-
Je něco špatně v tomto záznamu ?
Real-world study of children and young adults with myeloproliferative neoplasms: identifying risks and unmet needs
M. Sobas, JJ. Kiladjian, Y. Beauverd, N. Curto-Garcia, P. Sadjadian, LY. Shih, T. Devos, D. Krochmalczyk, S. Galli, M. Bieniaszewska, I. Seferynska, MF. McMullin, A. Armatys, A. Spalek, J. Waclaw, M. Zdrenghea, L. Legros, F. Girodon, K....
Jazyk angličtina Země Spojené státy americké
Typ dokumentu časopisecké články
NLK
Directory of Open Access Journals
od 2016
PubMed Central
od 2016
Europe PubMed Central
od 2016
ROAD: Directory of Open Access Scholarly Resources
od 2016
- MeSH
- dítě MeSH
- dospělí MeSH
- esenciální trombocytemie * MeSH
- lidé MeSH
- mladý dospělý MeSH
- myeloproliferativní poruchy * komplikace diagnóza epidemiologie MeSH
- polycythaemia vera * komplikace MeSH
- primární myelofibróza * genetika MeSH
- prospektivní studie MeSH
- trombóza * etiologie MeSH
- Check Tag
- dítě MeSH
- dospělí MeSH
- lidé MeSH
- mladý dospělý MeSH
- Publikační typ
- časopisecké články MeSH
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.
Biology of Cardiovascular Diseases University of Bordeaux INSERM UMR1034 Pessac France
Department of Cellular Biology INSERM UMRS 1131 St Louis Hospital AP HP Paris France
Department of Hematology and Transplantation Medical University and Clinical Center Gdansk Poland
Department of Hematology Annecy Genevois Hospital Centre Pringy France
Department of Hematology Collegium Medicum Jagiellonian University Krakow Poland
Department of Hematology Collegium Medicum Nicolaus Copernicus University of Torun Bydgoszcz Poland
Department of Hematology Holy Cross Oncology Center Kielce Poland
Department of Hematology Hospital del Mar Barcelona Spain
Department of Hematology Institute of Oncology and Hematology University Hospital Brest France
Department of Hematology Iuliu Hatieganu University of Medicine and Pharmacy Cluj Napoca Romania
Department of Hematology Jagiellonian University Hospital Krakow Poland
Department of Hematology Le Mans Hospital Le Mans France
Department of Hematology Medical University Lodz Poland
Department of Hematology Paul Brousse Hospital Paris France
Department of Hematology St Vincent De Paul Hospital Lille France
Department of Hematology State Hospital Opole Poland
Department of Hematology University Hospital Linkoping Sweden
Department of Hematology University Hospital Santiago de Compostela Spain
Department of Hematology University Medical Center Amsterdam Netherlands
Division of Hematology Department of Oncology Geneva University Hospitals Geneva Switzerland
Division of Hematology Oncology Chang Gung Memorial Hospital Taipei Taiwan
Hematology Belfast City Hospital Queen's University Belfast Belfast United Kingdom
Institute of Hematology and Blood Transfusion Prague Czech Republic
Institute of Hematology and Transfusion Medicine Warsaw Poland
Laboratory of Biological Hematology University Hospital Dijon France
Citace poskytuje Crossref.org
- 000
- 00000naa a2200000 a 4500
- 001
- bmc22024419
- 003
- CZ-PrNML
- 005
- 20221031101353.0
- 007
- ta
- 008
- 221017s2022 xxu f 000 0|eng||
- 009
- AR
- 024 7_
- $a 10.1182/bloodadvances.2022007201 $2 doi
- 035 __
- $a (PubMed)35802458
- 040 __
- $a ABA008 $b cze $d ABA008 $e AACR2
- 041 0_
- $a eng
- 044 __
- $a xxu
- 100 1_
- $a Sobas, Marta $u Department of Hematology, Blood Neoplasms and Bone Marrow Transplantation, Wroclaw Medical University, Wroclaw, Poland
- 245 10
- $a Real-world study of children and young adults with myeloproliferative neoplasms: identifying risks and unmet needs / $c M. Sobas, JJ. Kiladjian, Y. Beauverd, N. Curto-Garcia, P. Sadjadian, LY. Shih, T. Devos, D. Krochmalczyk, S. Galli, M. Bieniaszewska, I. Seferynska, MF. McMullin, A. Armatys, A. Spalek, J. Waclaw, M. Zdrenghea, L. Legros, F. Girodon, K. Lewandowski, A. Angona Figueras, J. Samuelsson, A. Abuin Blanco, P. Cony-Makhoul, A. Collins, C. James, R. Kusec, M. Lauermannova, MS. Noya, M. Skowronek, L. Szukalski, A. Szmigielska-Kaplon, M. Wondergem, I. Dudchenko, J. Gora Tybor, K. Laribi, A. Kulikowska de Nalecz, JL. Demory, K. Le Du, S. Zweegman, C. Besses Raebel, R. Skoda, S. Giraudier, M. Griesshammer, CN. Harrison, JC. Ianotto
- 520 9_
- $a 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.
- 650 _2
- $a dospělí $7 D000328
- 650 _2
- $a dítě $7 D002648
- 650 _2
- $a lidé $7 D006801
- 650 12
- $a myeloproliferativní poruchy $x komplikace $x diagnóza $x epidemiologie $7 D009196
- 650 12
- $a polycythaemia vera $x komplikace $7 D011087
- 650 12
- $a primární myelofibróza $x genetika $7 D055728
- 650 _2
- $a prospektivní studie $7 D011446
- 650 12
- $a esenciální trombocytemie $7 D013920
- 650 12
- $a trombóza $x etiologie $7 D013927
- 650 _2
- $a mladý dospělý $7 D055815
- 655 _2
- $a časopisecké články $7 D016428
- 700 1_
- $a Kiladjian, Jean-Jacques $u Université de Paris, Assistance Publique des Hôpitaux de Paris (AP-HP), Hôpital Saint-Louis, Centre d'Investigations Cliniques 1427, INSERM, Paris, France
- 700 1_
- $a Beauverd, Yan $u Division of Hematology, Department of Oncology, Geneva University Hospitals, Geneva, Switzerland $1 https://orcid.org/0000000279714326
- 700 1_
- $a Curto-Garcia, Natalia $u Department of Hematology, Guy's and St Thomas' National Health Service Foundation Trust, London, United Kingdom
- 700 1_
- $a Sadjadian, Parvis $u University Clinic for Hematology, Oncology, Hemostasis, and Palliative Care, Johannes Wesling Medical Center, University of Bochum, Minden, Germany
- 700 1_
- $a Shih, Lee Yung $u Division of Hematology-Oncology, Chang Gung Memorial Hospital, Taipei, Taiwan $1 https://orcid.org/0000000318667922
- 700 1_
- $a Devos, Timothy $u Department of Hematology, University Hospitals Leuven and Department of Microbiology and Immunology, Laboratory of Molecular Immunology (Rega Institute), Katholieke Universiteit Leuven, Leuven, Belgium $1 https://orcid.org/000000026881417X
- 700 1_
- $a Krochmalczyk, Dorota $u Department of Hematology, Collegium Medicum, Jagiellonian University, Krakow, Poland
- 700 1_
- $a Galli, Serena $u Department of Biomedicine, Experimental Hematology, University Hospital and University, Basel, Switzerland
- 700 1_
- $a Bieniaszewska, Maria $u Department of Hematology and Transplantation, Medical University and Clinical Center, Gdansk, Poland
- 700 1_
- $a Seferynska, Ilona $u Institute of Hematology and Transfusion Medicine, Warsaw, Poland
- 700 1_
- $a McMullin, Mary Frances $u Hematology, Belfast City Hospital, Queen's University Belfast, Belfast, United Kingdom $1 https://orcid.org/0000000207730204
- 700 1_
- $a Armatys, Anna $u Department of Hematology, Jagiellonian University Hospital, Krakow, Poland $1 https://orcid.org/0000000185375802
- 700 1_
- $a Spalek, Adrianna $u Department of Hematology, Jagiellonian University Hospital, Krakow, Poland
- 700 1_
- $a Waclaw, Joanna $u Department of Hematology, Collegium Medicum, Jagiellonian University, Krakow, Poland
- 700 1_
- $a Zdrenghea, Mihnea $u Department of Hematology, Iuliu Hatieganu University of Medicine and Pharmacy, Cluj-Napoca, Romania
- 700 1_
- $a Legros, Laurence $u Department of Hematology, Paul Brousse Hospital, Paris, France
- 700 1_
- $a Girodon, François $u Laboratory of Biological Hematology, University Hospital, Dijon, France $1 https://orcid.org/0000000331511068
- 700 1_
- $a Lewandowski, Krzysztof $u Department of Hematology and Bone Marrow Transplantation, University of Medical Sciences, Poznan, Poland $1 https://orcid.org/0000000309922020
- 700 1_
- $a Angona Figueras, Anna $u Department of Hematology, Hospital del Mar, Barcelona, Spain
- 700 1_
- $a Samuelsson, Jan $u Department of Hematology, University Hospital, Linkoping, Sweden
- 700 1_
- $a Abuin Blanco, Aitor $u Department of Hematology, University Hospital, Santiago de Compostela, Spain
- 700 1_
- $a Cony-Makhoul, Pascale $u Department of Hematology, Annecy-Genevois Hospital Centre, Pringy, France
- 700 1_
- $a Collins, Angela $u Department of Hematology, Norfolk and Norwich University Hospitals National Health Service Trust, Norwich, United Kingdom
- 700 1_
- $a James, Chloé $u Biology of Cardiovascular Diseases, University of Bordeaux, INSERM, UMR1034, Pessac, France $1 https://orcid.org/0000000195532701
- 700 1_
- $a Kusec, Rajko $u Department of Hematology, University Hospital Dubrava, School of Medicine, University of Zagreb, Zagreb, Croatia
- 700 1_
- $a Lauermannova, Marie $u Institute of Hematology and Blood Transfusion, Prague, Czech Republic
- 700 1_
- $a Noya, Maria Sol $u Department of Hematology, University Hospital Juan Canalejo-Complejo Hospitalario Universitario a Coruña, La Coruña, Spain
- 700 1_
- $a Skowronek, Malgorzata $u Department of Hematology, Holy Cross Oncology Center, Kielce, Poland $1 https://orcid.org/0000000264281003
- 700 1_
- $a Szukalski, Lukasz $u Department of Hematology, Collegium Medicum Nicolaus Copernicus, University of Torun, Bydgoszcz, Poland $1 https://orcid.org/0000000298855140
- 700 1_
- $a Szmigielska-Kaplon, Anna $u Department of Hematology, Medical University, Lodz, Poland $1 https://orcid.org/0000000207756412
- 700 1_
- $a Wondergem, Marielle $u Department of Hematology, Amsterdam University Medical Centers, Universiteit Amsterdam, Cancer Center, Amsterdam, Netherlands
- 700 1_
- $a Dudchenko, Iryna $u Department of Internal Medicine, Sumy State University, Medical Institute, Respiratory Medicine Center, Sumy, Ukraine $1 https://orcid.org/0000000270384455
- 700 1_
- $a Gora Tybor, Joanna $u Department of Hematology, Medical University, Lodz, Poland
- 700 1_
- $a Laribi, Kamel $u Department of Hematology, Le Mans Hospital, Le Mans, France
- 700 1_
- $a Kulikowska de Nalecz, Anna $u Department of Hematology, State Hospital, Opole, Poland
- 700 1_
- $a Demory, Jean-Loup $u Department of Hematology, St. Vincent De Paul Hospital, Lille, France
- 700 1_
- $a Le Du, Katell $u The Confluent, Private Hospital, Nantes, France
- 700 1_
- $a Zweegman, Sonja $u Department of Hematology, University Medical Center, Amsterdam, Netherlands
- 700 1_
- $a Besses Raebel, Carlos $u Department of Hematology, Hospital del Mar, Barcelona, Spain
- 700 1_
- $a Skoda, Radek $u Department of Biomedicine, Experimental Hematology, University Hospital and University, Basel, Switzerland
- 700 1_
- $a Giraudier, Stéphane $u Department of Cellular Biology, INSERM UMRS 1131, St Louis Hospital, AP-HP, Paris, France; and
- 700 1_
- $a Griesshammer, Martin $u University Clinic for Hematology, Oncology, Hemostasis, and Palliative Care, Johannes Wesling Medical Center, University of Bochum, Minden, Germany
- 700 1_
- $a Harrison, Claire N $u Department of Hematology, Guy's and St Thomas' National Health Service Foundation Trust, London, United Kingdom
- 700 1_
- $a Ianotto, Jean-Christophe $u Department of Hematology, Institute of Oncology and Hematology, University Hospital, Brest, France
- 773 0_
- $w MED00194912 $t Blood advances $x 2473-9537 $g Roč. 6, č. 17 (2022), s. 5171-5183
- 856 41
- $u https://pubmed.ncbi.nlm.nih.gov/35802458 $y Pubmed
- 910 __
- $a ABA008 $b sig $c sign $y p $z 0
- 990 __
- $a 20221017 $b ABA008
- 991 __
- $a 20221031101351 $b ABA008
- 999 __
- $a ok $b bmc $g 1854245 $s 1175709
- BAS __
- $a 3
- BAS __
- $a PreBMC
- BMC __
- $a 2022 $b 6 $c 17 $d 5171-5183 $e 20220913 $i 2473-9537 $m Blood advances $n Blood Adv $x MED00194912
- LZP __
- $a Pubmed-20221017
