Allali S, Taylor M, Albinni S, Amiranoff D, de Montalembert M
Department of general pediatrics and pediatric infectious diseases, Paris Descartes university, Necker-Enfants malades hospital, AP-HP, 149, rue de Sèvres, 75015 Paris, France; Laboratory of excellence, GR-Ex, 75015 Paris, France; Pediatric reference center for sickle cell disease, 75015 Paris, France.
Établissement français du sang, Necker-Enfants malades hospital, 149, rue de Sèvres, 75015 Paris, France.
Transfus Clin Biol. 2019 Sep;26(3):147-149. doi: 10.1016/j.tracli.2019.06.323. Epub 2019 Jun 29.
Thalassemia and sickle cell disease (SCD) are among the most common inherited diseases worldwide. Red blood cell transfusion is a cornerstone of their treatment, but its indications have significantly changed over the past years. New therapies are emerging in both syndromes: among them, hematopoietic stem cell transplantation is now routinely proposed, and gene therapy has shown promising preliminary results.
地中海贫血和镰状细胞病(SCD)是全球最常见的遗传性疾病。红细胞输血是其治疗的基石,但在过去几年中其适应症已发生显著变化。这两种综合征都出现了新的治疗方法:其中,造血干细胞移植现在已常规推荐,基因治疗也已显示出有希望的初步结果。