Table 4.
Data reporting, clinical relevance, and statistical analysis methods used for PRO data (n = 21)
| Variable | No. (%) |
|---|---|
| Data reporting | |
| Definition of study population | |
| Patients with a baseline assessment and at least 1 postbaseline assessment | 8 (38.1) |
| Intent to treat | 5 (23.8) |
| Patients who received at least 1 dose of study medication and completed at least 1 assessment | 4 (19.0) |
| Modified intent-to-treat population | 1 (4.8) |
| Intent-to-treat population with nonmissing baseline measurements | 1 (4.8) |
| Not clearly defined | 2 (9.5) |
| PRO completion rate | |
| Completion/compliance rate table included in the manuscript | 17 (81.0) |
| Clinical relevance threshold was prespecified as | |
| Change of X points from baseline within-patient or treatment group | 12 (57.1) |
| Difference of X points between arms at a certain timepoint | 2 (9.5) |
| Both | 3 (14.3) |
| Not reported | 4 (19.0) |
| Clinical relevance was justified and citeda | 17 (81.0) |
| Reporting of descriptive data | 18 (85.7) |
| Data analysis | |
| Primary statistical technique | |
| Time-to-event analysis | |
| Cox proportional hazard/Cox regression model | 11 (52.4) |
| Log rank test | 6 (28.6) |
| Brookmeyer and Crowley | 1 (4.8) |
| Magnitude of change from baseline analysis | |
| Mixed-model for repeated measures | 6 (28.6) |
| cLDA/LDA | 4 (19.0) |
| Mixed effects model | 3 (14.3) |
| ANCOVA | 2 (9.5) |
| t tests | 3 (14.3) |
| Chi-square | 2 (9.5) |
| Conventional Wald method | 1 (4.8) |
| ANOVA | 1 (4.8) |
| Linear regression | 1 (4.8) |
| Response trajectory over time | |
| Linear mixed effects model | 4 (19.0) |
| Symptom improvement rate at time t analysis | |
| Clopper Pearson | 2 (9.5) |
| Logistic regression | 2 (9.5) |
| Not reported or unclear | 2 (9.5) |
| PRO scores were compared at baseline between 2 arms | 13 (61.9) |
| Control for type I error | 3 (14.3) |
| PRO data analysis stratified by ethnicity/race | 0 |
| Handling of missing data | |
| Strategy to deal with missing data is definedb | 17 (81.0) |
| Detailed reasons for missing data by timepoint reported | 0 |
| PRO specific limitations stated in the discussion section | 16 (76.2) |
| PRO conclusion | |
| Improvement in key PROsc | 6 (28.6) |
| Stable PROsc | 1 (4.8) |
| Experimental arm is superior to control arm | 9 (42.9) |
| Similar outcomes between experimental arm and control arm | 3 (14.3) |
| Experimental arm is inferior to control arm | 2 (9.5) |
a
Articles that did not justify clinical relevance where those that did not specify the clinical relevance. Numbers are rounded to the nearest whole number. ANCOVA = analysis of covariance; ANOVA = analysis of variance; cLDA = constrained LDA; LDA = longitudinal data analysis; PRO = patient-reported outcome.
b
Two manuscripts stated that the approach to deal with missing data was “no imputations” and 1 stated that the approach to deal with missing data was “left as missing.”
c
Single-arm studies.