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.

Standardized Outcomes for Randomized Controlled Trials Targeting Early Interventions in Patients With Moderate-to-Severe Traumatic Brain Injury: Protocol for the Development of a Core Outcome Set.

Background: : With more than 60 million new cases around the world each year, traumatic brain injury (TBI) causes substantial mortality and morbidity. Managing TBI is a major human, social, and economic concern. In the last 20 years, there has been an increase in clinical trials in neurocritical care, leading mostly to negative results.

Impact of Country Income Level on Outcomes in Patients with Acute Brain Injury Requiring Invasive Mechanical Ventilation: A Secondary Analysis of the ENIO Study.

Background: Invasive mechanical ventilation can present complex challenges for patients with acute brain injury (ABI) in middle-income countries (MICs). We characterized the impact of country income level on weaning strategies and outcomes in patients with ABI.

Methods: A secondary analysis was performed on a registry of critically ill patients with ABI admitted to 73 intensive care units (ICUs) in 18 countries from 2018 to 2020.

International Expert Consensus on Relevant Health and Functioning Concepts to Assess in Users of Tobacco and Nicotine Products: Delphi Study.

Background: A Delphi study was conducted to reach a consensus among international clinical and health care experts on the most important health and functioning self-reported concepts when evaluating a switch from smoking cigarettes to using smoke-free tobacco and/or nicotine products (sf-TNPs).

Objective: The aim of this research was to identify concepts considered important to measure when assessing the health and functioning status of users of tobacco and/or nicotine products.

Methods: Experts (n=105), including health care professionals, researchers, and policy makers, from 26 countries with professional experience and knowledge of sf-TNPs completed a 3-round, adapted Delphi panel.

Chronic rhinosinusitis with nasal polyps (CRSwNP) symptom verbal response scales: content validity testing for use in adults with CRSwNP.

Background: PRO measures of symptoms in clinical trials have historically utilized visual and numerical scales but verbal descriptors may make it easier for patients to clearly differentiate between response options. This study assessed content validity and meaningful change in five verbal response scales (VRSs) used to assess chronic rhinosinusitis with nasal polyp (CRSwNP) symptom severity.

Methodology: This qualitative, semi-structured interview study recruited adults from the US, Germany, and China with confirmed moderate-to-severe CRSwNP.

Psychometric evaluation of the PROMIS parent proxy mobility item bank for use in Duchenne muscular dystrophy.

Aim: To evaluate the psychometric properties and measurement quality of the Patient-Reported Outcomes Measurement Information System Parent Proxy (PROMIS PP) Mobility item bank (v1.0, 23 items) for children with Duchenne muscular dystrophy (DMD), through Rasch statistical analysis.

Method: De-identified PROMIS PP Mobility items were completed by the caregivers of male patients with DMD, aged 4 to 12 years, as part of standard clinical care at the Nationwide Children's Hospital clinic; data were mined retrospectively from electronic health records.

Time to deterioration of patient-reported outcome endpoints in cancer clinical trials: targeted literature review and best practice recommendations.

Background: Time to deterioration (TTD) endpoints are often utilized in the analysis of patient-reported outcome (PRO) data in oncology clinical trials but different endpoint definitions and analysis frameworks exist that can impact result interpretation. This review examined the analysis, reporting and heterogeneity of TTD endpoints in the literature, the impact of analysis methods on results, and provides recommendations for future trials.

Methods: A targeted literature review of articles published between 2017 and 2022 was performed to collate TTD endpoints reported in oncology randomized controlled trials (RCTs).

Gender representation in cardiothoracic surgical academia: a call to support women across the globe.

Underrepresentation of women in surgical specialties persists, especially in academic leadership roles. Efforts to better understand disparities and support women cardiothoracic surgeons are ongoing.

Accelerating evidence generation: Addressing critical challenges and charting a path forward.

Efficient evidence generation to assess the clinical and economic impact of medical therapies is critical amid rising healthcare costs and aging populations. However, drug development and clinical trials remain far too expensive and inefficient for all stakeholders. On October 25-26, 2023, the Duke Clinical Research Institute brought together leaders from academia, industry, government agencies, patient advocacy, and nonprofit organizations to explore how different entities and influencers in drug development and healthcare can realign incentive structures to efficiently accelerate evidence generation that addresses the highest public health needs.

Response shift results of quantitative research using patient-reported outcome measures: a meta-regression analysis.

Purpose: Our objectives were to identify characteristics of response shift studies using patient-reported outcomes (PROMs) that explain variability in (1) the detection and (2) the magnitude of response shift effects.

Methods: We conducted a systematic review of quantitative studies published before June 2023. First, two-level multivariable logistic regression models (effect- and sample-levels) were used to explain variability in the probability of finding a response shift effect.

Which Chemotherapy-Related Terms were Difficult for Cancer Patients, and Who Would Have the Most Difficulties?

Purpose: This cross-sectional study aimed to examine which chemotherapy (CTx) terms were most difficult to understand for cancer patients and identify vulnerable patient populations who might need extra support to understand the terms.

Materials And Methods: We listed 56 CTx-related terms based on the experts' review, then 300 cancer patients and their caregivers completed a questionnaire that assessed literacy in CTx terms (LCT), functional health literacy, and empowerment. Descriptive analysis was performed to examine which CTx-related terms were most difficult for them.

Non-medical nitrous oxide misuse: From identifying a signal to unprecedented addictovigilance network communication.

Addictovigilance is a French health monitoring system, composed of 13 centres distributed across the national territory, that aims to monitor cases of abuse, misuse, or use disorders related to psychoactive substances. Fulfilling these missions requires a regional network that ensures efficient information flow, dissemination to health authorities, and subsequent downward communication to prevent risks. This was illustrated through the example of nitrous oxide misuse.

The factor structure of the Patient Health Questionnaire-9 in stroke: A comparison with a non-stroke population.

Background: It is unclear if certain post-stroke somatic symptoms load onto items of the Patient Health Questionnaire-9 (PHQ-9), a self-report depression questionnaire. We investigated these concerns in a stroke sample using factor analysis, benchmarked against a non-stroke comparison group.

Methods: The secondary dataset constituted 787 stroke and 12,016 non-stroke participants.

Views of Aging and Subjective Cognition in Middle-Aged and Older Adults: A Systematic Review.

Introduction: Views of aging have been linked with many important outcomes in older adults. Subjective cognition, or one's perception of their cognitive functioning, may be a valuable indicator of cognitive changes as individuals age, but is known to be impacted by a variety of factors. The aim of this systematic review was to synthesize the evidence on relationships between views of aging and subjective cognition, including whether and how these relationships may differ based on age.

Equimolar oxygen-nitrous oxide (EMONO) in dental pediatric care: which patients achieve therapeutic effects? A French national exploratory prospective observational multicenter study.

Purpose: Equimolar oxygen-nitrous oxide mixture (EMONO) is considered as a reference for dental care in children. Completion of dental treatment has usually been used as a criterion to assess EMONO treatment. However, other patient-reported outcomes are needed.

Patient-reported outcomes in early phase trials for patients with myelodysplastic syndromes.

Patients with myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) experience a wide range of symptoms due both to their underlying disease and the effects of treatment. Designing early phase trials to explore effective therapies in these patients should not only examine anti-tumor activity, but also consider the effects of treatments on how patients feel and function. Assessing symptomatic toxicities associated with new therapies in early phase trials from the patient perspective is best measured using patient-reported outcomes (PROs) and offers valuable insight and complementary information to the traditional adverse event reporting in cancer clinical trials.

Early palliative care and overall survival in patients with metastatic upper gastrointestinal cancers (EPIC): a multicentre, open-label, randomised controlled phase 3 trial.

Background: Early palliative care (EPC) leads to an improvement in quality of life and an unexpected survival benefit compared with oncological care for patients with metastatic lung cancer. The Early Palliative Integrated Care (EPIC) is aimed at examining whether EPC can improve overall survival in patients with metastatic upper gastrointestinal cancer.

Methods: We performed a multicentre, open-label, randomised phase-3 trial.