Juvenile myelomonocytic leukemia (JMML) is a rare but aggressive childhood leukemia. While hematopoietic stem cell transplantation is curative for some patients, approximately half of all patients will still have their leukemia return following a transplant.
Recent studies have identified that the number of changes in the DNA (mutational burden) and the degree of DNA methylation (a chemical change to DNA) are both predictive of the outcome in JMML. In an effort to improve outcomes for patients with newly diagnosed JMML, a multi-center study is underway that determines each patient’s treatment based on a blood test.
Under the auspices of the Therapeutic Advances in Childhood Leukemia & Lymphoma (TACL) consortium, a Phase 1/2 clinical trial led by UC San Francisco pediatric oncologist Elliot Stieglitz, MD, will risk stratify patients based on mutational burden and DNA methylation to receive different therapies.
“This is the first time we will stratify patients using a DNA methylation test and be able to tailor treatments to individual patients,” said Stieglitz, a UCSF associate professor of Pediatrics. “We know that patients with lower amounts of DNA methylation have better outcomes and may not require a stem cell transplant. Patients with higher amounts of DNA methylation have poorer outcomes and will require a stem cell transplant following their trial therapy.”