Table 2.
Efficacy endpoints: treatment success and survival
| mITT n/N (%) (95%CI) |
|
|---|---|
| Response at EOST | |
| Successa | 16/20 (80) (56.3–94.3) |
| Failure | 4/20 (20) (5.7–43.7) |
| Reasons for failure at EOST | |
| Persistent Candida spp. in blood culturesb | 3/20 |
| Deathc | 1/20 |
| Response at EOT | |
| Success | 15 (75.0) (50.9–91.3) |
| Failured | 5 (25.0) (8.7–49.1) |
| Response at 2 weeks after EOT | |
| Treatment success sustained | 12 (60.0) (36.1–80.9) |
| Clinical relapse | 2 (10.0) (1.2–31.7) |
| Death | 1 |
| Response at 4 weeks after EOT | |
| Treatment success sustained | 11 (55.0) (31.5–76.9) |
| Death | 1 |
| Survival at D30 | |
| Participant survival at D30 | 17/20 (85) |
| Median time to death (days) | 15 |
| Reasons for mortality through D30: | |
| Gram-negative (Acinetobacter) sepsis (D12) | 1/20 |
| Progression of underlying cancers (D15) | 1/20 |
| Worsening of interstitial pneumonia (D30) | 1/20 |
If there was no step-down anti-fungal treatment, EOT = EOST. Efficacy outcomes are up to the timepoint of failure. Percentages were calculated using the number of participants in the column heading as the denominator. 95% CIs were two-sided exact binomial CIs. CI, confidence interval; DRC, data review committee; EOST, end of study drug treatment; EOT, end of antifungal treatment; mITT, modified intent-to-treat; spp., species.
Success at EOST definition (composite): (i) eradication of Candida spp. from blood; and (ii) no use of other systemic antifungal through to EOST; and (iii) alive at EOST. Failure: any case not meeting definition of success.
C. glabrata (n = 1), C. albicans (n = 1), C. parapsilosis (n = 1).
Death on D12—investigator assessed cause as Gram-negative (Acinetobacter) bacteraemia/sepsis.
EOST + EOT; one additional failure from EOST.