Roxadustat Shows Noninferiority to rHuEPO-α for Chemotherapy-Induced Anemia Treatment

Roxadustat may be a viable alternative to rHuEPO-α for CIA management, particularly in resource-limited settings where oral administration offers logistical advantages, according to a recent open-label, phase 3 study.

The study evaluated the efficacy and safety of roxadustat, a hypoxia-inducible factor prolyl hydroxylase inhibitor, for chemotherapy-induced anemia (CIA) in patients with nonmyeloid malignancies in China. A total of 159 adults were randomized 1:1 to receive either oral roxadustat or subcutaneous recombinant human erythropoietin-α (rHuEPO-α) three times weekly for 12 weeks. The primary endpoint was the change in hemoglobin (Hb) levels from baseline to weeks 9 to 13.

Results showed that roxadustat achieved a least-squares mean (LSM) increase in Hb of 17.1 g/L, which was comparable to the 15.4 g/L increase with rHuEPO-α. Noninferiority was established with the lower bound of the 97.5% confidence interval for the difference (-3.4 g/L) above the predefined margin (-6.6 g/L). Secondary endpoints, including patient-reported outcomes and the percentage of responders, also supported noninferiority. Roxadustat had a higher compliance rate compared with rHuEPO-α (65.4% vs. 39.2%), potentially due to its oral formulation. Adverse events were comparable between groups, with no drug-related deaths reported.

While the results indicate that roxadustat may be a viable alternative to rHuEPO-α for CIA management, especially in a resource-constrained setting where its oral administration provides practical benefits. However, the open-label design and short duration (12 weeks) limit the study's ability to assess long-term outcomes and rare adverse events. The COVID-19 pandemic also introduced potential biases, particularly in treatment adherence and follow-up.

“These findings could have far-reaching implications for changing clinical practice as chemotherapy remains the major treatment modality for cancer in much of the world, and CIA prevalence is high,” the study authors concluded. “In addition to supporting a secondary indication for this new class of therapeutics, the convenience of an oral pill formulation may potentially lead to improved patient compliance as observed in this study.”

Reference
Lu S, Wu J, Jiang J, et al. Efficacy and Safety of Roxadustat for Anemia in Patients Receiving Chemotherapy for Nonmyeloid Malignancies: A Randomized, Open-Label, Active-Controlled Phase III Study. J Clin Oncol. 2025;43(2):143-153. doi:10.1200/JCO.23.02742