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 RajaIrene Roberts, Andrew M. Will, Isaac Yaniv, Christiane Vermylen, Nunzia Tannoia, Federico Garnier, Irina Ionescu, Mark C. Walters, Bertram H. Lubin, Eliane Gluckman

Risultato della ricerca: Contributo in rivistaArticolo in rivista


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 x 10(7)/kg (range, 1.2-10 x 10(7)/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.
Lingua originaleEnglish
pagine (da-a)2137-2143
Numero di pagine7
Stato di pubblicazionePubblicato - 2003


  • Anemia, Sickle Cell / therapy


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