A ELN-Multicenter Study on Phenotypic Evolution and Clinical Outcomes
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Polycythemia Vera, Thrombocythemia. Basic parameters: 18 years — 100 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Germany, Italy, Spain, United Kingdom
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
Unmasking Polycythemia Vera in Long-Standing Essential Thrombocythemia: A ELN-Multicenter Study on Phenotypic Evolution and Clinical Outcomes
Overview
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.
Primary outcome measures
- Nested case-control study [Time frame: Up to the baseline]
- Comparative retrospective cohort study [Time frame: Up to the baseline]
Secondary outcome measures (2)
- Nested case-control study [Time frame: At the time of progression]
- Comparative retrospective cohort study [Time frame: At the time of progression]
Eligibility criteria
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Other
Study locations
Italy · 17 centers
- Divisione Ematologia, Fondazione IRCCS Policlinico San Matteo — Pavia
- Divisione Ematologia, Ospedale San Bortolo — Vicenza
- A.S.O. SS. Antonio e Biagio e C.Arrigo, SC Ematologia — Alessandria
- ASST Papa Giovanni XXIII, SC Ematologia — Bergamo
- Policlinico S. Orsola - Malpighi — Bologna
- ASST-Spedali Civili — Brescia
- AOU Careggi, Divisione di Ematologia — Florence
- ASST Grande Ospedale Metropolitano Niguarda, Divisione di Ematologia — Milan
- … and 9 more centers
Germany · 3 centers
- University Hospital Halle — Halle
- Hannover Medical School — Hanover
- University Clinic for Hematology, Oncology, Hemostaseology and Palliative Care Johannes We — Minden
Spain · 2 centers
- IDIBAPS, Hospital clinic — Barcelona
- 12 Octubre University Hospital — Madrid
United States · 1 center
- Mayo Clinic — Rochester
United Kingdom · 1 center
- Guy's and St. Thomas' NHS Foundation Trust — London
Publications
- Barbui T, Thiele J, Alvarez-Larran A, Rambaldi A, Vannucchi AM, Tefferi A. From essential thrombocythemia to polycythemia vera (ET-2-PV): rationale and design of a multicenter ELN study. Ann Hematol. 2026 Mar 4;105(4):158. doi: 10.1007/s00277-026-06898-1. PMID 41776088
Identifiers
NCT: NCT07203768 · ET2PV