Bone marrow transplantation for severe combined immune deficiency
Bone marrow transplantation for severe combined immune deficiency
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DOI：
10.1001/jama.295.5.508
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发表时间：
2006-02-01
影响因子：
120.7
通讯作者：
Roifman, CM
中科院分区：
文献类型：
--
作者：
Grunebaum, E;Mazzolari, E;Roifman, CM
骨髓移植（BMT）使用的干细胞来自家族相关的HLA相合供者（RID），是严重联合免疫缺陷（SCID）患者的最佳治疗方法。在没有RID的情况下，经常使用HLA错配的相关供体（MMRD）。然而，与RIDS相比，使用MMRD进行BMT与生存率降低和长期免疫重建较差相关。HLA匹配的无关供体（MUD）的使用代表了BMT.Objective的另一种潜在替代方案，比较接受RID，MUD或MMRD BMT的SCID患者的结果和免疫重建。回顾性研究了1990年至2004年间94名诊断为SCID的婴儿的病历，这些婴儿在1名加拿大和1名意大利儿科转诊时接受了BMT中心分别有13例、41例和40例患者接受了RID、MUD和MMRD BMT.Main Outcome Measures生存率和移植物衰竭，沿着移植物抗宿主病、感染和其他并发症的发生率;对BMT.Results后存活超过2年的儿童进行免疫重建评估。13例接受RID BMT的患者中有12例（92.3%）存活，41例接受MUD BMT的患者中有33例（80.5%）存活，40例接受MMRD BMT的患者中有21例（52.5%）存活。与MMRD BMT相比，RID（P = 0.008）或MUD（P = 0.03）的生存率显著较高。接受MMRD BMT的患者移植失败和需要重复BMT的情况比接受MUD BMT的患者更常见。MUD BMT（94.7%）比MMRD BMT（61.1%）更常实现完整T细胞库的长期重建（P = .02）。急性移植物抗宿主病在MUD BMT后的患者中有73.1%，但在MMRD BMT后仅为45%（P = 0.009）。相反，间质性肺炎观察后MMRD BMT（14 [35.0%] 40）比后MUD BMT（3 [7.3%] 41; P = 0.002）更频繁。结论我们的研究表明，在没有一个相对相同的HLA，MUD BMT可能会提供更好的植入，免疫重建和生存的SCID患者比MMRD BMT。
Context Bone marrow transplantation (BMT) using stem cells obtained from a family-related, HLA-identical donor (RID) is the optimal treatment for patients with severe combined immune deficiency (SCID). in the absence of an RID, HLA-mismatched related donors (MMRDs) are often used. However, compared with RIDS, use of MMRDs for BMT is associated with reduced survival and inferior long-term immune reconstitution. Use of HLA-matched unrelated donors (MUDs) represents another potential alternative for BMT.Objective To compare outcomes and immune reconstitution in a large cohort of patients with SCID who received RID, MUD, or MMRD BMT.Design, Setting, and Patients Retrospective study of medical records from 94 infants diagnosed as having SCID who received BMT between 1990 and 2004 at 1 Canadian and 1 Italian pediatric referral center. Thirteen, 41, and 40 patients received RID, MUD, and MMRD BMT, respectively.Main Outcome Measures Survival and graft failure, along with incidence of graft-vs-host disease, infections, and other complications; immune reconstitution was assessed in children who survived for more than 2 years after BMT.Results Survival after RID BMT was highest. Twelve (92.3%) of 13 patients who received RID BMT, 33 (80.5%) of 41 who received MUD BMT, and 21 (52.5%) of 40 patients who received MMRD BMT survived. Compared with MMRD BMT, survival was significantly higher with RID (P = .008) or with MUD (P = .03). Graft failures and need for repeat BMT were more common in patients receiving MMRD BMT than in those who underwent MUD BMT. Long-term reconstitution of a full T-cell repertoire was achieved more frequently following MUD BMT (94.7%) than after MMRD BMT (61.1%) (P = .02). Acute graft-vs-host disease was documented in 73.1% of patients following MUD BMT but in only 45% after MMRD BMT (P = .009). Conversely, interstitial pneumonitis was observed more frequently after MMRD BMT (14 [35.0%] of 40) than after MUD BMT (3 [7.3%] of 41; P = .002).Conclusion Our study suggests that in the absence of a relative with identical HLA, MUD BMT may provide better engraftment, immune reconstitution, and survival for patients with SCID than MMRD BMT.