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. © 2011 American Society for Blood and Marrow Transplantation.
Ruggeri, A., Eapen, M., Scaravadou, A., Cairo, M. S., Bhatia, M., Kurtzberg, J., … Rocha, V. (2011). Umbilical cord blood transplantation for children with thalassemia and sickle cell disease. Biology of Blood and Marrow Transplantation, 17(9), 1375–1382. https://doi.org/10.1016/j.bbmt.2011.01.012