Where polycythemia vera treatment is heading

About 6,000 people in the US are diagnosed with polycythemia vera each year. A JAK2 gene change makes the bone marrow overproduce red blood cells, thickening the blood and raising the risk of clots, strokes and heart attacks. Most people live for decades with treatment: regular phlebotomy (blood removal), low-dose aspirin, and for higher-risk people hydroxyurea, interferon or ruxolitinib. New drugs that work through hepcidin can now keep red cells in check without blood draws.

• 82studies open or running
• 15phase 3 trials
• 281papers published this year
• 25NIH-funded projects

1. Now

   Nov 2026

   A first-in-class drug approvedHappened 76.9% no phlebotomy needed

   Rusfertide: 76.9% needed no phlebotomy in weeks 20-32 vs 32.9% on placebo. FDA approved August 2026.

   76.9%: needed no phlebotomy in weeks 20-32, vs 32.9% with placebo.

   • VERIFY trial (rusfertide)
   • REVIVE trial (rusfertide)

   A second hepcidin drug worksHappened 0 median phlebotomies

   Sapablursen cut median phlebotomies from 5 to 0 over 20 weeks.

   0: median phlebotomies in 20 weeks, down from 5.

   • Sapablursen phase 2 study

   Interferon as disease-modifying treatmentHappened 3.05 odds ratio vs phlebotomy

   Ropeginterferon roughly tripled the odds of normal blood counts compared with phlebotomy alone.

   3.05: odds ratio for complete blood-count response vs phlebotomy alone.

   • Ropeginterferon meta-analysis

2. 6 months

   by May 2027

   Rusfertide in practiceLikely

   Uptake of rusfertide and how it combines with hydroxyurea, interferon or ruxolitinib.

   • VERIFY trial (rusfertide)

3. 12 months

   by Nov 2027

   A new pill vs hydroxyureaPossible

   Italfarmaco's givinostat, an HDAC inhibitor, is compared with hydroxyurea (220 people); due July 2026, results expected.

   • GIV-IN-PV

   Blood thinners vs aspirinPossible

   AVAJAK (1,308 people) tests whether apixaban or rivaroxaban prevents clots better than aspirin; due July 2027.

   • AVAJAK

4. 3 years

   by late 2029

   Sapablursen phase 3Possible 0 median phlebotomies (phase 2)

   INTREPID (250 people) is due September 2028.

   0: median phlebotomies in 20 weeks, down from 5.

   • Sapablursen phase 2 study
   • INTREPID

   Which first treatment is bestPossible

   MITHRIDATE (586 people) compares ruxolitinib with hydroxyurea or interferon as first treatment for high-risk PV; due October 2028.

   • MITHRIDATE

5. 5 years

   by late 2031

   An LSD1 inhibitor for PVLong shot

   Merck's bomedemstat (380 people) is in phase 3; due June 2032.

   • Bomedemstat trial

The research paths

Borrowed from other cancers

Open to join now

• Needs frequent phlebotomy

  Sapablursen

  Phase 3 · 38 sites

  Locations and eligibility →

• Polycythemia vera

  Bomedemstat

  Phase 3 · 8 sites

  Locations and eligibility →

• High-risk, first treatment

  Ruxolitinib vs hydroxyurea or interferon

  Phase 3 · 55 sites

  Locations and eligibility →

• JAK2-positive MPN

  Blood thinners vs aspirin

  Phase 3 · 42 sites

  Locations and eligibility →

See every recruiting Polycythemia vera trial →

When the trials finish

Completion dates the trials registered. Results usually follow 3 to 12 months later.

What just happened

Standard treatment today

• Low risk (under 60, no clots)Phlebotomy to keep hematocrit under 45%, plus low-dose aspirin; interferon increasingly used.
• High risk (60+ or past clot)Add hydroxyurea or ropeginterferon.
• Not controlled or intolerantRuxolitinib; rusfertide to reduce phlebotomy needs.
• Progression to myelofibrosisTreated as myelofibrosis.

See Polycythemia vera trials recruiting now →