Pediatric Patient-Reported Outcomes Research Articles

Health-related quality of life for children with anterior cruciate ligament deficiency: Ensuring content validity of the new KIDS-KNEES-ACL questionnaire.

The existing knee-specific pediatric patient-reported outcome measures (PROMs) lack content and construct validity for children with anterior cruciate ligament (ACL) injury. This impairs their accuracy which can lead to false interpretations of data and inaccurate clinical guidelines. The purpose of this study was to develop a content-valid PROM for children with an ACL injury. The process adhered to the COnsensus-based Standards for the selection of health Measurement INstruments guidelines for PROM development. Informants were children with ACL deficiency and sampled based on age, sex, and treatment. Semistructured interviews were conducted exploring themes within the International Classification of Functioning, Disability and Health model. Interviews continued beyond data saturation. By thematic analysis and by probing items from the adult PROM 'KNEES-ACL', new themes and items emerged. Content coverage, relevance and understandability were continuously evaluated. All interviews were recorded and transcribed verbatim. The NVivo 12 software was used for data analysis and coding of items. A PROM of 60 items across nine subscales was formed. From cognitive interviews, 19 new items emerged. Forty-one of 55 items from KNEES-ACL were endorsed as relevant; however, all required rewording to ensure understandability. Substantial differences in the psychosocial impact between adults and children were observed. The children experienced a more considerable negative psychosocial impact caused by a loss of participation in sports, lower self-confidence and loss of social networks. This resulted in four new domains. The physical issues were similar to adults, with few exceptions. The 'KIDS-KNEES-ACL' 1.0 (qualitative version) was developed. This version will be subjected to psychometric analysis, resulting in adequate measurement properties of the final KIDS-KNEES-ACL 2.0. As the only adequate pediatric ACL-specific PROM, its use in clinical trials and databases will enhance PROM data quality, and strengthen clinical guidelines and thus the treatment of children with ACL injury. Not applicable.

Multi-omics analysis of the gut microbiome and metabolites associated with the psychoneurological symptom cluster in children with cancer receiving chemotherapy

BackgroundChildren with cancer receiving chemotherapy commonly report a cluster of psychoneurological symptoms (PNS), including pain, fatigue, anxiety, depression, and cognitive dysfunction. The role of the gut microbiome and its functional metabolites in PNS is rarely studied among children with cancer. This study investigated the associations between the gut microbiome–metabolome pathways and PNS in children with cancer across chemotherapy as compared to healthy children.MethodsA case–control study was conducted. Cancer cases were recruited from Children’s Healthcare of Atlanta and healthy controls were recruited via flyers. Participants reported PNS using the Pediatric Patient-Reported Outcomes Measurement Information System. Data for cases were collected pre-cycle two chemotherapy (T0) and post-chemotherapy (T1), whereas data for healthy controls were collected once. Gut microbiome and its metabolites were measured using fecal specimens. Gut microbiome profiling was performed using 16S rRNA V4 sequencing, and metabolome was performed using an untargeted liquid chromatography–mass spectrometry approach. A multi-omics network integration program analyzed microbiome–metabolome pathways of PNS.ResultsCases (n = 21) and controls (n = 14) had mean ages of 13.2 and 13.1 years. For cases at T0, PNS were significantly associated with microbial genera (e.g., Ruminococcus, Megasphaera, and Prevotella), which were linked with carnitine shuttle (p = 0.0003), fatty acid metabolism (p = 0.001) and activation (p = 0.001), and tryptophan metabolism (p = 0.008). Megasphaera, clustered with aspartate and asparagine metabolism (p = 0.034), carnitine shuttle (p = 0.002), and tryptophan (p = 0.019), was associated with PNS for cases at T1. Gut bacteria with potential probiotic functions, along with fatty acid metabolism, tryptophan, and carnitine shuttle, were more clustered in cancer cases than the control network and this linkage with PNS needs further studies.ConclusionsUsing multi-omics approaches, this study indicated specific microbiome–metabolome pathways linked with PNS in children with cancer across chemotherapy. Due to limitations such as antibiotic use in cancer cases, these findings need to be further confirmed in a larger cohort.

Patient-Reported Outcomes Measurement Information System Pediatric Symptom Profiles of Children With Cancer in China: A Latent Profile Analysis.

Unpleasant symptoms are common in children with cancer. However, research identifying subgroups of children with cancer who experience similar levels of self-reported symptoms in China is limited. This study aimed to classify the symptom profiles of children with cancer and detect the possible predictors of the profiles and their effect on children's quality of life (QoL). A total of 272 children aged 8 to 17 years completed the Chinese version of the Pediatric Patient-Reported Outcomes Measurement Information System short form measures, the Pediatric QOL Inventory general core and cancer modules. Latent profile analysis was used to identify symptom profiles, and ordinal logistic regression and analysis of variance were used to examine predictors of symptom profile membership and profile differences on QoL. The best fit was a 3-profile model: low, moderate, and severe symptom distress. Children who had been inpatients in the past 7 days and were currently under treatment are more likely to have severe symptoms. Participants in the low symptom distress profile reported significantly greater QoL than those in the other profiles. Children with cancer are heterogeneous in their experience of symptoms. Children's characteristics, such as inpatient history and treatment status, are predictors of profiles; different symptom profiles are associated with QoL. This study identified distinct groups of patients who predictably experience higher symptoms and their predictors, which could help to place children within a profile and perhaps allow nurses to provide targeted supportive care to match children's specific symptom profile.

Uptake of pediatric patient-reported outcome and experience measures and challenges associated with their implementation in Alberta: a mixed-methods study

BackgroundImplementing Patient-reported Outcome Measures (PROMs) and Patient-reported Experience Measures (PREMs) is an effective way to deliver patient- and family-centered care (PFCC). Although Alberta Health Services (AHS) is Canada's largest and fully integrated health system, PROMs and PREMs are yet to be routinely integrated into the pediatric healthcare system. This study addresses this gap by investigating the current uptake, barriers, and enablers for integrating PROMs and PREMs in Alberta's pediatric healthcare system.MethodsPediatric clinicians and academic researchers with experience using PROMs and PREMs were invited to complete a quantitative survey. Additionally, key stakeholders were qualitatively interviewed to understand current challenges in implementing pediatric PROMs and PREMs within AHS. Quantitative data gathered from 22 participants were descriptively analyzed, and qualitative data from 14 participants were thematically analyzed.ResultsParticipants identified 33 PROMs and 6 PREMs showing diversity in the types of pediatric PROMs and PREMs currently being used in Alberta and their mode of administration. The qualitatively identified challenges were associated with patients, family caregivers, and clinicians. The absence of system-level support, such as integration within electronic medical records, is considered a significant system-level challenge.ConclusionsThe significant variation in the types of PROMs and PREMs used, the rationale for their use, and their mode of administration demonstrate the diverse and sporadic use of these measures in Alberta. These findings highlight the need for province-wide uniform implementation of pediatric PROMs and PREMs in Alberta. Our results could benefit healthcare organizations in developing evidence-based PROM and PREM implementation strategies in pediatrics.

Swahili translation and cultural adaptation of the pediatric patient-reported outcomes version of the common terminology criteria for adverse events (PRO-CTCAE)

BackgroundThe pediatric patient-reported outcomes version of the common terminology criteria for adverse event measure was developed and validated for use in pediatric cancer clinical trials to better capture the symptom experiences through direct self-report. The study aim was to develop and validate a Swahili language version of the patient-reported outcomes version of the common terminology criteria for adverse event measure.MethodsThe pediatric version of 15 core symptom adverse events, and the corresponding questions, were selected from the patient-reported outcomes version of the common terminology criteria for adverse event library, then forward and back translated into Swahili by bilingual translators. The translated items were further refined using concurrent cognitive interviewing. Each round of interviews included five children, ages 8–17 years-old, receiving cancer therapy at Bugando Medical Centre, the cancer referral hospital for Northwest Tanzania, and continued until at least 80% of participants understood the question.ResultsThree rounds of cognitive interviews were completed involving 13 patients and 5 caregivers. Among patients, 50% of questions (19/38) were fully comprehended after the first interview round. Two Adverse Events (anxiety and peripheral neuropathy) were the most difficult for participants to understand, associated with education level and experience. Goal comprehension was achieved after three rounds of interviews with no further revisions required. All parents in the first cognitive interview group comprehended the survey, with no additional revisions.ConclusionA Swahili patient-reported outcomes version of the common terminology criteria for adverse event was effective in eliciting patient-reported Adverse Events related to cancer treatment, with good comprehension for children aged 8–17 years. This survey is important to incorporate patient self-reporting of symptomatic toxicities and is an effective tool to increase capacity for pediatric cancer clinical trials throughout East Africa, further reducing global disparities in cancer care.

Ethical and practical considerations related to data sharing when collecting patient-reported outcomes in care-based child health research

PurposeThe collection and use of patient reported outcomes (PROs) in care-based child health research raises challenging ethical and logistical questions. This paper offers an analysis of two questions related to PROs in child health research: (1) Is it ethically obligatory, desirable or preferable to share PRO data collected for research with children, families, and health care providers? And if so, (2) What are the characteristics of a model best suited to guide the collection, monitoring, and sharing of these data?MethodsA multidisciplinary team of researchers, providers, patient and family partners, and ethicists examined the literature and identified a need for focus on PRO sharing in pediatric care-based research. We constructed and analyzed three models for managing pediatric PRO data in care-based research, drawing on ethical principles, logistics, and opportunities to engage with children and families.ResultsWe argue that it is preferable to share pediatric PRO data with providers, but to manage expectations and balance the risks and benefits of research, this requires a justifiable data sharing model. We argue that a successful PRO data sharing model will allow children and families to have access to and control over their own PRO data and be engaged in decision-making around how PROs collected for research may be integrated into care, but require support from providers.ConclusionWe propose a PRO data sharing model that can be used across diverse research settings and contributes to improved transparency, communication, and patient-centered care and research.

Subjective Toxicity Profiles of Children With Cancer During Treatment: A Latent Class Analysis.

Children and adolescents may experience a variety of subjective adverse events (AEs) caused by cancer treatment. The identification of distinct groups of patients is crucial for guiding symptomatic AE management interventions to prevent AEs from worsening. The aim of this study was to identify subgroups of children with cancer experiencing similar patterns of subjective toxicities and evaluate differences among these subgroups in demographic and clinical characteristics. A cross-sectional survey was conducted of 356 children in China with malignancies who received chemotherapy within the past 7 days using the pediatric Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events. A latent class analysis (LCA) was conducted to identify subgroups of patients with distinct profiles of symptomatic AE occurrence. Nausea (54.5%), anorexia (53.4%), and headache (39.3%) were the top 3 AEs children experienced. Nearly all participants (97.8%) experienced ≥1 core AEs, and 30.3% experienced ≥5 AEs. The LCA results identified 3 subgroups ("high gastrotoxicity and low neurotoxicity" [53.2%], "moderate gastrotoxicity and high neurotoxicity" [23.6%], and "high gastrotoxicity and high neurotoxicity" [22.8%]). The subgroups were differentiated by monthly family per-capita income, time since diagnosis, and Karnofsky Performance Status score. Children experienced multiple subjective toxicities during chemotherapy, especially gastrotoxicity and neurotoxicity. Heterogeneity was found in the LCA in the patients' toxicities. The prevalence of toxicities could be distinguished by the children's characteristics. The results showing different subgroups in our study may assist clinical staff in focusing on patients with higher toxicities to provide effective interventions.

Translation and Validation of the Chichewa Pediatric Patient-Reported Outcome CTCAE Tool to Measure Treatment-Related Adverse Events Among Adolescent and Young Adult Lymphoma Patients in Malawi (TH114B)

Translation and Validation of the Chichewa Pediatric Patient-Reported Outcome CTCAE Tool to Measure Treatment-Related Adverse Events Among Adolescent and Young Adult Lymphoma Patients in Malawi (TH114B)

Association of Multimodal Pain Control with Patient-Reported Outcomes in Children Undergoing Surgery

IntroductionOur aim was to describe practices in multimodal pain management at US children's hospitals and evaluate the association between non-opioid pain management strategies and pediatric patient-reported outcomes (PROs). MethodsData were collected as part of the 18-hospital ENhanced Recovery In CHildren Undergoing Surgery (ENRICH-US) clinical trial. Non-opioid pain management strategies included use of preoperative and postoperative non-opioid analgesics, regional anesthetic blocks, and a biobehavioral intervention. PROs included perioperative nervousness, pain-related functional disability, health-related quality of life (HRQoL). Associations were analyzed using multinomial logistic regression models. ResultsAmong 186 patients, 62 (33%) received preoperative analgesics, 186 (100%) postoperative analgesics, 81 (44%) regional anesthetic block, and 135 (73%) used a biobehavioral intervention. Patients were less likely to report worsened as compared to stable nervousness following regional anesthetic block (relative risk ratio [RRR]:0.31, 95% confidence interval [CI]:0.11–0.85), use of a biobehavioral technique (RRR:0.26, 95% CI:0.10–0.70), and both in combination (RRR:0.08, 95% CI:0.02–0.34). There were no associations of non-opioid pain control modalities with pain-related functional disability or HRQoL. ConclusionUse of postoperative non-opioid analgesics have been largely adopted, while preoperative non-opioid analgesics and regional anesthetic blocks are used less frequently. Regional anesthetic blocks and biobehavioral interventions may mitigate postoperative nervousness in children. Level of evidenceIII.

Interpreting Patient-Reported Outcome Scores: Pediatric Inflammatory Bowel Disease as a Use Case.

To demonstrate how to interpret Patient-Reported Outcomes Measurement Information System (PROMIS) pediatric patient-reported outcome measure (PROM) scores for patients with pediatric inflammatory bowel disease (IBD). Using data from a prospective cohort study of patients ages 8 to 23 years with IBD (n=1049), we established disease-specific percentiles and computed the minimal clinically important difference (MCID) change score for 6 pediatric PROMs. We applied these results, general population percentiles, and the reliable change index to interpret PROM scores in a clinical trial sample of patients ages 8 to 20 years with IBD (n=294) in which PROMIS PROMs were obtained at baseline and 3 months later. Application of general population percentiles showed that the clinical trial sample at baseline had moderately worse self-reported health than the general population (22% of patients at or above the 95th percentile on Fatigue; 21% on Pain Interference). IBD-specific percentiles showed that the sample was somewhat worse than the reference IBD sample (8% of patients at or above the 95th percentile on Fatigue; 11% on Pain Interference). Application of the MCID threshold indicated that among the subgroup of patients that improved by 15 or more on the short Pediatric Crohn's Disease Activity Index (n=38), 45% also improved on IBD Symptoms, 47% for Fatigue, and 65% for Pain Interference. This study established IBD-specific percentiles for 6 pediatric PROMIS measures and demonstrated the application of percentiles and other methods for interpreting PROM scores.

Psychometric properties and CAT performance of the PROMIS pediatric sleep disturbance, sleep-related impairment, and fatigue item banks in Dutch children and adolescents.

This study's aim is to evaluate the psychometric properties of the pediatric Patient-Reported Outcomes Measurement Information System (PROMIS) sleep disturbance (SD), sleep-related impairment (SRI), and fatigue item banks in Dutch children and adolescents since they are yet to be validated in this population. Children and adolescents aged 8-18 years old (n = 1,325), representative of the Dutch general population, were invited. The following psychometric properties of both item banks were assessed: structural validity by fitting a graded response model (GRM) for which assumptions were checked and assessing item fit, reliability, and efficiency for the full-length item bank, short forms, and computerized adaptive test (CAT). Mean T scores and cutoff scores for mild, moderate, and severe scores were determined. A total of 527 children (response rate 39.7%) were included. SD did not meet the assumption of unidimensionality. The SRI and fatigue item banks showed sufficient structural validity. Three items showed GRM misfit; SRI item w026c and fatigue items 3224R1r and 4191R1r. Both item banks measured reliable (> .90) at the mean of the population and 2SD in the clinical direction. CATs outperformed short forms in efficiency. Mean T scores for the Dutch population were: SRI 47.5 (SD = 10.0) and fatigue 39.8 (SD = 12.4). Cutoffs included SRI minimal ≥ 34.7, moderate ≥ 55.6, severe ≥ 63.2 and fatigue minimal ≥ 23.9, moderate ≥ 49.9, severe ≥ 61.3. PROMIS pediatric SRI and fatigue item banks, short forms, and CAT showed sufficient structural validity and reliability in the Dutch population. Dutch reference values are provided. More research is needed for the PROMIS SD item bank. (PsycInfo Database Record (c) 2022 APA, all rights reserved).

The Effects of Lower Extremity Rotational Malalignment on Pediatric Patient-reported Outcomes Measurement and Information System (PROMIS) Scores.

Background:There is sparse literature demonstrating the relationship between lower limb pediatric idiopathic rotational malalignment (IRM) and patient-reported outcomes measurement information system (PROMIS) scores. Our goal is to determine and quantify the amount that IRM deformities, as measured with the 3D gait analysis, affect childrens’ pain interference, mobility, and peer relationship PROMIS domains. Secondary outcomes include investigating the potential relationships between IRM and various subgroups (Pediatric Outcomes Data Collection Instrument (PODCI), gender, Body Mass Index (BMI), femur Versus tibia). We also examine whether the PROMIS domains correlate with PODCI in this population.Methods:This study was a retrospective cohort, single institution, and consecutively recruited cases series. We identified 47 children over a 3-year period who were evaluated at the motion analysis center at our tertiary care hospital, with increased torsion of the femur or tibia. After exclusions, 25 children with IRM, documented PROMIS data and gait analysis were considered.Results:Femoral malrotation had a significant relationship with female gender (P=0.001) and increased BMI (P<0.001). Femoral malrotation had a correlation with PROMIS pain interference (P=0.016), whereas tibial rotation did not achieve significance (P=0.084). In the ANOVA regression analysis, there was a strong prediction of the PROMIS mobility domain when both malrotation and pain interference were present (P=0.007). There were Pearson Correlations of PROMIS and PODCI domains for Mobility Versus Sports (P=0.007) and Pain Interference Versus Comfort/Pain (P=0.004), respectively.Conclusion:The evident relationship between lower limb rotational malalignment and PROMIS scores signifies the likelihood for gait and pain disturbance. This in turn could show us that children are likely to be more debilitated and thus may benefit from timely correction. We aim to conduct this as a multicentre trial to confirm our findings.Level of Evidence:Level IV.

Pediatric Patient-Reported Outcomes in Chronic Disease

Managing chronic disease is increasingly important in pediatric health care. In internal medicine, patient-reported, patient-important outcomes are a key part of assessment of programs. In pediatrics, there has been less focus on patient-reported outcomes. These outcomes are more complex in pediatrics because the utility of patient-reported outcomes may differ by age and, in most cases, parent-reported outcomes are also relevant. In some instances, parents serve as a surrogate for the pediatric patient. In other circumstances, parent-reported outcomes may differ from child-reported outcomes. In this volume of the Journal, Forrest et al report on self-reported child health outcomes across 10 chronic conditions. Across all these conditions, children reported more anxiety, fatigue, pain, and restriction of mobility than reported in the general pediatric population. Disease activity was associated with poorer self-reported outcomes. These results emphasize the need to incorporate patient-reported outcomes in pediatric chronic disease management. Programs will need to develop strategies to improve these patient-reported outcomes. Article page 207

Translation Into Simplified Chinese and Cultural Validation of the Pediatric Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events Using Cognitive Interviewing.

The original English Pediatric Patient-Reported Outcome version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) captures symptomatic adverse events (AEs) in cancer clinical trials from the perspective of pediatric patients. A Chinese version was needed to encourage the use of the Pediatric PRO-CTCAE among Chinese pediatric oncology patients. This study translated and linguistically validated a simplified Chinese version of the Pediatric PRO-CTCAE for oncological patients aged 7 to 18 years. Following the Functional Assessment of Chronic Illness Therapy translation methodology, 130 questions were translated into Chinese. Semistructured cognitive interviews investigated the comprehensibility and clarity of terms for symptoms, attributes, and response options. Two rounds of interviews were conducted with 48 native Chinese-speaking children aged 7 to 18 years who were undergoing chemotherapy or radiotherapy treatment. Most items, response options, and recall periods were well understood by children across the age range in round 1. Nineteen items posed comprehension difficulties for 9 participants and were revised and retested without further difficulties. The Pediatric PRO-CTCAE was successfully developed and linguistically validated among Chinese oncology patients. The results indicated that the Chinese Pediatric PRO-CTCAE was semantically and conceptually equivalent to the English version. The availability of the simplified Chinese Pediatric PRO-CTCAE will facilitate the generation of patient-reported outcome data about symptomatic AEs for children with cancer in China and thus improve our understanding of children's experience of treatment-related symptoms.

COVID-19 Pandemic Associated With Increased Self-reported Depressive Symptoms in Patients With Congenital Craniofacial Diagnoses.

ObjectiveThe current study investigated the influence of the coronavirus (COVID-19) pandemic on patients with congenital craniofacial diagnoses.MethodsPatients (n = 66) with craniofacial diagnoses aged between 8 and 17 were prospectively evaluated with longitudinal psychosocial assessments using the anger, anxiety, depressive symptoms, and peer relationships instruments within the pediatric Patient-Reported Outcomes Measurement Information System (PROMIS). The COVID-19 cohort (n = 33) included patients with assessments within 2 years prior to the pandemic (t0) and during the pandemic (t1; March 2020 to March 2021). An age-matched comparison cohort (n = 33) with similar demographics and diagnoses included patients assessed twice over 3 years prior to the pandemic.ResultsAll PROMIS measures were in the average range clinically for both groups across time points. However, the COVID-19 group reported a significant increase in depressive symptoms during the pandemic (t1) compared to pre-pandemic (t0) scores (48.2 ± 10.1 vs 44.3 ± 9.4, P = .04, d = −0.37), while the comparison group did not demonstrate any differences in psychosocial functioning between t0 and t1. For the COVID-19 cohort, only the pandemic timeframe (r = 0.21, P = .03) was significantly associated with increased depressive symptom scores, and no other sociodemographic or medical variables were associated with depressive symptoms.ConclusionsSelf-reported depressive symptoms increased during the COVID-19 pandemic in patients with congenital craniofacial diagnoses. Longitudinal studies are needed to elucidate whether such changes will be persistent or compound known variables associated with psychosocial functioning.

The impact of school life and family involvement on Western China junior high school students\u2019 wellbeing at multidimensional levels

BackgroundThe focus of students’ health concerns has gradually progressed from the single factor of physical health to comprehensive health factors, and the physical and mental health of students are now generally considered together. This study focuses on exploring the status of junior high school students’ physical health and their subjective health assessment with the major societal factors that affect students’ lives: School Life and Family involvement. In addition, we explore the main factors influencing students’ subjective health.MethodsA cross-sectional survey was conducted with 190 Tibetan junior high school students in the Maozhuang Township. The intentional sampling was used to choose the research object. The structured questionnaire comprised four parts, namely social and demographic information, family condition, school life, and subjective health quality which was assessed by PROMIS (Chinese version of the Pediatric Patient-Reported Outcomes Measurement Information System).ResultThe average height and weight of boys and girls are statistically different (p-values of 0.026 and 0.044, respectively), but there is no statistically significant difference in BMI (Body Mass Index) between boys and girls (p-value of 0.194). The average values of the five dimensions of depression, anger, anxiety, fatigue, and peer relationships in the PROMIS of the research subjects were 58.9 ± 5.3, 53.3 ± 8.0, 58.1 ± 7.3, 52.8 ± 8.0, 39.3 ± 6.6. In the demographic dimension, the grade was the main factor influencing anger (p < 0.01) and fatigue (p < 0.01), while gender was related to peer relationships (p = 0.02). In the family dimension, the father’s educational level was related to peer relationships (p = 0.05), while the family financial situation was related to depression (p = 0.01). In the school life dimension, relationship with classmates was found to affect anger (p = 0.05), while homework was related to anxiety (p = 0.02) and fatigue (p = 0.05).Conclusionthe physical health index BMI and subjective health evaluation of students are worse than students of more developed areas in China. Their family environment and school life all have varying degrees of impact on the five subjective health outcomes. There are differences in gender and grade level. The government and society need to pay more attention to the physical and mental health of students in remote and underdeveloped areas and improve their health through a student nutrition plan and the establishment of mental health offices.

Using the Pediatric Patient-reported Outcomes–common Terminology Criteria for Adverse Events to Longitudinally Measure Symptom Adverse Events of Children With Advanced Cancer

Background: Children with advanced cancer are frequently enrolled on clinical trials and experience multiple symptoms. Inclusion of patient-reported outcomes in pediatric cancer trials to monitor for symptom adverse events (AEs) of treatment is limited. Objective: To (1) determine the feasibility and acceptability of administering the Ped-PRO-CTCAE biweekly over a 6-month period to children with advanced cancer, and (2) describe symptom AEs. Interventions/Methods: A multisite prospective longitudinal design was used to electronically collect information about symptom AEs using the Ped-PRO-CTCAE and acceptability of a biweekly assessment interval in children 2–18 of age with a diagnosis of advanced cancer who spoke English or Spanish. Descriptive statistics were used to analyze sample characteristics and symptom AE data. Results: Forty-nine children completed the study with a mean age of 11.1 years with diverse cancer diagnoses. Participants completed 85% of 515 administered symptom measures over an average of 16 weeks. Fatigue severity and frequency attributes for anxiety, pain, nausea, and anorexia were the most reported symptom AEs across all participants and time points, while severity attributes of pain, nausea, headache, and abdominal pain had the highest mean scores. Conclusions: Biweekly electronic symptom assessment using the Pediatric PRO-CTCAE is feasible and acceptable. Implications for Practice: Using the Ped-PRO-CTCAE to longitudinally measure symptom AEs in children with advanced cancer may support clinicians and researchers alike to understand patient-reported symptom experiences and toxicities from treatment across the continuum of care. What is Foundational: Inclusion of patient-reported symptom AE monitoring in therapeutic and nontherapeutic clinical trials is necessary to illuminate symptom experiences.

Self-Reported Health Outcomes of Children and Youth with 10 Chronic Diseases

To identify pediatric patient-reported outcomes (PROs) that are associated with chronic conditions and to evaluate the effects of chronic disease activity on PROs. Participants (8-24years old) and their parents were enrolled into 14 studies that evaluated Patient-Reported Outcome Measurement Information SystemPROs across 10 chronic conditions-asthma, atopic dermatitis, cancer, cancer survivors, chronic kidney disease, Crohn's disease, juvenile idiopathic arthritis, lupus, sickle cell disease, and type 1 diabetes mellitus. PRO scores were contrasted with the US general population of children using nationally representative percentiles. PRO-specific coefficients of variation were computed to illustrate the degree of variation in scores within vs between conditions. Condition-specific measures of disease severity and Cohen d effect sizes were used to examine PRO scores by disease activity. Participants included 2975 child respondents and 2392 parent respondents who provided data for 3409 unique children: 52% were 5-12years old, 52% female, 25% African American/Black, and 14% Hispanic. Across all 10 chronic conditions, children reported more anxiety, fatigue, pain, and mobility restrictions than the general pediatric population. Variation in PRO scores within chronic disease cohorts was equivalent to variation within the general population, exceeding between-cohort variation by factors of 1.9 (mobility) to 5.7 (anxiety). Disease activity was consistently associated with poorer self-reported health, and these effects were weakest for peer relationships. Chronic conditions are associated with symptoms and functional status in children and adolescents across 10 different disorders. These findings highlight the need to complement conventional clinical evaluations with those obtained directly from patients themselves using PROs.

Adolescents' understanding of chemotherapy-related adverse events: a concept elicitation study.

to document adolescents' understanding of chemotherapy-related core adverse events from the Pediatric Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events and thus begin the validation process of this tool's items with Brazilian adolescents. this is a prospective, qualitative study of concept elicitation. The participants were 17 adolescents aged 13-18 years and undergoing chemotherapy in three hospitals in São Paulo - SP, Brazil. Cognitive interviews were conducted with questions based on chemotherapy-related adverse events. Data were analyzed for responsiveness and missingness. adolescents could and were willing to provide descriptive information about their chemotherapy adverse events, including physical and emotional events. Some participants suggested alternative terms to name the adverse events and some used more complex terms, but most were satisfied with the primary terms used by the researchers. this study represents the first steps towards understanding how adolescent cancer patients identify, name, and describe these events by cognitive interviewing to help design future assessment instruments focused on this age group. (1) Adolescents' voice is a priority in reporting chemotherapy-related adverse events. (2) Adverse events (AEs) inquiry may best be initiated with open-ended questions. (3) Elicitation studies can include the voices of the target population. (4) Adolescents with cancer can self-report clinically relevant chemotherapy AEs.

Recommended scoring approach for the pediatric patient-reported outcomes version of the Common Terminology Criteria for Adverse Events.

Collecting symptom, function, and adverse event (AE) data directly from children and adolescents undergoing cancer care is more comprehensive and accurate than relying solely on their caregivers or clinicians for their interpretations. We developed the pediatric patient-reported outcomes version of the Common Terminology Criteria for Adverse Events (Ped-PRO-CTCAE) measurement system with input from children, parents, and clinicians. Here, we report how we determined the recommended Ped-PRO-CTCAE item scoring approach. Data from 271 patients were analyzed using three scoring approaches: (a) at the AE attribute (frequency, severity, interference) using ordinal and dichotomous measures; (b) a weighted composite AE item score by AE attribute (0.5 - frequency; 1.0 - severity; 1.5 - interference); and (c) overall number of AEs endorsed. Associations of each AE attribute, AE item score, and overall AE score with the Patient-Reported Outcome Measurement Information System (PROMIS) Pediatric measures were examined. The ability of the overall Ped-PRO-CTCAE AE score to identify patients with PROMIS symptom T-scores worse than reference population scores was assessed. Clinician preference for score information display was elicited through interviews with five pediatric oncology clinical trialists. The diverse scoring approaches yielded similar outcomes, including positive correlations of the Ped-PRO-CTCAE attributes, AE item score, and the overall AEs score with the PROMIS Pediatric measures. Clinicians preferred the most granular display of scoring information (actual score reported by the child and corresponding descriptive term). Although three scoring approaches yielded similar results, we recommend the AE attribute level of one score per Ped-PRO-CTCAE AE attribute for its simplicity of use in care and research.