Multicenter experience in hematopoietic stem cell transplantation for serious complications of common variable immunodeficiency
Küçük Resim Yok
Tarih
2015
Dergi Başlığı
Dergi ISSN
Cilt Başlığı
Yayıncı
Mosby-Elsevier
Erişim Hakkı
info:eu-repo/semantics/openAccess
Özet
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.
Açıklama
Anahtar Kelimeler
Common variable immunodeficiency, hypogammaglobulinemia, hematopoietic stem cell transplantation, immunologic reconstitution, immunoglobulin substitution/replacement, outcome, mortality, survival
Kaynak
Journal of Allergy and Clinical Immunology
WoS Q Değeri
Q1
Scopus Q Değeri
Q1
Cilt
135
Sayı
4
