Baseline demographics and disease characteristics in the intent-to-treat population
| . | 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) . | Total (N = 95) . |
|---|---|---|---|---|---|
| Age, y, median (min, max) | 69.5 (50, 89) | 65.0 (51, 81) | 75.0 (60, 88) | 72.0 (59, 83) | 71.0 (50, 89) |
| Male | 13 (59.1) | 7 (50.0) | 15 (71.4) | 23 (60.5) | 58 (61.1) |
| Race | |||||
| Asian | 2 (9.1) | 3 (21.4) | 1 (4.8) | 1 (2.6) | 7 (7.4) |
| Black or African American | 0 | 1 (7.1) | 0 | 2 (5.3) | 3 (3.2) |
| White | 16 (72.7) | 10 (71.4) | 18 (85.7) | 30 (78.9) | 74 (77.9) |
| Other | 1 (4.5) | 0 | 0 | 0 | 1 (1.1) |
| Not collected or reported | 3 (13.6) | 0 | 2 (9.5) | 5 (13.2) | 10 (10.5) |
| Ethnicity | |||||
| Hispanic or Latino | 2 (9.1) | 0 | 0 | 1 (2.6) | 3 (3.2) |
| Not Hispanic or Latino | 16 (72.7) | 14 (100.0) | 19 (90.5) | 32 (84.2) | 81 (85.3) |
| Unknown | 0 | 0 | 0 | 1 (2.6) | 1 (1.1) |
| Not reported | 4 (18.2) | 0 | 2 (9.5) | 4 (10.5) | 10 (10.5) |
| Disease diagnosis | |||||
| PMF | 15 (68.2) | 6 (42.9) | 13 (61.9) | 18 (47.4) | 52 (54.7) |
| Post-PV MF | 0 | 4 (28.6) | 2 (9.5) | 7 (18.4) | 13 (13.7) |
| Post-ET MF | 7 (31.8) | 4 (28.6) | 6 (28.6) | 13 (34.2) | 30 (31.6) |
| ECOG PS | |||||
| 0 | 9 (40.9) | 7 (50.0) | 3 (14.3) | 13 (34.2) | 32 (33.7) |
| 1 | 13 (59.1) | 7 (50.0) | 16 (76.2) | 19 (50.0) | 55 (57.9) |
| 2 | 0 | 0 | 2 (9.5) | 6 (15.8) | 8 (8.4) |
| Time since initial diagnosis to first screening | |||||
| ≤2 y | 10 (45.5) | 2 (14.3) | 6 (28.6) | 6 (15.8) | 24 (25.3) |
| >2 y | 12 (54.5) | 12 (85.7) | 15 (71.4) | 32 (84.2) | 71 (74.7) |
| Time on RUX before study, mo, median (min, max)∗ | — | 16.4 (5.1, 71.8) | — | 18.8 (6.7, 150.6) | 18.1 (5.1, 150.6) |
| DIPSS risk | |||||
| Intermediate-risk 1 | 1 (4.5) | 4 (28.6) | 1 (4.8) | 3 (7.9) | 9 (9.5) |
| Intermediate-risk 2 | 18 (81.8) | 9 (64.3) | 15 (71.4) | 30 (78.9) | 72 (75.8) |
| High-risk | 2 (9.1) | 1 (7.1) | 5 (23.8) | 5 (13.2) | 13 (13.7) |
| Unknown | 1 (4.5) | 0 | 0 | 0 | 1 (1.1) |
| Baseline transfusion burden per 12 wk† | |||||
| 4-5 units | — | — | 1 (4.8) | 6 (15.8) | 7 (11.9) |
| 6-12 units | — | — | 18 (85.7) | 32 (84.2) | 50 (84.7) |
| >12 units | — | — | 2 (9.5) | 0 | 2 (3.4) |
| Baseline hemoglobin levels (g/dL), median (min, max) | 8.7 (6.6, 9.7) | 8.3 (7.2, 9.2) | 7.3 (6.0, 9.5) | 8.2 (6.3, 9.9) | 8.3 (6.0, 9.9) |
| Baseline platelet categories (109/L) | |||||
| <100 | 6 (27.3) | 3 (21.4) | 8 (38.1) | 12 (31.6) | 29 (30.5) |
| 100-400 | 15 (68.2) | 10 (71.4) | 11 (52.4) | 24 (63.2) | 60 (63.2) |
| >400 | 1 (4.5) | 1 (7.1) | 2 (9.5) | 2 (5.3) | 6 (6.3) |
| Baseline serum erythropoietin (U/L), median (min, max) | 52.2 (7.0, 1996.6) | 157.5 (30.2, 2394.6) | 140.0 (15.9, 1594.4) | 164.6 (5.8, 1 977.2) | 133.3 (5.8, 2 394.6) |
| Baseline serum ferritin (ug/L), median (min, max) | 270.0 (27.0, 1456.7) | 136.7 (56.1, 1554.0) | 1168.2 (314.3, 4632.0) | 1 174.3 (170.2, 1 2189.0) | 759.8 (27.0, 1 2189.0) |
| Baseline spleen size,‡ cm, median (min, max)§ | 6.0 (1.0, 15.0) | 7.0 (1.0, 17.0) | 5.0 (2.0, 16.0) | 10.0 (0.0, 20.0) | — |
| . | 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) . | Total (N = 95) . |
|---|---|---|---|---|---|
| Age, y, median (min, max) | 69.5 (50, 89) | 65.0 (51, 81) | 75.0 (60, 88) | 72.0 (59, 83) | 71.0 (50, 89) |
| Male | 13 (59.1) | 7 (50.0) | 15 (71.4) | 23 (60.5) | 58 (61.1) |
| Race | |||||
| Asian | 2 (9.1) | 3 (21.4) | 1 (4.8) | 1 (2.6) | 7 (7.4) |
| Black or African American | 0 | 1 (7.1) | 0 | 2 (5.3) | 3 (3.2) |
| White | 16 (72.7) | 10 (71.4) | 18 (85.7) | 30 (78.9) | 74 (77.9) |
| Other | 1 (4.5) | 0 | 0 | 0 | 1 (1.1) |
| Not collected or reported | 3 (13.6) | 0 | 2 (9.5) | 5 (13.2) | 10 (10.5) |
| Ethnicity | |||||
| Hispanic or Latino | 2 (9.1) | 0 | 0 | 1 (2.6) | 3 (3.2) |
| Not Hispanic or Latino | 16 (72.7) | 14 (100.0) | 19 (90.5) | 32 (84.2) | 81 (85.3) |
| Unknown | 0 | 0 | 0 | 1 (2.6) | 1 (1.1) |
| Not reported | 4 (18.2) | 0 | 2 (9.5) | 4 (10.5) | 10 (10.5) |
| Disease diagnosis | |||||
| PMF | 15 (68.2) | 6 (42.9) | 13 (61.9) | 18 (47.4) | 52 (54.7) |
| Post-PV MF | 0 | 4 (28.6) | 2 (9.5) | 7 (18.4) | 13 (13.7) |
| Post-ET MF | 7 (31.8) | 4 (28.6) | 6 (28.6) | 13 (34.2) | 30 (31.6) |
| ECOG PS | |||||
| 0 | 9 (40.9) | 7 (50.0) | 3 (14.3) | 13 (34.2) | 32 (33.7) |
| 1 | 13 (59.1) | 7 (50.0) | 16 (76.2) | 19 (50.0) | 55 (57.9) |
| 2 | 0 | 0 | 2 (9.5) | 6 (15.8) | 8 (8.4) |
| Time since initial diagnosis to first screening | |||||
| ≤2 y | 10 (45.5) | 2 (14.3) | 6 (28.6) | 6 (15.8) | 24 (25.3) |
| >2 y | 12 (54.5) | 12 (85.7) | 15 (71.4) | 32 (84.2) | 71 (74.7) |
| Time on RUX before study, mo, median (min, max)∗ | — | 16.4 (5.1, 71.8) | — | 18.8 (6.7, 150.6) | 18.1 (5.1, 150.6) |
| DIPSS risk | |||||
| Intermediate-risk 1 | 1 (4.5) | 4 (28.6) | 1 (4.8) | 3 (7.9) | 9 (9.5) |
| Intermediate-risk 2 | 18 (81.8) | 9 (64.3) | 15 (71.4) | 30 (78.9) | 72 (75.8) |
| High-risk | 2 (9.1) | 1 (7.1) | 5 (23.8) | 5 (13.2) | 13 (13.7) |
| Unknown | 1 (4.5) | 0 | 0 | 0 | 1 (1.1) |
| Baseline transfusion burden per 12 wk† | |||||
| 4-5 units | — | — | 1 (4.8) | 6 (15.8) | 7 (11.9) |
| 6-12 units | — | — | 18 (85.7) | 32 (84.2) | 50 (84.7) |
| >12 units | — | — | 2 (9.5) | 0 | 2 (3.4) |
| Baseline hemoglobin levels (g/dL), median (min, max) | 8.7 (6.6, 9.7) | 8.3 (7.2, 9.2) | 7.3 (6.0, 9.5) | 8.2 (6.3, 9.9) | 8.3 (6.0, 9.9) |
| Baseline platelet categories (109/L) | |||||
| <100 | 6 (27.3) | 3 (21.4) | 8 (38.1) | 12 (31.6) | 29 (30.5) |
| 100-400 | 15 (68.2) | 10 (71.4) | 11 (52.4) | 24 (63.2) | 60 (63.2) |
| >400 | 1 (4.5) | 1 (7.1) | 2 (9.5) | 2 (5.3) | 6 (6.3) |
| Baseline serum erythropoietin (U/L), median (min, max) | 52.2 (7.0, 1996.6) | 157.5 (30.2, 2394.6) | 140.0 (15.9, 1594.4) | 164.6 (5.8, 1 977.2) | 133.3 (5.8, 2 394.6) |
| Baseline serum ferritin (ug/L), median (min, max) | 270.0 (27.0, 1456.7) | 136.7 (56.1, 1554.0) | 1168.2 (314.3, 4632.0) | 1 174.3 (170.2, 1 2189.0) | 759.8 (27.0, 1 2189.0) |
| Baseline spleen size,‡ cm, median (min, max)§ | 6.0 (1.0, 15.0) | 7.0 (1.0, 17.0) | 5.0 (2.0, 16.0) | 10.0 (0.0, 20.0) | — |
Baseline is defined as the last value on or before the first dose of luspatercept, or average if multiple values are present for the same date. Data are n (%) unless otherwise indicated.
DIPSS, Dynamic International Prognostic Scoring System; max, maximum; min, minimum; post-ET MF, post-essential thrombocythemia MF; post-PV MF, post-polycythemia vera MF; RUX, ruxolitinib.
Defined as sum durations of RUX on, or before, first luspatercept dose date, with overlapping periods excluded. Data not provided for cohort 1 and cohort 2 because these patients did not receive RUX treatment.
Data not provided for cohort 1 and cohort 3A because these cohorts comprised NTD patients, meaning they could not have received any RBC transfusions within the 12-week period immediately preceding C1D1.
Spleen length from the left costal margin.
Only patients with enlarged spleen included: cohort 1 (n = 16), cohort 3A (n = 12), cohort 2 (n = 15), and cohort 3B (n = 28). Spleen size change from baseline data were summarized by cohort only.