Novartis PV Nanomodules

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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:¹

5.6x risk of death 6.8x risk of progression

Risk of death¹

Risk of transformation to acute leukaemia or myelofibrosis¹

Adapted from Alvarez-Larran A et al, 2012¹

	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.¹

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)^2†

Symptom burden

Symptoms like pruritus and fatigue interfere with daily activities and are associated with depression^4-6

Treatment resistance

Median survival is 1.2 years from development of HU resistance (n=261)¹

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)^7‡

Treatment-related AEs

Serious AEs can lead to subtherapeutic dosing and discontinuation of HU treatment^8-10

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)¹

†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=0.02.²
‡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<0.0001.⁷

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

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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).¹¹

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

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
Marchioli R, Finazzi G et al. Cardiovascular events and intensity of treatment in polycythemia vera. N Engl J Med 2013;368(1):22-33
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
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
Lelonek E, Matusiak L et al. Burden of aquagenic pruritus in polycythaemia vera. Acta Derm Venereol 2018;98(2):185-190
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
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
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
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
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
JAKAVI^® Summary of Product Characteristics. Available at: www.medicines.org.uk (for GB) or www.emcmedicines.com (for NI). Accessed November 2024

Complete remission	Durable* resolution of disease-related signs, including palpable hepato-splenomegaly, large symptom improvement^† and	Durable* peripheral blood count remission HCT levels <45% without phlebotomies Platelet count ≤400 x 10⁹/L WBC count <10 x 10⁹/L and	Without progressive disease, and absence of any haemorrhagic or thrombotic event and	Bone marrow histological remission Presence of age-adjusted normocellularity and disappearance of trilinear hyperplasia Absence of >grade 1 reticulin fibrosis
Partial remission	Durable* resolution of disease-related signs, large symptoms improvement^† and	Durable* peripheral blood count remission and	Without progressive disease, and absence of any haemorrhagic or thrombotic event and	Without bone marrow histological remission (persistence of trilinear hyperplasia)
No response	Any response that does not satisfy partial remission

Timely management of polycythaemia vera: considerations and pitfalls