Ruemu Birhiray, MD

Panelists discuss how important end points for evaluating new myelofibrosis therapies include overall survival, symptom improvement, splenic volume reduction, hematologic parameters, disease modification, safety, and patient-reported outcomes, all of which help assess the impact of treatment on both clinical outcomes and quality of life.

Panelists discuss how investigational therapies beyond Janus kinase (JAK) inhibition, including epigenetic modulation, telomerase inhibition, PI3K/AKT/mTOR pathway inhibition, immune modulation, TGF-β inhibition, and novel anti-fibrotic agents, are showing promise in improving efficacy and targeting different disease pathways in myelofibrosis.

Panelists discuss how in polycythemia vera (PV), defining and managing treatment resistance or intolerance is crucial, requiring careful monitoring and timely intervention, including assessing adherence, evaluating mutations, and making therapeutic adjustments such as dose modifications, switching to alternative therapies like ruxolitinib or interferon, and employing a multidisciplinary approach to ensure optimal disease control and patient outcomes.

Panelists discuss how the choice between hydroxyurea (HU) and interferon (IFN) for cytoreductive therapy in polycythemia vera (PV) depends on factors such as patient age, long-term safety, tolerability, comorbidities, response to previous treatments, and patient preferences, with HU often preferred in older patients and IFN favored for younger patients, those intolerant to HU, or those seeking potential disease-modifying effects.

Panelists discuss how managing myelofibrosis patients with challenging cytopenias involves careful treatment selection, regular monitoring of blood counts, and tailored dose adjustments to balance disease control with the risks of exacerbating hematologic toxicities.

Panelists discuss how selecting and optimizing Janus kinase (JAK) inhibitor therapy in myelofibrosis involves personalizing treatment based on patient risk factors, comorbidities, and preferences while emphasizing regular monitoring to manage adverse effects and improve quality of life.

Panelists discuss how frequent phlebotomy requirements—typically more than 4 to 6 times per year—serve as a marker of inadequate disease control in polycythemia vera (PV), prompting consideration of cytoreductive therapy to improve hematologic stability, alleviate symptoms, and reduce thrombotic risk.

Panelists discuss how the decision to initiate cytoreductive therapy in polycythemia vera (PV) is driven by high-risk features such as age over 60 and thrombotic history, as well as factors like inadequate hematocrit control, symptom burden, and intolerance to phlebotomy, with therapy tailored to individual patient needs and goals.

Panelists discuss how managing asymptomatic splenomegaly in myelofibrosis requires a nuanced approach, balancing observation in low-risk patients with early intervention using Janus kinase (JAK) inhibitors in higher-risk cases, while emphasizing the importance of regular monitoring, patient preferences, and shared decision-making.

Panelists discuss how survival data from the COMFORT trials support the use of ruxolitinib in myelofibrosis, especially in intermediate- to high-risk patients with significant symptom burden or splenomegaly, while highlighting the need to consider patient selection, crossover effects, and real-world applicability when interpreting these results.

Panelists discuss how thrombotic risk assessment—anchored in age, thrombotic history, and additional factors like leukocytosis and JAK2 allele burden—guides personalized therapy in polycythemia vera (PV), balancing cytoreduction, symptom control, and prevention of vascular events.

Panelists discuss how integrating molecular markers—particularly JAK2 V617F allele burden—and clinical features such as symptom burden and thrombotic history inform diagnosis, risk stratification, and monitoring strategies in polycythemia vera to anticipate disease progression and guide personalized treatment.

Panelists discuss how initiating therapy in myelofibrosis requires a personalized approach that balances symptom burden, risk stratification, cytopenias, and patient goals, with treatment decisions—often involving Janus kinase (JAK) inhibitors—tailored to optimize both disease control and quality of life.

Panelists discuss how accurate risk stratification using clinical and molecular prognostic tools like DIPSS, MIPSS70, and GIPSS guides personalized treatment decisions in myelofibrosis, ranging from observation in low-risk patients to Janus kinase (JAK) inhibitor therapy and early transplant evaluation in higher-risk cases.

Panelists discuss how distinguishing pre-fibrotic myelofibrosis from other myeloproliferative neoplasms like essential thrombocythemia remains challenging due to overlapping clinical and molecular features, subjective histopathologic interpretation, and variable application of diagnostic criteria, underscoring the need for expert pathology review and multidisciplinary evaluation to guide accurate diagnosis and management.

Panelists discuss how evolving insights into myelofibrosis—driven by molecular profiling, risk-adaptive therapy, and emerging treatments—are reshaping clinical practice toward more personalized care, with increasing focus on managing cytopenias, treatment resistance, and long-term disease modification.