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Long-term survival and late deaths after hematopoietic cell transplantation for primary immunodeficiency diseases and inborn errors of metabolism.
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 articleSubject
Transplantation ChimeraHumans
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
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https://hdl.handle.net/10161/24685Published Version (Please cite this version)
10.1016/j.bbmt.2012.03.003Publication 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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Show full item recordScholars@Duke
Mitchell Eric Horwitz
Professor of Medicine
Allogeneic stem cell transplantation with a focus on the use of umbilical cord blood
grafts; Allogenic stem cell transplantation for Sickle Cell Disease; Prevention of
acute and chronic graft versus host disease; Improving immune recovery following alternative
donor stem cell transplantation using donor graft manipulation.
Joanne Kurtzberg
Jerome S. Harris Distinguished Professor of Pediatrics
Dr. Kurtzberg is an internationally renowned expert in pediatric hematology/oncology,
pediatric blood and marrow transplantation, umbilical cord blood banking and transplantation,
and novel applications of cord blood and birthing tissues in the emerging fields of
cellular therapies and regenerative medicine. Dr. Kurtzberg serves as the Director
of the Marcus Center for Cellular Cures (MC3), Director of the Pediatric Transplant
and Cellular Therapy Program, Director of the Carolina
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