, India, Singapore and Hong-Kong.
He is the Co-Organiser of the Imperial Morphology Courses along with Prof Barbara Bain.
Apart from education, his main interests are morphology and Myeloproliferative neoplasms.
Dr. Julie Kanter
Dr. Julie Kanter is an associate professor at the Medical University of South Carolina where she is the director of the Sickle Cell Disease Research Program and the Lifespan Comprehensive Sickle Cell Center. Dr. Kanter is a co-PI on two large implementation trails in sickle cell disease as well as a site PI for several sponsored research studies. Dr. Kanter is interested in identifying novel methods to enhance access to care for individuals living with sickle cell disease and for working to develop novel therapeutics to improve care outcomes. Dr. Kanter is part of the NHLBI sickle cell disease advisory panel and the American Society of Hematology government relations committee.
Dr. Rachel Kesse-Adu
Dr Rachel Kesse-Adu graduated from Imperial College School of medicine in London in 2002, completed haematology training at Kings College Hospital in 2012 and was appointed as a consultant haematologist at Guy’s and St Thomas’ NHS Foundation Trust the same year.
Along with another colleagues she manages a large haemoglobinopathy service with more than 750 registered patients, she has runs specialist clinics in cardiorespiratoy and urological complications of sickle cell disease (SCD). She also manages a general haematology clinics.
Dr. Fenella Kirkham
Fenella Kirkham is a paediatric neurologist at University hospital Southampton with an interest in acute paediatric neurological problems and sickle cell anaemia. She has developed an interest in the role of sleep in cognition in sickle cell disease and is currently analysing data from the East London and Sleep Asthma cohorts with her Post-docs, PhD and MSc students in Developmental Neurosciences at UCL Institute of Child Health. She collaborates with haematologists in England, Europe, the USA and Africa. She is running a Phase II randomised controlled trial of auto-adjusting continuous positive airways pressure at Guy’s and St Thomas’ and King’s College hospitals.
Dr. Marina Kleanthous
Professor, The Cyprus School of Molecular Medicine. Head, Molecular Genetics Thalassaemia Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus
Dr Marina Kleanthous (PhD 1990 Neurochemistry, University of Athens) is the Head of the Molecular Genetics Thalassaemia Department at the Cyprus Institute of Neurology and Genetics (CING and Professor at the Cyprus School of Molecular Medicine. She is actively involved in the diagnosis, prenatal diagnosis and Preimplantation Genetic Diagnosis (PGD) of haemoglobinopathies Her research interests include: Drug therapy for thalassaemia using HbF inducers, gene therapy for thalassaemia using gene addition and gene editing approaches, development of non-invasive prenatal diagnosis (NIPD) for thalassaemia by NGS and genomic and proteomic studies in thalassaemia. She has created the ITHANET, an international haemoglobinopathies portal.
Dr. Elizabeth Klings
Dr. Elizabeth Klings is an Associate Professor of Medicine at Boston University School of Medicine, the Director of the Center for Excellence in Sickle Cell Disease and an Attending Physician in the Section of Pulmonary, Critical Care and Allergy at Boston Medical Center. She came to Boston University after receiving Bachelors of Arts and Doctor of Medicine degrees at New York University in New York, New York. She completed her residency in Internal Medicine at Boston City Hospital and fellowship training in Pulmonary and Critical Care at Boston University School of Medicine.
Yaa is a mum.of two, an 11 and 6 year old, a daughter and son. Her daughter has sickle cell trait and her son had sickle cell disease. He was cured of Sickle Cell in 2013 when he undertook a haploidentical bone marrow transplant. He was 3 at the time.
She would describe herself as a ‘go getter’ and will not stop until she exhausts all avenues.
Chris Lambert is the Service Delivery Manager for Reference Haematology and operational lead of the Red Cell Protein Laboratory in the Viapath Laboratories at Kings College Hospital.
He has been at Kings College Hospital for over 20 years, working in the pre-natal diagnosis section for 8 years before moving to the protein section and subsequently taking on more senior managerial responsibility.
He works with the National Programme Centre for Sickle Cell and Thalassaemia delivering the training days and continues to work with the new providers delivering the training days and working on the on-line training module. The laboratory at KCH offers a comprehensive screening and diagnostic service for sickle cell and thalassaemia serving internal users and a reference service for external users working closely with the molecular laboratory.
Dr. Carsten Lederer
Dr Lederer received his PhD from the University of East Anglia for work in plant virology at the John Innes Centre, Norwich, UK. He is now a researcher at the Department of Molecular Genetics Thalassemia (MGTD; head: Marina Kleanthous) at the Cyprus Institute of Neurology and Genetics (CING), where he heads the MGTD Gene Therapy and Genome Editing unit. Current research focus is the gene therapy of β-globinopathies and particularly of β-thalassaemia by a) genome editing of disease modifiers, b) mutation-specific RNAi-supplementation of gene addition and c) homology-independent gene repair.
Dr Lederer is also assistant professor and course coordinator at the Cyprus School for Molecular Medicine, is executive board member of the Global Globin 2020 Challenge, curator of the ITHANET Portal, board member of the Cyprus Society of Human Genetics and a member of the European Society of Human Genetics and the Hellenic Society of Gene Therapy and Regenerative Medicine.
Dr. Stephan Lobitz
Stephan Lobitz is a paediatric haematologist from Germany. He has graduated from Charité – Universitaetsmedizin Berlin and earned a master’s degree in haemoglobinopathies from University College London. He has a long term experience with the diagnostics and treatment of sickle cell disease, thalassemia and other non-malignant blood disorders. Stephan is the spokesperson of Germany’s pediatric sickle cell disease program and the corresponding author of the national treatment guideline for children and adolescents with sickle cell disease. He is a member of the European Reference Network (ERN) EuroBloodNet.
Dr. Deepa Manwani
Dr. Deepa Manwani is the Director of the Hematology Program, Children’s Hospital at Montefiore (CHAM) and Professor of Pediatrics at Albert Einstein College of Medicine, Bronx, NY.
Dr Manwani completed her residency (1993-96) at NSUH – Cornell University Medical College, NY. She received her sub specialty training in pediatric hematology – oncology (1996 – 99) at Mount Sinai School of Medicine. She conducted basic science research in the field of globin gene regulation, under the mentorship of Dr James Bieker, PhD at Mount Sinai School of Medicine from 1997-2006. Her primary areas of research focused on exploring mechanisms of fetal hemoglobin reactivation with the ultimate goal of designing novel therapies for sickle cell disease and thalassemia. She has also studied erythropoiesis and red cell-macrophage adhesive interactions in the erythroblast island.
Mbololwa Mbikusita-Lewanika, BSc, MSc, PhD, AKC
Dr Mbololwa Mbikusita-Lewanika, Health Adviser, leads the Commonwealth Secretariat’s programme on health, with special focus on universal health coverage and non-communicable diseases. She previously spearheaded programmes on HIV/AIDS, tuberculosis and malaria, and contributed to programmes on maternal and child health, youth empowerment, human rights and civil society engagement.
Dr Mbikusita-Lewanika has a health science background and expertise in social development, with more than 30 years national and international experience in these fields. She has been working in the health field as a lecturer in pharmacology at the University of Zambia School of Medicine and at King’s College, London, training medical, dental, biomedical, veterinary, physiotherapy, nutrition and pharmacy students and technicians. As a social development professional she has a special interest in women’s health, the linkages between health, education and social development as well as social justice.
Samuel Misiani Mbunya, AMPATH Hematology Administrator Sam Mbunya has been AMPATH’s Hematology Administrator since December 2015, leading one of many Hematology programs ‐ mainly the Indiana Hemophilia & Thrombosis Center (IHTC) Program that deals with Sickle Cell Disease and Hemophilia. He also oversees administrative aspects in the Multiple Myeloma Program with Celgene. Sam’s current areas of management are with the AMPATH Hematology Reference and Research Laboratory, IT Point of Care digital platforms, Telemedicine/Telepathology, Outreach, and Revolving Fund Pharmacy programs. Before joining AMPATH, Sam worked at Pathologists Lancet Kenya LTD, a South African based diagnostic and pathology corporate company as their Consultant Laboratory Scientist, providing both administrative and technical assistance for diagnostic and pathology services. Sam received his Bachelor of Science degree in Biomedical Science from Kingston University London and his Post Graduate Diploma BioMedSci Research at University of Glasgow (UK). He is currently in an MBA program with the University of South Whales (UK).
Dr. Stephan Menzel
I graduated 1993 in Greifswald, Germany, with a thesis describing families with Mendelian forms of diabetes. With a fellowship from the German Academic Exchange Service, I took my sample collection to Graeme Bell’s lab at the University of Chicago, participating in the successful undertaking to ‘clone’ the genes responsible for diabetes in these and other families.
Moving back to Europe in 1997, I took up a position at the Wellcome Trust Centre for Human Genetics at the University of Oxford, where I studied the genetics of late-onset (type 2) diabetes using the fraught approach of genome-wide linkage scanning in small families.
I joined Swee Lay Thein’s lab at King’s College London in 2003, moving from diabetology into haematology. The lab had a long-standing interest in the persistence of fetal haemoglobin, a condition that can ameliorate sickle cell disease and beta thalassaemia.
Dr. Caterina Minniti
Dr. Minniti is Professor of Clinical Medicine and Pediatrics at Einstein College of Medicine. She is the Director of the Sickle Cell Center for Adults at Montefiore Hospital, whose mission is to provide exceptional, seamless, comprehensive, compassionate and individualized care, education, counseling and research for people living with sickle cell disease. Dr. Minniti is a clinical trial specialist and a translational researcher who believes that the best way to provide care for SCD patients is on a continuum, from birth to adulthood.
The focus of her research is in understanding mechanisms that lead to end organ damage in order to identify early biomarkers and targeted therapies. Her interests have spanned from stroke to pulmonary hypertension and most recently, she has focused on leg ulcers as they represent a window into the vasculopathy in SCD. She aims to develop pathogenetically based therapeutic approaches for preventing and treating SCD-related end organ damage. Before moving to New York, she was a member of the Hematology Branch of the National Institute of Heart Blood and Lung, where she developed a topical treatment for chronic leg ulcers in SCD.
Prof Mariane de Montalembert
Professor de Montalembert received her MD from the Paris Descartes Medical School in 1982 and her PhD in Ethics in 1994. She specialized in pediatrics, statistics (option clinical research), and transfusion. She is Associate Professor of Pediatrics and serves as the head of the Hemoglobin Diseases Unit at the Necker University Hospital, and the « ROFSED » healthcare network for SCD children in the Parisian area . Prof de Montalembert is a member of the French Society of Pediatrics, in which she chairs the Ethic Committee, of the French Society of Hematology, the European Network for rare and Congenital Anaemias (ENERCA), the European Reference Network in Rare Haematological Diseases(EuroBloodNet), the European Association (EHA), and the American Society of Hematology (ASH). She coordinates and participates to several clinical trials, especially on hydroxycarbamide use in SCD.
Luhanga Musumadi – MPH, BSc, RGN
Advanced Nurse Practitioner for Haemoglobinopathy and the Lead Nurse for Adolescent transition at Guy’s and St Thomas’ NHS Foundation Trust (GSTT).
Luhanga set up the transition service at GSTT in 2008 to support young people aged 13 – 24years, preparing them for transition while in Paediatrics and helping them settle into the adult services. The transition service provides a comprehensive multidisciplinary service addressing a range of issues concerning this age group from clinical to Social, Psychological, educational and general wellbeing.
His current research interests include the role of digital communication in current and future patient–clinician communication for NHS providers of specialist clinical services for young people.
He also teaches on the Adolescent care & Haemoglobinopathy Courses for post graduate Nurses at King’s College University, London.
Sharon Ndoro (RCN, BSc) is Senior Research and specialist Nurse for sickle cell and Thalassaemia at the Evelina London Children’s Hospital since 2012, responsible for the recruitment and follow up of patients in clinical and observational studies and also providing education and clinical support for children with haemoglobinopathy. I began my career in paediatric haematology in 2002 as a Sister at Royal London Hospital on a general haematology ward. In 2009-2012 worked as specialist haemoglobinopathy nurse at East London NHS Foundation Trust providing genetic counseling, providing support for newly diagnosed patients and affected patients both in patient and in the community.
Maria Pelidis received her MD at Harvard Medical School and completed her training in paediatrics at the Children’s Hospital of Philadelphia in 1993. She subsequently trained in children’s haematology/oncology at Johns Hopkins Hospital.
She is board certified in children’s medicine and children’s haematology/oncology by the American Board of Paediatrics.
She practised at Tufts New England Medical Center until 2009 when she moved to the United Kingdom and practised at St George’s Hospital and The Royal Marsden Hospital. She moved her practice to Evelina London Children’s Hospital in 2015.
Antonio Piga is Professor of Pediatrics at Turin University, where he serves as Dean of the School of Medicine, pole B.
Research fields include hemoglobinopathies, disorders of iron metabolism, non invasive methods for body iron assessment, and development of drugs as iron chelators, and modulators of bone marrow activity.
John B. Porter, MD, FRCP, FRCPath.
John Porter is Professor of Haematology and Consultant Haematologist at the University College London Hospitals in London, UK and head of the joint Red Cell Unit for UCLH and Whittington Hospitals. He graduated from the University of Cambridge in 1974 was awarded an FRCP by the Royal London College of Physicians in 1995 and FRCPath in Haematology by the Royal College of Pathologists in 1996.
His clinical and research focus has been treatments of thalassaemia and sickle cell disorders, with particular reference to iron overload, the mechanisms of iron chelation, the speciation and uptake of non-transferrin-bound iron (NTBI) species, the molecular basis of iron homeostasis in health and disease, and the actions and toxicities of mixed-ligand chelation therapies.
Dr Victoria Rathbone
Victoria is an academic clinical fellow in Histopathology, she started her registrar training at Guys and St Thomas’ NHS foundation trust and now works at University College Hospital London. Victoria will be presenting on behalf of Professor Sebastian Lucas. Sebastian Lucas was the professor of pathology at Guy’s & St Thomas’ Hospital from 1995-2012, and is now emeritus.
He has been interested in the pathology of sickle cell disease since encountering it in Kenya in the early 1980s. He has a wide experience of the pathology of sickle cell patients who have come to autopsy in London, along with much referred case work from other pathologists. Over the last thirty years, the clinical pathology of severe and fatal sickle cell disease has changed along with improved management protocols, and his presentation reflects these different patterns.
Dr. Heather Rawle
Dr Heather Rawle is a Consultant Clinical and Health Psychologist at Guys and St Thomas’ Hospital Foundation NHS Trust (GSTT). Heather has worked in the GSTT Haematology Health Psychology Service since 2002 and has led the service since 2016 when Dr Nicky Thomas (Consultant Health Psychologist) retired.
Heather provides psychological support using a range of psychological approaches such as cognitive behavioural therapy, acceptance and commitment therapy, solution-focussed therapy, and motivational interviewing. Heather is also responsible for the neuropsychological aspects of the service which supports patients who are concerned about their memory and other aspects of cognitive functioning. She is on the Peer Review Haemoglobin Disorders Steering Group, chairs the British Psychological Society Special Interest Group in Haemoglobinopathies, and is a member of the Haematology Institute Red Cell Working Group. She is co-lead psychologist for South Thames Sickle Cell and Thalassaemia Network and for NHS London Haemoglobinopathies meetings.
Heather obtained her Doctorate in Clinical Psychology from the University of Southampton, her Masters in Health Psychology from the University of Surrey, and her Postgraduate Diploma in Applied Clinical Neuropsychology from Institute of Psychiatry/King’s College London.
Prof. Giovanna Russo
Professor of Pediatrics, University of Catania; director of the Pediatric Hematology/Oncology Center, which provides health care for children with hematology/oncology/immunology diseseas. Her areas of clinical and research interests are hemoglobinopathies, sickle cell disease, rare anemias, platelet disorders.
She coordinates the Red Cell Working group of Italian Pediatric Hematology/Oncology Association, which has an ongoing programme to promote comprehensive care for sickle cell disease pediatric patients all over Italy.
Dr. Renu Saxena
Dr. R Saxena is currently Professor & Head, Department of Hematology, AIIMS. She did her MBBS, MD, and subsequently became Faculty at AIIMS, a premier tertiary care Medical School in India. Her major interests have been genotypic phenotypic correlations of Hemoglobinpathies and Hemostasis. She has more than 350 publications in National and International Journals. She has received 17 prestigious national and international awards for her contributions in hematology and also BC Roy award for eminent teacher by Medical Council of India. She is member of numerous national and international editorial boards of journals and a member of subject expert committees of national scientific bodies like Indian Council for Medical Research (ICMR), Department of Science and Technology (DST), Council of Scientific and Industrial Research (CSIR), Department of Biotechnology (DBT). Besides this she was Member Secretary Ethics Committee AIIMS for 05 years. She has been Principal Investigator for 26 extramural funded research projects.
Dr. Claire Sharpe
Dr Sharpe has been involved in the combined renal and sickle cell clinic at King’s College Hospital since its instigation in 2004 and has developed a strong interest in the underlying mechanisms and management of sickle cell nephropathy. She is actively involved in studying both its epidemiology and the outcomes of early treatment, focusing on patient education, early intervention and on-going monitoring in order to slow disease progression and minimize the number of patients requiring renal replacement therapy in the future. She has been invited to speak on the renal complications of sickle cell disease at many national meetings including at the Royal Society of Medicine Transplantation Immunology & Mucosal Biology MPhil/PhD.
On world sickle cell day, 19th June 2012, in collaboration with the Maldivian medical association,in an effort to break the sickle silence in the country, the very first newspaper supplement on sickle cell was published featuring my story and I presented patient perspective living with sickle cell at a session held for doctors and health care professsionals. Since then I volunteer with Maldivian thalassaemia society and has been an elected executive committee member since 2014.
As a sickle – beta thalassaemia Patient, I dedicate my efforts to create sickle cell awareness throughout my country and at different international platforms for patients, parents, medical professionals and policy makers. My mission is to share my story and stand up for my cause to break the stigma and empower patients & parents to win the battle against sickle cell and thalassemia for the sufferers.
Dr. Paul Telfer
Dr Paul Telfer is clinical lead for the haemoglobinopathy services at the Royal London Hospital since 1998, and clinical lead for the East London and Essex Clinical Haemoglobinopathy Network since 2004. He has been a committee member of the British Society for Haematology, UK Forum for Haemoglobin Disorders, the Clinical Advisory Group for Specialist Commissioning in Haemoglobinopathies, and is clinical advisor to the National Sickle Cell and Thalassaemia Screening Programme, the UK Thalassaemia Society, and Thalassaemia International Federation. He has co-authored national guidelines for management of children and adults with sickle cell disease, and thalassaemia. He has authored clinical research papers and is co-author of ‘Sickle Cell Disease in Clinical Practice’ published by Springer.
He is chief investigator for the SCAPE trial, the East London Newborn Sickle Cell Cohort Study and Cyprus Thalassaemia Cohort study. He has been principal investigator for ASSERT, SITT, DEEP, GBT440 and BELIEVE trials.
Dr. Alexis Thompson
Dr. Alexis Thompson is currently the Hematology Section Head at Ann and Robert H. Lurie Children’s Hospital of Chicago in Chicago, Illinois. She holds the A. Watson and Sarah Armour Endowed Chair for Blood Diseases and Cancer at Lurie Children’s. She is also a Professor of Pediatrics at the Northwestern University Feinberg School of Medicine. Her clinical interests include hemoglobinopathies (thalassemia and sickle cell disease), bone marrow failure syndromes and stem cell transplantation in pediatric patients. In her current position, she is an investigator on multi-center trials and as well as her own institutional clinical studies in thalassemia, sickle cell disease and ITP. Dr. Thompson is also the Associate Director for Equity and Minority Health at the Robert H. Lurie Cancer Center and Northwestern University Feinberg School of Medicine. Currently she is Vice President of the American Society of Hematology.
Dr. Marsha Treadwell
Dr. Treadwell is a clinical psychologist and a clinical and health services researcher at UCSF Benioff Children’s Hospital Oakland in Oakland, CA, USA. Research interests include: cross cultural models of health beliefs and practices; patient reported outcomes (assessment and measurement development); transition from pediatric to adult care; adherence with medical regimens; and factors influencing pain experiences. Dr. Treadwell is co-principal investigator for the Pacific Sickle Cell Regional Collaborative that aims to improve sickle cell disease clinical services and policy, and enhance workforce development. Dr. Treadwell is co-investigator on Exploring Perspectives on Genomics and Sickle Cell Public Health Interventions, an H3Africa consortium project. Dr. Treadwell obtained her PhD in clinical psychology from the University of Washington in Seattle, WA USA and her training in clinical research from the Department of Epidemiology and Biostatistics at UCSF.
Dr. Mark Velangi
Dr Mark Velangi graduated from the University of Edinburgh Medical School in 1991. He is a Consultant Paediatric Haematologist and, in 2006, joined the team at Birmingham Children’s Hospital which has a large sickle cell and thalassaemia unit. He has been the Lead for the Haemoglobinopathy service since 2014 and is also helping to develop the West Midlands Haemoglobinopathy network. He has developed a particular interest in acute complications of sickle cell disease.
Dr. Winfred Wang
Member, Department of Haematology, St. Jude Children’s Research Hospital
Dr Wang trained at the University of California, Berkeley (BA, Chemistry); University of Chicago (MD); Montefiore Hospital and Medical Center, Bronx, NY and Kauikeolani Children’s Hospital, Honolulu, HI (paediatric residency); University of California Medical Center, San Francisco (paediatric haematology fellowship)
Dr Wang has provided clinical care for children with non-malignant haematologic conditions for more than 40 years and has been an attending physician at St. Jude Children’s Research Hospital and LeBonheur Children’s Medical Center for the past 36 years. His major interests are clinical care of children with sickle cell disease and bone marrow failure disorders and clinical research in those areas. Most of his clinical research efforts are related to the use of hydroxyurea and the evaluation of the central nervous system in different populations of children with sickle cell disease. He was formerly the leader of the St. Jude Comprehensive Sickle Cell Center and the BABY HUG multi-center trial.
Other current appointments are Professor, Dept. of Paediatrics University of Tennessee Center for the Health Sciences and Attending Physician, LeBonheur Children’s Medical Center.
Dr. Russell Ware
Dr. Russell Ware obtained his MD and PhD degrees, and completed his Pediatric Hematology/Oncology fellowship, at Duke University Medical Center. In 2013, Dr. Ware joined Cincinnati Children’s Hospital Medical Center as Division Director of Hematology and Marjory Johnson Chair of Translational Hematology Research. He serves as Director of the Global Health Center, and leads the residency program pathways for research and global health.
Dr. Ware has been a national leader for NIH-funded clinical trials using hydroxyurea for children with sickle cell disease, including BABY HUG, SWiTCH, SCATE, and TWiTCH. His laboratory focuses primarily on genetic modifiers of sickle cell disease and variable responses to hydroxyurea therapy. He completed a sickle surveillance study in Uganda, and leads similar efforts now in Tanzania and Malawi. Dr. Ware has NIH and Doris Duke funding to introduce hydroxyurea safely and effectively into low-resource settings, working with partners in the Caribbean and sub-Saharan Africa.
Dr. Kimberly Wesley, Psy.D.
Kimberly Wesley is a pediatric psychologist at the Children’s Hospital of Philadelphia in Philadelphia, Pennsylvania. She earned her doctorate in clinical psychology from La Salle University prior to completing a postdoctoral fellowship at St. Jude Children’s Research Hospital in Memphis, Tennessee. While on fellowship, she worked under the mentorship of Dr. Jerlym Porter. Her primary research interests include evaluation of factors affecting quality of life in individuals diagnosed with chronic medical conditions and healthcare transition readiness assessment and intervention. Dr. Wesley is a member of the American Psychological Association and the Society of Pediatric Psychology.
Prof. Ambroise Wonkam
Prof Ambroise Wonkam is a specialist medical geneticist, in the Division of Human Genetics, Faculty of Health Sciences, University of Cape Town, South Africa.
After a MD training from the Faculty of Medicine and Biomedical Sciences, University of Yaoundé I (Cameroon), he completed a thesis in Cell Biology in the department of Morphology, University of Geneva (Switzerland) and a PhD in Human Genetics (University of Cape Town, South Africa). Other salient aspects of Prof Wonkam’s background include his education as a medical geneticist at a highly reputable genetics department in Geneva (Switzerland). He subsequently practices medical genetics in both European and African contexts.
His research interests are reflected in more than 100 peer-reviewed publications, which are in molecular, clinical, educational and ethical aspects of medical and human genetics.
His is member of the steering committee of H3Africa consortium, leading specifically the NIH/NHGRI funded SCD project. He has recently been granted from NIH/NHLBI 3.7m USD, to establish a Sickle Africa Data Coordinating Centre (SADaCC), at the University of Cape Town.
More conference speakers to be announced.
The 11th Annual Conference Academy for Sickle Cell and Thalassaemia Conference (ASCAT) is the essential event for all health care professionals who wish to learn more about the diagnosis and management of sickle cell disease and thalassaemia.
The conference provides unique exposure and access to network with international healthcare stakeholders including:
- A showcase to a highly relevant audience
- Access to industry influencers and decision makers
- Premium association with a leading edge authoritative event
- Access to key stakeholders
- Networking opportunities
For sponsorship enquiries, please contact email@example.com or call 020 7880 6244
This year’s sponsors and exhibitors include:
Platinum sponsor – Pfizer
At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value the discovery, development and manufacture of health care products. Our global portfolio includes medicines and vaccines as well as many of the world’s best-known consumer products.
Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world’s premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, Pfizer has worked to make a difference for all who rely on us. To learn more, please visit us at www.pfizer.com.
Silver sponsor – bluebird bio
With its lentiviral-based gene therapies, T cell immunotherapy expertise and gene editing capabilities, bluebird bio has built an integrated product platform with broad potential application to severe genetic diseases and cancer. bluebird bio’s gene therapy clinical programs include its Lenti-D™ product candidate for the treatment of cerebral adrenoleukodystrophy and its LentiGlobin™ BB305 product candidate for the treatment of transfusion-dependent ß-thalassemia and severe sickle cell disease. bluebird bio’s oncology pipeline is built upon the company’s leadership in lentiviral gene delivery and T cell engineering.
bluebird bio’s lead oncology program, bb2121, is an anti-BCMA CAR T program partnered with Celgene. bluebird bio also has discovery research programs utilizing megaTALs/homing endonuclease gene editing technologies with the potential for use across the company’s pipeline.
Silver sponsor – Novartis
In the UK, Novartis develops, manufactures and markets innovative medicines, devices and diagnostic tests which help improve patient outcomes. Based on four sites across the north and south of England, we employ approximately 1,500 people to serve healthcare needs across the whole of the UK, as well as supporting the global operations of Novartis by manufacturing the active pharmaceutical ingredients used worldwide in many medicines. In 2015 Novartis in the UK invested over £50million in R&D and is the largest commercial sponsor of clinical trials. For more information, please visit www.novartis.co.uk.
Silver sponsor – Addmedica
ADDMEDICA, founded in 2005, is a European pharmaceutical company focused on developing and marketing innovative products for rare diseases, serious conditions and unmet medical needs.
The mission of ADDMEDICA is to provide specific medical solutions to physicians, patients, and managed care organizations, by developing, registering and/or marketing a range of medical products in several rare and debilitating diseases.
ADDMEDICA has developed a strong expertise in hematology and pediatric fields.
In Europe, SCD affects more than 41,000 patients, more than 15,000 in UK; and before SIKLOS, no specific medicine has ever been registered for the treatment of SCD.
On June 29th, 2007, ADDMEDICA obtained a European Marketing Authorization for SIKLOS® 100mg & 1000mg as an orphan medicinal product in the prevention of recurrent, painful vaso-occlusive crisis, including chest syndrome in paediatric and adult patients suffering from SCD.
With the development of SIKLOS® 100mg and 1000mg (hydroxycarbamide), the company has made a significant contribution to the field of Sickle Cell Disease (SCD).
ADDMEDICA is actively engaged against SCD, and to facilitate disease management, the company also developed free app for patients. (http://www.sickle-o-scope.net/)
This engagement is also translated in high involvement in specific clinical research.
AstraZeneca is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialization of prescription medicines, primarily for the treatment of diseases in three therapy areas – Respiratory and Autoimmunity, Cardiovascular and Metabolic Diseases, and Oncology. The company is also active in inflammation, infection and neuroscience through numerous collaborations. AstraZeneca operates in over 100 countries. For more information: www.astrazeneca.com.
AstraZeneca is currently undertaking a development program with the anti-platelet drug Ticagrelor in Sickle Cell Disease. There are clinical and nonclinical evidence that platelets participate in the vaso-occlusive process and there is a rationale for the evaluation of antiplatelet therapies in management of Sickle Cell Disease. Currently, two studies have been completed, one in children 2-18 years and one in young adults (18-30 yrs), and preparations for next phase of development are ongoing. For more information – please contact firstname.lastname@example.org (study project leader) or email@example.com (senior physician).
Terumo BCT, a global leader in blood component, therapeutic apheresis and cellular technologies, is the only company with the unique combination of apheresis collections, manual and automated whole blood processing, and pathogen reduction. We believe in the potential of blood to do even more for patients than it does today. This belief inspires our innovation and strengthens our collaboration with customers.
Our therapeutic apheresis device, Spectra Optia® Apheresis System, can be used to perform red blood cell exchange (RBCX) procedures.
Global Blood Therapeutics
GBT is a clinical-stage biopharmaceutical company dedicated to discovering, developing and commercializing novel therapeutics to treat grievous blood-based disorders with significant unmet need. The team at GBT strives to understand the needs of the patients they hope to serve, and incorporate patients’ perspectives and needs into their development programs.
GBT is developing its lead product candidate, GBT440, as an oral, once-daily therapy for SCD. GBT is also investigating GBT440 for the treatment of hypoxemic pulmonary disorders in two ongoing Phase 2a studies in patients with idiopathic pulmonary fibrosis.
Sobi is an international specialty healthcare company dedicated to rare diseases. Our mission is to develop and deliver innovative therapies and services to improve the lives of patients. The product portfolio is primary focused on Haemophilia, Inflammation and Genetic diseases. We also market a portfolio of specialty and rare disease products across Europe, the Middle East, North Africa and Russia for partner companies. Sobi is a pioneer in biotechnology with world-class capabilities in protein biochemistry and biologics manufacturing.
The focus at ApoPharma is on the development of drugs for the treatment of debilitating and life-threatening diseases caused or worsened by iron. The main product is the iron chelator Ferriprox® (deferiprone), which is approved for the treatment of systemic iron overload in transfusion-dependent patients with thalassemia syndromes. In patients with thalassemia major, Ferriprox has been shown to be successful in reducing the incidence of iron-associated cardiac disease and in increasing survival, as described in the EMA Summary of Product Characteristics for Ferriprox. Current studies are exploring the use of deferiprone in other populations: iron-overloaded patients with sickle cell disease, and patients with conditions of local brain iron overload, including neurodegeneration with brain iron accumulation and Parkinson’s disease. For information about those studies, please contact ApoPharma at firstname.lastname@example.org.
The South Thames Sickle Cell and Thalassaemia Network is a haemoglobinopathy collaboration led by health professionals, including consultants, nurses, psychologists and others, at King’s College Hospital, Evelina Children’s Hospital and Guy’s and St Thomas’ Hospital NHS Foundation Trusts. The Network region includes hospitals across South London and South East England.
Our mission is to improve the patient experience by offering better treatment outcomes for people with sickle cell disease and thalassaemia. We seek to achieve this by providing a peer-support network and educational programme, producing patient information and guidelines for healthcare professionals, collaborating in specialist clinics, holding patient forums and workshops, producing a patient newsletter (Red Cell News), and contributing to research.
Hemanext is a medical technology company based in Bethesda MD, with operations in Cambridge and Avon MA. Our corporate mission is to support the transfusion medicine community in its efforts to save lives and improve patient outcomes. Our distinctive focus is on innovations that promote radical improvements in the quality, safety, efficacy and cost of transfusion therapy
We are developing the Hemanext RBC Processing System, an innovative blood processing and storage system for red blood cells that is designed to preserve their efficacy and safety characteristics during storage. Hemanext RBC removes oxygen, the fuel for oxidative damage, and aims to provide a more consistent and higher quality red blood cell product for transfusion patients.
Safer transfusion, better patient care. That’s our mission.
For more information, please visit: www.hemanext.com
Sickle Cell Society
The Sickle Cell Society is the only national charity in the UK that supports and represents people affected by sickle cell disease to improve their overall quality of life.
The Sickle Cell Society provides a helpline and information service, advice, advocacy, raises awareness of sickle cell disease (SCD) and the importance of screening/blood donations, mentoring, children’s activities, annual children’s holiday, assist into research, hold talks, patient education seminars, training and lobbying to improve treatments and services for people affected by SCD.
Sickle cell disease is the most common genetic blood disorder in the UK, 380,000 people are carrying the gene and roughly 15,000 actually have the condition. Sickle cell disease is a long term inherited condition that causes excruciating pain, can be life threatening, causes multiple problems such as specific severe infections, strokes, chronic fatigue, delayed growth and progressive tissue and organ damage.
United Kingdom Thalassaemia Society (UKTS)
At myTomorrows, our goal is to facilitate early access to medicines for patients with unmet medical needs. To achieve this, we have developed a Knowledge Base that combines the global WHO Clinical Trial registries, approved drugs and labels, all active Early Access Programs, and research from PubMed. Using this Knowledge Base, our medical team can assist you in finding the appropriate unlicensed medicine. In addition, we support you in organising regulatory approval and financial matters for the Early Access treatment.
Over the Wall
Over The Wall provides FREE residential activity camps for children and teenagers living with serious health challenges and their families, to enable them to reach beyond their perceived limitations and rediscover a whole new world of possibilities. Our camps help develop the confidence, self esteem, coping strategies and peer relationships of our campers.
We focus heavily on delivering extremely high quality, safe services that are great fun and at the same time transformational. The most common feedback we receive from those attending camp is that it has been the best experience of their lives and parents routinely write and tell us of the positive changes camp has had on their children.
Research carried out by Yale University has helped us quantify the benefits of attending camp and they include increasing self-confidence, self-esteem, independence, openness to try new things and an increased interest in social activities.
For more information, contact us:
Phone number: 02392 477 110
Your feedback is important.
Thank you for attending conference. We would love to know your general feedback and the sessions and speakers enjoyed the most. This is easy to do within the app.
To get started, make sure you have downloaded the app from the Apple App Store or Google Play. More details on how to do this can be found on the ASCAT Conference App tab.
1. If you haven’t already set up a profile, you need to do so in order to submit your feedback.
2. There are three general feedback questions available in the Activity Stream Tab. To reach these, tap the side bar and then ‘Activity Stream’.
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Next, make sure you rate the sessions and speakers.
4. To do this, tap the side bar and then ‘Schedule’.
5. Select the day from the top, and session that you attended.
6. Tap the rating bar, choose your rating and tap ‘Submit’. Then choose each of the speakers from that session.
7. Select the rating bar, choose your rating and tap ‘Submit’.
Remember to rate all the sessions you attended and speakers that you heard.
Dr Marina Kleanthous, Professor at The Cyprus School of Molecular Medicine, Head Molecular genetics, Thlassaemia Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus – Prevention of hamoglobinopathies: new methodologies and procedures
Dr Marsha Treadwell, Psychologist and Clinical Health services researcher, UCSF Benioff Children’s Hospital Oakland and Co-principal investigator, Pacific sickle cell regional collaborative – Search for a cure for sickle cell disease and thalassemia should be the priority – Yes
Dr Miguel Abboud, Professor and Chairman, Department of Pediatrics and Adolescent Medicine, American University of Beirut – Medical Center, Children’s Cancer Center of Lebanon, Beirut, Lebanon – New Therapies for sickle cell disease
24 June 2019