Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease

Franco Locatelli, Vanderson Rocha, William Reed, Françoise Bernaudin, Mehmet Ertem, Stelios Grafakos, Benedicte Brichard, Xiaxin Li, Arnon Nagler, Giovanna Giorgiani, Paul R. Haut, Joel A. Brochstein, Diane J. Nugent, Julie Blatt, Paul Woodard, Joanne Kurtzberg, Charles M. Rubin, Roberto Miniero, Patrick Lutz, Thirumalairaj Raja & 10 others Irene Roberts, Andrew M. Will, Isaac Yaniv, Christiane Vermylen, Nunzia Tannoia, Federico Garnier, Irina Ionescu, Mark C. Walters, Bertram H. Lubin, Eliane Gluckman

Research output: Contribution to journalArticle

300 Citations (Scopus)

Abstract

Allogeneic bone marrow transplantation (BMT) from HLA-identical siblings is an accepted treatment for both thalassemia and sickle cell disease (SCD). However, it is associated with decided risk of both transplant-related mortality (TRM) and chronic graft-versus-host disease (GVHD). We analyzed 44 patients (median age, 5 years; range, 1-20 years) given an allogeneic related cord blood transplant for either thalassemia (n = 33) or SCD (n = 11). Thirty children were given cyclosporin A (CsA) alone as GVHD prophylaxis, 10 received CsA and methotrexate (MTX), and 4 patients received other combinations of immunosuppressive drugs. The median number of nucleated cells infused was 4.0 × 107/kg (range, 1.2-10 × 107/kg). No patient died and 36 of 44 children remain free of disease, with a median follow-up of 24 months (range, 4-76 months). Only one patient with SCD did not have sustained donor engraftment as compared with 7 of the 33 patients with thalassemia. Three of these 8 patients had sustained donor engraftment after BMT from the same donor. Four patients experienced grade 2 acute GVHD; only 2 of the 36 patients at risk developed limited chronic GVHD. The 2-year probability of event-free survival is 79% and 90% for patients with thalassemia and SCD, respectively. Use of MTX for GVHD prophylaxis was associated with a greater risk of treatment failure. Related CBT for hemoglobinopathies offers a good probability of success and is associated with a low risk of GVHD. Optimization of transplantation strategies could further improve these results.

Original languageEnglish
Pages (from-to)2137-2143
Number of pages7
JournalBlood
Volume101
Issue number6
DOIs
Publication statusPublished - Mar 15 2003

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Thalassemia
Sickle Cell Anemia
Fetal Blood
Blood
Transplantation
Graft vs Host Disease
Grafts
Tissue Donors
Transplants
Bone Marrow Transplantation
Methotrexate
Cyclosporine
Bone
Hemoglobinopathies
Transplantation (surgical)
Homologous Transplantation
Drug Combinations
Immunosuppressive Agents
Treatment Failure
Disease-Free Survival

ASJC Scopus subject areas

  • Hematology

Cite this

Locatelli, F., Rocha, V., Reed, W., Bernaudin, F., Ertem, M., Grafakos, S., ... Gluckman, E. (2003). Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease. Blood, 101(6), 2137-2143. https://doi.org/10.1182/blood-2002-07-2090

Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease. / Locatelli, Franco; Rocha, Vanderson; Reed, William; Bernaudin, Françoise; Ertem, Mehmet; Grafakos, Stelios; Brichard, Benedicte; Li, Xiaxin; Nagler, Arnon; Giorgiani, Giovanna; Haut, Paul R.; Brochstein, Joel A.; Nugent, Diane J.; Blatt, Julie; Woodard, Paul; Kurtzberg, Joanne; Rubin, Charles M.; Miniero, Roberto; Lutz, Patrick; Raja, Thirumalairaj; Roberts, Irene; Will, Andrew M.; Yaniv, Isaac; Vermylen, Christiane; Tannoia, Nunzia; Garnier, Federico; Ionescu, Irina; Walters, Mark C.; Lubin, Bertram H.; Gluckman, Eliane.

In: Blood, Vol. 101, No. 6, 15.03.2003, p. 2137-2143.

Research output: Contribution to journalArticle

Locatelli, F, Rocha, V, Reed, W, Bernaudin, F, Ertem, M, Grafakos, S, Brichard, B, Li, X, Nagler, A, Giorgiani, G, Haut, PR, Brochstein, JA, Nugent, DJ, Blatt, J, Woodard, P, Kurtzberg, J, Rubin, CM, Miniero, R, Lutz, P, Raja, T, Roberts, I, Will, AM, Yaniv, I, Vermylen, C, Tannoia, N, Garnier, F, Ionescu, I, Walters, MC, Lubin, BH & Gluckman, E 2003, 'Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease', Blood, vol. 101, no. 6, pp. 2137-2143. https://doi.org/10.1182/blood-2002-07-2090
Locatelli, Franco ; Rocha, Vanderson ; Reed, William ; Bernaudin, Françoise ; Ertem, Mehmet ; Grafakos, Stelios ; Brichard, Benedicte ; Li, Xiaxin ; Nagler, Arnon ; Giorgiani, Giovanna ; Haut, Paul R. ; Brochstein, Joel A. ; Nugent, Diane J. ; Blatt, Julie ; Woodard, Paul ; Kurtzberg, Joanne ; Rubin, Charles M. ; Miniero, Roberto ; Lutz, Patrick ; Raja, Thirumalairaj ; Roberts, Irene ; Will, Andrew M. ; Yaniv, Isaac ; Vermylen, Christiane ; Tannoia, Nunzia ; Garnier, Federico ; Ionescu, Irina ; Walters, Mark C. ; Lubin, Bertram H. ; Gluckman, Eliane. / Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease. In: Blood. 2003 ; Vol. 101, No. 6. pp. 2137-2143.
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abstract = "Allogeneic bone marrow transplantation (BMT) from HLA-identical siblings is an accepted treatment for both thalassemia and sickle cell disease (SCD). However, it is associated with decided risk of both transplant-related mortality (TRM) and chronic graft-versus-host disease (GVHD). We analyzed 44 patients (median age, 5 years; range, 1-20 years) given an allogeneic related cord blood transplant for either thalassemia (n = 33) or SCD (n = 11). Thirty children were given cyclosporin A (CsA) alone as GVHD prophylaxis, 10 received CsA and methotrexate (MTX), and 4 patients received other combinations of immunosuppressive drugs. The median number of nucleated cells infused was 4.0 × 107/kg (range, 1.2-10 × 107/kg). No patient died and 36 of 44 children remain free of disease, with a median follow-up of 24 months (range, 4-76 months). Only one patient with SCD did not have sustained donor engraftment as compared with 7 of the 33 patients with thalassemia. Three of these 8 patients had sustained donor engraftment after BMT from the same donor. Four patients experienced grade 2 acute GVHD; only 2 of the 36 patients at risk developed limited chronic GVHD. The 2-year probability of event-free survival is 79{\%} and 90{\%} for patients with thalassemia and SCD, respectively. Use of MTX for GVHD prophylaxis was associated with a greater risk of treatment failure. Related CBT for hemoglobinopathies offers a good probability of success and is associated with a low risk of GVHD. Optimization of transplantation strategies could further improve these results.",
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AU - Bernaudin, Françoise

AU - Ertem, Mehmet

AU - Grafakos, Stelios

AU - Brichard, Benedicte

AU - Li, Xiaxin

AU - Nagler, Arnon

AU - Giorgiani, Giovanna

AU - Haut, Paul R.

AU - Brochstein, Joel A.

AU - Nugent, Diane J.

AU - Blatt, Julie

AU - Woodard, Paul

AU - Kurtzberg, Joanne

AU - Rubin, Charles M.

AU - Miniero, Roberto

AU - Lutz, Patrick

AU - Raja, Thirumalairaj

AU - Roberts, Irene

AU - Will, Andrew M.

AU - Yaniv, Isaac

AU - Vermylen, Christiane

AU - Tannoia, Nunzia

AU - Garnier, Federico

AU - Ionescu, Irina

AU - Walters, Mark C.

AU - Lubin, Bertram H.

AU - Gluckman, Eliane

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N2 - Allogeneic bone marrow transplantation (BMT) from HLA-identical siblings is an accepted treatment for both thalassemia and sickle cell disease (SCD). However, it is associated with decided risk of both transplant-related mortality (TRM) and chronic graft-versus-host disease (GVHD). We analyzed 44 patients (median age, 5 years; range, 1-20 years) given an allogeneic related cord blood transplant for either thalassemia (n = 33) or SCD (n = 11). Thirty children were given cyclosporin A (CsA) alone as GVHD prophylaxis, 10 received CsA and methotrexate (MTX), and 4 patients received other combinations of immunosuppressive drugs. The median number of nucleated cells infused was 4.0 × 107/kg (range, 1.2-10 × 107/kg). No patient died and 36 of 44 children remain free of disease, with a median follow-up of 24 months (range, 4-76 months). Only one patient with SCD did not have sustained donor engraftment as compared with 7 of the 33 patients with thalassemia. Three of these 8 patients had sustained donor engraftment after BMT from the same donor. Four patients experienced grade 2 acute GVHD; only 2 of the 36 patients at risk developed limited chronic GVHD. The 2-year probability of event-free survival is 79% and 90% for patients with thalassemia and SCD, respectively. Use of MTX for GVHD prophylaxis was associated with a greater risk of treatment failure. Related CBT for hemoglobinopathies offers a good probability of success and is associated with a low risk of GVHD. Optimization of transplantation strategies could further improve these results.

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