Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease.
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.
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; Eurocord Transplant Group,
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