Investigating Low-Dose Subcutaneous Decitabine Treatment for Patients With Myelofibrosis
According to phase 2 prospective study results, a high International Prognostic Scoring System (IPSS) risk score, high baseline fetal hemoglobin level, and sustained decreases in circulating CD34+ cell counts were associated with a response to hypomethylating agent (HMA) decitabine treatment for patients with myelofibrosis, supporting the merit of additional exploration of the utilization of solo or combination HMA therapy for these patients.
“Myelofibrosis in the chronic phase is a challenging disease entity to treat, and conventional treatment options are geared towards symptom palliation,” said lead study investigator Chenyu Lin, MD, Duke University School of Medicine, Durham, North Carolina, and coauthors.
This multicenter trial included 21 patients with MF (18 chronic-phase, 2 accelerated-phase, 1 blast-phase) who were treated with a 10-day schedule of subcutaneous decitabine at 0.3 mg/kg per day. Results demonstrated an OR rate of 33% (95% [confidence interval] CI, 15 to 57), which primarily manifested as an improvement in cytopenias. The median duration of response was 7 months, with a range of 3 to 44 months.
According to the measured safety profile, all patients experienced at least 1 grade 3 or 4 cytopenia. Researchers noted non-hematologic toxicities were less frequent. Those most often seen were fatigue, anorexia, and hypocalcemia.
“Given the lack of effective therapies in myelofibrosis with severe cytopenias, this study supports further investigation into the use of hypomethylating agents as single agents or in combination therapies,” concluded Dr Lin and colleagues.
Source:
Lin C, Patel A, Huo D, et al. A multicenter phase 2 clinical trial of low-dose subcutaneous decitabine in myelofibrosis. Blood Adv. Published online September 9, 2024. doi: 10.1182/bloodadvances.2024013215
