Service d'hémato-immunologie pédiatrique, hôpital Robert-Debré, AP-HP, 48, boulevard Sérurier, 75935 Paris cedex 19, France.
C R Biol. 2013 Mar;336(3):148-51. doi: 10.1016/j.crvi.2012.09.004. Epub 2012 Oct 12.
Hematopoietic stem cell transplantation (HSCT) is the one and only curative therapy available for patient with severe sickle cell disease (SCD). Until today, several hundreds of patients have undergone geno-identical HSCT. More than 200 patients were transplanted in France. The first indication was cerebral vasculopathy. Among both malignant and non-malignant diseases treated with HSCT, the success rate obtained in SCD patients appears as the best one. From the year 2000, more than 95% of transplanted patients survived the HSCT procedure and more than 90% are completely cured and experience a very satisfying health condition post-transplantation. However, the current standard procedure includes a myeloablative conditioning regimen for warranting engraftment. Such regime is linked to severe long-term side effects such as hypofertility. Due to the excellent obtained results, we have to think about a possible widening of indications, a decrease of conditioning intensity and toxicity, and about HSCT from alternative stem cell sources, such as mismatch family donor, unrelated volunteer donor or unrelated cord blood.
造血干细胞移植（HSCT）是治疗严重镰状细胞病（SCD）患者的唯一有效方法。迄今为止，已有数百名患者接受了基因匹配的 HSCT。其中 200 多名患者在法国接受了移植。第一个适应症是脑血管病变。在接受 HSCT 治疗的恶性和非恶性疾病中，SCD 患者的成功率似乎是最高的。自 2000 年以来，超过 95%的接受移植的患者成功完成了 HSCT 手术，超过 90%的患者完全治愈，并且在移植后健康状况非常令人满意。然而，目前的标准程序包括使用清髓性预处理方案来保证植入。这种方案与严重的长期副作用有关，如生育能力下降。由于取得了优异的结果，我们必须考虑扩大适应症、降低预处理的强度和毒性，并考虑使用替代干细胞来源进行 HSCT，如不匹配的家族供体、无关的志愿供体或无关的脐带血。
