prikaz prve stranice dokumenta Real-world study of children and young adults with myeloproliferative neoplasms: identifying risks and unmet needs
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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 Više autora...

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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, br. 17, 2022, str. 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, br. 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), str. 5171-5183. doi: 10.1182/bloodadvances.2022007201

Sobas M, Kiladjian JJ, Beauverd Y, Curto Garcia N, Sadjadian P, Shih LY, i sur.. Real-world study of children and young adults with myeloproliferative neoplasms: identifying risks and unmet needs. Blood Advances [Internet]. 13.09.2022. [pristupljeno 09.10.2024.];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, br. 17, str. 5171-5183, Rujan 2022. [Online]. Dostupno na: https://urn.nsk.hr/urn:nbn:hr:105:654839. [Citirano: 09.10.2024.]

Za citiranje koristite ovu mrežnu adresu: https://urn.nsk.hr/urn:nbn:hr:105:654839
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Naslov (engleski)Real-world study of children and young adults with myeloproliferative neoplasms: identifying risks and unmet needs
AutorMarta Sobas
AutorJean-Jacques Kiladjian
AutorYan Beauverd
AutorNatalia Curto-Garcia
AutorParvis Sadjadian
AutorLee Yung Shih
AutorTimothy Devos
AutorDorota Krochmalczyk
AutorSerena Galli
AutorMaria Bieniaszewska
AutorIlona Seferynska
AutorMary Frances McMullin
AutorAnna Armatys
AutorAdrianna Spalek
AutorJoanna Waclaw
AutorMihnea Zdrenghea
AutorLaurence Legros
AutorFrançois Girodon
AutorKrzysztof Lewandowski
AutorAnna Angona Figueras
AutorJan Samuelsson
AutorAitor Abuin Blanco
AutorPascale Cony-Makhoul
AutorAngela Collins
AutorChloé James
AutorRajko Kušec
AutorMarie Lauermannova
AutorMaria Sol Noya
AutorMalgorzata Skowronek
AutorLukasz Szukalski
AutorAnna Szmigielska-Kaplon
AutorMarielle Wondergem
AutorIryna Dudchenko
AutorJoanna Gora Tybor
AutorKamel Laribi
AutorAnna Kulikowska de Nalecz
AutorJean-Loup Demory
AutorKatell Le Du
AutorSonja Zweegman
AutorCarlos Besses Raebel
AutorRadek Skoda
AutorStéphane Giraudier
AutorMartin Griesshammer
AutorClaire N. Harrison
AutorJean-Christophe Ianotto
Autorova ustanovaSveučilište u Zagrebu
Medicinski fakultet
(KATEDRA ZA INTERNU MEDICINU)
Znanstveno / umjetničko
područje, polje i grana		BIOMEDICINA I ZDRAVSTVO
Kliničke medicinske znanosti
Interna medicina
Sažetak (engleski)
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.
Ključne riječi (engleski)
Jezikengleski
Vrsta publikacijeZnanstveni rad - Izvorni znanstveni rad
Status objaveObjavljen
Vrsta recenzijeRecenziran
Verzija publikacijeObjavljena verzija rada (izdavačev PDF)
Naslov časopisaBlood Advances
Brojčani podacivol. 6, br. 17, str. 5171-5183
p-ISSN2473-9529
e-ISSN2473-9537
DOIhttps://doi.org/10.1182/bloodadvances.2022007201
URN:NBNurn:nbn:hr:105:654839
Datum objave publikacije2022-09-13
URL dokumentahttps://ashpublications.org/bloodadvances/article/6/17/5171/485820/Real-world-study-of-children-and-young-adults-with
Vrsta resursaTekst
Prava pristupaOtvoreni pristup
Uvjeti korištenjaIkona licence Imenovanje-Nekomercijalno-Bez prerada 4.0 međunarodna (CC BY-NC-ND 4.0)
Datum i vrijeme pohrane2023-04-21 07:12:21