Thalassemia
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]
Advances
Recent research provides new data for clinical decision-making in patients with thalassemia:
- Related donor HCT for sickle cell disease (SCD) or thalassemia major can yield overall survival of >95% at 6 years [1]
- A reduced-toxicity HCT conditioning regimen using treosulfan can yield a 5-year overall survival of 93% in thalassemia [2]
- Risk stratification based on liver size, liver fibrosis, and chelation history in patients with thalassemia correlates well with HCT outcomes [3]
Outcomes
Review outcomes for allogeneic transplantation in patients with thalassemia below. View additional slides showing demographic data and hematopoietic cell transplant trends.
Data in this section have been prepared by CIBMTR® (Center for International Blood and Marrow Transplant Research), our research program.
Figure 1: Thalassemia Survival, Unrelated HCT
Referral Timing Guidelines
These guidelines highlight disease categories that include patients at risk for disease progression and who should be referred for a consultation for hematopoietic cell transplantation. [5]
Transplant Consultation Guidelines: Hemoglobinopathies
- Sickle Cell Disease
- With aggressive course (stroke, end-organ complications, frequent pain crises)
- Transfusion-Dependent Thalassemias
- At diagnosis
View complete Referral Timing Guidelines
References
- 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
- 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
- Mehta PA, Faulkner LB. Hematopoietic cell transplantation for thalassemia: A global perspective. Biol Blood Marrow Transplant. 2013; 19(1 Suppl): S70-S73. Access
- Tolar J, Sodani P, Symons H. Alternative donor transplant of benign primary hematologic disorders. Bone Marrow Transplant. 2015; 50(5): 619-627. Access
- NMDP/Be The Match and ASBMT Recommended Timing for Transplant Consultation, 2018. Download (PDF)