Outcome of patients with hemoglobinopathies given either cord blood or bone marrow transplantation from an HLA-identical sibling

Franco Locatelli, Nabil Kabbara, Annalisa Ruggeri, Ardeshir Ghavamzadeh, Irene Roberts, Chi Kong Li, Françoise Bernaudin, Christiane Vermylen, Jean Hugues Dalle, Jerry Stein, Robert Wynn, Catherine Cordonnier, Fernando Pinto, Emanuele Angelucci, Gérard Socié, Eliane Gluckman, Mark C. Walters, Vanderson Rocha

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Abstract

We analyzed the outcomes of 485 patients with thalassemia major (TM) or sickle cell disease (SCD) receiving HLA-identical sibling cord blood transplantation (CBT, n = 96) or bone marrow transplantation (BMT, n = 389). Compared with patients given BMT, CBT recipients were significantly younger (median age 6 vs 8 years, P = .02), and were treated more recently (median year 2001 vs 1999, P <.01). A higher proportion of patients with TM belonging to classes II-III of the Pesaro classification received BMT (44%) compared with CBT (39%, P <.01). In comparison with patients receiving BMT(n = 259, TM; n = 130, SCD), those given CBT (n = 66, TM; n = 30, SCD) had slower neutrophil recovery, less acute graft-versus-host disease (GVHD) and none had extensive chronic GVHD. With a median follow-up of 70 months, the 6-year overall survival was 95% and 97% after BMT and CBT, respectively (P = .92). The 6-year disease-free survival (DFS) was 86% and 80% in TM patients after BMT and CBT, respectively, whereas DFS in SCD patients was 92% and 90%, respectively. The cell dose infused did not influence outcome of patients given CBT. In multivariate analysis, DFS did not differ between CBT and BMT recipients. Patients with TMor SCD have excellent outcomes after both HLA-identical sibling CBT and BMT.

Original languageEnglish
Pages (from-to)1072-1078
Number of pages7
JournalBlood
Volume122
Issue number6
DOIs
Publication statusPublished - Aug 8 2013

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Hemoglobinopathies
Bone Marrow Transplantation
Fetal Blood
Siblings
Bone
Blood
beta-Thalassemia
Sickle Cell Anemia
Disease-Free Survival
Graft vs Host Disease
Grafts
Neutrophils
Multivariate Analysis
Transplantation
Survival
Recovery

ASJC Scopus subject areas

  • Hematology
  • Biochemistry
  • Cell Biology
  • Immunology

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Outcome of patients with hemoglobinopathies given either cord blood or bone marrow transplantation from an HLA-identical sibling. / Locatelli, Franco; Kabbara, Nabil; Ruggeri, Annalisa; Ghavamzadeh, Ardeshir; Roberts, Irene; Li, Chi Kong; Bernaudin, Françoise; Vermylen, Christiane; Dalle, Jean Hugues; Stein, Jerry; Wynn, Robert; Cordonnier, Catherine; Pinto, Fernando; Angelucci, Emanuele; Socié, Gérard; Gluckman, Eliane; Walters, Mark C.; Rocha, Vanderson.

In: Blood, Vol. 122, No. 6, 08.08.2013, p. 1072-1078.

Research output: Contribution to journalArticle

Locatelli, F, Kabbara, N, Ruggeri, A, Ghavamzadeh, A, Roberts, I, Li, CK, Bernaudin, F, Vermylen, C, Dalle, JH, Stein, J, Wynn, R, Cordonnier, C, Pinto, F, Angelucci, E, Socié, G, Gluckman, E, Walters, MC & Rocha, V 2013, 'Outcome of patients with hemoglobinopathies given either cord blood or bone marrow transplantation from an HLA-identical sibling', Blood, vol. 122, no. 6, pp. 1072-1078. https://doi.org/10.1182/blood-2013-03-489112
Locatelli, Franco ; Kabbara, Nabil ; Ruggeri, Annalisa ; Ghavamzadeh, Ardeshir ; Roberts, Irene ; Li, Chi Kong ; Bernaudin, Françoise ; Vermylen, Christiane ; Dalle, Jean Hugues ; Stein, Jerry ; Wynn, Robert ; Cordonnier, Catherine ; Pinto, Fernando ; Angelucci, Emanuele ; Socié, Gérard ; Gluckman, Eliane ; Walters, Mark C. ; Rocha, Vanderson. / Outcome of patients with hemoglobinopathies given either cord blood or bone marrow transplantation from an HLA-identical sibling. In: Blood. 2013 ; Vol. 122, No. 6. pp. 1072-1078.
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abstract = "We analyzed the outcomes of 485 patients with thalassemia major (TM) or sickle cell disease (SCD) receiving HLA-identical sibling cord blood transplantation (CBT, n = 96) or bone marrow transplantation (BMT, n = 389). Compared with patients given BMT, CBT recipients were significantly younger (median age 6 vs 8 years, P = .02), and were treated more recently (median year 2001 vs 1999, P <.01). A higher proportion of patients with TM belonging to classes II-III of the Pesaro classification received BMT (44{\%}) compared with CBT (39{\%}, P <.01). In comparison with patients receiving BMT(n = 259, TM; n = 130, SCD), those given CBT (n = 66, TM; n = 30, SCD) had slower neutrophil recovery, less acute graft-versus-host disease (GVHD) and none had extensive chronic GVHD. With a median follow-up of 70 months, the 6-year overall survival was 95{\%} and 97{\%} after BMT and CBT, respectively (P = .92). The 6-year disease-free survival (DFS) was 86{\%} and 80{\%} in TM patients after BMT and CBT, respectively, whereas DFS in SCD patients was 92{\%} and 90{\%}, respectively. The cell dose infused did not influence outcome of patients given CBT. In multivariate analysis, DFS did not differ between CBT and BMT recipients. Patients with TMor SCD have excellent outcomes after both HLA-identical sibling CBT and BMT.",
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AU - Locatelli, Franco

AU - Kabbara, Nabil

AU - Ruggeri, Annalisa

AU - Ghavamzadeh, Ardeshir

AU - Roberts, Irene

AU - Li, Chi Kong

AU - Bernaudin, Françoise

AU - Vermylen, Christiane

AU - Dalle, Jean Hugues

AU - Stein, Jerry

AU - Wynn, Robert

AU - Cordonnier, Catherine

AU - Pinto, Fernando

AU - Angelucci, Emanuele

AU - Socié, Gérard

AU - Gluckman, Eliane

AU - Walters, Mark C.

AU - Rocha, Vanderson

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N2 - We analyzed the outcomes of 485 patients with thalassemia major (TM) or sickle cell disease (SCD) receiving HLA-identical sibling cord blood transplantation (CBT, n = 96) or bone marrow transplantation (BMT, n = 389). Compared with patients given BMT, CBT recipients were significantly younger (median age 6 vs 8 years, P = .02), and were treated more recently (median year 2001 vs 1999, P <.01). A higher proportion of patients with TM belonging to classes II-III of the Pesaro classification received BMT (44%) compared with CBT (39%, P <.01). In comparison with patients receiving BMT(n = 259, TM; n = 130, SCD), those given CBT (n = 66, TM; n = 30, SCD) had slower neutrophil recovery, less acute graft-versus-host disease (GVHD) and none had extensive chronic GVHD. With a median follow-up of 70 months, the 6-year overall survival was 95% and 97% after BMT and CBT, respectively (P = .92). The 6-year disease-free survival (DFS) was 86% and 80% in TM patients after BMT and CBT, respectively, whereas DFS in SCD patients was 92% and 90%, respectively. The cell dose infused did not influence outcome of patients given CBT. In multivariate analysis, DFS did not differ between CBT and BMT recipients. Patients with TMor SCD have excellent outcomes after both HLA-identical sibling CBT and BMT.

AB - We analyzed the outcomes of 485 patients with thalassemia major (TM) or sickle cell disease (SCD) receiving HLA-identical sibling cord blood transplantation (CBT, n = 96) or bone marrow transplantation (BMT, n = 389). Compared with patients given BMT, CBT recipients were significantly younger (median age 6 vs 8 years, P = .02), and were treated more recently (median year 2001 vs 1999, P <.01). A higher proportion of patients with TM belonging to classes II-III of the Pesaro classification received BMT (44%) compared with CBT (39%, P <.01). In comparison with patients receiving BMT(n = 259, TM; n = 130, SCD), those given CBT (n = 66, TM; n = 30, SCD) had slower neutrophil recovery, less acute graft-versus-host disease (GVHD) and none had extensive chronic GVHD. With a median follow-up of 70 months, the 6-year overall survival was 95% and 97% after BMT and CBT, respectively (P = .92). The 6-year disease-free survival (DFS) was 86% and 80% in TM patients after BMT and CBT, respectively, whereas DFS in SCD patients was 92% and 90%, respectively. The cell dose infused did not influence outcome of patients given CBT. In multivariate analysis, DFS did not differ between CBT and BMT recipients. Patients with TMor SCD have excellent outcomes after both HLA-identical sibling CBT and BMT.

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