Umbilical cord blood transplantation for children with thalassemia and sickle cell disease.

Ruggeri, Annalisa

Eapen, Mary

Scaravadou, Andromachi

Cairo, Mitchell S

Bhatia, Monica

Kurtzberg, Joanne

Wingard, John R

Fasth, Anders

Lo Nigro, Luca

Ayas, Mouhab

Purtill, Duncan

Boudjedir, Karim

Chaves, Wagnara

Walters, Mark C

Wagner, John

Gluckman, Eliane

Rocha, Vanderson

Eurocord Registry

Center for International Blood and Marrow Transplant Research

New York Blood Center

2022-03-23T20:00:54Z

2022-03-23T20:00:54Z

2011-09

2022-03-23T20:00:54Z

We examined the efficacy of unrelated cord blood (CB) transplantation in children with thalassemia (n = 35) and sickle cell disease (n = 16), using data reported to 3 registries. Donor-recipient pairs were matched at HLA-A and -B (antigen level) and DRB1 (allele level) in 7 or HLA mismatched at 1 (n = 18), 2 (n = 25), or 3 loci (n = 1). Transplant conditioning was myeloablative (n = 39) or reduced intensity (n = 12). Neutrophil recovery with donor chimerism was documented in 24 patients; 11 patients developed grade II-IV acute graft-versus-host disease (aGVHD) and 10 patients, chronic GVHD (cGVHD). Overall survival (OS) and disease-free survival (DFS) were 62% and 21% for thalassemia and 94% and 50% for sickle cell disease (SCD), respectively. In multivariate analysis, engraftment rate (hazard ratio [HR] 2.2, P = .05) and DFS (HR 0.4, P = .01) were higher with cell dose >5 × 10(7)/kg. The 2-year probability of DFS was 45% in patients who received grafts with cell dose >5 × 10(7)/kg and 13% with lower cell dose. Primary graft failure was the predominant cause of treatment failure occurring in 20 patients with thalassemia and 7 patients with SCD. Primary graft failure was fatal in 5 patients with thalassemia. These results suggest that only CB units containing an expected infused cell dose >5 × 10(7)/kg should be considered for transplantation for hemoglobinopathy.

S1083-8791(11)00034-6

1083-8791

1523-6536

https://hdl.handle.net/10161/24672

eng

Elsevier BV

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

10.1016/j.bbmt.2011.01.012

Eurocord Registry

Center for International Blood and Marrow Transplant Research

New York Blood Center

Neutrophils

Transplantation Chimera

Humans

Anemia, Sickle Cell

Thalassemia

Graft vs Host Disease

Histocompatibility Testing

Treatment Outcome

Transplantation Conditioning

Cord Blood Stem Cell Transplantation

Cell Count

Registries

Survival Analysis

Graft Survival

Child

Female

Male

Umbilical cord blood transplantation for children with thalassemia and sickle cell disease.

Journal article

Kurtzberg, Joanne|0000-0002-3370-0703

1375

1382

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

17