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-Garcia; Lee Yung Shih; Timothy Devos; Dorota Krochmalczyk; Serena Galli; Maria Bieniaszewska; Ilona Seferynska; Mary Frances McMullin; Anna Armatys; Adrianna Spalek; Joanna Waclaw; Mihnea Tudor Zdrenghea; Laurence Legros; Francois Girodon; Krzysztof Lewandowski; Beatriz Bellosillo; Jan Samuelsson; Aitor Abuin Blanco; Pascale Cony-Makhoul; Angela Collins; Chloe James; Rajko Kusec; Marie Lauermannova; Maria Soledad Noya; Malgorzata Skowronek; Lukasz Szukalski; Anna Szmigielska-Kaplon; Marielle Wondergem; Iryna Dudchenko; Joanna Gora-Tybor; Kamel Laribi; Anna Kulikowska de Nałęcz; Jean Loup Demory; Katell Le Dû; Sonja Zweegman; Carlos Besses Raebel; Radek C. Skoda; Stephane Giraudier; Martin Griesshammer; Jean Jacques Kiladjian; Claire N. Harrison

doi:10.1038/s41375-025-02545-2

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-Garcia
, Lee Yung Shih
, Timothy Devos
, Dorota Krochmalczyk
, Serena Galli
, Maria Bieniaszewska
, Ilona Seferynska
, Mary Frances McMullin
, Anna Armatys
, Adrianna Spalek
, Joanna Waclaw
, Mihnea Tudor Zdrenghea
, Laurence Legros
, Francois Girodon
, Krzysztof Lewandowski

Beatriz Bellosillo, Jan Samuelsson, Aitor Abuin Blanco, Pascale Cony-Makhoul, Angela Collins, Chloe James, Rajko Kusec, Marie Lauermannova, Maria Soledad Noya, Malgorzata Skowronek, Lukasz Szukalski, Anna Szmigielska-Kaplon, Marielle Wondergem, Iryna Dudchenko, Joanna Gora-Tybor, Kamel Laribi, Anna Kulikowska de Nałęcz, Jean Loup Demory, Katell Le Dû, Sonja Zweegman, Carlos Besses Raebel, Radek C. Skoda, Stephane Giraudier, Martin Griesshammer, Jean Jacques Kiladjian, Claire N. Harrison

^*Corresponding author for this work

University of Geneva
CHU de Brest
Guy's and St Thomas' NHS Foundation Trust
Wrocław Medical University
Ruhr University Bochum
Chang Gung Memorial Hospital
KU Leuven
Jagiellonian University Medical College
University of Zurich
Medical University of Gdańsk
Institute of Hematology and Blood Transfusion
Queen's University Belfast
Jagiellonian University in Kraków
Iuliu Hatieganu University of Medicine and Pharmacy
Université Paris-Saclay
Université de Bourgogne
University of Medical Sciences Poznan
Hospital del Mar
Linköping University
Servicio de Hematología. Hospital Universitario Lucus Augusti
Annecy-Genevois Hospital
Norfolk and Norwich University Hospitals NHS Foundation Trust
University of Bordeaux
University Hospital Dubrava
Hospital Juan Canalejo
Holy Cross Oncology Center
Nicolaus Copernicus University in Toruń
Medical University of Łódź
Academic Medical Center
Sumy State University
Le Mans Hospital
State Hospital
Hôpital Saint Vincent de Paul
Private Hospital
University of Basel
Université Paris Cité

Research output: Contribution to journal › Journal Article › peer-review

17 Scopus 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 × 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.

Original language	English
Pages (from-to)	1135-1145
Number of pages	11
Journal	Leukemia
Volume	39
Issue number	5
DOIs	https://doi.org/10.1038/s41375-025-02545-2
State	Published - 05 2025
Externally published	Yes

Bibliographical note

Publisher Copyright:
© The Author(s) 2025.

Keywords

Adolescent
Adult
Calreticulin/genetics
Female
Follow-Up Studies
Humans
Male
Mutation
Polycythemia Vera/drug therapy
Primary Myelofibrosis/etiology
Prognosis
Retrospective Studies
Risk Factors
Thrombocythemia, Essential/drug therapy
Thrombosis/etiology
Young Adult

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10.1038/s41375-025-02545-2

Cite this

Beauverd, Y., Ianotto, J. C., Thaw, K. H., Sobas, M., Sadjadian, P., Curto-Garcia, N., Shih, L. Y., Devos, T., Krochmalczyk, D., Galli, S., Bieniaszewska, M., Seferynska, I., McMullin, M. F., Armatys, A., Spalek, A., Waclaw, J., Zdrenghea, M. T., Legros, L., Girodon, F., ... Harrison, C. N. (2025). Impact of treatment for adolescent and young adults with essential thrombocythemia and polycythemia vera. Leukemia, 39(5), 1135-1145. https://doi.org/10.1038/s41375-025-02545-2

@article{cf7f870b0bf54bdc91b2bd3abcf731d5,

title = "Impact of treatment for adolescent and young adults with essential thrombocythemia and polycythemia vera",

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 {\texttimes} 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.",

keywords = "Adolescent, Adult, Calreticulin/genetics, Female, Follow-Up Studies, Humans, Male, Mutation, Polycythemia Vera/drug therapy, Primary Myelofibrosis/etiology, Prognosis, Retrospective Studies, Risk Factors, Thrombocythemia, Essential/drug therapy, Thrombosis/etiology, Young Adult",

author = "Yan Beauverd and Ianotto, \{Jean Christophe\} and Thaw, \{Kyaw Htin\} and Marta Sobas and Parvis Sadjadian and Natalia Curto-Garcia and Shih, \{Lee Yung\} and Timothy Devos and Dorota Krochmalczyk and Serena Galli and Maria Bieniaszewska and Ilona Seferynska and McMullin, \{Mary Frances\} and Anna Armatys and Adrianna Spalek and Joanna Waclaw and Zdrenghea, \{Mihnea Tudor\} and Laurence Legros and Francois Girodon and Krzysztof Lewandowski and Beatriz Bellosillo and Jan Samuelsson and \{Abuin Blanco\}, Aitor and Pascale Cony-Makhoul and Angela Collins and Chloe James and Rajko Kusec and Marie Lauermannova and Noya, \{Maria Soledad\} and Malgorzata Skowronek and Lukasz Szukalski and Anna Szmigielska-Kaplon and Marielle Wondergem and Iryna Dudchenko and Joanna Gora-Tybor and Kamel Laribi and \{Kulikowska de Na{\l}{\c e}cz\}, Anna and Demory, \{Jean Loup\} and \{Le D{\^u}\}, Katell and Sonja Zweegman and \{Besses Raebel\}, Carlos and Skoda, \{Radek C.\} and Stephane Giraudier and Martin Griesshammer and Kiladjian, \{Jean Jacques\} and Harrison, \{Claire N.\}",

note = "Publisher Copyright: {\textcopyright} The Author(s) 2025.",

year = "2025",

month = may,

doi = "10.1038/s41375-025-02545-2",

language = "英语",

volume = "39",

pages = "1135--1145",

journal = "Leukemia",

issn = "0887-6924",

publisher = "Springer Nature",

number = "5",

}

Beauverd, Y, Ianotto, JC, Thaw, KH, Sobas, M, Sadjadian, P, Curto-Garcia, N, Shih, LY, Devos, T, Krochmalczyk, D, Galli, S, Bieniaszewska, M, Seferynska, I, McMullin, MF, Armatys, A, Spalek, A, Waclaw, J, Zdrenghea, MT, Legros, L, Girodon, F, Lewandowski, K, Bellosillo, B, Samuelsson, J, Abuin Blanco, A, Cony-Makhoul, P, Collins, A, James, C, Kusec, R, Lauermannova, M, Noya, MS, Skowronek, M, Szukalski, L, Szmigielska-Kaplon, A, Wondergem, M, Dudchenko, I, Gora-Tybor, J, Laribi, K, Kulikowska de Nałęcz, A, Demory, JL, Le Dû, K, Zweegman, S, Besses Raebel, C, Skoda, RC, Giraudier, S, Griesshammer, M, Kiladjian, JJ & Harrison, CN 2025, 'Impact of treatment for adolescent and young adults with essential thrombocythemia and polycythemia vera', Leukemia, vol. 39, no. 5, pp. 1135-1145. https://doi.org/10.1038/s41375-025-02545-2

TY - JOUR

T1 - Impact of treatment for adolescent and young adults with essential thrombocythemia and polycythemia vera

AU - Beauverd, Yan

AU - Ianotto, Jean Christophe

AU - Thaw, Kyaw Htin

AU - Sobas, Marta

AU - Sadjadian, Parvis

AU - Curto-Garcia, Natalia

AU - Shih, Lee Yung

AU - Devos, Timothy

AU - Krochmalczyk, Dorota

AU - Galli, Serena

AU - Bieniaszewska, Maria

AU - Seferynska, Ilona

AU - McMullin, Mary Frances

AU - Armatys, Anna

AU - Spalek, Adrianna

AU - Waclaw, Joanna

AU - Zdrenghea, Mihnea Tudor

AU - Legros, Laurence

AU - Girodon, Francois

AU - Lewandowski, Krzysztof

AU - Bellosillo, Beatriz

AU - Samuelsson, Jan

AU - Abuin Blanco, Aitor

AU - Cony-Makhoul, Pascale

AU - Collins, Angela

AU - James, Chloe

AU - Kusec, Rajko

AU - Lauermannova, Marie

AU - Noya, Maria Soledad

AU - Skowronek, Malgorzata

AU - Szukalski, Lukasz

AU - Szmigielska-Kaplon, Anna

AU - Wondergem, Marielle

AU - Dudchenko, Iryna

AU - Gora-Tybor, Joanna

AU - Laribi, Kamel

AU - Kulikowska de Nałęcz, Anna

AU - Demory, Jean Loup

AU - Le Dû, Katell

AU - Zweegman, Sonja

AU - Besses Raebel, Carlos

AU - Skoda, Radek C.

AU - Giraudier, Stephane

AU - Griesshammer, Martin

AU - Kiladjian, Jean Jacques

AU - Harrison, Claire N.

PY - 2025/5

Y1 - 2025/5

N2 - 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.

AB - 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.

KW - Adolescent

KW - Adult

KW - Calreticulin/genetics

KW - Female

KW - Follow-Up Studies

KW - Humans

KW - Male

KW - Mutation

KW - Polycythemia Vera/drug therapy

KW - Primary Myelofibrosis/etiology

KW - Prognosis

KW - Retrospective Studies

KW - Risk Factors

KW - Thrombocythemia, Essential/drug therapy

KW - Thrombosis/etiology

KW - Young Adult

UR - https://www.scopus.com/pages/publications/105000059303

U2 - 10.1038/s41375-025-02545-2

DO - 10.1038/s41375-025-02545-2

M3 - 文章

C2 - 40074852

AN - SCOPUS:105000059303

SN - 0887-6924

VL - 39

SP - 1135

EP - 1145

JO - Leukemia

JF - Leukemia

IS - 5

ER -

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

Abstract

Bibliographical note

Keywords

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