Impact of treatment for adolescent and young adults with essential thrombocythemia and polycythemia vera

Essential thrombocythemia (ET) and polycythemia vera (PV) are rare in adolescent and young adult (AYA). These conditions, similar to those in older patients, are linked with thrombotic complications and the potential progression to secondary myelofibrosis (sMF). This retrospective study of ET and PV patients diagnosed before age 25 evaluated complication rates and impact of cytoreductive drugs on outcomes. Among 348 patients (278 ET, 70 PV) with a median age of 20 years, the of thrombotic events was 1.9 per 100 patient-years. Risk factors for thrombosis included elevated white blood cell count (>11 × 10<sup>9</sup>/L) (HR: 2.7, p = 0.012) and absence of splenomegaly at diagnosis (HR: 5.7, p = 0.026), while cytoreductive drugs did not reduce this risk. The incidence of sMF was 0.7 per 100 patient-years. CALR mutation (HR: 6.0, p < 0.001) and a history of thrombosis (HR: 3.8, p = 0.015) were associated with sMF risk. Interferon as a first-line treatment significantly improved myelofibrosis-free survival compared to other treatments or the absence of cytoreduction (p = 0.046). Although cytoreduction did not affect thrombotic event, early interferon use reduced sMF risk. These findings support interferon use to mitigate sMF risk in AYA ET and PV patients.

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Publication Details

Journal
Leukemia
Published
2025-03-12
DOI
https://doi.org/10.1038/s41375-025-02545-2
Citations
7
Primary Topic
Myeloproliferative Neoplasms: Diagnosis and Treatment
Type
article
Field-Weighted Citation Impact
11.38
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article

Impact of treatment for adolescent and young adults with essential thrombocythemia and polycythemia vera

Carlos Besses Raebel, Martin Grießhammer, Chloé James, Jean‐Jacques Kiladjian et al.
7 citations
Leukemia
Myeloproliferative Neoplasms: Diagnosis and Treatment
11.38
article

Impact of treatment for adolescent and young adults with essential thrombocythemia and polycythemia vera

Carlos Besses Raebel, Martin Grießhammer, Chloé James, Jean‐Jacques Kiladjian, Rajko Kušec, Jean‐Christophe Ianotto, Kamel Laribi, Łukasz Szukalski, Dorota Krochmalczyk, Joanna Wącław, Serena Galli, François Girodon, Anna Armatys, Natalia Curto‐García, Marta Sobas, Katell Le Dû, Pascale Cony‐Makhoul, Claire N. Harrison, Iryna Dudchenko, Parvis Sadjadian, Jan Samuelsson, Radek C. Skoda, Krzysztof Lewandowski, Ilona Seferyńska, Maria Bieniaszewska, Małgorzata Skowronek, Lee‐Yung Shih, Mihnea Zdrenghea, Adrianna Spałek, Beatríz Bellosillo, Joanna Góra‐Tybor, Anna Szmigielska‐Kapłon, Yan Beauverd, Laurence Legros, Anna Kulikowska de Nałęcz, Mary Frances McMullin, Sonja Zweegman, Stéphane Giraudier, Timothy Devos, Marie Lauermannová, Angela Collins, María Soledad Noya, Aitor Abuín Blanco, Jean‐Loup Demory, Kyaw Htin Thaw, Mariëlle J. Wondergem
article en
7 citations

Abstract

Essential thrombocythemia (ET) and polycythemia vera (PV) are rare in adolescent and young adult (AYA). These conditions, similar to those in older patients, are linked with thrombotic complications and the potential progression to secondary myelofibrosis (sMF). This retrospective study of ET and PV patients diagnosed before age 25 evaluated complication rates and impact of cytoreductive drugs on outcomes. Among 348 patients (278 ET, 70 PV) with a median age of 20 years, the of thrombotic events was 1.9 per 100 patient-years. Risk factors for thrombosis included elevated white blood cell count (>11 × 10<sup>9</sup>/L) (HR: 2.7, p = 0.012) and absence of splenomegaly at diagnosis (HR: 5.7, p = 0.026), while cytoreductive drugs did not reduce this risk. The incidence of sMF was 0.7 per 100 patient-years. CALR mutation (HR: 6.0, p < 0.001) and a history of thrombosis (HR: 3.8, p = 0.015) were associated with sMF risk. Interferon as a first-line treatment significantly improved myelofibrosis-free survival compared to other treatments or the absence of cytoreduction (p = 0.046). Although cytoreduction did not affect thrombotic event, early interferon use reduced sMF risk. These findings support interferon use to mitigate sMF risk in AYA ET and PV patients.

LeukemiaVol. 39(5)
Universidade da Coruña (ES), University of Geneva (CH), Poznan University of Medical Sciences (PL), National Health Service (GB), Queen's University Belfast (GB), Jagiellonian University (PL), University of Ulster (GB), Université de Bordeaux (FR), Inserm (FR), University of Opole (PL), Chang Gung University (TW), University of Zagreb (HR), Guy's and St Thomas' NHS Foundation Trust (GB), Iuliu Hațieganu University of Medicine and Pharmacy (RO), Rega Institute for Medical Research (BE), Sumy State University (UA), Université Paris-Saclay (FR), Norfolk and Norwich University Hospitals NHS Foundation Trust (GB), University Hospital Dubrava (HR), University Hospital of Basel (CH), Belfast City Hospital (GB), Nicolaus Copernicus University (PL), Chang Gung Memorial Hospital (TW), Wroclaw Medical University (PL), Instytut Hematologii i Transfuzjologi (PL), Institute of Haematology and Blood Transfusion (CZ), Assistance Publique – Hôpitaux de Paris (FR), University Hospital of Zurich (CH), Holy Cross University (PL), Medical University of Lodz (PL), Centre Hospitalier du Mans (FR), Hospital Del Mar (ES), Centre Hospitalier Régional Universitaire de Brest (FR), Centre Hospitalier Annecy Genevois (FR), Hôpital privé du Confluent (FR), Complexo Hospitalario Universitario A Coruña (ES), Maison des Sciences sociales et des Humanités de Dijon (FR), Hôpital Saint-Louis (FR), Hospital Universitario Lucus Augusti (ES), Collegium Medicum in Bydgoszcz (PL), Amsterdam University Medical Centers (NL), Linköping University Hospital (SE), Bicêtre Hospital (FR), Johannes Wesling Klinikum Minden (DE), Hôpital Saint-Vincent-de-Paul (FR), Biologie des maladies cardiovasculaires, Gdańsk Medical University (PL), KU Leuven (BE)
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Myeloproliferative Neoplasms: Diagnosis and Treatment
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