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. 2025 May;39(5):1135-1145.
doi: 10.1038/s41375-025-02545-2. Epub 2025 Mar 12.

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

Yan Beauverd  1 , Jean-Christophe Ianotto  2 , Kyaw Htin Thaw  3 , Marta Sobas  4 , Parvis Sadjadian  5 , Natalia Curto-Garcia  3 , Lee-Yung Shih  6 , Timothy Devos  7 , Dorota Krochmalczyk  8 , Serena Galli  9 , Maria Bieniaszewska  10 , Ilona Seferynska  11 , Mary Frances McMullin  12 , Anna Armatys  13 , Adrianna Spalek  13 , Joanna Waclaw  8 , Mihnea Tudor Zdrenghea  14 , Laurence Legros  15 , Francois Girodon  16 , Krzysztof Lewandowski  17 , Beatriz Bellosillo  18 , Jan Samuelsson  19 , Aitor Abuin Blanco  20 , Pascale Cony-Makhoul  21 , Angela Collins  22 , Chloe James  23 , Rajko Kusec  24 , Marie Lauermannova  25 , Maria Soledad Noya  26 , Malgorzata Skowronek  27 , Lukasz Szukalski  28 , Anna Szmigielska-Kaplon  29 , Marielle Wondergem  30 , Iryna Dudchenko  31 , Joanna Gora-Tybor  29 , Kamel Laribi  32 , Anna Kulikowska de Nałęcz  33 , Jean-Loup Demory  34 , Katell Le Dû  35 , Sonja Zweegman  30 , Carlos Besses Raebel  18 , Radek C Skoda  36 , Stephane Giraudier  37 , Martin Griesshammer  5 , Jean-Jacques Kiladjian  38 , Claire N Harrison  39
Affiliations

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

Yan Beauverd et al. Leukemia. 2025 May.

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 × 109/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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Conflict of interest statement

Competing interests: YB reports advisory board membership (AOP), speaker fee (AOP), congress support (Abbvie), J-CI reports advisory board membership (Abbvie, Novartis), grant (Novartis), congresses support (Novartis, Pfizer, GSK), MS reports lectures (AOP Orphan, BMS, GSK, Novartis), conferences (Novartis), membership advisory board (BMS, GSK), TD reports consultancy fee (GSK, Morphosys, BMS, Novartis, Incyte) and honorarium (Janssen, Sobi, Alexion), MFM reports advisory board membership (Novartis, BMS, Incyte, GSK), speaker bureau participation (Novartis, AOP, GSK), and clinical trial support (BMS, AOP, GSK), LL reports research funding (Amgen, BMS, Incyte, Novartis), honoraria (BMS, GSK, Novartis, Pfizer), hospitality (Novartis, Pfizer), FG reports sponsored UPR (Abbvie), CJ reports conference (Abbvie), MW reports speaker fees (AOP), educational talks (Novartis, BMS, Sanofi), data safety monitoring board (Keros therapeutics), MG report consultancy (AOP Orphan, Novartis, BMS, AbbVie, Pfizer, Roche, Janssen, Gilead, AstraZeneca, Sierra, Lilly, GSK) and honorarium (AOP Orphan, Novartis, BMS, AbbVie, Pfizer, Roche, Janssen, Gilead, AstraZeneca, Sierra, Lilly, GSK), J-JK reports consultancy fee (Novartis, GSK, Abbvie), advisory boards membership (BMS, Incyte, AOP, PharmaEssentia), CNH reports research funding (Celgene (BMS), Constellation, GSK, Novartis), and Advisory board membership (AbbVie, AOP, BMS, CTI, IMAGO, Incyte, Novartis, Galacteo, Geron, GSK, Incyte, Janssen, Keros, MSD, SOBI, Morphosys). The remaining authors declare no competing financial interests.

Figures

Fig. 1
Fig. 1
Univariate and multivariate analysis for risk factors associated with thrombotic risk in the entire cohort. CVRF cardiovascular risk factors, ET essential thrombocythemia, HCT hematocrit, LDH lactate dehydrogenase, PLT platelet, PV polycythemia vera, TN triple-negative, WBC white blood count.
Fig. 2
Fig. 2
Thrombosis-free survival according to the first management (IFN vs. HU vs. ANA). ANA anagrelide, HU hydroxycarbamide, IFN interferon.
Fig. 3
Fig. 3
Univariate and multivariate analysis for risk factors associated with risk of myelofibrosis progression in the entire cohort. CVRF cardiovascular risk factors, ET essential thrombocythemia, HCT hematocrit, LDH lactate dehydrogenase, PLT platelet, PV polycythemia vera, TN triple-negative, WBC white blood count.
Fig. 4
Fig. 4. Myelofibrosis-free survival.
A According to the first management (No IFN vs. IFN). B According to the first management (No CYTO vs. IFN vs. HU vs. ANA). ANA anagrelide, HU hydroxycarbamide, IFN interferon, No CYTO no cytoreduction, No IFN No interferon.
Fig. 5
Fig. 5
Myelofibrosis-free survival according to the number of lines of cytoreduction.
See this image and copyright information in PMC

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