A conceptual model for advanced/metastatic gastric or gastroesophageal junction cancer: a review of qualitative studies and results from patient interviews.

Background: Despite approvals of new first-line immunotherapies for advanced/metastatic gastric cancer/gastroesophageal junction cancer (aGC/GEJC), patients' median survival is around 14 months and their health-related quality of life (HRQoL) is affected by disease-related symptoms and treatment-related side effects. Using a targeted literature review (TLR) and patient interviews, this study identified disease- and treatment-related concepts that are important to patients with aGC/GEJC and their HRQoL.

Methods: A TLR was conducted to identify primary qualitative studies from 2018 to 2021 on patients' experiences with aGC/GEJC.

A Consistent Lack of Consistency: Definitions, Evidentiary Expectations and Potential Use of Meaningful Change Data in Clinical Outcome Assessments Across Stakeholders. Results from a DIA Working Group Literature Review and Survey.

Background: Clinical outcome assessments (COAs) measure how patients feel or function and can be used to understand which patients experience benefits of treatment and which do not. Interpretation of COA data is influenced by how meaningful change is defined. We aimed to compare how different stakeholders define, assess, and use meaningful change for decisions that impact patients.

Relevance of patient-centered actigraphy measures in pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension: a qualitative interview study.

Background: Pulmonary arterial hypertension (PAH) and chronic thromboembolic pulmonary hypertension (CTEPH) are severe, progressive diseases characterized by key symptoms such as dyspnea and fatigue. These symptoms impair physical functioning, with patients struggling to perform their daily activities. One traditional measure of physical functioning and exercise capacity is the 6-minute walk test (6MWT).

Assessing usability of electronic patient-reported outcome measures in older people with and without a rare dermatologic disorder.

Background: Robust and well-defined data collection is important when using electronic patient-reported outcome measures (ePROMs) in clinical studies. Questions have been raised as to whether older age may be a barrier to data collection due to patients' unfamiliarity with electronic devices. Older adults may also have underlying health conditions that affect their ability to fill out patient-reported outcome measures (PROMs) on electronic devices.

Development of the celiac disease symptom diary version 2.1 (CDSD 2.1) patient-reported outcome measure.

Purpose: For patients with celiac disease (CeD), the only current management option is adherence to a strict gluten-free diet (GFD); however, many patients on a GFD continue to experience symptoms with a significant impact on quality of life. Potential new treatments for CeD are under development and a validated patient-reported outcome measure is required to evaluate their utility in clinical trials. The purpose of this article is to provide a history of the development of the Celiac Disease Symptom Diary (CDSD) 2.

Life after Lockdown: An Exploratory Qualitative Study of Behaviors and Impacts of Avoiding COVID-19 in Individuals at High Risk of Severe COVID-19 and Their Caregivers.

This exploratory qualitative study involved semi-structured interviews with adults and caregivers of adults at high risk of severe COVID-19, addressing current COVID-19 avoidance and protective behaviors and how these behaviors impacted their lives. Results were interpreted in a separate think tank session. Insights were developed into a conceptual model of COVID-19 avoidance and protective behaviors and the associated impacts on health-related quality of life and overall functioning.

Three Novel Patient-Reported Outcome Measures to Assess the Patient Experience With Daily and Weekly HIV Oral Antiretroviral Therapy.

Background: Patient-reported outcome measures (PROMs) can provide data on the barriers and facilitators of adherence to daily oral antiretroviral therapy (OART) regimens. We aimed to develop PROMs to understand the perspectives of people with HIV (PWH) on (1) facilitators/barriers to daily OART regimen adherence and (2) a hypothetical switch to a long-acting (LA)-OART regimen.

Methods: Following the US food and drug administration patient-reported outcome guidance, targeted literature reviews and concept elicitation interviews with clinicians (n = 7) and PWH (n = 28) were conducted to develop conceptual models (CMs) of facilitators/barriers to OART regimen adherence.

Development and Content Validation of the Patient's Qualitative Assessment of Treatment - Real-World (PQAT-RW): An Instrument to Evaluate Benefits and Disadvantages of Treatments in Real-World Settings.

Purpose: Quantifying patient-perceived benefits and disadvantages of treatments in a real-world setting is increasingly important in healthcare decision-making. The Patient's Qualitative Assessment of Treatment (PQAT) assesses patient-perceived benefits and disadvantages of treatment, and associated trade-offs potentially influencing patients' willingness to continue treatment. It has then been modified to capture patients' perceived magnitude of benefits and disadvantages of treatment quantitatively, as well as qualitatively (PQATv2).

The Association Between Physical Distancing Behaviors to Avoid COVID-19 and Health-Related Quality of Life in Immunocompromised and Nonimmunocompromised Individuals: Patient-Informed Protocol for the Observational, Cross-Sectional EAGLE Study.

Background: Immunocompromised individuals are known to respond inadequately to SARS-CoV-2 vaccines, placing them at high risk of severe or fatal COVID-19. Thus, immunocompromised individuals and their caregivers may still practice varying degrees of social or physical distancing to avoid COVID-19. However, the association between physical distancing to avoid COVID-19 and quality of life has not been comprehensively evaluated in any study.

The Role of Patient-Reported Outcomes to Measure Treatment Satisfaction in Drug Development.

Treatment satisfaction is a person's rating of his or her treatment experience, including processes and outcomes. It is directly related to treatment adherence, which may be predictive of treatment effectiveness in clinical and real-world research. Consequently, patient-reported outcome (PRO) instruments have been developed to incorporate patient experience throughout various stages of drug development and routine care.

Disability Associated with Chronic Graft-Versus-Host Disease After Allogeneic Hematopoietic Stem Cell Transplantation: Analysis of a Cross-Sectional US Patient Survey.

Introduction: Chronic graft-versus-host disease (GVHD) following hematopoietic stem cell transplantation (HSCT) is associated with poor health-related quality of life (HRQoL) and functional status. However, few studies have evaluated chronic GVHD-related disability and specific activity limitations from a patient perspective. The objective of this analysis was to assess physical, cognitive, and work disability, and describe factors predictive of disability in patients with chronic GVHD in the potentially employable general workforce.

Patient-Reported Outcomes From the Phase III HIMALAYA Study of Tremelimumab Plus Durvalumab in Unresectable Hepatocellular Carcinoma.

Purpose: In the phase III HIMALAYA study (ClinicalTrials.gov identifier: NCT03298451) in unresectable hepatocellular carcinoma (uHCC), the Single Tremelimumab Regular Interval Durvalumab (STRIDE) regimen significantly improved overall survival versus sorafenib, and durvalumab monotherapy was noninferior to sorafenib. Patient-reported outcomes (PROs), a secondary outcome from HIMALAYA, are reported here.

The health care experience of adults with metabolic dysfunction-associated steatohepatitis and influence of PNPLA3: A qualitative study.

Background: Metabolic dysfunction-associated steatohepatitis (MASH) is a progressive form of metabolic dysfunction-associated steatotic liver disease, for which there is limited information about patient experience, including the patient journey.

Methods: In this study, we conducted interviews with patients with MASH to qualitatively evaluate the patient journey and help elucidate the experiences of this patient population. We also investigated if the patatin-like phospholipase domain-containing protein 3 (PNPLA3) I148M variant (non-Hispanic) or being of Hispanic ethnicity may influence patient experiences because these 2 subgroups develop advanced liver disease more frequently than other patient groups.

Health-related quality of life measures provide information on the contributors, components, and consequences of frailty in HIV: a systematic mapping review.

Purpose: Frailty in HIV is extensively explored in epidemiological and clinical studies; it is infrequently assessed as an outcome in routine care. The focus on health-related quality of life (HRQL) measures in HIV presents a unique opportunity to understand frailty at a larger scale. The objective was to identify the extent to which generic and HIV-related HRQL measures capture information relevant to frailty.

The impact of different data handling strategies in exploratory and confirmatory factor analysis of diary measures: an evaluation using simulated and real-world asthma nighttime symptoms diary data.

Background: Daily diaries are an important modality for patient-reported outcome assessment. They typically comprise multiple questions, so understanding their underlying structure is key to appropriate analysis and interpretation. Structural evaluation of such measures poses challenges due to the high volume of repeated measurements.

Synchronous and asynchronous telerehabilitation methods produce similar benefits in individuals with non-specific neck pain.

Introduction: Evidence exists on clinical benefits of synchronous and asynchronous telerehabilitation for patients with non-specific neck pain (NSNP); however, limited studies are comparing synchronous and asynchronous telerehabilitation (TR) programs in this population. The aim of this study was to estimate the relative effectiveness of an 8-week synchronous or asynchronous TR in improving pain, functional disability, kinesiophobia, and mobility in patients with NSNP.

Materials And Methods: This was a randomized, controlled clinical trial carried out on 60 individuals with NSNP.

Story completion (SC) - an epistemological approach to the interpretation of patient-reported outcomes?

Introduction: Patient-reported outcome measures (PROMs) quantitative data are increasingly accompanied by qualitative narrative data, which does not always provide the insights required to inform healthcare.

Areas Covered: This perspective considers how story completion (SC) may be used to gain a deeper insight on people's experiences and guide interpretation of the meaning of PROM responses.

Expert Opinion: Story completion (SC) is an epistemological approach involving participants telling a story in response to a pre-determined 'stem' they have been presented with.

Perspectives of patients with advanced or metastatic non-small cell lung cancer on symptoms, impacts on daily activities, and thresholds for meaningful change: a qualitative research study.

Introduction: Advanced or metastatic non-small cell lung cancer (NSCLC) is associated with significant symptom burden. It is important to understand the impact of these disease-and treatment-related symptoms on patients' daily lives and explore from a patient perspective what constitutes a meaningful change in NSCLC symptoms.

Methods: Patient experience of advanced or metastatic NSCLC was explored in this prospective, non-interventional qualitative research study recruiting patients from the United States (US).

A powerful partnership: researchers and patients working together to develop a patient-facing summary of clinical trial outcome data.

Objective: Availability of easy-to-understand patient-reported outcome (PRO) trial data may help individuals make more informed healthcare decisions. Easily interpretable, patient-centric PRO data summaries and visualizations are therefore needed. This three-stage study explored graphical format preferences, understanding, and interpretability of clinical trial PRO data presented to people with prostate cancer (PC).