The Burden of Pain Symptoms in Individuals with Uterine Fibroids-Results from a Prospective Observational Study in the USA.

Purpose: To characterize the burden of uterine fibroids (UF) in individuals experiencing heavy menstrual bleeding (HMB) and moderate-to-severe UF-associated pain in terms of symptoms experienced, impact on work and activities, and pain medication use both on menstrual and non-menstrual days.

Patients And Methods: This prospective, real-world, observational study enrolled 350 participants in the USA with a self-reported UF diagnosis, HMB, and moderate-to-severe pain due to UF. Data collection took place from February 9 to July 19, 2021.

Patient Preferences for Attributes of Androgen Deprivation Therapies in Prostate Cancer: A Discrete Choice Experiment with Latent Class Analysis.

Introduction: Medical androgen deprivation therapy (ADT) options have expanded for patients with advanced prostate cancer (PC). Historically, ADT was primarily available in long-acting injectable formulations. In 2020, the first oral formulation was US Food and Drug Administration-approved for adults with advanced PC.

Impact of relugolix combination therapy on functioning and quality of life in women with endometriosis-associated pain.

Objective: To evaluate the effect of relugolix combination therapy (relugolix CT; 40 mg relugolix, 1 mg estradiol, and 0.5 mg norethisterone acetate) for up to 2 years in the SPIRIT long-term extension study on functioning and health-related quality of life (QoL), using the Endometriosis Health Profile (EHP)-30 questionnaire, and assess how changes in QoL domains correlated with improvements in dysmenorrhea as well as nonmenstrual pelvic pain (NMPP).

Design: Long-term extension study of the SPIRIT phase 3 trials.

Development of a Novel Patient-Reported Outcome Measure to Assess Symptoms and Impacts of Androgen Deprivation Therapy for Advanced Prostate Cancer.

Introduction: This qualitative research study was conducted to develop a novel, comprehensive, patient-reported outcome measure (PRO), the "Symptoms and Impacts of Androgen Deprivation Therapy (ADT) for Prostate Cancer" (SIADT-PC), assessing hormonal therapy-related symptoms and their impacts on men with advanced prostate cancer.

Methods: Concept elicitation (CE) interviews were conducted among adult men with prostate cancer to evaluate their experiences with ADT. Based on key symptom and impact concepts mentioned, an initial PRO measure was developed.

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.

Endometriosis Symptoms and Their Impacts on the Daily Lives of US Women: Results from an Interview Study.

Objective: This interview study sought to capture patients' experiences and perceptions of endometriosis symptoms and their impacts on daily life, as described by women in their own words. Using open-ended questions and a concept-elicitation approach, this study assessed the signs and symptoms of endometriosis and their impacts on different aspects of quality of life, including daily activities, functioning, and well-being.

Materials And Methods: This interview study included US women with moderate-to-severe endometriosis-associated pain who completed one of two Phase 3, randomized, double-blind, placebo-controlled trials (SPIRIT 1 or SPIRIT 2; ClinicalTrials.

Quality of life with relugolix combination therapy for uterine fibroids: LIBERTY randomized trials.

Background: Symptomatic uterine fibroids are burdensome to live with; they are associated with symptom-related distress, affect daily activities, and reduce health-related quality of life. The LIBERTY randomized clinical trials showed that oral relugolix combination therapy (40 mg relugolix, 1 mg estradiol, and 0.5 mg norethindrone acetate once daily) markedly improved fibroid-associated symptoms and conditions, including heavy menstrual bleeding, pain, and anemia, and was well-tolerated.

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.

Relugolix Combination Therapy for Uterine Leiomyoma-Associated Pain in the LIBERTY Randomized Trials.

Objective: To assess the effect of once-daily relugolix combination therapy (relugolix-CT: relugolix 40 mg, estradiol 1 mg, and norethindrone acetate 0.5 mg) compared with placebo on moderate-to-severe pain in women with uterine leiomyomas and heavy menstrual bleeding.

Methods: Two replicate, multinational, double-blind, 24-week, randomized, phase 3 studies (LIBERTY 1 and 2) were conducted in premenopausal women with uterine leiomyoma-associated heavy menstrual bleeding (80 mL or greater per cycle for two cycles or 160 mL or greater during one cycle).

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.

Clostridium difficile Infection-Daily Symptoms (CDI-DaySyms™) questionnaire: psychometric characteristics and responder thresholds.

Background: The purpose of the current study was to determine the final content validation, psychometric characteristics, clinically meaningful improvement, and responder thresholds of the Clostridium difficile infection (CDI)-Daily Symptoms (CDI-DaySyms™) patient-reported outcome (PRO) questionnaire.

Methods: This validation study was part of two phase III studies (NCT01987895 and NCT01983683) conducted in patients with mild-to-moderate or severe CDI who completed the CDI-DaySyms™ daily throughout the treatment period. The questionnaire was evaluated in three stages: final PRO item content validation (Stage I); psychometric evaluation of reliability and construct validity (Stage II); and determination of clinically meaningful improvement and responder thresholds using distribution-based methods (Stage III).

Development and Validation of the FSIQ-RMS: A New Patient-Reported Questionnaire to Assess Symptoms and Impacts of Fatigue in Relapsing Multiple Sclerosis.

Objectives: A new patient-reported outcome (PRO) instrument to measure fatigue symptoms and impacts in relapsing multiple sclerosis (RMS) was developed in a qualitative stage, followed by psychometric validation and migration from paper to an electronic format.

Methods: Adult patients with relapsing-remitting multiple sclerosis (RRMS) were interviewed to elicit fatigue-related symptoms and impacts. A draft questionnaire was debriefed in cognitive interviews with further RRMS patients, and revised.

Psychometric Validation of the Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT) Questionnaire: Results of the SYMPHONY Trial.

Background: Disease-specific patient-reported outcome (PRO) instruments are important in assessing the impact of disease and treatment. The Pulmonary Arterial Hypertension-Symptoms and Impact Questionnaire is the first instrument for quantifying pulmonary arterial hypertension (PAH) symptoms and impacts developed according to the 2009 US Food and Drug Administration PRO guidance; previous qualitative research in patients with PAH supported its initial content validity.

Methods: Content finalization and psychometric validation were conducted by using data from A Study of Macitentan in Pulmonary Arterial Hypertension to Validate the PAH-SYMPACT (SYMPHONY), a single-arm, 16-week trial with macitentan 10 mg in US patients with PAH.

The CDI-DaySyms: Content Development of a New Patient-Reported Outcome Questionnaire for Symptoms of Clostridium difficile Infection.

Objectives: To develop a patient-reported outcome (PRO) questionnaire for symptoms of Clostridium difficile infection (CDI) following the US Food and Drug Administration PRO guidelines.

Methods: Patients' experiences of CDI symptoms were elicited in open-ended discussions with patients and nurses at five US sites (stage 1). A draft PRO measure was developed after demonstration of concept saturation.

Macitentan Improves Health-Related Quality of Life for Patients With Pulmonary Arterial Hypertension: Results From the Randomized Controlled SERAPHIN Trial.

Background: Pulmonary arterial hypertension (PAH) leads to reduced health-related quality of life (HRQoL). The objectives of this analysis were to evaluate the effect of macitentan on HRQoL in patients with PAH in the Study with an Endothelin Receptor Antagonist in Pulmonary Arterial Hypertension to Improve Clinical Outcome (SERAPHIN) study. The association between baseline HRQoL and long-term outcomes was also investigated.

Development of the Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT®) questionnaire: a new patient-reported outcome instrument for PAH.

Background: Regulators and clinical experts increasingly recognize the importance of incorporating patient-reported outcomes (PROs) in clinical studies of therapies for pulmonary arterial hypertension (PAH). No PAH-specific instruments have been developed to date in accordance with the 2009 FDA guidance for the development of PROs as endpoints in clinical trials. A qualitative research study was conducted to develop a new instrument assessing PAH symptoms and their impacts following the FDA PRO guidance.

An observational cohort study of patients with newly diagnosed digital ulcer disease secondary to systemic sclerosis registered in the EUSTAR database.

Objectives: This study describes clinical characteristics, prognostic factors, and quality of life in patients with newly diagnosed (incident) digital ulcers (DU).

Methods: Observational cohort study of 189 consecutive SSc patients with incident DU diagnosis identified from the EUSTAR database (22 centres in 10 countries). Data were collected from medical charts and during one prospective visit between 01/2004 and 09/2010.

Characterizing pulmonary hypertension-related hospitalization costs among Medicare Advantage or commercially insured patients with pulmonary arterial hypertension: a retrospective database study.

Objectives: This study assessed pulmonary hypertension (PH)-related hospitalizations, including readmissions, among US patients with pulmonary arterial hypertension (PAH), a rare disease characterized by high morbidity and premature mortality.

Study Design: Analysis of claims data (January 1, 2007-April 30, 2011) from adult enrollees with commercial or Medicare Advantage with Part D coverage from a large US health plan.

Methods: Patients with PAH were identified based on ≥ 1 medical claim with a PH-related diagnostic code (International Classification of Diseases, Ninth Edition, Clinical Modification code 416.

Effect of macitentan on hospitalizations: results from the SERAPHIN trial.

Objectives: This study sought to evaluate the effect of macitentan on hospitalization of patients with symptomatic pulmonary arterial hypertension (PAH).

Background: PAH is a progressive, life-threatening disease often requiring hospitalization.

Methods: In the multicenter, double-blind, randomized, event-driven, phase III SERAPHIN (Study with an Endothelin Receptor Antagonist in Pulmonary arterial Hypertension to Improve cliNical outcome) trial, patients with symptomatic PAH were randomized (1:1:1) to receive placebo or 3 mg or 10 mg of macitentan.

Functional impairment of systemic scleroderma patients with digital ulcerations: results from the DUO Registry.

Objectives: Digital ulcers (DUs) are frequent manifestations of systemic scleroderma (SSc). This study assessed functional limitations due to DUs among patients enrolled in the Digital Ulcer Outcome (DUO) Registry, an international, multicentre, observational registry of SSc patients with DU disease.

Methods: Patients completed at enrolment a DU-specific functional assessment questionnaire with a 1-month recall period, measuring impairment in work and daily activities, and hours of help needed from others.

Burden of illness in idiopathic pulmonary fibrosis.

Background: Idiopathic pulmonary fibrosis is a life-threatening condition, and few data concerning the impact on healthcare utilization and associated costs are available. The objective of this study was to describe the burden of illness (comorbidity, healthcare resource utilization, and associated costs) in patients with idiopathic pulmonary fibrosis.

Methods: Two cohorts (patients with idiopathic pulmonary fibrosis and matched controls) were retrospectively identified from US claims databases between January 1, 2001 and September 30, 2008.

Quality of life and healthcare resource use associated with angiographic vasospasm after aneurysmal subarachnoid hemorrhage.

Background And Purpose: In this analysis of data from a large clinical trial in aneurysmal subarachnoid hemorrhage, the impact of angiographic vasospasm (aVSP) on specific patient outcomes and inpatient healthcare resource use was assessed.

Methods: This was a post hoc analysis of exploratory end points collected for 409 patients with aneurysmal subarachnoid hemorrhage in the Clazosentan to Overcome Neurological Ischemia and Infarction Occurring After Subarachnoid Hemorrhage (CONSCIOUS-1) trial. Central reviewers graded severity of aVSP as none, mild, moderate, or severe based on comparison of catheter angiograms obtained at baseline and 7 to 11 days after aneurysmal subarachnoid hemorrhage.