Elliot Stieglitz, MD
Associate Professor
Pediatrics
School of Medicine

elliot.stieglitz@ucsf.edu

Juvenile myelomonocytic leukemia (JMML) is a blood cancer that affects young children and is difficult to diagnose. Currently available therapies cure only half of patients, with some children experiencing an aggressive disease course while some children get better with very little treatment. We have now shown that the presence of secondary mutations at diagnosis predicts a poor outcome.

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We have also shown that DNA methylation profiling can identify patients most likely to experience favorable outcomes.

We recently completed a clinical trial 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 and met its primary objective. This trial was based on extensive pre-clinical testing and a strong genomic rationale. This trial marks an important step towards more effectively treating patients with Ras driven leukemia using targeted agents.

Our current trial (NCT05849662) risk-stratifies patients with newly diagnosed JMML to receive different intensities of therapy based on the number of mutations and DNA methylation signatures at diagnosis. It also combines trametinib with standard-of-care therapy for newly diagnosed patients.

Awards

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  • R Accelerated Award, Alex's Lemonade Stand Foundation, 2024-2028
  • Translational Research Grant, Leukemia Lymphomoa Society, 2023-2027
  • Research Grant, Cookies for Kids' Cancer, 2023-2024
  • R37, NIH NCI, 2022-2027
  • Translational Research Award, V Foundation, 2019-2021
  • Research Award, Pediatric Cancer Research Foundation, 2019-2020
  • Individual Research Award, American Cancer Society, 2016-2017
  • Translational Research Training in Hematology, ASH/EHA, 2016
  • Young Investigator Award, Alex's Lemonade Stand Foundation, 2015-2017
  • Fellow in Clinical Research, St. Baldrick's Foundation Fellowship, 2014-2016

Education & Training

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  • Pediatric Hematology Oncology Fellowship University of California, San Francisco 2014
  • Pediatric Residency New York Presbyterian - Weill Cornell 2011
  • MD School of Medicine Stony Brook 2008

Websites

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Grants and Projects

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Publications (95)

Top publication keywords:
MutationPyrimidinonesPrecursor B-Cell Lymphoblastic Leukemia-LymphomaPrecursor Cell Lymphoblastic Leukemia-LymphomaHematologic NeoplasmsHematopoietic Stem Cell TransplantationMyelodysplastic SyndromesMyeloproliferative Disordersfms-Like Tyrosine Kinase 3ErwiniaMyelodysplastic-Myeloproliferative DiseasesGene Expression Regulation, LeukemicDrug HypersensitivityGATA2 Transcription FactorLeukemia, Myelomonocytic, Juvenile

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