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Long-term survival and late deaths after hematopoietic cell transplantation for primary immunodeficiency diseases and inborn errors of metabolism.

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Date
2012-09
Authors
Eapen, Mary
Ahn, Kwang Woo
Orchard, Paul J
Cowan, Morton J
Davies, Stella M
Fasth, Anders
Hassebroek, Anna
Ayas, Mouhab
Bonfim, Carmem
O'Brien, Tracey A
Gross, Thomas G
Horwitz, Mitchell
Horwitz, Edwin
Kapoor, Neena
Kurtzberg, Joanne
Majhail, Navneet
Ringden, Olle
Szabolcs, Paul
Veys, Paul
Baker, K Scott
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Abstract
It is uncertain whether late mortality rates after hematopoietic cell transplantation for severe combined immunodeficiency (SCID), non-SCID primary immunodeficiency diseases (non-SCID PIDD), and inborn errors of metabolism (IEM) return to rates observed in the general population, matched for age, sex, and nationality. We studied patients with SCID (n = 201), non-SCID PIDD (n = 405), and IEM (n = 348) who survived for at least 2 years after transplantation with normal T cell function (SCID) or >95% donor chimerism (non-SCID PIDD and IEM). Importantly, mortality rate was significantly higher in these patients compared with the general population for several years after transplantation. The rate decreased toward the normal rate in patients with SCID and non-SCID PIDD beyond 6 years after transplantation, but not in patients with IEM. Active chronic graft-versus-host disease at 2 years was associated with increased risk of late mortality for all diseases (hazard ratio [HR], 1.87; P = .05). In addition, late mortality was higher in patients with non-SCID PIDD who received T cell-depleted grafts (HR 4.16; P = .007) and in patients with IEM who received unrelated donor grafts (HR, 2.72; P = .03) or mismatched related donor grafts (HR, 3.76; P = .01). The finding of higher mortality rates in these long-term survivors for many years after transplantation confirms the need for long-term surveillance.
Type
Journal article
Subject
Transplantation Chimera
Humans
Metabolism, Inborn Errors
Severe Combined Immunodeficiency
Graft vs Host Disease
Immunologic Deficiency Syndromes
Chronic Disease
Transplantation Conditioning
Hematopoietic Stem Cell Transplantation
Transplantation, Homologous
Survival Analysis
Time Factors
International Cooperation
Adolescent
Child
Child, Preschool
Infant
United States
Europe
Female
Male
Unrelated Donors
Permalink
https://hdl.handle.net/10161/24685
Published Version (Please cite this version)
10.1016/j.bbmt.2012.03.003
Publication Info
Eapen, Mary; Ahn, Kwang Woo; Orchard, Paul J; Cowan, Morton J; Davies, Stella M; Fasth, Anders; ... Baker, K Scott (2012). Long-term survival and late deaths after hematopoietic cell transplantation for primary immunodeficiency diseases and inborn errors of metabolism. Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 18(9). pp. 1438-1445. 10.1016/j.bbmt.2012.03.003. Retrieved from https://hdl.handle.net/10161/24685.
This is constructed from limited available data and may be imprecise. To cite this article, please review & use the official citation provided by the journal.
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Scholars@Duke

Horwitz

Mitchell Eric Horwitz

Professor of Medicine
Allogeneic stem cell transplantation using umbilical cord blood grafts; Allogenic stem cell transplantation for Sickle Cell Disease; Prevention of acute graft versus host disease through donor stem cell graft manipulation; Improving immune recovery following alternative donor stem cell transplantation using donor graft manipulation or third party thymus transplantation.
Kurtzberg

Joanne Kurtzberg

Jerome S. Harris Distinguished Professor of Pediatrics
Dr. Kurtzberg conducts both clinical and laboratory-based translational research efforts, all involving various aspects of normal and malignant hematopoiesis. In the laboratory, her early work focused on studies determining the mechanisms that regulate the choice between the various pathways of differentiation available to the pluripotent hematopoietic stem cell. Her laboratory established a CD7+ cell line, DU.528, capable of multilineage differentiation as well as self-renewal, and subse
Alphabetical list of authors with Scholars@Duke profiles.
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