Givinostat for the treatment of polycythemia vera

被引：2

作者：

Pieri, Lisa ^{[1
,2
]}

Guglielmelli, Paola ^{[1
,2
]}

Finazzi, Guido ^{[3
]}

Vannucchi, Alessandro M. ^{[1
,2
]}

机构：

[1] Univ Florence, Lab Congiunto MMPC, Azienda Osped Univ Careggi, Dept Expt & Clin Med, I-50134 Florence, Italy

[2] Azienda Osped Univ Careggi, Hematol Unit, Florence, Italy

[3] Osped Papa Giovanni XXIII, Hematol Unit, Bergamo, Italy

来源：

EXPERT OPINION ON ORPHAN DRUGS | 2014年 / 2卷 / 08期

关键词：

givinostat; histone deacethylase inhibitors; polycythemia vera; pruritus; DEACETYLASE INHIBITOR ITF2357; POST-ESSENTIAL THROMBOCYTHEMIA; INTERNATIONAL-WORKING-GROUP; TYROSINE KINASE JAK2; PRIMARY MYELOFIBROSIS; MYELOPROLIFERATIVE NEOPLASMS; PHASE-II; ANTILEUKEMIC ACTIVITY; ACTIVATING MUTATION; HEMATOPOIETIC STEM;

D O I：

10.1517/21678707.2014.934223

中图分类号：

R9 [药学];

学科分类号：

1007 ;

摘要：

Introduction: Polycythemia vera (PV) is a myeloproliferative neoplasm for which no curative treatment currently exists. However, the discovery of abnormally activated JAK/STAT signaling pushed the clinical development of JAK1/JAK2 inhibitors and other target drugs, including inhibitors of histone deacetylases (HDACi). Areas covered: We reviewed preclinical studies and clinical trials with givinostat, a Class I and II HDACi, through PubMed research and communications to the meeting of the most important hematology associations. Expert opinion: Proof of evidence has been obtained with givinostat earlier in JAK2V617F mutated cells and then in two clinical trials in patients with PV. Treatment resulted effective in inducing hematological response, symptomatic improvement and reduction of enlarged spleen, with good tolerability. Further clinical development is required to more clearly define the role of Givinostat in the therapeutic armamentarium of PV and exploit the full activity of the drug against the myeloproliferative clone.

引用

页码：841 / 850

页数：10

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