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

Publication Title

Blood Adv

Document Type

Article

Publication Date

11-26-2024

Keywords

oregon; portland; chiles; Humans; Decitabine; Primary Myelofibrosis; Male; Female; Aged; Middle Aged; Azacitidine; Aged, 80 and over; Treatment Outcome; Injections, Subcutaneous; Adult; Prospective Studies; Antimetabolites, Antineoplastic

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 3/4 cytopenia. Nonhematologic toxicities were less frequent, with fatigue, anorexia, and hypocalcemia being the most common. Given the lack of effective therapies in MF with severe cytopenias, this study supports further investigation into the use of hypomethylating agents as single agents or in combination therapies. This trial was registered at www.ClinicalTrials.gov as #NCT00095784.

Area of Special Interest

Cancer

Specialty/Research Institute

Oncology

Specialty/Research Institute

Hematology

DOI

10.1182/bloodadvances.2024013215

Plum Print visual indicator of research metrics
PlumX Metrics
  • Usage
    • Abstract Views: 3
  • Captures
    • Readers: 6
  • Mentions
    • News Mentions: 3
see details

Share

COinS