NCT07203768

A ELN-Multicenter Study on Phenotypic Evolution and Clinical Outcomes

Unmasking Polycythemia Vera in Long-Standing Essential Thrombocythemia: A ELN-Multicenter Study on Phenotypic Evolution and Clinical Outcomes

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 678 (estimated)
• Sponsor: FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS · Academic / Other
• Sex: All
• Age: 18 Years – 100 Years
• Healthy volunteers: Not accepted

Summary

International multicenter retrospective observational study consisting of two parts: a nested case-control study (part A) and a comparative retrospective cohort study (part B).

Detailed description

The study will include ET patients (diagnosed according to "International consensus classification of myeloid neoplasms and acute leukemias - 2022" criteria) who are JAK2V617F positive and have progressed to PV (diagnosed according to "International consensus classification of myeloid neoplasms and acute leukemias - 2022" criteria) by 31/12/2020. The date of PV diagnosis will be defined as the index date and this group of patients will be defined as ET-to-PV cases. Part A - Nested case-control study Controls for ET-to-PV cases will be patients diagnosed with ET, according to the "International consensus classification of myeloid neoplasms and acute leukemias - 2022" criteria, JAK2V617F positive who did not progress to PV after a comparable period of time. This control group will be selected using a 1:1 matched case-control design. For each patient who progressed to PV (case), a control will be selected with the following characteristics: * year of ET diagnosis (+/-2 years as compared to the date of the case); * age at ET diagnosis (+/-3 years as compared to the age of the case); * duration of ET disease (+/-2 years as compared to the period between the diagnosis of ET and the index date of the respective case). Part B - Comparative retrospective cohort study Patients diagnosed with de novo PV (no progression from previous ET) according to the "International consensus classification of myeloid neoplasms and acute leukemias - 2022" data criteria, will be recruited using a 1:1 matching procedure according to the following characteristics: * year of PV diagnosis (+/-2 years as compared to the date of the case); * age at PV diagnosis (+/-3 years as compared to the age of the case); * duration of PV disease (+/-2 years as compared to the period between PV diagnosis and the last follow-up of the respective case). Furthermore, to have at least 5 years of follow-up, de novo PV patients must be diagnosed no later than 2020.

Conditions

• Polycythemia Vera
• Thrombocythemia

Timeline

• Start date: 2025-12-01
• Primary completion: 2026-04-30
• Completion: 2026-07-31
• First posted: 2025-10-02
• Last updated: 2025-12-26

Locations

25 sites across 5 countries: United States, Germany, Italy, Spain, United Kingdom