Table 2. Efficacy and effectiveness indicators.
| Efficacy and effectiveness indicators | n | N | % | (95% CI) |
|---|---|---|---|---|
| Hospitalization period | ||||
| Number of patients (ITT population) | 629 | 629 | 100 | |
| In-hospital fatalities | 10 | 629 | 1.6 | |
| Discharged alive (primary endpoint) | 619 | 629 | 98.4 | (97.1–99.1) |
| Follow-up period | ||||
| Fatalities | 18 | 629 | 2.8 | |
| Relapses (confirmed and probable relapses1) | 8 | 629 | 1.3 | |
| At 24 months | ||||
| Clinical cure at 24 months (effectiveness, secondary endpoint) | ||||
| mITT population | 577 | 613 | 94.1 | (91.8–95.7) |
| PP2 population | 525 | 561 | 93.6 | (91.0–95.3) |
| Sensitivity analysis | ||||
| ITT CLTFU considered as cured | 593 | 629 | 94.3 | (92.0–95.8) |
| ITT CLTFU considered as failures | 577 | 629 | 91.7 | (89.3–93.8) |
| Probability of cure per subpopulation of interest | ||||
| Overall, mITT population | 577 | 613 | 93.9 | (91.6–95.6) |
| Children 0–11 years | 93 | 99 | 93.1 | (85.0–96.9) |
| Pregnant or breastfeeding women | 46 | 47 | 97.9 | (85.8–99.7) |
| Other patients | 438 | 467 | 93.6 | (91.0–95.5) |
| Previous HAT history | ||||
| Within 2 years2 | 77 | 82 | 93.3 | (84.5–97.2) |
| Probability of cure per site | ||||
| Kasai Oriental Province | ||||
| Dipumba | 129 | 138 | 93.2 | (87.2–96.4) |
| Katanda | 121 | 132 | 91.4 | (85.0–95.2) |
| Ngandajika | 84 | 89 | 94.2 | (86.5–97.5) |
| Bandundu Province | ||||
| Bandundu | 94 | 98 | 95.9 | (89.4–98.4) |
| Kwamouth | 94 | 97 | 96.9 | (90.7–99.0) |
| Yasa Bonga | 55 | 59 | 93.2 | (82.9–97.4) |
Abbreviations: ITT, intention to treat; mITT, modified intention to treat; CLTFU, completely lost to follow up.
1confirmed relapses (patients with detected trypanosomes in any body fluid), probable relapses (patients with no evidence of trypanosomes but >20 WBC/mm3 in CSF or clinical evidence).
2Previous HAT history within 2 years, can be considered as previous treatment failure.