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

Blood. 2003 Mar 15;101(6):2137-43. doi: 10.1182/blood-2002-07-2090. Epub 2002 Nov 7.


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.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Acute Disease
  • Anemia, Sickle Cell / therapy*
  • Blood Platelets
  • Child
  • Child, Preschool
  • Chronic Disease
  • Cord Blood Stem Cell Transplantation*
  • Cyclosporine / therapeutic use
  • Disease-Free Survival
  • Female
  • Graft Survival
  • Graft vs Host Disease / epidemiology
  • Graft vs Host Disease / prevention & control
  • Humans
  • Immunosuppressive Agents / therapeutic use
  • Infant
  • Male
  • Methotrexate / therapeutic use
  • Neutrophils
  • Survival Rate
  • Thalassemia / therapy*
  • Transplantation, Homologous
  • Treatment Outcome


  • Immunosuppressive Agents
  • Cyclosporine
  • Methotrexate