Ferry C, Gemayel G, Rocha V, Labopin M, Esperou H, Robin M, de Latour R P, Ribaud P, Devergie A, Leblanc T, Gluckman E, Baruchel A, Socié G
Hematopoietic Stem Cell Transplant, Hôpital Saint Louis, Paris, France.
Bone Marrow Transplant. 2007 Aug;40(3):219-24. doi: 10.1038/sj.bmt.1705710. Epub 2007 May 28.
We analyzed long-term outcomes and psycho-social aspects in 112 children with malignancies surviving 1 year after hematopoietic stem cell transplantation. At 10 years, overall survival was 75+/-5%, TRM 18+/-4% and relapse 14+/-3%; 10-year cumulative incidence of infections was 31+/-4%, cataract 44+/-4%, pulmonary dysfunction 20+/-4%, bone and joint complications 29+/-5%, hypothyroidism 36+/-4%, cardiac complications 11+/-3% and secondary malignancies 7+/-3%. Total body irradiation (TBI) was the most significant risk factor associated with cataract, pulmonary impairment, osteoarticular complications and hypothyroidism. Chronic graft-versus-host disease was associated with higher incidence of pulmonary dysfunction. The number of complications per patient increased with time. Half of the patients had psychological disturbance, 13 signs of depression and 16 a history of eating behavior disorders; 54% of patients with one or more long-term complications had psychological problems. Sixty-nine patients had learning difficulties and 36 achieved normal scholarship. With increased follow-up, development of late effects and of psycho-social disturbance are of major concern. While the use of single-dose TBI has now been abandoned, other risk factors are still of concern in the early 2000s.
我们分析了112例造血干细胞移植术后存活1年的恶性肿瘤患儿的长期预后及心理社会方面的情况。10年时，总生存率为75±5%，移植相关死亡率为18±4%，复发率为14±3%；10年感染累积发生率为31±4%，白内障为44±4%，肺功能障碍为20±4%，骨与关节并发症为29±5%，甲状腺功能减退为36±4%，心脏并发症为11±3%，继发性恶性肿瘤为7±3%。全身照射（TBI）是与白内障、肺损伤、骨关节并发症和甲状腺功能减退相关的最显著危险因素。慢性移植物抗宿主病与肺功能障碍的较高发生率相关。每位患者的并发症数量随时间增加。一半的患者有心理障碍，13例有抑郁迹象，16例有饮食行为障碍史；有一项或多项长期并发症的患者中54%有心理问题。69例患者有学习困难，36例学业成绩正常。随着随访时间的增加，迟发效应和心理社会障碍的发生成为主要关注点。虽然现在已不再使用单剂量TBI，但在21世纪初其他危险因素仍令人担忧。
