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Table 1 Methods used to investigate psychometric and other properties of the MP and UF-DBD

From: Validation of a menstrual pictogram and a daily bleeding diary for assessment of uterine fibroid treatment efficacy in clinical studies

Statistical analysis PRO scores Time points Reference measures Population Interpretation
Item performance/variability – extent to which potential available response option for each item is selected by patients
 Descriptive statistics • MP
• UF-DBD item levela
• Monthly and bleeding episode sum scores
• RND
• EOT
NA All patients, by study • Distributional properties
• Floor and ceiling effects
Reliability/Test-retest reliability – ability to give reproducible, consistent scores over a short time period in stable patients
 1. Descriptive statistics,
Wilcox signed rank test
 2. Intraclass correlation coefficient
• MP
• UF-DBD
• Monthly and bleeding episode sum scores
• SCR2, RND
• T2, EOT
• AHb
• PGI-S
Stable patients by study:
 • AH method: MP and UF-DBD in ASTEROID 1c
 • PGI-S scores: UF-DBD and MP in ASTEROID 1 and 2
UF-DBD
 • ICC ≥0.50: moderate
MP
 • ICC < 0.40: poor
 • 0.40 to 0.59: moderate
 • 0.60 to 0.74: good
 • 0.75+: excellent
Construct validity – extent to which a scale measures the intended construct
Known-groups validity – ability of measure to discriminate between patient groups differing in levels of condition severity
  1. Jonckheere-Terpstra test
  2. Kruskal-Wallis test
• MP
• UF-DBD
• Monthly and bleeding episode sum scores
• RND
• EOT
• AHb
• PGI-S
All patients, by studyc Significance of ordered difference between known groups
Convergent and divergent validity – extent of association between a measure and other measures or variables based on an expected relationship
  1. Spearman rank correlation
  2. Scatterplots
• MP
• UF-DBD
• Monthly and bleeding episode sum scores
• RND
• EOT
• pooled RND and EOT
• MP
• UF-DBD
• UF-DSD v3
• UF-IS v3
• UFS-QoL
• SF-36 v2®
All patients, by study Strength of correlations
 • 0.10 to 0.29: weak
 • 0.30 to 0.49: moderate
 • 0.50 to 1.0: strong
Criterion validity – extent of relation between PRO instrument scores and a known gold standard measure of the same concept
 1. Spearman rank correlation
 2. Scatterplots
• MP
• UF-DBD
• Monthly and bleeding episode sum scores
• RND
• EOT
• AHb All patients with AH measurements Strength of correlations
 • 0.10 to 0.29: weak
 • 0.30 to 0.49: moderate
 • 0.50 to 1.0: strong
Responsiveness – ability to detect change when a change in the measured concept has occurred
 1. Spearman rank correlation, Scatterplots
 2. Kruskal-Wallis test, Jonckheere-Terpstra test
• MP
• UF-DBD
• Monthly and bleeding episode sum scores
• RND
• EOT
Correlation analysis
 • MP
 • UF-DBD
 • UF-DSD v3d
 • UF-IS v3d
 • UFS-QoLd
 • AHb
 • PGI-S
Definition of change
 • AHb
 • PGI-S
All patients, by studyc Strength of correlations
 • 0.10 to 0.29: weak
 • 0.30 to 0.49: moderate
 • 0.50 to 1.0: strong
Significant or significant ordered difference across the groups
Missing data
 Descriptive statistics; frequencies and percentages of missing data (daily scores over time) • MP
• UF-DBD
• RND
• EOT
AH ASTEROID 1, patients with AH measurements: all patients except for Japanese centerse and only US patients  
Comparability of methods (AH, MP, UF-DBD)
 1. Cross-tabulation of benchmark scores (HMB eligibility, responder status and amenorrhea, calculation of sensitivity, specificity, PPV and NPV)
 2. Kaplan–Meier curves; descriptive statistics, histograms of difference
• MP
• UF-DBD
• RND
• EOT
AH ASTEROID 1, patients with AH measurements  
  1. AH Alkaline hematin method, EOT End of treatment, ICC Intraclass correlation coefficient, MP (MP SAP-cv3) Menstrual pictogram superabsorbent polymer-containing version 3, NA Not applicable/available, NPV Negative predictive value, PPV Positive predictive value, PGI-S Patient Global Impression of Severity, PRO Patient-reported outcomes, RND Randomization, SF-36 v2® Short-Form 36 Health Survey Version 2, SCR Screening, T Treatment, UF-DBD Uterine Fibroid Daily Bleeding Diary, UF-DSD v3b Uterine Fibroid Daily Symptom Diary version 3, UF-IS v3 Uterine Fibroid Impact Scale version 3, UFS-QoL Uterine Fibroid Symptom and Quality of Life Questionnaire
  2. aMonthly sum scores were based on the 28 days prior to and including the visit date; bleeding episode sum scores were collected on or closest to the visit date within 27 days prior
  3. bASTEROID 1 only
  4. cFor analyses including the AH method, only data from patients with AH measurements in ASTEROID 1 were used
  5. dUse of total instrument scores only
  6. eAH measurements were not performed in patients from Japanese centers; therefore, these patients were excluded from analysis