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.

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Published Version (Please cite this version)

10.1016/j.bbmt.2012.03.003

Publication Info

Eapen, Mary, Kwang Woo Ahn, Paul J Orchard, Morton J Cowan, Stella M Davies, Anders Fasth, Anna Hassebroek, Mouhab Ayas, et al. (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.

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Scholars@Duke

Horwitz

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.


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