Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: Entering a new century, do we do better?
Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: Entering a new century, do we do better?
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DOI：
10.1016/j.jaci.2010.06.015
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发表时间：
2010-09-01
影响因子：
14.2
通讯作者：
Landais, Paul
中科院分区：
文献类型：
--
作者：
Gennery, Andrew R.;Slatter, Mary A.;Landais, Paul
背景：造血干细胞移植仍然是大多数严重联合免疫缺陷 (SCID) 或其他原发性免疫缺陷 (非 SCID PID) 患者的唯一治疗方法。 目的： 分析 1968 年至 2005 年间欧洲中心接受治疗的 SCID 和非 SCID PID 患者的长期结局。对数秩检验比较了组间的生存率。 Cox 比例风险模型评估了独立预测因素对患者生存的影响。结果：在 SCID 患者中，从 2000 年到 2005 年，基因相同供体 (n = 25) 的生存率为 90%。使用不匹配的亲属 (n = 96) 的生存率有所提高 (66%)，与使用不相关的捐赠者的生存率相似 (n = 46; 69%; P = .005)。多变量分析表明，1995 年之后的移植、年龄较小、B+ 表型、基因相同和表型相同的供体、无呼吸障碍或移植前病毒感染与更好的预后相关。对于非 SCID PID，与 SCID 患者相比，我们确认，在 2000 年至 2005 年期间，使用无关供体 (n = 124) 的 3 年生存率与基因相同供体 (n = 73) 相似，均为 79%。表型相合移植 (n = 23) 的存活率为 76%，不匹配相关供体移植的存活率更差 (n = 47; 46%; P = .016)。结论：这是此类患者规模最大、随访时间最长的队列研究。不同的患者群体会出现具体的问题。尽管各组均有改善，但 B-SCID 患者的生存率比其他 SCID 患者更差。对于非 SCID PID，尽管现在治疗的病症更多，但其生存率比 SCID 更差。现在需要分析各个疾病类别，以便更好地了解特定疾病的预后并规划最佳治疗方案。 (J 过敏临床免疫学杂志 2010 年；126:602-10。)
Background: Hematopoietic stem cell transplantation remains the only treatment for most patients with severe combined immunodeficiencies (SCIDs) or other primary immunodeficiencies (non-SCID PIDs).Objective: To analyze the long-term outcome of patients with SCID and non-SCID PID from European centers treated between 1968 and 2005.Methods: The product-limit method estimated cumulative survival; the log-rank test compared survival between groups. A Cox proportional-hazard model evaluated the impact of independent predictors on patient survival.Results: In patients with SCID, survival with genoidentical donors (n = 25) from 2000 to 2005 was 90%. Survival using a mismatched relative (n = 96) has improved (66%), similar to that using an unrelated donor (n = 46; 69%; P = .005). Transplantation after year 1995, a younger age, B+ phenotype, genoidentical and phenoidentical donors, absence of respiratory impairment, or viral infection before transplantation were associated with better prognosis on multivariate analysis. For nonSCID PID, in contrast with patients with SCID, we confirm that, in the 2000 to 2005 period, using an unrelated donor (n = 124) gave a 3-year survival rate similar to a genoidentical donor (n = 73), 79% for both. Survival was 76% in phenoidentical transplants (n = 23) and worse in mismatched related donor transplants (n = 47; 46%; P = .016).Conclusion: This is the largest cohort study of such patients with the longest follow-up. Specific issues arise for different patient groups. Patients with B-SCID have worse survival than other patients with SCID, despite improvements in each group. For non-SCID PID, survival is worse than SCID, although more conditions are now treated. Individual disease categories now need to be analyzed so that disease-specific prognosis may be better understood and the best treatments planned. (J Allergy Clin Immunol 2010;126:602-10.)