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Transfusion-dependent thalassemias

Transplant advances and outcomes

Although advances in supportive care and drug therapies have significantly improved the prognosis in beta thalassemia major, hematopoietic cell transplantation (HCT) remains the only treatment with a potential to cure this hemoglobinopathy. [1-4]

Recommended timing for transplant consultation

  • At diagnosis

References

  1. Locatelli F, Kabbara N, Ruggeri A, et al. Outcome of patients with hemoglobinopathies given either cord blood or bone marrow transplantation from an HLA-identical sibling. Blood. 2013; 122(6): 1072-1078.  Access
  2. Bernardo ME, Piras E, Vacca A, et al. Allogeneic hematopoietic stem cell transplantation in thalassemia major: results of a reduced-toxicity conditioning regimen based on the use of treosulfan. Blood. 2012; 120(2): 473-476. Access
  3. Mehta PA, Faulkner LB. Hematopoietic cell transplantation for thalassemia: A global perspective. Biol Blood Marrow Transplant. 2013; 19(1 Suppl): S70-S73. Access
  4. Tolar J, Sodani P, Symons H. Alternative donor transplant of benign primary hematologic disorders. Bone Marrow Transplant. 2015; 50(5): 619-627.  Access
  5. NMDP and ASTCT Recommended Timing for Transplant Consultation. Download PDF     
  6. National Comprehensive Cancer Network. NCCN Guidelines. 2023. Access