Baseline patient and treatment characteristics
| Characteristic . | ITT population . | Patients with neutropenia* . | Patients with thrombocytopenia† . | ||||||
|---|---|---|---|---|---|---|---|---|---|
| Luspatercept (n = 153) . | Placebo (n = 76) . | Total (n = 229) . | Luspatercept (n = 15) . | Placebo (n = 10) . | Total (n = 25) . | Luspatercept (n = 8) . | Placebo (n = 6) . | Total (n = 14) . | |
| Age, median, y (range) | 71 (40-95) | 72 (26-91) | 71 (26-95) | 72 (60-86) | 69.5 (43-79) | 72 (43-86) | 72.5 (58-79) | 73.5 (65-80) | 73.5 (58-80) |
| Male, n (%) | 94 (61.4) | 50 (65.8) | 144 (62.9) | 9 (60.0) | 6 (60.0) | 15 (60.0) | 5 (62.5) | 5 (83.3) | 10 (71.4) |
| MDS WHO 2008 classification, n (%) | |||||||||
| RS and multilineage dysplasia | 1 (0.7) | 0 | 1 (0.4) | NA | NA | NA | NA | NA | NA |
| RCMD-RS | 145 (94.8) | 74 (97.4) | 219 (95.6) | 15 (100.0) | 9 (90.0) | 24 (96.0) | 8 (100.0) | 6 (100.0) | 14 (100.0) |
| RARS | 7 (4.6) | 2 (2.6) | 9 (3.9) | NA | 1 (10.0) | 1 (4.0) | NA | NA | NA |
| Mutated SF3B1,‡ n/N with data (%) | 138/148 (93.2) | 64/74 (86.5) | 202/222 (91.0) | 12 (80.0) | 10 (100.0) | 22 (88.0) | 5 (62.5) | 4 (66.7) | 9 (64.3) |
| ANC, mean, ×109/L (SD) | 2.8 (2.1) | 2.7 (2.0) | 2.8 (2.0) | 0.8 (0.19) | 0.8 (0.11) | 0.8 (0.16) | 3.6 (5.00) | 1.8 (0.96) | 2.8 (3.83) |
| ANC category, n (%) | |||||||||
| <0.5 × 109/L | 1 (0.7) | 0 | 1 (0.4) | 1 (6.7) | 0 | 1 (4.0) | NA | NA | NA |
| 0.5 to <1.0 × 109/L | 14 (9.2) | 10 (13.2) | 24 (10.5) | 14 (93.3) | 10 (100.0) | 24 (96.0) | 2 (25.0) | 2 (33.3) | 4 (28.6) |
| ≥1.0 × 109/L | 138 (90.2) | 66 (86.8) | 204 (89.1) | NA | NA | NA | 6 (75.0) | 4 (66.7) | 10 (71.4) |
| Platelet count, mean, ×109/L (SD) | 259 (123) | 252 (124) | 257 (123) | 160.5 (58.13) | 179.1 (80.97) | 167.9 (67.20) | 78.1 (14.16) | 84.7 (12.74) | 80.9 (13.48) |
| Platelet count category, n (%) | |||||||||
| <100 × 109/L | 8 (5.2) | 6 (7.9) | 14 (6.1) | 2 (13.3) | 2 (20.0) | 4 (16.0) | 8 (100.0) | 6 (100.0) | 14 (100.0) |
| 100-400 × 109/L | 128 (83.7) | 61 (80.3) | 189 (82.5) | 13 (86.7) | 8 (80.0) | 21 (84.0) | NA | NA | NA |
| >400 × 109/L | 17 (11.1) | 9 (11.8) | 26 (11.4) | NA | NA | NA | NA | NA | NA |
| ICT use, n (%) | 71 (46.4) | 40 (52.6) | 111 (48.5) | 10 (66.7) | 8 (80.0) | 18 (72.0) | 3 (37.5) | 5 (83.3) | 8 (57.1) |
| Characteristic . | ITT population . | Patients with neutropenia* . | Patients with thrombocytopenia† . | ||||||
|---|---|---|---|---|---|---|---|---|---|
| Luspatercept (n = 153) . | Placebo (n = 76) . | Total (n = 229) . | Luspatercept (n = 15) . | Placebo (n = 10) . | Total (n = 25) . | Luspatercept (n = 8) . | Placebo (n = 6) . | Total (n = 14) . | |
| Age, median, y (range) | 71 (40-95) | 72 (26-91) | 71 (26-95) | 72 (60-86) | 69.5 (43-79) | 72 (43-86) | 72.5 (58-79) | 73.5 (65-80) | 73.5 (58-80) |
| Male, n (%) | 94 (61.4) | 50 (65.8) | 144 (62.9) | 9 (60.0) | 6 (60.0) | 15 (60.0) | 5 (62.5) | 5 (83.3) | 10 (71.4) |
| MDS WHO 2008 classification, n (%) | |||||||||
| RS and multilineage dysplasia | 1 (0.7) | 0 | 1 (0.4) | NA | NA | NA | NA | NA | NA |
| RCMD-RS | 145 (94.8) | 74 (97.4) | 219 (95.6) | 15 (100.0) | 9 (90.0) | 24 (96.0) | 8 (100.0) | 6 (100.0) | 14 (100.0) |
| RARS | 7 (4.6) | 2 (2.6) | 9 (3.9) | NA | 1 (10.0) | 1 (4.0) | NA | NA | NA |
| Mutated SF3B1,‡ n/N with data (%) | 138/148 (93.2) | 64/74 (86.5) | 202/222 (91.0) | 12 (80.0) | 10 (100.0) | 22 (88.0) | 5 (62.5) | 4 (66.7) | 9 (64.3) |
| ANC, mean, ×109/L (SD) | 2.8 (2.1) | 2.7 (2.0) | 2.8 (2.0) | 0.8 (0.19) | 0.8 (0.11) | 0.8 (0.16) | 3.6 (5.00) | 1.8 (0.96) | 2.8 (3.83) |
| ANC category, n (%) | |||||||||
| <0.5 × 109/L | 1 (0.7) | 0 | 1 (0.4) | 1 (6.7) | 0 | 1 (4.0) | NA | NA | NA |
| 0.5 to <1.0 × 109/L | 14 (9.2) | 10 (13.2) | 24 (10.5) | 14 (93.3) | 10 (100.0) | 24 (96.0) | 2 (25.0) | 2 (33.3) | 4 (28.6) |
| ≥1.0 × 109/L | 138 (90.2) | 66 (86.8) | 204 (89.1) | NA | NA | NA | 6 (75.0) | 4 (66.7) | 10 (71.4) |
| Platelet count, mean, ×109/L (SD) | 259 (123) | 252 (124) | 257 (123) | 160.5 (58.13) | 179.1 (80.97) | 167.9 (67.20) | 78.1 (14.16) | 84.7 (12.74) | 80.9 (13.48) |
| Platelet count category, n (%) | |||||||||
| <100 × 109/L | 8 (5.2) | 6 (7.9) | 14 (6.1) | 2 (13.3) | 2 (20.0) | 4 (16.0) | 8 (100.0) | 6 (100.0) | 14 (100.0) |
| 100-400 × 109/L | 128 (83.7) | 61 (80.3) | 189 (82.5) | 13 (86.7) | 8 (80.0) | 21 (84.0) | NA | NA | NA |
| >400 × 109/L | 17 (11.1) | 9 (11.8) | 26 (11.4) | NA | NA | NA | NA | NA | NA |
| ICT use, n (%) | 71 (46.4) | 40 (52.6) | 111 (48.5) | 10 (66.7) | 8 (80.0) | 18 (72.0) | 3 (37.5) | 5 (83.3) | 8 (57.1) |
ANC, absolute neutrophil count; ICT, iron chelation therapy; ITT, intention to treat; NA, not applicable; RARS, refractory anemia with RS; RCMD-RS, refractory cytopenia with multilineage dysplasia and RS; SD, standard deviation; WHO, World Health Organization.
Patients from the ITT population with neutropenia defined per IWG 2006 criteria as neutrophil level <1 × 109/L.
Patients from the ITT population with thrombocytopenia defined per IWG 2006 criteria as platelet level <100 × 109/L.
No patients with SF3B1 mutation had RS <15%.