Survey Of Caregivers Research Articles

Caregiver and Youth Characteristics That Influence Trust in Digital Health Platforms in Pediatric Care: Mixed Methods Study.

Combining patient-generated health data and digital health platforms may improve patient experience and population health, mitigate rising health care costs, reduce clinician burnout, and enable health equity. However, lack of trust may be a notable barrier to the data-sharing required by such platforms. Understanding sociodemographic, health, and personal characteristics will enable developers and implementers of such technologies to consider these in their technical design requirements. This study aims to understand relationships between sociodemographic characteristics of caregivers of children or adolescents and trust in and willingness to use digital platforms to store and share personal health information for clinical care and research. This study used a mixed methods approach, including surveys of caregivers of youth aged <18 years living in Canada or the United States and youth aged 16 to 17 years living in Canada, as well as web-based bulletin board discussions to further explore topics of trust in data sharing. Sociodemographic and survey data were tabulated and explored using proportional odds ordinal regression models. Comments from web-based group discussions were analyzed thematically using a coding approach to identify issues important to the participants. Survey data from 1128 caregivers (female participants: n=549, 48.7%; 36-50 years old: n=660, 58.5%; Canadian: n=603, 53.5%; urban population: n=494, 43.8%) were collected, of which 685 (60.7%) completed all questions. Data from 173 youth (female participants: n=73, 42.2%; urban population: n=94, 54.3%) were collected, of which 129 (74.6%) completed all questions, and data were available for analysis. Furthermore, among 40 participants, 23 (58%) caregivers contributed to the web-based discussion boards. Related to trust, living in a rural area (vs urban; odds ratio [OR] 0.66, 95% CI 0.46-0.95) resulted in lower concern for data privacy and security, while having an undergraduate (OR 1.82, 95% CI 1.30-2.55) or graduate degree (vs secondary or trade school; OR 2.50, 95% CI 1.68-3.73) resulted in higher levels of concern. Living with a chronic disease (OR 1.81, 95% CI 1.35-2.44) increased levels of concern regarding data privacy and security. Interestingly, those with chronic disease were more willing to use digital platforms for clinical care and share personal health information for not-for-profit research. Caregivers were most concerned about data breaches involving data from their children but also highlighted that digital platforms would allow for better coordination of care for their children. Our research confirms the willingness of caregivers and youth to use digital platforms for both clinical care delivery and research and suggests that the value of a digital platform may outweigh the risks of its use. Engagement of end users in co-designing such platforms has the potential to enhance digital trust. However, digital trust varies across sociodemographic groups; therefore, diverse end user engagement is necessary when designing digital applications.

The economic burden of varicella among children in France: a caregiver survey.

A substantial economic burden is associated with pediatric varicella in France, primarily due to the productivity loss among caregivers. •Although varicella is considered a mild disease, it poses a significant burden on caregivers and society. Our study aimed to estimate the economic burden of varicella among children in France from family and societal perspectives to support policymakers in understanding the value of UVV in France. • We conducted an online survey of caregivers of 185 French children (≤17 years) diagnosed with varicella and collected data on varicella-related healthcare resource use, missed school days and workdays. Our study estimated annual societal burden of pediatric varicella in France. Out of 185 children with varicella, most (95.1%) had at least one outpatient visit, 10.3% had at least one emergency room visit, and 2.2% were hospitalized. 61.1% of parents with sick child, missed work and median productivity loss was 2.0 workdays. Additionally, 170 children who attended school or preschool missed a median of 5.0 school days. • The societal burden of pediatric varicella in France was estimated to be €450,427,578 (95% CI, €357,144,618 - €543,710,538), with indirect costs accounting for 85%. Pediatric varicella is associated with a substantial economic burden in France, primarily due to high productivity losses among parents and caregivers.

6811 RESTORE: a Real-World Study of Setmelanotide in Patients With MC4R Pathway Diseases

Abstract Disclosure: C. Huber: Employee; Self; Rhythm Pharmaceuticals, Inc.. Stock Owner; Self; Rhythm Pharmaceuticals, Inc. B. Sweeney: Consulting Fee; Self; Rhythm Pharmaceuticals, Inc., Novo Nordisk. Research Investigator; Self; Rhythm Pharmaceuticals, Inc.. Speaker; Self; Rhythm Pharmaceuticals, Inc. J. Pomeroy: Research Investigator; Self; Rhythm Pharmaceuticals, Inc. U. Mallya: Employee; Self; Rhythm Pharmaceuticals, Inc.. Stock Owner; Self; Rhythm Pharmaceuticals, Inc. S. Zhang: Other; Self; Employer received funding from Rhythm Pharmaceuticals, Inc. to support this research. M. Yang: Other; Self; Employer received funding from Rhythm Pharmaceuticals, Inc. to support this research. S. Malhotra: Employee; Self; Rhythm Pharmaceuticals, Inc.. Stock Owner; Self; Rhythm Pharmaceuticals, Inc. A.M. Haqq: Advisory Board Member; Self; Rhythm Pharmaceuticals, Inc., Novo Nordisk, Foundation for Prader-Willi Research USA. Grant Recipient; Self; Weston Family Microbiome Initiative and Canadian Institutes of Health Research. Background: Setmelanotide has demonstrated efficacy in clinical trials of patients with obesity due to rare melanocortin-4 receptor pathway diseases. Real-world evidence can provide a greater understanding of patient outcomes, such as hyperphagia and quality of life, in a clinical practice setting. Real-world Evidence of SeTmelanotide fOr Hyperphagia and ChRonic Weight ManagEment (RESTORE) is a prospective, observational, longitudinal study aimed at assessing patient- and caregiver-reported real-world effectiveness of setmelanotide among patients with Bardet-Biedl syndrome and proopiomelanocortin, proprotein convertase subtilisin/kexin type 1, or leptin receptor deficiency and their caregivers. Methods: Patients aged ≥6 years and caregivers aged ≥18 years who are able to respond to a survey in English and consented to the Rhythm Intune Program are eligible for the study. Patients must have been prescribed, but not yet initiated, setmelanotide, not have been previously treated with setmelanotide, and not have previously participated in or be currently enrolled in any clinical trial, including setmelanotide trials. Caregivers must be confirmed legal guardians of patients without prior exposure to setmelanotide, have cared for their patient for ≥6 months, and not be paid professional caregivers. Participants will complete secure online surveys at baseline (ie, before treatment) and at 6 posttreatment time points over a 1-year study period. Patient surveys collect information on hyperphagia-related signs and symptoms, medical history, treatment adherence, weight management programs and treatment, comorbidities/complications, comedications, disease impact on psychological well-being and health-related quality of life, treatment satisfaction, work/school/activity status, and patient global assessment. Caregiver surveys include assessments on psychological well-being, treatment satisfaction, and impact on activities of daily living, work status, and family dynamics. A subset of study patients will be invited to receive a wearable device (CentrePoint® Insight Watch) to assess activity and sleep patterns. Treatments and outcomes among patients will be described and compared with baseline for all post-treatment initiation assessment time points. Current status: RESTORE is the first study to assess the real-world effectiveness of setmelanotide, has been IRB approved, and is actively enrolling. Presentation: 6/3/2024

The Telehealth Education for Asthma Connecting Hospital and Home (TEACHH) pilot study

Objective To evaluate the feasibility of a novel intervention of health literacy-informed, telemedicine-enhanced asthma education and home management support for hospitalized children and caregivers, and assess caregiver perspectives of the intervention. Methods We conducted a pilot randomized trial of the Telehealth Education for Asthma Connecting Hospital and Home (TEACHH) intervention vs. standardized care (SC) for children (5–13 yrs) hospitalized with asthma. Participants in TEACHH received health literacy-informed teaching prior to discharge, including pictorial materials (e.g. flipchart, action plan), color- and shape-coded medication labels, and medication demonstration. Two Zoom-based follow-up teaching visits were completed within 1-month of discharge. Feasibility was assessed by tracking visit completion, and we measured preliminary outcomes using health records (i.e. total asthma-related acute healthcare visits) and blinded surveys of caregivers 2-, 4-, and 6-months post-discharge (i.e. symptom-free days, quality of life). We interviewed caregivers about their perceptions of TEACHH. Transcripts were coded inductively. Results We enrolled 26 children and interviewed 14 caregivers (9 TEACHH, 5 SC). All inpatient sessions were completed, as were 77% of virtual visits. Both groups experienced improved symptoms and quality of life over time. Caregivers valued the teaching, involvement of children, visual tools, and color-coded information of TEACHH. They described child-specific benefits, greater support after discharge, and improved asthma-related communication, and indicated that other families would benefit from similar teaching. Conclusions A novel program of patient-centered asthma education was feasible in both hospital and home settings and well received by caregivers. A larger study is needed to assess the impact of TEACHH on childhood asthma morbidity. Clinicaltrials.gov identifier NCT04995692 (Registration date 8/9/2021)

Barriers in Accessing Orthodontic Care for Patients with Orofacial Clefts: Insights from a Florida-Based Survey and National Database Analysis.

To assess the barriers to obtaining care for patients with orofacial clefts through a survey of Florida-based orthodontists and families and an analysis of the Pediatric Health Information System (PHIS) database. A cross-sectional study utilizing multiple-choice questionnaires completed by Florida orthodontists and caregivers of patients who attended a Florida-based cleft and craniofacial clinic. Additionally, data from the PHIS database were analyzed to investigate national factors affecting the age of alveolar bone grafting (ABG). Craniofacial team in Florida. The survey included 39 orthodontists (7.1% response rate) and 48 caregivers (41% response rate) The PHIS study included 1182 patients. Barrier to orthodontic care and age of ABG. Orthodontic Survey: Among the surveyed orthodontists, 71% treated cleft/craniofacial patients, 37% accepted Medicaid, and 55% provided pro-bono care. Poor reimbursement was identified as the most common barrier (58%). Caregiver Survey: Most patients were insured by Medicaid (67%), with 55% incurring out-of-pocket expenses. PHIS Database: The average age of ABG was 10.3 years (SD = 3.2). Government funding was associated with a 6.0-month delay in ABG (p = 0.047) and residing in non-Medicaid expanded states was linked to a 6.0-month delay (p = 0.023). Post-Medicaid expansion status was also associated with a delay (p = 0.004). Access to oral care is difficult for patients with OFC. Despite both federal and state mandates, many financial and non-financial barriers still exist in accessing orthodontic care and a majority of patients experience significant out-of-pocket expenses despite statutorily mandated insurance coverage.

Utilization of Real-World Data in the Intervention to Reduce Early Peanut Allergy in Children (iREACH) Trial

ObjectiveTo demonstrate the use of linked, real-world data in the context of a pediatric practice-based clinical trial. ApproachThe Intervention to Reduce Early Peanut Allergy in Children (iREACH) is a two-arm, cluster randomized, controlled trial. The intervention included clinician education and clinical decision support to increase pediatric clinician adherence to the NIAID’s 2017 Prevention of Peanut Allergy guidelines during infants’ 4- and 6-month well child care visits. Electronic health record (EHR) data were shared with the data coordinating center under a Health Insurance Portability and Accountability Act (HIPAA) waiver. All caregivers of eligible infants were invited to complete two surveys via REDCap, an online survey platform. The consents included an optional HIPAA authorization to link EHR data with survey responses and authorization to collect and link administrative data from the Special Supplemental Nutrition Program for Women, Infants, and Children (WIC) program, if applicable. ResultsOverall, 30 Illinois pediatric practices participated. EHR data was collected for 18,480 infants of whom 4,331 had any caregiver survey data (23.4% response rate). Permission to link survey responses to EHR data was obtained for 3,950 infants among those with any survey data (91.2%). Consent for linking WIC data with study data was obtained for 229 infants and 194 mothers. ConclusionsAuthorization for data linkage in the consent forms allowed investigators to amass data to compare caregiver and clinician responses, generate hypotheses, and propose future research outside the scope of the main trial. ImplicationsMultiple real-world data sources can be effectively combined to extend the findings of clinical trials.

Inappropriate trusting behaviour in dementia.

Inappropriate trusting behaviour may have significant social, financial and other consequences for people living with dementia. However, its clinical associations and predictors have not been clarified. Here we addressed this issue in canonical syndromes of frontotemporal dementia (FTD) and Alzheimer's disease (AD). In 34 patients with AD and 73 with FTD (27 behavioural variant (bv)FTD, 22 semantic variant primary progressive aphasia (svPPA), 24 nonfluent/agrammatic variant (nfv)PPA) we recorded inappropriate trusting and other abnormal socio-emotional behaviours using a semi-structured caregiver survey. Patients were comprehensively characterised using a general cognitive assessment and the Revised Self-Monitoring Scale (RSMS; an informant index of socioemotional awareness). Inappropriate trusting was more frequent in svPPA (55%) and bvFTD (44%) than nfvPPA (17%) or AD (24%). After adjusting for age, sex, education and Mini-Mental State Examination (MMSE) score, inappropriate trusting was significantly more likely in svPPA (odds ratio 3.61; 95% confidence interval 1.41-8.75) and bvFTD (3.01, 1.23-6.65) than AD. Significant predictors of inappropriate trusting comprised apathy in svPPA, disinhibition and altered pain responsiveness in bvFTD, and lower MMSE and RSMS (self-presentation) scores in AD. Dementia syndromes vary in prevalence and predictors of abnormal trusting behaviour, with implications for clinical counselling and safeguarding.

Dysphagia in primary progressive aphasia: Clinical predictors and neuroanatomical basis.

Dysphagia is an important feature of neurodegenerative diseases and potentially life-threatening in primary progressive aphasia (PPA) but remains poorly characterized in these syndromes. We hypothesized that dysphagia would be more prevalent in nonfluent/agrammatic variant (nfv)PPA than other PPA syndromes, predicted by accompanying motor features, and associated with atrophy affecting regions implicated in swallowing control. In a retrospective case-control study at our tertiary referral centre, we recruited 56 patients with PPA (21 nfvPPA, 22 semantic variant [sv]PPA, 13 logopenic variant [lv]PPA). Using a pro forma based on caregiver surveys and clinical records, we documented dysphagia (present/absent) and associated, potentially predictive clinical, cognitive, and behavioural features. These were used to train a machine learning model. Patients' brain magnetic resonance imaging scans were assessed using voxel-based morphometry and region-of-interest analyses comparing differential atrophy profiles associated with dysphagia presence/absence. Dysphagia was significantly more prevalent in nfvPPA (43% vs. 5% svPPA and no lvPPA). The machine learning model revealed a hierarchy of features predicting dysphagia in the nfvPPA group, with excellent classification accuracy (90.5%, 95% confidence interval = 77.9-100); the strongest predictor was orofacial apraxia, followed by older age, parkinsonism, more severe behavioural disturbance, and more severe cognitive impairment. Significant grey matter atrophy correlates of dysphagia in nfvPPA were identified in left middle frontal, right superior frontal, and right supramarginal gyri and right caudate. Dysphagia is a common feature of nfvPPA, linked to underlying corticosubcortical network dysfunction. Clinicians should anticipate this symptom particularly in the context of other motor features and more severe disease.

An Inventory of Rare Disease Registries in the Canadian Landscape

What Was the Question? There is a lack of centralized information about rare disease registries (RDRs) in Canada. To address this, we created an inventory of RDRs in Canada and international RDRs that include patients living in Canada. What Did We Do? Information about RDRs was identified through a search of published literature, grey literature, consultations with members of the rare disease community, and a survey with the registry holders. What Did We Find? We identified 148 RDRs, of which 66 are RDRs in Canada and 82 international RDRs. In total, 21% of RDRs in Canada and 11% of international RDRs capture rare cancer(s). About a half of RDRs in Canada (53%) and international RDRs (46%) collaborate with other national or international registries or networks. Most RDRs in Canada (73%) and international RDRs (86%) identify patients for RDR enrollment through health care provider referrals. Electronic medical records (68%), clinician-reported data (68%), and medical chart abstraction (60%) are the most common sources of data for RDRs in Canada compared to international RDRs, for which patient (79%) and caregiver (61%) surveys are most common. Most RDRs in Canada collect clinical data (95%), laboratory and diagnostic data (85%), health outcomes data (83%), and treatment data (78%), and less commonly patient-generated data (55%) and caregiver data (15%). What Does This Mean? The information in the inventory will help guide future initiatives for improving the RDR landscape in Canada. Most RDRs in Canada source data from electronic health records, clinician-reported data, and medical charts. Fewer RDRs in Canada implement patient and caregiver surveys. RDRs in Canada include information relevant to decision-makers as most collect clinical data, health outcomes data, and treatment data, and about half collect health resource utilization data. This inventory will support future initiatives to assess the suitability of RDRs for generating decision-grade real-world evidence.

Geographical location as a determinant of caregiver burden: a rural-urban analysis of the informal caregiving, health, and healthcare survey in Ghana

BackgroundThe caregiving scholarship widely acknowledges informal caregivers’ contributions to maintaining older adults’ health and well-being. However, informal caregivers encounter economic, physical, social, financial and psychological challenges when caring for older adults. The caregiving literature has shown variations in caregiving intensity and motivation between rural and urban informal caregivers of older adults. This situation is likely to result in rural-urban disparities in caregiver burden. However, the literature on predictors of caregiver burden is more focused on demographic, socio-economic, caregiving and health-related factors with very little attention to geographical dynamics. For this reason, the effects of demographic, socio-economic, caregiving, and health-related factors on the variations in caregiver burden between rural and urban informal caregivers of older adults are yet to be known in the sub-Saharan African context, including Ghana. Notably, the impact of geographical location on caregiver burden is mainly missing in the informal caregiving literature in Ghana. Situated within the stress process model, we determine the association between geographical location and caregiver burden among informal caregivers of older adults in Ghana.MethodsThis study employed data from a large cross-sectional survey on informal caregiving, health, and healthcare among caregivers of older adults aged 50 years or above (N = 1,853) in Ghana. We selected the World Health Organization Impact of Caregiving Scale to measure caregiver burden. Generalized multivariable linear regression models were employed to determine the association between geographical location and caregiver burden among informal caregivers of older adults. We reported beta values and standard errors with significance levels of 0.05 or less.ResultsThe results showed that rural informal caregivers of older adults significantly have a decreased caregiver burden compared to urban informal caregivers (β = -1.64; SE = 0.41). Also, participants across all the self-rated health categories (poor/very poor: β = 12.63; SE = 1.65; fair: β = 9.56; SE = 1.07; good: β = 11.00; SE = 0.61, very good: β = 7.03; SE = 0.49) have a significantly increased caregiver burden for the full sample and for both rural (poor/very poor: β = 13.88; SE = 2.4; fair: β = 6.11; SE = 1.62; good: β = 9.97; SE = 0.96, very good: β = 6.06; SE = 0.71) and urban (poor/very poor: β = 11.86; SE = 2.25; fair: β = 12.33; SE = 1.42; good: β = 11.80; SE = 0.79, very good: β = 7.90; SE = 0.67) participants. This study further revealed that participants with no financial support needs reported a decreased caregiver burden compared to those with financial support needs for the full sample (β = -2.92, p-value < 0.01) and for both rural (β = -3.20; p-value < 0.01) and urban (β =-2.70; p-value < 0.01) participants.ConclusionThe findings from this study underscore geographical location differences in caregiver burden among informal caregivers of older adults in Ghana. Given these findings, the need to consider geographical location variations in providing welfare and health support programs to lessen caregiver burden among informal caregivers of older adults is welcomed. In line with the stress process model, such welfare and health programs should consider background, context, and stressor factors that contribute to variations in caregiver burden between rural and urban informal caregivers of older adults in Ghana and other sub-Saharan African countries.

Harm to children from others' drinking: A survey of caregivers in Australia.

This study aimed to identify the prevalence and types of harm to children from others' drinking in Australia, as indicated by caregivers, and examine socio-demographic characteristics of caregivers who indicated a child was affected by others' drinking. A subsample of 854 adult respondents, who were caregivers of children under 18 years from the 2021 Australian Alcohol's Harm to Others study, were asked questions about whether children in their care had been negatively affected by others' drinking in the past year. Weighted prevalence estimates of overall and specific harms to children are presented. Logistic regressions were conducted to determine characteristics associated with indicating harms to children from others' drinking. Over 17% of caregivers (95% confidence interval [CI] = 13.0-19.0) indicated that one or more children in their care had been affected by others' drinking in the past 12 months. Verbal abuse (6.2%; 95% CI = 4.3-8.3) was the most common harm indicated, followed by financial harm (4.3%; 95% CI = 2.7-6.2). One percent of caregivers (95% CI = 0.4-2.3) indicated a child was physically hurt and less than 1% (95% CI = 0.2-1.4) indicated a child was the subject of a child protection call due to someone's drinking. Women and caregivers over 65 years were more likely to indicate a child had been affected compared with men and caregivers under 65 years. Caregivers who drank five or more drinks at least three times per week were four times more likely to indicate a child was affected compared with abstainers. Living in a household with someone who drinks heavily and who had negatively affected the child's caregiver was associated with an increased likelihood of child harm. In 2021, a weighted survey estimate for caregivers in Australia indicated that almost one in six children had been affected by others' drinking. Heavier drinking of caregivers and other household members was the most substantial predictor for indicating a child had been negatively affected by others' drinking.

Palliative Care for Dialysis-Dependent Pediatric Patients: A Survey of Providers, Nurses, and Caregivers.

Dialysis-dependent pediatric patients and their families face significant biopsychosocial burdens and low health-related quality of life. Palliative care consultations can alleviate some degree of suffering for patients and families but remain underutilized within pediatric nephrology. Little is known about how providers, nurses, and caregivers perceive palliative care integration into the multidisciplinary care of dialysis-dependent pediatric patients. This study surveyed pediatric nephrology providers (physicians and advanced practice providers), inpatient pediatric nephrology bedside nurses, and caregivers of dialysis-dependent pediatric patients at a freestanding tertiary care children's hospital in Dallas, Texas. Participants completed a survey regarding knowledge about, experiences with, and perceptions of palliative care in pediatric nephrology. 10 providers, 20 nurses, and 18 caregivers completed the survey. Although 80% of providers and all nurses agreed that palliative care would benefit dialysis-dependent pediatric patients, most believed that palliative care is not as important in pediatric nephrology as it should be. 70% of providers and 45% of nurses believed that they understand the scope of palliative care. 90% of providers and all nurses desired more palliative care education. Of the 22% of caregivers whose child had already received palliative care services, all found the consultation to be helpful. Many providers and nurses worried that a palliative care consultation would signal to families that the nephrology team would be giving up on their child and that their child is approaching the end of life. However, no caregivers thought that a consultation would mean that the nephrology team would be giving up on their child and only 6% worried that it would indicate that their child is approaching the end of life. These data support further palliative care education for pediatric nephrology providers and nurses and more robust and systematic involvement of subspecialty palliative care for dialysis-dependent pediatric patients.

Burden of illness in Rett syndrome: initial evaluation of a disorder-specific caregiver survey

BackgroundRett syndrome (RTT) is a severe X-linked neurodevelopmental disorder associated with multiple neurologic impairments. Previous studies have shown challenges to the quality of life of individuals with RTT and their caregivers. However, instruments applied to quantify disease burden have not adequately captured the impact of these impairments on affected individuals and their families. Consequently, an international collaboration of stakeholders aimed at evaluating Burden of Illness (BOI) in RTT was organized.MethodsBased on literature reviews and qualitative interviews with parents of children and adults with RTT, a caregiver questionnaire was constructed to evaluate 22 problems (inclusive of core characteristics, functional impairments, and comorbidities) often experienced with RTT, rated mainly with a 5-level Likert scale. The questionnaire was administered anonymously online to an international sample of 756 caregivers (predominantly parents) of girls and women with RTT. Descriptive statistics were used to identify problems of high frequency and impact on affected individuals and caregivers. Chi-square tests characterized the relationship between problem severity and impact responses, while nonparametric ANOVAs of raw and z-score adjusted scores identified agreement between severity and impact on individual and caregiver. Secondary inferential tests were used to determine the roles of age, clinical type, and country of residence on BOI in RTT.ResultsThere was variability in reported frequency of problems, with the most prevalent, severe and impactful being those related to the core features of RTT (i.e., communication and fine and gross motor impairments). Chi-square analyses demonstrated interdependence between severity and impact responses, while ANOVAs showed that many problems had disproportionately greater impact than severity, either on affected individuals (e.g., hand stereotypies) or their caregivers (e.g., sleep difficulties, seizures, pain, and behavioral abnormalities). With certain exceptions (e.g., breath-holding, seizures), age, clinical type, or country of residence did not influence these BOI profiles.ConclusionsOur data demonstrate that core features and related impairments are particularly impactful in RTT. However, problems with mild severity can also have disproportionate impact on affected individuals and, particularly, on their caregivers. Future analyses will examine the role of factors such as treatment outcomes, healthcare services, and healthcare provider’s perspectives, in these BOI profiles.

Facilitators of &amp; Barriers to Caregiver Adherence to Home Therapy Recommendations for Brachial Plexus Birth Injury: A Survey Study

Abstract Date Presented 03/21/24 This poster presents findings from a survey of caregivers of infants with brachial plexus birth injury regarding facilitators of and barriers to adherence to home therapy recommendations; best practices for making home recommendations are identified. Primary Author and Speaker: Jennifer Wingrat Additional Authors and Speakers: Shayna Gertz, Amy Mae Miller

A Caregiver Survey on Medical and Behavior Analytic Treatments for Idiopathic Toe Walking

A Caregiver Survey on Medical and Behavior Analytic Treatments for Idiopathic Toe Walking

Cost-Utility Analysis of Bilateral Cochlear Implants for Children With Severe-to-Profound Sensorineural Hearing Loss in Taiwan.

Cochlear implants are an option for children with sensorineural hearing loss who do not benefit from hearing aids. Although bilateral cochlear implantation (CI) has been shown to enhance hearing performance and quality of life, its cost-effectiveness remains unclear. This study aimed to evaluate the cost-effectiveness of bilateral CI compared with bimodal hearing for children with sensorineural hearing loss in Taiwan from both the perspectives of patients and Taiwan's National Health Insurance Administration (TNHIA). A four-state Markov model was utilized in the study, including "use the first internal device," "use the second internal device," "use the third internal device," and "death." Health utility values were obtained from a local survey of health professionals and then adjusted by a scale to reflect both the negative impact of aging on hearing and the time needed to develop the full benefit of treatment in the earliest years of life. The cost data were derived from a caregiver survey, hospital databases, clinical experts, and the TNHIA. The incremental cost-effectiveness ratio (ICER) was calculated over the lifetime horizon and presented as cost per quality-adjusted life year (QALY) to evaluate the cost-effectiveness of simultaneous bilateral CI, sequential bilateral CI, and bimodal hearing. In addition, one-way sensitivity analyses and probabilistic sensitivity analyses were conducted to investigate the impact of uncertainty and the robustness of the model. The base-case analysis showed that children with bilateral CI gained more QALYs while incurring more costs when compared with those with bimodal hearing. From the TNHIA perspective, compared with bimodal hearing, the ICER of simultaneous bilateral CI was New Taiwan Dollars 232,662 per QALY whereas from the patient perspective, the ICER was New Taiwan Dollars 1,006,965 per QALY. Moreover, simultaneous bilateral CI dominated sequential bilateral CI from both perspectives. Compared with bimodal hearing, the ICER of sequential bilateral CI did not exceed twice the gross domestic product per capita in Taiwan from either perspective. One-way sensitivity analysis demonstrated that the utility gain of bilateral CI compared with bimodal hearing was the most impactful parameter from both perspectives. Probabilistic sensitivity analysis confirmed the robustness of the base-case analysis results. Our findings reveal that bilateral CI was cost-effective when using the threshold of one to three times the 2022 gross domestic product per capita in Taiwan from both the TNHIA and patient perspectives. Future research incorporating cost and effectiveness data from other dimensions is needed to help decision-makers assess the cost-effectiveness of bilateral CI more comprehensively.

Evaluation of the Arthur Project: Evidence-Based Mentoring in a Social Work Framework with a Social Justice Approach

Low-income students from underrepresented communities experience significant educational inequalities, including chronic absenteeism, mental health crises, trauma exposure, and social injustices. These conditions escalate risks of dropping out and justice system involvement. Middle-school youth must navigate intense social and emotional change. To address such challenges, The Arthur Project (TAP) provides intensive, in-school, therapeutic mentoring and family support. The program teaches students essential life skills, such as problem-solving, communication, and academic engagement. Students are empowered to strengthen their resilience, perseverance, self-actualization, and confidence. TAP provides up to 500 h of therapeutic mentoring yearly over three years, delivered individually and in small groups by social work interns. The research question was as follows: Does structured, intensive in-school therapeutic mentoring with social work interns increase the socioemotional, cognitive, noncognitive, and academic skills and engagement of underserved middle-school students? This evaluation for the school year 2022–2023 is based on the program of 200 students and 30 mentors, with quantitative mentee, mentor, teacher, and caregiver surveys and qualitative interview data. The program and evaluation methodology are described, with results and conclusions. Students showed significant improvement in all domains; mentors reported student increases in confidence, perseverance, problem-solving, and communication; and teachers and caregivers reported students’ increased academic engagement. The Arthur Project program can become a national middle-school mentoring model to address widespread student inequalities.

P38 Sources of Financial Support to Households Caring for Individuals with Duchenne Muscular Dystrophy: Results from a Caregiver Survey

P38 Sources of Financial Support to Households Caring for Individuals with Duchenne Muscular Dystrophy: Results from a Caregiver Survey

PCR124 Burden of Fracture and Fracture Management Among Individuals with Osteogenesis Imperfecta: Results from a Survey of Adults and Caregivers

PCR124 Burden of Fracture and Fracture Management Among Individuals with Osteogenesis Imperfecta: Results from a Survey of Adults and Caregivers

Nonseizure- and seizure-related benefits of cannabidiol treatment in the real world: plain language summary of the results from a caregiver survey

What is this summary about? Lennox-Gastaut syndrome (LGS) and Dravet syndrome (DS) are rare forms of epilepsy that start in childhood and cause life-long disability. People with LGS and DS experience many seizure types and nonseizure-related problems with cognition (ability to think and understand), learning, behaviour and communication. People with LGS and DS depend on caregivers to assist with daily activities. Although many medications are available to treat seizures associated with LGS and DS, the effect on nonseizure outcomes is unclear. A highly purified (defined as a substance that has undergone a special process to remove impurities) oral (by mouth) solution of cannabidiol (CBD; Epidiolex®) from plants is approved by the United States (US) Food and Drug Administration (FDA) for the treatment of seizures associated with LGS, DS and tuberous sclerosis complex in people who are at least 1 year old. In clinical studies, treatment with CBD reduced the number of seizures. Common side effects (secondary, typically unintended effects of the medication) were increased liver enzyme levels, sleepiness, decreased appetite, diarrhea, fever, vomiting, tiredness, rash, problems with sleep and infections. CBD may also improve nonseizure outcomes, such as cognition, behavior and quality of life. The BEhavior, COgnition and More with Epidiolex® (BECOME) survey was designed to find out whether caregivers of people with LGS or DS taking CBD noticed changes in nonseizure and seizure outcomes. What are the key takeaways? Caregivers of people with LGS or DS taking CBD noticed improvements in cognition, language and communication, emotional and social functioning, physical functioning, sleep and ability to do daily activities, as well as a reduction in the number and severity of seizures. People with reduction in seizures were more likely to experience nonseizure benefits, but some people without seizure reduction also experienced improvements in nonseizure outcomes. Caregivers reported that both seizure and nonseizure effects were important when thinking about treatment options. What were the main conclusions reported by the researchers? The BECOME survey provides a unique caregiver perspective on nonseizure outcomes that may be affected by CBD. These results support further study of CBD for nonseizure outcomes in people with LGS or DS.