Table 1.
Targeted treatment options for anemic lower-risk MDS patients.
| Therapeutic Compound | Phase | Included Patients | Ongoing/Recent Trial Number | Efficacy | Reference |
|---|---|---|---|---|---|
| EPO-α | III | LR-MDS patients with Hb 10 g/dL, serum erythropoietin <500 mU/mL | NCT01381809 | HI-E: 45.9% vs. 4.4% (placebo) | [5] |
| Luspatercept | III | RBC-transfusion depended, RS+ LR-MDS patients | NCT02631070 | HI-E: 52.9% vs. 11.8% (placebo) | [5] |
| Lenalidomide | III | RBC-transfusion depended LR-MDS patients with del5q | NCT00179621 | RBC-TI: 42.6–56.1% vs. 5.9% (placebo) | [5] |
| Roxadustat | III | LR-MDS patients with low RBD-transfusion burden (LTB) | NCT03263091 | ongoing | ongoing |
| Imetelstat | II/III | RBC-transfusion depended LR-MDS patients relapsed/refractory to ESA | NCT02598661 | HI-E: 71%, RBC-TI: 37% | [5], ongoing |