The survival graphs below illustrate outcomes of unrelated donor transplants facilitated by the National Marrow Donor Program® (NMDP) for patients with severe aplastic anemia and Fanconi anemia.
NMDP outcomes for adult and pediatric patients with severe aplastic anemia (SAA)
The majority of adult (≥18 years of age) patients transplanted for SAA have failed prior immunosuppressive therapy. Optimal and safer conditioning regimens for unrelated donor hematopoietic cell transplantation for patients with severe aplastic anemia are being studied through the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) Protocol 0301. [1]
Figure 1.
Severe aplastic anemia: Survival of marrow transplant patients, by age (pediatric or adult), 2000-2009. (NMDP data)
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NMDP outcomes for pediatric patients with Fanconi anemia
Figure 2.
Fanconi anemia: Survival of pediatric non-myeloablative transplant patients, by cell source, 2000-2009. (NMDP data)
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References
- BMT CTN Protocol 0301. Fludarabine-based conditioning for allogeneic marrow transplantation from HLA-compatible unrelated donors in severe aplastic anemia. 2007.
https://web.emmes.com/study/bmt2/protocol/0301_protocol/
0301_protocol.html