Table 4.
Reasons for stopping randomized treatment during follow-up
| ERN/LRPT | Placebo | Excessa (SE) | P* | |
|---|---|---|---|---|
| Number randomized | 12 838 | 12 835 | ||
| Total not continuing randomized treatment | 3256 (25.4%) | 2136 (16.6%) | 8.7% (0.5%) | <0.0001 |
| Medical reasons | ||||
| Skin | ||||
| Pruritis | 432 | 90 | ||
| Rash | 132 | 47 | ||
| Flushing | 106 | 14 | ||
| Other skin | 27 | 9 | ||
| Any skin reason | 697 (5.4%) | 160 (1.2%) | 4.2% (0.2%) | <0.0001 |
| Gastrointestinal (GI) | ||||
| Any upper GI | 227 | 104 | ||
| Any lower GI | 205 | 73 | ||
| Other GI | 63 | 42 | ||
| Any GI reason | 495 (3.9%) | 219 (1.7%) | 2.1% (0.2%) | <0.0001 |
| Hepatobiliary | ||||
| Abnormal alanine transaminase | 38 | 30 | ||
| Other hepatobiliary | 14 | 13 | ||
| Any hepatobiliary reason | 52 (0.4%) | 43 (0.3%) | 0.1% (0.1%) | 0.36 |
| Musculoskeletal | ||||
| Muscle symptoms | 151 | 90 | ||
| Rheumatological | 18 | 21 | ||
| Gout | 26 | 8 | ||
| Abnormal creatine kinase | 25 | 5 | ||
| Other musculoskeletal | 5 | 4 | ||
| Any musculoskeletal reason | 225 (1.8%) | 128 (1.0%) | 0.8% (0.1%) | <0.0001 |
| Diabetes | ||||
| New-onset diabetes mellitus | 13 | 5 | ||
| Major diabetes complication | 2 | 0 | ||
| Other diabetes-related reason | 104 | 45 | ||
| Any diabetes-related reason | 119 (0.9%) | 50 (0.4%) | 0.5% (0.1%) | <0.0001 |
| Other medical | ||||
| Pre-syncope/syncope | 23 | 16 | ||
| Palpitations | 8 | 2 | ||
| Other cardiovascular | 75 | 80 | ||
| Respiratory | 20 | 13 | ||
| Cancer | 65 | 62 | ||
| Other | 185 | 150 | ||
| Contraindicated medication | 9 | 4 | ||
| Medical advice | 135 | 94 | ||
| Any other medical reason | 520 (4.1%) | 421 (3.3%) | 0.8% (0.2%) | 0.001 |
| Any medical reason | 2107 (16.4%) | 1020 (7.9%) | 8.5% (0.4%) | <0.0001 |
| Non-medical reasons | ||||
| Patient wishes | 626 | 580 | ||
| Difficulty swallowing tablets | 258 | 361 | ||
| Other | 265 | 175 | ||
| Any non-medical reason | 1149 (8.9%) | 1116 (8.7%) | 0.3% (0.4%) | 0.47 |
| Any reason for stopping | 3256 (25.4%) | 2136 (16.6%) | 8.7% (0.5%) | <0.0001 |
aExcess is defined as the absolute percentage of patients who had the event in the ERN/LRPT group minus the percentage who had the event in the placebo group.
*P-values are calculated from z tests comparing the proportion of patients who had the event in the ERN/LRPT group with the proportion of patients who had the event in the placebo group.