Table 5. Secondary efficacy variables at months 3, 6, 12, 18, and 24 (per-protocol dataset).
| Pafuramidine | Pentamidine | ||
|---|---|---|---|
| WHO Efficacy Criteria | 100 mg BID | 4 mg/kg QD | |
| Time Point | n/N (%) | n/N (%) | 95% CIa |
| Cure | |||
| Month 3 | 2/135 (1.5) | 1/137 (0.7) | -3.2, 1.7 |
| Month 6 | 124/135 (91.9) | 131/137 (95.6) | -2.0, 9.5 |
| Month 12 | 117/133 (88.0) | 122/129 (94.6) | -0.2, 13.4 |
| Month 18 | 99/131 (75.6) | 109/129 (84.5) | -0.7, 18.6 |
| Month 24 | 86/135 (63.7) | 83/130 (63.8) | -11.4, 11.7 |
| Probable cure | |||
| Month 3b | 131/135 (97.0) | 135/137 (98.5) | -2.0, 5.0 |
| Month 6b | 5/135 (3.7) | 3/137 (2.2) | -5.5, 2.5 |
| Month 12b | 4/133 (3.0) | 4/129 (3.1) | -4.1, 4.3 |
| Month 18b | 16/131 (12.2) | 13/129 (10.1) | -9.8, 5.5 |
| Month 24c | 28/135 (20.7) | 34/130 (26.2) | -4.8, 15.6 |
| Probable relapse | |||
| Month 3 | 0 | 0 | -- |
| Month 6 | 2/135 (1.5) | 1/137 (0.7) | -3.2, 1.7 |
| Month 12 | 5/133 (3.8) | 1/129 (0.8) | -6.6, 0.6 |
| Month 18 | 7/131 (5.3) | 1/129 (0.8) | -8.7, -0.4 |
| Month 24 | 11/135 (8.1) | 3/130 (2.3) | -11.1, -0.6 |
| Relapsed | |||
| Month 3 | 2/135 (1.5) | 1/137 (0.7) | -3.2, 1.7 |
| Month 6 | 2/135 (1.5) | 1/137 (0.7) | -3.2, 1.7 |
| Month 12 | 3/133 (2.3) | 1/129 (0.8) | -4.4, 1.5 |
| Month 18 | 4/131 (3.1) | 3/129 (2.3) | -4.7, 3.2 |
| Month 24 | 5/135 (3.7) | 3/130 (2.3) | -5.5, 2.7 |
| Death | |||
| Month 3 | 0 | 0 | -- |
| Month 6 | 2/135 (1.5) | 1/137 (0.7) | -3.2, 1.7 |
| Month 12 | 4/133 (3.0) | 1/129 (0.8) | -5.5, 1.0 |
| Month 18 | 5/131 (3.8) | 3/129 (2.3) | -5.7, 2.7 |
| Month 24e | 5/135 (3.7) | 7/130 (5.4) | -3.3, 6.7 |
BID = twice a day, CI = confidence interval, QD = once a day, WHO = World Health Organization
a Confidence intervals based on normal approximation to the binomial.
b Includes cases of “uncertain evolution” [27]
c Includes cases classified as probable cure based on oral information
d One patient in the pentamidine group was classified as a treatment failure at end of treatment and is included in Relapse at Month 3.
e One death was reported in the pafuramidine group subsequent to the last patient visit and is not included in the efficacy endpoints.