Chronic GvHD is a leading cause of serious complications after stem cell transplants, an important treatment option for people with certain types of myeloma, leukemia and lymphoma
Rye Brook, N.Y., August 16, 2024 – The U.S. Food and Drug Administration this week approved axatilimab-csfr (Niktimvo™) for the treatment of chronic graft-versus host disease (cGvHD) in children and adults. GvHD is a common complication of stem cell transplants and happens when the donor’s cells (the graft) see the patient’s healthy cells (the host) as foreign and attack them.
“This new treatment will help more blood cancer patients successfully navigate the challenge of GvHD after stem cell transplant,” says Dr. Gwen Nichols, LLS Chief Medical Officer. “While doctors have learned a lot about how to prevent this serious complication, it still impacts many people.”
Up to half of patients will develop chronic GvHD following a stem cell transplant. It can last for years or even a lifetime, with symptoms ranging from mild to life-threatening. LLS currently has $17 million committed to research into ways to improve stem cell transplants, including $8 million for nine research projects focused on better outcomes for patients with GvHD.
Axatilimab uses a different approach than other GvHD treatments. It reduces levels of a protein called CSF-1R that drives both the inflammation and scarring (fibrosis) that are common in chronic GvHD. In a clinical trial, three in four patients (all of whom had tried at least two previous cGvHD treatments) responded to axatilimab within six months. Six in 10 (60%) of them maintained their response for at least 12 months.
“Every new treatment that improves quality of life and health outcomes for people with blood cancer is an important step forward in our mission,” says Dr. Nichols. “Improving outcomes is about more than just research; LLS also provides free information and support for patients, survivors and families, and we advocate for policies that help them access the best care.”
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