RESPONSE Trial Findings: Managing Polycythemia Vera in Hydroxyurea-Inadequate Responders

Opinion
Video

Panelists discuss how ruxolitinib provides comprehensive benefits for polycythemia vera patients beyond count control, highlighting its remarkable ability to rapidly alleviate severe pruritus (often within 48 hours) and other constitutional symptoms that remain resistant to conventional therapies like hydroxyurea and interferon while also effectively managing cytokine-driven and spleen-related symptoms that significantly impact quality of life.

Summary of Ruxolitinib Benefits in Polycythemia Vera

Ruxolitinib’s Impact Beyond Hematocrit Control

  • Comprehensive disease control:
    • Effective at maintaining hematocrit <45%
    • Reduces white blood cell count
    • Controls platelet counts
    • Addresses splenomegaly
  • Symptom management:
    • Pruritus relief: Dramatic improvement in aquagenic pruritus and general itching
      • Patients report significant relief within 48 hours
      • Enables return to normal activities (eg, warm showers) previously avoided
      • This benefit is unique to ruxolitinib among available polycythemia vera therapies
    • Constitutional symptoms: Superior relief compared to other available therapies
      • Hydroxyurea, interferon, and supportive care often ineffective for symptom control
      • Cytokine-driven symptoms respond particularly well
      • Spleen-related discomfort also improves

Clinical Application

  • Ruxolitinib offers a comprehensive solution for patients with:
    • Inadequate hematocrit control on maximum hydroxyurea
    • Hydroxyurea intolerance
    • Significant symptom burden, particularly pruritus

The panel emphasized that while the RESPONSE trial data supports ruxolitinib’s efficacy in controlling objective disease parameters, its profound impact on patient quality of life through symptom relief represents an equally important benefit in clinical practice.

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