A Single-arm Trial of Roxadustat Combined With Retinoic Acid in the Treatment of Refractory Low-risk MDS

A Single-arm Trial of Roxadustat Combined With Retinoic Acid in the Treatment of Refractory Low-risk MDS

Roxadustat has been approved for low-risk MDS clinical trials, but the trial results are not available. For refractory low-risk MDS, the effective rate of roxadustat treatment is about 20-30%, and roxadustat combined with retinoic acid may have better efficacy in the treatment of refractory low-risk MDS.

Patients with low-risk myelodysplastic syndrome who are refractory to the regular treatments have to live on transfusions which lead to poor quality of life (QoL) and survival. Roxadustat, an oral hypoxia-inducible factor prolyl hydroxylase inhibitor, which increases both endogenous EPO and iron metabolism, has shown promising results in several phase 3 clinical trials for chronic kidney disease.

Roxadustat comprehensively improves iron metabolism by upregulating DCYTB and DMT1 through the HIF pathway, which can promote the iron absorption and utilization. Detail mechanisms include upregulating transferrin receptors to increase iron uptake, upregulating transferrin to promote iron transport and downregulating ferritin levels to indirectly improve iron absorption and transportation.

Patients with refractory low-risk MDS were selected and given roxadustat 150mg po qod combined with retinoic acid 20mg po bid.

Inclusion Criteria:

• Low-risk MDS unresponsive to first-line therapy

Exclusion Criteria:

• myelofibrosis
• Severe heart and kidney function bu'quan