Polycythaemia vera (PV)
Polycythaemia vera is a slow blood cancer in which a single faulty gene, JAK2, makes the bone marrow produce too many red cells. Thick blood causes clots, so treatment thins it (blood removal, aspirin) and, for higher-risk patients, calms the marrow with hydroxyurea, interferon or ruxolitinib; a hepcidin mimic, rusfertide, now controls red cell counts without regular blood removal.
Overview
Polycythaemia vera is one of the classical myeloproliferative neoplasms. Almost every case carries a mutation in JAK2 (V617F in about 95 percent, exon 12 in most of the rest) that keeps the red-cell growth signal switched on without erythropoietin. The result is a high haematocrit, often with raised platelets and white cells, an enlarged spleen, itching after warm water, burning red hands and feet, and above all a raised risk of arterial and venous thrombosis, including clots in unusual places such as the hepatic veins. Diagnosis rests on the blood count, the JAK2 mutation, a hypercellular marrow and a low erythropoietin level. Treatment is risk-adapted: everyone has low-dose aspirin and phlebotomy to keep the haematocrit under 45 percent, a target proven by the CYTO-PV trial; people over 60 or with a prior clot add a cytoreductive drug, hydroxyurea or ropeginterferon alfa-2b, with ruxolitinib for those hydroxyurea fails. Rusfertide, a hepcidin mimetic that starves red-cell production of iron, was approved in 2026 for phlebotomy-dependent disease. Over decades a minority progress to post-PV myelofibrosis and a few percent to acute leukaemia, which is why the field is now chasing molecular remission with interferon and JAK2 V617F-selective inhibitors.
State of the art
- Ropeginterferon alfa-2b is the first drug shown to shrink the JAK2 mutant clone in a randomised trial, and its long-term molecular responses raise the prospect of changing the course of the disease rather than only controlling counts.
- Ruxolitinib gives durable haematocrit and symptom control after hydroxyurea fails, and in MAJIC-PV a complete response tracked with fewer clots and progressions.
- Rusfertide, approved in 2026, is the first mechanistically new PV drug in a decade: by mimicking hepcidin it starves red-cell production of iron and made three quarters of patients phlebotomy-free in VERIFY.
- The unmet needs are a treatment that prevents progression to myelofibrosis and leukaemia, and a way to select who needs cytoreduction beyond age and clot history.
Show survival figures (1)
Averages across everyone diagnosed, often years ago. Your stage, subtype, age, fitness and the treatment you receive matter more than the average, and the numbers are improving quickly.
- Keeping the haematocrit under 45 percent cuts major clots and cardiovascular death to about a third, the single most important result in PV (CYTO-PV).
Anatomy and lymph node drainage
Leukaemias, myeloma and MDS live in the marrow and blood; lymphomas grow in lymph nodes and spleen. The node stations are the disease map, not a route of spread, and staging counts them.
- Bone marrow (leukaemia, MDS, MPN, myeloma)JAK2 V617F-positive (about 95 percent) · JAK2 exon 12-mutated (about 3 percent; often isolated erythrocytosis) · Post-PV myelofibrosis (spent phase) · PV in blast phase (transformation to acute myeloid leukaemia)
- Lymph node germinal centre (lymphomas)
- Spleen
- Blood (leukaemic phase)
- Lytic bone lesions (myeloma)
- Skin and extranodal sites
- cervical
- axillary
- mediastinal
- para-aortic and mesenteric
- inguinal
In lymphoma the node stations are the disease itself; staging (Ann Arbor / Lugano) counts how many regions and sides of the diaphragm are involved.
Same organ: Acute myeloid leukaemia, Acute lymphoblastic leukaemia, Chronic lymphocytic leukaemia, Chronic myeloid leukaemia (CML), Diffuse large B-cell lymphoma, Follicular lymphoma, Hodgkin lymphoma, Mantle cell lymphoma, Multiple myeloma, Non-Hodgkin lymphoma (all types), Myelodysplastic syndromes / neoplasms (MDS), Myeloproliferative neoplasms (PV, ET, myelofibrosis), Essential thrombocythaemia (ET), Waldenström macroglobulinaemia, Hairy cell leukaemia, Peripheral T-cell lymphomas (including cutaneous T-cell lymphoma), Blastic plasmacytoid dendritic cell neoplasm (BPDCN), Burkitt lymphoma, HIV-associated (AIDS-related) lymphomas, Chronic myelomonocytic leukaemia and MDS/MPN overlap neoplasms, Systemic mastocytosis, Erdheim-Chester disease, Rosai-Dorfman disease and other histiocytic neoplasms, Langerhans cell histiocytosis (LCH), Post-transplant lymphoproliferative disorder (PTLD)
Around one to two new cases per 100,000 people a year, most diagnosed in their sixties; with treatment most people live for decades, and the main dangers are clots, bleeding and, late on, scarring of the marrow or leukaemia.
- AI in radiologyEstablished
- cfDNA fragmentomicsEstablished
- Colorectal cancer screening (colonoscopy, FIT, stool DNA, blood)Standard of care
- DNA methylation profilingEstablished
- HCC surveillance in cirrhosis (ultrasound + AFP)Standard of care
- High-risk pancreatic surveillance (CAPS / PRECEDE)Established
- A 28-day national pathway for people with a positive multi-cancer blood test
- A breath test to rule out cancer in people with vague symptoms
- A cancer blood test for older people arriving at A&E with unexplained symptoms
- A legislated, publicly reported 28-day standard from urgent referral to diagnosis
- A live national dashboard of stage at diagnosis as the scorecard for early detection
- A single 'cancer check at 60' appointment bundling all screening tests
Background: Alpha-fetoprotein (AFP), Barrett's oesophagus, CA 19-9, Early detection, Faecal immunochemical test (FIT). Also on OnCo: Symptoms and red flags · Early detection roadmap.
Cases by country
No country-level case numbers. This cancer is not mapped to a GLOBOCAN site.
Full blood count, JAK2 V617F and exon 12 testing, serum erythropoietin, and bone marrow biopsy showing trilineage growth; WHO criteria (haemoglobin above 16.5 g/dL in men or 16.0 in women, or haematocrit above 49 or 48 percent). Secondary causes of a high red count (smoking, sleep apnoea, kidney or liver tumours, testosterone) are excluded first.
Low-dose aspirin unless contraindicated, phlebotomy to a haematocrit under 45 percent (CYTO-PV), and control of cardiovascular risk factors.
Aspirin and phlebotomy alone; ropeginterferon alfa-2b is an option when phlebotomy is poorly tolerated or symptoms persist (Low-PV).
Add cytoreduction: hydroxyurea, or ropeginterferon alfa-2b (PROUD-PV and CONTINUATION-PV), the latter preferred in younger patients and in pregnancy.
Ruxolitinib (RESPONSE, RESPONSE-2, MAJIC-PV) for haematocrit control, spleen shrinkage and symptom relief; interferon if not already tried.
Rusfertide, a hepcidin mimetic given by weekly injection, keeps the haematocrit under 45 percent and removes the need for phlebotomy in most patients (VERIFY).
Antihistamines, aspirin for erythromelalgia, interferon or ruxolitinib for severe aquagenic pruritus.
Managed as myelofibrosis: JAK inhibitors for spleen and symptoms, momelotinib for anaemia, allogeneic stem cell transplant for fit higher-risk patients.
Subtypes & biomarkers
top- JAK2 V617F-positive (about 95 percent)
- JAK2 exon 12-mutated (about 3 percent; often isolated erythrocytosis)
- Masked PV (haemoglobin below the WHO threshold but marrow and mutation typical)
- Post-PV myelofibrosis (spent phase)
- PV in blast phase (transformation to acute myeloid leukaemia)
- JAK2 V617F allele burden (falls with interferon; a marker of molecular response)
- JAK2 exon 12 mutations
- Haematocrit, with treatment targeting below 45 percent
- Serum erythropoietin (low in PV, high in secondary erythrocytosis)
- Leukocyte count above 11 x 10^9/L (thrombosis risk)
- Additional mutations in TET2, ASXL1, SRSF2, IDH1/2 (progression risk)
- Age over 60 and prior thrombosis (the two risk factors that define high-risk disease)
How often this target appears
- 1892Vaquez describes the disease
Louis Henri Vaquez reports a patient with persistent excess red cells and an enlarged spleen.
- 1903Osler defines a clinical syndrome
William Osler collects cases of chronic cyanosis with polycythaemia and splenomegaly.
- 1951Dameshek groups the myeloproliferative disorders
PV, essential thrombocythaemia, myelofibrosis and CML proposed as related diseases of the marrow.
- 1967Polycythemia Vera Study Group founded
Louis Wasserman's cooperative group runs the first randomised PV trials, comparing phlebotomy, radiophosphorus and chlorambucil, and shows the leukaemia risk of alkylating agents.
- 2005JAK2 V617F discovered
Four groups report the mutation in almost all PV; the first molecular marker for BCR-ABL1-negative MPN and the target for the JAK inhibitors that followed.
- 2013CYTO-PV proves the haematocrit target
Keeping the haematocrit under 45 percent gives fewer cardiovascular deaths and major clots than a 45 to 50 percent target.
- 2014Ruxolitinib approved for PV
FDA approval in December 2014 for hydroxyurea-resistant or intolerant PV on the RESPONSE trial; EU approval followed in 2015.
- 2019Ropeginterferon alfa-2b approved in Europe
The first interferon licensed for PV, on PROUD-PV and CONTINUATION-PV; FDA approval followed in November 2021.
- 2021Low-PV: interferon helps low-risk patients too
Ropeginterferon plus phlebotomy kept more low-risk patients at target than phlebotomy alone.
- 2023MAJIC-PV links complete response to fewer events
Ruxolitinib beat best available therapy after hydroxyurea failure, and complete responders had better event-free survival.
- 2025VERIFY: a hepcidin mimetic replaces phlebotomy
Rusfertide added to standard care made most patients phlebotomy-free over weeks 20 to 32.
- 2026Rusfertide approved
FDA approval for phlebotomy-dependent polycythaemia vera.
What is in development for Polycythaemia vera (PV), drawn from the whole corpus: 12 items. Drugs are grouped by the most advanced trial phase they have reached anywhere; approved treatments sit under standard of care. Technologies are the methods being tested for this cancer, trials are the studies recorded here, and ideas are proposals not yet in a trial.
Drugs in phase 3 · 2
Trials under way · 1
- Study on Efficacy and Safety of Givinostat Versus Hydroxyurea in Patients With Polycythemia Vera · phase 3 · Italfarmaco
Trials reported · 7
- CYTO-PV · phase 3 · 2013 · positive
- Low-PV · phase 2 · 2021 · positive
- MAJIC-PV · phase 2 · 2023 · positive
- PROUD-PV and CONTINUATION-PV · phase 3 · 2020 · positive
- RESPONSE · phase 3 · 2015 · positive
- RESPONSE-2 · phase 3 · 2017 · positive
- VERIFY · phase 3 · 2025 · positive
Ideas not yet in a trial · 2
Open problems and what is being done
No treatment has yet been shown to prevent progression to myelofibrosis or leukaemia; interferon's molecular responses are the strongest hint.
Risk stratification still rests on age and clot history; leukocyte count, allele burden and additional mutations are not yet built into treatment decisions.
Whether low-risk patients should have early cytoreduction (Low-PV suggests yes for interferon) remains unsettled and depends on cost and tolerability.
and how the field plans to fix it →What is being done about thisCost and accessAvailable now- HPV & HBV vaccinationStandard of care
- ImatinibApproved
- Trastuzumab biosimilarsApproved
In trials- Gefitinib vs gefitinib plus pemetrexed-carboplatin in EGFR-mutant lung cancer (Tata Memorial)Positive
- IARC India HPV vaccine dose study (one, two or three doses)Positive
- IMAGINE (varnimcabtagene autoleucel, Immuneel)Positive
- Low-dose nivolumab plus metronomic chemotherapy (Tata Memorial)Positive
- Low-dose olanzapine for cancer anorexia (Tata Memorial)Positive
- METRO PLUS (Tata Memorial Centre, Varanasi)Positive
Ideas and roadmaps- 90-day reliance approval for cancer drugs cleared by two stringent regulators
- A cheap old tablet to restore appetite
- A combination pricing rule so two-drug regimens are not priced as two monopolies
- A coordinated reserve and shared schedule for the world's medical isotope reactors
- A dedicated global financing window for cancer, modelled on the Global Fund
- A delinked market-entry reward paid by payers when a repurposed generic wins approval
Background: Accelerated approval, Biosimilar, Real-world evidence. Also on OnCo: Financial help · Coverage by country · HTA decisions.
Aquagenic pruritus and fatigue are under-treated and poorly measured in trials.
and how the field plans to fix it →What is being done about thisSide effects and quality of lifeAvailable now- Cardio-oncologyEstablished
- Exercise & lifestyle oncologyEstablished
- Geriatric assessmentEstablished
- IMRT / IGRT (modern external beam)Standard of care
- Oncology nutrition assessment and medical nutrition therapyEstablished
- Proton therapyEstablished
In trialsIdeas and roadmaps- A cheap old tablet to restore appetite
- A coordinated FLASH radiotherapy evidence programme with shared dose-rate standards
- A dedicated programme for cachexia and treatment toxicity research
- A dietitian in every gastrointestinal and head and neck tumour board
- A funded programme of organ-preservation trials to avoid radical surgery
- A lifelong late-effects registry linked to every treatment for adult survivors
Background: CTCAE toxicity grading (grade 3-4 adverse events), De-escalation, escalation and response-adapted therapy, Immune-related adverse events (irAEs), Quality of life, Toxicity grade. Also on OnCo: Side effects by symptom · Immune-related side effects · Toxicity compare · Survivorship planner.
Hepcidin mimetics control counts but their effect on thrombosis and long-term outcomes is not yet known.
Trials
topTrials recruiting now
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Landmark trials
Expert centres
topExpert centres
- via Ruxolitinib, Myeloproliferative neoplasms (PV, ET, myelofibrosis)
Questions to ask
topQuestions to ask your oncologist about Polycythaemia vera
Newly diagnosed
- What is my exact diagnosis, stage, and grade, and which tests established them?Why: Everything else follows from an accurate stage and subtype.
- Which biomarkers have been tested on my tumour (for example JAK2 V617F allele burden, JAK2 exon 12 mutations, Haematocrit, with treatment targeting below 45 percent, Serum erythropoietin, Leukocyte count above 11 x 10^9/L), and what were the results?Why: These results decide eligibility for targeted therapy, immunotherapy, and trials.
- Which subtype is my cancer, and does that change the recommended treatment?Why: Recognised subtypes for this cancer include JAK2 V617F-positive, JAK2 exon 12-mutated, Masked PV.
- Is germline (inherited) genetic testing recommended for me or my family?Why: Inherited variants can change treatment and matter for relatives.
Diagnosis
- For my situation (diagnosis), which of the standard options do you recommend and why?Why: Guideline options include: Full blood count, JAK2 V617F and exon 12 testing, serum erythropoietin, and bone marrow biopsy showing trilineage growth; WHO criteria (haemoglobin above 16.5 g/dL in men or 16.0 in women, or haematocrit above 49 or 48 percent). Secondary causes of a high red count (smoking, sleep apnoea, kidney or liver tumours, testosterone) are excluded first.
All patients
- For my situation (all patients), which of the standard options do you recommend and why?Why: Guideline options include: Low-dose aspirin unless contraindicated, phlebotomy to a haematocrit under 45 percent (CYTO-PV), and control of cardiovascular risk factors.
- Am I a candidate for Aspirin, and what side effects should I expect?Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
- How do the results of CYTO-PV apply to someone like me?Why: Trial populations differ from individual patients; ask how closely you match.
Low risk (under 60, no prior clot)
- For my situation (low risk (under 60, no prior clot)), which of the standard options do you recommend and why?Why: Guideline options include: Aspirin and phlebotomy alone; ropeginterferon alfa-2b is an option when phlebotomy is poorly tolerated or symptoms persist (Low-PV).
- Am I a candidate for Ropeginterferon alfa-2b, and what side effects should I expect?Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
- How do the results of Low-PV apply to someone like me?Why: Trial populations differ from individual patients; ask how closely you match.
High risk (over 60 or prior clot)
- For my situation (high risk (over 60 or prior clot)), which of the standard options do you recommend and why?Why: Guideline options include: Add cytoreduction: hydroxyurea, or ropeginterferon alfa-2b (PROUD-PV and CONTINUATION-PV), the latter preferred in younger patients and in pregnancy.
- Am I a candidate for Hydroxyurea (hydroxycarbamide), Ropeginterferon alfa-2b, and what side effects should I expect?Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
- How do the results of PROUD-PV and CONTINUATION-PV apply to someone like me?Why: Trial populations differ from individual patients; ask how closely you match.
Hydroxyurea resistance or intolerance
- For my situation (hydroxyurea resistance or intolerance), which of the standard options do you recommend and why?Why: Guideline options include: Ruxolitinib (RESPONSE, RESPONSE-2, MAJIC-PV) for haematocrit control, spleen shrinkage and symptom relief; interferon if not already tried.
- Am I a candidate for Ruxolitinib, and what side effects should I expect?Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
- How do the results of RESPONSE and RESPONSE-2 apply to someone like me?Why: Trial populations differ from individual patients; ask how closely you match.
Phlebotomy-dependent disease
- For my situation (phlebotomy-dependent disease), which of the standard options do you recommend and why?Why: Guideline options include: Rusfertide, a hepcidin mimetic given by weekly injection, keeps the haematocrit under 45 percent and removes the need for phlebotomy in most patients (VERIFY).
- Am I a candidate for Rusfertide, and what side effects should I expect?Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
- How do the results of VERIFY apply to someone like me?Why: Trial populations differ from individual patients; ask how closely you match.
Itching and burning extremities
- For my situation (itching and burning extremities), which of the standard options do you recommend and why?Why: Guideline options include: Antihistamines, aspirin for erythromelalgia, interferon or ruxolitinib for severe aquagenic pruritus.
Post-PV myelofibrosis
- For my situation (post-pv myelofibrosis), which of the standard options do you recommend and why?Why: Guideline options include: Managed as myelofibrosis: JAK inhibitors for spleen and symptoms, momelotinib for anaemia, allogeneic stem cell transplant for fit higher-risk patients.
- Am I a candidate for Ruxolitinib, Momelotinib, and what side effects should I expect?Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
Any stage
- Are there clinical trials I could join, for example of Givinostat, Bomedemstat, Study on Efficacy and Safety of Givinostat Versus Hydroxyurea in Patients With Polycythemia Vera, Clearing the JAK2 clone in polycythaemia vera: interferon plus mutant-selective inhibitors as a route to treatment-free remission?Why: Trials are how the next standard of care is set; asking early keeps options open.
- Would a second opinion at a high-volume centre change anything, and can you help arrange it?Why: Rare or high-stakes decisions benefit from a centre that treats many similar patients.
- What supportive care (symptom control, nutrition, exercise, mental health, financial help) is available from the start?Why: Supportive care improves quality of life and helps patients complete treatment.
- I read that “No treatment has yet been shown to prevent progression to myelofibrosis or leukaemia; interferon's molecular responses are the strongest hint”. How does that affect my plan?Why: Open problems are where trials and second opinions matter most.
- I read that “Risk stratification still rests on age and clot history; leukocyte count, allele burden and additional mutations are not yet built into treatment decisions”. How does that affect my plan?Why: Open problems are where trials and second opinions matter most.
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Direct links plus the targets, companies, and technologies of this cancer's products.
technologies
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2drugs
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8ideas
2Latest papers
topQuery for this cancer: (TITLE:"Polycythaemia vera" OR ABSTRACT:"Polycythaemia vera" OR TITLE:"Polycythemia vera" OR ABSTRACT:"Polycythemia vera" OR TITLE:"Primary polycythaemia" OR ABSTRACT:"Primary polycythaemia" OR TITLE:"Vaquez disease" OR ABSTRACT:"Vaquez disease" OR TITLE:"Vaquez-Osler disease" OR ABSTRACT:"Vaquez-Osler disease") AND (treatment OR therapy OR trial OR survival OR diagnosis). Results are unfiltered search hits about Polycythaemia vera (PV), not a curated reading list.
Similar pages
not linked directly; found by shared links- CancerEssential thrombocythaemia (ET)
Shares Erythromelalgia, Aspirin, Post-PV myelofibrosis (spent phase), JAK2 V617F and the tag mpn.
- TrialPT-1 (Primary Thrombocythaemia 1)
Shares Hydroxyurea (hydroxycarbamide), Myeloproliferative neoplasms (PV, ET, myelofibrosis) and the tag mpn.
- TrialMAJIC-ET
Shares Ruxolitinib and the tag mpn.
- PersonClaire Harrison
Shares Myeloproliferative neoplasms (PV, ET, myelofibrosis) and the tag mpn.
- CompanyItalfarmaco
Shares Study on Efficacy and Safety of Givinostat Versus Hydroxyurea in Patients With Polycythemia Vera, Givinostat, Myeloproliferative neoplasms (PV, ET, myelofibrosis).
- Key paperMOMENTUM: momelotinib versus danazol for myelofibrosis patients with anaemia after a prior JAK inhibitor
Shares Momelotinib, JAK2, Ruxolitinib, Myeloproliferative neoplasms (PV, ET, myelofibrosis).
- Key paperCOMFORT-I: ruxolitinib, the first JAK inhibitor, versus placebo for myelofibrosis
Shares Momelotinib, JAK2, Ruxolitinib, Myeloproliferative neoplasms (PV, ET, myelofibrosis).
- TreatmentPipobroman
Shares Hydroxyurea (hydroxycarbamide), Ruxolitinib, Myeloproliferative neoplasms (PV, ET, myelofibrosis).