New Stem Cell Transplantation Preparation Regimen for Patients with Myeloid Disorders
The FDA approved treosulfan and fludarabin for patients with hematologic disorders prior to stem cell transplantation.
The U.S. Food and Drug Administration (FDA) has approved treosulfan (Grafapex) with fludarabine for people with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) who are preparing to undergo a hematopoietic stem cell transplantation (HSCT). The approval covers patients 1 year and older.
Stem cell transplantation offers a way for patients with myeloid blood disorders, including AML and MDS, to replace dysfunctional or cancerous blood cells with stem cells that will mature into healthy blood cells. Before receiving the transplant, patients undergo myeloablative conditioning, usually in the form of high-dose chemotherapy or radiation, to kill the dysfunctional cells and make room in the bone marrow for the transplanted stem cells.
Treosulfan is thought to kill cells by disrupting DNA replication and cell division. This is the first FDA approval for treosulfan.
This approval was based on results from the randomized, active-controlled phase III MC-FludT.14/L trial, in which 570 patients with either AML or MDS were randomly assigned (1:1) to receive either treosulfan or busulfan, both in combination with fludarabine, prior to receiving HSCT.
Patients who received treosulfan and fludarabine had a 33% lower risk of death from any cause, compared with those who received busulfan and fludarabine. When survival was analyzed by disease type, patients with AML in the treosulfan arm were found to have a 27% lower risk of death than those in the busulfan arm, and patients with MDS in the treosulfan arm were found to have a 36% lower risk of death than those in the busulfan arm.
The recommended dose for treosulfan is 10 g/m2 of body surface area, given on the second, third, and fourth days prior to HSCT. The recommended dose for fludarabine is 30mg/m2 of body surface area, given on the second, third, fourth, fifth, and sixth days prior to HSCT.
AML is a cancer of the blood and bone marrow characterized by an overproduction of immature white blood cells. According to federal statistics, it was estimated that 20,800 individuals would be diagnosed with AML and 11,220 patients would die of the disease in the United States in 2024.
MDS refers to a group of rare blood cancers in which the patient’s body does not produce mature blood cells. MDS has been estimated to impact approximately 4 out of every 100,000 individuals in the United States each year.
The FDA rendered its decision on January 21, 2025. Check this resource for updated information on all therapeutics regulated by the FDA.
