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

Wehr, Claudia and Gennery, Andrew R. and Lindemans, Caroline and Schulz, Ansgar and Hoenig, Manfred and Marks, Reinhard and Recher, Mike and Gruhn, Berd and Holbro, Andreas and Heijnen, Ingmar and Meyer, Deborah and Grigoleit, Goetz and Einsele, Hermann and Baumann, Ulrich and Witte, Thorsten and Sykora, Karl-Walter and Goldacker, Sigune and Regairaz, Lorena and Aksoylar, Serap and Ardeniz, Ömur and Zecca, Marco and Zdziarski, Przemyslaw and Meyts, Isabelle and Matthes-Martin, Susanne and Imai, Kohsuke and Kamae, Chikako and Fielding, Adele and Seneviratne, Suranjith and Mahlaoui, Nizar and Slatter, Mary A. and Gungor, Tayfun and Arkwright, Peter D. and van Montfrans, Joris and Sullivan, Kathleen E. and Grimbacher, Bodo and Cant, Andrew and Peter, Hans-Hartmut and Finke, Juergen and Gaspar, H. Bobby and Warnatz, Klaus and Rizzi, Marta and Inborn Errors Working Party of the European Society for, Blood and Marrow, Transplantation and the European Society for, Immunodeficiency. (2015) Multicenter experience in hematopoietic stem cell transplantation for serious complications of common variable immunodeficiency. J Allergy Clin Immunol, 135 (4). pp. 988-97e6.

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Official URL: https://edoc.unibas.ch/62407/

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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.
Faculties and Departments:03 Faculty of Medicine > Departement Biomedizin > Department of Biomedicine, University Hospital Basel > Immunodeficiency (Recher)
UniBasel Contributors:Recher, Mike
Item Type:Article, refereed
Article Subtype:Research Article
ISSN:1097-6825 (Electronic) 0091-6749 (Linking)
Note:Publication type according to Uni Basel Research Database: Journal article
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Last Modified:24 Nov 2018 11:20
Deposited On:24 Nov 2018 11:20

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