Impact of treatment for adolescent and young adults with essential thrombocythemia and polycythemia vera
Yan Beauverd, Jean‐Christophe Ianotto, Kyaw Htin Thaw, Marta Sobas, Parvis Sadjadian, Natalia Curto‐García, Lee‐Yung Shih, Timothy Devos, Dorota Krochmalczyk, Serena Galli, Maria Bieniaszewska, Ilona Seferyńska, Mary Frances McMullin, Anna Armatys, Adrianna Spałek, Joanna Wącław, Mihnea Zdrenghea, Laurence Legros, François Girodon, Krzysztof Lewandowski, Beatríz Bellosillo, Jan Samuelsson, Aitor Abuín Blanco, Pascale Cony‐Makhoul, Angela Collins, Chloé James, Rajko Kušec, Marie Lauermannová, María Soledad Noya, Małgorzata Skowronek, Łukasz Szukalski, Anna Szmigielska‐Kapłon, Mariëlle J. Wondergem, Iryna Dudchenko, Joanna Góra‐Tybor, Kamel Laribi, Anna Kulikowska de Nałęcz, Jean‐Loup Demory, Katell Le Dû, Sonja Zweegman, Carlos Besses Raebel, Radek C. Skoda, Stéphane Giraudier, Martin Grießhammer, Jean‐Jacques Kiladjian, Claire Harrison
Abstract
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 9 /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.