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] 

Outcomes 

Data in this section have been prepared by the CIBMTR® (Center for International Blood and Marrow Transplant Research®). The CIBMTR is a research collaboration between the National Marrow Donor Program® (NMDP)/Be The Match® and the Medical College of Wisconsin. 

Figure 1. Pediatric Survival after Allogeneic HCT for Thalassemia 

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HCT Consultation Timing Guidelines  

The National Marrow Donor Program® (NMDP)/Be The Match® and the American Society for Transplantation and Cellular Therapy (ASTCT) have jointly developed guidelines for transplant consultation and referral timing based on disease characteristics. [5] The National Comprehensive Cancer Network Clinical Practice Guidelines (NCCN Guidelines®) were consulted in developing these guidelines and are a valuable tool in determining risk stratification. [6] 

Our 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 

Transfusion-Dependent Thalassemias 

• At diagnosis 

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View complete HCT Consultation Timing Guidelines

Clinical Trials Search and Support  

The NMDP/Be The Match offers the Be The Match® Jason Carter Clinical Trials Search and Support (CTSS) program, which can provide clinical trial navigation to your patients. The CTSS Program was created to help people with blood cancers or blood disorders and their families find and join clinical trials.

For more information, visit Clinical Trials Search and Support.  

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/Be The Match and ASTCT Recommended Timing for Transplant Consultation. Download (PDF)
6. National Comprehensive Cancer Network. NCCN Guidelines. 2023. Access