Therapeutic options for MDS in 2023, licensed or recommended according to guidelines (amended)
| Procedure/treatment option United Sates: formally licensed US EU: formally licensed EU . | Lower-risk MDS . | Higher-risk MDS . | MDS-RS . | Del(5q) MDS . | Comment . |
|---|---|---|---|---|---|
| Transfusion therapy | Yes | Yes | Yes | Yes | Indicated for symptomatic anemia |
| Chelation therapy in TD patients∗ | Yes | If planned HSCT | Yes | Yes | If estimated survival >1-2 y ∗US and ∗EU |
| ESAs | Yes US and EU | Rarely | Yes US and EU | Yes US and EU | Firstline treatment for symptomatic anemia of all lower-risk MDSs. Poor response rate if S-EPO >500 (200) U/L. |
| Trombomimetics | If severe thrombocytopenia | No | No | No | Not licensed for MDS but can be used as in immune-mediated thrombocytopenia |
| Luspatercept | Yes US | No | Yes US, EU | No | Currently licensed only for MDS-RS; recent phase 3 study shows efficacy in other lower-risk MDSs as well |
| Immunosuppressive treatment | Occasionally | No | No | No | Younger patients; severe pancytopenia and hypo/normoplastic BM without high-risk genetics |
| Lenalidomide | No | No | No | Yes | Licensed for TD del(5q) MDS, at ESA failure/ineligibility |
| HMAs | Yes US | Yes US and EU | No | No | Could be used in LR-MDS if aggravating cytopenia or progression |
| Aza | Yes US | Yes US and EU | No | Yes US | Firstline treatment for higher-risk MDS. In United States also approved for lower-risk MDS. |
| Decitabine | Yes (IPSS ≥ INT-1) US | Yes US | No | Yes (IPSS ≥ INT-1) US | Licensed for AML in the EU Licensed for AML and MDS in the US |
| Oral decitabine/cedazuridine | Yes (IPSS ≥ INT-1) US | Yes (IPSS ≥ INT-1) | No | Yes (IPSS ≥ INT-1) US | Not inferior to HMA in randomized studies |
| HMA + venetoclax | No | See comment | No | No | Licensed for AML only. Phase 3 study for MDS is pending. Caution if used as a rescue treatment for MDS: toxicity is usually higher than in AML. |
| Targeted AML drugs (IDH1 + 2 inhibitors) | No | See comment | No | No | Not licensed for MDS. In clinical trials including AML and MDS. For other targeted drugs, see text. |
| Chemotherapy | No | See comment | No | No | Inferior to aza (not tested for CPX). May be relevant for MDS with AML-like genetics and as bridging therapy to allo-HSCT. |
| Allo-HSCT | If adverse risk profile | Yes | Occasionally, if refractory TD | If refractory TD or high-risk genetics | Complex decision making involving MDS risk, comorbidities, and patients’ preferences |
| Procedure/treatment option United Sates: formally licensed US EU: formally licensed EU . | Lower-risk MDS . | Higher-risk MDS . | MDS-RS . | Del(5q) MDS . | Comment . |
|---|---|---|---|---|---|
| Transfusion therapy | Yes | Yes | Yes | Yes | Indicated for symptomatic anemia |
| Chelation therapy in TD patients∗ | Yes | If planned HSCT | Yes | Yes | If estimated survival >1-2 y ∗US and ∗EU |
| ESAs | Yes US and EU | Rarely | Yes US and EU | Yes US and EU | Firstline treatment for symptomatic anemia of all lower-risk MDSs. Poor response rate if S-EPO >500 (200) U/L. |
| Trombomimetics | If severe thrombocytopenia | No | No | No | Not licensed for MDS but can be used as in immune-mediated thrombocytopenia |
| Luspatercept | Yes US | No | Yes US, EU | No | Currently licensed only for MDS-RS; recent phase 3 study shows efficacy in other lower-risk MDSs as well |
| Immunosuppressive treatment | Occasionally | No | No | No | Younger patients; severe pancytopenia and hypo/normoplastic BM without high-risk genetics |
| Lenalidomide | No | No | No | Yes | Licensed for TD del(5q) MDS, at ESA failure/ineligibility |
| HMAs | Yes US | Yes US and EU | No | No | Could be used in LR-MDS if aggravating cytopenia or progression |
| Aza | Yes US | Yes US and EU | No | Yes US | Firstline treatment for higher-risk MDS. In United States also approved for lower-risk MDS. |
| Decitabine | Yes (IPSS ≥ INT-1) US | Yes US | No | Yes (IPSS ≥ INT-1) US | Licensed for AML in the EU Licensed for AML and MDS in the US |
| Oral decitabine/cedazuridine | Yes (IPSS ≥ INT-1) US | Yes (IPSS ≥ INT-1) | No | Yes (IPSS ≥ INT-1) US | Not inferior to HMA in randomized studies |
| HMA + venetoclax | No | See comment | No | No | Licensed for AML only. Phase 3 study for MDS is pending. Caution if used as a rescue treatment for MDS: toxicity is usually higher than in AML. |
| Targeted AML drugs (IDH1 + 2 inhibitors) | No | See comment | No | No | Not licensed for MDS. In clinical trials including AML and MDS. For other targeted drugs, see text. |
| Chemotherapy | No | See comment | No | No | Inferior to aza (not tested for CPX). May be relevant for MDS with AML-like genetics and as bridging therapy to allo-HSCT. |
| Allo-HSCT | If adverse risk profile | Yes | Occasionally, if refractory TD | If refractory TD or high-risk genetics | Complex decision making involving MDS risk, comorbidities, and patients’ preferences |
BM, bone marrow; EU, European Union; INT, intermediate; LR-MDS, low-risk MDS; US, United States.
Licensed for treatment of chronic iron overload, irrespective of diagnosis. Variation in licensing patterns between countries.