Friday, December 5, 2025, Orlando, Florida, 3:15 PM – 5:15 PM Eastern Time
Expert Second Opinion: Investigators Discuss the Optimal Management of Myelofibrosis and Systemic Mastocytosis
A CME-Accredited Friday Satellite Symposium Preceding the 67th ASH Annual Meeting
Event Details
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2:45 PM – 3:15 PM — Registration and Light Snack
3:15 PM – 5:15 PM — Educational Meeting
Location
Hyatt Regency Orlando
9801 International Drive
Orlando, Florida
Meeting Room
Regency Ballroom TUV (Convention Level)
Professor Claire Harrison
Professor of Myeloproliferative Neoplasms
Guy’s and St Thomas’ NHS Foundation Trust
London, United Kingdom
Andrew T Kuykendall, MD
Associate Member, Department of Malignant Hematology
Moffitt Cancer Center
Associate Professor, Department of Oncologic Sciences
University of South Florida
Tampa, Florida
Stephen T Oh, MD, PhD
Associate Professor of Medicine
Co-Chief, Division of Hematology
Washington University School of Medicine
St Louis, Missouri
Jeanne Palmer, MD
Associate Professor of Medicine
Mayo Clinic in Arizona
Phoenix, Arizona
Raajit K Rampal, MD, PhD
Associate Member, Director
MPN and Rare Hematologic Malignancies Program
Director, Center for Hematologic Malignancies
Memorial Sloan Kettering Cancer Center
New York, New York
Moderator
Neil Love, MD
Research To Practice
Miami, Florida
This activity is supported by educational grants from Blueprint Medicines, Bristol Myers Squibb, GSK, and Incyte Corporation.
Agenda
2:45 PM – 3:15 PM — Registration and Light Snack
3:15 PM – 5:15 PM — Educational Meeting
MODULE 1: Current Clinical Decision-Making for Myelofibrosis (MF) in the Absence of Severe Cytopenias — Dr Palmer
- Appropriate threshold for initiating active therapy for patients with MF; published data supporting early intervention versus observation for intermediate- and high-risk disease
- Factors influencing the choice of therapy for patients with MF without severe cytopenias
- Published research database supporting the use of ruxolitinib for patients with intermediate- and high-risk MF; impact of ruxolitinib on symptom control and survival
- Initial dosing of and dose-modification strategies for ruxolitinib
- Key efficacy and safety findings with fedratinib for newly diagnosed MF and for MF that is intolerant or resistant to ruxolitinib; selection of patients to receive fedratinib
- Spectrum, frequency and severity of common and unique toxicities associated with ruxolitinib and fedratinib; optimal approach to monitoring and management of treatment-related adverse events (AEs)
MODULE 2: Managing MF in Patients with Anemia — Dr Oh
- Incidence of MF-associated anemia; differences between momelotinib and other approved JAK inhibitors and rationale for its activity in patients with anemia
- Key findings from the Phase III MOMENTUM study of momelotinib versus danazol for symptomatic, anemic patients previously treated with a JAK inhibitor
- Efficacy and safety of momelotinib compared to ruxolitinib in the subset of patients with JAK inhibitor-naïve disease and severe anemia in the Phase III SIMPLIFY-1 trial
- FDA approval of momelotinib for patients with MF and disease-related anemia; current role in disease management
- Comparative tolerability/toxicity profile of momelotinib versus other approved JAK inhibitors
- Retrospective analysis of the PERSIST-2 study to evaluate the utility of pacritinib for MF and anemia; role, if any, of pacritinib in treatment for this subset of patients
MODULE 3: Managing MF in Patients with Thrombocytopenia — Dr Rampal
- Incidence of thrombocytopenia in patients with newly diagnosed MF and in those with prior JAK inhibitor exposure
- Mechanistic similarities and differences between pacritinib and other approved JAK inhibitors; implications for the safety of pacritinib for patients with low platelet counts
- Published clinical research findings with pacritinib for MF, including among patients with baseline thrombocytopenia
- FDA approval of pacritinib for patients with MF and severe thrombocytopenia; optimal use in clinical practice
- Design, entry criteria and key endpoints of the ongoing confirmatory Phase III PACIFICA trial of pacritinib versus physician’s choice of therapy for patients with MF and severe thrombocytopenia; estimated completion date
- Incidence and severity of treatment-related AEs, including hemorrhage and cardiovascular events, reported with pacritinib; optimal monitoring and management
MODULE 4: Promising Novel Agents Under Investigation for MF — Prof Harrison
- Mechanism of action of luspatercept; biological rationale for its investigation for MF-associated anemia
- Emerging outcomes from and potential research and clinical implications of the Phase III INDEPENDENCE study evaluating luspatercept with concomitant JAK inhibitor therapy for patients with MF-associated anemia receiving red blood cell transfusions
- Current clinical role, if any, of luspatercept for patients with MF experiencing anemia; ongoing ODYSSEY study attempting to further elucidate the potential utility of luspatercept
- Rationale for the inhibition of BET proteins in MF; key efficacy and safety findings from the Phase III MANIFEST-2 study combining pelabresib with ruxolitinib for patients with JAK inhibitor-naïve MF
- Available data with and ongoing evaluation of other investigational BET inhibitors, such as INCB57643 and NUV868, in MF
- Mechanism of antitumor activity of navtemadlin; key efficacy and safety data from the Phase III BOREAS trial evaluating navtemadlin monotherapy for patients with MF who were resistant or refractory to JAK inhibitors
- Other promising investigational agents and strategies for patients with MF
MODULE 5: Current and Future Management of Systemic Mastocytosis (SM) — Dr Kuykendall
- Rationale for targeting the KIT D816V mutation in patients with SM; mechanism of action of avapritinib
- Published efficacy and safety findings from the pivotal Phase II PIONEER trial of avapritinib versus placebo for patients with indolent SM whose symptoms were not adequately controlled with standard therapy
- Outcomes achieved in key studies, such as EXPLORER and PATHFINDER, evaluating avapritinib for advanced SM
- Selection of patients with various SM subtypes for whom treatment with avapritinib would be appropriate
- Mechanistic similarities and differences between avapritinib and next-generation KIT D816V inhibitors, such as elenestinib and bezuclastinib; implications for efficacy and tolerability
- Preliminary safety and efficacy data with elenestinib for patients with indolent SM whose symptoms are not adequately controlled by best supportive care; ongoing evaluation for indolent SM in the HARBOR study
- Available and emerging results with and ongoing investigation of bezuclastinib for nonadvanced and advanced SM
- Other promising agents and strategies in clinical development for patients with SM
CE Information
Target Audience
This activity is intended for hematologists, medical oncologists, hematology-oncology fellows and other healthcare providers involved in the treatment of myelofibrosis (MF) and systemic mastocytosis (SM).
Learning Objectives
Upon completion of this activity, participants should be able to
- Use an understanding of disease biology and natural history to effectively counsel patients diagnosed with MF regarding their long-term prognosis.
- Analyze how patient age, performance status, prior therapeutic exposure and other biological and disease-related factors affect the selection and sequencing of therapy for primary and secondary MF.
- Appraise available research findings informing the safety and efficacy of approved JAK inhibitors for patients with MF, including those with thrombocytopenia, anemia or compromised renal function.
- Evaluate published research findings with JAK inhibitors for patients with MF and anemia to optimize therapeutic decision-making.
- Assess available research findings with novel investigational strategies, and consider the future clinical application of these approaches for patients with MF.
- Use an understanding of disease biology to appropriately classify SM, and effectively counsel patients diagnosed with this disease regarding their long-term prognosis.
- Recall the rationale for targeting the KIT D816V mutation in SM, and appreciate the current role of selective kinase inhibitors of D816V-mutated KIT for patients with indolent and advanced disease.
CME Credit Form
A CME credit link will be given to each participant as part of the meeting course materials.
Accreditation Statement
Research To Practice is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.
Credit Designation Statement
Research To Practice designates this live activity for a maximum of 2 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
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Unlabeled/Unapproved Uses Notice
This educational activity may contain discussion of published and/or investigational uses of agents that are not indicated by the Food and Drug Administration. Research To Practice does not recommend the use of any agent outside of the labeled indications. Please refer to the official prescribing information for each product for discussion of approved indications, contraindications and warnings. The opinions expressed are those of the presenters and are not to be construed as those of the provider or grantors.
Content Validation and Disclosures
Research To Practice (RTP) is committed to providing its participants with high-quality, unbiased and state-of-the-art education and adheres to the ACCME’s Standards for Integrity and Independence in Accredited Continuing Education. Any individuals in a position to control the content of an accredited continuing education activity, including faculty, planners, reviewers and others, are required to disclose all relevant financial relationships with ineligible entities (commercial interests). All relevant conflicts of interest will have been mitigated prior to the commencement of this activity. In addition, all activity content is reviewed by RTP scientific staff and an external, independent physician reviewer for fair balance, scientific objectivity of studies referenced and patient care recommendations.
FACULTY — The following faculty reported relevant financial relationships with ineligible entities:
Prof Harrison — Advisory Committees: Galecto Inc, Geron Corporation, GSK, Incyte Corporation, Karyopharm Therapeutics, Keros Therapeutics, Novartis, Silence Therapeutics, Sobi; Consulting Agreements: Galecto Inc, Geron Corporation, GSK, Incyte Corporation, Karyopharm Therapeutics, Keros Therapeutics, Novartis, Silence Therapeutics, Sobi, Takeda Pharmaceutical Company Limited; Contracted Research: Galecto Inc, Geron Corporation, GSK, Incyte Corporation, Karyopharm Therapeutics, Novartis, Silence Therapeutics, Sobi; Data and Safety Monitoring Boards/Committees: Galecto Inc, Incyte Corporation, Novartis, Silence Therapeutics; Speakers Bureaus: AOP Health, GSK, Incyte Corporation, Novartis. Dr Kuykendall — Advisory Committees: AbbVie Inc, Blueprint Medicines, Bristol Myers Squibb, Cogent Biosciences, CTI BioPharma, a Sobi Company, Incyte Corporation, Karyopharm Therapeutics, PharmaEssentia; Consulting Agreements: AbbVie Inc, Karyopharm Therapeutics, MorphoSys; Contracted Research: Blueprint Medicines, Bristol Myers Squibb, Geron Corporation, Janssen Biotech Inc, MorphoSys, Protagonist Therapeutics; Data and Safety Monitoring Boards/Committees: Geron Corporation. Dr Oh — Consulting Agreements: AbbVie Inc, Bristol Myers Squibb, Cogent Biosciences, CTI BioPharma, a Sobi Company, Geron Corporation, GSK, Incyte Corporation, Morphic Therapeutic, a wholly owned subsidiary of Lilly, MorphoSys, Protagonist Therapeutics; Stock Options — Private Companies: Harmonic Discovery, Phoenix Molecular Designs. Dr Palmer — Presentation (Money to Institution — Not Speakers Bureau): CTI BioPharma, a Sobi Company. Dr Rampal — Advisory Committees: AbbVie Inc, Blueprint Medicines, Bristol Myers Squibb, Cogent Biosciences, CTI BioPharma, a Sobi Company, Disc Medicine, Galecto Inc, GSK, Incyte Corporation, Jazz Pharmaceuticals Inc, Kartos Therapeutics, Karyopharm Therapeutics, MorphoSys, Novartis, Opna Bio, PharmaEssentia, Roche Laboratories Inc, Stemline Therapeutics Inc, Sumitomo Dainippon Pharma Oncology Inc, Zentalis Pharmaceuticals; Contracted Research: BioMed Valley Discoveries, Incyte Corporation, MorphoSys, Ryvu Therapeutics, Stemline Therapeutics Inc, Zentalis Pharmaceuticals; Data and Safety Monitoring Boards/Committees: Merck.
MODERATOR — Dr Love is president and CEO of Research To Practice. Research To Practice receives funds in the form of educational grants to develop CME activities from the following companies: Aadi Bioscience, AbbVie Inc, ADC Therapeutics, Agendia Inc, Alexion Pharmaceuticals, Amgen Inc, Array BioPharma Inc, a subsidiary of Pfizer Inc, Arvinas, Astellas, AstraZeneca Pharmaceuticals LP, Aveo Pharmaceuticals, Bayer HealthCare Pharmaceuticals, BeOne, Black Diamond Therapeutics Inc, Blueprint Medicines, Boehringer Ingelheim Pharmaceuticals Inc, Bristol Myers Squibb, Celcuity, Clovis Oncology, Coherus BioSciences, Corcept Therapeutics Inc, CTI BioPharma, a Sobi Company, Daiichi Sankyo Inc, Eisai Inc, Elevation Oncology Inc, Exact Sciences Corporation, Exelixis Inc, Genentech, a member of the Roche Group, Genmab US Inc, Geron Corporation, Gilead Sciences Inc, GSK, Helsinn Therapeutics (US) Inc, Hologic Inc, ImmunoGen Inc, Incyte Corporation, Ipsen Biopharmaceuticals Inc, Jazz Pharmaceuticals Inc, Johnson & Johnson, Karyopharm Therapeutics, Kite, A Gilead Company, Kura Oncology, Legend Biotech, Lilly, MEI Pharma Inc, Merck, Mersana Therapeutics Inc, Mirati Therapeutics Inc, Mural Oncology Inc, Natera Inc, Novartis, Novartis Pharmaceuticals Corporation on behalf of Advanced Accelerator Applications, Novocure Inc, Nuvalent, Pfizer Inc, Pharmacyclics LLC, an AbbVie Company, Puma Biotechnology Inc, Regeneron Pharmaceuticals Inc, Rigel Pharmaceuticals Inc, R-Pharm US, Sanofi, Seagen Inc, Servier Pharmaceuticals LLC, SpringWorks Therapeutics Inc, Stemline Therapeutics Inc, Sumitomo Pharma America, Syndax Pharmaceuticals, Taiho Oncology Inc, Takeda Pharmaceuticals USA Inc, TerSera Therapeutics LLC, and Tesaro, A GSK Company.
RESEARCH TO PRACTICE CME PLANNING COMMITTEE MEMBERS, STAFF AND REVIEWERS
Planners, scientific staff and independent reviewers for Research To Practice have no relevant conflicts of interest to disclose.
This activity is supported by educational grants from Blueprint Medicines, Bristol Myers Squibb, GSK, and Incyte Corporation.
Location
Hyatt Regency Orlando
9801 International Drive
Orlando, FL 32819
+1 407 284 1234
Meeting Room
Regency Ballroom TUV (Convention Level)
Directions
The Hyatt Regency Orlando hotel is connected by pedestrian bridges to the Orange County Convention Center, where the ASH Annual Meeting is taking place.
Registration
There is no registration fee for this event. For the in-person symposium in Orlando, preregistration (below) is required as seating is limited for this program.
NOTICE:
Registration for this event is independent of registration for the ASH Annual Meeting.
Hyatt Regency Orlando is conveniently connected by pedestrian bridge to the Orange County Convention Center, where the 67th ASH Annual Meeting is taking place.
If you have any questions, please feel free to contact us at Meetings@ResearchToPractice.com or (800) 233-6153.
NOTICE:
Registration for this event is independent of registration for the ASH Annual Meeting and Exposition.
Please note, we will stream this event over Zoom. After registering you will receive a separate confirmation from Zoom with the viewing instructions.
REGISTRATION FOR WEBCAST »Photography and/or video recording may be taken during the educational program by Research To Practice and used in future educational offerings.
Research To Practice fully complies with the legal requirements of the ADA. If you require any physical, dietary or other accommodations, please call us at (800) 233-6153 before the event.
If you have any questions, please feel free to contact us via email at Meetings@ResearchToPractice.com, or call (800) 233-6153.