Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies:: report of the European experience 1968-99
Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies:: report of the European experience 1968-99
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DOI：
10.1016/s0140-6736(03)12513-5
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发表时间：
2003-02-15
期刊：
影响因子：
168.9
通讯作者：
Fischer, A
中科院分区：
文献类型：
--
作者：
Antoine, C;Müller, S;Fischer, A
背景异基因造血干细胞移植可以治疗多种原发性免疫缺陷。这份欧洲报告的重点是1968年和1999年12月之间，原发性immunodeficiency.Methods，该报告包括来自37个中心在18个国家，参加了在欧洲登记的干细胞移植严重联合免疫缺陷（SCID）和其他免疫缺陷疾病（非SCID）的数据，这样的程序的长期结果。研究了919例患者的1082例移植（475例SCID患者中566例，444例非SCID患者中512例;由于数据不足，排除了4例手术）。结果：在SCID中，HLA相合移植后持续植入的3年生存率显著高于不相合移植后（77% vs 54%; p=0.002），生存率随时间推移而提高。在HLA不匹配的干细胞移植中，B（-）SCID的预后比B（+）SCID差。然而，随着时间的推移，改善发生在两个SCID表型。在非SCID患者中，基因型HLA匹配、表型HLA匹配、HLA不匹配相关和无关供体移植后3年生存率分别为71%、42%、42%和59%（p=0.0006）。急性移植物抗宿主病预测预后不良，无论捐助者的起源，除了在相关的HLA-相同的移植在SCID.Interpretation生存随着时间的推移的改善表明更有效的预防和治疗疾病相关的和程序相关的并发症，如感染和移植物抗宿主病。一个重要的因素是通过使用更有效的T细胞耗竭方法更好地预防HLA不一致背景下的移植物抗宿主病。对于非SCID，干细胞移植可以提供治疗，来自无关供体的移植物几乎与来自遗传上HLA相同的亲属的移植物一样有益。
Background Transplantation of allogeneic haemopoietic stem cells can cure several primary immunodeficiencies. This European report focuses on the long-term results of such procedures done between 1968 and December, 1999, for primary immunodeficiencies.Methods The report includes data from 37 centres in 18 countries, which participated in a European registry for stem-cell transplantation in severe combined immunodeficiencies (SCID) and in other immunodeficiency disorders (non-SCID). 1082 transplants in 919 patients were studied (566 in 475 SCID patients, 512 in 444 non-SCID patients; four procedures excluded owing to insufficient data). Minimum follow-up of 6 months was required.Findings In SCID, 3-year survival with sustained engraftment was significantly better after HLA-identical than after mismatched transplantation (77% vs 54%; p=0.002) and survival improved over time. In HLA-mismatched stem-cell transplantation, B(-) SCID had poorer prognosis than B(+) SCID. However, improvement with time occurred in both SCID phenotypes. In non-SCID, 3-year survival after genotypically HLA-matched, phenotypically HLA-matched, HLA-mismatched related, and unrelated-donor transplantation was 71%, 42%, 42%, and 59%, respectively (p=0.0006). Acute graft versus host disease predicted poor prognosis whatever the donor origin except in related HLA-identical transplantation in SCID.Interpretation The improvement in survival over time indicates more effective prevention and treatment of disease-related and procedure-related complications-eg, infections and graft versus host disease. An important factor is better prevention of graft versus host disease in the HLA-non-identical setting by use of more efficient methods of T-cell depletion. For non-SCID, stem-cell transplantation can provide a cure, and grafts from unrelated donors are almost as beneficial as those from genetically HLA-identical relatives.