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

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

2022-03-23T20:25:00Z

2022-03-23T20:25:00Z

2012-09

2022-03-23T20:25:00Z

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.

S1083-8791(12)00113-9

1083-8791

1523-6536

https://hdl.handle.net/10161/24685

eng

Elsevier BV

Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation

10.1016/j.bbmt.2012.03.003

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

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

Journal article

Horwitz, Mitchell|0000-0001-9863-8464

Kurtzberg, Joanne|0000-0002-3370-0703

1438

1445

9

Duke

School of Medicine

Clinical Science Departments

Institutes and Centers

Pathology

Pediatrics

Duke Cancer Institute

Institutes and Provost's Academic Units

Initiatives

Duke Innovation & Entrepreneurship

Pediatrics, Transplant and Cellular Therapy

Published

18