Considerations in Choosing a Treatment for Myelofibrosis

Video

An expert describes the factors he considers when choosing a treatment for a patient with myelofibrosis, and how he judges treatment response.

Case: A 76-Year-Old Woman with Myelofibrosis

Initial presentation

  • SM is a 76 y/o woman who visits her primary care physician for her annual checkup. She reports having fatigue, occasional night sweats and abdominal discomfort under left rib for the past 2 months. She also mentioned that she feels pain in her bones, but she just attributes that to her “old age.”
    • SM spends most of her days gardening followed by baking, her 2 favorite activities. Lately, she feels tired after just spending 1 hour gardening and has no energy to bake afterwards.
    • Since her last examination a year ago, SM has lost close to 12 lbs. She says she has not been dieting and has not noticed a change in her eating habits.
    • PMH:
      • Hypertension (controlled)
      • Glaucoma
      • Asthma (controlled with inhalers)
    • SMH: (-) nonsmoker; has an occasional drink during book club with her girlfriend
    • FH: she lives alone but her daughter and sister live close by; no family history of cancer
    • PE: abdominal exam reveals spleen palpable 4cm below left coastal margin visible bruising

Clinical workup

  • Current Labs: (baseline labs were within normal limits at her last examination one year ago)
    • Platelet count: 44 x 109/L
    • Hgb: 8g/dL
    • WBC count: 27 x 109/L
  • Bone Marrow Biopsy
    • Shows a hypercellular bone marrow, increase in atypical appearing megakaryocytes and MF-2 fibrosis, no increase in blasts by CD34+ IHC, aspirate without spicules
  • Molecular testing
    • JAK-V617F mutation: positive at 44%, NGS panel only identified TET2 mutation at a VAF of 17%

Treatment

  • SM complaining of increasing fatigue and abdominal pain and on PE, shows splenomegaly
  • Treatment started with pacritinib 200mg BID
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