{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,29]],"date-time":"2026-09-29T01:27:49Z","timestamp":1790645269863,"version":"4.1.0"},"reference-count":54,"publisher":"American Society of Hematology","issue":"17","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2022,9,13]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Myeloproliferative neoplasms (MPNs) are uncommon in children\/young adults. Here, we present data on unselected patients diagnosed before 25 years of age included from 38 centers in 15 countries. Sequential patients were included. We identified 444 patients, with median follow-up 9.7 years (0-47.8). Forty-nine (11.1%) had a history of thrombosis at diagnosis, 49 new thrombotic events were recorded (1.16% patient per year [pt\/y]), perihepatic vein thromboses were most frequent (47.6% venous events), and logistic regression identified JAK2V617F mutation (P = .016) and hyperviscosity symptoms (visual disturbances, dizziness, vertigo, headache) as risk factors (P = .040). New hemorrhagic events occurred in 44 patients (9.9%, 1.04% pt\/y). Disease transformation occurred in 48 patients (10.9%, 1.13% pt\/y), usually to myelofibrosis (7.5%) with splenomegaly as a novel risk factor for transformation in essential thrombocythemia (ET) (P= .000) in logistical regression. Eight deaths (1.8%) were recorded, 3 after allogeneic stem cell transplantation. Concerning conventional risk scores: International Prognostic Score for Essential Thrombocythemia-Thrombosis\u00a0and new International Prognostic Score for Essential Thrombocythemia-Thrombosis\u00a0differentiated ET patients in terms of thrombotic risk. Both scores identified high-risk patients with the same median thrombosis-free survival of 28.5 years. No contemporary scores were able to predict survival for young ET or polycythemia vera patients. Our data represents the largest real-world study of MPN patients age &amp;lt; 25 years at diagnosis. Rates of thrombotic events and transformation were higher than expected compared with the previous literature. Our study provides new and reliable information as a basis for prospective studies, trials, and development of harmonized international guidelines for the specific management of young patients with MPN.<\/jats:p>","DOI":"10.1182\/bloodadvances.2022007201","type":"journal-article","created":{"date-parts":[[2022,7,8]],"date-time":"2022-07-08T16:01:45Z","timestamp":1657296105000},"page":"5171-5183","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":46,"title":["Real-world study of children and young adults with myeloproliferative neoplasms: identifying risks and unmet needs"],"prefix":"10.1182","volume":"6","author":[{"given":"Marta","family":"Sobas","sequence":"first","affiliation":[{"name":"1Department of Hematology, Blood Neoplasms and Bone Marrow Transplantation, Wroclaw Medical University, Wroclaw, Poland;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jean-Jacques","family":"Kiladjian","sequence":"additional","affiliation":[{"name":"2Universit\u00e9 de Paris, Assistance Publique des H\u00f4pitaux de Paris (AP-HP), H\u00f4pital Saint-Louis, Centre d\u2019Investigations Cliniques 1427, INSERM, Paris, France;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7971-4326","authenticated-orcid":false,"given":"Yan","family":"Beauverd","sequence":"additional","affiliation":[{"name":"3Division of Hematology, Department of Oncology, Geneva University Hospitals, Geneva, Switzerland;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Natalia","family":"Curto-Garcia","sequence":"additional","affiliation":[{"name":"4Department of Hematology, Guy's and St Thomas' National Health Service Foundation Trust, London, United Kingdom;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Parvis","family":"Sadjadian","sequence":"additional","affiliation":[{"name":"5University Clinic for Hematology, Oncology, Hemostasis, and Palliative Care, Johannes Wesling Medical Center, University of Bochum, Minden, Germany;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1866-7922","authenticated-orcid":false,"given":"Lee Yung","family":"Shih","sequence":"additional","affiliation":[{"name":"6Division of Hematology-Oncology, Chang Gung Memorial Hospital, Taipei, Taiwan;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6881-417X","authenticated-orcid":false,"given":"Timothy","family":"Devos","sequence":"additional","affiliation":[{"name":"7Department of Hematology, University Hospitals Leuven and Department of Microbiology and Immunology, Laboratory of Molecular Immunology (Rega Institute), Katholieke Universiteit Leuven, Leuven, Belgium;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dorota","family":"Krochmalczyk","sequence":"additional","affiliation":[{"name":"8Department of Hematology, Collegium Medicum, Jagiellonian University, Krakow, Poland;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Serena","family":"Galli","sequence":"additional","affiliation":[{"name":"9Department of Biomedicine, Experimental Hematology, University Hospital and University, Basel, Switzerland;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maria","family":"Bieniaszewska","sequence":"additional","affiliation":[{"name":"10Department of Hematology and Transplantation, Medical University and Clinical Center, Gdansk, Poland;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ilona","family":"Seferynska","sequence":"additional","affiliation":[{"name":"11Institute of Hematology and Transfusion Medicine, Warsaw, Poland;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0773-0204","authenticated-orcid":false,"given":"Mary Frances","family":"McMullin","sequence":"additional","affiliation":[{"name":"12Hematology, Belfast City Hospital, Queen's University Belfast, Belfast, United Kingdom;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8537-5802","authenticated-orcid":false,"given":"Anna","family":"Armatys","sequence":"additional","affiliation":[{"name":"13Department of Hematology, Jagiellonian University Hospital, Krakow, Poland;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Adrianna","family":"Spalek","sequence":"additional","affiliation":[{"name":"13Department of Hematology, Jagiellonian University Hospital, Krakow, Poland;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joanna","family":"Waclaw","sequence":"additional","affiliation":[{"name":"8Department of Hematology, Collegium Medicum, Jagiellonian University, Krakow, Poland;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mihnea","family":"Zdrenghea","sequence":"additional","affiliation":[{"name":"14Department of Hematology, Iuliu Hatieganu University of Medicine and Pharmacy, Cluj-Napoca, Romania;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laurence","family":"Legros","sequence":"additional","affiliation":[{"name":"15Department of Hematology, Paul Brousse Hospital, Paris, France;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3151-1068","authenticated-orcid":false,"given":"Fran\u00e7ois","family":"Girodon","sequence":"additional","affiliation":[{"name":"16Laboratory of Biological Hematology, University Hospital, Dijon, France;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0992-2020","authenticated-orcid":false,"given":"Krzysztof","family":"Lewandowski","sequence":"additional","affiliation":[{"name":"17Department of Hematology and Bone Marrow Transplantation, University of Medical Sciences, Poznan, Poland;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anna","family":"Angona Figueras","sequence":"additional","affiliation":[{"name":"18Department of Hematology, Hospital del Mar, Barcelona, Spain;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jan","family":"Samuelsson","sequence":"additional","affiliation":[{"name":"19Department of Hematology, University Hospital, Linkoping, Sweden;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aitor","family":"Abuin Blanco","sequence":"additional","affiliation":[{"name":"20Department of Hematology, University Hospital, Santiago de Compostela, 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Dubrava, School of Medicine, University of Zagreb, Zagreb, Croatia;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marie","family":"Lauermannova","sequence":"additional","affiliation":[{"name":"25Institute of Hematology and Blood Transfusion, Prague, Czech Republic;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maria Sol","family":"Noya","sequence":"additional","affiliation":[{"name":"26Department of Hematology, University Hospital Juan Canalejo-Complejo Hospitalario Universitario a Coru\u00f1a, La Coru\u00f1a, Spain;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6428-1003","authenticated-orcid":false,"given":"Malgorzata","family":"Skowronek","sequence":"additional","affiliation":[{"name":"27Department of Hematology, Holy Cross Oncology Center, Kielce, Poland;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9885-5140","authenticated-orcid":false,"given":"Lukasz","family":"Szukalski","sequence":"additional","affiliation":[{"name":"28Department of Hematology, Collegium Medicum Nicolaus Copernicus, University of Torun, Bydgoszcz, Poland;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0775-6412","authenticated-orcid":false,"given":"Anna","family":"Szmigielska-Kaplon","sequence":"additional","affiliation":[{"name":"29Department of Hematology, Medical University, Lodz, Poland;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marielle","family":"Wondergem","sequence":"additional","affiliation":[{"name":"30Department of Hematology, Amsterdam University Medical Centers, Universiteit Amsterdam, Cancer Center, Amsterdam, 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