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.

• Authors: Mary Eapen, Kwang Woo Ahn, Paul J Orchard, Morton J Cowan, Stella M Davies, Anders Fasth, Anna Hassebroek, Mouhab Ayas, Carmem Bonfim, Tracey A O'Brien, Thomas G Gross, Mitchell Horwitz, Edwin Horwitz, Neena Kapoor, Joanne Kurtzberg, Navneet Majhail, Olle Ringden, Paul Szabolcs, Paul Veys, K Scott Baker
• Journal: Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation (Biol Blood Marrow Transplant) Vol. 18 Issue 9 Pg. 1438-45 (Sep 2012) ISSN: 1523-6536 [Electronic] United States
• PMID: 22430083 (Publication Type: Journal Article, Multicenter Study, Research Support, N.I.H., Extramural, Research Support, U.S. Gov't, Non-P.H.S., Research Support, U.S. Gov't, P.H.S.)
• Copyright: Copyright © 2012 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.

Topics

• Adolescent
• Child
• Child, Preschool
• Chronic Disease
• Europe
• Female
• Graft vs Host Disease (prevention & control)
• Hematopoietic Stem Cell Transplantation
• Humans
• Immunologic Deficiency Syndromes (immunology, mortality, therapy)
• Infant
• International Cooperation
• Male
• Metabolism, Inborn Errors (immunology, mortality, therapy)
• Severe Combined Immunodeficiency (immunology, mortality, therapy)
• Survival Analysis
• Time Factors
• Transplantation Chimera
• Transplantation Conditioning
• Transplantation, Homologous
• United States
• Unrelated Donors