Hôpital Necker-Enfants malades, Service de pédiatrie générale et maladies infectieuses, 149 rue de Sèvres, 75015 Paris
- Key words: hydroxyurea, hematopoietic stem cell transplant, gene therapy
- DOI : 10.1684/mtp.2018.0657
- Page(s) : 281-4
- Published in: 2017
Understanding of the pathophysiological mechanisms in sickle cell disease (SCD) has been recently greatly increased, opening new therapeutic ways. So far, the most used treatment is Hydroxycarbamide (previously licensed as Hydrea®, licensed in Europe for SCD as Siklos®). A more than 20 years-experience in children has evidenced a dramatic decrease in the rate of painful events, acute chest syndrome, and transfusion needs. New drugs are undergoing development, especially ones targeting interactions between white and red cells, and vascular endothelium. Hematopoietic stem cell transplant (bone marrow transplant) is a curative treatment, which has been used for more than 30 years. Excellent results are observed in patients transplanted from HLA-identical siblings, but less than 20% of patients have such donor. Also, short and long-term risks have to be weighted against benefits. A recent favorable outcome after gene therapy offers a real hope.