prikaz prve stranice dokumenta Real-world study of children and young adults with myeloproliferative neoplasms: identifying risks and unmet needs
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Scientific paper - Original scientific paper
Real-world study of children and young adults with myeloproliferative neoplasms: identifying risks and unmet needs
Blood Advances, 6 (2022), 17; 5171-5183. https://doi.org/10.1182/bloodadvances.2022007201
Sobas, Marta; Kiladjian, Jean-Jacques; Beauverd, Yan; Curto-Garcia, Natalia; Sadjadian, Parvis; Shih, Lee Yung; Devos, Timothy; Krochmalczyk, Dorota; Galli, Serena; Bieniaszewska, Maria; Seferynska, Ilona; McMullin, Mary Frances; Armatys, Anna; Spalek, Adrianna; Waclaw, Joanna; Zdrenghea, Mihnea; Legros, Laurence; Girodon, François; Lewandowski, Krzysztof; Angona Figueras, Anna; Samuelsson, Jan; Abuin Blanco, Aitor; Cony-Makhoul, Pascale; Collins, Angela; James, Chloé; Kušec, Rajko; Lauermannova, Marie; Noya, Maria Sol; Skowronek, Malgorzata; Szukalski, Lukasz; Szmigielska-Kaplon, Anna; Wondergem, Marielle; Dudchenko, Iryna; Gora Tybor, Joanna; Laribi, Kamel; Kulikowska de Nalecz, Anna; Demory, Jean-Loup; Le Du, Katell; Zweegman, Sonja; Besses Raebel, Carlos; Skoda, Radek; Giraudier, Stéphane; Griesshammer, Martin; Harrison, Claire N.; Ianotto, Jean-Christophe More authors...

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Sobas, M., Kiladjian, J. J., Beauverd, Y., Curto Garcia, N., Sadjadian, P., Shih, L. Y. ... Ianotto, J. C. (2022). Real-world study of children and young adults with myeloproliferative neoplasms: identifying risks and unmet needs. Blood Advances, 6. (17), 5171-5183. doi: 10.1182/bloodadvances.2022007201

Sobas, Marta, et al. "Real-world study of children and young adults with myeloproliferative neoplasms: identifying risks and unmet needs." Blood Advances, vol. 6, no. 17, 2022, pp. 5171-5183. https://doi.org/10.1182/bloodadvances.2022007201

Sobas, Marta, Jean Jacques Kiladjian, Yan Beauverd, Natalia Curto Garcia, Parvis Sadjadian, Lee Yung Shih, Timothy Devos, et al. "Real-world study of children and young adults with myeloproliferative neoplasms: identifying risks and unmet needs." Blood Advances 6, no. 17 (2022): 5171-5183. https://doi.org/10.1182/bloodadvances.2022007201

Sobas, M., et al. (2022) 'Real-world study of children and young adults with myeloproliferative neoplasms: identifying risks and unmet needs', Blood Advances, 6(17), pp. 5171-5183. doi: 10.1182/bloodadvances.2022007201

Sobas M, Kiladjian JJ, Beauverd Y, Curto Garcia N, Sadjadian P, Shih LY, and sur.. Real-world study of children and young adults with myeloproliferative neoplasms: identifying risks and unmet needs. Blood Advances [Internet]. 2022 September 13 [cited 2024 August 19];6(17):5171-5183. doi: 10.1182/bloodadvances.2022007201

M. Sobas, et al., "Real-world study of children and young adults with myeloproliferative neoplasms: identifying risks and unmet needs", Blood Advances, vol. 6, no. 17, pp. 5171-5183, September 2022. [Online]. Available at: https://urn.nsk.hr/urn:nbn:hr:105:654839. [Accessed: 19 August 2024]

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Metadata
Title (english)Real-world study of children and young adults with myeloproliferative neoplasms: identifying risks and unmet needs
AuthorMarta Sobas
AuthorJean-Jacques Kiladjian
AuthorYan Beauverd
AuthorNatalia Curto-Garcia
AuthorParvis Sadjadian
AuthorLee Yung Shih
AuthorTimothy Devos
AuthorDorota Krochmalczyk
AuthorSerena Galli
AuthorMaria Bieniaszewska
AuthorIlona Seferynska
AuthorMary Frances McMullin
AuthorAnna Armatys
AuthorAdrianna Spalek
AuthorJoanna Waclaw
AuthorMihnea Zdrenghea
AuthorLaurence Legros
AuthorFrançois Girodon
AuthorKrzysztof Lewandowski
AuthorAnna Angona Figueras
AuthorJan Samuelsson
AuthorAitor Abuin Blanco
AuthorPascale Cony-Makhoul
AuthorAngela Collins
AuthorChloé James
AuthorRajko Kušec
AuthorMarie Lauermannova
AuthorMaria Sol Noya
AuthorMalgorzata Skowronek
AuthorLukasz Szukalski
AuthorAnna Szmigielska-Kaplon
AuthorMarielle Wondergem
AuthorIryna Dudchenko
AuthorJoanna Gora Tybor
AuthorKamel Laribi
AuthorAnna Kulikowska de Nalecz
AuthorJean-Loup Demory
AuthorKatell Le Du
AuthorSonja Zweegman
AuthorCarlos Besses Raebel
AuthorRadek Skoda
AuthorStéphane Giraudier
AuthorMartin Griesshammer
AuthorClaire N. Harrison
AuthorJean-Christophe Ianotto
Author's institutionUniversity of Zagreb
School of Medicine
(Department of Internal Medicine)
Scientific / art field,
discipline and subdiscipline		BIOMEDICINE AND HEALTHCARE
Clinical Medical Sciences
Internal Medicine
Abstract (english)
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 and new International Prognostic Score for Essential Thrombocythemia-Thrombosis differentiated 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 < 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.
Keywords (english)
Languageenglish
Publication typeScientific paper - Original scientific paper
Publication statusPublished
Peer reviewPeer review
Publication versionPublished version
Journal titleBlood Advances
Numberingvol. 6, no. 17, pp. 5171-5183
p-ISSN2473-9529
e-ISSN2473-9537
DOIhttps://doi.org/10.1182/bloodadvances.2022007201
URN:NBNurn:nbn:hr:105:654839
Publication2022-09-13
Document URLhttps://ashpublications.org/bloodadvances/article/6/17/5171/485820/Real-world-study-of-children-and-young-adults-with
Type of resourceText
Access conditionsOpen access
Terms of useLicence Attribution-NonCommercial-NoDerivatives 4.0 International (CC BY-NC-ND 4.0) icon
Created on2023-04-21 07:12:21