All PV therapies may be associated with treatment-related effects. It is important for patients to understand the difference between manageable effects and those that may warrant medical evaluation [2][5].

Treatment-related effects that have been reported with various PV therapies may include:

• Hematologic parameters requiring monitoring

• Dermatologic manifestations

• Gastrointestinal effects

• Constitutional symptoms

Important: This information is not intended to discourage the use of any particular therapy. All PV treatments have associated benefit-risk profiles. Patients should report any new or concerning symptoms to their healthcare provider for proper evaluation.

The European LeukemiaNet (ELN) has published criteria to help standardize the assessment of response to cytoreductive therapy in PV. These criteria provide a framework for clinical decision-making [4].

According to published literature, various scenarios have been described in which treatment modifications may be considered. However, the application of these criteria requires individualized clinical judgment [4][5].

Note: The presence of any specific finding does not automatically indicate a need for treatment change. Clinical context, severity, persistence, and impact on disease control must all be considered by the treating physician

Multiple studies have examined treatment patterns and outcomes in PV patients receiving various therapies. These observational studies provide context for understanding real-world clinical practice [2][3][4][6][7].

Published literature suggests variability in treatment response and tolerability across patient populations. Healthcare utilization patterns have also been examined in some studies [3][6][7].

Important: Study results may not be generalizable to all patients or clinical settings. Treatment decisions should be based on individual patient assessment, not population-level data alone.

Patients should maintain regular communication with their healthcare team. Specific situations that may warrant discussion include [1][4]:

• Changes in symptoms or new symptoms

• Difficulty maintaining prescribed treatment regimen

• Laboratory values outside target ranges

• Concerns about current treatment

• Interest in understanding all available treatment options

Multiple therapeutic options are available for the management of PV. Treatment selection should be individualized based on patient-specific factors [1].

Treatment modalities that may be considered include:

Phlebotomy: May be used alone or in combination with other therapies

Cytoreductive therapies: Multiple agents are available with different mechanisms of action and administration schedules

Aspirin: May be considered for cardiovascular risk management in appropriate patients

Supportive care measures: Including management of symptoms and complications

Other Treatment Options

Additional therapeutic options exist and may be appropriate depending on individual clinical circumstances. Patients should discuss all available options with their hematologist [1].

Treatment decisions in PV should involve collaborative discussion between patients and their healthcare team. Factors to consider include [1]:

• Individual treatment goals and priorities

• Risk stratification and disease characteristics

• Comorbidities and other medications

• Administration schedule and practical considerations

• Benefit-risk profile of each treatment option

• Patient preferences and quality of life considerations

Regardless of the treatment approach, regular monitoring is essential in PV management. Monitoring parameters may include [1]:

• Complete blood counts

• Hematocrit levels

• Symptom assessment

• Evaluation for treatment-related effects

• Assessment for disease-related complications

Your healthcare provider will determine the appropriate monitoring schedule based on your individual situation.

• What are my current treatment goals?

• What treatment options are available for my specific situation?

• What are the potential benefits and risks of each treatment option?

• What monitoring will be required with different treatment approaches?

• What symptoms or changes should prompt me to contact you?

• How do we know if my current treatment is working effectively?

• What factors should we consider when evaluating treatment options?

• Are there clinical trials available that might be appropriate for me?

1. Kuykendall AT. Treatment of hydroxyurea-resistant/intolerant polycythemia vera: a discussion of best practices. Ann Hematol. 2023;102(5):985-993.

2. Demuynck T, Verhoef G, Delforge M, Vandenberghe P, Devos T. Polycythemia vera and hydroxyurea resistance/intolerance: a monocentric retrospective analysis. Ann Hematol. 2019;98(6):1421-1426.

3. Chiaranairungrot K, et al. Prevalence and clinical outcomes of polycythemia vera and essential thrombocythemia with hydroxyurea resistance or intolerance. Hematology. 2022;27(1):813-819.

4. Alvarez-Larrán 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.

5. Lebowa WM, Ignatowicz A, Ząber J, Sacha T. Current status of hydroxyurea in treatment of polycythemia vera. Acta Haematol Pol. 2024;55(3):145-150.

6. Manuel L, Milligan G, Graham A, Taylor-Stokes G. Assessing the level of resistance/intolerance to hydroxyurea therapy amongst patients with polycythemia vera in Europe. Value Health. 2015;18(7):A436.

7. Ellis MH, et al. Clinical and Economic Implications of Hydroxyurea Intolerance in Polycythemia Vera in Routine Clinical Practice. J Clin Med. 2024;13(12):3390.

8. Gisslinger H, et al. Polycythemia Vera Patients Respond Better to Ropeginterferon Alfa-2b Than HU/BAT Irrespective of Pretreatment or Mutational Status; Results from 5 Years' Treatment in a Randomized, Controlled Setting in the PROUD-PV/Continuation-PV Trials. Blood. 2021;138:3660.

9. Gisslinger H, et al. Event-free survival in patients with polycythemia vera treated with ropeginterferon alfa-2b versus best available treatment. Leukemia. 2023;37(10):2129-2132.