BACKGROUND: In some women, uterine fibroids are associated with severe, disabling symptoms. There is a lack of high-quality evidence supporting the effectiveness of most interventions for symptomatic uterine fibroids. In part, this is due to the lack of available disease-specific instruments with comprehensive validation evidence that measure treatment benefit from the patient perspective.
OBJECTIVE: The aim of this study was to develop an electronic patient-reported outcomes (PRO) instrument that measures treatment benefit from the patient's perspective that is easily administered and practical for use in clinical trials. Rigorous methods that are consistent with the US FDA's PRO Guidance to Industry were employed.
METHODS: The study took place in two phases: a content development phase (eight focus groups; three sets of cognitive interviews) and a prospective non-intervention usability pilot phase. Both phases were conducted in the US. The study population comprised women diagnosed with symptomatic uterine fibroids. A total of 68 women (mean age 40 years) participated in the eight focus groups; 27 women (mean age 41 years) who were not part of the focus groups participated in the cognitive interviews. Fourteen additional women (mean age 39 years) participated in a usability pilot. Efforts were made to recruit a diverse population with respect to race and education.
RESULTS: After completing eight focus groups, no new symptom concepts or severity-level measurement ideas were introduced, indicating that concept saturation was achieved. Fourteen draft items were developed during the focus groups for testing in the cognitive interviews. Every symptom represented by the draft items was endorsed by at least two-thirds of the participants in the cognitive interviews. After completing three rounds of cognitive interviews, the Fibroid Symptom Diary© (FSD) contained eight items that assessed bleeding severity, menstrual cramping, and fibroid-related fatigue. An open-field item, tailored to each participant, was also included to assess the most bothersome fibroid-related pain. However, to accommodate electronic administration of the diary this item was replaced, prior to the usability pilot, with three pain-specific items (i.e. abdominal pain, low back pain, and pain during intercourse) that were most commonly expressed during the development phase. The final FSD includes 11 items: five addressing menstrual bleeding or spotting; one each relating to cramping (distinct from other pain), fatigue, and bloating; and three that address other fibroid-related pain. The average time for completing the diary was 1-2 minutes per day. A total of 118 daily diary records were collected from 14 participants (average of nine daily diary completions per participant; range 5-18 days) in the usability pilot. Seven participants completed the diary every day. Most participants experienced the majority of the symptoms included in the FSD.
CONCLUSIONS: The FSD captures the concepts most important to women with uterine fibroids and has strong evidence of content validity as required by the FDA PRO Guidance to Industry. Once fully validated, the FSD may replace other measures for assessing changes in symptoms and treatment benefit that are both burdensome to patients and cumbersome to trial sponsors.
