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Permanent URL to this publication: http://dx.doi.org/10.5167/uzh-41625

Gennery, A R; Slatter, M A; Grandin, L; Taupin, P; Cant, A J; Veys, P; Amrolia, P J; Gaspar, H B; Davies, E G; Friedrich, W; Hoenig, M; Notarangelo, L D; Mazzolari, E; Porta, F; Bredius, R G M; Lankester, A C; Wulffraat, N M; Seger, R; Güngör, T; Fasth, A; Sedlacek, P; Neven, B; Blanche, S; Fischer, A; Cavazzana-Calvo, M; Landais, P (2010). Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: entering a new century, do we do better? Journal of Allergy and Clinical Immunology, 126(3):602-610.e11.

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Abstract

BACKGROUND: Hematopoietic stem cell transplantation remains the only treatment for most patients with severe combined immunodeficiencies (SCIDs) or other primary immunodeficiencies (non-SCID PIDs).

OBJECTIVE: To analyze the long-term outcome of patients with SCID and non-SCID PID from European centers treated between 1968 and 2005.

METHODS: The product-limit method estimated cumulative survival; the log-rank test compared survival between groups. A Cox proportional-hazard model evaluated the impact of independent predictors on patient survival.

RESULTS: In patients with SCID, survival with genoidentical donors (n = 25) from 2000 to 2005 was 90%. Survival using a mismatched relative (n = 96) has improved (66%), similar to that using an unrelated donor (n = 46; 69%; P = .005). Transplantation after year 1995, a younger age, B(+) phenotype, genoidentical and phenoidentical donors, absence of respiratory impairment, or viral infection before transplantation were associated with better prognosis on multivariate analysis. For non-SCID PID, in contrast with patients with SCID, we confirm that, in the 2000 to 2005 period, using an unrelated donor (n = 124) gave a 3-year survival rate similar to a genoidentical donor (n = 73), 79% for both. Survival was 76% in phenoidentical transplants (n = 23) and worse in mismatched related donor transplants (n = 47; 46%; P = .016).

CONCLUSION: This is the largest cohort study of such patients with the longest follow-up. Specific issues arise for different patient groups. Patients with B-SCID have worse survival than other patients with SCID, despite improvements in each group. For non-SCID PID, survival is worse than SCID, although more conditions are now treated. Individual disease categories now need to be analyzed so that disease-specific prognosis may be better understood and the best treatments planned.
Copyright (c) 2010 American Academy of Allergy, Asthma & Immunology. Published by Mosby, Inc. All rights reserved.

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Additional indexing

Item Type:Journal Article, refereed, original work
Communities & Collections:04 Faculty of Medicine > University Children's Hospital Zurich > Medical Clinic
DDC:610 Medicine & health
Language:English
Date:2010
Deposited On:22 Jan 2011 08:24
Last Modified:27 Nov 2013 22:01
Publisher:Elsevier
ISSN:0091-6749
Publisher DOI:10.1016/j.jaci.2010.06.015
PubMed ID:20673987

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