The FDA approval will benefit children and adults with acute myeloid leukemia (AML), and myelodysplastic syndromes (MDS).
Rye Brook, N.Y., January 28, 2025 – The FDA approved treosulfan (Grafapex™) as a preparative treatment prior to stem cell transplant in adults and children 1 year of age and older who have acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).
The drug, which is a form of chemotherapy, is used in combination with another chemo drug called fludarabine.
“Even as we are moving away from chemotherapy for many blood cancer patients, it remains an essential treatment for some people with AML and MDS in preparation for a stem cell transplant,” says Lore Gruenbaum, Ph.D., LLS Chief Scientific Officer. “Treosulfan improves survival while minimizing side effects, which is extremely important for the growing number of older people with these aggressive forms of blood cancer who are considered eligible for stem cell transplant.”
In a 570-patient clinical trial, those who received the treosulfan/fludarabine combination were more likely to have event-free survival two years after treatment compared to patients treated with another chemo drug commonly used to prepare patients for stem cell transplant (64.0% compared to 50.4%). Of note, event-free survival was defined as a patient remaining free of certain complications, disease recurrence or side effects that the treatment was intended to prevent or delay.
LLS is dedicated to further improving the lives of transplant patients
LLS has been at the forefront of supporting transplant research, beginning in the 1950s when transplant was first developed.
“We currently support some of the world’s leading researchers at premier academic institutions who strive to find safer and effective ways to improve treatment outcomes for patients who receive a transplant,” says Dr. Gruenbaum.
View LLS’s latest academic grants dedicated to transplant in patients with blood cancer
Just last week, Dr. Gruenbaum wrote about recent and anticipated progress expected in the treatment of AML. Because it is one of the most aggressive and difficult to treat blood cancers, LLS dedicates more than 25% of its comprehensive research portfolio budget to AML.
If you or a loved one need personalized disease, treatment or support information, you can contact one of our Information Specialists: https://www.lls.org/support-resources/information-specialists