1. Post hoc subgroup analyses of treatment failure at 18 months' follow‐up.
| Criterion | Subgroup | Analysis | Fexinidazole | NECT | RR (95% CI) | Test for subgroup differencesa |
| Signs and symptoms score* ≥ 12 at entry (no lumbar puncture required) | Symptom score ≥ 12 | ITT | 15/91 | 1/45 | 7.42 (1.01 to 54.40) | P = 0.08, I² = 67.8% |
| Symptom score < 12 | 10/173 | 5/85 | 0.98 (0.35 to 2.78) | |||
| Symptom score ≥ 12 | mITT | 14/90 | 0/44 | 14.34 (0.88 to 235.00) | P = 0.14, I² = 53.2% | |
| Symptom score < 12 | 9/172 | 3/83 | 1.45 (0.40 to 5.21) | |||
| Symptom score ≥ 12 | EP | 14/90 | 0/44 | 14.34 (0.88 to 235.00) | P = 0.46, I² = 0% | |
| Symptom score < 12 | 8/168 | 1/81 | 3.86 (0.49 to 30.32) | |||
| Signs and symptoms score* ≥ 10 at entry (no lumbar puncture required) | Symptom score ≥10 | ITT | 19/112 | 3/58 | 3.28 (1.01 to 10.63) | P = 0.18, I² = 45.6% |
| Symptom score < 10 | 6/152 | 3/72 | 0.95 (0.24 to 3.68) | |||
| Symptom score ≥10 | mITT | 18/111 | 1/56 | 9.08 (1.24 to 66.29) | P = 0.12, I² = 59.1% | |
| Symptom score < 10 | 5/151 | 2/71 | 1.18 (0.23 to 5.91) | |||
| Symptom score ≥10 | EP | 17/110 | 1/56 | 8.65 (1.18 to 63.37) | P = 0.77, I² = 0% | |
| Symptom score < 10 | 5/148 | 0/69 | 5.17 (0.29 to 92.16) | |||
| Presence of trypanosomes in CSF at entry | With trypanosomes in CSF | ITT | 22/175 | 4/90 | 2.83 (1.01 to 7.96) | P = 0.17, I² = 46.9% |
| No trypanosomes in CSF | 3/88 | 2/40 | 0.68 (0.12 to 3.92) | |||
| With trypanosomes in CSF | mITT | 20/173 | 2/88 | 5.09 (1.22, 21.27) | P = 0.32, I² = 0% | |
| No trypanosomes in CSF | 3/88 | 1/39 | 1.33 (0.14 to 12.38) | |||
| With trypanosomes in CSF | EP | 19/172 | 1/87 | 9.61 (1.31 to 70.61) | P = 0.54, I² = 0% | |
| No trypanosomes in CSF | 3/85 | 0/38 | 3.17 (0.17 to 59.98) | |||
| Presence of > 100 WBC/μL in CSF at entry | WBC > 100 cells/μL | ITT | 22/161 | 3/80 | 3.64 (1.12 to 11.81) | P = 0.04, I² = 75.5% |
| WBC ≤ 100 cells/μL | 3/103 | 3/50 | 0.49 (0.10 to 2.32) | |||
| WBC > 100 cells/μL | mITT | 21/160 | 1/78 | 10.24 (1.40 to 74.72) | P = 0.03, I² = 79.5% | |
| WBC ≤ 100 cells/μL | 2/109 | 2/49 | 0.45 (0.07 to 3.10) | |||
| WBC > 100 cells/μL | EP | 20/157 | 0/77 | 20.24 (1.24 to 330.28) | P = 0.10, I² = 62.6% | |
| WBC ≤ 100 cells/μL | 2/101 | 1/48 | 0.95 (0.09 to 10.23) | |||
| Presence of > 400 WBC/μL in CSF at entry | WBC > 400 cells/μL | ITT | 13/79 | 1/34 | 5.59 (0.76 to 41.09) | P = 0.19, I² = 42.3% |
| WBC ≤ 400 cells/μL | 12/185 | 5/96 | 1.25 (0.45 to 3.43) | |||
| WBC > 400 cells/μL | mITT | 13/79 | 1/34 | 5.59 (0.76 to 41.09) | P = 0.53, I² = 0% | |
| WBC ≤ 400 cells/μL | 10/183 | 2/93 | 2.54 (0.57 to 11.36) | |||
| WBC > 400 cells/μL | EP | 12/78 | 0/33 | 10.76 (0.66 to 176.58) | P = 0.67, I² = 0% | |
| WBC ≤ 400 cells/μL | 10/180 | 1/92 | 5.11 (0.66 to 39.32) |
*Five significant signs and symptoms, based on the standard list of HAT warning symptoms: sleepiness, pruritus, tremor, asthenia, and recurrent headache with respectively 5, 4, 3, 2, and 1 points. aAnalysis not shown.
CI: confidence interval; CSF: cerebrospinal fluid; EP: evaluable population; g‐HAT: gambiense human African trypanosomiasis; ITT: intention‐to‐treat; mITT: modified intention‐to‐treat; NECT: nifurtimox combined with eflornithine; RR: risk ratio; WBC: white blood cell count