Randomized Trial of Lower-Dose Roxadustat Efficacy and Safety in Non-Dialysis-Dependent CKD-Associated Anemia

低剂量罗沙司他治疗非透析依赖性慢性肾病相关性贫血的随机试验

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Abstract

INTRODUCTION: Different starting doses of roxadustat are used for treating anemia in chronic kidney disease (CKD). We tested the noninferiority of weight-based lower starting dose compared with standard starting dose roxadustat for anemia in stage 3 to 5 CKD without dialysis. METHODS: Patients were randomized (1:1) and stratified by CKD stage to receive weight-based standard (< 60 kg: 70 mg 3 times per week [TIW]; ≥ 60 kg: 100 mg TIW) or 1-step-lower (< 60 kg: 50 mg TIW; ≥ 60 kg: 70 mg TIW) roxadustat starting dose for 16 weeks. The primary endpoint was mean hemoglobin change from baseline over weeks 12 to 16. The secondary endpoints included the proportion achieving hemoglobin 100 to 120 g/l, hemoglobin variability, and rescue therapy. RESULTS: Overall, 254 patients were randomized. The mean (SD) baseline hemoglobin was 89.88 (6.90) g/l. Most patients had stage 4 (39.0%) or stage 5 (40.2%) CKD. Mean hemoglobin increased from baseline at weeks 12 to 16 (lower: 21.57 g/l; standard: 26.35 g/l), but noninferiority was not met. A comparable proportion achieved hemoglobin of 100 to 120 g/l (lower: 46.0%; standard: 47.2%). The hemoglobin increase was comparable in CKD stage 3 to 4, but less with the lower dose in CKD stage 5 (17.28 vs. 26.71 g/l). The lower dose exhibited a lower hemoglobin rate of change (lower: 2.917; standard: 3.376) and less drug exposure. Drug-related adverse event rates were comparable. CONCLUSION: The proportion of patients who achieved the hemoglobin target was comparable between the doses. The lower starting dose had less hemoglobin fluctuation and is recommended for stage 3 to 4 CKD.

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