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Journal of Allergy and Clinical Immunology

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Multicenter experience in hematopoietic stem cell transplantation for serious complications of common variable immunodeficiency - 03/04/15

Doi : 10.1016/j.jaci.2014.11.029 
Claudia Wehr, MD a, Andrew R. Gennery, MD b, Caroline Lindemans, MD, PhD c, Ansgar Schulz, MD d, Manfred Hoenig, MD d, Reinhard Marks, MD e, Mike Recher, MD f, Bernd Gruhn, MD g, Andreas Holbro, MD h, Ingmar Heijnen, PhD i, Deborah Meyer, BSc j, Goetz Grigoleit, MD k, Hermann Einsele, MD k, Ulrich Baumann, MD l, Thorsten Witte, MD m, Karl-Walter Sykora, MD n, Sigune Goldacker, MD a, Lorena Regairaz, MD o, Serap Aksoylar, MD p, Ömur Ardeniz, MD q, Marco Zecca, MD r, Przemyslaw Zdziarski, MD s, Isabelle Meyts, MD t, Susanne Matthes-Martin, MD u, Kohsuke Imai, MD v, Chikako Kamae, MD w, Adele Fielding, MD x, Suranjith Seneviratne, MD y, Nizar Mahlaoui, MD, MSc, MPH z, Mary A. Slatter, MD aa, Tayfun Güngör, MD j, Peter D. Arkwright, MD bb, Joris van Montfrans, MD cc, Kathleen E. Sullivan, MD, PhD dd, Bodo Grimbacher, MD a, Andrew Cant, MD b, Hans-Hartmut Peter, MD a, Juergen Finke, MD e, H. Bobby Gaspar, MD ee, Klaus Warnatz, MD a, ⁎ , Marta Rizzi, MD, PhD a, ⁎
on behalf of the

Inborn Errors Working Party of the European Society for Blood and Marrow Transplantation and the European Society for Immunodeficiency

a Center for Chronic Immunodeficiency (CCI), University Medical Center Freiburg and the University of Freiburg, Freiburg, Germany 
b Department of Paediatric Immunology, Newcastle Upon Tyne Hospitals Foundation Trust, Newcastle Upon Tyne, United Kingdom 
c Pediatric Blood and Bone Marrow Transplantation Program, UMC Utrecht, Utrecht, The Netherlands 
d Department of Pediatrics, University Medical Center Ulm, Ulm, Germany 
e Department of Hematology and Oncology, University Medical Center Freiburg, Freiburg, Germany 
f Clinic for Primary Immunodeficiency, Medical Outpatient Clinic and Immunodeficiency Laboratory, Department of Biomedicine, University Hospital, Basel, Switzerland 
g Klinik für Kinder- und Jugendmedizin, Universitätsklinikum Jena, Friedrich-Schiller-Universität Jena, Jena, Germany 
h Division of Hematology and Stem Cell Transplant Team, University Hospital Basel, Basel, Switzerland 
i Medical Immunology, Laboratory Medicine, University Hospital Basel, Basel, Switzerland 
j University Children's Hospital, Zurich, Switzerland 
k Department of Hematology/Oncology, University Medical Center Würzburg, Würzburg, Germany 
l Paediatric Pulmonology, Allergy and Neonatology, Hannover Medical School, Hannover, Germany 
m Clinic for Immunology and Rheumatology, Hannover Medical School, Hannover, Germany 
n Department of Pediatric Hematology and Oncology, University Hospital Hannover, Hannover, Germany 
o Unidad de Immunología, Hospital de Niños Sor María Ludovica La Plata, Buenos Aires, Argentina 
p Department of Pediatric Hematology & Oncology and BMT Center, Ege University, Bornova-Izmir, Turkey 
q Division of Allergy and Clinical Immunology, Ege University Medical Faculty, Izmir, Turkey 
r Oncoematologia Pediatrica, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy 
s Lower Silesian Center for Cellular Transplantation, Wroclaw, Poland 
t Department of Paediatrics, University Hospital Leuven, Leuven, Belgium 
u St Anna Kinderspital, Medical University, Vienna, Austria 
v Department of Pediatrics, Tokyo Medical and Dental University, Tokyo, Japan 
w Department of Pediatrics, National Defense Medical College, Saitama, Japan 
x University College London, London, United Kingdom 
y Immunology Department, Royal Free London, London, United Kingdom 
z Unité d'Immuno-Hématologie et Rhumatologie Pédiatrique, Hôpital Necker-Enfants Malades, French National Reference Center for PIDs (CEREDIH), Stem Cell Transplantation for PIDs in Europe (SCETIDE) registry, Assistance Publique–Hôpitaux de Paris, Paris, France 
aa Institute of Cellular Medicine, Newcastle University, Newcastle upon Tyne, United Kingdom 
bb University of Manchester, Royal Manchester Children's Hospital, Manchester, United Kingdom 
cc Pediatric Immunology and Infectious Disease, UMC Utrecht, Utrecht, The Netherlands 
dd Division of Allergy and Immunology, Children's Hospital of Philadelphia, Philadelphia, Pa 
ee Center of Immunodeficiency, Molecular Immunology Unit, Institute of Child Health, London, United Kingdom 

∗Corresponding author: Marta Rizzi, MD, PhD, Center of Chronic Immunodeficiency, University Medical Center Freiburg, Engesserstrasse 4, 79108 Freiburg, Germany.∗Klaus Warnatz, MD, Center of Chronic Immunodeficiency, University Medical Center Freiburg, Breisacherstrasse 117, 79106 Freiburg, Germany.

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.

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Key words : Common variable immunodeficiency, hypogammaglobulinemia, hematopoietic stem cell transplantation, immunologic reconstitution, immunoglobulin substitution/replacement, outcome, mortality, survival

Abbreviations used : aGvHD, BM, cGvHD, CMV, CVID, ESID, GvHD, HCT-CI, HSCT, MAC, PID, RIC


Plan


 Supported by the German Federal Ministry of Education and Research (BMBF 01 EO 0803). C.L. is supported by a clinical fellowship from the Dutch Cancer Society (2013-5883).
 Disclosure of potential conflict of interest: C. Wehr has received research and travel support from the German Federal Ministry of Education and Research. C. Lindemans has received research support from the Dutch Cancer Society (2013-5883). A. Schulz is employed by Medical Center Ulm. M. Recher has received research support from a Swiss National Science Foundation Professorship Grant (PP00P3_144863). U. Baumann has received research support from EURO-PADnet (FP7/2007-2011). K.-W. Sykora has received travel support from EUSA Pharma. S. Goldacker has received research support from Octapharma. A. Fielding has received consultancy fees from Amgen. S. Seneviratne is employed by the Royal Free Hospital, London. K. E. Sullivan has received consultancy fees from the Immune Deficiency Foundation, is employed by UpToDate, and has received research support from Baxter. B. Grimbacher has received research support from BMBF (01E01303 and 012X1306F) and the European Union (EU); is employed by UCL and UKL-FR; has received research support from BMBF (01E01303 and 012X1306F), the EU, and Helmholtz (DZIF 8000805-3); and has received lecture fees from CSL, Baxter, and Biotest. H.-H. Peter has provided expert testimony for Pfizer and has received lecture fees. K. Warnatz has received lecture fees from Baxter, GlaxoSmithKline, CSL Behring, Pfizer, Biotest, Novartis Pharma, Stallergenes AG, Roche, Meridian HealthComms, Octapharma, and the American Academy of Allergy, Asthma & Immunology; has received payment for manuscript preparation from UCB Pharma; and has received payment for development of educational presentations from European Society for Immunodeficiency. M. Rizzi has received research support from Pfizer (Europe Aspire Award [10/2013-09/2014]) and Novartis (Stiftung für Klinische Forschung Grant [11/2014-10-2016]). The rest of the authors declare that they have no relevant conflicts of interest.


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Vol 135 - N° 4

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