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Multicenter experience in hematopoietic stem cell transplantation for serious complications of common variable immunodeficiency
Journal
J Allergy Clin Immunol
Volume
135
Number
4
Pages / Article-Number
988-97.e6
Keywords
Adolescent; Adult; Cause of Death; Child; Common Variable Immunodeficiency/complications/mortality/*therapy; Female; Follow-Up Studies; Graft Survival; Graft vs Host Disease/etiology/prevention & control; *Hematopoietic Stem Cell Transplantation/adverse effects; *Hematopoietic Stem Cells; Humans; Male; Middle Aged; Retrospective Studies; Transplantation Conditioning; Treatment Outcome; Young Adult
Mesh terms
Adolescent; Adult; Cause of Death; Child; Common Variable Immunodeficiency, therapy; Female; Follow-Up Studies; Graft Survival; Graft vs Host Disease, prevention & control; Hematopoietic Stem Cell Transplantation, adverse effects; Hematopoietic Stem Cells; Humans; Male; Middle Aged; Retrospective Studies; Transplantation Conditioning; Treatment Outcome; Young Adult
Abstract
BACKGROUND: Common variable immunodeficiency (CVID) is usually well controlled with immunoglobulin substitution and immunomodulatory drugs. A subgroup of patients has a complicated disease course with high mortality. For these patients, investigation of more invasive, potentially curative treatments, such as allogeneic hematopoietic stem cell transplantation (HSCT), is warranted. OBJECTIVE: We sought to define the outcomes of HSCT for patients with CVID. METHODS: Retrospective data were collected from 14 centers worldwide on patients with CVID receiving HSCT between 1993 and 2012. RESULTS: Twenty-five patients with CVID, which was defined according to international criteria, aged 8 to 50 years at the time of transplantation were included in the study. The indication for HSCT was immunologic dysregulation in the majority of patients. The overall survival rate was 48%, and the survival rate for patients undergoing transplantation for lymphoma was 83%. The major causes of death were treatment-refractory graft-versus-host disease accompanied by poor immune reconstitution and infectious complications. Immunoglobulin substitution was stopped in 50% of surviving patients. In 92% of surviving patients, the condition constituting the indication for HSCT resolved. CONCLUSION: This multicenter study demonstrated that HSCT in patients with CVID was beneficial in most surviving patients; however, there was a high mortality associated with the procedure. Therefore this therapeutic approach should only be considered in carefully selected patients in whom there has been extensive characterization of the immunologic and/or genetic defect underlying the CVID diagnosis. Criteria for patient selection, refinement of the transplantation protocol, and timing are needed for an improved outcome.