Considerations for hematopoietic stem cell transplantation in primary immunodeficiency disorders

作     者：Tatyana Gavrilova 

作者机构：Division of Allergy and Immunology Montefiore Medical Center 

出 版 物：《World Journal of Transplantation》 (世界移植杂志)

年 卷 期：2019年第9卷第3期

页      面：48-57页

学科分类：10[医学] 

主　　题：Primary immunodeficiency disorders Hematopoietic stem cell transplant Autoimmunity Conditioning regimens Engraftment 

摘      要：Primary immunodeficiency disorders (PIDs) result from inborn errors in *** to infections and oftentimes severe autoimmunity pose life-threatening risks to patients with these *** cell transplant (HCT) remains the only curative option for *** combined immunodeficiency disorders (SCID) most commonly present at the time of birth and typically require emergent HCT in the first few weeks of *** poses an unusual challenge for *** source and conditioning regimen often impact the outcome of immune reconstitution after HCT in *** use of matched or unmatched, as well as related versus unrelated donor has resulted in variable outcomes for different subsets of ***, there is significant variability in the success of engraftment even for a single patient’s lymphocyte *** certain cell lines do well without a conditioning regimen, others will not reconstitute unless conditioning is *** 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 *** 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.