Available therapies in LR-MDS
| Agent . | Mechanism of action . | Population . | Identifier/Trial Name . | Phase . | Status . | Ref. . |
|---|---|---|---|---|---|---|
| Epoietin alfa | ESA | LR-MDS with anemia | NCT01381809 EPOANE3021 | 3 | EMA approved | 5 |
| Darbepoietin alfa | ESA | LR-MDS with anemia | NCT00095264 | 2 | Off-label | 6 |
| Lenalidomide | Immune regulatory agent | LR-MDS del5(q) | NCT00179621 | 3 | FDA/EMA approved | 7 |
| LR-MDS without del(5q) | NCT01029262 | 3 | Off-label | 8 | ||
| Azacitidine | HMA | MDS with pancytopenia | NCT01720225 | 2/3 | FDA approved | 9 |
| Decitabine | HMA | MDS with pancytopenia | NCT01720225 | 2/3 | FDA approved | 9 |
| Eltrombopag | TPO mimetic | MDS without excess blasts, with thrombocytopenia or pancytopenia | NCT02928419 EQoL-MDS | 2 | Off-label | 10 |
| ATG/CSA | Immunosuppressive | LR-MDS with pancytopenia | Retrospective study | NA | Off-label | 11 |
| Luspatercept | TGF-β inhibitor | MDS-RS or with SF3B1 mutation | NCT02631070MEDALIST | 3 | FDA/EMA approved | 12 |
| LR-MDS with anemia | NCT:03682536 COMMANDS | 3 | Off-label | 13 |
| Agent . | Mechanism of action . | Population . | Identifier/Trial Name . | Phase . | Status . | Ref. . |
|---|---|---|---|---|---|---|
| Epoietin alfa | ESA | LR-MDS with anemia | NCT01381809 EPOANE3021 | 3 | EMA approved | 5 |
| Darbepoietin alfa | ESA | LR-MDS with anemia | NCT00095264 | 2 | Off-label | 6 |
| Lenalidomide | Immune regulatory agent | LR-MDS del5(q) | NCT00179621 | 3 | FDA/EMA approved | 7 |
| LR-MDS without del(5q) | NCT01029262 | 3 | Off-label | 8 | ||
| Azacitidine | HMA | MDS with pancytopenia | NCT01720225 | 2/3 | FDA approved | 9 |
| Decitabine | HMA | MDS with pancytopenia | NCT01720225 | 2/3 | FDA approved | 9 |
| Eltrombopag | TPO mimetic | MDS without excess blasts, with thrombocytopenia or pancytopenia | NCT02928419 EQoL-MDS | 2 | Off-label | 10 |
| ATG/CSA | Immunosuppressive | LR-MDS with pancytopenia | Retrospective study | NA | Off-label | 11 |
| Luspatercept | TGF-β inhibitor | MDS-RS or with SF3B1 mutation | NCT02631070MEDALIST | 3 | FDA/EMA approved | 12 |
| LR-MDS with anemia | NCT:03682536 COMMANDS | 3 | Off-label | 13 |
CSA, cyclosporine A; EMA, European Medicines Agency; HMA, hypomethylating agent; NA, not applicable; TPO, thrombopoietin.