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International multicenter retrospective observational study consisting of two parts: a nested case-control study (part A) and a comparative retrospective cohort study (part B).
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:
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:
Furthermore, to have at least 5 years of follow-up, de novo PV patients must be diagnosed no later than 2020.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ET to PV patients | Patients JAK2V617F positive and who have progressed to PV by 31/12/2020. | ||
| ET patients | Patients 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. | ||
| PV de novo patients | Patients diagnosed with de novo PV (no progression from previous ET). This control group will be recruited using a 1:1 matching procedure. |
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| Measure | Description | Time Frame |
|---|---|---|
| Nested case-control study | To identify proliferative risk factors for progression from ET to PV in JAK2V617F positive patients. | Up to the baseline |
| Comparative retrospective cohort study | To evaluate the different disease prognosis in ET patients who have progressed to PV compared to de novo PV patients. | Up to the baseline |
| Measure | Description | Time Frame |
|---|---|---|
| Nested case-control study | To describe characteristics of ET patients who progressed to PV, including the timing of progression. | At the time of progression |
| Comparative retrospective cohort study |
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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:
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Nested case-control study: the prevalence of at least one of the following proliferative conditions in the control group, a neutrophil/lymphocyte ratio (N/L ratio) above the median, an LDH above the median, being JAK2V617F homozygous and having splenomegaly.
Comparative retrospective cohort study: it is known that the incidence of the composite outcome (vascular events, cancers, progression to myelofibrosis, progression to acute myeloid leukemia, death) in PV de novo patients.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| TIZIANO BARBUI, MD | Contact | +39 0352675134 | tbarbui@fondazionefrom.it | |
| FRANCESCA FENILI | Contact | sperimentazioni@fondazionefrom.it |
| Name | Affiliation | Role |
|---|---|---|
| TIZIANO BARBUI, MD | FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Mayo Clinic | Active, not recruiting | Rochester | Minnesota | 55905-0001 | United States | |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41776088 | Derived | 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. |
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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.
| At the time of progression |
| University Hospital Halle |
| Not yet recruiting |
| Halle |
| Germany |
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| Hannover Medical School | Recruiting | Hanover | Germany |
|
| University Clinic for Hematology, Oncology, Hemostaseology and Palliative Care Johannes Wesling Medical Center | Recruiting | Minden | Germany |
|
| Divisione Ematologia, Fondazione IRCCS Policlinico San Matteo | Not yet recruiting | Pavia | Lombardy | 27100 | Italy |
|
| Divisione Ematologia, Ospedale San Bortolo | Recruiting | Vicenza | Veneto | Italy |
|
| A.S.O. SS. Antonio e Biagio e C.Arrigo, SC Ematologia | Not yet recruiting | Alessandria | 15121 | Italy |
|
| ASST Papa Giovanni XXIII, SC Ematologia | Recruiting | Bergamo | Italy |
|
| Policlinico S. Orsola - Malpighi | Not yet recruiting | Bologna | Italy |
|
| ASST-Spedali Civili | Not yet recruiting | Brescia | 25123 | Italy |
|
| AOU Careggi, Divisione di Ematologia | Recruiting | Florence | Italy |
|
| ASST Grande Ospedale Metropolitano Niguarda, Divisione di Ematologia | Not yet recruiting | Milan | Italy |
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| Fondazione IRCCS Cà Grande - Op. Maggiore Policlinico | Not yet recruiting | Milan | Italy |
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| Ospedale San Raffaele | Not yet recruiting | Milan | Italy |
|
| ASST MONZA Ospedale San Gerardo Clinica Ematologica | Not yet recruiting | Monza | 20900 | Italy |
|
| Azienda Ospedaliera Universitaria Federico II di Napoli Divisione di Ematologia e Trapianti del Midollo | Not yet recruiting | Naples | 80131 | Italy |
|
| Azienda Ospedaliero Universitaria Maggiore della Carità di Novara SCDU Ematologia | Recruiting | Novara | 28100 | Italy |
|
| Presidio Ospedaliero di Pescara | Not yet recruiting | Pescara | Italy |
|
| Fondazione Policlinico Universitario Gemelli | Recruiting | Roma | Italy |
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| Umberto I Policlinico | Not yet recruiting | Roma | Italy |
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| Azienda Ospedaliera Universitaria Integrata - Ospedale Borgo Roma, Unità di Ematologia | Not yet recruiting | Verona | Italy |
|
| IDIBAPS, Hospital clinic | Recruiting | Barcelona | Spain |
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| 12 Octubre University Hospital | Recruiting | Madrid | Spain |
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| Guy's and St. Thomas' NHS Foundation Trust | Recruiting | London | United Kingdom |
|
| ID | Term |
|---|---|
| D011087 | Polycythemia Vera |
| D013922 | Thrombocytosis |
| ID | Term |
|---|---|
| D019046 | Bone Marrow Neoplasms |
| D019337 | Hematologic Neoplasms |
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
| D001855 | Bone Marrow Diseases |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D009196 | Myeloproliferative Disorders |
| D001791 | Blood Platelet Disorders |
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