Qualitative Exploration of the Visual Function Impairments and Health-Related Quality of Life Impacts of Amblyopia in Adult and Pediatric Populations.

INTRODUCTION: Amblyopia is a reduction in vision in one or both eyes due to impaired development of the visual pathway. This study explored the experience of amblyopia and treatment from the patient, caregiver, and clinician perspectives. METHODS: A targeted literature review, including a review of social media listening (SML) studies, was conducted. Next, qualitative interviews were conducted with amblyopia patients, caregivers of children with amblyopia, and ophthalmologists with experience treating patients with amblyopia. The findings informed the development of a disease model. Amblyopia clinical experts provided input at key stages. RESULTS: Twelve data sources were reviewed, including qualitative studies in the literature and SML studies. Overall, 133 patients/caregivers were interviewed (23 adults, 16 adolescents, 47 child-caregiver dyads), plus 10 ophthalmologists from the United States, France, and Germany. Reduced visual acuity, impaired depth perception, impaired peripheral vision, and double vision were the most frequently reported symptoms. Amblyopia impacted daily activities (reading, using digital devices), the ability to move around, school/work (productivity, seeing the board in class), emotional well-being (frustration, sadness), and social functioning (difficulty socializing). Treatments, including patching and corrective lens, also impacted daily activities (using digital devices, sports/leisure), mobility (bumping into things), and work/school (tasks taking longer) as well as emotional well-being (embarrassment), and social functioning (bullying/stigma). CONCLUSION: The findings contribute valuable insights into the adult and pediatric experience of amblyopia from a multi-stakeholder perspective. The findings were used to critically assess existing clinical outcome assessments and supported the development of patient- and observer-reported outcome measures for use in amblyopia clinical trials.

Cardiac complications of COVID-19 infection.

Since the start of the COVID-19 pandemic, studies emerged reporting the occurrence of cardiovascular complications in patients affected by SARS-CoV-2. Initial data were likely skewed by higher risk populations and those with severe disease. Recent, larger studies have corroborated this association and provide estimates for risk of cardiovascular complications. Patients affected by COVID-19 are at increased risk of myocardial infarction, myocarditis, venous thromboembolism, arrhythmias, and exacerbation of heart failure. Furthermore, a subset of patients who recover from the acute illness have persistent symptoms, a condition termed "long COVID", and management of these symptoms is challenging. Clinicians treating patients affected by COVID-19 should remain vigilant for cardiac complications during the acute illness, particularly in high-risk populations.

The Amblyopia Quality of Life (AmbQoL): Development and Content Validation of a Novel Health-Related Quality of Life Instrument for Use in Adult and Pediatric Amblyopia Populations.

INTRODUCTION: Amblyopia is a neurodevelopmental vision disorder, characterized by poor vision in one or both eyes. Given the lack of existing clinical outcome assessments (COA) considered fit-for-purpose for amblyopia clinical trials, this study developed new COAs to assess amblyopia symptoms and health-related quality of life (HRQoL) impacts in adult and pediatric amblyopia populations that conform with best practice standards and regulatory guidelines. METHODS: Findings from a targeted qualitative literature review informed the development of three versions of the new Amblyopia Quality of Life Questionnaire (AmbQoL): a patient-reported outcome measure (PRO) for individuals aged 13 years and older, a PRO for children aged 9-12 years, and an observer-reported outcome measure (ObsRO) for caregivers of children aged 4-8 years. Qualitative interviews were conducted with the target populations, and with ophthalmologists experienced in treating amblyopia patients to evaluate the content validity for further development of the AmbQoL. A translatability assessment was conducted to ensure cultural appropriateness and usability across multiple languages. Feedback from Food and Drug Administration (FDA) was also sought on the instruments, and clinical experts provided input at key stages. RESULTS: Interviews were conducted with 112 patients/caregivers and ten ophthalmologists from the USA, France, and Germany. The instructions, items, and response options were well understood across all AmbQoL versions. Feedback from the patients, caregivers, ophthalmologists, the translatability assessment, the FDA, and the expert clinicians informed minor wording modifications to enhance clarity and translatability. Some items were removed due to low relevance. The study resulted in a 23-item adult/adolescent PRO, 24-item child PRO, and 12-item ObsRO, each employing a 7-day recall period. CONCLUSION: Each AmbQoL version has documented support for its face and content validity for use in amblyopia populations aged ≥ 4 years. Further research is necessary to evaluate the psychometric measurement properties of the AmbQoL instruments to enable their use in amblyopia treatment trials.

The endometriosis daily diary: qualitative research to explore the patient experience of endometriosis and inform the development of a patient-reported outcome (PRO) for endometriosis-related pain.

PURPOSE: Endometriosis is a chronic disorder of the female reproductive system characterized by debilitating symptoms, particularly endometriosis-related pain (ERP). Patient-reported outcome (PRO) measures of symptoms and impacts are required to assess disease severity in ERP clinical studies and clinical practice. A content-valid instrument was developed by modifying the Dysmenorrhea Daily Diary (DysDD) to form the Endometriosis Daily Diary (EDD), an electronic PRO administered via handheld device. METHODS: Qualitative research with US females with ERP was conducted in three stages: (1) Development of an endometriosis conceptual model based on qualitative literature and conduct of concept elicitation (CE) interviews (N = 30). (2) Cognitive debriefing (CD) interviews (N = 30) conducted across two rounds to assess relevance and understanding of the EDD, with modifications between interview rounds. (3) Pilot testing to assess usability/feasibility of administrating the EDD daily on an electronic handheld device (N = 15). Clinical experts provided guidance throughout the study. RESULTS: The conceptual model provided a comprehensive summary of endometriosis to inform modifications to the DysDD, forming the EDD. CD results demonstrated that EDD items were relevant for most participants. Instructions, items, response scales, and recall period were well-understood. The resulting daily diary assesses severity of cyclic and non-cyclic pelvic pain, dyspareunia, impact of ERP on functioning and daily life, symptoms associated with ERP, and bowel symptoms. Participants were able to complete the diary daily and found the device easy to use. CONCLUSION: The EDD demonstrated good content validity in females experiencing ERP. The next step is to perform psychometric validation in an ERP sample.

Understanding the relationship between the 32-item motor function measure and daily activities from an individual with spinal muscular atrophy and their caregivers' perspective: a two-part study.

BACKGROUND: The 32-item Motor Function Measure (MFM32) is a clinician-reported outcome measure used to assess the functional abilities of individuals with neuromuscular diseases, including those with spinal muscular atrophy (SMA). This two-part study explored the relationship between the functional abilities assessed in the MFM32 and activities of daily living (ADLs) from the perspective of individuals with Type 2 and Type 3 (non-ambulant and ambulant) SMA and their caregivers through qualitative interviews and a quantitative online survey. METHODS: In-depth, semi-structured, qualitative interviews were conducted with individuals with SMA and caregivers from the US. Subsequently, a quantitative online survey was completed by individuals with SMA or their caregivers from France, Germany, Italy, Poland, Spain, Canada, the United States (US) and the UK. In both parts of the study, participants were asked to describe the ADLs considered to be related to the functional abilities assessed in the MFM32. Results from the qualitative interviews informed the content of the quantitative online survey. RESULTS: Qualitative interviews were conducted with 15 adult participants, and 217 participants completed the quantitative online survey. From the qualitative interviews, all of the functional abilities assessed in the patient-friendly MFM32 were deemed as related to one or more ADL. The specific ADLs that participants considered related to the patient-friendly MFM32 items could be grouped into 10 key ADL domains: dressing, mobility/transferring, self-care, self-feeding, reaching, picking up and holding objects, physical activity, writing and technology use, social contact/engagement, toileting and performing work/school activities. These results were confirmed by the quantitative online survey whereby the ADLs reported to be related to each patient-friendly MFM32 item were consistent and could be grouped into the same 10 ADL domains. CONCLUSION: This study provides in-depth evidence from the patient/caregiver perspective supporting the relevance of the patient-friendly MFM32 items to the ADLs of individuals with Type 2 and Type 3 SMA.

A Patient-Centered Evaluation of Meaningful Change on the 32-Item Motor Function Measure in Spinal Muscular Atrophy Using Qualitative and Quantitative Data.

The 32-item Motor Function Measure (MFM32) is an assessment of motor function used to evaluate fine and gross motor ability in patients with neuromuscular disorders, including spinal muscular atrophy (SMA). Reliability and validity of the MFM32 have been documented in individuals with SMA. Through semi-structured qualitative interviews (N = 40) and an online survey in eight countries (N = 217) with individuals with Types 2 and 3 SMA aged 2-59 years old and caregivers, the meaning of changes on a patient-friendly version of the MFM32 was explored. In an independent analysis of clinical trial data, anchor- and distribution-based analyses were conducted in a sample of individuals with Type 2 and non-ambulant Type 3 SMA to estimate patient-centered quantitative MFM32 meaningful change thresholds. The results from this study demonstrate that, based on patient and caregiver insights, maintaining functional ability as assessed by a patient-friendly version of the MFM32 is an important outcome. Quantitative analyses using multiple anchors (median age range of 5-8 years old across anchor groups) indicated that an ~3-point improvement in MFM32 total score represents meaningful change at the individual patient level. Overall, the qualitative and quantitative findings from this study support the importance of examining a range of meaningful change thresholds on the MFM32 including ≥0 points change reflecting stabilization or improvement and ≥3 points change reflecting a higher threshold of improvement. Future research is needed to explore quantitative differences in meaningful change on the MFM32 based on age and functional subgroups.

Content validity of patient-reported outcomes for use in lower-risk myelodysplastic syndromes.

BACKGROUND: The lower-risk (low and intermediate-1 risk based on IPSS) myelodysplastic syndrome (MDS) has a negative impact on patients' health-related quality of life (HRQoL). Patient Reported Outcomes (PROs) instruments, which are used to collect patients' HRQoL data, should have established content validity in the target population to ensure that the instrument is comprehensive and comprehensible. The present study was conducted to evaluate the content validity of the Quality of Life in Myelodysplasia Scale (QUALMS) and the Functional Assessment of Cancer Therapy-Anemia (FACT-An) PRO instruments in patients with lower-risk MDS. METHODS: In this cross-sectional, qualitative study, 16 patients aged ≥18 years with lower-risk MDS, who were RBC transfusion dependent, literate and fluent in US-English were interviewed. Interviews were semi-structured comprising of two parts: concept elicitation (CE) explored symptoms and impacts important to patients, and cognitive debriefing (CD) assessed understanding and relevance of the QUALMS and FACT-An. A conceptual model was developed, which was used to map the concepts that emerged during CE onto the QUALMS and FACT-An to assess concept coverage and suitability of the instruments. RESULTS: The median age of participants was 67.5 years (range: 51-91), with half being female (n = 8). Nine (56.2%) participants had intermediate-1-risk MDS and 10 (62.5%) were relapsed or refractory to erythropoiesis-stimulating agent treatment. Fatigue/tiredness (100.0%), shortness of breath (87.5%), weakness (81.2%), and low energy (75.0%) were reported most commonly and were the most bothersome symptoms as well. Of seven high-level HRQoL domains identified, activities of daily living (n = 16, 100.0%), physical functioning (n = 15, 93.8%), emotional wellbeing (n = 13, 81.3%), social functioning (n = 12, 75.0%), sleep disturbance (n = 9, 56.3%), and impact on work (n = 9, 56.3%) were the most commonly reported. For CD, the QUALMS and FACT-An were found to be mostly relevant and very well understood; response options were easy to use, and recall period was appropriate. CONCLUSION: Both QUALMS and FACT-An demonstrated a strong face and content validity in patients with lower-risk MDS, suggesting that these instruments are appropriate for assessing HRQoL in this population.

Idiopathic Pulmonary Fibrosis and the Elderly: Diagnosis and Management Considerations.

Idiopathic pulmonary fibrosis (IPF) is a severe and progressive fibrosing interstitial lung disease, which ultimately results in respiratory failure and death. The median age at diagnosis is 66 years, and the incidence increases with age, making this a disease that predominantly affects the elderly population. IPF can often be difficult to diagnose, as its symptoms--cough, dyspnoea and fatigue--are non-specific and can often be attributed to co-morbidities such as heart failure and chronic obstructive pulmonary disease. Making an accurate diagnosis of IPF is imperative, as new treatments that appear to slow the progression of IPF have recently become available. Pirfenidone and nintedanib are two such treatments, which have shown efficacy in randomised controlled trials. As with all new treatments, caution must be advocated in the elderly, as these patients often lie outside the narrow clinical trial cohorts that are studied, and the benefits of therapy must be weighed against potential toxicities. Both medications, while relatively safe, have been associated with adverse effects, particularly gastrointestinal symptoms such as nausea, diarrhoea and anorexia. In this review, we highlight measures to improve recognition and accurate diagnosis of IPF, as well as co-morbidities that often affect the diagnosis and disease course. The gold standard for IPF diagnosis is a multidisciplinary meeting whereby clinicians, radiologists and histopathologists reach a consensus after interactive discussion. In many cases, a lung biopsy may not be available because of high risk or patient choice, particularly in the elderly. In these cases, there is debate as to whether a biopsy is required, given the high rates of IPF in patients over the age of 70 years with interstitial changes on computed tomography. We also discuss the management of IPF, drawing particular attention to specific issues affecting the elderly population, especially with regard to polypharmacy and end-of-life care. Through this article, we endeavour to improve awareness of this devastating disease and thus improve recognition of the disease and its outcomes in elderly patients.

Validity and reliability of the Behavioural Assessment Tool for Cognition and Higher Function (BATCH) in neuropsychiatric patients.

OBJECTIVE: Patients with mental health disorders frequently have difficulties with their cognitive functioning. Assessment of cognition in this population can be complicated by psychiatric symptomatology, making standard written and verbal assessment methods difficult. In these situations, observations by nursing and allied staff of patients' routine activities provide an important source of information about cognitive ability. The Behavioural Assessment Tool for Cognition and Higher Function (BATCH) was developed to record observations of patients' daily functioning under subheadings that reflect cognitive domains. Its capacity to provide a measure of cognitive function through observational means was assessed in a neuropsychiatric unit. METHOD: A consecutive sample of 76 adult neuropsychiatry inpatients were assessed over 6 months using BATCH. BATCH measures the frequency of given behaviours grouped under 10 functional and cognitive domains: orientation, attention/concentration, personal responsibility, volition, adaptation, problem-solving/judgement, executive function, memory, language, and visuospatial function. Data from routine standardized cognitive (Mini-Mental Status Examination, MMSE; Neuropsychiatry Unit Cognitive Screening Tool, NUCOG), psychiatric (Neuropsychiatric Inventory; Health of the Nation Outcome Scale) and functional (Bristol Activities of Daily Living Scale; Barthel Index) instruments were collected to determine the relative contribution of cognitive function to scores on the BATCH. RESULTS: A strong correlation was found between total BATCH scores and total NUCOG and MMSE scores. BATCH and NUCOG subdomains correlated significantly in all subscales. BATCH demonstrated very high internal consistency. Linear regression analysis showed that the strongest determinant of BATCH scores was cognitive function as measured on the NUCOG. A significant subscale x group effect showed lower BATCH scores in memory, orientation, attention, executive function and language in dementia sufferers compared to psychiatric and neurological patient groups. CONCLUSION: BATCH scores correlated strongly with pencil-and-paper measures of cognitive function. BATCH provides clinicians with a means of assessing cognitive function through behavioural observation, thus enabling assessment of patients with behavioural disturbance or severe psychopathology. This tool has practical application for adult and aged clients across all observational mental health settings.

The development and validation of a carer questionnaire to assess cognitive function in neuropsychiatric patients.

OBJECTIVES: The carer history is an integral part of the assessment of patients with cognitive impairment. We aimed to develop a comprehensive yet concise carer questionnaire, the CogRisk, which captures actuarial risk variables for cognitive impairment in addition to key symptoms suggestive of cognitive decline in a number of cognitive domains, and to then assess its validity and reliability in a neuropsychiatric population. METHOD: Carers of patients assessed for cognitive impairment completed the CogRisk, and patients were clinically assessed using the Mini-Mental State Examination (MMSE) and Neuropsychiatry Unit COGnitive assessment tool (NUCOG). Reliability was assessed using test-retest and interrater measures and measures of internal consistency. Construct and concurrent validity was assessed using correlation between total and subscale scores on the CogRisk, total scores on the NUCOG and MMSE, and subscale scores on the NUCOG. Predictive validity was determined using measures of sensitivity and specificity and using receiver operating characteristic (ROC) methods. RESULTS: The CogRisk was completed by all carers in less than 10 min. The total CogRisk score correlated significantly with total MMSE and NUCOG scores (r=-0.511 and -0.563, respectively) and remained highly significant when age and education were controlled for. Internal consistency of CogRisk items was high (alpha=0.943). Intrarater reliability of the CogRisk was high with an intraclass correlation coefficient of .978 (P<.001), and interrater reliability between carers was also high at 0.868 (P<.05). Sensitivity and specificity for the detection of dementia were .70 and .73, respectively, with area under the ROC curve not significantly different from that of the MMSE or NUCOG. CONCLUSION: The CogRisk is a brief carer-rated tool of a patient's cognitive functioning developed for use within a neuropsychiatric setting. It exhibited good concurrent validity, internal consistency, and interrater and intrarater reliability. The CogRisk also demonstrated good sensitivity and specificity for dementia. The CogRisk provides carer information, which complements the clinical assessment and can be used to focus on direct carer interview.