Analysis

Are the risks of treatment to cure a child with severe sickle cell disease too high?

BMJ 2017; 359 doi: https://doi.org/10.1136/bmj.j5250 (Published 23 November 2017) Cite this as: BMJ 2017;359:j5250
  1. Mariane de Montalembert, professor1,
  2. Valentine Brousse, consultant paediatrician1,
  3. Subarna Chakravorty, consultant paediatric haematologist2,
  4. Antonio Pagliuca, professor3,
  5. John Porter, professor4,
  6. Paul Telfer, consultant haematologist5,
  7. Ajay Vora, professor6,
  8. David C Rees, professor2
  1. 1Department of Pediatrics, Reference Centre for Sickle Cell Disease, Hôpital Universitaire Necker-Enfants Malades, APHP, Paris, France
  2. 2Department of Paediatric Haematology, King’s College Hospital, London SE5 9RS, UK
  3. 3Department of Haematological Medicine, King’s College Hospital, London
  4. 4Department of Haematology, University College London Hospitals
  5. 5Department of Paediatric Haematology and Oncology, Barts Health NHS Trust, Royal London Hospital
  6. 6Department of Haematology, Great Ormond Street Hospital for Children, London
  1. Correspondence to: D Rees david.rees2{at}nhs.net

Haematopoietic stem cell transplantation from alternative donors is curative for children with sickle cell disease, but risk of death is high. David Rees and colleagues call for greater caution in its use

Key messages

  • Curative treatment for sickle cell disease is potentially available to all patients using haematopoietic stem cell transplantation from alternative donors, but the risks of these procedures are very high

  • We don’t know when these high risk transplants should be offered to children or how parents should be counselled to give appropriate consent

  • In high income countries, non-transplant treatments are being developed for sickle cell disease that are likely to improve prognosis

  • We need to develop safer transplants and to explore the ethics of offering high risk procedures to children with sickle cell disease and other chronic conditions

Sickle cell disease is one of the most common severe inherited conditions in the world. Around 300 000 babies are born with sickle cell disease each year, mostly in Africa, although there are roughly 100 000 affected people in the US and 50 000 in Europe.1 The prognosis for children born with the condition today varies enormously, particularly with geography: only about 20% of babies born in Africa survive to adulthood,2 whereas more than 93% of children survive to adulthood in Europe3 and the US,4 thanks to basic medical care, screening programmes, vaccinations, prophylactic antibiotics, blood transfusions, stroke prevention, and hydroxyurea.1 Median survival is 60 years in some high income countries, such as the UK .5

Children severely affected with sickle cell disease (around 5%) fail to respond to treatment with hydroxyurea or regular blood transfusion. These children may have acute complications, including frequent attacks of severe pain and acute chest syndrome, or have evidence of progressive organ damage such as cerebrovascular disease. They are …

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