67% With MDS, AML From Fanconi Anemia Alive at 5 Years Following Allo-HSCT

Two-thirds of patients who received allogeneic hematopoietic stem cell transplantation (allo-HSCT) to treat myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) resulting from Fanconi anemia (FA) were still alive at the 5-year mark, according to findings from a small retrospective study just published in Bone Marrow Transplantation.

Authors described allo-HSCT as “the only curative approach” for MDS or AML arising in patients with FA. “However,” they wrote, “[HSCT] approaches are inconsistent and limited data on outcomes exist.”

The rare disease FA is an inherited bone marrow disorder linked to birth defects that leads to failure of bone marrow, the spongy material inside the bones where stem cells develop. Children with FA experience the loss of hematopoietic stem cells and cytopenia, and it is common for MDS or AML to develop before they reach adolescence, according to the American Society of Hematology.

To understand the long-term outcomes of the only known treatment for MDS/AML in these patients, authors retrospectively studied 30 patients with FA and MDS/AML who received “first allogeneic [HSCT] with a T-cell depleted (TCD) graft” at Memorial Sloan Kettering Cancer Center, from January 1, 1999, through June 2020. As described in an abstract in Bone Marrow Transplantation, the patients received transplants on successive protocols that included cytoreduction as well graft-vs-host disease prophylaxis.

Authors noted that 50% of the patients were already 20 or older, meaning they were considered high-risk, and a majority of patients had mismatched grafts. Results were as follows:

• All but 2 patients (93%) saw durable hematopoietic engraftment.
• After a median follow-up of 8.7 years, 5-year overall survival was 66.8% and disease-free survival was 53.8%.
• No significant differences in survival were seen in high-risk patients, such as those 20 years or older, those diagnosed with AML, or those with an alternative donor graft. Also, no significant differences were seen when patients were stratified by conditioning regimen.
• 5-year cumulative incidences of relapse were 24.3%, and 5-year nonrelapse mortality (NRM) was 21.9%. NRM was higher in patients who were 20 years or older at HSCT, but no other differences were seen.

“We herein demonstrate promising outcomes following allogeneic [HSCT] for patients with FA and MDS/AML using TCD grafts,” the authors wrote, calling for prospective studies to evaluate this approach with other HSCT platforms.

Reference

Satty AM, Klein E, Mauguen A, et al. T-cell depleted allogeneic hematopoietic stem cell transplant for the treatment of Fanconi anemia and MDS/AML. Bone Marrow Transplant. Published online September 29, 2023. doi:10.1038/s41409-023-02113-1