Table 3 Comparison of PRO quality reporting over time in Bladder Cancer RCTs with PROs as a secondary outcome
| Methodological issue | Category |
RCT with PROs (Jan.2004 –Mar. 2014) (n. 9), N. (%) |
RCTs with PROs (Apr.2014 – Jun.2018) (n. 8), N. (%) | P-value |
|---|---|---|---|---|
| Title and abstract | ||||
| The PRO should be identified as an outcome in the abstract | No | 1 (11.1) | 3 (37.5) | 0.29 |
| Yes | 8 (88.9) | 5 (62.5) | ||
| Introduction, background, and objectives | ||||
| The PRO hypothesis should be stated and specify the relevant PRO domain if applicable | No | 5 (55.6) | 5 (62.5) | 1 |
| Yes | 2 (22.2) | 1 (12.5) | ||
| N/A (if explorative) | 2 (22.2) | 2 (25) | ||
| Methods | ||||
| Outcomes | ||||
| The mode of administration of the PRO tool and the methods of collecting data should be described | No | 7 (77.8) | 5 (62.5) | 0.62 |
| Yes | 2 (22.2) | 3 (37.5) | ||
| Electronic mode of PRO administrationa | No | 1 (11.1) | 2 (25) | 1 |
| Yes | 1 (11.1) | 0 (0) | ||
| N/A | 7 (77.8) | 6 (75) | ||
| The rationale for choice of the PRO instrument used should be provided | No | 4 (44.4) | 4 (50) | 1 |
| Yes | 5 (55.6) | 4 (50) | ||
| Evidence of PRO instrument validity and reliability should be provided or cited | No | 4 (44.4) | 3 (37.5) | 0.44 |
| Yes, for all PRO instruments | 5 (55.6) | 3 (37.5) | ||
| Yes, only for some PRO instruments | 0 (0) | 2 (25) | ||
| The intended PRO data collection schedule should be provided | No | 2 (22.2) | 1 (12.5) | 1 |
| Yes | 7 (77.8) | 7 (87.5) | ||
| PROs should be identified in the trial protocol post-hoc analyses | No | 9 (100) | 4 (50) | 0.03a |
| Yes | 0 (0) | 4 (50) | ||
| The status of PRO as either a primary or secondary outcome should be stated | No | 2 (22.2) | 3 (37.5) | 0.62 |
| Yes | 7 (77.8) | 5 (62.5) | ||
| Statistical methods | ||||
| There should be evidence of appropriate statistical analysis and tests of statistical significance for each PRO hypothesis tested | No | 0 (0) | 2 (25) | 0.223 |
| Yes | 2 (22.2) | 0 (0) | ||
| N/A | 7 (77.8) | 6 (75) | ||
| The extent of missing data should be statedb | No | 8 (88.9) | 2 (25) | 0.015a |
| Yes | 1 (11.1) | 6 (75) | ||
| Statistical approaches for dealing with missing data should be explicitly statedb | No | 9 (100) | 6 (75) | 0.206 |
| Yes | 0 (0) | 2 (25) | ||
| Results | ||||
| Participant flow | ||||
| A flow diagram or a description of the allocation of participants and those lost to follow-up should be provided for PROs specifically | No | 7 (77.8) | 5 (62.5) | 0.62 |
| Yes | 2 (22.2) | 3 (37.5) | ||
| The reasons for missing data should be explained | No | 8 (88.9) | 5 (62.5) | 0.294 |
| Yes | 1 (11.1) | 3 (37.5) | ||
| Baseline data | ||||
| The study patients characteristics should be described including baseline PRO scores | No | 6 (66.7) | 3 (37.5) | 0.347 |
| Yes | 3 (33.3) | 5 (62.5) | ||
| Outcomes and estimation | ||||
| PRO outcomes also reported in a graphical formata | No | 5 (55.6) | 6 (75) | 0.62 |
| Yes | 4 (44.4) | 2 (25) | ||
| Discussion | ||||
| Limitations | ||||
| The limitations of the PRO components of the trial should be explicitly discussed | No | 5 (55.6) | 4 (50) | 1 |
| Yes | 4 (44.4) | 4 (50) | ||
| Generalizability | ||||
| Generalizability issues uniquely related to the PRO results should be discussed | No | 5 (55.6) | 6 (75) | 0.62 |
| Yes | 4 (44.4) | 2 (25) | ||
| Interpretation | ||||
| PROs are interpreted (Not only re-stated)a | No | 2 (22.2) | 5 (62.5) | 0.153 |
| Yes | 7 (77.8) | 3 (37.5) | ||
| The clinical significance of the PRO findings should be discussed | No | 6 (66.7) | 6 (75) | 1 |
| Yes | 3 (33.3) | 2 (25) | ||
| Methodology used to assess clinical significance is discusseda | Anchor based (e.g., minimal important difference) | 1 (11.1) | 0 (0) | 1 |
| Distribution based (e.g. effect size) | 1 (11.1) | 2 (25) | ||
| Both | 1 (11.1) | 0 (0) | ||
| Missing | 6 (66.7) | 6 (75) | ||
| The PRO results should be discussed in the context of the other clinical trial outcomes | No | 2 (22.2) | 1 (12.5) | 1 |
| Yes | 7 (77.8) | 7 (87.5) | ||