Essential thrombocythemia, myelofibrosis respond to telomere targeting
Treatment with a drug that targets the chromosome-lengthening enzyme telomerase led to hematologic responses in all treated patients with essential thrombocythemia, a preliminary clinical trial showed.
In 16 of 18 cases, patients achieved complete hematologic responses with imetelstat. Additionally, seven of eight patients with Janus kinase 2 (JAK2) V617F mutations had molecular responses, and mutant allele burdens were reduced by as much as 66%.
Most adverse events were mild or moderate, and grade ≥3 neutropenia occurred in four of the 18 patients. All 18 patients had at least one abnormal liver-function test, as reported online in the New England Journal of Medicine.
“Imetelstat … had a clinically significant effect on disease burden in patients with essential thrombocythemia who had not had a response to previous treatment or who had had unacceptable side effects from conventional therapies,” Gabriela M. Baerlocher, MD, of University Hospital of Bern in Switzerland, and co-authors wrote in conclusion.
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