A ELN-Multicenter Study on Phenotypic Evolution and Clinical Outcomes

Not yet recruiting

• Conditions: Polycythemia Vera; Thrombocythemia

• Registration Number: NCT07203768
• Lead Sponsor: FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS

Brief 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.

Study Timeline

• Study First Submitted: 2025-09-23
• Status Verified: 2025-10
• Study First Submitted QC: 2025-10-01
• Last Update Submitted: 2025-10-01
• Study First Posted: 2025-10-02
• Last Update Posted: 2025-10-02
• Study Start: 2025-11-01
• Primary Completion: 2026-04-30
• Study Completion: 2026-07-31

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 678

Inclusion Criteria

- 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.

Exclusion Criteria

• None.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Nested case-control study	Up to the baseline	To identify proliferative risk factors for progression from ET to PV in JAK2V617F positive patients.
Comparative retrospective cohort study	Up to the baseline	To evaluate the different disease prognosis in ET patients who have progressed to PV compared to de novo PV patients.

Secondary Outcome Measures

Name	Time	Method
Nested case-control study	At the time of progression	To describe characteristics of ET patients who progressed to PV, including the timing of progression.
Comparative retrospective cohort study	At the time of progression	To compare the incidence of different outcomes (vascular events, progression to myelofibrosis, progression to acute myeloid leukaemia, tumours, death) in ET patients who progressed to PV and in de novo PV patients.

Trial Locations

Locations (25)

Mayo Clinic; 🇺🇸 Rochester, Minnesota, United States

University Hospital Halle; 🇩🇪 Halle, Germany

Hannover Medical School; 🇩🇪 Hanover, Germany

University Clinic for Hematology, Oncology, Hemostaseology and Palliative Care Johannes Wesling Medical Center; 🇩🇪 Minden, Germany

Divisione Ematologia, Fondazione IRCCS Policlinico San Matteo; 🇮🇹 Pavia, Lombardy, Italy

Divisione Ematologia, Ospedale San Bortolo; 🇮🇹 Vicenza, Veneto, Italy

A.S.O. SS. Antonio e Biagio e C.Arrigo, SC Ematologia; 🇮🇹 Alessandria, Italy

ASST Papa Giovanni XXIII, SC Ematologia; 🇮🇹 Bergamo, Italy

ASST-Spedali Civili; 🇮🇹 Brescia, Italy

Ospedale Businco; 🇮🇹 Cagliari, Italy

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