Adverse events. Values are n (%) unless otherwise specified.
| Variables | Placebo-controlled, Weeks 0 to 52* | Open-label/Study Termination | ||
|---|---|---|---|---|
| Placebo | POM 1 mg QD | Placebo* | POM 1 mg QD | |
| Patients, n | 12 | 10 | 6 | 2 |
| AE summary | ||||
| ≥ 1 AE | 12 (100.0) | 9 (90.0) | 5 (83.3) | 2 (100.0) |
| ≥ 1 SAE | 1 (8.3) | 4 (40.0) | 0 (0.0) | 1 (50.0) |
| ≥ 1 severe AE | 1 (8.3) | 4 (40.0) | 0 (0.0) | 0 (0.0) |
| AE leading to drug withdrawal/discontinuation | 0 (0.0) | 4 (40.0) | 0 (0.0) | 0 (0.0) |
| Death | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) |
| AE reported by ≥ 2 patients in any treatment group | ||||
| Constipation | 1 (8.3) | 3 (30.0) | 0 (0.0) | 0 (0.0) |
| Diarrhea | 3 (25.0) | 1 (10.0) | 0 (0.0) | 0 (0.0) |
| Nausea | 2 (16.7) | 0 (0.0) | 0 (0.0) | 0 (0.0) |
| Bronchitis | 0 (0.0) | 2 (20.0) | 0 (0.0) | 0 (0.0) |
| Upper respiratory tract infection | 3 (25.0) | 1 (10.0) | 0 (0.0) | 0 (0.0) |
| Influenza | 2 (16.7) | 0 (0.0) | 2 (33.3) | 0 (0.0) |
| Urinary tract infection | 2 (16.7) | 0 (0.0) | 2 (33.3) | 0 (0.0) |
| Ligament sprain | 0 (0.0) | 2 (20.0) | 0 (0.0) | 0 (0.0) |
| Arthralgia | 4 (33.3) | 4 (40.0) | 0 (0.0) | 0 (0.0) |
| Headache | 3 (25.0) | 1 (10.0) | 0 (0.0) | 0 (0.0) |
| Dyspnea | 2 (16.7) | 0 (0.0) | 0 (0.0) | 0 (0.0) |
| Oropharyngeal pain | 2 (16.7) | 0 (0.0) | 0 (0.0) | 0 (0.0) |
| Productive cough | 0 (0.0) | 0 (0.0) | 2 (33.3) | 0 (0.0) |
| Rash | 1 (8.3) | 2 (20.0) | 0 (0.0) | 0 (0.0) |
| Skin ulcer | 2 (16.7) | 0 (0.0) | 2 (33.3) | 0 (0.0) |
| SAE | ||||
| ≥ 1 SAE | 1 (8.3) | 4 (40.0) | 0 (0.0) | 1 (50.0) |
| Infections and infestations | ||||
| Pneumonia | 1 (8.3) | 1 (10.0) | 0 (0.0) | 0 (0.0) |
| Upper respiratory tract infection | 0 (0.0) | 1 (10.0) | 0 (0.0) | 0 (0.0) |
| Sepsis | 1 (8.3) | 0 (0.0) | 0 (0.0) | 0 (0.0) |
| Renal and urinary disorders | ||||
| Renal failure | 0 (0.0) | 1 (10.0) | 0 (0.0) | 0 (0.0) |
| Respiratory, thoracic, mediastinal disorders | ||||
| Pulmonary embolism | 0 (0.0) | 1 (10.0) | 0 (0.0) | 0 (0.0) |
| Acute respiratory failure | 0 (0.0) | 0 (0.0) | 0 (0.0) | 1 (50.0) |
| Chronic respiratory failure | 0 (0.0) | 0 (0.0) | 0 (0.0) | 1 (50.0) |
| Pulmonary hypertension | 0 (0.0) | 0 (0.0) | 0 (0.0) | 1 (50.0) |
| AE leading to discontinuation | ||||
| C-reactive protein increased | 0 (0.0) | 1 (10.0) | 0 (0.0) | 0 (0.0) |
| Renal failure | 0 (0.0) | 1 (10.0) | 0 (0.0) | 0 (0.0) |
| Pulmonary embolism | 0 (0.0) | 1 (10.0) | 0 (0.0) | 0 (0.0) |
| Toxic skin eruption | 0 (0.0) | 1 (10.0) | 0 (0.0) | 0 (0.0) |
↵* Placebo-controlled period includes all data through Week 52. The placebo group in the open-label extension phase includes patients initially randomized to receive placebo in the double-blind treatment phase who are now receiving POM. AE: adverse event; POM: pomalidomide; QD: once daily; SAE: serious AE.