A 55-year-old man with polycythaemia vera (JAK2 V617F mutation, Hct 58%, splenomegaly, platelets 780 × 10⁹/L) had a prior deep vein thrombosis. Risk stratification places him in high-risk category. Which agent, beyond phlebotomy and low-dose aspirin, has shown myelosuppressive benefit and reduction in thrombotic events specifically in PV?
- A Ruxolitinib — superior to hydroxyurea in reducing haematocrit and thrombosis in the RESPONSE trial
- B Hydroxyurea — first-line cytoreductive therapy for high-risk PV based on randomised trials ✓
- C Interferon-alpha-2a — first-line for high-risk PV with superior molecular remission over hydroxyurea
- D Busulfan — first-line cytoreduction in patients aged > 50 years with high-risk PV
Explanation
Hydroxyurea is the established first-line cytoreductive therapy for high-risk PV (age ≥ 60 or prior thrombosis), demonstrated to reduce thrombotic events in the PVSG-08 trial. Ruxolitinib is a JAK1/2 inhibitor approved for PV refractory or intolerant to hydroxyurea, as shown in the RESPONSE trial (superior haematocrit control and symptom improvement vs. best available therapy). Ropeginterferon alpha-2b (PROUD-PV/CONTINUATION-PV) showed superior molecular responses but is not yet universally standard first-line. Busulfan is alkylating and leukaemogenic, used as last resort.
Reference: Harrison's Principles of Internal Medicine, 21st ed.
High-yield for: NEET PGINI-CETNExTFMGEUSMLEPLABMRCP
Written and medically reviewed by the StethoPrep medical team.