Tony Hagen is senior managing editor for The Center for Biosimilars®.
Investigators produced what they described as the first evidence supporting the use of biosimilar erythropoiesis-stimulating agents (ESAs) in patients with myelofibrosis-related anemia.
A retrospective study of patients with anemia in myelofibrosis (MF) treated with biosimilar erythropoiesis-stimulating agents (ESAs) demonstrated this to be an effective and well-tolerated option, according to data to be presented at the EHA20 Virtual, the 25th European Hematology Association Congress.
Investigators said ESAs are used to manage anemia in hematological malignancies, but scarce evidence exists for the use of these agents in the treatment of MF, and available results are divergent: Response rates range from 23% to 69%, according to the abstract presented at EHA25 Virtual. Until this study, there were no data on the use of biosimilar ESAs in this setting, investigators said.
The Italian study enrolled patients (N = 40) with MF who received biosimilar ESA (B-ESA) for at least 1 month to treat anemia (hemoglobin [Hb] ≤10 g/dL). B-ESA was given off label with patient consent.
Investigators reported anemia response (AR), without significant toxicities, and concluded that patients who achieved AR demonstrated a significant survival advantage compared with those who had no response (NR).
They also concluded that transfusion dependency at baseline was a negative predictor for AR, suggesting that outcomes could be improved via earlier B-ESA treatment in patients with MF.
AR included complete and partial responses (CR and PR, respectively). PR was defined as a transfusion decrease of more than 50% or sustained Hb increase between 1 and 2 g/dL.
The study included slightly more male than female patients and 21 patients with primary MF and 19 with secondary MF. Median age at baseline was 73 years. At start of treatment, 17.5% of patients were at high risk and 70% and 12.5% of patients at intermediate-2 and intermediate-1 risk MF, respectively.
Patients began treatment with B-ESA at a median of 8.5 months from MF diagnosis, and the median dose of B-ESA was 40,000 U/week subcutaneously. Investigators said 29 patents (72.5%) received B-ESA before, at start of, or during ruxolitinib treatment.
The median Hb level at baseline was 9.2 g/dL (range, 8.2-10.0), and just 3 patients were transfusion dependent.
Investigators said 34 patients (85%) achieved an AR, with 28 CR (70%), 6 PR (15%), and 6 NR (15%). The median time to response was 1.7 months, and median B-ESA exposure time was 12.4 months. Six patients (17%) lost response to therapy after a median 12.9 months.
“The only significant predictor of poor response rate was transfusion dependency at baseline (Fisher’s exact test, P = .002),” investigators wrote.
Inzoli E, Pugliese N, Renso R, et al. Use of biosimilar erythropoiesis stimulating agents for anemia in myelofibrosis: a multicenter real-life experience on 40 patients. Presented at: EHA25 Virtual; June 11-21, 2020. Abstract EP1119. library.ehaweb.org/eha/2020/eha25th/293608/elena.inzoli.use.of.biosimilar.erythropoiesis.stimulanting.agents.for.anemia.html?f=listing%3D0%2Abrowseby%3D8%2Asortby%3D1%2Asearch%3Dbiosimilar