Self-Reported Adherence to Cancer Therapy: Development and Validation of the Domains of Subjective Extent of Nonadherence-Cancer Measure.

Purpose: Patients with cancer take different types of medications with varying schedules and settings. They are also sometimes instructed to stop medications due to toxicity. To measure self-reported nonadherence in this heterogeneous population, we modified and evaluated a measure originally developed to assess nonadherence to daily oral antihypertensives, the Domains of Subjective Extent of Nonadherence (DOSE-Nonadherence).

A Systematic Review of Health-Related Quality-of-Life Measurement in Patients With Complicated Intraabdominal Infections.

Background: In this systematic review, we assessed health-related quality-of-life (HRQoL) measures in patients with complicated intraabdominal infections (cIAIs) commonly associated with sepsis and evaluated the impact of cIAIs on HRQoL.

Methods: Following a prospectively registered protocol in PROSPERO (CRD42021255827), we searched electronic databases, including MEDLINE (PubMed), EMBASE (Elsevier), and CINAHL (EBSCO), from database inception through September 2022 to identify studies that measured HRQoL in patients with cIAIs.

Results: We identified 93 articles that included 45 HRQoL measures: 32 generic, 5 condition-specific, and 8 symptom-specific.

Meaningful Score Differences and Meaningful Score Regions of the Patient-Reported Outcomes Measurement Information System® Pediatric Asthma Impact Scale.

Objectives: To estimate meaningful score differences (MSDs) and meaningful score regions (MSRs) for the Patient-Reported Outcomes Measurement Information System® (PROMIS®) Pediatric Asthma Impact Scale to enhance score interpretability.

Methods: Secondary analysis included 106 children with asthma (8-17 years of age) who completed weekly surveys for 4 weeks. Repeated measures correlations examined the magnitude of association of the PROMIS Pediatric Asthma Impact Scale with other asthma measures.

Family caregiver observations of pain behaviors in infants and young children less than 3 years of age.

Pain in very young, preverbal children is often inadequately assessed and thus poorly treated, leading to short- and long-term negative consequences. Family caregivers are often the primary observers of young children's pain, but their perspectives are often overlooked. There are no well-designed or validated caregiver-reported pain measures for children under 3 years of age.

Prevalence of symptomatic toxicities for novel therapies in adult oncology trials: a scoping review.

Background: Patients' self-report of their symptoms can provide important data for the evaluation of treatment benefit and tolerability of oncology drugs. Contemporary treatment approaches, including immunotherapy and molecular targeted therapies, have unique toxicities based on their novel mechanisms of action. This scoping review aimed to summarize evidence from existing reviews and clinical practice guidelines to examine the type and prevalence of toxicities including symptomatic adverse events (sympAEs) for adult cancer patients to inform clinical care and therapeutic trials.

Attitudes toward patient engagement in clinical pain research: Insights from individuals with chronic pain in the United States.

The value of engaging people with lived experience into chronic pain research is becoming increasingly recognized, yet the perspectives of individuals with chronic pain who have not previously participated in research are underexplored. This study aims to fill this gap by assessing the attitudes, preferences, and barriers related to patient engagement among adults living with chronic pain in the United States (US). An online survey was developed in collaboration with an advisory board and community engagement studio and distributed through Qualtrics panels from December 2023-January 2024.

Financial Toxicity in Spina Bifida: Development of a Patient and Caregiver Questionnaire.

Objective: To develop and validate the first patient- and caregiver-reported measure of spina bifida-specific "financial toxicity" (SBFT), that is, the negative side effects of healthcare-related expenditures. Assessing financial toxicity is a growing area of research in both oncology and benign urology.

Methods: We developed a 20-item questionnaire and quantitatively validated it in a population of SB patients and caregivers.

Mapping immune checkpoint inhibitor side effects to item libraries for use in real-time side effect monitoring systems.

Background: Monitoring for the side effects of novel therapies using patient-reported outcomes (PROs) is critical for ensuring patient safety. Existing static patient-reported outcome measures may not provide adequate coverage of novel side effects. Item libraries provide a flexible approach to monitoring for side effects using customized item lists, but the ideal process for matching side effects to items sourced from multiple item libraries is yet to be established.

Symptom monitoring with electronic patient-reported outcomes during cancer treatment: final results of the PRO-TECT cluster-randomized trial.

Symptoms are often underdetected during cancer treatment. To determine if symptom monitoring with electronic patient-reported outcomes (PROs) improves clinical outcomes, we conducted a cluster-randomized trial in which 52 oncology practices were assigned to PRO or usual care. At PRO practices, patients with metastatic cancer were invited to complete weekly symptom surveys.

Development of CARRA/PReS-endorsed consensus Core and Expanded Datasets in childhood-onset systemic lupus erythematosus for international registry-based research.

Objectives: Childhood-onset systemic lupus erythematosus (cSLE), representing 15%-20% of individuals with SLE, has been difficult to study globally due to differences between registries. This initiative, supported by Childhood Arthritis Rheumatology Research Alliance (CARRA) and Paediatric Rheumatology European Society (PReS), aims to create Core and Expanded cSLE Datasets to standardise and enhance research worldwide.

Methods: 21 international cSLE experts and 4 patients participated in a Delphi process (questionnaires, 2 topic-specific focus groups and 3 virtual consensus meetings) to create 2 standardised cSLE datasets.

Comparison of patient-reported symptoms with multi-item patient-reported outcome measures of fatigue, anxiety, and depression in the clinical care of women undergoing chemotherapy for early breast cancer.

Background: As patient-reported symptoms are increasingly incorporated into routine clinical practice and captured in electronic medical records these data can be used to conduct health-related quality of life research studies. This study compares symptom reports from the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) and its precursor patient reported symptom monitoring (PRSM) (hereafter PRSM/PRO-CTCAE) with multi-item patient-reported outcome (PRO) scales for fatigue (Functional Assessment of Chronic Illness Therapy/FACIT-Fatigue) and depression and anxiety (Mental Health Index/MHI).

Methods: This is a secondary analysis of data collected from women with early breast cancer (Stage I-III) scheduled for chemotherapy who completed PRSM/PRO-CTCAE, FACIT-Fatigue, and MHI scales pre- and post-chemotherapy.

Assessing patient-reported outcomes in primary sclerosing cholangitis: an update.

Purpose Of Review: Patient-reported outcome (PRO) measures validated in primary sclerosing cholangitis (PSC) are needed for clinical trials. This review describes the recent US Food & Drug Administration (FDA) Patient-Focused Drug Development (PFDD) guidelines, existing PRO measures used in PSC studies, and the design of PSC-specific symptom measures adherent with the guidelines.

Recent Findings: FDA released updated guidance reflecting best practices for the design and evaluation of clinical outcome assessments (including PROs) and the design of trial endpoints.

Recalling what we thought we knew about recall periods: a qualitative descriptive study of how adults diagnosed with cancer use recall periods for patient-reported outcome items about physical function.

Objectives: In cancer studies, assessment of patients' physical function can provide insight into cancer-related symptoms and the side effects of treatment. Physical function can be assessed using patient-reported outcome measures (PROMs), which may or may not include a recall period-the amount of time the questionnaire asks the respondent to think back to answer the questions. More understanding is needed about how patients interpret and respond to items posed with different recall periods, and which recall period they actually use.

Health-related quality of life profiles of adults with arthritis and/or fibromyalgia: a cross-sectional study.

Purpose: Adults with arthritis experience poor health-related quality of life (HRQOL), though research often focuses on single HRQOL outcomes or summary scores. We aimed to identify HRQOL profiles in adults with different arthritis types and determine risk and protective factors.

Methods: Data including PROMIS-29 Profile v2.

Designing a Measure of Body Image: Cognitive Interview Findings from an Adolescent and Young Adult Cancer Sample.

A cancer diagnosis in adolescence and young adulthood significantly impacts a person's quality of life, particularly concerning identity, self-esteem, and subsequently, body image. This study aims to develop a psychometrically-sound patient-reported outcome measure of body image for adolescent and young adult (AYA) oncology patients that was guided by the National Institutes of Health's Patient-Reported Outcomes Measurement Information System® (PROMIS) Scientific Standards and our past concept elicitation interviews with AYAs. We conducted a multi-step approach involving item identification, refinement, generation; translatability and reading level review; and cognitive interviews.

Development and evaluation of a questionnaire to capture environmental and occupational inhalational exposures in adults with fibrotic interstitial lung disease.

Background: Identification of exposures in patients with interstitial lung diseases (ILDs) is essential for diagnosis and management and can be facilitated through the use of exposure questionnaires. However, for most ILDs, a patient-focused questionnaire is lacking. Cognitive interviewing is a methodology used to evaluate sources of understanding and misunderstanding in a questionnaire and to provide evidence of content validity.

Pain Interference in Juvenile Idiopathic Arthritis.

Objective: Despite treatment advances, pain remains a serious problem for many children with juvenile idiopathic arthritis (JIA). To better understand pain in children with JIA and identify potentially modifiable factors, this study evaluated Patient-Reported Outcomes Measurement Information System (PROMIS) Pediatric Pain Interference (PI) and its relationships with other pain measures and demographic, clinical, psychosocial, and functional variables.

Methods: This cross-sectional, observational, multicenter study used descriptive statistics and a mix of bivariate and multivariable analyses to describe PI and characterize relationships with other measures and variables.

Designing Patient-Reported Measures of Fertility: Cognitive Interview Findings from Adolescents and Young Adults with Cancer.

Fertility concerns (FC) are central to the well-being of many adolescent and young adult (AYA) cancer survivors. Clinical conversations about FC and fertility preservation are suboptimal, increasing patient distress. The goal of this project was to establish content validity and comprehensibility of self-report questions on FCs for AYAs with cancer.

The FACT-GP5 as a global tolerability measure: responsiveness and robustness to missing assessments.

Purpose: The Functional Assessment of Cancer Therapy item (FACT-GP5) has the potential to provide an understanding of global treatment tolerability from the patient perspective. Longitudinal evaluations of the FACT-GP5 and challenges posed by data missing-not-at-random (MNAR) have not been explored. Robustness of the FACT-GP5 to missing data assumptions and the responsiveness of the FACT-GP5 to key side-effects are evaluated.

Asking the "Right" Questions about Financial Hardship: Using Cognitive Interviews with Adolescents and Young Adults with Cancer and Their Caregivers to Inform Measure Development.

Financial hardship as a result of cancer treatment can have a significant and lasting negative impact on adolescents and young adults (AYAs) and their families. To address a lack of developmentally informed and psychometrically sound measures of financial hardship for AYAs and their caregivers, we used rigorous measurement development methods recommended by the National Institutes of Health's Patient-Reported Outcomes Measurement Information System® (PROMIS®) to determine comprehensibility and relevance of measure content. Our multi-step approach involved item identification, refinement, and generation; translatability and reading level review; and cognitive interviews.

Use of patient-reported global assessment measures in clinical trials of chronic pain treatments: ACTTION systematic review and considerations.

Establishing clinically meaningful changes in pain experiences remains important for clinical trials of chronic pain treatments. Regulatory guidance and pain measurement initiatives have recommended including patient-reported global assessment measures (eg, Patient-Global Impression of Change [PGIC]) to aid interpretation of within-patient differences in domain-specific clinical trial outcomes (eg, pain intensity). The objectives of this systematic review were to determine the frequency of global assessment measures inclusion, types of measures, domains assessed, number and types of response options, and how measures were analyzed.

Understanding reliability of the observer-reported communication ability measure within Angelman syndrome through the lens of generalizability theory.

Aims: Caregivers rate improved communication ability as one of the most desired outcomes for successful interventions for individuals with Angelman syndrome (AS). When measuring communication ability in clinical trials, the reliability of such measures is critical for detecting significant changes over time. This study examined the reliability of the Observed-Reported Communication Ability (ORCA) measure completed by caregivers of individuals with AS.

Emotional and functional well-being in long-term breast cancer survivorship.

Purpose: Emotional and functional well-being (EWB and FWB) are important components of mental health and quality of life. This study aims to evaluate long-term EWB and FWB in breast cancer (BC) survivors.

Methods: The Carolina Breast Cancer Study Phase 3 oversampled Black and younger (< 50 years in age) women so that they each represent approximately 50% of the study population and assessed participants' EWB and FWB with the Functional Assessment of Cancer Therapy-Breast (FACT-B) at 5- (baseline), 25-, and 84-months post diagnosis.