期刊
JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
卷 135, 期 4, 页码 988-+出版社
MOSBY-ELSEVIER
DOI: 10.1016/j.jaci.2014.11.029
关键词
Common variable immunodeficiency; hypogammaglobulinemia; hematopoietic stem cell transplantation; immunologic reconstitution; immunoglobulin substitution/replacement; outcome; mortality; survival
资金
- German Federal Ministry of Education and Research (BMBF) [01 EO 0803]
- Dutch Cancer Society [2013-5883]
- MRC [G0501468] Funding Source: UKRI
- Medical Research Council [G0501468] Funding Source: researchfish
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.
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