Özdoğu Hakan, Boğa Can
Turk J Haematol. 2015 Sep;32(3):195-205. doi: 10.4274/tjh.2014.0311.
Sickle cell disease-related organ injuries cannot be prevented despite hydroxyurea use, infection prophylaxis, and supportive therapies. As a consequence, disease-related mortality reaches 14% in adolescents and young adults. Hematopoietic stem cell transplantation is a unique curative therapeutic approach for sickle cell disease. Myeloablative allogeneic hematopoietic stem cell transplantation is curative for children with sickle cell disease. Current data indicate that long-term disease-free survival is about 90% and overall survival about 95% after transplantation. However, it is toxic in adults due to organ injuries. In addition, this curative treatment approach has several limitations, such as difficulties to find donors, transplant-related mortality, graft loss, graft-versus-host disease (GVHD), and infertility. Engraftment effectivity and toxicity for transplantations performed with nonmyeloablative reduced-intensity regimens in adults are being investigated in phase 1/2 trials at many centers. Preliminary data indicate that GVHD could be prevented with transplantations performed using reduced-intensity regimens. It is necessary to develop novel regimens to prevent graft loss and reduce the risk of GVHD.
尽管使用了羟基脲、进行了感染预防以及采取了支持性治疗，但镰状细胞病相关的器官损伤仍无法预防。因此，青少年和年轻成年人中与疾病相关的死亡率达到了14%。造血干细胞移植是镰状细胞病一种独特的治愈性治疗方法。清髓性异基因造血干细胞移植对患有镰状细胞病的儿童具有治愈作用。目前的数据表明，移植后长期无病生存率约为90%，总生存率约为95%。然而，由于器官损伤，它对成年人有毒性。此外，这种治愈性治疗方法有几个局限性，比如难以找到供体、移植相关死亡率、移植物丢失、移植物抗宿主病（GVHD）以及不育。许多中心正在进行1/2期试验，研究非清髓性低强度方案在成人移植中的植入有效性和毒性。初步数据表明，使用低强度方案进行移植可以预防GVHD。有必要开发新的方案来预防移植物丢失并降低GVHD风险。
