Faculty Collaboration Database

Long-term survival and late deaths after hematopoietic cell transplantation for primary immunodeficiency diseases and inborn errors of metabolism. Biol Blood Marrow Transplant 2012 Sep;18(9):1438-45

Date

03/21/2012

Pubmed ID

22430083

Pubmed Central ID

PMC3390445

DOI

10.1016/j.bbmt.2012.03.003

Scopus ID

2-s2.0-84865199175 (requires institutional sign-in at Scopus site)   38 Citations

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.

Author List

Eapen M, Ahn KW, Orchard PJ, Cowan MJ, Davies SM, Fasth A, Hassebroek A, Ayas M, Bonfim C, O'Brien TA, Gross TG, Horwitz M, Horwitz E, Kapoor N, Kurtzberg J, Majhail N, Ringden O, Szabolcs P, Veys P, Baker KS

Authors

Kwang Woo Ahn PhD Professor in the Institute for Health and Equity department at Medical College of Wisconsin
Mary Eapen MBBS, DCh, MRCPI, MS Professor in the Medicine department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

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