Elliot Stieglitz, MD
Associate Professor, Pediatric Hematology/Oncology, UCSF
Juvenile myelomonocytic leukemia (JMML) is a blood cancer that affects young children and is often difficult to diagnose and treat. Currently available therapies cure only half of patients, with some children experiencing an aggressive disease course while others get better with very little treatment. We have now shown that the presence of more than one mutation at diagnosis portends a poor outcome. We have also shown that DNA methylation profiling can identify patients who are most likely to experience favorable outcomes with little to no therapy.
We recently completed a clinical trial conducted through the Children's Oncology Group for patients with relapsed and refractory JMML. This trial tested the safety and efficacy of the oral MEK inhibitor, trametinib. This trial met its primary objective with half of all patients experiencing an objective response. Our next trial will risk-stratify patients with newly diagnosed JMML to receive different therapies tailored to the risk of relapse predicted by both the number of mutations and DNA methylation signatures at diagnosis. This trial will move trametinib into the front-line setting in combination with azacitidine. This clinical trial will be conducted through the Therapeutic Advances in Childhood Leukemia & Lymphoma (TACL) consortium and is funded by the National Cancer Institute (R37).
Yeshiva University, New York, B.A., 06/03, Biology
Stony Brook School of Medicine, New York, M.D., 06/08, Medicine
NYPH - Weill Cornell Medical Center, New York, Residency, 06/11, Pediatrics
University of California, San Francisco, Fellowship, 07/14, Pediatric Heme/Onc