Gavrilova Tatyana
Division of Allergy and Immunology, Montefiore Medical Center, Bronx, NY 10461, United States.
World J Transplant. 2019 Jul 31;9(3):48-57. doi: 10.5500/wjt.v9.i3.48.
Primary immunodeficiency disorders (PIDs) result from inborn errors in immunity. Susceptibility to infections and oftentimes severe autoimmunity pose life-threatening risks to patients with these disorders. Hematopoietic cell transplant (HCT) remains the only curative option for many. Severe combined immunodeficiency disorders (SCID) most commonly present at the time of birth and typically require emergent HCT in the first few weeks of life. HCT poses an unusual challenge for PIDs. Donor source and conditioning regimen often impact the outcome of immune reconstitution after HCT in PIDs. The use of matched or unmatched, as well as related versus unrelated donor has resulted in variable outcomes for different subsets of PIDs. Additionally, there is significant variability in the success of engraftment even for a single patient's lymphocyte subpopulations. While certain cell lines do well without a conditioning regimen, others will not reconstitute unless conditioning is used. The decision to proceed with a conditioning regimen in an already immunocompromised host is further complicated by the fact that alkylating agents should be avoided in radiosensitive PIDs. This manuscript reviews some of the unique elements of HCT in PIDs and evidence-based approaches to transplant in patients with these rare and challenging disorders.
原发性免疫缺陷病（PID）由先天性免疫缺陷引起。感染易感性以及通常严重的自身免疫性给患有这些疾病的患者带来了危及生命的风险。造血细胞移植（HCT）仍然是许多患者唯一的治愈选择。严重联合免疫缺陷病（SCID）最常在出生时出现，通常需要在出生后的头几周内紧急进行HCT。HCT对PID构成了特殊挑战。供体来源和预处理方案通常会影响PID患者HCT后免疫重建的结果。使用匹配或不匹配的供体，以及相关供体与无关供体，导致不同亚型PID的结果各不相同。此外，即使对于单个患者的淋巴细胞亚群，植入成功率也存在显著差异。虽然某些细胞系在没有预处理方案的情况下表现良好，但其他细胞系除非使用预处理，否则不会重建。在已经免疫受损的宿主中决定是否进行预处理方案会因以下事实而更加复杂：在对放射敏感的PID中应避免使用烷化剂。本文综述了PID患者HCT的一些独特要素以及针对这些罕见且具有挑战性疾病患者的基于证据的移植方法。
