Real-world study of children and young adults with myeloproliferative neoplasms: identifying risks and unmet needs

Blood Advances - Tập 6 - Trang 5171-5183 - 2022
Marta Sobas1, Jean-Jacques Kiladjian2, Yan Beauverd3, Natalia Curto-Garcia4, Parvis Sadjadian5, Lee Yung Shih6, Timothy Devos7, Dorota Krochmalczyk8, Serena Galli9, Maria Bieniaszewska10, Ilona Seferynska11, Mary Frances McMullin12, Anna Armatys13, Adrianna Spalek13, Joanna Waclaw8, Mihnea Zdrenghea14, Laurence Legros15, François Girodon16, Krzysztof Lewandowski17, Anna Angona Figueras18
1Department of Hematology, Blood Neoplasms and Bone Marrow Transplantation, Wroclaw Medical University, Wroclaw, Poland
2Université de Paris, Assistance Publique des Hôpitaux de Paris (AP-HP), Hôpital Saint-Louis, Centre d'Investigations Cliniques 1427, INSERM, Paris, France
3Division of Hematology, Department of Oncology, Geneva University Hospitals, Geneva, Switzerland
4Department of Hematology, Guy's and St Thomas' National Health Service Foundation Trust, London, United Kingdom
5University Clinic for Hematology, Oncology, Hemostasis, and Palliative Care, Johannes Wesling Medical Center, University of Bochum, Minden, Germany
6Division of Hematology-Oncology, Chang Gung Memorial Hospital, Taipei, Taiwan
7Department of Hematology, University Hospitals Leuven and Department of Microbiology and Immunology, Laboratory of Molecular Immunology (Rega Institute), Katholieke Universiteit Leuven, Leuven, Belgium
8Department of Hematology, Collegium Medicum, Jagiellonian University, Krakow, Poland
9Department of Biomedicine, Experimental Hematology, University Hospital and University, Basel, Switzerland
10Department of Hematology and Transplantation, Medical University and Clinical Center, Gdansk, Poland
11Institute of Hematology and Transfusion Medicine, Warsaw, Poland
12Hematology, Belfast City Hospital, Queen's University Belfast, Belfast, United Kingdom
13Department of Hematology, Jagiellonian University Hospital, Krakow, Poland
14Department of Hematology, Iuliu Hatieganu University of Medicine and Pharmacy, Cluj-Napoca, Romania
15Department of Hematology, Paul Brousse Hospital, Paris, France
16Laboratory of Biological Hematology, University Hospital, Dijon, France
17Department of Hematology and Bone Marrow Transplantation, University of Medical Sciences, Poznan, Poland
18Department of Hematology, Hospital del Mar, Barcelona, Spain

Tóm tắt

AbstractMyeloproliferative 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.

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Blood Advances

Tập 6

5171-5183

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