"An Overview of Current Research, Diagnosis, and Treatment of JMML"
Juvenile Myelomonocytic Leukemia (JMML) is a rare form of childhood leukemia that affects approximately 1 per million children, meaning that very few hematologist/oncologists have ever treated a patient with JMML. The current survival rate for kids with JMML is <15% without treatment, ~55% with a bone marrow transplant, and up to an additional third of relapsed patients with a second bone marrow transplant. These rates are far below the survival rate for children with ALL. Chemotherapy has so far proven ineffective at inducing a lasting remission for patients.
Currently, few standardized treatment protocols targeted to JMML exist. One is administered by the European Working Group on the Myelodysplastic Syndromes (EWOG-MDS), and one by the Japanese Pediatric Leukemia/Lymphoma Study Group (JPLSG). These protocols overlap in research objectives but are not similar enough for sample populations to be combined. Only the Japanese protocol is currently part of a funded study; the other is a voluntary regimen until funding is available for a full trial to commence.
Though research is published and shared worldwide regarding JMML by various individuals, the potential for international cooperation for research and treatment is not maximized or centrally coordinated.
Based on this information, The JMML Foundation, whose mission is “to cure JMML and to improve the quality of life of JMML patients and families through research, education, advocacy, and charity,” has determined the need for web-based CME-certified training on JMML and a recurring International JMML Symposium. The goal of the symposium will be “to establish an international scientific forum, driven simply by a focus on the JMML disease and not by country or institutional goals, that combines clinical, basic, and translational research in order to improve survivability among JMML patients.” The goals of this web-based CME-certified training program, consisting of material presented at the December 2009 JMML Foundation International Symposium (slide presentations, transcripts, and audio and video of all presentations) are to encourage more physicians and researchers to collaborate in the search for a cure for Juvenile Myelomonocytic Leukemia (JMML) by presenting an overview of current international research, and to provide up-to-date training on JMML diagnosis and clinical options to physicians worldwide who are treating a child with JMML.
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Contents: A discussion of the basic characteristics of the JMML disease, current translational studies in the U.S., and proposed topics for further consideration and collaboration in future research and clinical studies.
Learning objective: At the conclusion of this activity you will be able to discuss current translational studies and questions that must be answered to advance translational research for aimed at curing JMML
Faculty name and affiliation: Mignon Loh, MD, University of California, San Francisco
Faculty disclosure: Dr. Loh has no disclosures to report.
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3. "Juvenile Myelomonocytic Leukemia (JMML) Preclinical Models (Overview)"
Contents: An overview of the murine models that have been developed to shed light on the genetics of JMML and facilitate the process of testing new agents for JMML.
Learning objective: At the conclusion of this activity you will be able to discuss pre-clinical models that have been used or will be used to facilitate understanding of JMML
Faculty name and affiliation: Rebecca Chan, MD, PhD, Indiana University School of Medicine
Faculty disclosure: Dr. Chan has no disclosures to report.
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Contents: A discussion of the previous WHO-accepted diagnostic criteria and the revised diagnostic criteria agreed-upon by international experts in JMML research and clinical treatment in December 2008, as well as a brief overview of the results of historical clinical studies for JMML.
Learning objectives: At the conclusion of this activity you will be able to...
a. Describe the most current JMML diagnostic criteria proposed at the 2008 JMML International Working Group meeting.
b. Explain the effectiveness of various clinical options for treating children with JMML.
Faculty name and affiliation: Christian Flotho, MD, PhD, University of Freiburg, Germany
Faculty disclosure: Dr. Flotho has no disclosures to report.
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The JMML Foundation wishes to extend a huge thanks to each of the four presenters: Drs. Ben Braun, Mignon Loh, Becky Chan, and Christian Flotho. These accomplished researchers and clinicians developed this training voluntarily for the benefit of children with JMML worldwide and the physicians who treat them. You are family to us!
This CME activities and related materials were produced by The JMML Foundation and CME Outfitters with support from the National Cancer Institute (NCI) and the National Institutes of Health (NIH) Office of Rare Diseases (ORD). All materials above are copyright 2009 by the JMML Foundation.