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A phase II study of G ivinostat in combination with hydroxycarbamide in patients with polycythaemia vera unresponsive to hydroxycarbamide monotherapy
Author(s) -
Finazzi Guido,
Vannucchi Alessandro M.,
Martinelli Vincenzo,
Ruggeri Marco,
Nobile Francesco,
Specchia Giorgina,
Pogliani Enrico Maria,
Olimpieri Odoardo Maria,
Fioritoni Giuseppe,
Musolino Caterina,
Cilloni Daniela,
Sivera Piera,
Barosi Giovanni,
Finazzi Maria Chiara,
Tollo Silvia Di,
Demuth Tim,
Barbui Tiziano,
Rambaldi Alessandro
Publication year - 2013
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/bjh.12332
Subject(s) - hydroxycarbamide , medicine , tolerability , anagrelide , polycythaemia , polycythemia vera , adverse effect , gastroenterology , pharmacology , essential thrombocythemia , chemotherapy
Summary Givinostat, a histone‐deacetylase inhibitor ( HDAC i), inhibits proliferation of cells bearing the JAK 2 V617F mutation and has shown significant activity with good tolerability in patients with chronic myeloproliferative neoplasms ( MPN ). In this multicentre, open‐label, phase II study, 44 patients with polycythaemia vera ( PV ), unresponsive to the maximum tolerated doses ( MTD ) of hydroxycarbamide ( HC ), were treated with Givinostat (50 or 100 mg/d) in combination with MTD of HC . The European LeukaemiaNet response criteria were used to assess the primary endpoint after 12 weeks of treatment. Complete or partial response was observed in 55% and 50% of patients receiving 50 or 100 mg of Givinostat, respectively. Control of pruritus was observed in 64% and 67% of patients in the 50 and 100 mg groups, respectively. The combination of Givinostat and HC was well tolerated: eight patients (18%) discontinued, four in each treatment arm; grade 3 adverse events were reported in one patient (4·5%) in each treatment arm. The combined use of Givinostat and HC was safe and clinically effective in HC ‐unresponsive PV patients.