Baseline patient characteristics (mITT population)
| . | Placebo (n = 51) . | Lenalidomide 5 mg (n = 47) . | Lenalidomide 10 mg (n = 41) . | Overall (N = 139) . |
|---|---|---|---|---|
| Age, y | ||||
| Median | 70 | 66 | 68 | 69 |
| Range | 39-85 | 40-86 | 36-84 | 36-86 |
| Female sex, n (%) | 41 (80.4) | 37 (78.7) | 28 (68.3) | 106 (76.3) |
| Time since diagnosis, y | ||||
| Median | 2.4 | 2.8 | 2.5 | 2.7 |
| Range | 0.2-14.3 | 0.2-17.1 | 0.2-14.9 | 0.2-17.1 |
| Transfusion burden, units/8 wk | ||||
| Median | 6 | 7 | 6 | 6 |
| Range | 4-12 | 1*-25 | 2-12 | 1*-25 |
| IPSS risk category (central review), n (%) | ||||
| Low | 29 (56.9) | 19 (40.4) | 20 (48.8) | 68 (48.9) |
| Intermediate-1 | 22 (43.1) | 28 (59.6) | 21 (51.2) | 71 (51.1) |
| WPSS risk category, n (%) | ||||
| Very low | 0 | 0 | 0 | 0 |
| Low | 2 (3.9) | 7 (14.9) | 2 (4.9) | 11 (7.9) |
| Intermediate | 33 (64.7) | 23 (48.9) | 26 (63.4) | 82 (59.0) |
| High | 15 (29.4) | 17 (36.2) | 13 (31.7) | 45 (32.4) |
| Very high | 0 | 0 | 0 | 0 |
| Missing | 1 (2.0) | 0 | 0 | 1 (0.7) |
| FAB classification (central review), n (%) | ||||
| RA | 35 (68.6) | 32 (68.1) | 28 (68.3) | 95 (68.3) |
| RARS | 8 (15.7) | 5 (10.6) | 8 (19.5) | 21 (15.1) |
| RAEB | 3 (5.9) | 7 (14.9) | 5 (12.2) | 15 (10.8) |
| CMML | 1 (2.0) | 2 (4.3) | 0 | 3 (2.2) |
| Other or missing | 4 (7.8) | 1 (2.1) | 0 | 5 (3.6) |
| WHO classification, n (%) | ||||
| RA | 6 (11.8) | 7 (14.9) | 2 (4.9) | 15 (10.8) |
| RARS | 2 (3.9) | 0 | 2 (4.9) | 4 (2.9) |
| RAEB-1 | 7 (13.7) | 5 (10.6) | 4 (9.8) | 16 (11.5) |
| RAEB-2 | 1 (2.0) | 2 (4.3) | 1 (2.4) | 4 (2.9) |
| RCMD | 1 (2.0) | 2 (4.3) | 1 (2.4) | 4 (2.9) |
| 5q− syndrome | 27 (52.9) | 24 (51.1) | 16 (39.0) | 67 (48.2) |
| Unknown | 5 (9.8) | 7 (14.9) | 14 (34.1) | 26 (18.7) |
| Missing | 2 (3.9) | 0 | 1 (2.4) | 3 (2.2) |
| EPO level, n (%) | ||||
| ≤ 500 mIU/mL | 21 (41.2) | 13 (27.7) | 14 (34.1) | 48 (34.5) |
| > 500 mIU/mL | 24 (47.1) | 24 (51.1) | 21 (51.2) | 69 (49.6) |
| Missing | 6 (11.8) | 10 (21.3) | 6 (14.6) | 22 (15.8) |
| Prior EPO use, n (%) | 24 (47.1) | 24 (51.1) | 24 (58.5) | 72 (51.8) |
| Karyotype, n (%) | ||||
| Isolated del5q31 | 38 (74.5) | 35 (74.5) | 33 (80.5) | 106 (76.3) |
| del5q31 plus ≥ 1 additional abnormality† | 13 (25.5) | 12 (25.5) | 8 (19.5) | 33 (23.7) |
| ANC 0.5-1.0 × 109/L, n (%) | 3 (5.9) | 10 (21.3) | 3 (7.3) | 16 (11.5) |
| Platelet count, n (%) | ||||
| < 150 × 109/L | 8 (15.7) | 11 (23.4) | 8 (19.5) | 27 (19.4) |
| 25-50 × 109/L | 0 | 1 (2.1) | 1 (2.4) | 2 (1.4) |
| ≥ 150 × 109/L | 43 (84.3) | 36 (76.6) | 33 (80.5) | 112 (80.6) |
| . | Placebo (n = 51) . | Lenalidomide 5 mg (n = 47) . | Lenalidomide 10 mg (n = 41) . | Overall (N = 139) . |
|---|---|---|---|---|
| Age, y | ||||
| Median | 70 | 66 | 68 | 69 |
| Range | 39-85 | 40-86 | 36-84 | 36-86 |
| Female sex, n (%) | 41 (80.4) | 37 (78.7) | 28 (68.3) | 106 (76.3) |
| Time since diagnosis, y | ||||
| Median | 2.4 | 2.8 | 2.5 | 2.7 |
| Range | 0.2-14.3 | 0.2-17.1 | 0.2-14.9 | 0.2-17.1 |
| Transfusion burden, units/8 wk | ||||
| Median | 6 | 7 | 6 | 6 |
| Range | 4-12 | 1*-25 | 2-12 | 1*-25 |
| IPSS risk category (central review), n (%) | ||||
| Low | 29 (56.9) | 19 (40.4) | 20 (48.8) | 68 (48.9) |
| Intermediate-1 | 22 (43.1) | 28 (59.6) | 21 (51.2) | 71 (51.1) |
| WPSS risk category, n (%) | ||||
| Very low | 0 | 0 | 0 | 0 |
| Low | 2 (3.9) | 7 (14.9) | 2 (4.9) | 11 (7.9) |
| Intermediate | 33 (64.7) | 23 (48.9) | 26 (63.4) | 82 (59.0) |
| High | 15 (29.4) | 17 (36.2) | 13 (31.7) | 45 (32.4) |
| Very high | 0 | 0 | 0 | 0 |
| Missing | 1 (2.0) | 0 | 0 | 1 (0.7) |
| FAB classification (central review), n (%) | ||||
| RA | 35 (68.6) | 32 (68.1) | 28 (68.3) | 95 (68.3) |
| RARS | 8 (15.7) | 5 (10.6) | 8 (19.5) | 21 (15.1) |
| RAEB | 3 (5.9) | 7 (14.9) | 5 (12.2) | 15 (10.8) |
| CMML | 1 (2.0) | 2 (4.3) | 0 | 3 (2.2) |
| Other or missing | 4 (7.8) | 1 (2.1) | 0 | 5 (3.6) |
| WHO classification, n (%) | ||||
| RA | 6 (11.8) | 7 (14.9) | 2 (4.9) | 15 (10.8) |
| RARS | 2 (3.9) | 0 | 2 (4.9) | 4 (2.9) |
| RAEB-1 | 7 (13.7) | 5 (10.6) | 4 (9.8) | 16 (11.5) |
| RAEB-2 | 1 (2.0) | 2 (4.3) | 1 (2.4) | 4 (2.9) |
| RCMD | 1 (2.0) | 2 (4.3) | 1 (2.4) | 4 (2.9) |
| 5q− syndrome | 27 (52.9) | 24 (51.1) | 16 (39.0) | 67 (48.2) |
| Unknown | 5 (9.8) | 7 (14.9) | 14 (34.1) | 26 (18.7) |
| Missing | 2 (3.9) | 0 | 1 (2.4) | 3 (2.2) |
| EPO level, n (%) | ||||
| ≤ 500 mIU/mL | 21 (41.2) | 13 (27.7) | 14 (34.1) | 48 (34.5) |
| > 500 mIU/mL | 24 (47.1) | 24 (51.1) | 21 (51.2) | 69 (49.6) |
| Missing | 6 (11.8) | 10 (21.3) | 6 (14.6) | 22 (15.8) |
| Prior EPO use, n (%) | 24 (47.1) | 24 (51.1) | 24 (58.5) | 72 (51.8) |
| Karyotype, n (%) | ||||
| Isolated del5q31 | 38 (74.5) | 35 (74.5) | 33 (80.5) | 106 (76.3) |
| del5q31 plus ≥ 1 additional abnormality† | 13 (25.5) | 12 (25.5) | 8 (19.5) | 33 (23.7) |
| ANC 0.5-1.0 × 109/L, n (%) | 3 (5.9) | 10 (21.3) | 3 (7.3) | 16 (11.5) |
| Platelet count, n (%) | ||||
| < 150 × 109/L | 8 (15.7) | 11 (23.4) | 8 (19.5) | 27 (19.4) |
| 25-50 × 109/L | 0 | 1 (2.1) | 1 (2.4) | 2 (1.4) |
| ≥ 150 × 109/L | 43 (84.3) | 36 (76.6) | 33 (80.5) | 112 (80.6) |
WPSS indicates World Health Organization classification-based Prognostic Scoring System; FAB, French-American-British; RARS, RA with ringed sideroblasts; CMML, chronic myelomonocytic leukemia; WHO, World Health Organization; RCMD, refractory cytopenia with multilineage dysplasia; and ANC, absolute neutrophil count.
Only 2 patients had a transfusion burden of 1 unit/8 weeks.
Two patients (3.9%) in the placebo group, 2 patients (4.3%) in the lenalidomide 5 mg group, and 3 patients (7.3%) in the lenalidomide 10 mg group had del5q31 plus ≥ 2 additional cytogenetic abnormalities.