Addressing Current Challenges and Unmet Needs in Myelofibrosis

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John Mascarenhas, MD, looks to the future of myelofibrosis treatment, noting present challenges and treatment strategies in development that may address those unmet needs.

Case: A 62-Year-Old Man with Myelofibrosis

Clinical Presentation:

  • A 62-year-old man presented with symptoms of fatigue, night sweats, and increased bruising.
  • PMH: type 2 diabetes, hypercholesteremia, and hypertension
  • 1 year prior, patient had a “normal” physical and blood work.
    • PCP noticed lower Hg (11 to 9.5 g/dL) and Plt (350 to 195 cells/m3)
  • Patient was referred to Hem/Onc – first available appoint in 2 months.

Initial Clinical Workup and Diagnosis (Post-PCP Visit):

  • Exam: fatigue and night sweats worsening; bone pain.
  • Labs: RBC 3.40 x 1012/L; Hb 8.7 g/dL; Hct 36%; MCV 94fL; WBC 28.0 x 109/L; Plt 75 x 109/; LDH 330 IU/L
  • Spleen: 5 cm below LCM
  • Bone Marrow Fibrosis: Grade 2
  • Bone Marrow Blasts: 3%
  • Blood Smear: leukoerythroblastosis; 1% blasts by manual count/flow cytometry
  • Molecular Analysis: JAK V617F+
  • ECOG PS 2
  • Diagnosis: Primary MF

Current Treatments:

  • Patient was initiated on pacritinib.
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