Stepping out of antiquity: An update on emerging drugs for the treatment of polycythemia vera

被引:5
作者
Castillo Tokumori, Franco [1 ]
Komrokji, Rami [2 ]
Kuykendall, Andrew T. [2 ]
机构
[1] Univ S Florida, Coll Med, Internal Med, Tampa, FL 33620 USA
[2] H Lee Moffitt Canc Ctr & Res Inst, Dept Malignant Hematol, 12902 USF Magnolia Dr, Tampa, FL 33612 USA
关键词
Epigenetic modification; hepcidin; interferon; jak inhibition; jak; stat pathway; mdm2; inhibition; myeloproliferative neoplasms; polycythemia vera; rare disease; INTERFERON-ALPHA; ESSENTIAL THROMBOCYTHEMIA; ROPEGINTERFERON ALPHA-2B; LOW TOXICITY; PHASE-II; HYDROXYUREA; RUXOLITINIB; RISK; EFFICACY; THERAPY;
D O I
10.1080/14728214.2021.1945579
中图分类号
R9 [药学];
学科分类号
1007 ;
摘要
Introduction: Polycythemia vera is a chronic hematologic malignancy frequently presented with constitutional symptoms and associated with an increased risk of thrombosis, hemorrhage, and progression to myelofibrosis or acute myeloid leukemia. Current treatment strategies reduce thrombohemorrhagic risk by controlling blood counts and inhibiting platelets, but often fail to address disease-related symptoms or biologically modify the disease. Areas covered: We review the current paradigm for treating polycythemia vera, highlight areas of unmet need, review therapeutic agents in late stage clinical development, and provide an overarching view of how these emerging agent may fit into the future armamentarium of polycythemia vera treatments. Expert opinion: The shift from focusing solely on secondary prevention of thrombohemorrhagic events to a comprehensive treatment strategy that additionally aims to improve quality of life and prevent disease progression has resulted in a rapidly evolving therapeutic landscape that promises to move the treatment of polycythemia vera out of antiquity into the modern age.
引用
收藏
页码:209 / 218
页数:10
相关论文
共 79 条
[1]  
Abu Zeinah G., 2019, ASH ANN M DEC 6 10 2
[2]  
Abu-Zeinah G, 2021, LEUKEMIA
[3]   Assessment and prognostic value of the European LeukemiaNet criteria for clinicohematologic response, resistance, and intolerance to hydroxyurea in polycythemia vera [J].
Alvarez-Larran, Alberto ;
Pereira, Arturo ;
Cervantes, Francisco ;
Arellano-Rodrigo, Eduardo ;
Hernandez-Boluda, Juan-Carlos ;
Ferrer-Marin, Francisca ;
Angona, Anna ;
Gomez, Montse ;
Muina, Begona ;
Guillen, Helga ;
Teruel, Anabel ;
Bellosillo, Beatriz ;
Burgaleta, Carmen ;
Vicente, Vicente ;
Besses, Carles .
BLOOD, 2012, 119 (06) :1363-1369
[4]  
[Anonymous], 2016, BLOOD
[5]   Exposure to Hydroxyurea and Pregnancy Outcomes in Patients With Sickle Cell Anemia [J].
Ballas, Samir K. ;
McCarthy, William F. ;
Guo, Nan ;
DeCastro, Laura ;
Bellevue, Rita ;
Barton, Bruce A. ;
Waclawiw, Myron A. .
JOURNAL OF THE NATIONAL MEDICAL ASSOCIATION, 2009, 101 (10) :1046-1051
[6]   Ropeginterferon alfa-2b versus phlebotomy in low-risk patients with polycythaemia vera (Low-PV study): a multicentre, randomised phase 2 trial [J].
Barbui, Tiziano ;
Vannucchi, Alessandro Maria ;
De Stefano, Valerio ;
Masciulli, Arianna ;
Carobbio, Alessandra ;
Ferrari, Alberto ;
Ghirardi, Arianna ;
Rossi, Elena ;
Ciceri, Fabio ;
Bonifacio, Massimiliano ;
Iurlo, Alessandra ;
Palandri, Francesca ;
Benevolo, Giulia ;
Pane, Fabrizio ;
Ricco, Alessandra ;
Carli, Giuseppe ;
Caramella, Marianna ;
Rapezzi, Davide ;
Musolino, Caterina ;
Siragusa, Sergio ;
Rumi, Elisa ;
Patriarca, Andrea ;
Cascavilla, Nicola ;
Mora, Barbara ;
Cacciola, Emma ;
Mannarelli, Carmela ;
Loscocco, Giuseppe Gaetano ;
Guglielmelli, Paola ;
Betti, Silvia ;
Lunghi, Francesca ;
Scaffidi, Luigi ;
Bucelli, Cristina ;
Vianelli, Nicola ;
Bellini, Marta ;
Finazzi, Maria Chiara ;
Tognoni, Gianni ;
Rambaldi, Alessandro .
LANCET HAEMATOLOGY, 2021, 8 (03) :E175-E184
[7]   A reappraisal of the benefit-risk profile of hydroxyurea in polycythemia vera: A propensity-matched study [J].
Barbui, Tiziano ;
Vannucchi, Alessandro Maria ;
Finazzi, Guido ;
Finazzi, Maria Chiara ;
Masciulli, Arianna ;
Carobbio, Alessandra ;
Ghirardi, Arianna ;
Tognoni, Gianni .
AMERICAN JOURNAL OF HEMATOLOGY, 2017, 92 (11) :1131-1136
[8]   White blood cell counts and thrombosis in polycythemia vera: a subanalysis of the CYTO-PV study [J].
Barbui, Tiziano ;
Masciulli, Arianna ;
Marfisi, Maria Rosa ;
Tognoni, Giovanni ;
Finazzi, Guido ;
Rambaldi, Alessandro ;
Vannucchi, Alessandro .
BLOOD, 2015, 126 (04) :560-561
[9]   Philadelphia chromosome-negative classical myeloproliferative neoplasms: revised management recommendations from European LeukemiaNet [J].
Barbuil, Tiziano ;
Tefferi, Ayalew ;
Vannucchi, Alessandro M. ;
Passamonti, Francesco ;
Silvers, Richard T. ;
Hoffman, Ronald ;
Verstovsek, Srdan ;
Mesa, Ruben ;
Kiladjian, Jean-Jacques ;
Hehlmann, Rudiger ;
Reiter, Andreas ;
Cervantes, Francisco ;
Harrison, Claire ;
Mc Mullin, Mary Frances ;
Hasselbalch, Hans Carl ;
Koschmieder, Steffen ;
Marchetti, Monia ;
Bacigalupo, Andrea ;
Finazzil, Guido ;
Kroeger, Nicolaus ;
Griesshammer, Martin ;
Birgegard, Gunnar ;
Barosi, Giovanni .
LEUKEMIA, 2018, 32 (05) :1057-1069
[10]   INCREASED INCIDENCE OF ACUTE-LEUKEMIA IN POLYCYTHEMIA-VERA ASSOCIATED WITH CHLORAMBUCIL THERAPY [J].
BERK, PD ;
GOLDBERG, JD ;
SILVERSTEIN, MN ;
WEINFELD, A ;
DONOVAN, PB ;
ELLIS, JT ;
LANDAW, SA ;
LASZLO, J ;
NAJEAN, Y ;
PISCIOTTA, AV ;
WASSERMAN, LR .
NEW ENGLAND JOURNAL OF MEDICINE, 1981, 304 (08) :441-447