RESPONSE Trial and Hydroxyurea Resistance


Dr. Kuykendall comments on the high rates of hematocrit control and spleen response with ruxolitinib vs standard therapy in the RESPONSE trial for hydroxyurea-resistant/intolerant PV, and how these efficacy findings inform his treatment choices.

Case: Management of Advanced Polycythemia Vera in a 67-Year-Old-Male

Clinical Presentation:

  • 67-year-old male presented with frequent headache and dizziness
  • Prior medical history is notable for smoking.
  • PMH: unremarkable, no splenomegaly

Initial Clinical Workup and Diagnosis:

  • Labs:
    • Hemoglobin, 20.5 g/dL
    • White blood cell count, 13 K/L
    • Platelet count, 380 K/L
    • Hematocrit, 68%
  • JAK2-V617F mutation- variant allele frequency of 65%
  • Bone marrow biopsy: trilineage proliferation and pleiomorphic megakaryocytes
  • The patient started phlebotomy, aspirin, and Hydroxyurea (HU)

Initial Treatments:

  • The patient started phlebotomy, aspirin and HU.
    • Over the next year the patient underwent several phlebotomies and remained on aspirin and 2000 mg/day of HU

Disease Progression and Subsequent Treatments:

February 2019

  • 2 phlebotomies since the last visit 3 months earlier
  • He is also experiencing fatigue and night sweats.
  • Labs
    • Hgb, 15.5 g/dL
    • WBC, 13 K/L
    • Platelet count, 380 K/L
    • Hematocrit, 47%
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