| Title: |
Impact of treatment for adolescent and young adults with essential thrombocythemia and polycythemia vera. |
| Authors: |
Beauverd, Yan; Ianotto, Jean-Christophe; Thaw, Kyaw Htin; Sobas, Marta; Sadjadian, Parvis; Curto-Garcia, Natalia; Shih, Lee-Yung; Devos, Timothy; Krochmalczyk, Dorota; Galli, Serena; Bieniaszewska, Maria; Seferynska, Ilona; Mcmullin, Mary Frances; Armatys, Anna; Spalek, Adrianna; Waclaw, Joanna; Zdrenghea, Mihnea Tudor; Legros, Laurence; Girodon, Francois; Lewandowski, Krzysztof; Bellosillo, Beatriz; Samuelsson, Jan; Abuin Blanco, Aitor; Cony-Makhoul, Pascale; Collins, Angela; James, Chloe; Kusec, Rajko; Lauermannova, Marie; Noya, Maria Soledad; Skowronek, Malgorzata; Szukalski, Lukasz; Szmigielska-Kaplon, Anna; Wondergem, Marielle; Dudchenko, Iryna; Gora-Tybor, Joanna; Laribi, Kamel; Kulikowska de Nałęcz, Anna; Demory, Jean-Loup; Le Dû, Katell; Zweegman, Sonja; Besses Raebel, Carlos; Skoda, Radek C; Giraudier, Stephane; Griesshammer, Martin; Kiladjian, Jean-Jacques; Harrison, Claire N |
| Contributors: |
Centre Hospitalier Régional Universitaire de Brest (CHRU Brest); Hôpital Bicêtre AP-HP, Le Kremlin-Bicêtre; Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP); Université Paris-Saclay; Centre Hospitalier Universitaire de Dijon - Hôpital François Mitterrand (CHU Dijon); Biologie des maladies cardiovasculaires = Biology of Cardiovascular Diseases (BMC); Université de Bordeaux (UB)-Institut National de la Santé et de la Recherche Médicale (INSERM); Centre Hospitalier Le Mans (CH Le Mans); Hématopoïèse normale et pathologique : émergence, environnement et recherche translationnelle Paris ((UMR_S1131 / U1131)); Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Paris Cité (UPCité); Hopital Saint-Louis AP-HP (AP-HP) |
| Source: |
ISSN: 0887-6924. |
| Publisher Information: |
CCSD; Springer Nature |
| Publication Year: |
2025 |
| Subject Terms: |
[SDV.MHEP.HEM]Life Sciences [q-bio]/Human health and pathology/Hematology; [SDV.CAN]Life Sciences [q-bio]/Cancer; [SDV.SP.PHARMA]Life Sciences [q-bio]/Pharmaceutical sciences/Pharmacology; [SDV.SPEE]Life Sciences [q-bio]/Santé publique et épidémiologie |
| Description: |
International audience ; 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/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. |
| Document Type: |
article in journal/newspaper |
| Language: |
English |
| DOI: |
10.1038/s41375-025-02545-2 |
| Availability: |
https://hal.science/hal-05055971; https://hal.science/hal-05055971v1/document; https://hal.science/hal-05055971v1/file/BMC_Leukemia_2025_Beauverd.pdf; https://doi.org/10.1038/s41375-025-02545-2 |
| Rights: |
https://creativecommons.org/licenses/by/4.0/ ; info:eu-repo/semantics/OpenAccess |
| Accession Number: |
edsbas.25396F37 |
| Database: |
BASE |