Myelofibrosis Management: Clinical Approaches and Treatment Algorithms With Case-Based Application
Panelists discuss how treatment goals for intermediate-risk myelofibrosis patients focus on achieving meaningful clinical outcomes including relieving symptoms, preventing worsening of anemia, maintaining transfusion independence, reducing symptomatic splenomegaly, and ultimately improving survival while considering patient-specific factors like age and transplant eligibility.
Summary of Myelofibrosis Treatment Approach and Case Discussion
Treatment Algorithm Overview
- Lower-risk myelofibrosis:
- Observation is appropriate for asymptomatic patients
- For symptomatic or spleen-related issues: Janus kinase (JAK) inhibitors (including pacritinib for platelets <50,000/μL), or peginterferon alfa-2a
- Higher-risk myelofibrosis with platelets >50,000/μL:
- Four JAK inhibitor options per NCCN guidelines:
- Ruxolitinib and fedratinib: Category 1 recommendation
- Momelotinib: Category 2A recommendation
- Pacritinib: Category 2B recommendation
- Continual monitoring for response or progression
- Switch to alternate JAK inhibitor if no/lost response
- Consider different therapy approach for disease progression
- Four JAK inhibitor options per NCCN guidelines:
Case Presentation: Intermediate-Risk Myelofibrosis
Patient Profile:
- 68-year-old woman with mild fatigue
- Spleen 7 cm below left costal margin
- No known comorbidities
- Labs: white blood cells 23,000/μL, hemoglobin (Hb) 9.7 g/dL, platelets 450,000/μL, <1% blasts
- JAK2 V617F mutation positive, normal karyotype
- Risk assessment: DIPSS intermediate-1 risk, MIPSS70+ version 2.0 intermediate risk
- Patient not interested in transplant
Treatment Considerations
JAK Inhibitor Selection:
- JAK inhibitor recommended for significant symptomatic splenomegaly
- Adequate platelet count (450,000/μL) allows flexibility in JAK inhibitor choice
- For Hb 9.7 g/dL: momelotinib or pacritinib may offer anemia benefits
- Ruxolitinib remains reasonable despite potential anemia concerns
Treatment Goals:
- Spleen-related:
- Reduce pain and discomfort
- Improve ability to eat full meals
- Maintain patient weight
- Anemia management:
- Maintain Hb >9.0 g/dL
- Avoid transfusion dependence
- Symptom improvement:
- Address constitutional symptoms
- Improve quality of life
- Long-term objectives:
- Prevent worsening cytopenias
- Potentially improve survival outcomes
The panel emphasized the importance of establishing clear treatment goals, monitoring response, and considering JAK inhibitor characteristics when selecting therapy for intermediate-risk myelofibrosis patients.
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