Department of Paediatric Immunology, Newcastle upon Tyne Hospital NHS Foundation Trust, United Kingdom.
Ann N Y Acad Sci. 2011 Nov;1238:122-31. doi: 10.1111/j.1749-6632.2011.06243.x.
Hematopoietic stem cell transplantation (HSCT) is now highly successfully curing a widening range of primary immunodeficiencies (PIDs). Better tissue typing, matching of donors, less toxic chemotherapy, better virus detection and treatment, improved supportive care, and graft-versus-host disease prophylaxis mean up to a 90% cure for severe combined immunodeficiency patients and a 70-80% cure for other PIDs given a matched unrelated donor, and rising to 95% for young patients with specific PIDs, such as Wiskott-Aldrich syndrome. Precise molecular diagnosis, detailed data on prognosis, and careful pre-HSCT assessment of infective lung and liver damage will ensure an informed benefit analysis of HSCT and the best outcome. It is now recognized that the best treatment option for chronic granulomatous disease is HSCT, which can also be curative for CD40 ligand deficiency and complex immune dysregulation disorders.
造血干细胞移植（HSCT）现在已经成功治愈了越来越多的原发性免疫缺陷病（PID）。更好的组织配型、供者匹配、毒性更低的化疗、更好的病毒检测和治疗、改进的支持性护理以及移植物抗宿主病预防意味着对于严重联合免疫缺陷病患者，高达 90%的治愈率，对于其他 PID 患者，如果有匹配的无关供者，则治愈率为 70-80%，对于特定 PID 患者（如 Wiskott-Aldrich 综合征），治愈率高达 95%。精确的分子诊断、详细的预后数据以及 HSCT 前对感染性肺和肝损伤的仔细评估将确保 HSCT 的知情获益分析和最佳结果。现在已经认识到，慢性肉芽肿病的最佳治疗选择是 HSCT，它也可以治愈 CD40 配体缺乏症和复杂免疫失调疾病。
