HU-F-AIM - A Prospective, Interventional Study to Evaluate HU-resistance in Polycythemia Vera Patients Who Meet Predictive Parameters Identified in the Machine Learning Project PV-AIM
The purpose of this study is to confirm the predictive factors for hydroxyurea (HU) failure (hemoglobin (HGB) \<15.5 g/dL (9.62 mmol/L) and red blood cell distribution width (RDW) ≥17%) identified by machine learning in the polycythemia vera advanced integrated model (PV-AIM) project in the real-life setting.
• Signed informed consent must be obtained prior to participation in the study
• Patients ≥18 years
• Confirmed diagnosis of Polycythemia vera (according to WHO 2008, 2016, or 2022 criteria) (Tefferi and Vardiman 2008, Arber et al 2016, Khoury et al 2022)
• Eastern Cooperative Oncology Group (ECOG) ≤ 2
• No previous pharmacologic cytoreductive therapy (including investigational drugs)
• No phlebotomy in last 14 days
• HU-eligible
‣ High-risk: age ≥ 60 years and/or prior history of thrombosis
⁃ Low-risk: showing at least one of the defined criteria
• Signs of disease progression (myeloproliferation):
‣ Increase in spleen size or symptomatic splenomegaly
⁃ Platelet increase to \> 1,000,000/µl
⁃ WBC increase to \> 15,000/µl or higher
⁃ Frequent (\> 10 per year) or increasing frequency of phlebotomies
∙ Increasing risk of thromboembolism and bleeding:
‣ New thromboembolism and/or hemorrhagic complications
⁃ Microcirculation disorders despite acetyl salicylic acid (ASA) 2x 100 mg/day
⁃ Restricted feasibility or intolerance of phlebotomies
⁃ Symptomatic iron deficiency
⁃ Uncontrolled increase in hematocrit
⁃ Severe or distressing disease-related symptoms
• Female participants of childbearing potential should have a negative serum pregnancy test within 72 hours prior to receiving the first dose of study treatment.