Polycythemia vera treatment algorithm 2018

Area of Science:

• Hematology
• Oncology
• Internal Medicine

Background:

• Polycythemia vera (PV) is a myeloproliferative neoplasm with a generally favorable prognosis, especially in younger patients.
• Current therapies for PV primarily aim to prevent thrombosis and manage symptoms, not to alter the disease's natural history or prolong survival.
• There is a lack of critical appraisal regarding the added value of newly approved drugs for PV.

Purpose of the Study:

• To evaluate the current management strategies for polycythemia vera.
• To discuss the limitations of current clinical trial endpoints in PV.
• To advocate for appropriate therapeutic approaches based on risk stratification.

Main Methods:

• Review of recent survival data for polycythemia vera patients.
• Analysis of current treatment paradigms and drug indications.
• Discussion of clinical trial endpoints and their relevance.

Main Results:

• Mature survival data confirm a favorable prognosis in PV patients younger than 60, with a median survival of 24 years.
• Existing drugs for PV have not demonstrated survival prolongation or alteration of the disease's natural course.
• Current clinical trial endpoints often focus on palliation rather than clinically relevant outcomes like thrombosis or survival.

Conclusions:

• Conservative management (phlebotomy and aspirin) is recommended for low-risk PV.
• Cytoreductive therapy (hydroxyurea, pegylated interferon-α, busulfan) is indicated for high-risk PV.
• JAK2 inhibitor therapy may be considered for refractory symptoms like pruritus or splenomegaly.