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

Publication ,  Journal Article
Ruggeri, A; Eapen, M; Scaravadou, A; Cairo, MS; Bhatia, M; Kurtzberg, J; Wingard, JR; Fasth, A; Lo Nigro, L; Ayas, M; Purtill, D; Boudjedir, K ...
Published in: Biol Blood Marrow Transplant
September 2011

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.

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Published In

Biol Blood Marrow Transplant

DOI

EISSN

1523-6536

Publication Date

September 2011

Volume

17

Issue

9

Start / End Page

1375 / 1382

Location

United States

Related Subject Headings

  • Treatment Outcome
  • Transplantation Conditioning
  • Transplantation Chimera
  • Thalassemia
  • Survival Analysis
  • Registries
  • Neutrophils
  • Male
  • Immunology
  • Humans
 

Citation

APA
Chicago
ICMJE
MLA
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Ruggeri, A., Eapen, M., Scaravadou, A., Cairo, M. S., Bhatia, M., Kurtzberg, J., … New York Blood Center, . (2011). Umbilical cord blood transplantation for children with thalassemia and sickle cell disease. Biol Blood Marrow Transplant, 17(9), 1375–1382. https://doi.org/10.1016/j.bbmt.2011.01.012
Ruggeri, Annalisa, Mary Eapen, Andromachi Scaravadou, Mitchell S. Cairo, Monica Bhatia, Joanne Kurtzberg, John R. Wingard, et al. “Umbilical cord blood transplantation for children with thalassemia and sickle cell disease.Biol Blood Marrow Transplant 17, no. 9 (September 2011): 1375–82. https://doi.org/10.1016/j.bbmt.2011.01.012.
Ruggeri A, Eapen M, Scaravadou A, Cairo MS, Bhatia M, Kurtzberg J, et al. Umbilical cord blood transplantation for children with thalassemia and sickle cell disease. Biol Blood Marrow Transplant. 2011 Sep;17(9):1375–82.
Ruggeri, Annalisa, et al. “Umbilical cord blood transplantation for children with thalassemia and sickle cell disease.Biol Blood Marrow Transplant, vol. 17, no. 9, Sept. 2011, pp. 1375–82. Pubmed, doi:10.1016/j.bbmt.2011.01.012.
Ruggeri A, Eapen M, Scaravadou A, Cairo MS, Bhatia M, Kurtzberg J, Wingard JR, Fasth A, Lo Nigro L, Ayas M, Purtill D, Boudjedir K, Chaves W, Walters MC, Wagner J, Gluckman E, Rocha V, Eurocord Registry, Center for International Blood and Marrow Transplant Research, New York Blood Center. Umbilical cord blood transplantation for children with thalassemia and sickle cell disease. Biol Blood Marrow Transplant. 2011 Sep;17(9):1375–1382.
Journal cover image

Published In

Biol Blood Marrow Transplant

DOI

EISSN

1523-6536

Publication Date

September 2011

Volume

17

Issue

9

Start / End Page

1375 / 1382

Location

United States

Related Subject Headings

  • Treatment Outcome
  • Transplantation Conditioning
  • Transplantation Chimera
  • Thalassemia
  • Survival Analysis
  • Registries
  • Neutrophils
  • Male
  • Immunology
  • Humans