Clinical Decision-Making Framework for Initiating Myelofibrosis Therapy

EP: 1.The Evolving Landscape of Myelofibrosis: Current Understanding and Clinical Patterns

EP: 2.Diagnostic Challenges in Myelofibrosis: Distinguishing Prefibrotic Disease

EP: 3.Prognostic Stratification on Myelofibrosis: From Models to Clinical Application

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EP: 4.Clinical Decision-Making Framework for Initiating Myelofibrosis Therapy

EP: 5.Early Intervention in Myelofibrosis: Interpreting Survival Evidence

EP: 6.Splenomegaly Management in Myelofibrosis: Symptomatic and Asymptomatic Disease

EP: 7.JAK Inhibitor Therapy: Personalized Selection and Optimization Strategies

EP: 8.Navigating Cytopenia Challenges: Strategic Management Approaches in Myelofibrosis

EP: 9.Novel Therapeutic Pathways in Myelofibrosis: Beyond JAK Inhibition

EP: 10.Defining Treatment Success: End points in Myelofibrosis Clinical Development

Summary for Physicians:

Clinical Decision-Making When Initiating Therapy in Myelofibrosis:

Initiating treatment for myelofibrosis (MF) requires a careful balance between disease control and quality-of-life (QOL) considerations. The decision-making process is guided by an integrated assessment of symptom burden, risk stratification, cytopenias, and patient goals.

Key Drivers of Initial Therapy:

• Symptom Burden:
• Significant constitutional symptoms (eg, fatigue, night sweats, weight loss) or splenomegaly-related discomfort are major triggers for initiating therapy.
• Patients with poor performance status due to symptom load may benefit from early intervention, typically with a JAK inhibitor.

• Risk Stratification:
• Prognostic tools such as DIPSS, MIPSS70, or GIPSS help determine overall disease risk and guide whether disease-modifying therapy or transplant evaluation should be prioritized.
• Intermediate- and high-risk patients are more likely to require treatment upfront, whereas low-risk patients may be observed if asymptomatic.

• Cytopenias:
• Anemia and thrombocytopenia influence treatment selection. For example:
• Ruxolitinib may be used with caution in patients with adequate platelets.
• Momelotinib may be preferred when anemia is a prominent feature.
• Pacritinib can be considered in patients with severe thrombocytopenia.

Balancing Disease Control and Quality of Life:

• Tailoring Therapy to Patient Goals:
• For some patients, maintaining or improving QOL through symptom relief is the primary goal, especially in older individuals or those with comorbidities.
• Others, particularly younger patients with high-risk features, may prioritize disease modification and transplant candidacy.

• JAK Inhibitor Selection:
• The choice of agent depends on both disease characteristics (eg, cytopenias, splenomegaly) and patient-specific factors such as treatment preferences, tolerability, and access.
  
• Ongoing Reassessment:
• Treatment decisions are dynamic and revisited regularly as the disease evolves or as the patient’s symptoms and goals change over time.

In summary, initiating therapy in MF involves a personalized approach, where symptom burden, disease biology, and patient values are weighed to select the most appropriate and tolerable treatment strategy—balancing clinical benefit with quality of life.