What Are the Outcomes of Patients With Fanconi Anemia Undergoing HSCT?

Patients with fanconi anemia with moderately severe renal insufficiency can safely and effectively undergo hematopoietic stem cell transplantation (HSCT) with chemotherapy dose adjustments, according to a recent study.

The results were presented at the 2024 Tandem Transplant & Cellular Therapy Meetings of ASTCT^® and CIBMTR^® by Smitha Hosahalli Vasanna, a Pediatric Blood and Marrow Transplant Fellow at the University of Minnesota.

The researchers also determined that requiring dialysis before HSCT is associated with “very poor outcomes,” and therefore HSCT should be avoided in these patients if possible.

As a conditioning regimen, all 20 patients received cyclophosphamide and mesna, and 19 patients received fludarabine. The cyclophosphamide dose was reduced by 50% in five patients, while the fludarabine dose was reduced by 20% in six patients, 50% in four patients, and 75% in one patient. Four patients did not require any dose reductions in either drug, while five patients had dose adjustments in both drugs.

Of six patients who required dialysis during HSCT, two received chemotherapy dose reductions and five died within the first two years of HSCT. At one year, the probability of survival for the nine patients who did not require dialysis before HSCT and received chemotherapy dose reductions is 65%.

At a median of 10 days after HSCT, all but one of the evaluable patients achieved neutrophil engraftment. One patient developed grade II-IV acute graft-versus-host disease (GVHD), and no patients developed chronic GVHD.

Reference

Hosahalli Vasanna S, Nelson K, Cao Q, et al. Successful hematopoietic cell transplantation in patients with fanconi anemia with severe renal insufficiency. Abstract #395. Presented at the 2024 Tandem Transplantation & Cellular Therapy Meetings of ASTCT® and CIBMTR®; February 21-24, 2024; San Antonio, Texas.