Factor | Single-arm trial (n = 82) | Randomized controlled trial (n = 151) | Total (n = 233) | Univariable logistic regression analysis | Multivariable logistic regression analysiss | ||||
---|---|---|---|---|---|---|---|---|---|
Odds ratio | 95% CI | p value | Odds ratio | 95% CI | p value | ||||
Prevalence | |||||||||
< 1/100,000 | 13 (15.9%) | 26 (17.2%) | 39 | Reference | Reference | ||||
≥ 1/100,000 | 69 (84.1%) | 125 (82.8%) | 194 | 0.91 | 0.44–1.88 | 0.7899 | 1.31 | 0.52–3.34 | 0.561 |
Severity of the disease outcome | |||||||||
High mortality | 67 (81.7%) | 67 (44.4%) | 134 | Reference | Reference | ||||
Others | 15 (18.3%) | 84 (55.6%) | 99 | 5.60 | 2.94–10.68 | < 0.0001* | 5.63 | 2.64-12.00 | < 0.0001* |
Drug usage | |||||||||
Mono therapy | 78 (95.1%) | 128 (84.8%) | 206 | Reference | Reference | ||||
Combination therapy | 4 (4.9%) | 23 (15.2%) | 27 | 3.50 | 1.17–10.51 | 0.0253* | 2.95 | 1.80-18.57 | 0.0032* |
Target age segment | |||||||||
Children with or without adult | 24 (29.3%) | 55 (36.4%) | 79 | Reference | Reference | ||||
Adult only | 58 (70.7%) | 96 (63.6%) | 154 | 0.72 | 0.40–1.29 | 0.2714 | 1.72 | 0.75–3.97 | 0.2027 |
Primary efficacy endpoint | |||||||||
Pharmacodynamic/Response biomarker | 69 (84.1%) | 67 (44.4%) | 136 | Reference | Reference | ||||
Clinical outcome | 13 (15.9%) | 84 (55.6%) | 97 | 6.65 | 3.39–13.06 | < 0.0001* | 5.57 | 2.57–12.06 | 0.0001* |
Therapeutic clasiffication | |||||||||
Oncology | 63 (76.8%) | 46 (30.5%) | 109 | Reference | |||||
Nononcology | 19 (23.2%) | 105 (69.5%) | 124 | 7.57 | 4.08–14.06 | < 0.0001* | N/A |