TY - JOUR
T1 - Real world study of children and young adults with myeloproliferative neoplasms identifying risks and unmet needs
AU - Sobas, Marta
AU - Kiladjian, Jean-Jacques
AU - Beauverd, Yan
AU - Curto-Garcia, Natalia
AU - Sadjadian, Parvis
AU - Shih, Lee Yung
AU - Devos, Timothy
AU - Krochmalczyk, Dorota
AU - Galli, Serena
AU - Bieniaszewska, Maria
AU - Seferynska, Ilona
AU - McMullin, Mary Frances Frances
AU - Armatys, Anna
AU - Spalek, Adrianna
AU - Wącław, Joanna
AU - Zdrenghea, Mihnea
AU - Legros, Laurence
AU - Girodon, François
AU - Lewandowski, Krzysztof
AU - Angona Figueras, Anna
AU - Samuelsson, Jan
AU - Abuin Blanco, Aitor
AU - Cony-Makhoul, Pascale
AU - Collins, Angela
AU - James, Chloé
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 - Góra-Tybor, Joanna
AU - Laribi, Kamel
AU - Kulikowska de Nałęcz, Anna
AU - Demory, Jean-Loup
AU - Le Du, Katell
AU - Zweegman, Sonja
AU - Besses, Carles
AU - Skoda, Radek C
AU - Giraudier, Stéphane
AU - Griesshammer, Martin
AU - Harrison, Claire N
AU - Ianotto, Jean-Christophe
PY - 2022/9/13
Y1 - 2022/9/13
N2 - Myeloproliferative neoplasms (MPN) are uncommon in children/young adults. Here we present data on unselected patients diagnosed before 25 years of age included from 38 centres 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 % pt/year), peri-hepatic vein thromboses were most frequent (47.6% venous events) and logistic regression identified JAK2V617F mutation (p=0.016) and hyperviscosity symptoms (visual disturbances, dizziness, vertigo, headache) as risk factors (p=0.040). New hemorrhagic events occurred in 44 patients (9.9%, 1.04 % pt/y). Disease transformation occurred in 48 patients (10.9%, 1.13 % pt/year), usually to myelofibrosis (7.5%) with splenomegaly as a novel risk factor for transformation in ET (p= 0.000) in logistical regression. Eight deaths (1.8%) were recorded, three after allogeneic stem cell transplantation. Concerning conventional risk scores: IPSET-T and IPSET-NT differentiated ET patients in terms of thrombotic risk. Both scores identified high-risk patients with the same median thrombosis-free survival of 28.5y. No contemporary scores were able to predict survival for young ET or PV 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.
AB - Myeloproliferative neoplasms (MPN) are uncommon in children/young adults. Here we present data on unselected patients diagnosed before 25 years of age included from 38 centres 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 % pt/year), peri-hepatic vein thromboses were most frequent (47.6% venous events) and logistic regression identified JAK2V617F mutation (p=0.016) and hyperviscosity symptoms (visual disturbances, dizziness, vertigo, headache) as risk factors (p=0.040). New hemorrhagic events occurred in 44 patients (9.9%, 1.04 % pt/y). Disease transformation occurred in 48 patients (10.9%, 1.13 % pt/year), usually to myelofibrosis (7.5%) with splenomegaly as a novel risk factor for transformation in ET (p= 0.000) in logistical regression. Eight deaths (1.8%) were recorded, three after allogeneic stem cell transplantation. Concerning conventional risk scores: IPSET-T and IPSET-NT differentiated ET patients in terms of thrombotic risk. Both scores identified high-risk patients with the same median thrombosis-free survival of 28.5y. No contemporary scores were able to predict survival for young ET or PV 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.
U2 - 10.1182/bloodadvances.2022007201
DO - 10.1182/bloodadvances.2022007201
M3 - Article
C2 - 35802458
SN - 2473-9529
VL - 6
SP - 5171
EP - 5183
JO - Blood Advances
JF - Blood Advances
IS - 17
ER -