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

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 ArdenizMarco 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

Research output: Contribution to journalArticlepeer-review

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.

Original languageEnglish
Pages (from-to)988-997.e6
JournalJournal of Allergy and Clinical Immunology
Volume135
Issue number4
DOIs
Publication statusPublished - Apr 1 2015

Keywords

  • Common variable immunodeficiency
  • hematopoietic stem cell transplantation
  • hypogammaglobulinemia
  • immunoglobulin substitution/replacement
  • immunologic reconstitution
  • mortality
  • outcome
  • survival

ASJC Scopus subject areas

  • Immunology and Allergy
  • Immunology
  • Medicine(all)

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