TABLE 3.
Patient‐reported outcomes (PRO) reporting according to ISOQOL PRO Guidelines and study design
| Variable | Total | Open‐label | Blinded | P | |||
|---|---|---|---|---|---|---|---|
| No. | (%) | No. | (%) | No. | (%) | ||
| 110 | (100) | 68 | (100) | 42 | (100) | ||
| Title and abstract | |||||||
| The PRO should be identified as an outcome in the abstract. | .69 | ||||||
| No | 9 | (8.2) | 5 | (7.3) | 4 | (9.5) | |
| Yes | 101 | (91.8) | 63 | (92.7) | 38 | (90.5) | |
| The title of the paper should be explicit as to the RCT including a PRO * | .63 | ||||||
| No | 17 | (44.7) | 11 | (47.8) | 6 | (40.0) | |
| Yes | 21 | (55.3) | 12 | (52.2) | 9 | (60.0) | |
| Introduction, background, and objectives | |||||||
| The PRO hypothesis should be stated and specify the relevant PRO domain, if applicable. | .58 | ||||||
| No | 26 | (23.6) | 16 | (23.5) | 10 | (23.8) | |
| Yes | 36 | (32.7) | 20 | (29.4) | 16 | (38.1) | |
| N/A (if explorative) | 48 | (43.7) | 32 | (47.1) | 16 | (38.1) | |
| The introduction should contain a summary of PRO research that is relevant to the RCT * | .22 | ||||||
| No | 9 | (23.7) | 7 | (30.4) | 2 | (13.3) | |
| Yes | 29 | (76.3) | 16 | (69.6) | 13 | (86.7) | |
| Additional details regarding the hypothesis should be provided, including the rationale for the selected domains, the expected directions of change, and the time points for assessment * | 0.09 | ||||||
| No | 31 | (81.6) | 21 | (91.3) | 10 | (66.7) | |
| Yes | 7 | (18.4) | 2 | (8.7) | 5 | (33.3) | |
| Methods | |||||||
| Outcomes | |||||||
| The mode of administration of the PRO tool and the methods of collecting data should be described | .61 | ||||||
| No | 89 | (80.9) | 54 | (79.4) | 35 | (83.3) | |
| Yes | 21 | (19.1) | 14 | (20.6) | 7 | (16.7) | |
| The rationale for the choice of the PRO instrument used should be provided. | .02 | ||||||
| No | 47 | (42.7) | 23 | (33.8) | 24 | (57.1) | |
| Yes | 63 | (57.3) | 45 | (66.2) | 18 | (42.9) | |
| Evidence of PRO instrument validity and reliability should be provided or cited. | .23 | ||||||
| No | 31 | (28.2) | 17 | (25.0) | 14 | (33.3) | |
| Yes, for All PRO instruments | 54 | (49.1) | 32 | (47.1) | 22 | (52.4) | |
| Yes, only for some PRO instruments | 25 | (22.7) | 19 | (27.9) | 6 | (14.3) | |
| The intended PRO data collection schedule should be provided. | .60 | ||||||
| No | 11 | (10.0) | 6 | (8.8) | 5 | (11.9) | |
| Yes | 99 | (90.0) | 62 | (91.2) | 37 | (88.1) | |
| PRO should be identified in the trial protocol; post hoc analyses should be identified. | <.01 | ||||||
| No | 67 | (60.9) | 49 | (72.1) | 18 | (42.9) | |
| Yes | 43 | (39.1) | 19 | (27.9) | 24 | (57.1) | |
| The status of PRO as either a primary or secondary outcome should be stated. | .07 | ||||||
| No | 11 | (10.0) | 10 | (14.7) | 1 | (2.4) | |
| Yes | 88 | (80.0) | 50 | (73.5) | 38 | (90.5) | |
| Unclear | 11 | (10.0) | 8 | (11.8) | 3 | (7.1) | |
| A citation for the original development of the PRO instrument should be provided * | .57 | ||||||
| No | 14 | (36.8) | 7 | (30.4) | 7 | (46.8) | |
| Yes | 13 | (34.2) | 9 | (39.2) | 4 | (26.7) | |
| Yes, only for some PRO instruments | 11 | (29.0) | 7 | (30.4) | 4 | (26.7) | |
| Windows for valid PRO responses should be specified and justified as being appropriate for the clinical context * | .46 | ||||||
| No | 18 | (47.4) | 12 | (52.2) | 6 | (40.0) | |
| Yes | 20 | (52.6) | 11 | (47.8) | 9 | (60.0) | |
| Sample size | |||||||
| There should be a power sample size calculation relevant to the PRO based on a clinical rationale * | .72 | ||||||
| No | 14 | (36.8) | 9 | (39.1) | 5 | (33.3) | |
| Yes | 24 | (63.2) | 14 | (60.9) | 10 | (66.7) | |
| Statistical methods | |||||||
| There should be evidence of appropriate statistical analysis and tests of statistical significance for each PRO hypothesis tested. | .57 | ||||||
| No | 5 | (4.5) | 3 | (4.4) | 2 | (4.8) | |
| Yes | 33 | (30.0) | 18 | (26.5) | 15 | (35.7) | |
| N/A (if PRO hypotheses were not stated) | 72 | (65.5) | 47 | (69.1) | 25 | (59.5) | |
| The extent of missing data should be stated. a | .44 | ||||||
| No | 32 | (29.1) | 18 | (26.5) | 14 | (33.3) | |
| Yes | 78 | (70.9) | 50 | (73.5) | 28 | (66.7) | |
| Statistical approaches for dealing with missing data should be explicitly stated. a | .68 | ||||||
| No | 81 | (73.6) | 51 | (75.0) | 30 | (71.4) | |
| Yes | 29 | (26.4) | 17 | (25.0) | 12 | (28.6) | |
| The manner in which multiple comparisons have been addressed should be provided * | .80 | ||||||
| No | 27 | (71.1) | 16 | (69.6) | 11 | (73.3) | |
| Yes | 11 | (28.9) | 7 | (30.4) | 4 | (26.7) | |
| Results | |||||||
| Participant flow | |||||||
| A flow diagram or a description of the allocation of participants and those lost to follow‐up should be provided for PRO specifically. | .37 | ||||||
| No | 61 | (55.5) | 40 | (58.8) | 21 | (50.0) | |
| Yes | 49 | (44.5) | 28 | (41.2) | 21 | (50.0) | |
| The reasons for missing data should be explained. | 0.60 | ||||||
| No | 70 | (63.6) | 42 | (61.8) | 28 | (66.7) | |
| Yes | 40 | (36.4) | 26 | (38.2) | 14 | (33.3) | |
| Baseline data | |||||||
| The study patients’ characteristics should be described, including baseline PRO scores. | .88 | ||||||
| No | 35 | (31.8) | 22 | (32.4) | 13 | (30.9) | |
| Yes | 75 | (68.2) | 46 | (67.6) | 29 | (69.1) | |
| Outcomes and estimation | |||||||
| Are PRO outcomes also reported in a graphical format? b | .44 | ||||||
| No | 39 | (35.5) | 26 | (38.2) | 13 | (30.9) | |
| Yes | 71 | (64.5) | 42 | (61.8) | 29 | (69.1) | |
| The analysis of PRO data should account for survival differences between treatment groups, if relevant * | .45 | ||||||
| No | 1 | (2.6) | 1 | (4.3) | 0 | (0) | |
| Yes | 5 | (13.2) | 2 | (8.7) | 3 | (20.0) | |
| N/A (if not relevant) | 32 | (84.2) | 20 | (87.0) | 12 | (80.0) | |
| Results should be reported for all PRO domains (if multidimensional) and items identified by the reference instrument * | .97 | ||||||
| No | 10 | (26.3) | 6 | (26.1) | 4 | (26.7) | |
| Yes | 28 | (73.7) | 17 | (73.9) | 11 | (73.3) | |
| The proportion of patients achieving predefined responder definitions should be provided where relevant * | .11 | ||||||
| No | 4 | (10.5) | 1 | (4.4) | 3 | (20.0) | |
| Yes | 7 | (18.4) | 3 | (13.0) | 4 | (26.7) | |
| N/A (if not relevant) | 27 | (71.1) | 19 | (82.6) | 8 | (53.3) | |
| Discussion | |||||||
| Limitations | |||||||
| The limitations of the PRO components of the trial should be explicitly discussed | .60 | ||||||
| No | 70 | (63.6) | 42 | (61.8) | 28 | (66.7) | |
| Yes | 40 | (36.4) | 26 | (38.2) | 14 | (33.3) | |
| Generalizability | |||||||
| Generalizability issues uniquely related to the PRO results should be discussed. | .42 | ||||||
| No | 47 | (42.7) | 27 | (39.7) | 20 | (47.6) | |
| Yes | 63 | (57.3) | 41 | (60.3) | 22 | (52.4) | |
| Interpretation | |||||||
| Are PRO interpreted (not just restated)? b | .23 | ||||||
| No | 28 | (25.5) | 20 | (29.4) | 8 | (19.1) | |
| Yes | 82 | (74.5) | 48 | (70.6) | 34 | (80.9) | |
| The clinical significance of the PRO findings should be discussed. | .12 | ||||||
| No | 74 | (67.3) | 42 | (61.8) | 32 | (76.2) | |
| Yes | 36 | (32.7) | 26 | (38.2) | 10 | (23.8) | |
| The PRO results should be discussed in the context of the other clinical trial outcomes. | .19 | ||||||
| No | 15 | (13.6) | 7 | (10.3) | 8 | (19.1) | |
| Yes | 95 | (86.4) | 61 | (89.7) | 34 | (80.9) | |
| Other information | |||||||
| Protocol | |||||||
| A copy of the instrument should be included if it has not been published previously * | .35 | ||||||
| No | 15 | (39.5) | 11 | (47.8) | 4 | (26.7) | |
| Yes | 12 | (31.5) | 7 | (30.4) | 5 | (33.3) | |
| N/A (if the instrument is already published or known in the literature) | 11 | (29.0) | 5 | (21.8) | 6 | (40.0) | |
a
These items were originally combined in the ISOQOL recommended standards but have been split in this report to better investigate possible discrepancies between documentation of PRO missing data (ie, reporting how many patients did not complete a given questionnaire at any given time point) versus actual reporting of statistical methods to address this issue.
b
These items were not included in the ISOQOL recommended standards but have been evaluated in our study and reported in this table to have a wider outlook on the level of reporting.
*
Additional standards only for PRO as primary outcome, number of trials = 38.