Caregiver Proxy Research Articles

An exploratory qualitative study on factors influencing the level of agreement in symptom reports in child-caregiver dyads.

Clinicians often rely on caregiver proxy symptom reports to treat cancer-related symptoms in children. Research has described disagreement between children's and caregivers' symptom reports. Factors influencing the level of agreement is an understudied area. Thus, this study aimed to examine potential factors contributing to the level of agreement between symptom reports provided by children and their caregivers. Sixteen child-caregiver dyads participated separately in semi-structured interviews after completing a brief symptom measure independently using an electronic device. Child and caregiver quantitative symptom responses were reviewed in real-time and incorporated into the semi-structured interview. Sample characteristics and the level of agreement between symptom reports were calculated using descriptive statistics. Transcribed participant interviews were analyzed using content analysis. Nearly half of child-caregiver dyads exhibited a moderate (37.5%, n = 6) or low (18.75%, n = 3) level of agreement on the abbreviated symptom measure. Qualitative analysis identified 5 themes: recognizing symptoms, experiencing symptoms, communicating symptoms, re-assessing and treating symptoms, and influencing individual and relationship factors. Influencing individual, including a child's tendencies or personality traits, and relationship factors intersected the other themes, partially explained their symptom perceptions, and served to facilitate or hinder symptom communication. Symptom communication is an important part of the symptom cycle, comprised of symptom recognition, experience, and management. Individual and relational factors may influence discrepancies in symptom perceptions between the child and caregiver. Clinicians and researchers should consider developing interventions to enhance symptom communication and promote collaboration between children and their caregivers to address symptom suffering during cancer treatment.

Beyond presence of symptoms: Self-reported psychosocial distress interference among outpatient youth with cancer and other life-limiting conditions.

Youth with life-limiting conditions face significant psychosocial challenges (e.g., symptoms of anxiety, depression, and pain) throughout illness and treatment. Without appropriate intervention, this can negatively affect long-term outcomes (e.g., disease management, health-related quality of life). Prompt identification and appropriate attention to distress can mitigate these effects. We aimed to determine the prevalence and severity of distress interference among outpatient youth with cancer and other life-limiting conditions, using the Checking IN screener. Within a larger study across four hospital centers, English-speaking pediatric outpatients aged 8-21, and a caregiver-proxy-reporter, completed a brief distress screener. Descriptive analyses were used to characterize the sample and evaluate reported distress symptoms. Checking IN was completed by 100 participants, aged 8-21 (M=14.27, SD=3.81); caregivers completed an equivalent proxy screener. Youth most frequently endorsed fatigue (moderate: n=50, 50.0%; high: n=21, 21.0%), paying attention (moderate: n=45, 45.0%; high: n=16, 16.0%), and sleep difficulty (moderate: n=46, 46.0%; high: n=13; 13.0%) as problematic. Caregivers proxy reported fatigue (moderate: n=46, 46.0%; high: n=32, 32.0%), worry (moderate: n=56, 56.0%; high: n=10, 10.0%), and sleep difficulty (moderate: n=47, 47.0%; high: n=14; 14.0%) as most problematic. Group differences between youth and caregiver responses were not significant. Youth self-report via Checking IN can detect psychosocial distress interference. By directing resources based on real-time assessment of symptom interference, there is potential to simplify outpatient psychosocial screening and improve referral timeliness and specificity, thus allowing for more effective attention to evolving symptoms of distress.

Medication adherence for people with acquired communication disorders: A systematic review.

Conditions such as stroke, dementia and neurodegeneration are major contributors to the incidence of acquired communication disorders in Europe. Pharmacological interventions play a central role in the management and treatment of these conditions, though many patients with an acquired communication disorder may be at a higher risk of medication non-adherence than their peers. The objectives of the current review were to identify, in the context of people with acquired communication disorders: factors that influence medication adherence; current interventions targeting medication adherence; and current measures of medication adherence. This study was conducted and reported in accordance with both PRISMA and SWiM guidelines. Two authors independently screened the results of a literature search, assessed risk of bias and extracted relevant data. Eight studies were identified for inclusion. The results of this review indicate that patient-related factors are most indicative of medication non-adherence in a population with acquired communication disorders, followed by socioeconomic factors and medication-related factors. Despite the recognized importance of medication adherence, no gold standard of assessment or intervention currently exists for this population. Half of the included studies replaced patients with communication difficulties with caregiver proxies, thus reducing opportunities for patients to have agency over their own healthcare. The term "acquired communication disorders" encompasses a range of conditions with diverse aetiologies, presentations and needs, and future research should be tailored to specific patient groups most at risk of medication non-adherence, namely those with aphasia and cognitive-communication impairments. Patients should be empowered to participate in future research to ensure the literature accurately represents their lived experience.

Self and proxy symptom reporting in glioma patient-caregiver dyads: the role of psychosocial function in rating accuracy

BackgroundIllness-related communication and depressive symptoms within families may play an important role in caregivers’ ability to accurately understand patients’ symptom burden. We examined the associations between these psychosocial factors and symptom accuracy in patients with glioma and their family caregivers.MethodsPatients and caregivers (n = 67 dyads) completed measures of depressive symptoms (CES-D), illness communication (CICS), and QOL (SF-36). Patients reported on their own cancer-related symptoms (MDASI-BT) while caregivers reported on their perception of the patients’ symptoms (i.e., proxy reporting). Paired t-tests and difference scores were used to test for agreement (absolute value of difference scores between patients and caregiver proxy symptom and interference severity reports) and accuracy (caregiver underestimation, overestimation, or accurate estimation of patient symptom and interference severity).ResultsClinically significant disagreement was found for all means scores of the MDASI-BT subscales except for gastrointestinal symptoms and general symptoms. Among caregivers, 22% overestimated overall symptom severity and 32% overestimated overall symptom interference. In addition, 13% of caregivers underestimated overall symptom severity and 21% of caregivers underestimated overall symptom interference. Patient illness communication was associated with agreement of overall symptom severity (r=−0.27, p = 0.03) and affective symptom subscale (r=−0.34, p < 0.01). Caregivers’ reporting of illness communication (r=−0.33, p < 0.01) and depressive symptoms (r = 0.46, p < 0.0001) were associated with agreement of symptom interference. Caregiver underestimating symptom severity was associated with lower patient physical QOL (p < 0.01); caregiver underestimating symptom interference was associated with lower patient physical QOL (p < 0.0001) and overestimating symptom interference was associated with lower patient physical QOL (p < 0.05). Patient and caregiver mental QOL was associated with caregiver underestimating (p < 0.05) and overestimating (p < 0.05) symptom severity.ConclusionThe psychosocial context of the family plays an important role in the accuracy of symptom understanding. Inaccurately understanding patients experience is related to poor QOL for both patients and caregivers, pointing to important targets for symptom management interventions that involve family caregivers.

Linking 24-hour movement behavior guidelines to academic engagement and social-emotional functioning among youth with chronic pain

BackgroundGrowing evidence documents the health benefits of meeting the 24-h movement behavior (24-HMB) guidelines (including recommendations concerning physical activity [PA], screen time [ST], and sleep [SL]) regardless of age or health status. However, the association between meeting the 24-HMB guidelines and academic engagement, social functioning, and emotional functioning has not been investigated in children and adolescents (youth) with chronic pain. Therefore, this cross-sectional study examined the potential links between meeting the 24-HMB guidelines and indicators of the above-mentioned outcomes in a nationally representative sample of U.S. youth with chronic pain. MethodsData from caregiver proxy reports for 7918 U.S. children and adolescents with chronic pain, aged between 6 and 17 years, were retrieved from the 2020 National Survey of Children's Health. Exposures of interest included the isolated and combined 24-HMB guidelines, while outcomes included measures of academic engagement, social functioning, and emotional functioning. To determine the association between the variables mentioned above, a logistic regression analysis was conducted using adjustment for confounding variables (e.g., age, sex, ethnicity, household federal poverty level [FPL] status, education level of primary caregivers, premature birth, and overweight). ResultsA low (4.53 %) proportion of U.S. youth with chronic pain met the combined 24-HMB guidelines. As compared to the reference group (i.e., did not meet the 24-HMB guidelines), youth with chronic pain who met the combined 24-HMB guidelines (referring to PA + ST, PA + SL, PA + ST + SL) showed significantly better performance on our measures of interest (e.g., learning interest/curiosity, difficulty in making friends, resilience; p < 0.05 for all). ConclusionsIn youth with chronic pain, meeting the 24-HMB guidelines is associated with better academic engagement, social functioning, and emotional functioning. However future intervention studies are necessary to provide more robust evidence before more firm conclusions can be drawn. Our results suggest that fostering a healthy lifestyle behavior in this vulnerable population might support their social-emotional and cognitive development (e.g., academic engagement).

Discrepancies between self- and proxy-rated quality of life in people living with dementia.

The aim of this study was to analyze discrepancies between self- and proxy-rated health-related quality of life (HRQoL), measured with the EuroQol 5 Dimension 5 Level survey (EQ-5D-5L), in people living with dementia (PlwD) and their caregivers on an individual response level. EQ-5D-5L, sociodemographic and clinical data were obtained from baseline data of n = 174 dyads of a cluster-randomized, controlled intervention trial. Self- and proxy-rated EQ-5D-5L health profiles were evaluated in terms of response distribution and agreement (weighted Kappa), and discrepancies in individual dimension level were analyzed using the Paretian Classification of Health Change (PCHC) as well as the presence and degree of inconsistencies between ratings. PlwD had a mean age of 80.1, nearly the half were female and 82.3% were mildly to moderately cognitively impaired. PlwD reported a higher utility index than caregiver proxies (mean 0.75vs. 0.68, 83% of PlwD>0.5). According to the PCHC and inconsistency approach, 95% of PlwD rated their health differently compared to proxies; 66% with divergent responses in at least three EQ-5D-5L dimensions. Nine dyads (5%) showed identical ratings. Discrepancies of one higher or lower EQ-5D-5L response represented the most frequent discrepancy (35.4%). Caregivers were two times more likely to report "moderate problems," representing the middle of the 5-point Likert scale. Usual activities had the lowest agreement between ratings (weighted kappa = 0.23). In PlwD reporting no or some problems in EQ-5D-5L-dimensions, proxies were more likely to report more problems and vice versa, especially in the more observable dimension usual activities and less likely in the less observable domains pain/discomfort and anxiety/depression. The central tendency bias observed in proxy-ratings could be associated with assessment uncertainties, resulting in an underestimation (overestimation) in PlwD reporting better (worse) health. This diverging trend extends the knowledge from previous studies and underlines the need for more methodological research in this area. People living with dementia (PlwD) rate their health differently than proxies.Proxy-ratings over- or underestimate PlwD health when self-ratings are low or high.Proxies indicate a possible central tendency bias.Further research is needed to understand influencing factors.

Household income and health-related quality of life in children receiving treatment for acute myeloid leukemia: Potential impact of selection bias in health equity research.

Examine the influence of household income on health-related quality of life (HRQOL) among children with newly diagnosed acute myeloid leukemia (AML). Secondary analysis of data prospectively collected from pediatric patients receiving treatment for AML at 14 hospitals across the United States. Household income was self-reported on a demographic survey. The examined mediators included the acuity of presentation and treatment toxicity. Caregiver proxy reported assessment of patient HRQOL from the Peds QL 4.0 survey. Children with AML (n = 131) and caregivers were prospectively enrolled to complete PedsQL assessments. HRQOL scores were better for patients in the lowest versus highest income category (mean ± SD: 76.0 ± 14 household income <$25,000 vs. 59.9 ± 17 income ≥$75,000; adjusted mean difference: 11.2, 95% CI: 2.2-20.2). Seven percent of enrolled patients presented with high acuity (ICU-level care in the first 72 h), and 16% had high toxicity (any ICU-level care); there were no identifiable differences by income, refuting mediating roles in the association between income and HRQOL. Enrolled patients were less likely to be Black/African American (9.9% vs. 22.2%), more likely to be privately insured (50.4% vs. 40.7%), and more likely to have been treated on a clinical trial (26.7% vs. 18.5%) compared to eligible unenrolled patients not enrolled. Evaluations of potential selection bias on the association between income and HRQOL suggested differences in HRQOL may be smaller than observed or even in the opposing direction. While primary analyses suggested lower household income was associated with superior HRQOL, differential participation may have biased these results. Future studies should partner with patients/families to identify strategies for equitable participation in clinical research.

The Effect of Outdoor Play, Physical Activity, and Screen Time Use on the Emotional Wellbeing of Children and Youth during a Health Crisis

Long-standing research has highlighted the critical role of physical and outdoor activities for children’s health and wellbeing. During the COVID-19 pandemic, children around the world experienced heightened stress and poor mental health, which coincided with a decrease in active play and increase in screen-based activities. This study examined how physical, outdoor, and screen-based activities impacted emotional wellbeing during the post-lockdown period of COVID-19. Caregiver proxy report of children’s activities and emotional wellbeing was collected from 190 caregivers through an online survey in February-April 2021. Overall, we observed low levels of physical and outdoor activities and high levels of screen activities in children, especially those aged 5 and older. Unadjusted models suggested that time spent in physical activity and online socializing predicted child wellbeing positively and negatively, respectively. However, only child age, parent-child co-play, parent stress, and family life satisfaction emerged as significant predictors of child wellbeing in the fully adjusted model. The findings facilitate a deeper understanding of children and youth’s outdoor engagement during the pandemic and highlight the critical role of parents and families in supporting children’s emotional wellbeing and healthy recreation under sustained stress and disruption.

Health-related quality of life in patients with diverse rare diseases: An online survey

PurposeRare diseases substantially contribute to population morbidity and mortality. Understanding rare disease health-related quality of life (HRQL) is essential for evaluating platform-based interventions that aim to tackle multiple rare diseases at a time. However, most HRQL studies focus on single or select group of rare diseases, often in a single country. Our study aimed to identify patient- and disease-specific correlates of HRQL across diverse rare diseases. MethodsWe conducted an international online survey of rare disease patients and caregiver proxies affected by a systematically identified sample of rare diseases. We calculated EQ-5D scores and conducted multivariate linear regression to examine sociodemographic and disease predictors of EQ-5D-5L visual analog scale (VAS) and utility scores (United States only). ResultsA total of 1053 individuals affected by 103 different rare diseases participated, including 660 patients and 393 caregiver proxies. Disability status and disease prevalence correlated with poorer HRQL across models (P < .05). Increased pain and decreased ability to perform usual activities also correlated with lower VAS for both adult patients and caregiver proxies (P < .05). Being unemployed approached significance as a correlate of both lower caregiver proxy VAS and lower patient utility scores. ConclusionOur results suggest that across rare diseases, lower HRQL is associated with a reduced rare disease prevalence and disability status, among other predictors. Understanding the key correlates of HRQL is essential for developing interventions for improving health care delivery and quality of life for rare disease patients and families.

Inter-reporter differences in symptom burdens in Japanese children with cancer.

Recent studies about inter-reporter differences and patient-reported outcomes (PROs) in childhood cancer from Western countries showed that caregiver proxy reports tend to overestimate symptom burdens in comparison with children's self-reports. However, the results from Western countries may not be generalizable to Asian countries. This paper is a secondary analysis of a validation study of the Japanese pediatric version of the Memorial Symptom Assessment Scale including 88 dyads of children aged 7-12 years and 74 dyads of children aged 13-18 years and their caregivers. The study assessed the inter-reporter differences of eight and 31 symptom burdens calculated as symptom scores in children aged 7-12 years and 13-18 years, respectively, and the association between inter-reporter differences and the characteristics of children and caregivers. The majority of children and caregivers scored equally at the dyadic level for almost all symptoms. However, 37.5% of symptoms in children aged 7-12 years and 10.0% of symptoms in children aged 13-18 years showed significant inter-reporter differences, suggesting a general tendency of caregivers to underestimate their children's symptom burden. The caregiver's age was the characteristic most frequently associated with magnitude of inter-reporter differences. Caregiver proxy reports may be a reliable source of PROs in Japanese children with cancer, as self-reported and caregiver proxy-reported symptom burdens were generally concordant. However, as some significant inter-reporter differences were observed, an effort should be made within the medical community to evaluate the parent-child relationship to minimize inter-reporter differences and achieve better symptom management.

Associations of 24-hour movement behaviors with externalizing and internalizing problems among children and adolescents prescribed with eyeglasses/contact lenses

BackgroundEmerging evidence points towards the psychological benefits of meeting 24-hour movement behavior (24-HMB) guidelines, but such associations have not yet been investigated among children and adolescents of prescribed eyeglasses/contact lenses. To this end, we examined associations of meeting 24-HMB guidelines with internalizing and externalizing challenges in this population. MethodsWe used data from the 2021 National Survey of Children's Health, a cross-sectional survey including a representative sample of US children and adolescents. Data on movement behaviors (physical activity [PA], sedentary behavior operationalized via screen time [ST], and sleep duration [SL]) and internalizing and externalizing problems were collected through caregiver proxy reports. Caregivers completed questionnaires for 6030 (2799 girls) US children and adolescents of prescribed eyeglasses/contact lenses. Logistic regression analyses were conducted to determine the above-presented associations. ResultsOnly 7.1 % of those prescribed eyeglasses/contact lenses met all three 24-HMB guidelines, while they were more likely to meet SL guideline alone (32.1 %) in relation to other independent guidelines including PA (2.5 %) and ST (10.9 %). Compared to not meeting any of the three 24-HMB guidelines, meeting at least two guidelines (25.22 %) was significantly linked to lower odds of internalizing problems and externalizing problems. ConclusionMeeting at least two components of the 24-HMB guidelines was beneficially linked to internalizing and externalizing problems. Thus, strategies or intervention programs that focus on meeting 24-HMB guidelines should be implemented among children and adolescents of those prescribed eyeglasses/contact lenses to foster coping with psychological issues.

Self and proxy symptom reporting in patient-caregiver dyads coping with a primary brain tumor: The role of psychosocial function in rating agreement.

223 Background: Patients with glioma experience high levels of symptom burden with often sudden performance declines. As such, family caregivers tend to report on the patient’s symptoms; yet previous research examining the validity of proxy symptom reporting points to inconsistent findings. While prior studies focused on identifying patient clinical variables that may be related to agreement, caregiver characteristics may also be important for accurately perceiving patient symptoms. We examined the associations between symptom agreement and patient and caregiver perceived illness communication, depressive symptoms, and QOL. Methods: As part of a behavioral intervention trial, baseline assessments were collected prior to randomization in 67 dyads. We report on patient symptom burden (MDASI-BT) and patient and caregiver depressive symptoms (CES-D), illness communication (CICS), and QOL (SF-36). Paired t-tests, difference scores and inter-correlations coefficients (ICCs) were used to test for agreement, accuracy and congruence in proxy symptom scores, respectively. Bivariate correlations were calculated to examine the association between psychosocial function variables and symptom agreement. Dyadic level associations between symptom agreement and QOL were examined using multi-level modeling. Results: Thirty-nine percent of patients (63% male; 93% non-Hispanic White; mean age=47 years; 84% Grade III-IV) and 49% of caregivers (79% female; 90% non-Hispanic White; mean age=51 years) endorsed clinical levels of depression. Clinically significant disagreement was found for all means scores of the MDASI-BT subscales except for GI symptoms and general symptoms. 22% of caregivers overestimated and 13% underestimated overall symptom severity and 32% overestimated and 21% underestimated symptom interference. ICCs of patient and caregiver proxy ratings were significant (except cognitive and GI subscales) ranging from small to moderate coefficients (.09 - .50). Patient illness communication was associated with agreement in overall symptom severity (r=-.27, p=.03) and affective symptom subscale (r=-.34, p&lt;.01). Caregiver illness communication (r=-.33, p&lt;.01) and depressive symptoms (r=.46, p&lt;.0001) were associated with symptom interference agreement. Caregiver overestimation of patient symptom severity was significantly associated with poor caregiver physical QOL, while underestimation was significantly associated with poor patient physical and mental QOL and poor caregiver mental QOL. Conclusions: The psychosocial context of the family plays an important role in the accuracy of proxy symptom reporting. Moreover, inaccurately understanding patients' experience is related to poor QOL for both patients and caregivers, which points to important targets for symptom management interventions that involve family caregivers.

Community Teens' COVID-19 Experience: Implications for Engagement Moving Forward.

Data collected from pediatric primary care settings during the pandemic suggest an increase in internalizing symptoms and disparities in care based upon minoritized identity status(es). To inform care moving forward, the current study characterized the pandemic and related technology usage experiences of teenaged pediatric patients from communities with high hardship indexes. As part of a larger mixed-methods study, 17 teens (Mean age = 15.99 ± .99) and 10 caregivers independently voiced experiences related to the pandemic during remote focus group and interview sessions. Thematic analyses were used to assess qualitative data; descriptive analyses were used to characterize qualitative data. Despite no direct queries about the pandemic, 41% of teens and 40% of caregivers described their lived experiences during the pandemic. Two subthemes emerged within the primary theme of COVID-19: (1) Wellness/Mental Health and (2) Smartphone Use and Utility. Although distress and negative effects were voiced, questionnaire data indicated normative psychosocial functioning for both teen self-report and caregiver proxy report. Informed by the voiced experiences of teens and their caregivers from communities with high hardship indexes, methods for better assessing and managing internalizing symptoms in teen patients are presented. A multi-modal and multi-informant approach that leverages technology to garner information about teens' experiences and deliver care may help improve the well-being of teens in communities systemically burdened with disparities.

Comparative analysis of pediatric anxiety measures in clinical sample: evaluation of the PROMIS pediatric anxiety short forms.

Examine the psychometric properties, validity in relation to a legacy measure, and diagnostic accuracy of the PROMIS Anxiety Short Form 2.0 (PROMIS A-SF) Caregiver and Youth Reports in a clinical sample. Participants were 301 youth and caregivers referred to a behavioral health clinic by their pediatrician. Participants and their caregivers completed PROMIS A-SF (youth and caregiver proxy), SCARED (youth and caregiver proxy), and a semi-structured interview. Descriptive, correlational, test-retest reliability, and receiver operating characteristic (ROC) analyses were conducted for both measures. PROMIS A-SF measures were highly correlated with SCARED total scores and the panic subscale. PROMIS A-SF measures had AUCs ranging from .49-.79 for the detection of any of three primary subtypes of anxiety: Generalized Anxiety, Separation Anxiety, and Social Anxiety. Dimensional anxiety subtypes, such as Social Anxiety may not be well detected on the PROMIS youth measure. Use of the PROMIS A-SF as a part of Evidence Based Assessment process is discussed.

HOW CAN PATIENT PORTALS SUPPORT CAREGIVERS PROVIDING END-OF-LIFE CARE?: A MIXED-METHODS STUDY

Abstract Patient portal design is rarely centered around patient caregivers—family members, friends and other designated persons who may access these digital tools for the patient’s health needs. This study examines: (a) portal features used by caregivers during their loved-one’s end-of-life; and (b) caregiver perceptions regarding the use of patient portals for caregiving near the end-of-life.Using sequential mixed-methods, we conducted a retrospective cohort analysis of caregiver proxy (N=137) use from 2016-2019 during their loved-ones’ (N=5,284) last 12 months of life and interviews with (N=31) 16-former and 15-current caregiver proxies. Caregiver proxy portal use in this population was lower than expected; however, caregivers who used the portal as proxies found value in tools for caregiving near the end-of-life. To leverage the patient portal to better support caregivers, future strategies should target caregiver portal adoption, awareness of beneficial portal tools, and the addition of caregiver specific resources and education.

Children and caregiver proxy quality of life from peanut oral immunotherapy trials.

Health-related quality of life (HRQoL) is significantly and substantially reduced in individuals with peanut allergy due to many factors associated with unanticipated or potentially fatal reactions. Further insight on the impact of peanut oral immunotherapy in managing peanut allergy on HRQoL is needed. The aim of this analysis was to assess effects of peanut (Arachis hypogaea) allergen powder-dnfp (PTAH), a biologic drug for peanut oral immunotherapy, on HRQoL from three phase 3 and two follow-on trials of PTAH. HRQoL assessments from participants aged 4-17 in the PALISADE (ARC003), ARC004 (PALISADE follow-on), ARTEMIS (ARC010), RAMSES (ARC007), and ARC011 (RAMSES follow-on) trials were included in this analysis. Responses on the Food Allergy Quality of Life Questionnaire (FAQLQ) and Food Allergy Independent Measure (FAIM) were evaluated by age group and respondent (self or caregiver proxy). Data were analyzed with descriptive statistics and Student t tests. Baseline FAQLQ and FAIM total scores appeared comparable between PTAH- and placebo-treated participants. Self and caregiver proxy-reported total scores on the FAQLQ for PTAH-treated participants generally improved at trial exit versus baseline; FAIM total scores improved throughout all trials. The tendency for improvement in FAQLQ total scores from baseline for PTAH appeared larger in self versus caregiver proxy-reports. Between treatment groups, PTAH was generally favored in the PALISADE and ARTEMIS trials; differences varied in the RAMSES trial based on age and respondent types. PTAH for the management of peanut allergy in children appeared to have a beneficial effect on HRQoL in trials. Improvements were seen despite rigors of trial participation.

Influence of Household Income on Health-Related Quality of Life in Children with Acute Myeloid Leukemia

Introduction Treatment of pediatric acute myeloid leukemia (AML) requires multiple, consecutive cycles of intense chemotherapy with prolonged neutropenia and high risk for infectious complications. This treatment may have unique implications for patient health-related quality of life (HRQOL)-how patients perceive their disease impacting overall health and well-being. Patients and their families also experience significant economic burdens associated with disease and treatment. In particular, patients from socioeconomically vulnerable families disproportionately experience economic burden, which may subsequently worsen HRQOL. Few studies have explored the influence of household income (HHI) on HRQOL during AML therapy. Furthermore, the mechanisms through which HHI may influence HRQOL have not been described. To fill this gap, we examined the association between HHI and HRQOL and explored whether differences in presentation acuity or treatment toxicity mediate this association. Methods Secondary analyses were performed using data from a multi-institution, prospective cohort study in pediatric patients receiving treatment for newly diagnosed AML. Patients and their primary caregivers were recruited to complete prospective assessments of patient HRQOL, and a detailed survey providing sociodemographic information including annual HHI (<$25K; $25K-49K; $50K-74K; and >$75K). HRQOL was measured using caregiver proxy responses to the PedsQL 4.0 Generic Core Scales; overall score and separate scores for the psychosocial and physical health subscales were evaluated. Daily-level inpatient resource utilization (RU) data were obtained via a merge with the Pediatric Health Information System database. High acuity at initial presentation was defined as any ICU-level RU within the first 72 hours following first leukemia admission. High treatment toxicity was defined as any ICU-level RU requirement during treatment. Covariates including patient age at diagnosis, sex, race/ethnicity, risk classification, clinical trial enrollment, and inpatient vs outpatient neutropenia management were obtained from electronic medical record abstraction. Linear regression models compared HRQOL by categories of HHI (reference: HHI >$75K). Demographic and clinical characteristics were compared for patients who enrolled on the study and those who were eligible but were not enrolled to evaluate for potential selection bias. Results 225 eligible patients were identified from 14 institutions, and 60% of eligible patients enrolled on the study. Patients with lower HHI were generally younger, more likely to be non-White, their caregivers were less likely to be college educated and more likely to be unemployed compared to those with higher HHI (Table 1). Overall HRQOL scores were low (mean ±sd: 65.7 ±1.8). Patients in the lowest HHI category had higher (better) overall HRQOL as compared with patients in the highest HHI category (mean ± sd: 76.0 ±3.3 for HHI <$25K vs. 59.9 ±2.3 for HHI>$75K, adjusted mean difference: 11.2, 95% CI: 2.2-20.2). Subscale assessments revealed larger differences on the psychosocial domain than physical domain (Table 2). Overall, 6% of patients presented with high acuity and 16% had high toxicity; there were no clear trends in presentation acuity or toxicity by HHI (Table 2). HRQOL scores were comparable for patients with (62.0 ±19.5) and without high toxicity (66.2 ±18.2). Enrolled patients were less likely to be Black or African American (9.6% vs 20%), more likely to be privately insured (51.1% vs 42.2%), and more likely to have been treated on a clinical trial (27.4% vs 17.8%) than eligible patients who did not enroll on the study. Conclusion Patient HRQOL during AML therapy is low overall. Contrary to our hypothesis, lower HHI was associated with superior HRQOL, particularly in the psychosocial health domain. HRQOL did not differ by toxicity, and toxicity did not differ meaningfully by HHI, refuting a mediating role in this association. These findings could potentially indicate differences in perspective on HRQOL amidst cancer therapy. Future studies to qualitatively investigate patient- and caregiver-reported drivers of HRQOL among low and high-income cohorts are warranted. Given the observed differences in characteristics for enrolled versus unenrolled patients, we cannot rule out a potential selection bias. Formal quantitative bias adjustment is ongoing. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

HEALTH-RELATED QUALITY OF LIFE IN CHILDREN AND CAREGIVER PROXIES FROM TRIALS OF PEANUT ORAL IMMUNOTHERAPY

<h3>Introduction</h3> We assessed health-related quality of life (HRQoL) of pediatric participants across phase 3 and follow-on trials for peanut (<i>Arachis hypogaea</i>) allergen powder-dnfp (PTAH). <h3>Methods</h3> HRQoL data were analyzed from PALISADE (ARC003), ARC004 (PALISADE follow-on; daily dosing cohorts), ARTEMIS (ARC010), RAMSES (ARC007), and ARC011 (RAMSES follow-on) using the Food Allergy Quality of Life Questionnaire (FAQLQ) and Food Allergy Independent Measure (FAIM). Assessments were completed by caregiver proxies of participants aged 4-17 years and participants aged 8-17 years at prespecified time points. Scores were summarized descriptively, and mean changes were reported for between groups (PTAH vs placebo) and within groups (PTAH only) from the screening to exit visit. <h3>Results</h3> Baseline FAQLQ and FAIM mean total scores appeared comparable between placebo- and PTAH-treated participants. Self- and caregiver proxy-reported total scores on the FAQLQ and FAIM for PTAH-treated participants generally improved over time from baseline to exit throughout all trials (<b>Table</b>). PTAH was generally favored in PALISADE and ARTEMIS versus placebo. PTAH-treated participants reported statistically significant changes in scores for self-reported responses (mean change) versus placebo for domains relating to allergy avoidance and dietary restrictions (-1.46 for ages 8-12, ARTEMIS) and risk of accidental exposure (-1.44 for ages 8-12, ARTEMIS) on the FAQLQ, and death (-0.91 for ages 8-12, PALISADE; -1.29 for ages 13-17; ARTEMIS) and severe reaction (-0.77 for ages 8-12, RAMSES; -1.57 for ages 13-17, ARTEMIS) on the FAIM. <h3>Conclusion</h3> PTAH for peanut allergy management appeared to be associated with an improvement in reported HRQoL in children.

Patient Responses to the Term Pain Catastrophizing: Thematic Analysis of Cross-sectional International Data

Pain catastrophizing is understood as a negative cognitive and emotional response to pain. Researchers, advocates and patients have reported stigmatizing effects of the term in clinical settings and the media. We conducted an international study to investigate patient perspectives on the term pain catastrophizing. Open-ended electronic patient and caregiver proxy surveys were promoted internationally by collaborator stakeholders and through social media. 3,521 surveys were received from 47 countries (77.3% from the U.S.). The sample was mainly female (82.1%), with a mean age of 41.62 (SD 12.03) years; 95% reported ongoing pain and pain duration > 10 years (68.4%). Forty-five percent (n = 1,295) had heard of the term pain catastrophizing; 12% (n = 349) reported being described as a ‘pain catastrophizer’ by a clinician with associated high levels of feeling blamed, judged, and dismissed. We present qualitative thematic data analytics for responses to open-ended questions, with 32% of responses highlighting the problematic nature of the term. We present the patients’ perspective on the term pain catastrophizing, its material effect on clinical experiences, and associations with negative gender stereotypes. Use of patient-centered terminology may be important for favorably shaping the social context of patients’ experience of pain and pain care. PerspectiveOur international patient survey found that 45% had heard of the term pain catastrophizing, about one-third spontaneously rated the term as problematic, and 12% reported the term was applied to them with most stating this was a negative experience. Clinician education on patient-centered terminology may improve care and reduce stigma.

Pilot Randomized Control Trial of an App-Based CBT Program for Reducing Anxiety in Individuals with ASD without Intellectual Disability.

This study developed and tested the effectiveness of an app-based cognitive behavioral therapy (CBT) program in alleviating anxiety among adolescents and adults with autism without co-occurring intellectual disability. Thirty participants from 15 to 35years old were randomly assigned to either the intervention or waitlist control group, and self- and caregiver proxy report questionnaires were administered, accompanied by direct behavior observation before and after the intervention period. There was a significant decrease in anxiety level, an increase in positive affect, and a decline in stereotypic behaviors, hyperactivity, noncompliance, and inappropriate speech in proxy reports for the intervention group, compared to the control group. A significant rise in passive response in the direct observation was also seen in the intervention group.