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

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.

• Authors: Claudia Wehr, Andrew R Gennery, Caroline Lindemans, Ansgar Schulz, Manfred Hoenig, Reinhard Marks, Mike Recher, Bernd Gruhn, Andreas Holbro, Ingmar Heijnen, Deborah Meyer, Goetz Grigoleit, Hermann Einsele, Ulrich Baumann, Thorsten Witte, Karl-Walter Sykora, Sigune Goldacker, Lorena Regairaz, Serap Aksoylar, Ömur Ardeniz, Marco Zecca, Przemyslaw Zdziarski, Isabelle Meyts, Susanne Matthes-Martin, Kohsuke Imai, Chikako Kamae, Adele Fielding, Suranjith Seneviratne, Nizar Mahlaoui, Mary A Slatter, Tayfun Güngör, Peter D Arkwright, Joris van Montfrans, Kathleen E Sullivan, Bodo Grimbacher, Andrew Cant, Hans-Hartmut Peter, Juergen Finke, H Bobby Gaspar, Klaus Warnatz, Marta Rizzi, Inborn Errors Working Party of the European Society for Blood and Marrow Transplantation and the European Society for Immunodeficiency
• Journal: The Journal of allergy and clinical immunology (J Allergy Clin Immunol) Vol. 135 Issue 4 Pg. 988-997.e6 (Apr 2015) ISSN: 1097-6825 [Electronic] United States
• PMID: 25595268 (Publication Type: Journal Article, Multicenter Study, Research Support, Non-U.S. Gov't)
• Copyright: Copyright © 2014 American Academy of Allergy, Asthma & Immunology. Published by Elsevier Inc. All rights reserved.

Topics

• 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