Faraci M, Békássy A N, De Fazio V, Tichelli A, Dini G
Bone Marrow Transplantation Unit, Department of Haematology-Oncology, G Gaslini Children's Research Institute, Genova, Italy.
Bone Marrow Transplant. 2008 Jun;41 Suppl 2:S49-57. doi: 10.1038/bmt.2008.55.
Non-endocrine events represent a heterogeneous group of complications occurring in children who survive long term after haematopoietic SCT. This review highlights the late sequel in a growing child. The preparative regimen itself with high-dose chemotherapy and/or radiotherapy (TBI) or the treatment given before the transplant procedure may cause organ damage with permanent sequel. Immune reconstitution and chronic GvHD have crucial role in occurrence of clinical abnormalities and late severe infections. Autoimmune syndromes may occur after use of novel transplant modalities (cord blood transplantation, reduced intensity conditioning regimen and haploidentical T-cell-depleted SCTs). Exposure to chemo- and/or radiotherapy increases the risk of second malignant neoplasms. Surveillance strategy focusing on each potential complication risk at continuous follow-up will allow vigilant post transplant care. Each paediatrician must be well versed in appropriate monitoring of these complications. Guidelines and recommendations are provided for serious problems occurring at follow-up, which must rapidly be identified so that appropriate intervention can be initiated. To achieve cure at a lowest possible price in terms of suffering and cost expenditures for health care is an extended frontier of paediatric haematopoietic SCT and biggest challenge for a paediatrician.
非内分泌事件是造血干细胞移植后长期存活儿童中出现的一组异质性并发症。本综述重点介绍了成长中儿童的晚期后遗症。高剂量化疗和/或放疗（全身照射）的预处理方案本身或移植手术前给予的治疗可能会导致器官损伤并留下永久性后遗症。免疫重建和慢性移植物抗宿主病在临床异常和晚期严重感染的发生中起关键作用。使用新型移植方式（脐血移植、减低强度预处理方案和单倍体T细胞去除的造血干细胞移植）后可能会出现自身免疫综合征。接触化疗和/或放疗会增加患第二原发性恶性肿瘤的风险。在持续随访中针对每种潜在并发症风险的监测策略将有助于进行警惕的移植后护理。每位儿科医生都必须精通对这些并发症的适当监测。针对随访中出现的严重问题提供了指南和建议，必须迅速识别这些问题以便启动适当的干预措施。以尽可能低的痛苦代价和医疗保健成本支出实现治愈是儿科造血干细胞移植的一个拓展前沿领域，也是儿科医生面临的最大挑战。
