Myelofibrosis in Clinical Practice: Disease Landscape, Treatment Approaches, and Evolving Management Strategies

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.

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 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 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 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 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 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 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 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 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 the evolution of myelofibrosis care has led to advancements in pharmacotherapy, risk stratification, and emerging therapies while highlighting ongoing unmet needs such as long-term disease control, treatment resistance, anemia and thrombocytopenia management, and the need for personalized treatment approaches.

Panelists discuss how JAK inhibitor therapy, while effective in treating myelofibrosis and other hematologic disorders, increases the risk of skin cancers, particularly non-melanoma skin cancers, and emphasize the importance of regular screening, patient education, and preventive measures to manage this risk.