Long-term outcome after hematopoietic stem cell transplantation of a single-center cohort of 90 patients with severe combined immunodeficiency
Long-term outcome after hematopoietic stem cell transplantation of a single-center cohort of 90 patients with severe combined immunodeficiency
复制标题
DOI：
10.1182/blood-2008-09-177923
复制
发表时间：
2009-04-23
期刊：
影响因子：
20.3
通讯作者：
Fischer, Alain
中科院分区：
文献类型：
--
作者：
Neven, Benedicte;Leroy, Sandrine;Fischer, Alain
异基因造血干细胞移植（HSCT）是治疗严重联合免疫缺陷（SCID）的有效方法。现在需要对HSCT的长期结果进行详细评估，即临床事件的发生以及免疫重建的质量和稳定性。我们对90例患者队列中HSCT的长期结局进行了单中心回顾性分析，随访时间为2 - 34年（中位数，14年）。收集临床事件和免疫重建数据。几乎一半的患者经历了一个或多个重大的临床事件，包括持续的慢性移植物抗宿主病（GVHD），自身免疫和炎症表现，机会性和非机会性感染，慢性人乳头瘤病毒（HPV）感染，并需要营养支持。与严重的HPV感染的显着例外，这些并发症往往变得不太常见15 - 15年后HSCT。多变量分析表明，这些事件的发生与非基因相同的供体，Artemis SCID的诊断和免疫重建的质量相关。在大多数情况下，HSCT能够长期生存，很少有后遗症。然而，相对迟发性并发症的发生是一个需要采取具体预防措施的问题，需要对患者进行仔细随访。（血。2009; 113：4114-4124）
Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative treatment for severe combined immunodeficiency (SCID). Detailed assessment of the longterm outcome of HSCT, ie, the occurrence of clinical events and the quality and stability of immune reconstitution, is now required. We performed a single-center retrospective analysis of the long-term outcome of HSCT in 90-patient cohort followed for between 2 and 34 years (median, 14 years). Clinical events and immune reconstitution data were collected. Almost half the patients have experienced one or more significant clinical events, including persistent chronic graft-versus-host disease (GVHD), autoimmune and inflammatory manifestations, opportunistic and nonopportunistic infections, chronic human papilloma virus (HPV) infections, and a requirement for nutritional support. With the notable exception of severe HPV infection, these complications tend to become less common 15 15 years later after HSCT. A multivariate analysis showed that the occurrence of these events correlated with non-genoidentical donors, diagnosis of Artemis SCID, and quality of immune reconstitution. In most cases, HSCT enables long-term survival with infrequent sequelae. However, the occurrence of relatively late-onset complications is a concern that requires specific means of prevention and justifies careful patient follow-up. (Blood. 2009; 113: 4114-4124)