Table 1 Characteristics of 77 included studies, by non-inferiority and superiority trials
| Characteristic | Non-inferiority trials (N = 11) |
Superiority trials (N = 66) | ||
|---|---|---|---|---|
| n | % | n | % | |
| Journal of publication | ||||
| Journal of Clinical Oncology | 3 | 27.3 | 25 | 37.9 |
| Blood | 2 | 18.2 | 10 | 15.2 |
| Pediatric Blood & Cancer | 0 | 0.0 | 6 | 9.1 |
| Leukemia | 1 | 9.1 | 4 | 6.1 |
| Lancet | 2 | 18.2 | 1 | 1.5 |
| Cancer | 0 | 0.0 | 4 | 6.1 |
| Lancet Oncology | 1 | 9.1 | 3 | 4.5 |
| New England Journal of Medicine | 1 | 9.1 | 2 | 3 |
| Other | 1 | 9.1 | 11 | 16.7 |
| Region of publication | ||||
| Europe | 6 | 54.5 | 23 | 34.8 |
| North America | 4 | 36.4 | 41 | 62.1 |
| Other | 1 | 9.1 | 2 | 3 |
| Year of publication | ||||
| 1976 to 1989 | 1 | 9.1 | 4 | 6.1 |
| 1990 to 2003 | 4 | 36.4 | 28 | 42.4 |
| 2004 to 2016 | 6 | 54.5 | 34 | 51.5 |
| Source of funding | ||||
| Non-industry | 10 | 90.9 | 56 | 84.8 |
| Industry and non-industry | 0 | 0.0 | 2 | 3 |
| Not stated | 1 | 9.1 | 8 | 12.1 |
| Study participants | ||||
| Exclusively children | 7 | 63.6 | 41 | 62.1 |
| Adults included | 4 | 36.4 | 25 | 37.9 |
| Disease site | ||||
| Hematological | 7 | 63.6 | 43 | 65.2 |
| Central nervous system tumor | 1 | 9.1 | 11 | 16.7 |
| Non-central nervous system solid tumor | 3 | 27.3 | 12 | 18.2 |
| RCT study design | ||||
| 2 × 2 factorial | 0 | 0.0 | 4 | 6.1 |
| Greater than 2 arms | 1 | 9.1 | 5 | 7.6 |
| Two-armed | 10 | 90.9 | 57 | 86.3 |
| RCT trial group | ||||
| POG | 1 | 9.1 | 15 | 22.7 |
| CCG | 1 | 9.1 | 12 | 18.2 |
| COG | 0 | 0.0 | 8 | 12.1 |
| BFM | 1 | 9.1 | 9 | 13.6 |
| UK MRC | 1 | 9.1 | 4 | 6.1 |
| Other | 7 | 63.6 | 18 | 27.3 |
| Outcome | ||||
| Time-to-event | 10 | 90.9 | 56 | 84.8 |
| Dichotomous | 1 | 9.1 | 10 | 15.2 |
| Intervention in question | ||||
| Chemotherapy | 9 | 81.8 | 57 | 86.4 |
| Multimodal therapy | 0 | 0.0 | 2 | 3 |
| Hemopoietic stem-cell transplant | 1 | 9.1 | 6 | 9.1 |
| Radiation therapy | 1 | 9.1 | 1 | 1.5 |