Telomerase Inhibitor Imetelstat in Patients with Essential Thrombocythemia
Author(s) -
Gabriela M. Baerlocher,
Elisabeth Oppliger Leibundgut,
Oliver G. Ottmann,
Gary Spitzer,
Olatoyosi Odenike,
Michael A. McDevitt,
Alexander Röth,
Michael Daskalakis,
Bart Burington,
Monic J. Stuart,
David S. Snyder
Publication year - 2015
Publication title -
new england journal of medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 19.889
H-Index - 1030
eISSN - 1533-4406
pISSN - 0028-4793
DOI - 10.1056/nejmoa1503479
Subject(s) - essential thrombocythemia , medicine , adverse effect , gastroenterology , neutropenia , platelet , surgery , chemotherapy
Imetelstat, a 13-mer oligonucleotide that is covalently modified with lipid extensions, competitively inhibits telomerase enzymatic activity. It has been shown to inhibit megakaryocytic proliferation in vitro in cells obtained from patients with essential thrombocythemia. In this phase 2 study, we investigated whether imetelstat could elicit hematologic and molecular responses in patients with essential thrombocythemia who had not had a response to or who had had unacceptable side effects from prior therapies.
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