Fanconi Anemia Outcomes

The survival graphs below illustrate outcomes of unrelated donor hematopoietic cell transplants (bone marrow, PBSC, or cord blood — BMT) facilitated by the National Marrow Donor Program (NMDP) for both adult and pediatric patients.

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 adult and pediatric marrow transplant patients 1998-2006. (NMDP data)

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Marrow transplants facilitated by the NMDP, 1998-2006
Severe Aplastic Anemia	# of Transplants	Kaplan-Meier 5-Year Survival*
Pediatric (<18) Patients	218	59% ± 7%
Adult (> 18) Patients	166	55% ± 8%

* 95% confidence interval

NMDP outcomes for pediatric patients with Fanconi anemia

Figure 2.
Fanconi anemia: Survival of pediatric non-myeloablative marrow transplant patients 1998-2006. (NMDP data)

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Pediatric non-myeloablative marrow transplants facilitated by the NMDP, 1998-2006
Disease	# of Transplants	Kaplan-Meier 5-Year Survival*
Fanconi anemia (< 18 years)	64	56% ± 13%

* 95% confidence interval

References

1. 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/bmt/protocol/0301_protocol/
   0301_protocol.html