Roxadustat for the Treatment of Anemia in Patients with Lower-Risk MDS

Anemia is the predominant cytopenia in myelodysplastic syndromes (MDS), and treatment options are limited. Roxadustat, which is a hypoxia-inducible factor prolyl hydroxylase inhibitor, may be a potential treatment of anemia of chronic kidney disease. A study published in the American Journal of Hematology assessed the treatment’s efficacy.

In this phase 3, randomized, double-blind, placebo-controlled study, called MATTERHORN, researchers assessed the efficacy and safety of roxadustat in anemia of lower risk MDS in 24 patients. In an open-label (OL), dose-selection, lead-in phase, the population of interest was assigned to 1 of 3 roxadustat starting doses (n=8 each): 1.5, 2.0, and 2.5 mg/kg.

The primary endpoint of the OL phase was defined as the proportion of patients with transfusion independence (TI) for ≥8 consecutive weeks in the first 28 treatment weeks. The secondary endpoint was stipulated as the proportion of patients with a ≥50% reduction in RBC transfusions over any 8-week period compared with baseline. The researchers also assessed for adverse events. The follow-up period was 52 weeks.

According to the results, TI was achieved in 9 patients (37.5%) at 28 and 52 weeks. Seven of those patients were receiving 2.5 mg/kg dose when TI was achieved. The researchers observed a ≥50% reduction in RBC transfusions was achieved in 54.2% and 58.3% of patients at 28 and 52 weeks, respectively. Overall, the results were promising. Roxadustat dosed thrice weekly was well tolerated, with no serious adverse events, and no progression to acute myeloid leukemia.