Impact of ELN clinical signs and symptoms on the thrombotic risk in polycythemia vera patients treated with front-line hydroxyurea
The European LeukemiaNet recently proposed specific Clinical Signs and Symptoms (CSSs) that may trigger cytoreduction in patients with polycythemia vera (PV) at low thrombotic risk (LR). To evaluate the impact of CSSs on the thrombotic risk of patients at LR, high risk by age only (HR-AGE) or by previous thrombosis (HR-THRO), we conducted a multicenter cooperative study (NCT06134102) involving 739 PV patients treated with first-line hydroxyurea. At hydroxyurea start, 443 patients had at least one CSS. In patients with and without CSSs, the incidence rate ratio of thrombosis was 2.2 and 0.7 per 100 patient-years, respectively (p < 0.001), and the thrombosis-free survival (TFS) adjusted for delayed entry at 5 years was 88.7% and 96.1% (p < 0.001). The best 5-years TFS was observed in LR and HR-AGE with no CSSs (LR, 100%; HR-AGE: 98.1%). LR, HR-AGE patients with CSSs and HR-THRO patients without CSSs had comparable TFS (89.2%, 92.1% and 88.8%, respectively). TFS of HR-THRO patients was 80.2%. In multivariate analysis including each CSS, inadequate hematocrit control (HR: 2.32, p < 0.001), relevant CVRFs (HR: 2.87, p = 0.006), progressive splenomegaly (HR: 4.02, p = 0.03) and previous thrombosis (HR: 3.76, p < 0.001) remained significantly associated with thrombotic risk. CSSs identify an increased thrombotic risk phenotype across all risk categories.
Authors
- Fabrizio Cavalca (ORCID: https://orcid.org/0000-0002-5819-9476)
- Francesca Palandri (ORCID: https://orcid.org/0000-0001-8367-5668)
- Roberto Latagliata (ORCID: https://orcid.org/0000-0002-7741-862X)
- Alessia Tieghi (ORCID: https://orcid.org/0000-0002-6911-4095)
- Mario Tiribelli (ORCID: https://orcid.org/0000-0001-9449-2621)
- Mirko Farina (ORCID: https://orcid.org/0000-0002-2980-9028)
- Alessandra D’Addio
- Elena Rossi (ORCID: https://orcid.org/0000-0001-9152-435X)
- Valerio De Stefano (ORCID: https://orcid.org/0000-0002-5178-5827)
- Giuseppe A. Palumbo (ORCID: https://orcid.org/0000-0003-1859-6319)
- Erika Morsia (ORCID: https://orcid.org/0000-0003-4453-6985)
- Massimo Breccia (ORCID: https://orcid.org/0000-0003-1163-6162)
- Filippo Branzanti
- Elena Maria Elli (ORCID: https://orcid.org/0000-0001-5576-5412)
- Florian Heinrich Heidel (ORCID: https://orcid.org/0000-0003-2438-1955)
- Giulia Benevolo
- Francesco Cavazzini
- Alessandra Dedola
Institutions
- Università Cattolica del Sacro Cuore (IT)
- Marche Polytechnic University (IT)
- University of Udine (IT)
- University of Ferrara (IT)
- Policlinico Umberto I (IT)
- Agostino Gemelli University Polyclinic (IT)
- Medizinische Hochschule Hannover (DE)
- University of Catania (IT)
- Azienda Ospedaliera Citta' della Salute e della Scienza di Torino (IT)
- Azienda Sanitaria Unità Locale di Reggio Emilia (IT)
- Azienda Unità Sanitaria Locale Della Romagna (IT)
- Azienda Ospedaliera San Gerardo (IT)
- Istituto di Ematologia di Bologna (IT)
- Azienda USL di Bologna (IT)
- Ospedale di Belcolle (IT)
- Tecnologie Avanzate (Italy) (IT)
- Istituti di Ricovero e Cura a Carattere Scientifico (IT)
- Gruppo Italiano per il Trapianto di Midollo Osseo (IT)
- University of Brescia (IT)
- Catholic University of America (US)
- University of Bologna (IT)
Publication Details
- Journal
- Leukemia
- Published
- 2025-05-28
- DOI
- https://doi.org/10.1038/s41375-025-02646-y
- Citations
- 4
- Primary Topic
- Myeloproliferative Neoplasms: Diagnosis and Treatment
- Type
- article
- Field-Weighted Citation Impact
- 6.50
Funders
- Deutsche Forschungsgemeinschaft
- Ministero della Salute