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A multicenter phase 2 clinical trial of low-dose subcutaneous decitabine in myelofibrosis.


ABSTRACT:

Abstract

Myelofibrosis (MF) in the chronic phase is a challenging disease to treat, and conventional treatment options are geared toward symptom palliation. In this prospective, multicenter, phase 2 trial, 21 patients with MF (18 chronic phase, 2 accelerated phase, and 1 blast phase) were treated with a 10-day schedule of subcutaneous decitabine at 0.3 mg/kg per day. The overall response rate was 33% (95% confidence interval, 15-57), primarily manifested as an improvement in cytopenias. The median duration of response was 7 months (range, 3-44). A high International Prognostic Scoring System risk score, high baseline fetal hemoglobin level, and sustained decrease in circulating CD34+ cell counts were associated with response to decitabine. All patients experienced at least 1 grade

SUBMITTER: Lin C 

PROVIDER: S-EPMC11599980 | biostudies-literature | 2024 Nov

REPOSITORIES: biostudies-literature