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Multicenter experience in hematopoietic stem cell transplantation for serious complications of common variable immunodeficiency

Lookup NU author(s): Professor Andrew GenneryORCiD, Dr Mary Slatter, Professor Andrew Cant

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Abstract

© 2014 American Academy of Allergy, Asthma & Immunology. 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.


Publication metadata

Author(s): Wehr C, Gennery AR, Lindemans C, Schulz A, Hoenig M, Marks R, Recher M, Gruhn B, Holbro A, Heijnen I, Meyer D, Grigoleit G, Einsele H, Baumann U, Witte T, Sykora K-W, Goldacker S, Regairaz L, Aksoylar S, Ardeniz O, Zecca M, Zdziarski P, Meyts I, Matthes-Martin S, Imai K, Kamae C, Fielding A, Seneviratne S, Mahlaoui N, Slatter MA, Gungor T, Arkwright PD, Van Montfrans J, Sullivan KE, Grimbacher B, Cant A, Peter H-H, Finke J, Gaspar HB, Warnatz K, Rizzi M

Publication type: Article

Publication status: Published

Journal: Journal of Allergy and Clinical Immunology

Year: 2015

Volume: 135

Issue: 4

Pages: 988-997.e6

Print publication date: 01/04/2015

Online publication date: 14/01/2015

Acceptance date: 19/11/2014

ISSN (print): 0091-6749

ISSN (electronic): 1097-6825

Publisher: Mosby Inc.

URL: https://doi.org/10.1016/j.jaci.2014.11.029

DOI: 10.1016/j.jaci.2014.11.029

PubMed id: 25595268


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