Table 2.
Primary Efficacy Analysis in Per-Protocol and Modified Intention-to-Treat Populations.*
| Variable | Per-Protocol Analysis | Modified Intention-to-Treat Analysis | ||||||
|---|---|---|---|---|---|---|---|---|
| Control Group (N = 510) | Isoniazid Group (N = 514) | Ethambutol Group (N = 524) | All Patients (N = 1548) | Control Group (N = 555) | Isoniazid Group (N = 568) | Ethambutol Group (N = 551) | All Patients (N = 1674) | |
| Favorable outcome — no. (%) | ||||||||
|
| ||||||||
| Patients with outcome | 467 (92) | 436 (85) | 419 (80) | 1322 (85) | 468 (84) | 436 (77) | 419 (76) | 1323 (79) |
|
| ||||||||
| Culture-negative status at 18 mo | 409 (80) | 389 (76) | 367 (70) | 1165 (75) | 410 (74) | 389 (68) | 367 (67) | 1166 (70) |
|
| ||||||||
| Unable to produce sputum | 0 | 2 (<1) | 0 | 2 (<1) | 0 | 2 (<1) | 0 | 2 (<1) |
|
| ||||||||
| Unable to produce sputum at 18 mo but culture-negative status earlier | 49 (10) | 31 (6) | 35 (7) | 115 (7) | 49 (9) | 31 (5) | 35 (6) | 115 (7) |
|
| ||||||||
| Missing data on L–J culture at 18 mo and MGIT negative | 9 (2) | 14 (3) | 17 (3) | 40 (3) | 9 (2) | 14 (2) | 17 (3) | 40 (2) |
|
| ||||||||
| Unfavorable outcome — no. (%)† | ||||||||
|
| ||||||||
| Patients with outcome | 43 (8) | 78 (15) | 105 (20) | 226 (15) | 87 (16) | 132 (23) | 132 (24) | 351 (21) |
|
| ||||||||
| 6-Mo treatment phase | ||||||||
|
| ||||||||
| Nonviolent death | 5 (1) | 6 (1) | 7 (1) | 18 (1) | 5 (1) | 6 (1) | 7 (1) | 18 (1) |
|
| ||||||||
| Treatment failure‡ | ||||||||
|
| ||||||||
| Culture-confirmed | 3 (1) | 4 (1) | 1 (<1) | 8 (1) | 3 (1) | 4 (1) | 1 (<1) | 8 (<1) |
|
| ||||||||
| Not culture-confirmed | 4 (1) | 1 (<1) | 4 (1) | 9 (1) | 4 (1) | 1 (<1) | 4 (1) | 9 (1) |
|
| ||||||||
| Adverse reaction | NA | NA | NA | NA | 18 (3) | 15 (3) | 9 (2) | 42 (3) |
|
| ||||||||
| Withdrawal of consent | NA | NA | NA | NA | 8 (1) | 18 (3) | 8 (1) | 34 (2) |
|
| ||||||||
| Relocation | NA | NA | NA | NA | 2 (<1) | 4 (1) | 4 (1) | 10 (1) |
|
| ||||||||
| Other investigator decision | NA | NA | NA | NA | 2 (<1) | 5 (1) | 0 | 7 (<1) |
|
| ||||||||
| No completion of treatment | NA | NA | NA | NA | 13 (2) | 10 (2) | 6 (1) | 29 (2) |
|
| ||||||||
| Follow-up | ||||||||
|
| ||||||||
| Relapse after culture-negative status | 12 (2) | 46 (9) | 64 (12) | 122 (8) | 13 (2) | 46 (8) | 64 (12) | 123 (7) |
|
| ||||||||
| Retreated for tuberculosis | 14 (3) | 17 (3) | 27 (5) | 58 (4) | 14 (3) | 18 (3) | 27 (5) | 59 (4) |
|
| ||||||||
| Death from tuberculosis or respiratory distress | 2 (<1) | 0 | 0 | 2 (<1) | 2 (<1) | 0 | 0 | 2 (<1) |
|
| ||||||||
| No culture-negative status | ||||||||
|
| ||||||||
| Ever | 1 (<1) | 1 (<1) | 0 | 2 (<1) | 1 (<1) | 2 (<1) | 0 | 3 (<1) |
|
| ||||||||
| At last visit | 2 (<1) | 3 (1) | 2 (<1) | 7 (<1) | 2 (<1) | 3 (1) | 2 (<1) | 7 (<1) |
|
| ||||||||
| Adjusted difference from control in rate of unfavorable outcome — percentage points (97.5% CI) | NA | 6.1 (1.7–10.5) | 11.4 (6.7–16.1) | NA | NA | 7.8 (2.7–13.0) | 9.0 (3.8–14.2) | NA |
The treatment phase was defined as any time from randomization to 32 weeks after randomization (26 weeks plus 6-week window). L–J denotes Lowenstein–Jensen solid medium, and NA not applicable.
During follow-up, the relapse and retreatment categories include patients during the scheduled end of active treatment (after month 4 for the moxifloxacin-containing groups and month 6 for the control group). In the per-protocol analysis, data from 24-locus mycobacterial-interspersed- repetitive-unit analysis were missing for 9 of 17 patients with treatment failure, 42 of 122 patients with relapse, and 38 of 58 patients who were retreated for tuberculosis.
Listed are patients who were receiving active treatment in whom treatment failed.