Childhood Health Assessment Questionnaire Research Articles

Assessing the Menstrual Cycle and Related Problems in Adolescents with a Genetic Syndrome Associated with Intellectual Disability

Study ObjectiveThe aim of this study was to assess the experience and quality of life (QoL) related to menstruation in adolescents with a genetic syndrome accompanying intellectual disability (ID). MethodsThis prospective cross-sectional study was conducted on 49 adolescents with a genetic syndrome accompanied by ID, which was defined by the Wechsler Intelligence Scale for Children-Revised, and 50 unaffected controls. In a survey created by the authors, demographic information, menstrual history, and information regarding menstrual difficulties, school abstinence, dysmenorrhea, and premenstrual changes were collected. The Childhood Health Assessment Questionnaire was used to evaluate physical impairment, whereas the QoL scale was utilized to evaluate QoL in general and during menstruation. Data were collected from caregivers and additionally from the participants with mild ID, whereas in the control group, data were collected from the participants. ResultsMenstrual history was similar between the 2 groups. Menstruation-related school absenteeism was higher among the ID group (8% vs 40.5%, P < .001). Mothers reported that 73% of their daughters needed help with menstruation care. Social, school, psychosocial functioning, and total QoL scores during menstruation were significantly lower in the ID group when compared with the controls. A significant decrease in physical, emotional, social, psychosocial functioning, and total QoL score occurred during menstruation in the ID group. None of the mothers requested menstrual suppression. ConclusionAlthough menstrual patterns in the 2 groups were similar, QoL decreased significantly while menstruating in the ID group. Despite a decrease in QoL, an increase in school absenteeism, and a high percentage of needing assistance while menstruating, none of the mothers requested menstrual suppression.

Musculoskeletal Ultrasound and the Assessment of Disease Activity in Juvenile Idiopathic Arthritis.

To determine the frequency of subclinical synovitis on musculoskeletal ultrasonography (MSUS) in juvenile idiopathic arthritis (JIA) and correlate patient- and provider-reported outcome measures with MSUS synovitis. JIA patients with an active joint count (AJC) of >4 underwent a 42-joint MSUS performed at baseline and 3 months. B-mode and power Doppler images were obtained and scored (range 0-3) for each of the 42 joints. Outcomes evaluated included physician global assessment of disease activity (PhGA), patient global assessment of disease activity (PtGA), patient pain, Childhood Health Assessment Questionnaire (C-HAQ), and AJC. Subclinical synovitis was defined as synovitis detected by MSUS only. Generalized estimation equations were used to test the relationship between clinical arthritis (positive/negative) and subclinical synovitis (positive/negative). Spearman's correlation coefficients (rs ) were calculated to determine the association between MSUS synovitis and patient- and physician-reported outcomes. In 30 patients, subclinical synovitis was detected in 30% of joints. Clinical arthritis of the fingers, wrists, and knee joints was significantly associated with MSUS synovitis in these joints. PtGA and the C-HAQ had a moderate (rs =0.44, P= 0.014) to weak (rs =0.37, P=0.045) correlation with MSUS synovitis. There was a statistically significant strong correlation between MSUS synovitis and PhGA (rs =0.61, P=0.001), but a weak correlation with AJC (rs =0.37, P=0.048) at the follow-up visit. Subclinical synovitis was commonly observed in this cohort of JIA patients. The fair-to-moderate correlation of MSUS synovitis with patient- and provider-reported outcomes suggests that MSUS assesses a different, possibly more objective, domain not determined by traditional JIA outcome measurements.

Upper limb training using visual feedback for children with cerebral palsy

Purpose: This study aimed to investigate that if upper limb visual feedback training added to the usual therapy was effective on upper limb functions in children with cerebral palsy. Methods: Thirty-eight children with cerebral palsy (4-18 years) participated in the study. The participants were randomly assigned into two groups. Study group (n=19, mean age 8.50±3.40, 53% female) received visual feedback training plus usual therapy twice a week for 8 weeks, 16 sessions in total, while control group (n=19, mean age 10.60±3.80, 42% female) received only usual therapy. The participants’ Gross Motor Function Classification System levels were between I-III and spasticity levels were between ‘0’ to ‘1+’ according to Modified Ashworth Scale. Upper limb range of motion (ROM), grip and pinch strength, hand skills (Minnesota Manual Dexterity Test (MMDT)), and functional abilities (Childhood Health Assessment Questionnaire (CHAQ)) were assessed before and after treatment. Results: The change amount in shoulder flexion (p=0.004) and abduction (p=0.01), supination (p=0.019) and wrist flexion (p=0.004) and extension (p=0.00) ROM values; bilateral MMDT scores (p=0.003) were statistically significantly improved in study group compared to control group. Conclusion: Adding visual feedback training to the usual therapy program of children with cerebral palsy was found to be effective in some outcome measurements such as upper limb ROM and hand skills. In the long-term rehabilitation process, this new training method may be an alternative additive option for children, families, and physiotherapists.

Economic evaluation of infliximab, synthetic triple therapy and methotrexate in the treatment of newly diagnosed juvenile idiopathic arthritis

BackgroundEvaluation of costs and short-term cost-effectiveness of infliximab plus methotrexate (IFX + MTX); triple therapy of hydroxychloquine, sulphasalazine, and methotrexate (TRIPLE); or methotrexate monotherapy (MTX) in patients with new-onset polyarticular juvenile idiopathic arthritis (JIA).MethodsIn a prospective multicenter study (ACUTE-JIA), costs and health outcomes of 60 randomized patients with new-onset disease-modifying anti-rheumatic drug (DMARD)-naïve polyarticular JIA were analyzed during the first year. A mapping algorithm was used to obtain utility values from Child Health Assessment Questionnaire (CHAQ). Wallace criteriae were used to assess clinically inactive disease (CID). Linear regression with non-parametric bootstrapping was used to adjust imbalances at baseline.ResultsUsing prices for IFX biosimilar, adjusted annual mean (SD) costs of treatment (€) were 21,164 (4158), 12,136 (5286), and 18,300 (8635) on IFX + MTX, TRIPLE, and MTX, respectively. Incremental cost-effectiveness ratio (ICER) for IFX + MTX as compared with TRIPLE or MTX were 3442 € or 678 € per additional month spent in CID. Mean (SD) quality-adjusted life years (QALYs) for IFX + MTX, TRIPLE and MTX were 0.755 (0.065), 0.725 (0.062), and 0.686 (0.124). ICER for IFX + MTX vs TRIPLE was 294,433 €, and for IFX + MTX vs MTX 31,435 € per QALY gained.ConclusionsIn short-term, biosimilar IFX + MTX can be considered cost-effective when compared with MTX alone. TRIPLE was cost-effective when compared with MTX and showed cost advantage when compared with IFX + MTX. Cost per time spent in CID showed similar results than ICER evaluations.Trial registrationThis trial was primarily registered with the Ethical Board of Helsinki District University Hospital (https://www.hus.fi), clinical trial number 211864, and later with ClinicalTrials.gov, number NCT01015547.

Modified Juvenile Spondyloarthritis Disease Activity Index in the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry.

To validate the Juvenile Spondyloarthritis Disease Activity Index (JSpADA), and modified versions thereof, in a North American cohort of patients with enthesitis-related arthritis (ERA). We utilized the Childhood Arthritis and Rheumatology Research Alliance Registry database ERA cohort to validate the JSpADA and its modifications (JSpADA6-no Schober, no C-reactive protein [CRP]/erythrocyte sedimentation rate [ESR]; JSpADA7-no Schober; and JSpADA7-no CRP/ESR) using the Outcome Measures in Rheumatology principles of face validity, discriminative validity, and responsiveness to change. There were 51 subjects (64 visits) with complete JSpADA data with a mean age of 13.7 years and disease duration of 30.9 months. Subjects were predominantly White (84.3%), and 56.9% were male and 50% were HLA-B27 positive. The JSpADA showed high correlation with the clinical 10-joint Juvenile Arthritis Disease Activity Score (cJADAS10; r = 0.81), moderate-to-high correlation with physician global assessment (PGA; r = 0.69), and low-to-fair correlation with Childhood Health Assessment Questionnaire (CHAQ; r = 0.22). The modifications of the JSpADA (JSpADA7-no Schober; JSpADA7-no CRP/ESR; and JSpADA6-no Schober, no CRP/ESR) performed similarly with high correlation with cJADAS10 (r = 0.81, 0.79, and 0.80, respectively), moderate-to-high correlation with PGA (r = 0.65, 0.67, 0.64, respectively), and low-to-fair correlation with CHAQ (r = 0.35, 0.34, 0.39, respectively). All modified versions of JSpADA had good responsiveness to change. All versions of JSpADA had excellent discriminative validity. We propose the term modified JSpADA for the modification of JSpADA with 6 elements (JSpADA6-no Schober, no CRP/ESR). This shorter disease activity index may improve implementation of JSpADA in both clinical practice and research trials.

Portuguese Cross-Cultural Adaptations of the Pediatric International Knee Documentation Committee Score

The aim of this study was to translate the Pediatric International Knee Documentation Committee Score (Pedi-IKDC) into European Portuguese language. The Pedi-IKDC was originally developed in the English language and its main construct is functional assessment of knee disorders in children and adolescents. The original English version of the questionnaire was translated to European Portuguese using the forwardbackwards method. Patients aged eight to 17 with knee disorders were considered eligible for the study. An online platform was implemented to collect patient responses, including general patient information, the questionnaires Pedi-IKDC, EuroQol-5 Dimension Youth and Childhood Health Assessment Questionnaire. Three surveys were sent: at the moment of first evaluation (T0), after two (T1) and ten (T2) weeks. These surveys followed the Consensus-based Standards for the Selection of Health Measurement Instruments - COSMIN Checklist recommendations. The internal consistency, reliability, error of measurement, structural and construct validity (by means of correlation with previously validated scales), responsiveness and interpretability (floor/ceiling effects, MIC and ROC curve) were evaluated. Forty-seven patients completed T0, 42 patients completed T1 and 40 patients completed T2. The factorial analysis confirmed that the scale has one dimension. Cronbach alpha (α) was 0.94; interclass correlation coefficient was 0.92; smallest detectable change was 19.04 for individuals and 3.31 for groups; standard error of measurement was 6.87; minimum important change was 18.48; floor and ceiling effects were absent. More than 75% of the hypotheses tested for construct validity were confirmed, showing its adequacy. The variation of scores between T0 and T2 correlated with the clinical evolution of the participants (r = 0.421, p < 0.05). The Portuguese version of Pedi-IKDC demonstrated good psychometric properties, being a valuable tool for clinical assessment of pediatric patients with knee disorder.

Baseline characteristics of children with juvenile dermatomyositis enrolled in the first year of the new Childhood Arthritis and Rheumatology Research Alliance registry

BackgroundTo report baseline characteristics, patient reported outcomes and treatment of children with Juvenile Dermatomyositis (JDM) in the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry.MethodsChildren newly diagnosed with JDM were enrolled in the CARRA Registry from 41 pediatric rheumatology centers. Baseline patient demographics, disease characteristics, assessments, patient reported outcome and treatments were recorded.ResultsIn the first year, 119 JDM participants were enrolled. Most were female (63.4%), and white (72.3%) with a median diagnosis age 8.0 years (IQR 4.0–11.5), and median age of disease onset 7.0 years (IQR 3.5–7.5). They had characteristic rashes (92.4%), elevated muscle enzymes (83.2%), physician global score 4.0 (IQR 2.5–5.0) and manual muscle testing score 63.5 (IQR 51.0–75.0). Calcinosis (3.4%) and interstitial lung disease (< 1%) were uncommon. Myositis specific antibodies were measured and reported in nearly half of participants enrolled where anti-MJ followed by Anti-p155/140 were most common (11/49 and 7/53 respectively).Childhood Health Assessment Questionnaire (CHAQ) results showed mild-moderate disability (median 0.750, IQR 0.030–1.875), as did patient/parent global assessments of disease activity (median 3, patient IQR: 1.75–5.25; parent IQR: 1–7). Patient Reported Outcomes Measurement Information System (PROMIS®) Pediatric Global Health 7 scores, Pain Interference, Physical Function scores for Mobility, and Upper Extremity Function were commonly worse than 95% of the general pediatric population.ConclusionsIn its inaugural year, 119 JDM patients were successfully enrolled in participapte in the New CARRA Registy. This registry will provide the necessary foundation to advance clinical research to improve outcomes using traditional measures and patient reported outcomes. With the CARRA biorepository, this infrastructure will enable future translational research. Together, these efforts may aid in future clinical trials, including comparative effectiveness trials.

Comprehensive Assessment of Quality of Life, Functioning, and Mental Health in Children With Juvenile Idiopathic Arthritis and Noninfectious Uveitis.

Pediatric uveitis can lead to sight-threatening complications and can impact quality of life (QoL) and functioning. We aimed to examine health-related QoL, mental health, physical disability, vision-related functioning (VRF), and vision-related QoL in children with juvenile idiopathic arthritis (JIA), JIA-associated uveitis (JIA-U), and other noninfectious uveitis. We hypothesized that there will be differences based on the presence of eye disease. A multicenter cross-sectional study was conducted at four sites. Patients with JIA, JIA-U, or noninfectious uveitis were enrolled. Patients and parents completed the Pediatric Quality of Life Inventory (PedsQL; health-related QoL), the Revised Childhood Anxiety and Depression Scale (RCADS; anxiety/depression), the Childhood Health Assessment Questionnaire (C-HAQ; physical disability), and the Effects of Youngsters' Eyesight on Quality of Life (EYE-Q) (VRF/vision-related QoL). Clinical characteristics and patient-reported outcome measures were compared by diagnosis. Of 549 patients, 332 had JIA, 124 had JIA-U, and 93 had other uveitis diagnoses. Children with JIA-U had worse EYE-Q scores compared to those with JIA only. In children with uveitis, those with anterior uveitis (JIA-U and uveitis only) had less ocular complications, better EYE-Q scores, and worse C-HAQ and PedsQL physical summary scores compared to those with nonanterior disease. In children with anterior uveitis, those with JIA-U had worse PedsQL physical summary and C-HAQ scores than anterior uveitis only. Further, EYE-Q scores were worse in children with bilateral uveitis and more visual impairment. There were no differences in RCADS scores among groups. We provide a comprehensive outcome assessment of children with JIA, JIA-U, and other uveitis diagnoses. Differences in QoL and function were noted based on underlying disease. Our results support the addition of a vision-specific measure to better understand the impact of uveitis.

OP0221 EFFICACY OF SECUKINUMAB IN ENTHESITIS-RELATED ARTHRITIS: RESULTS FROM A RANDOMISED, DOUBLE-BLIND, PLACEBO-CONTROLLED, TREATMENT WITHDRAWAL, PHASE 3 STUDY (JUNIPERA)

BackgroundEnthesitis-related Arthritis (ERA) is a category of juvenile idiopathic arthritis (JIA) characterised by heterogeneous and insidious manifestations comprising axial and/or peripheral arthritis, and enthesitis.1 Secukinumab (SEC) demonstrated efficacy and safety in both ERA and juvenile psoriatic arthritis (JPsA) in the JUNIPERA trial.2ObjectivesTo evaluate the rate of flare risk reduction and efficacy of SEC on axial and peripheral manifestations in patients (pts) with active ERA.MethodsERA pts (2 to &lt;18 years of age) with active disease (both ≥3 active joints and ≥1 active enthesitis site) were included. In the open-label (OL) treatment-period (TP)1, s.c. SEC (75/150 mg in pts &lt;50/ ≥50 kg) was administered at baseline (BL), and at Week (Wk) 1–4, 8 and 12. Pts who achieved at least JIA-ACR30 response at Wk 12 were randomised into the double-blinded TP2 to continue SEC or placebo (PBO) every 4 wk until a disease flare, or up to Wk 100. The primary endpoint was time to flare in ERA and JPsA pts. The juvenile spondyloarthritis disease activity Index (JSpADA) is a disease activity assessment tool that contains 8 items to measure axial and peripheral disease activity.3 Evaluation of axial and peripheral manifestations at the end of TP1 and TP2 in pts who experienced these symptoms at BL included modified Schober test (lumbar flexion), inflammatory back pain, FABER (Flexion, ABduction, External Rotation) test, JIA-ACR responses, Juvenile Arthritis Disease Activity Score (JADAS)-27, and resolution of enthesitis and dactylitis for peripheral disease. These outcomes were also used to assess JIA disease course at the end of TP2.ResultsA total of 52/86 (60.5%) pts with ERA were enrolled in the OL period TP1 (mean age, 13.7 years; male, 78.8%). In total, 51/52 (98.1%) pts completed TP1 and 41/44 (93.2%) completed TP2. At BL, mean JADAS-27 was 14.8, mean JSpADA index was 3.9, mean enthesitis and dactylitis counts were 2.7 and 0.4, respectively, mean number of active joints was 6.2 and of mean joints with limited range of motion 4.9. The relative risk reduction of experiencing a disease flare in TP2 was 55% (HR 0.45, 95% CI: 0.16–1.28, p=0.075) in ERA pts (Figure 1). The overall axial and peripheral disease symptoms improved over time and are presented in the Table 1. At the end of TP1, 84.6% (44/52) of pts achieved JIA-ACR 30 and 65.4% (34/52) achieved JIA-ACR 70. Clinically relevant reduction of functional ability as assessed by Childhood Health Assessment Questionnaire (CHAQ) also occurred (see Table 1).Table 1.Resolution of axial and peripheral disease symptoms and JIA ACR responses at the end of TP1 and 2Clinical response, mean (SD) change from BL (unless otherwise stated)TP1-Wk 12End of TP2*SEC (N=52)SEC (N=22)PBO (N=22)JSpADA index−2.4 (1.7)−2.7 (1.7)−2.3 (2.1)JSpADA Schöber, %58.3100.0100.0Inflammatory back pain, %77.8100.050.0FABER test, %52.6100.083.3Clinical sacroiliitis, %53.3100.050.0Enthesitis−2.2 (1.9)−2.5 (2.1)−1.3 (1.8)Dactylitis−0.2 (0.8)−0.2 (1)−0.1 (0.4)JIA ACR30, %84.690.968.2JIA ACR50, %78.881.868.2JIA ACR70, %65.468.254.5JIA ACR90, %32.745.550.0JIA ACR100, %26.936.445.5Inactive disease, %38.550.050.0CHAQ−0.5 (0.5)−0.6 (0.7)−0.4 (0.5)CRP, median (SD) change from BL−1.8 (38.7)−5.8 (38.3)0 (35.9)JADAS-27−9.6 (7.5)−11.0 (8.9)−7.6 (8.9)Resolution of enthesitis#, %72.378.683.3Resolution of dactylitis#, %5066.70*End of TP2 is based on individual pts’ last visit at TP2. #At BL, in TP1, enthesitis (n= 46); dactylitis (n=5). In TP2, no. of pts who had presence at BL and showed complete resolution at the end of TP2: enthesitis, SEC 14, PBO 18; dactylitis, SEC 3, PBO, 0. CRP, C-reactive proteinConclusionIn pts with ERA, SEC demonstrated longer time to disease flare vs PBO and exhibited rapid and sustained improvement of axial and peripheral manifestations up to Wk 104.

Key measurement concepts and appropriate clinical outcome assessments in pediatric achondroplasia clinical trials

BackgroundThis study aimed to identify fit-for-purpose clinical outcome assessments (COAs) to evaluate physical function, as well as social and emotional well-being in clinical trials enrolling a pediatric population with achondroplasia. Qualitative interviews lasting up to 90 min were conducted in the US with children/adolescents with achondroplasia and/or their caregivers. Interviews utilized concept elicitation methodology to explore experiences and priorities for treatment outcomes. Cognitive debriefing methodology explored relevance and understanding of selected COAs.ResultsInterviews (N = 36) were conducted with caregivers of children age 0–2 years (n = 8) and 3–7 years (n = 7) and child/caregiver dyads with children age 8–11 years (n = 15) and 12–17 years (n = 6). Children/caregivers identified pain, short stature, impacts on physical functioning, and impacts on well-being (e.g. negative attention/comments) as key bothersome aspects of achondroplasia. Caregivers considered an increase in height (n = 9/14, 64%) and an improvement in limb proportion (n = 11/14, 71%) as successful treatment outcomes. The Childhood Health Assessment Questionnaire (CHAQ) and Quality of Life in Short Stature Youth (QoLISSY-Brief) were cognitively debriefed. CHAQ items evaluating activities, reaching, and hygiene were most relevant. QoLISSY-Brief items evaluating reaching, height bother, being treated differently, and height preventing doing things others could were most relevant. The CHAQ and QoLISSY-Brief instructions, item wording, response scales/options and recall period were well understood by caregivers and adolescents age 12–17. Some children aged 8–11 had difficulty reading, understanding, or required caregiver input. Feedback informed minor amendments to the CHAQ and the addition of a 7-day recall period to the QoLISSY-Brief. These amendments were subsequently reviewed and confirmed in N = 12 interviews with caregivers of children age 0–11 (n = 9) and adolescents age 12–17 (n = 3).ConclusionsAchondroplasia impacts physical functioning and emotional/social well-being. An increase in height and improvement in limb proportion are considered to be important treatment outcomes, but children/adolescents and their caregivers expect that a successful treatment should also improve important functional outcomes such as reach. The CHAQ (adapted for achondroplasia) and QoLISSY-Brief are relevant and appropriate measures of physical function and emotional/social well-being for pediatric achondroplasia trials; patient-report is recommended for age 12–17 years and caregiver-report is recommended for age 0–11 years.

P007 Novel integration of a digital patient-reported outcome measure into an NHS electronic health record

Abstract Background/Aims The Childhood Health Assessment Questionnaire (CHAQ) and 0-10cm visual analogue scale (VAS) for self-reporting global-wellbeing form part of the Core Outcome Variables measuring disease activity in juvenile idiopathic arthritis. However, reported barriers to using paper-based CHAQs include: impacts on consultation time and issues with scoring and transcribing CHAQ data into health records. To address these issues we aimed to develop a digital CHAQ (eCHAQ) for patients attending paediatric rheumatology clinics at Alder Hey Children’s Hospital. Additional objectives included: developing automated invitations to complete eCHAQs; achieving automated scoring and integration of CHAQ data into the hospital’s Electronic Health Record (EHR); investigating the acceptability of the eCHAQ to patients and clinical staff; and studying the effects of the system on CHAQ completion rates. Methods The eCHAQ was developed collaboratively by the Alder Hey Information Technology team, clinicians, and a technology provider, Aire Logic. Acceptability was measured using a patient survey (patient and parent ePROM questionnaire), and a survey (Technology Acceptance Model 2 questionnaire) and focus group involving clinical staff. CHAQ completion rates were measured using a before- after study of data from clinical records. Results The eCHAQ was implemented in April 2021 and approximately 500 eCHAQ invitations are sent monthly. Technical features include automated generation of SMS invitations to complete eCHAQs; automated scoring of CHAQ assessments; and unique URLs (webpage addresses) enabling automated re-integration of CHAQ data into the EHR. Patients/care-givers (n = 24) scored the eCHAQ highly across Likert scale items designed to assess accessibility (median response = 5 [strong agreement]; interquartile range [IQR] = 0); and acceptability (median response = 5 [strong agreement]; IQR = 0). Clinical staff (n = 7; consultants, nurse specialists and occupational therapists) also scored the eCHAQ highly across domains tested using Likert scale items including “Perceived Usefulness” (median response = 7 [strong agreement]; IQR = 0.25) and “Output Quality” (median response = 7 [strong agreement]; IQR = 1). Thematic analysis of a focus group discussion involving 10 members of the Rheumatology MDT identified that strengths of the eCHAQ system included improved access to clinical data, improved data quality and time saved. Concerns relating to access to digital technologies were highlighted. Use of the eCHAQ resulted in a significant increase in the CHAQ completion rate. Before the implementation of the eCHAQ, documentation of a CHAQ score in the EHR occurred in 33/100 (33%) assessed records compared to 47/65 (72%) after the introduction of the eCHAQ system (chi-square = 11.51; p &amp;lt; 0.05). Conclusion Development of a highly-automated digital CHAQ, with integration directly into an NHS EHR, was technically feasible. Introduction of the eCHAQ was found to be highly acceptable to patients and clinical staff and was associated with a significant improvement in CHAQ completion rates. Disclosure M. Neame: None. D. Reilly: None. D. Bond: Other; Employee of private technology provider involved in the development of the reported intervention. A. Puthiyaveetil: None. L. McCann: None. K. Mahmood: None. B. Almeida: None. C.E. Pain: None. V. Furfie: None. G. Cleary: None.

Development and validation of the Kids Disability Screen for children with juvenile idiopathic arthritis: results from the CAPRI Registry.

The aim of this study was to develop and validate a brief disability screen for children with JIA, the Kids Disability Screen (KDS). A total of 216 children enrolled in the Canadian Alliance of Pediatric Rheumatology Investigators (CAPRI) Registry in 2017-2018 formed a development cohort, and 220 children enrolled in 2019-2020 formed a validation cohort. At every clinic visit, parents answered two questions derived from the Childhood Health Assessment Questionnaire (CHAQ): 'Is it hard for your child to run and play BECAUSE OF ARTHRITIS?' ('Hard' 0-10), and 'Does your child usually need help from you or another person BECAUSE OF ARTHRITIS?' ('Help', 0-10). We used 36-fold cross-validation and tested nine different mathematical methods to combine the answers and optimize psychometric properties. The results were confirmed in the validation cohort. Expressed as the mean of the two answers, KDS best balanced ease of use and psychometric properties, while a LASSO regression model combining the two answers with other patient characteristics [estimated CHAQ [eCHAQ]) had the highest responsiveness. In the validation cohort, 22.7%, 25.9% and 28.6% of patients had a score of 0 at enrolment for the KDS, eCHAQ and CHAQ, respectively. Responsiveness was 0.67, 0.74 and 0.62, respectively. Sensitivity to detect a CHAQ > 0 was 0.90 and specificity 0.56, KDS detecting some disability in 44% of children with a CHAQ = 0. This simple KDS has psychometric properties comparable with those of a full CHAQ and may be used at every clinic visit to identify those children who need a full disability assessment.

Psychometric Evaluation of the Polish Version of the Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD).

PurposeThe assessment of the quality of life is an important element of the clinical examination of the patient. The aim of this study was translation and cross-cultural adaptation of the “Caregiver Priorities and Child Health Index of Life with Disabilities” (CPCHILD) questionnaire into Polish language, and testing of reliability and validity of the CPCHILD-PL for children with cerebral palsy (CP).Material and MethodsA Polish version of CPCHILD was created according to internationally accepted guidelines. Parents (n=77) of 51 boys/26 girls between 3 and 17 years with CP with Gross Motor Function Classification System I–V (GMFCS I–V) participated. To assess the reliability each domain and the total measure was tested for internal consistency and test-retest reliability. Convergent validity was evaluated by correlating the CPCHILD-PL with the CHAQ (Childhood Health Assessment Questionnaire) questionnaire.ResultsTest-retest reliability assessed by Spearman correlation coefficient for the final result of CPCHILD-PL and for most of domains were above 0.90. The values of Cronbach’s-α coefficient (measuring internal consistency) were high for all domains (except for domain 5: Health) and the entire CPCHILD-PL, with the range 0.88–0.96. The comparison between CPCHILD-PL and the Disability Index (DI) of the CHAQ showed a negative correlation. The higher the DI, the lower the CPCHILD result. The Spearman’s rank coefficient was −0.75.ConclusionThe Polish version for the CPCHILD for children with CP seems to be reliable and valid tool for assessing health-related quality of life from the caregiver perspective. It can be used in research and clinical practice for evaluation and comparison of health-related quality of life in children with CP in different countries.

Social determinants of health influence disease activity and functional disability in Polyarticular Juvenile Idiopathic Arthritis

BackgroundSocial determinants of health (SDH) greatly influence outcomes during the first year of treatment in rheumatoid arthritis, a disease similar to polyarticular juvenile idiopathic arthritis (pJIA). We investigated the correlation of community poverty level and other SDH with the persistence of moderate to severe disease activity and functional disability over the first year of treatment in pJIA patients enrolled in the Childhood Arthritis and Rheumatology Research Alliance Registry.MethodsIn this cohort study, unadjusted and adjusted generalized linear mixed effects models analyzed the effect of community poverty and other SDH on disease activity, using the clinical Juvenile Arthritis Disease Activity Score-10, and disability, using the Child Health Assessment Questionnaire, measured at baseline, 6, and 12 months.ResultsOne thousand six hundred eighty-four patients were identified. High community poverty (≥20% living below the federal poverty level) was associated with increased odds of functional disability (OR 1.82, 95% CI 1.28-2.60) but was not statistically significant after adjustment (aOR 1.23, 95% CI 0.81-1.86) and was not associated with increased disease activity. Non-white race/ethnicity was associated with higher disease activity (aOR 2.48, 95% CI: 1.41-4.36). Lower self-reported household income was associated with higher disease activity and persistent functional disability. Public insurance (aOR 1.56, 95% CI 1.06-2.29) and low family education (aOR 1.89, 95% CI 1.14-3.12) was associated with persistent functional disability.ConclusionHigh community poverty level was associated with persistent functional disability in unadjusted analysis but not with persistent moderate to high disease activity. Race/ethnicity and other SDH were associated with persistent disease activity and functional disability.

Assessing the Validity and Reliability of the Effects of Youngsters' Eyesight on Quality of Life Questionnaire Among Children With Uveitis.

The Effects of Youngsters' Eyesight on Quality of Life (EYE-Q) questionnaire measures vision-related functioning (VRF) and vision-related quality of life (VRQoL) in children with uveitis. Our aim was to revise the alpha version of the EYE-Q to refine VRF and VRQoL subscales and to assess the validity of the EYE-Q. Children with juvenile idiopathic arthritis (JIA), JIA-associated uveitis, and other noninfectious uveitis were enrolled. Patients and parents completed the EYE-Q, Pediatric Quality of Life Inventory (overall quality of life), and Childhood Health Assessment Questionnaire (physical functioning). The development site completed the alpha version of the EYE-Q, and the composite sites completed the beta version. We compared item-subscale correlations, internal consistency, and construct and discriminant validity among the different versions. Of the 644 patients enrolled, 61.6% completed the alpha version, and 38.4% the beta version of the EYE-Q. Mean ± SD patient age was 11.1 ± 4.2 years, and 70% were female. Fewer White patients (73.5%) completed the alpha version compared to the beta version (86.2%; P < 0.001). With the exception of patient-reported VRF, both versions had similar item-subscale correlations. Version comparisons on scale internal consistencies indicated significant differences for parent- and patient-reported VRF, but each scale had a Cronbach's α of >0.80 beta. When data were combined, the EYE-Q showed significant differences between JIA-only and uveitis patients on all parent and patient scores, except for patient-reported VRF. The EYE-Q appears to be a valid measure of VRF and VRQoL in pediatric uveitis. Our results suggest it may be used as an outcome measure in multicenter pediatric uveitis studies.

Quality of life problems in children with juvenile idiopathic arthritis and its subtype associated with uveitis (literature review and own research)

Background. The problem of the quality of life (QoL) is relevant nowadays, especially for patients with chronic diseases, including juvenile idiopathic arthritis (JIA) and the subtype of JIA associated with uveitis (JIA-u). The purpose was to carry out a literature review on the QoL in children with JIA and JIA-u, with an analysis of existing tools, the possibility of their use in children, and to assess the QoL indicators of children with JIA, who were treated in a specialized department of the hospital. Materials and methods. The search was performed in the PubMed, Medline, and Google databases using the keywords: JIA, rheumatoid arthritis, uveitis, JIA-u, arthritis with uveitis, QoL, QoL in children. Inclusion criteria were as follows: publication after 2010, use of questionnaires to assess QoL, the sample of patients over 30 people. The type of instrument for assessing QoL, the sample nature, and the QoL indicators of patients were analyzed. The study was carried out at the premises of the State Institution “Institute for Children and Adolescents Health Care of the National Academy of Medical Sciences of Ukraine” and involved 41 children with JIA (22 with polyarticular, and 19 with oligoarticular variants): 25 girls and 16 boys aged 3–17 years with disease duration of 40.2 ± 6.2 months. Thirty-six of them received methotrexate, and 5 — sulfasalazine. The disease activity was assessed by the Juvenile Arthritis Disease Activity Score (JADAS27) and the functional state — by the Childhood Health Assessment Questionnaire (CHAQ). For quality of life assessment, the Pediatric Quality of Life InventoryTM validated for Ukraine was used. Results. The analysis included 73 foreign scientific papers from 2011 to 2021, among which 11 are fully devoted to the assessment of QoL in children with JIA, and 62 partially cover this topic in children and adults with arthritis. In Ukraine, these issues are not discussed enough (6 articles on the evaluation of QoL in children), the factors for its deterioration remain unclear. The level of activity according to JADAS27 in children in subgroups with both poly- and oligoarticular variants was high (11.20 ± 7.04 and 8.9 ± 4.2 points), and the functional state (on CHAQ) — 0.19 ± 0.17. In children with JIA, QoL indicators are reduced, especially on the scales of emotional and school functioning, and closely correlate with disease activity (r = 0.784, p &lt; 0.05). Conclusions. The analysis of the conducted studies showed that decreased disease activity is not accompanied by the QoL indicators optimization, which remain reduced. Recently, the situation with the QoL assessment in domestic medicine has been improving, relevant questionnaires are being actively introduced. However, the ambiguity of the results in world medicine and the presence of a small number of works on this topic determine the urgency of the problem of QoL in children with JIA in Ukraine.

A randomized, double-blind, placebo-controlled 12-week trial of infliximab in patients with juvenile-onset spondyloarthritis

ObjectiveTo assess the efficacy and safety of infliximab versus placebo in the treatment of patients with juvenile-onset spondyloarthritis (JoSpA).MethodsPhase III, randomized, double-blind, placebo-controlled trial of 12 weeks that included patients ≤ 18 years old with JoSpA not responding to nonsteroidal anti-inflammatory drugs, sulfasalazine, or methotrexate. Patients were randomly assigned 1:1 to the infusion of infliximab 5mg/kg or placebo; completers entered then an open-label extension (OLE) period of 42 weeks. The primary endpoint was the number of active joints. Secondary outcomes included the assessment of disease activity, tender entheses, spinal mobility, serum C-reactive protein (CRP), the Bath Ankylosing Spondylitis Disease Activity and Functional Index, and the Childhood Health Assessment Questionnaire (CHAQ).ResultsWe randomized 12 patients to infliximab and 14 to placebo. No significant differences were found between groups at baseline. At week 12, the mean number of active joints was 1.4 (SD 2.4) in the infliximab group and 4.1 (SD 3.0) in the placebo group (p = 0.0002). A repeated-measures mixed model analysis that included all endpoints in the study demonstrated sustained favourable outcomes of infliximab for active joints, tender joints, swollen joints, and tender enthesis counts, as well as for CHAQ and CRP (p < 0.01). Adverse events were more frequent in the infliximab group, including infections and infusion reactions, but none of them was serious.ConclusionInfliximab is efficacious for patients with JoSpA with an inadequate response to conventional treatment. No serious adverse events with the use of infliximab were observed.

The state of physical functionning of patients with juvenile idiopathic arthritis in the assessment of quality of life

Summary. Juvenile idiopathic arthritis is a severe chronic childhood disease that affects not only the joints but is also accompanied by various comorbid conditions, among which eye damage (uveitis) is the most common. In addition to a significant impact on the general condition of the child, this disease also affects the main indicators of quality of life: physical activity, emotional activity, activity in educational institutions, and the social sphere. During the period of active study of Juvenile idiopathic arthritis patients quality of life, a decrease in its overall level is noted due to almost all components, but physical activity shows the lowest values in some studies, which is associated with joint damage, activity, and duration of the disease. Objective. To determine the state of physical functioning and assess the overall level of quality of life in patients with JIA, considering the subtype of the disease duration and the therapy complex. Materials and Methods. The study was carried out at SI "Institute for Children and Adolescents Health Care of the NAMS of Ukraine", Kharkiv, from November 2020 till November 2021. There 118 patients with juvenile idiopathic arthritis were examined, including 47 with polyarticular, 43 with oligoarticular, 28 with uveitis-associated subtypes. The investigation involved 77 girls and 41 boys in age from 2 till 18 years old. The therapy by methotrexate was provided in 111 patients, among them 30 had methotrexate with immunobiological therapy (29 adalimumab, 1 – tocilizumab), 6 – sulfasalazine. The disease duration due to disease subtype was in children with polyarthritis – (49,2±6,7), oligoarthritis – (35,4±4,2), uveitis-associated subtypes of juvenile idiopathic arthritis – (76,8±10,2) months. Disease activity was assessed using the Juvenile Arthritis Disease Activity Score 27-joint reduced count questionnaire, functional state according to he Child Health Assessment Questionnaire and quality of life according to PedsQLTM 4.0 Generic Core Scales. Results. It was found that high juvenile idiopathic arthritis activity was observed in 31 (26.2%) patients, equally often in all subgroups of children. Index functional state did not show a significant decrease either in the whole group or in each of the arthritis subgroups. The overall indicator of quality of life in children with juvenile idiopathic arthritis was reduced in the whole group (71.2±1.4 and 72.9±1.4 per week and month). There was no significant difference between the quality of life indicators of boys and girls. at the same time, physical activity indicators were the lowest in the group with polyarticular juvenile idiopathic arthritis, regardless of gender and age of children, and especially low in children with polyarthritis in the first year of the disease. They also turned out to be worse in children with uveitis-associated subtypes of juvenile idiopathic arthritis with the disease from one to three years. The highest level of the physical component of quality of life was observed in children with oligoarthritis older than 14 years and in children under 8 years of age in the uveitis-associated subtypes of juvenile idiopathic arthritis group. There was no significant effect on the physical indicators of quality of life of the start treatment timing. (р≤0,05). Conclusions. A decrease in quality of life and its physical component is typical for children with different types of juvenile idiopathic arthriti (oligoarticular, polyarticular, and uveitis-associated subtypes of juvenile idiopathic arthritis). It has been established that children with polyarticular subtype of arthritis have the greatest decrease in quality of life and physical functioning.

P013 Serum osteoprotegerin and RANKL in patients with Juvenile Idiopathic Arthritis and their correlation with bone mineral density

Abstract Background Juvenile idiopathic arthritis (JIA) is the most common chronic rheumatic arthropathy of childhood and is associated with low bone mass, and may hasten the onset of osteoporosis later in life1. Bone loss occurs because of an imbalance between osteoclasts-activating factors like receptor activator of nuclear factor-κB ligand (RANKL) and its inhibitor osteoprotegerin (OPG) 2. Dual energy X-ray absorptiometry (DXA) is the preferred method for measuring bone mineral density (BMD) in children and to identify and follow individuals at risk for fracture 3. The objective is the Evaluation of serum levels of osteoprotegerin and RANKL and their correlation with BMD in JIA patients. Methods Forty JIA patients (according to the revised classification criteria of ILAR) and 40 healthy children individually matched for age, sex and race were included in this study. Children excluded from the study were those with primary and secondary causes of osteoporosis (such as chronic illness). All patients were assessed clinically by: age, sex, body mass index, type of JIA, disease duration and disease activity (by Juvenile Arthritis Disease Activity Score; JADAS 10). The functional disability was assessed by the Childhood Health Assessment Questionnaire (CHAQ). Blood samples were collected from JIA patients and healthy controls to determine serum levels of OPG and RANKL by ELISA. DXA scans were done using GE Healthcare Lunar DPX, Madison, Wisconsin. Bone mineral density of the L1-L4 lumbar spine and total body less head (TBLH) was evaluated in g/cm2 and expressed as Z score for age, sex according to the reference data given for this equipment. Results The study included 40 patients (25 females) with a mean age of 11.14 years and median disease duration of 2.5 years. As regard JIA type, 45% of patients were oligoarticular, 32.5% were polyarticular, and 22.5% were systemic JIA. Median JADAS 10 was 13.95. Patients (especially polyarticular JIA) had significantly higher serum RANKL levels and lower serum OPG and OPG/RANKL ratio when compared with controls (with p-value &amp;lt;0.001, 0.032 and &amp;lt;0.001 respectively). A diagnosis of low BMD (BMD Z-score ≤ -2) was given in 25% of patients (15% polyarticular and 10% systemic) by DXA of lumbar spine, and 20% (10% polyarticular and 10% systemic) by DXA of TBLH. On the other hand, no patient was given a diagnosis of osteoporosis (BMD Z-score ≤ -2 and a significant fracture history). Low BMD at lumbar spine and TBLH was negatively correlated with serum RANKL while positively correlated with OPG/RANKL ratio. Moreover, low BMD at lumbar spine was positively correlated with serum OPG level Conclusion High RANKL and low OPG levels appear to be associated with low bone mass in JIA patients. Patients with JIA (especially polyarticular and systemic subtype) are at increased risk of low bone mineral mass. Disclosure of Interests None declared

Low-energy laser therapy application on knee joints as an auxiliary treatment in patients with polyarticular juvenile idiopathic arthritis: a dual-arm randomized clinical trial.

Patients with juvenile idiopathic arthritis (JIA) always experience persistent pain and stiffness which induces muscle weakness, fatigue, and functional limitations. This study evaluated whether applying low-energy laser therapy (LLT) on the knee joint could be an effective adjuvant intervention for patients with JIA. Sixty children with polyarticular JIA participated and were randomly allocated to receive either LLT (wavelength λ = 903nm; power output of 50 mW; and energy of 1.5J) plus exercises (LLT group) or exercises alone (control group). Pain, peak concentric torque of quadriceps muscles, fatigue, and functional status were measured by the visual analogue scale, isokinetic testing system, Pediatric Quality of Life Inventory Multidimensional Fatigue Scale, and Childhood Health Assessment Questionnaire, respectively pre- and post-intervention, and at 6-month follow-up. Per the mixed-model analysis of variance, the LLT group showed a statistically more favorable improvement in pain (P = .003, ηp2 = .014), fatigue perception (P = .004, ηp2 = .015), and functional status (P = .022, ηp2 = .09) across the three assessment occasions, as compared to the control group. However, no significant difference was demonstrated between both groups concerning peak concentric torque (all P > .05). Incorporation of LLT into the standard physical rehabilitation program for patients with JIA has the potential to induce more conducive improvements in pain, fatigue, and functional performance, but is not effective for improving muscle performance.