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Umbilical Cord Bloo...
Umbilical Cord Blood Transplantation for Children with Thalassemia and Sickle Cell Disease.
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Ruggeri, Annalisa (author)
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Eapen, Mary (author)
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Scaravadou, Andromachi (author)
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Cairo, Mitchell S (author)
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Bhatia, Monica (author)
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Kurtzberg, Joanne (author)
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Wingard, John R (author)
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- Fasth, Anders, 1945 (author)
- Gothenburg University,Göteborgs universitet,Institutionen för kliniska vetenskaper, Avdelningen för pediatrik,Institute of Clinical Sciences, Department of Pediatrics
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Lo Nigro, Luca (author)
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Ayas, Mouhab (author)
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Purtill, Duncan (author)
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Boudjedir, Karim (author)
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Chaves, Wagnara (author)
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Walters, Mark C (author)
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Wagner, John (author)
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Gluckman, Eliane (author)
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Rocha, Vanderson (author)
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(creator_code:org_t)
- Elsevier BV, 2011
- 2011
- English.
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In: Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation. - : Elsevier BV. - 1523-6536.
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http://www.bbmt.org/...
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https://gup.ub.gu.se...
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https://doi.org/10.1...
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Abstract
Subject headings
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- 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.
Subject headings
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Pediatrik (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Pediatrics (hsv//eng)
Keyword
- hemoglobulinopathies
- hemotopoietic stem cell transplantation
Publication and Content Type
- ref (subject category)
- art (subject category)
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- By the author/editor
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Ruggeri, Annalis ...
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Eapen, Mary
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Scaravadou, Andr ...
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Cairo, Mitchell ...
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Bhatia, Monica
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Kurtzberg, Joann ...
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show more...
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Wingard, John R
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Fasth, Anders, 1 ...
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Lo Nigro, Luca
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Ayas, Mouhab
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Purtill, Duncan
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Boudjedir, Karim
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Chaves, Wagnara
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Walters, Mark C
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Wagner, John
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Gluckman, Eliane
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Rocha, Vanderson
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show less...
- About the subject
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- MEDICAL AND HEALTH SCIENCES
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MEDICAL AND HEAL ...
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and Clinical Medicin ...
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and Pediatrics
- Articles in the publication
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Biology of blood ...
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Biology of Blood ...
- By the university
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University of Gothenburg