Secondary end points
| . | Cohort 1 No RUX NTD (n = 22) . | Cohort 3A RUX NTD (n = 14) . | Cohort 2 No RUX TD (n = 21) . | Cohort 3B RUX TD (n = 38) . |
|---|---|---|---|---|
| Time to, and duration of, response | ||||
| Evaluable patients, n | 3 | 2 | 2 | 10 |
| Time to anemia response,∗ d; median (min, max) | 63.0 (41, 68) | 63.5 (42, 85) | 2.0 (2, 2) | 37.0 (2, 71) |
| Longest duration of anemia response,† d; median (min, max) | 126.0 (84, 1163) | 88.5 (84, 93) | NA (602, 644) | 448.0 (85, 1582) |
| Transfusion burden reduction‡ | ||||
| Evaluable patients, n | 21 | 38 | ||
| Patients with transfusion burden reduction during primary treatment period, n (%) | ||||
| Primary treatment period | – | – | 10 (47.6) | 19 (50.0) |
| Entire treatment period | – | – | 10 (47.6) | 20 (52.6) |
| Hb levels§ | ||||
| Evaluable patients, n | 22 | 14 | 21 | 38 |
| Hb increase, g/dL; median (range) | ||||
| Primary treatment period | 0.8 (−1.0, 2.6) | 1.2 (0.2, 2.0) | 0.4 (−1.1, 1.9) | 0.2 (−1.7, 2.5) |
| Entire treatment period | 0.8 (−1.0, 3.0) | 1.2 (0.2, 2.4) | 0.4 (−1.1, 2.7) | 0.3 (−1.8, 2.6) |
| Symptom and fatigue reduction | ||||
| Symptom evaluable patients, n | 22 | 14 | 21 | 38 |
| Patients with mean MPN-SAF TSS of ≥50% reduction from baseline, n (%) | ||||
| Primary treatment period | 2 (9.1) | 3 (21.4) | 2 (9.5) | 6 (15.8) |
| Entire treatment period | 2 (9.1) | 3 (21.4) | 2 (9.5) | 6 (15.8) |
| Patients with mean MPN-SAF fatigue of ≥50% reduction from baseline, n (%) | ||||
| Primary treatment period | 5 (22.7) | 3 (21.4) | 1 (4.8) | 5 (13.2) |
| Entire treatment period | 4 (18.2) | 3 (21.4) | 0 | 5 (13.2) |
| . | Cohort 1 No RUX NTD (n = 22) . | Cohort 3A RUX NTD (n = 14) . | Cohort 2 No RUX TD (n = 21) . | Cohort 3B RUX TD (n = 38) . |
|---|---|---|---|---|
| Time to, and duration of, response | ||||
| Evaluable patients, n | 3 | 2 | 2 | 10 |
| Time to anemia response,∗ d; median (min, max) | 63.0 (41, 68) | 63.5 (42, 85) | 2.0 (2, 2) | 37.0 (2, 71) |
| Longest duration of anemia response,† d; median (min, max) | 126.0 (84, 1163) | 88.5 (84, 93) | NA (602, 644) | 448.0 (85, 1582) |
| Transfusion burden reduction‡ | ||||
| Evaluable patients, n | 21 | 38 | ||
| Patients with transfusion burden reduction during primary treatment period, n (%) | ||||
| Primary treatment period | – | – | 10 (47.6) | 19 (50.0) |
| Entire treatment period | – | – | 10 (47.6) | 20 (52.6) |
| Hb levels§ | ||||
| Evaluable patients, n | 22 | 14 | 21 | 38 |
| Hb increase, g/dL; median (range) | ||||
| Primary treatment period | 0.8 (−1.0, 2.6) | 1.2 (0.2, 2.0) | 0.4 (−1.1, 1.9) | 0.2 (−1.7, 2.5) |
| Entire treatment period | 0.8 (−1.0, 3.0) | 1.2 (0.2, 2.4) | 0.4 (−1.1, 2.7) | 0.3 (−1.8, 2.6) |
| Symptom and fatigue reduction | ||||
| Symptom evaluable patients, n | 22 | 14 | 21 | 38 |
| Patients with mean MPN-SAF TSS of ≥50% reduction from baseline, n (%) | ||||
| Primary treatment period | 2 (9.1) | 3 (21.4) | 2 (9.5) | 6 (15.8) |
| Entire treatment period | 2 (9.1) | 3 (21.4) | 2 (9.5) | 6 (15.8) |
| Patients with mean MPN-SAF fatigue of ≥50% reduction from baseline, n (%) | ||||
| Primary treatment period | 5 (22.7) | 3 (21.4) | 1 (4.8) | 5 (13.2) |
| Entire treatment period | 4 (18.2) | 3 (21.4) | 0 | 5 (13.2) |
Data are n (%) unless otherwise indicated.
TSS, total symptom score; max, maximum; min, minimum; NA, not applicable; RUX, ruxolitinib.
Time to anemia response for cohorts 1 and 3A defined as time between first administration of luspatercept and the first Hb increase of ≥1.5 g/dL from a baseline that starts a consecutive 84-day period of consecutive increase ≥1.5 g/dL without RBC transfusions; for cohorts 2 and 3B defined as time between first administration of LUSPA and the first day of an RBC transfusion–free period of 12 weeks between day 1 and day 168 (primary treatment period). Assessed only in evaluable patients with anemia response.
Duration of anemia response defined as last day of longest response, first date of longest response +1, assessed from day 1 to end of treatment (entire treatment period). Median duration of anemia response was not calculable for cohort 2 because of cohort 2 only containing 2 patients.
Patients achieving ≥50% RBC transfusion burden reduction over any 12-week period.
Change from baseline calculated based on average Hb measurements collected during the period, Hb measures followed the 14/3 rule (only Hb values >14 days after transfusion used unless there is another transfusion within 3 days after Hb assessment).