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Timely management of polycythaemia vera: considerations and pitfalls

JAKAVI® (ruxolitinib) is indicated for adult patients with PV who are resistant to or intolerant of hydroxyurea (also referred to as hydroxycarbamide in the UK).1

Treatment goals and treatment options in polycythaemia vera

The goal of polycythaemia vera (PV) treatment is comprehensive disease control2,3

PV treatment goals
  • No curative pharmacotherapies are currently available4
  • No treatments have been shown to reduce risk of transformation to leukaemia or myelofibrosis4

Timely intervention may help maintain disease control4

Given the association of thrombotic events with mortality in patients with PV, timely intervention is essential to control haematocrit level and minimise risk.5-7 Pharmacotherapy has been shown to have an impact on symptom reduction and quality of life.6,8

Despite the substantial symptom burden and risks associated with PV, there may be delays to patients receiving appropriate treatment.9

Real-world data from patients and clinicians

In a survey of 219 clinicians and 699 patients with myeloproliferative neoplasms (MPNs), including 223 patients with PV:9

    90% of patients experienced MPN related symptoms
  • 47% of clinicians were found to adopt a ‘watch and wait’ approach for >25% of patients with PV at diagnosis
  • 23% of all patients on a ‘watch and wait’ management when surveyed had a moderate-to-high symptom burden (n=10/44)

When physicians were asked about treatment:

  • 68% were likely to recommend drug treatment for patients with PV experiencing severe symptoms
  • 61% were likely to recommend drug treatment for patients with PV experiencing splenomegaly‑related symptoms

However, according to ELN guidelines, cytoreductive treatment is recommended for:5,10

  • all patients at high risk of thrombosis (≥60 years, history of thrombotic events)
  • patients at low thrombotic risk with symptomatic progressive splenomegaly, severe disease-related symptoms, persistent leukocytosis or poor tolerance to phlebotomy

Risks of untreated PV

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Survival

Median survival in patients with untreated PV is estimated as 1.5 years.11

Phlebotomy and pharmacotherapy can increase life expectancy.11,12

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Thrombosis

Uncontrolled haematocrit (>45%) due to erythrocytosis is related to an increase in risk of thrombotic events and mortality.7

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Quality of life

Uncontrolled symptoms lead to a substantial decrease in quality of life for patients with PV.9,13

Treatment options

ELN recommendations:5,10

Click to learn about each management option

Low risk treatment options
High risk treatment options

*Not all recombinant interferon alfa therapies are licensed for the treatment of PV. Please refer to the individual Summaries of Product Characteristics before prescribing.

How is response to first-line treatment monitored? 

ELN: European LeukemiaNet

Adverse events should be reported. Reporting forms and information can be found at www.mhra.gov.uk/yellowcard. Adverse events should also be reported to Novartis online through the pharmacovigilance intake (PVI) tool at www.novartis.com/report or alternatively email medinfo.uk@novartis.com or call 01276 698370

References
  1. JAKAVI® Summary of Product Characteristics. Available at: medicines.org.uk.
  2. Passamonti F. How I treat polycythemia vera. Blood 2012;120(2):275-284
  3. Benevolo G, Vassallo F et al. Polycythemia vera (PV): update on emerging treatment options. Ther Clin Risk Manag 2021;17:209-221.
  4. Fox S, Griffin L, Robinson Harris D. Polycythemia vera: rapid evidence review. Am Fam Physician 2021;103(11):680-687.
  5. Barbui T, Tefferi A et al. Philadelphia chromosome-negative classical myeloproliferative neoplasms: revised management recommendations from European LeukemiaNet. Leukemia 2018;32(5):1057-1069.
  6. Baumeister J, Chatain N et al. Progression of myeloproliferative neoplasms (MPN): diagnostic and therapeutic perspectives. Cells 2021;10(12).
  7. Griesshammer M, Kiladjian J J, Besses C. Thromboembolic events in polycythemia vera. Ann Hematol 2019;98(5):1071-1082.
  8. Vannucchi A M, Kiladjian J J et al. Ruxolitinib versus standard therapy for the treatment of polycythemia vera. N Engl J Med 2015;372(5):426-435.
  9. Harrison C N, Koschmieder S et al. The impact of myeloproliferative neoplasms (MPNs) on patient quality of life and productivity: results from the international MPN Landmark survey. Ann Hematol 2017;96(10):1653-1665.
  10. Marchetti M, Vannucchi A M et al. Appropriate management of polycythaemia vera with cytoreductive drug therapy: European LeukemiaNet 2021 recommendations. Lancet Haematol 2022;9:e301-e311.
  11. Tefferi A, Vannucchi A M, Barbui T. Polycythemia vera: historical oversights, diagnostic details, and therapeutic views. Leukemia 2021;35(12):3339-3351.
  12. Tefferi A, Rumi E et al. Survival and prognosis among 1545 patients with contemporary polycythemia vera: an international study. Leukemia 2013;27(9):1874-1881.
  13. Harrison C, Mathias J et al. UK results from the myeloproliferative neoplasms (MPN) landmark survey on the symptom, emotional and economic burden of MPN. Br J Haematol 2019;186(3):e1-e4.
  14. Michiels J J, Berneman Z et al. Platelet-mediated erythromelalgic, cerebral, ocular and coronary microvascular ischemic and thrombotic manifestations in patients with essential thrombocythemia and polycythemia vera: a distinct aspirin-responsive and coumadin-resistant arterial thrombophilia. Platelets 2006;17(8):528-544.
  15. NHS. Side effects of low-dose aspirin. Available at: nhs.uk. [Accessed August 2025].
  16. Leukemia and Lymphoma Society. Polycythemia vera facts. Available at: lls.org. [Accessed August 2025].
  17. Carobbio A, Finazzi G et al. Leukocytosis is a risk factor for thrombosis in essential thrombocythemia: interaction with treatment, standard risk factors, and Jak2 mutation status. Blood 2007;109(6)2310-2313.
  18. McMullin M F, Harrison C N et al. A guideline for the diagnosis and management of polycythaemia vera. A British Society for Haematology Guideline. Br J Haematol 2019;184(2):176-191.
  19. Hydroxyurea Summary of Product Characteristics.
  20. Ropeginterferon alfa-2b Summary of Product Characteristics.
  21. Nazha A, Gerds A T. Where to turn for second-line cytoreduction after hydroxyurea in polycythemia vera? Oncologist 2016;21(4):475-480.
  22. Busulfan Summary of Product Characteristics.
  23. Barbui T, Barosi G et al. Philadelphia-negative classical myeloproliferative neoplasms: critical concepts and management recommendations from European LeukemiaNet. J Clin Oncol 2011;29(6):761-770.