Definition of red cell transfusion-dependence and anemia response criteria in MF
| Clinical trial . | TDA . | TRA . | Non-TDA . | Response criteria for TDA . | Response criteria for non-TDA . |
|---|---|---|---|---|---|
| SIMPLIFY-1 Phase 3 study momelotinib vs ruxolitinib in JAKi-naïve MF Mesa et al19 | ≥4 units or Hb <8 g/dL in the 8 wk before randomization | Not defined | Not meeting criteria for TDA | TD rate at week 24 (proportion of patients who were TD) Transfusion rate (average number of units per subject-month) | TI rate at week 24 (proportion of patients who were TI) No transfusions and all Hb ≥8 g/dL in the prior 12 wk leading up to week 24 |
| SIMPLIFY-2 Phase 3 study momelotinib vs best available therapy in JAKi-treated MF Harrison et al20 | |||||
| MOMENTUM Phase 3 study momelotinib vs danazol in JAKi-treated MF Verstovsek et al21 | ≥4 units in the 8 wk before randomization with each transfusion trigger of Hb ≤9.5 g/dL | 1-3 units in the 8 wk before randomization | Hb <10 g/dL without transfusions in the 8 wk before randomization | TI rate at week 24 No transfusion and all Hb ≥8 g/dL in the prior 12 wk Transfusion rate (average number of units per subject-month) | ≥2 g/dL increase in Hb over a rolling ≥12 consecutive weeks |
| PERSIST-2 Phase 3 study pacritinib vs best available therapy in JAKi-treated MF Mascarenhas et al22 | ≥2 units per month in the 12 wk before cycle 1 d 1 | Not defined | Hb <10 g/dL without transfusions in the 12 wk before cycle 1 d 1 | TI rate at week 24 Absence of transfusions in the prior 12 wk | ≥2 g/dL increase in Hb for ≥8 wk before week 24 Applicable to patients with baseline Hb <10 g/dL |
| ACE-536-MF-001 Phase 2 study luspatercept in anemia-related to MF Gerds et a11 | 4-12 units in 12 wk before cycle 1 d 1 Transfusion threshold symptomatic anemia (Hb ≤9.5 g/dL) | Not defined | Hb ≤9.5 g/dL on ≥3 different days and without transfusions in 12 wk before cycle 1 d 1 | Absence of transfusions for ≥12 wk | ≥1.5 g/dL increase in baseline Hb for ≥12 wk without transfusion |
| Established anemia response criteria | |||||
| GALE Gale et al23 | ≥2 units per month in the prior 12 wk | Not defined | No transfusions in the prior 12 wk | No transfusions over any 12-wk interval with no minimum Hb requirement | No transfusions over any 12-wk interval with no minimum Hb requirement |
| IWG-MRT Tefferi et al18 | ≥6 units for Hb <8.5 g/dL in the 12 wk before screening Most recent transfusion within 28 d before screening | Not defined | Not meeting criteria for TDA | No transfusions over a rolling ≥12 consecutive weeks, +Hb ≥8.5 g | ≥2 g/dL increase in baseline Hb If transfused in prior month, pretransfusion Hb used as baseline Applicable to patients with baseline Hb <10 g/dL |
| Clinical trial . | TDA . | TRA . | Non-TDA . | Response criteria for TDA . | Response criteria for non-TDA . |
|---|---|---|---|---|---|
| SIMPLIFY-1 Phase 3 study momelotinib vs ruxolitinib in JAKi-naïve MF Mesa et al19 | ≥4 units or Hb <8 g/dL in the 8 wk before randomization | Not defined | Not meeting criteria for TDA | TD rate at week 24 (proportion of patients who were TD) Transfusion rate (average number of units per subject-month) | TI rate at week 24 (proportion of patients who were TI) No transfusions and all Hb ≥8 g/dL in the prior 12 wk leading up to week 24 |
| SIMPLIFY-2 Phase 3 study momelotinib vs best available therapy in JAKi-treated MF Harrison et al20 | |||||
| MOMENTUM Phase 3 study momelotinib vs danazol in JAKi-treated MF Verstovsek et al21 | ≥4 units in the 8 wk before randomization with each transfusion trigger of Hb ≤9.5 g/dL | 1-3 units in the 8 wk before randomization | Hb <10 g/dL without transfusions in the 8 wk before randomization | TI rate at week 24 No transfusion and all Hb ≥8 g/dL in the prior 12 wk Transfusion rate (average number of units per subject-month) | ≥2 g/dL increase in Hb over a rolling ≥12 consecutive weeks |
| PERSIST-2 Phase 3 study pacritinib vs best available therapy in JAKi-treated MF Mascarenhas et al22 | ≥2 units per month in the 12 wk before cycle 1 d 1 | Not defined | Hb <10 g/dL without transfusions in the 12 wk before cycle 1 d 1 | TI rate at week 24 Absence of transfusions in the prior 12 wk | ≥2 g/dL increase in Hb for ≥8 wk before week 24 Applicable to patients with baseline Hb <10 g/dL |
| ACE-536-MF-001 Phase 2 study luspatercept in anemia-related to MF Gerds et a11 | 4-12 units in 12 wk before cycle 1 d 1 Transfusion threshold symptomatic anemia (Hb ≤9.5 g/dL) | Not defined | Hb ≤9.5 g/dL on ≥3 different days and without transfusions in 12 wk before cycle 1 d 1 | Absence of transfusions for ≥12 wk | ≥1.5 g/dL increase in baseline Hb for ≥12 wk without transfusion |
| Established anemia response criteria | |||||
| GALE Gale et al23 | ≥2 units per month in the prior 12 wk | Not defined | No transfusions in the prior 12 wk | No transfusions over any 12-wk interval with no minimum Hb requirement | No transfusions over any 12-wk interval with no minimum Hb requirement |
| IWG-MRT Tefferi et al18 | ≥6 units for Hb <8.5 g/dL in the 12 wk before screening Most recent transfusion within 28 d before screening | Not defined | Not meeting criteria for TDA | No transfusions over a rolling ≥12 consecutive weeks, +Hb ≥8.5 g | ≥2 g/dL increase in baseline Hb If transfused in prior month, pretransfusion Hb used as baseline Applicable to patients with baseline Hb <10 g/dL |
IWG-MRT, IWG for Myelofibrosis Research and Treatment; TD, transfusion-dependent; TI, transfusion independent; TRA, transfusion-requiring anemia.