Investigators Discuss the Optimal Management of Myelofibrosis and Systemic Mastocytosis — Symposium Video Proceedings

Faculty

TARGET AUDIENCE
This program 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

• 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.

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
Video Program: Research To Practice designates this enduring material for a maximum of 2.25 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

AMERICAN BOARD OF INTERNAL MEDICINE (ABIM) — MAINTENANCE OF CERTIFICATION (MOC)
Successful completion of these CME activities, which includes participation in the evaluation components and post-tests, enables the participant to earn up to 2.25 (video) Medical Knowledge MOC points in the American Board of Internal Medicine’s (ABIM) Maintenance of Certification (MOC) program. Participants will earn MOC points equivalent to the amount of CME credits claimed for each activity. It is the CME activity provider’s responsibility to submit participant completion information to ACCME for the purpose of granting ABIM MOC credit.

Please note, these programs have been specifically designed for the following ABIM specialties: medical oncology and hematology.

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HOW TO USE THIS CE ACTIVITY
Video Program: This CME activity consists of a video component. To receive credit, the participant should review the CME information, watch the video, complete the post-test with a score of 80% or better and fill out the evaluation located at ResearchToPractice.com/ASHMF25/Video/CME.

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 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:

Professor Claire Harrison
Professor of Myeloproliferative Neoplasms
Guy’s and St Thomas’ NHS Foundation Trust
London, United Kingdom

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.

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

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.

Stephen T Oh, MD, PhD
Professor of Medicine
Co-Chief, Division of Hematology
Washington University School of Medicine
St Louis, Missouri

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.

Jeanne Palmer, MD
Associate Professor of Medicine
Mayo Clinic in Arizona
Phoenix, Arizona

Presentation (Money to Institution — Not Speakers Bureau): CTI BioPharma, a Sobi Company.

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

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, Biotheranostics Inc, A Hologic Company, 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, 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, Nuvation Bio Inc, 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.

These educational activities 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 publisher or grantors.

These activities are supported by educational grants from Blueprint Medicines, Bristol Myers Squibb, GSK, and Incyte Corporation.

Release date: January 2026
Expiration date: January 2027

After completing the post-test, learners may download and review the answers here in order to identify further areas of study.

Dr Palmer

Al-Ali HK et al. Primary analysis of JUMP, a phase 3b, expanded-access study evaluating the safety and efficacy of ruxolitinib in patients with myelofibrosis, including those with low platelet counts. Br J Haematol 2020;189(5):888-903. Abstract

Al-Ali HK et al. Impact of ruxolitinib treatment on the hemoglobin dynamics and the negative prognosis of anemia in patients with myelofibrosis. Leuk Lymphoma 2016;57(10):2464-7. Abstract

Cervantes F et al. Efficacy and safety of a novel dosing strategy for ruxolitinib in the treatment of patients with myelofibrosis and anemia: The REALISE phase 2 study. Leukemia 2021;35(12):3455-65. Abstract

Gerds A et al. Ruxolitinib rechallenge can improve constitutional symptoms and splenomegaly in patients with myelofibrosis: A case series. Clin Lymphoma Myeloma Leuk 2018;18(11):e463-8. Abstract

Gupta V et al. The impact of anemia on overall survival in patients with myelofibrosis treated with ruxolitinib in the COMFORT studies. Haematologica 2016;101(12):e482-4. Abstract

Harrison CN et al. Overall and progression-free survival in patients treated with fedratinib as first-line myelofibrosis (MF) therapy and after prior ruxolitinib (RUX): Results from the JAKARTA and JAKARTA2 trials. EHA 2021;Abstract S203.

Harrison CN et al. Fedratinib in patients with myelofibrosis previously treated with ruxolitinib: An updated analysis of the JAKARTA2 study using stringent criteria for ruxolitinib failure. Am J Hematol 2020;95(6):594-603. Abstract

Harrison CN et al. Case series of potential Wernicke’s encephalopathy in patients treated with fedratinib. ASH 2017;Abstract 4197.

Harrison CN et al. Janus kinase-2 inhibitor fedratinib in patients with myelofibrosis previously treated with ruxolitinib (JAKARTA-2): A single-arm, open-label, non-randomised, phase 2, multicentre study. Lancet Haematol 2017;4(7):e317-24. Abstract

Harrison CN et al. Long-term findings from COMFORT-II, a phase 3 study of ruxolitinib vs best available therapy for myelofibrosis. Leukemia 2016;30(8):1701-7. Abstract

Harrison CN et al. JAK inhibition with ruxolitinib versus best available therapy for myelofibrosis. N Engl J Med 2012;366(9):787-98. Abstract

Kuykendall AT et al. Between a rux and a hard place: Evaluating salvage treatment and outcomes in myelofibrosis after ruxolitinib discontinuation. Ann Hematol;97(3):435-41. Abstract

Maffioli M et al. A prognostic model to predict survival after 6 months of ruxolitinib in patients with myelofibrosis. Blood Adv 2022;6(6):1855-64. Abstract

Newberry KJ et al. Clonal evolution and outcomes in myelofibrosis after ruxolitinib discontinuation. Blood 2017;130(9):1125-1131. Abstract

Palandri F et al. Life after ruxolitinib: Reasons for discontinuation, impact of disease phase, and outcomes in 218 patients with myelofibrosis. Cancer 2020;126(6):1243-52. Abstract

Pardanani A et al. Safety and efficacy of fedratinib in patients with primary or secondary myelofibrosis: A randomized clinical trial. JAMA Oncol 2015;1(5):643-51. Abstract

Scott BL et al. Myeloablative versus reduced-intensity hematopoietic cell transplantation for acute myeloid leukemia and myelodysplastic syndromes. J Clin Oncol 2017;35(11):1154-61. Abstract

Verstovsek S et al. Long-term outcomes of 107 patients with myelofibrosis receiving JAK1/JAK2 inhibitor ruxolitinib: Survival advantage in comparison to matched historical controls. Blood 2012;120(6):1202-9. Abstract

Verstovsek S et al. A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis. N Engl J Med 2012;366(9):799-807. Abstract

Zhang Q et al. The Janus kinase 2 inhibitor fedratinib inhibits thiamine uptake: A putative mechanism for the onset of Wernicke’s encephalopathy. Drug Metab Dispos 2014;42(10):1656-62. Abstract

Dr Oh

Gerds AT et al. Momelotinib versus danazol in symptomatic patients with anaemia and myelofibrosis previously treated with a JAK inhibitor (MOMENTUM): An updated analysis of an international, double-blind, randomised phase 3 study. Lancet Haematol 2023;10(9):e735-46. Abstract

Gupta V et al. Momelotinib vs. ruxolitinib in myelofibrosis patient subgroups by baseline hemoglobin levels in the SIMPLIFY-1 trial. Leuk Lymphoma 2024;65(7):965-77. Abstract

Naymagon L, Mascarenhas L. Myelofibrosis-related anemia: Current and emerging therapeutic strategies. Hemasphere 2017;1(1). Abstract

Nicolosi M et al. Sex and degree of severity influence the prognostic impact of anemia in primary myelofibrosis: Analysis based on 1109 consecutive patients. Leukemia 2018;32(5):1254-8. Abstract

Oh ST et al. Changes in bone marrow fibrosis during momelotinib or ruxolitinib therapy do not correlate with efficacy outcomes in patients with myelofibrosis. EJHaem 2024;5(1):105-16. Abstract

Oh ST et al. Pacritinib is a potent ACVR1 inhibitor with significant anemia benefit in patients with myelofibrosis. Blood Adv 2023;7(19):5835-42. Abstract

Oh ST et al. ACVR1/JAK1/JAK2 inhibitor momelotinib reverses transfusion dependency and suppresses hepcidin in myelofibrosis phase 2 trial. Blood Adv 2020;4(18):4282-91. Abstract

Tefferi A et al. One thousand patients with primary myelofibrosis: The Mayo Clinic experience. Mayo Clin Proc 2012;87(1):25-33. Abstract

Verstovsek S et al. Momelotinib versus danazol in symptomatic patients with anaemia and myelofibrosis (MOMENTUM): Results from an international, double-blind, randomised, controlled, phase 3 study. Lancet 2023;401(10373):269-80. Abstract

Verstovsek S et al. MOMENTUM: Momelotinib vs danazol in patients with myelofibrosis previously treated with JAKi who are symptomatic and anemic. Future Oncol 2021;17(12):1449-58. Abstract

Verstovsek S et al. Robust overall survival and sustained efficacy outcomes during long term exposure to momelotinib in JAK inhibitor naïve and previously JAK inhibitor treated intermediate/high risk myelofibrosis patients. ASH 2020;Abstract 54.

Dr Rampal

Ajufo H et al. Pacritinib response is associated with overall survival in myelofibrosis: PERSIST-2 landmark analysis of survival. Eur J Haematol 2025;114(2):238-47. Abstract

Alhuraiji A et al. Clinical features and outcome of patients with poor-prognosis myelofibrosis based on platelet count <50 x 109/L: A single-center experience in 1100 myelofibrosis patients. ASCO 2016;Abstract 7068.

Gerds AT et al. Determining the recommended dose of pacritinib: Results from the PAC203 dose-finding trial in advanced myelofibrosis. Blood Adv 2020;4(22):5825-35. Abstract

Hernandez-Boluda JC et al. Clinical characteristics, prognosis and treatment of myelofibrosis patients with severe thrombocytopenia. Br J Haematol 2018;181(3):397-400. Abstract

Marcellino BK et al. The myelodepletive phenotype in myelofibrosis: Clinical relevance and therapeutic implication. Clin Lymphoma Myeloma Leuk 2020;20(7):415-21. Abstract

Masarova L et al. Severe thrombocytopenia in myelofibrosis is more prevalent than previously reported. Leuk Res 2020;91:106338. Abstract

Masarova L et al. Significance of thrombocytopenia in patients with primary and postessential thrombocythemia/polycythemia vera myelofibrosis. Eur J Haematol 2018;100(3):257-63. Abstract

Mascarenhas J et al. Pacritinib vs best available therapy, including ruxolitinib, in patients with myelofibrosis: A randomized clinical trial. JAMA Oncol 2018;4(5):652-9. Abstract

Scotch AH et al. Symptom burden profile in myelofibrosis patients with thrombocytopenia: Lessons and unmet needs. Leuk Res 2017;63:34-40. Abstract

Vachhani P et al. Platelet response in pacritinib-treated patients with cytopenic myelofibrosis: A retrospective analysis of PERSIST-2 and PAC203 studies. ASH 2023;Abstract 4554.

Prof Harrison

Bose P et al. Odyssey: A phase 2 open-label study of momelotinib in combination with luspatercept in patients with transfusion-dependent myelofibrosis. ASH 2024;Abstract 3191.2.

Fenaux P et al. Luspatercept for the treatment of anemia in myelodysplastic syndromes and primary myelofibrosis. Blood 2019;133(8):790-4. Abstract

Gerds AT et al. Safety and efficacy of luspatercept for the treatment of anemia in patients with myelofibrosis. Blood Adv 2024;8(17):4511-22. Abstract

Mascarenhas J et al. INCA33989 is a novel, first in class, mutant calreticulin-specific monoclonal antibody that demonstrates safety and efficacy in patients with essential thrombocythemia (ET). EHA 2025;Abstract LBA4002.

Mascarenhas J et al. Results from the randomized, multicenter, global phase 3 BOREAS study: Navtemadlin versus best available therapy in JAK inhibitor relapsed/refractory myelofibrosis. ASH 2024;Abstract 1000.

Mead A et al. Interim analysis of Promise, a clinical study combining the BET inhibitor OPN-2853 with ruxolitinib in patients with advanced myelofibrosis experiencing an inadequate response to ruxolitinib. ASH 2024;Abstract 3186.

Vachhani P et al. Disease-modifying activity of navtemadlin (NVTM) correlated with survival outcomes in Janus kinase inhibitor (JAKI) relapsed or refractory (R/R) myelofibrosis (MF) patients (PTS). EHA 2023;Abstract S214.

Vannucchi AM et al. Pelabresib in combination with ruxolitinib for Janus kinase inhibitor-naive patients with myelofibrosis: 72-week follow-up with long-term efficacy outcomes of the phase III MANIFEST-2 study. EHA 2025;Abstract S223.

Dr Kuykendall

Bose P et al. Initial results from Summit: An ongoing, 3-part, multi-center, randomized, double-blind, placebo-controlled phase 2 clinical study of bezuclastinib in adult patients with nonadvanced systemic mastocytosis (NonAdvSM). ASH 2023;Abstract 77.

Castells M et al. Efficacy and safety of avapritinib in indolent systemic mastocytosis (ISM): Results from the double-blind placebo-controlled PIONEER study. AAAAI 2023;Abstract 627.

Chifotides HT, Bose P. SOHO state of the art update and next questions: Current and emerging therapies for systemic mastocytosis. Clin Lymphoma Myeloma Leuk 2025;25(1):1-12. Abstract

DeAngelo DJ et al. An updated analysis on safety and efficacy of avapritinib in patients with advanced systemic mastocytosis from the Explorer clinical study: Long-term efficacy and safety. ASH 2022;Abstract.

Gotlib J et al. Efficacy and safety of avapritinib in advanced systemic mastocytosis: Interim analysis of the phase 2 PATHFINDER trial. Nat Med 2021;27(12):2192-9. Abstract

Mesa RA et al. Patient-reported outcomes among patients with systemic mastocytosis in routine clinical practice: Results of the TouchStone SM patient survey. Cancer 2022 Oct;128(20):3691-9. Abstract

Mukherjee S et al. Decreased survival among patients with indolent systemic mastocytosis: A population-level retrospective cohort analysis using healthcare claims dataset. ASH 2023;Abstract 75.

Pardanani A. Systemic mastocytosis in adults: 2019 update on diagnosis, risk stratification and management. Am J Hematol 2019;94(3):363-77. Abstract

Radia DH et. al. Avapritinib as first-line therapy in patients with advanced systemic mastocytosis: Efficacy and safety from the PATHFINDER clinical study. ASH 2022;Abstract.

Tashi T et al. Elenestinib, an investigational, next generation KIT D816V inhibitor, reduces mast cell burden, improves symptoms, and has a favorable safety profile in patients with indolent systemic mastocytosis: Analysis of the HARBOR trial. ASH 2023;Abstract 76.

Valent P et al. Harmonization of diagnostic criteria in mastocytosis for use in clinical practice: WHO vs ICC vs AIM/ECNM. J Allergy Clin Immunol Pract 2024;12(12):3250-60.e5. Abstract