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J Allergy Clin Immunol. 2015 Apr;135(4):988-97.e6. doi: 10.1016/j.jaci.2014.11.029. Epub 2015 Jan 14.

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

Author information

1
Center for Chronic Immunodeficiency (CCI), University Medical Center Freiburg and the University of Freiburg, Freiburg, Germany.
2
Department of Paediatric Immunology, Newcastle Upon Tyne Hospitals Foundation Trust, Newcastle Upon Tyne, United Kingdom.
3
Pediatric Blood and Bone Marrow Transplantation Program, UMC Utrecht, Utrecht, The Netherlands.
4
Department of Pediatrics, University Medical Center Ulm, Ulm, Germany.
5
Department of Hematology and Oncology, University Medical Center Freiburg, Freiburg, Germany.
6
Clinic for Primary Immunodeficiency, Medical Outpatient Clinic and Immunodeficiency Laboratory, Department of Biomedicine, University Hospital, Basel, Switzerland.
7
Klinik für Kinder- und Jugendmedizin, Universitätsklinikum Jena, Friedrich-Schiller-Universität Jena, Jena, Germany.
8
Division of Hematology and Stem Cell Transplant Team, University Hospital Basel, Basel, Switzerland.
9
Medical Immunology, Laboratory Medicine, University Hospital Basel, Basel, Switzerland.
10
University Children's Hospital, Zurich, Switzerland.
11
Department of Hematology/Oncology, University Medical Center Würzburg, Würzburg, Germany.
12
Paediatric Pulmonology, Allergy and Neonatology, Hannover Medical School, Hannover, Germany.
13
Clinic for Immunology and Rheumatology, Hannover Medical School, Hannover, Germany.
14
Department of Pediatric Hematology and Oncology, University Hospital Hannover, Hannover, Germany.
15
Unidad de Immunología, Hospital de Niños Sor María Ludovica La Plata, Buenos Aires, Argentina.
16
Department of Pediatric Hematology & Oncology and BMT Center, Ege University, Bornova-Izmir, Turkey.
17
Division of Allergy and Clinical Immunology, Ege University Medical Faculty, Izmir, Turkey.
18
Oncoematologia Pediatrica, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
19
Lower Silesian Center for Cellular Transplantation, Wroclaw, Poland.
20
Department of Paediatrics, University Hospital Leuven, Leuven, Belgium.
21
St Anna Kinderspital, Medical University, Vienna, Austria.
22
Department of Pediatrics, Tokyo Medical and Dental University, Tokyo, Japan.
23
Department of Pediatrics, National Defense Medical College, Saitama, Japan.
24
University College London, London, United Kingdom.
25
Immunology Department, Royal Free London, London, United Kingdom.
26
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.
27
Institute of Cellular Medicine, Newcastle University, Newcastle upon Tyne, United Kingdom.
28
University of Manchester, Royal Manchester Children's Hospital, Manchester, United Kingdom.
29
Pediatric Immunology and Infectious Disease, UMC Utrecht, Utrecht, The Netherlands.
30
Division of Allergy and Immunology, Children's Hospital of Philadelphia, Philadelphia, Pa.
31
Center of Immunodeficiency, Molecular Immunology Unit, Institute of Child Health, London, United Kingdom.
32
Center for Chronic Immunodeficiency (CCI), University Medical Center Freiburg and the University of Freiburg, Freiburg, Germany. Electronic address: klaus.warnatz@uniklinik-freiburg.de.
33
Center for Chronic Immunodeficiency (CCI), University Medical Center Freiburg and the University of Freiburg, Freiburg, Germany. Electronic address: marta.rizzi@uniklinik-freiburg.de.

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.

KEYWORDS:

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

PMID:
25595268
DOI:
10.1016/j.jaci.2014.11.029
[Indexed for MEDLINE]

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