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

Annalisa Ruggeri; Mary Eapen; Andromachi Scaravadou; Mitchell S. Cairo; Monica Bhatia; Joanne Kurtzberg; John R. Wingard; Anders Fasth; Luca Lo Nigro; Mouhab Ayas; Duncan Purtill; Karim Boudjedir; Wagnara Chaves; Mark C. Walters; John Wagner; Eliane Gluckman; Vanderson Rocha

doi:10.1016/j.bbmt.2011.01.012

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

Annalisa Ruggeri, Mary Eapen, Andromachi Scaravadou, Mitchell S. Cairo, Monica Bhatia, Joanne Kurtzberg, John R. Wingard, Anders Fasth, Luca Lo Nigro, Mouhab Ayas, Duncan Purtill, Karim Boudjedir, Wagnara Chaves, Mark C. Walters, John Wagner, Eliane Gluckman, Vanderson Rocha

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Abstract

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.

Original language	English (US)
Pages (from-to)	1375-1382
Number of pages	8
Journal	Biology of Blood and Marrow Transplantation
Volume	17
Issue number	9
DOIs	https://doi.org/10.1016/j.bbmt.2011.01.012
State	Published - Sep 2011

Bibliographical note

Funding Information:
This work was funded in part by the National Cancer Institute, National Heart Lung and Blood Institute and the National Institute of Allergy and Infectious Diseases ( U24-CA76518 ).

Keywords

Cord blood transplantation
Graft failure
Sickle cell disease
Thalassemia

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10.1016/j.bbmt.2011.01.012

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Ruggeri, A., Eapen, M., Scaravadou, A., Cairo, M. S., Bhatia, M., Kurtzberg, J., Wingard, J. R., Fasth, A., Lo Nigro, L., Ayas, M., Purtill, D., Boudjedir, K., Chaves, W., Walters, M. C., Wagner, J., Gluckman, E., & Rocha, V. (2011). Umbilical cord blood transplantation for children with thalassemia and sickle cell disease. Biology of Blood and Marrow Transplantation, 17(9), 1375-1382. https://doi.org/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 2011, 'Umbilical cord blood transplantation for children with thalassemia and sickle cell disease', Biology of Blood and Marrow Transplantation, vol. 17, no. 9, pp. 1375-1382. https://doi.org/10.1016/j.bbmt.2011.01.012

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abstract = "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.",

keywords = "Cord blood transplantation, Graft failure, Sickle cell disease, Thalassemia",

author = "Annalisa Ruggeri and Mary Eapen and Andromachi Scaravadou and Cairo, {Mitchell S.} and Monica Bhatia and Joanne Kurtzberg and Wingard, {John R.} and Anders Fasth and {Lo Nigro}, Luca and Mouhab Ayas and Duncan Purtill and Karim Boudjedir and Wagnara Chaves and Walters, {Mark C.} and John Wagner and Eliane Gluckman and Vanderson Rocha",

note = "Funding Information: This work was funded in part by the National Cancer Institute, National Heart Lung and Blood Institute and the National Institute of Allergy and Infectious Diseases ( U24-CA76518 ). ",

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AU - Ruggeri, Annalisa

AU - Eapen, Mary

AU - Scaravadou, Andromachi

AU - Cairo, Mitchell S.

AU - Bhatia, Monica

AU - Kurtzberg, Joanne

AU - Wingard, John R.

AU - Fasth, Anders

AU - Lo Nigro, Luca

AU - Ayas, Mouhab

AU - Purtill, Duncan

AU - Boudjedir, Karim

AU - Chaves, Wagnara

AU - Walters, Mark C.

AU - Wagner, John

AU - Gluckman, Eliane

AU - Rocha, Vanderson

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PY - 2011/9

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N2 - 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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KW - Sickle cell disease

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

Abstract

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