How I treat myelodysplastic syndromes

被引:0
作者
Zhao, Lin-Pierre [1 ,2 ,3 ]
Sebert, Marie [1 ,2 ,4 ]
机构
[1] Univ Paris Cite, IRSL, Paris, France
[2] Hop St Louis, AP HP, Serv Hematol Clin, Paris, France
[3] INSERM, UMR 1160, Paris, France
[4] CNRS, UMR7212, INSERM, U944, Paris, France
来源
HEMATOLOGIE | 2023年 / 29卷 / 05期
关键词
ACUTE MYELOID-LEUKEMIA; HEALTH-ORGANIZATION CLASSIFICATION; CHRONIC MYELOMONOCYTIC LEUKEMIA; TRANSFUSION-DEPENDENT PATIENTS; STEM-CELL TRANSPLANTATION; COLONY-STIMULATING FACTOR; RANDOMIZED PHASE-III; LOWER-RISK MDS; INTENSIVE CHEMOTHERAPY; PROGNOSTIC IMPACT;
D O I
10.1684/hma.2023.1823
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Myelodysplastic syndromes (MDS) are clonal myeloid haemopathies mainly affecting elderly subjects. The recently updated international classifications of the World Health Organisation and the International Consensus Classification now distinguish MDS on the basis of cytological abnormalities (excess blasts) and their genetic profile. Prognostic stratification of patients is essential to guide therapeutic management, the aim of which is to improve symptoms and quality of life in patients with low-risk MDS, or to slow leukaemic transformation in high-risk patients. Therapeutic options have evolved in recent years, with the appearance of new compounds that have obtained marketing authorisation (luspatercept), and other promising targeted therapies (isocitrate dehydrogenase inhibitor, venetoclax), the development of which has been based on a better understanding of the biology of MDS.
引用
收藏
页码:303 / 315
页数:13
相关论文
共 107 条
[21]   Clinical and prognostic significance of small paroxysmal nocturnal hemoglobinuria clones in myelodysplastic syndrome and aplastic anemia [J].
Fattizzo, Bruno ;
Ireland, Robin ;
Dunlop, Alan ;
Yallop, Deborah ;
Kassam, Shireen ;
Large, Joanna ;
Gandhi, Shreyans ;
Muus, Petra ;
Manogaran, Charles ;
Sanchez, Katy ;
Consonni, Dario ;
Barcellini, Wilma ;
Mufti, Ghulam J. ;
Marsh, Judith C. W. ;
Kulasekararaj, Austin G. .
LEUKEMIA, 2021, 35 (11) :3223-3231
[22]   Luspatercept in Patients with Lower-Risk Myelodysplastic Syndromes [J].
Fenaux, Pierre ;
Platzbecker, Uwe ;
Mufti, Ghulam J. ;
Garcia-Manero, Guillermo ;
Buckstein, Rena ;
Santini, Valeria ;
Diez-Campelo, Maria ;
Finelli, Carlo ;
Cazzola, Mario ;
Ilhan, Osman ;
Sekeres, Mikkael A. ;
Falantes, Jose F. ;
Arrizabalaga, Beatriz ;
Salvi, Flavia ;
Giai, Valentina ;
Vyas, Paresh ;
Bowen, David ;
Selleslag, Dominik ;
DeZern, Amy E. ;
Jurcic, Joseph G. ;
Germing, Ulrich ;
Goetze, Katharina S. ;
Quesnel, Bruno ;
Beyne-Rauzy, Odile ;
Cluzeau, Thomas ;
Voso, Maria-Teresa ;
Mazure, Dominiek ;
Vellenga, Edo ;
Greenberg, Peter L. ;
Hellstrom-Lindberg, Eva ;
Zeidan, Amer M. ;
Ades, Lionel ;
Verma, Amit ;
Savona, Michael R. ;
Laadem, Abderrahmane ;
Benzohra, Aziz ;
Zhang, Jennie ;
Rampersad, Anita ;
Dunshee, Diana R. ;
Linde, Peter G. ;
Sherman, Matthew L. ;
Komrokji, Rami S. ;
List, Alan F. .
NEW ENGLAND JOURNAL OF MEDICINE, 2020, 382 (02) :140-151
[23]   A phase 3 randomized, placebo-controlled study assessing the efficacy and safety of epoetin-α in anemic patients with low-risk MDS [J].
Fenaux, Pierre ;
Santini, Valeria ;
Spiriti, Maria Antonietta Aloe ;
Giagounidis, Aristoteles ;
Schlag, Rudolf ;
Radinoff, Atanas ;
Gercheva-Kyuchukova, Liana ;
Anagnostopoulos, Achilles ;
Oliva, Esther Natalie ;
Symeonidis, Argiris ;
Berger, Mathilde Hunault ;
Goetze, Katharina S. ;
Potamianou, Anna ;
Haralampiev, Hari ;
Wapenaar, Robert ;
Milionis, Iordanis ;
Platzbecker, Uwe .
LEUKEMIA, 2018, 32 (12) :2648-2658
[24]   A randomized phase 3 study of lenalidomide versus placebo in RBC transfusion-dependent patients with Low-/Intermediate-1-risk myelodysplastic syndromes with del5q [J].
Fenaux, Pierre ;
Giagounidis, Aristoteles ;
Selleslag, Dominik ;
Beyne-Rauzy, Odile ;
Mufti, Ghulam ;
Mittelman, Moshe ;
Muus, Petra ;
Boekhorst, Peter Te ;
Sanz, Guillermo ;
del Canizo, Consuelo ;
Guerci-Bresler, Agnes ;
Nilsson, Lars ;
Platzbecker, Uwe ;
Luebbert, Michael ;
Quesnel, Bruno ;
Cazzola, Mario ;
Ganser, Arnold ;
Bowen, David ;
Schlegelberger, Brigitte ;
Aul, Carlo ;
Knight, Robert ;
Francis, John ;
Fu, Tommy ;
Hellstrom-Lindberg, Eva .
BLOOD, 2011, 118 (14) :3765-3776
[25]   Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study [J].
Fenaux, Pierre ;
Mufti, Ghulam J. ;
Hellstrom-Lindberg, Eva ;
Santini, Valeria ;
Finelli, Carlo ;
Giagounidis, Aristoteles ;
Schoch, Robert ;
Gattermann, Norbert ;
Sanz, Guillermo ;
List, Alan ;
Gore, Steven D. ;
Seymour, John F. ;
Bennett, John M. ;
Byrd, John ;
Backstrom, Jay ;
Zimmerman, Linda ;
McKenzie, David ;
Beach, C. L. ;
Silverman, Lewis R. .
LANCET ONCOLOGY, 2009, 10 (03) :223-232
[26]   Phase 2, single-arm trial to evaluate the effectiveness of darbepoetin alfa for correcting anaemia in patients with myelodysplastic syndromes [J].
Gabrilove, Janice ;
Paquette, Ronald ;
Lyons, Roger M. ;
Mushtaq, Chaudhry ;
Sekeres, Mikkael A. ;
Tomita, Dianne ;
Dreiling, Lyndah .
BRITISH JOURNAL OF HAEMATOLOGY, 2008, 142 (03) :379-393
[27]   Molecular Responses Are Observed across Mutational Spectrum in Treatment-Naive Higher-Risk Myelodysplastic Syndrome Patients Treated with Venetoclax Plus Azacitidine [J].
Garcia, Jacqueline S. ;
Wei, Andrew H. ;
Jacoby, Meagan A. ;
Fong, Chun Yew ;
Borate, Uma ;
Baer, Maria R. ;
Cunningham, Ilona ;
Odenike, Olatoyosi ;
Jurcic, Joseph G. ;
Nowak, Daniel ;
Peterlin, Pierre ;
Platzbecker, Uwe ;
Dunshee, Diana ;
Zhou, Ying ;
Hoffman, David ;
Sun, Yan ;
Popovic, Relja ;
Ainsworth, Barrett ;
Naqvi, Kiran ;
Kye, Steve ;
Hogdal, Leah ;
Garcia-Manero, Guillermo .
BLOOD, 2021, 138
[28]   Guadecitabine (SGI-110) in patients with intermediate or high-risk myelodysplastic syndromes: phase 2 results from a multicentre, open-label, randomised, phase 1/2 trial [J].
Garcia-Manero, Guillermo ;
Roboz, Gail ;
Walsh, Katherine ;
Kantarjian, Hagop ;
Ritchie, Ellen ;
Kropf, Patricia ;
O'Connell, Casey ;
Tibes, Raoul ;
Lunin, Scott ;
Rosenblat, Todd ;
Yee, Karen ;
Stock, Wendy ;
Griffiths, Elizabeth ;
Mace, Joseph ;
Podoltsev, Nikolai ;
Berdeja, Jesus ;
Jabbour, Elias ;
Issa, Jean-Pierre J. ;
Hao, Yong ;
Keer, Harold N. ;
Azab, Mohammad ;
Savona, Michael R. .
LANCET HAEMATOLOGY, 2019, 6 (06) :E317-E327
[29]   A Phase II Study of Nivolumab or Ipilimumab with or without Azacitidine for Patients with Myelodysplastic Syndrome (MDS) [J].
Garcia-Manero, Guillermo ;
Sasaki, Koji ;
Montalban-Bravo, Guillermo ;
Daver, Naval G. ;
Jabbour, Elias J. ;
Alvarado, Yesid ;
DiNardo, Courtney D. ;
Ravandi, Farhad ;
Borthakur, Gautam ;
Bose, Prithviraj ;
Pemmaraju, Naveen ;
Naqvi, Kiran ;
Cortes, Jorge E. ;
Kadia, Tapan M. ;
Konopleva, Marina Y. ;
Colla, Simona ;
Yang, Hui ;
Rausch, Caitlin R. ;
Gasior, Yvonne ;
Bueso-Ramos, Carlos E. ;
Kanagal-Shamanna, Rashmi ;
Patel, Keyur P. ;
Kantarjian, Hagop M. .
BLOOD, 2018, 132
[30]   International scoring system for evaluating prognosis in myelodysplastic syndromes [J].
Greenberg, P ;
Cox, C ;
LeBeau, MM ;
Fenaux, P ;
Morel, P ;
Sanz, G ;
Sanz, M ;
Vallespi, T ;
Hamblin, T ;
Oscier, D ;
Ohyashiki, K ;
Toyama, K ;
Aul, C ;
Mufti, G ;
Bennett, J .
BLOOD, 1997, 89 (06) :2079-2088