Open‐label, Phase 2 study of roxadustat for the treatment of anemia in patients receiving chemotherapy for non‐myeloid malignancies

Abstract

Anemia is a common side effect of myelosuppressive chemotherapy; however, chemotherapy‐induced anemia (CIA) management options are suboptimal. We evaluated the efficacy and safety of roxadustat in this setting. This open‐label Phase 2 study included patients with non‐myeloid malignancies and CIA (hemoglobin [Hb] ≤10 g/dL) who had planned concurrent myelosuppressive chemotherapy for ≥8 additional weeks. Oral roxadustat was administered for ≤16 weeks (starting dose 2.0 or 2.5 mg/kg, then titrated every 4 weeks). The primary efficacy endpoint was mean maximum change in Hb within 16 weeks of baseline without red blood cell (RBC) transfusion. Patients were assigned to roxadustat 2.0 (n = 31) or 2.5 mg/kg (n = 61) starting doses, and 89 were assessed for efficacy. The mean (standard deviation) maximum Hb change from baseline without RBC transfusion was 2.4 (1.5) and 2.5 (1.5) g/dL in the roxadustat 2.0 and 2.5 mg/kg groups, respectively. Median (range) time to Hb increase of ≥2 g/dL was 71 (57–92) days. Twelve patients (14.5%) had RBC transfusions (Week 5 to the end of treatment). Roxadustat was efficacious regardless of tumor type and chemotherapy regimen. Deep vein thrombosis (DVT) and pulmonary embolism (PE) occurred in 14 (15.2%) and nine (9.8%) patients, respectively, and three had serious adverse events attributed to roxadustat in the opinion of the investigators (PE: n = 2 [2.2%]; DVT: n = 1 [1.1%]). Roxadustat increased Hb in patients with CIA regardless of tumor type and chemotherapy regimen. Adverse events were consistent with observations in patients with advanced‐stage malignancies.

Anemia in patients receiving anticancer treatments: focus on novel therapeutic approaches

“…However, this difference did not reach statistical significance ( p =0.22) ( 116 ). A recent phase 2 open-label trial has investigated the efficacy and safety of Roxadustat for CCRA in various non-myeloid malignancies, including pancreas, breast, lung, and ovarian cancer, who have planned concurrent myelosuppressive therapies ( 117 ). Subgroup analyses demonstrated the efficacy of Roxadustat in correcting Hb regardless of tumor type and chemotherapy received.…”

A preliminary study of roxadustat in the treatment of aplastic anemia patients with inadequate erythroid responses

“…Roxadustat is currently approved for treating anemia in CKD in China, Japan, Chile, South Korea, Europe, and the UK [9,[15][16][17][18], and has shown efficacy in improving anemia in patients with chemotherapy and low-risk myelodysplastic syndrome (MDS) [9,19]. Hence, we hypothesized that roxadustat might have a therapeutic effect in AA patients with inadequate erythroid responses to immunosuppressive therapy.…”