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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

The risks of uncontrolled polycythaemia vera

Patients intolerant of or resistant to HU risk serious negative outcomes

In a retrospective study of 261 patients with PV treated with HU in Spain, HU resistance versus non-resistance was associated with a:2

5.6x risk of death 6.8x risk of progression

Risk of death2

Risk of transformation to acute leukaemia or myelofibrosis2

Adapted from Alvarez-Larran A et al, 20122

Whole population From start of HU therapy After development of HU resistance
Median survival 19 years 18 years 1.2 years

*Survival curves and HR adjusted for other relevant prognostic factors including age, sex, haematological values at diagnosis, thrombosis, haemorrhage and change in white blood cell count.2

CI: confidence interval; HR: hazard ratio

There remains a high unmet need in patients resistant or intolerant to conventional therapies:

Elevated haematocrit

Risk of death due to CV causes or major thrombosis increases 4x in patients with HCT 45%‑50% (n=365)3†

Symptom burden

Symptoms like pruritus and fatigue interfere with daily activities and are associated with depression5-7

Treatment resistance

Median survival is 1.2 years from development of HU resistance (n=261)2

Phlebotomy dependence

Patients on HU requiring ≥3 phlebotomies per year have a 3.3x increased risk of thrombosis than those requiring <3 phlebotomies (n=533)8‡

Treatment-related AEs

Serious AEs can lead to subtherapeutic dosing and discontinuation of HU treatment9-11

Uncontrolled leukocyte count

Sustained leukocytosis (>10 x109/L) despite HU treatment can increase the risk of transformation to AML or myelofibrosis and death (n=261)2

†Incidence of death from CV causes or major thrombosis: 1.1 per 100 person‑years in patients with HCT <45% (n=182); 4.4 per 100 person‑years in patients with HCT 45%-50% (n=183); HR: 2.69; 95% CI: 1.19-6.12; p=0.02.
‡Rate of thrombosis for patients receiving HU and ≥3 phlebotomies per year: 20.5% at 3 years (n=85; median number of phlebotomies 4, range 3-23); rate of thrombosis for patients receiving HU and <3 phlebotomies per year: 5.3% at 3 years (n=448); p<0.0001.8

Continue to re-test your knowledge 

AE: adverse event; AML: acute myeloid leukaemia; CV: cardiovascular; HCT: haematocrit; HU hydroxyurea; PV: polycythaemia vera

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. Alvarez-Larran A, Pereira A et al. Assessment and prognostic value of the European LeukemiaNet criteria for clinicohematologic response, resistance, and intolerance to hydroxyurea in polycythemia vera. Blood 2012;119(6):1363-1369.
  3. Marchioli R, Finazzi G et al. Cardiovascular events and intensity of treatment in polycythemia vera. N Engl J Med 2013;368(1):22-33.
  4. 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.
  5. 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.
  6. Lelonek E, Matusiak L et al. Burden of aquagenic pruritus in polycythaemia vera. Acta Derm Venereol 2018;98(2):185-190.
  7. McFarland D C, Shaffer K M et al. Associations of physical and psychologic symptom burden in patients with Philadelphia chromosome-negative myeloproliferative neoplasms. Psychosomatics 2018;59(5):472-480.
  8. Alvarez-Larran A, Perez-Encinas M et al. Risk of thrombosis according to need of phlebotomies in patients with polycythemia vera treated with hydroxyurea. Haematologica 2017;102(1):103-109.
  9. Grunwald M R, Kuter D J et al. Treatment patterns and blood counts in patients with polycythemia vera treated with hydroxyurea in the United States: an analysis from the REVEAL study. Clin Lymphoma Myeloma Leuk 2020;20(4):219-225.
  10. Najean Y, Rain J D. Treatment of polycythemia vera: the use of hydroxyurea and pipobroman in 292 patients under the age of 65 years. Blood 1997;90(9):3370-3377.
  11. Stegelmann F, Wille K et al. Significant association of cutaneous adverse events with hydroxyurea: results from a prospective non-interventional study in BCR-ABL1-negative myeloproliferative neoplasms (MPN) - on behalf of the German Study Group-MPN. Leukemia 2021;35(2):628-631.