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

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.

A plain language summary of the perspectives of women who were interviewed about their experiences with uterine fibroids.

WHAT IS THIS SUMMARY ABOUT?: This summary describes what researchers learned during interviews of women with uterine fibroids and heavy menstrual bleeding (or period bleeding). At this time, little is known about how women perceive the impact of uterine fibroids on their lives and more information is needed. The goal of this study was to provide new information about the symptoms women have and how these symptoms affect their everyday lives. These interviews were done to better understand how uterine fibroid symptoms affect the lives of women in their own words. WHAT WERE THE RESULTS?: Thirty women from the United States, who had completed a clinical trial for a new treatment for heavy menstrual bleeding and uterine fibroids, agreed to be interviewed. The women described what their experiences with uterine fibroids were and the impact these experiences with uterine fibroids had on their lives before participating in the clinical trial. The most common symptoms of uterine fibroids the women described were heavy bleeding with their menstrual periods, pain in the pelvis or groin area, the passing of blood clots, and anemia (or low hemoglobin in red blood cells). Women said their symptoms affected them physically, emotionally, socially, and financially. They also said their symptoms made it hard to do daily activities, sleep, have a sex life, and go to work or school. WHAT DO THE RESULTS MEAN?: Women who have heavy menstrual bleeding and uterine fibroids experience various uterine fibroid symptoms, and these symptoms affect most parts of the their lives.

The Burden of Uterine Fibroids from the Perspective of US Women Participating in Open-Ended Interviews.

Background: Research on women's perspective of uterine fibroids (UF) experiences using their own words is limited. This study aimed to provide new insights on the symptoms experienced and their impacts on daily life. Methods: Interview substudy in 30 US women with heavy menstrual bleeding (HMB) associated with UF who completed one of two phase 3, randomized, double-blind, placebo-controlled trials (LIBERTY 1 and 2; ClinicalTrials.gov identifiers: NCT03049735, NCT03103087). Women who consented to participate in this substudy were interviewed after their last clinical trial study visit. Concepts (i.e., symptoms and impacts) of importance to women were determined via open-ended questions, and the frequency of symptoms and their impacts, including the relationship between pain and menstruation, were assessed. Data were analyzed using established qualitative research methods, including grounded theory and constant comparative methods, and concept saturation was assessed. Results: Fifteen unique symptoms of UF emerged: the most commonly reported were HMB (n = 30, 100.0%), pelvic pain (n = 28, 93.3%), and passing of blood clots (n = 24, 80.0%). In total, 25 unique impacts were identified across eight concepts: physical impacts, activities of daily living, sleep, emotional impacts, sex life, social impacts, work and school, and financial impacts. Concept saturation was achieved for both symptoms and impacts. Conclusion: This study provides data on the symptoms experienced by women with HMB associated with UF, as well as the negative impacts of these symptoms as reported using their own words. The study findings confirm the significant burden associated with symptomatic UF.

Patient Perspectives on Health-Related Quality of Life in Diffuse Large B-Cell Lymphoma Treated with Car T-Cell Therapy: A Qualitative Study.

INTRODUCTION: Chimeric antigen receptor T-cell (CAR T) therapy offers a potentially curative option for patients with relapsed and refractory hematologic malignancies, including diffuse large B-cell lymphoma (DLBCL). Patient-reported experiences with CAR T therapy are limited and have not been well characterized. The purpose of this qualitative study was to explore patient descriptions of key domains of health-related quality of life (HRQoL) in DLBCL patients treated with CAR T therapy. METHODS: A targeted literature review was initially conducted to inform the development of the interview guide comprising predetermined open-ended questions. Two focus groups were conducted with a total of 18 patients with DLBCL identified from patient advisory boards. Focus group sessions were recorded and transcribed verbatim. MAXQDA 18.2.0 qualitative data analysis software was utilized to facilitate a constant-comparative coding process to identify key concepts. RESULTS: Eight domain impairments (social functioning, emotional functioning, fatigue, physical functioning, cognitive functioning, role functioning, sleep, and pain/discomfort) were identified from the qualitative analysis and endorsed by DLBCL patients treated with CAR T. Compared with before CAR T therapy, patients reported increased impairment in every domain during or immediately after CAR T therapy. This impairment improved for each domain 6 months after CAR T therapy except for pain/discomfort. Compared with before CAR T therapy, improvement in impairment for each domain was observed 6 months after CAR T therapy except for fatigue, sleep, and pain/discomfort. CONCLUSION: This study provides meaningful information regarding the impact of CAR T therapy on HRQoL in patients with DLBCL throughout their treatment journey. Health care professionals and investigators can utilize these data in examining existing patient-reported outcome (PRO) measures that are used in DLBCL clinical trials and to better understand the needs of DLBCL survivors.

Results from Patient Interviews on Fatigue in Progressive Multiple Sclerosis and Evaluation of Fatigue Patient-Reported Outcome (PRO) Instruments.

INTRODUCTION: Fatigue is one of the most common and debilitating symptoms of multiple sclerosis (MS) but is challenging to assess and has not been comprehensively characterized in patients with progressive MS. This study aimed to (1) obtain qualitative evidence from patients with progressive MS to characterize MS-related fatigue concepts and their impacts on health-related quality of life (HRQoL), and (2) evaluate the conceptual frameworks of existing MS-specific fatigue patient-reported outcome (PRO) instruments using study data to determine the most suitable PRO instrument in this population. METHODS: Qualitative interviews were conducted with 30 US participants with confirmed progressive MS and fatigue in the last 6 months to assess their MS-related fatigue. Data were compared with concepts in existing PRO instruments to evaluate their relevance in progressive MS. RESULTS: Physical and mental concepts of fatigue were identified and characterized distinctly from patients with progressive MS. Most patients characterized fatigue as occurring daily and lasting several hours, with negative impacts on HRQoL. Concept mapping to existing MS-specific fatigue PRO instruments supported the Fatigue Severity Impact Questionnaire-Relapsing Multiple Sclerosis (FSIQ-RMS) as the most suitable existing option for assessing fatigue in patients with progressive MS, as it separates physical and mental aspects of fatigue and includes every highly endorsed concept reported by the interviewed patients. CONCLUSIONS: This qualitative study identified meaningful physical and mental fatigue concepts in patients with progressive MS and preliminarily supports the use of the FSIQ-RMS for this population. More research is needed to fully validate this instrument for progressive MS.

Evaluation and Validation of the Modified Reflux Symptom Questionnaire-Electronic Diary in Patients With Persistent Gastroesophageal Reflux Disease.

OBJECTIVES: This study aimed to examine the validity of the modified Reflux Symptom Questionnaire-electronic Diary (mRESQ-eD) through patient input and psychometric testing of the questionnaire to support use in clinical trials in patients with persistent gastroesophageal reflux disease (GERD) and in accordance with Food and Drug Administration guidance on patient-reported outcome instruments. METHODS: Cognitive interviews were conducted with patients (n = 30) to evaluate the interpretability and content validity of draft mRESQ-eD items. Patient data from a phase 2b clinical study (ClinicalTrials.gov identifier: NCT02637557) on persistent GERD served to aid in the construction of weekly scores for heartburn severity, regurgitation severity, and total GERD severity. These scores' psychometric properties were also evaluated. RESULTS: Minor modifications were made to the draft mRESQ-eD based on patient feedback to improve interpretability and clarity of the instrument. Psychometric analysis suggested that an 8-item version of the mRESQ-eD was best suited to the clinical data. The internal consistency was found to be high (Coefficient ω = 0.95). Retest reliability and convergent validity were strong for a heartburn weekly severity score, regurgitation weekly severity score, and total GERD severity score. DISCUSSION: The final 8-item mRESQ-eD is a reliable and valid instrument with good psychometric properties for use in clinical trials in patients with persistent GERD. The mRESQ-eD may be considered for inclusion in clinical trials for persistent GERD and potentially positioned, in consultation with Food and Drug Administration, as endpoints to characterize treatment benefit.