Lucarelli G, Polchi P, Galimberti M, Izzi T, Delfini C, Manna M, Agostinelli F, Baronciani D, Giorgi C, Angelucci E
Lancet. 1985 Jun 15;1(8442):1355-7. doi: 10.1016/s0140-6736(85)91784-2.
30 patients with homozygous beta-thalassaemia aged 6 months to 7 years received allogeneic marrow transplants following busulphan (Bu) and cyclophosphamide (Cy). Post-transplant immunosuppression was with methotrexate (MTX) or MTX and Cy. The first 6 patients received 16 mg/kg Bu and 200 mg/kg Cy. 3 died of transplant-related complications and 3 survived without thalassaemia 701-762 days after transplantation. The subsequent 24 patients received 14 mg/kg Bu and 200 mg/kg Cy. 1 died on day 28 without engraftment and 23 survived 64-624 days after transplantation. 19 of the 23 surviving patients are without thalassaemia while 4 patients with initial engraftment became thalassaemic again in 32-46 days and survived 253-624 days after transplantation. The latest death was 50 days after transplantation. The probability of developing grade 2 or greater acute graft versus host disease (GVHD) in patients with sustained engraftment was 23%. 5 patients had chronic GVHD which is still active and causing disability in 3 patients 358, 456, and 477 days after transplantation. The actuarial survival was 86% and the actuarial disease-free survival was 73%.
30例年龄在6个月至7岁的纯合子β地中海贫血患者在接受白消安（Bu）和环磷酰胺（Cy）治疗后接受了异基因骨髓移植。移植后免疫抑制采用甲氨蝶呤（MTX）或MTX联合Cy。前6例患者接受16mg/kg Bu和200mg/kg Cy。3例死于移植相关并发症，3例移植后701 - 762天无地中海贫血存活。随后的24例患者接受14mg/kg Bu和200mg/kg Cy。1例在第28天未植入而死亡，23例移植后64 - 624天存活。23例存活患者中19例无地中海贫血，4例最初植入的患者在32 - 46天再次出现地中海贫血，移植后存活253 - 624天。最近1例死亡发生在移植后50天。持续植入患者发生2级或更高级别急性移植物抗宿主病（GVHD）的概率为23%。5例发生慢性GVHD，其中3例在移植后358、456和477天仍处于活动期并导致残疾。精算生存率为86%，无病生存率为73%。
