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Development of the Bleeding and Pelvic Discomfort Scale for Use in Women With Heavy Menstrual Bleeding Associated With Uterine Fibroids

      Highlights

      • Uterine fibroids (UFs) are the most common type of benign uterine tumor; patients experience a high symptom burden that affects their health-related quality of life and activities of daily living. There is currently a need for a concise measure of cardinal UF symptoms, which is appropriate for use in clinical trials.
      • The bleeding and pelvic discomfort (BPD) scale was derived from the symptom severity scale of the Uterine Fibroid Symptom and Quality of Life questionnaire to obtain a patient-centric symptom endpoint for clinical trial use. Patient exit interviews confirmed the cardinal UF symptoms included in the BPD; its psychometric properties were found to be good, including reliability, known-groups validity, and ability to detect change.
      • The meaningful change threshold for the BPD, derived based on anchor-based methods supported by patient input, can be used to assess whether a patient is a responder to treatment, that is, experiences a relevant and meaningful improvement.

      Abstract

      Objectives

      This study aimed to define a cardinal symptom burden measure based on items from the Uterine Fibroid Symptom and Quality of Life questionnaire for use as a clinical trial endpoint.

      Methods

      Exploratory factor analysis was computed to assess the Uterine Fibroid Symptom and Quality of Life symptom severity scale factor structure, using phase 2 data. Pooled blinded data from phase 3 studies were used for the confirmatory factor analysis and the psychometric evaluation of the new measure. Exit interviews in 30 patients from phase 3 studies provided additional qualitative evidence. A meaningful change threshold was determined using anchor-based analyses supported by patient feedback in the exit interviews.

      Results

      Three factors emerged from the exploratory factor analysis. Factor 1, called the bleeding and pelvic discomfort (BPD) scale, consists of cardinal symptoms, measuring menstrual distress owing to heavy bleeding, passing blood clots, and feeling tightness or pressure in pelvic area. Patients generally understood the items in the scale and the recall period as intended. The BPD scale had good item performance and internal consistency reliability, strong item-to-total correlations, good item discrimination, known-groups validity, and ability to detect change. A 20-point change on the BPD scale was determined as the clinically meaningful change threshold.

      Conclusions

      The BPD scale assesses symptom burden owing to bleeding, passing blood clots, and pelvic pressure. The subscale is based on a subset of items selected to measure the cardinal symptoms of uterine fibroids in a clinical trial setting. The responder threshold evaluates whether patients experience a meaningful treatment benefit over the on-treatment period.

      Keywords

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