Definitions of transfusion-dependence and anemia response criteria in MDSs
Clinical trial . | TDA . | TRA . | Non-TDA . | Response criteria for TDA . | Response criteria for non-TDA . |
|---|---|---|---|---|---|
| Epoetin alfa Phase 3 study in low-risk MDS Fenaux et al24 | 1-4 units in 8 wk before baseline visit | Not applicable | Hb ≤10 g/dL without transfusions in the prior 8 wk | TI per IWG-2006 No transfusions for ≥8 consecutive weeks + increase in Hb by ≥1.5 g/dL Baseline Hb value taken before the last transfusion preceding enrollment | IWG-2006 ≥1.5 g/dL increase in Hb for ≥8 wk Modified IWG-2006 Increase in Hb ≥1.5 g/dL lasting <8 wk was considered a response if epoetin alfa was discontinued and when restarting at lower dose, Hb increased by ≥1.5 g/dL |
| Darbepoetin alpha Phase 3 study in low-risk MDS Platzbecker et al25 | 1-4 units in each of 2 consecutive 8-wk periods before randomization | Not applicable | Not applicable | TI per IWG-2006 No transfusions over ≥8 consecutive weeks + increase in Hb by ≥1.5 g/dL | Not applicable |
| MEDALIST Phase 3 study Luspatercept vs placebo in MDS with ring sideroblasts Fenaux et al26 | ≥2 units in 8 wk during the 16 wk before randomization | Not applicable | Not applicable | TI rate at week 24 No transfusions over ≥8 consecutive weeks | Not applicable |
| COMMANDS Phase 3 study Luspatercept vs epoetin alfa in low-risk MDS Platzbecker et al27 | 2-6 units in 8 wk for ≥8 wk before randomization | Not applicable | Not applicable | TI rate at week 24 No transfusions over ≥12 consecutive weeks + concurrent mean Hb increase ≥1.5 g/dL | Not applicable |
Clinical trial . | TDA . | TRA . | Non-TDA . | Response criteria for TDA . | Response criteria for non-TDA . |
|---|---|---|---|---|---|
| Epoetin alfa Phase 3 study in low-risk MDS Fenaux et al24 | 1-4 units in 8 wk before baseline visit | Not applicable | Hb ≤10 g/dL without transfusions in the prior 8 wk | TI per IWG-2006 No transfusions for ≥8 consecutive weeks + increase in Hb by ≥1.5 g/dL Baseline Hb value taken before the last transfusion preceding enrollment | IWG-2006 ≥1.5 g/dL increase in Hb for ≥8 wk Modified IWG-2006 Increase in Hb ≥1.5 g/dL lasting <8 wk was considered a response if epoetin alfa was discontinued and when restarting at lower dose, Hb increased by ≥1.5 g/dL |
| Darbepoetin alpha Phase 3 study in low-risk MDS Platzbecker et al25 | 1-4 units in each of 2 consecutive 8-wk periods before randomization | Not applicable | Not applicable | TI per IWG-2006 No transfusions over ≥8 consecutive weeks + increase in Hb by ≥1.5 g/dL | Not applicable |
| MEDALIST Phase 3 study Luspatercept vs placebo in MDS with ring sideroblasts Fenaux et al26 | ≥2 units in 8 wk during the 16 wk before randomization | Not applicable | Not applicable | TI rate at week 24 No transfusions over ≥8 consecutive weeks | Not applicable |
| COMMANDS Phase 3 study Luspatercept vs epoetin alfa in low-risk MDS Platzbecker et al27 | 2-6 units in 8 wk for ≥8 wk before randomization | Not applicable | Not applicable | TI rate at week 24 No transfusions over ≥12 consecutive weeks + concurrent mean Hb increase ≥1.5 g/dL | Not applicable |
| Established anemia response criteria . | Definitions . | Erythroid response . |
|---|---|---|
| IWG-2006 Cheson et al28 | Screening period, 8 wk for evaluation of transfusion burden and baseline Hb Pretreatment Hb <11 g/dL TDA (≥4 units in 8 wk for Hb <9 g/dL) TI (<4 units in 8 wk for Hb <9 g/dL) | ≥1.5 g/dL increase in Hb for ≥8 wk Reduction of ≥4 units transfusions/8 wk compared with the pretreatment transfusion in the prior 8 wk |
| Revised IWG-2018 Platzbecker et al29 | Screening period, 16 wk for evaluation of transfusion burden and baseline Hb Baseline Hb: Mean of all available Hb values during 16-wk screening period. Values before transfusion should be used for TD patients and should be ≥7 d apart Pretreatment Hb <10 g/dl NTD (0 units in 16 wk) LTB (3-7 units in 16 wk in at least 2 transfusion episodes, maximum 3 in 8 wk) HTB (≥8 units in 16 wk, ≥4 in 8 wk) | NTD Hb ≥1.5 g/dL for ≥8 wk over 16-24 wk compared with the lowest mean of 2 Hb values within 16 wk before treatment LTB TI, defined by the absence of transfusions for ≥8 wk over 16-24 wk HTB Major response: TI, defined by the absence of transfusions for ≥8 wk over 16-24 wk Minor response: Transfusion reduction by ≥50% over a minimum of 16 wk Same transfusion policy should be applied compared with 16 wk before treatment |
| Established anemia response criteria . | Definitions . | Erythroid response . |
|---|---|---|
| IWG-2006 Cheson et al28 | Screening period, 8 wk for evaluation of transfusion burden and baseline Hb Pretreatment Hb <11 g/dL TDA (≥4 units in 8 wk for Hb <9 g/dL) TI (<4 units in 8 wk for Hb <9 g/dL) | ≥1.5 g/dL increase in Hb for ≥8 wk Reduction of ≥4 units transfusions/8 wk compared with the pretreatment transfusion in the prior 8 wk |
| Revised IWG-2018 Platzbecker et al29 | Screening period, 16 wk for evaluation of transfusion burden and baseline Hb Baseline Hb: Mean of all available Hb values during 16-wk screening period. Values before transfusion should be used for TD patients and should be ≥7 d apart Pretreatment Hb <10 g/dl NTD (0 units in 16 wk) LTB (3-7 units in 16 wk in at least 2 transfusion episodes, maximum 3 in 8 wk) HTB (≥8 units in 16 wk, ≥4 in 8 wk) | NTD Hb ≥1.5 g/dL for ≥8 wk over 16-24 wk compared with the lowest mean of 2 Hb values within 16 wk before treatment LTB TI, defined by the absence of transfusions for ≥8 wk over 16-24 wk HTB Major response: TI, defined by the absence of transfusions for ≥8 wk over 16-24 wk Minor response: Transfusion reduction by ≥50% over a minimum of 16 wk Same transfusion policy should be applied compared with 16 wk before treatment |
HTB, high transfusion burden; LTB, low transfusion burden; NTD, non–transfusion dependent; TI, transfusion independent.