Caregiver Priorities And Child Health Index Of Life With Disabilities Research Articles

Development of algorithms for estimating the Child Health Utility 9D from Caregiver Priorities and Child Health Index of Life with Disability.

The primary aim was to determine Child Health Utility 9D (CHU9D) utilities from the Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD) for non-ambulatory children with cerebral palsy (CP). One hundred and eight surveys completed by Australian parents/caregivers of children with CP were analysed. Spearman's coefficients were used to investigate the correlations between the two instruments. Ordinary least square, robust MM-estimator, and generalised linear models (GLM) with four combinations of families and links were developed to estimate CHU9D utilities from either the CPCHILD total score or CPCHILD domains scores. Internal validation was performed using 5-fold cross-validation and random sampling validation. The best performing algorithms were identified based on mean absolute error (MAE), concordance correlation coefficient (CCC), and the difference between predicted and observed means of CHU9D. Moderate correlations (ρ 0.4-0.6) were observed between domains of the CHU9D and CPCHILD instruments. The best performing algorithm when considering the CPCHILD total score was a generalised linear regression (GLM) Gamma family and logit link (MAE=0.156, CCC=0.508). Additionally, the GLM Gamma family logit link using CPCHILD comfort and emotion, quality of life, and health domain scores also performed well (MAE=0.152, CCC=0.552). This study established algorithms for estimating CHU9D utilities from CPCHILD scores for non-ambulatory children with CP. The determined algorithms can be valuable for estimating quality-adjusted life years for cost-utility analysis when only the CPCHILD instrument is available. However, further studies with larger sample sizes and external validation are recommended to validate these findings.

Cannabidiol Use Patterns and Efficacy for Children Who Have Cerebral Palsy.

Cannabidiol (CBD)-containing supplements are used by children with cerebral palsy (CP), but the prevalence and efficacy of their use have not been studied. We sought to describe CBD use patterns and perceived efficacy in the pediatric population with CP, evaluating any association between CBD use and health-related quality of life. Patients with CP were prospectively enrolled, and caregivers were offered the Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD) Questionnaire and a survey assessing CBD use. Of 119 participants, 20 (16.8%) endorsed CBD use (CBD+) and 99 (83.2%) denied it (CBD-). Participants in the CBD+ group had worse functional status (85% Gross Motor Function Classification System level IV-V for CBD+ vs 37.4% for CBD-, P<.001) and lower health-related quality of life (mean CPCHILD score of 49.3 for CBD+ vs 62.2 for CBD-, P=.001). Spasticity was the rationale most cited for CBD use (29%), followed by pain and anxiety (both 22.6%). CBD was perceived to be most effective for improving emotional health, spasticity, and pain. Fifty percent of the patients in the CBD+ group underwent surgery in the previous 2 years and most endorsed a general benefit in the postoperative setting. The most common side effects noted were fatigue and increased appetite (both 12%). Most participants endorsed no side effects (60%). CBD may serve as a useful adjunct for some children with CP, especially those with worse disease severity. Caregivers perceive CBD as offering some benefits, particularly in the domains of emotional health, spasticity, and pain. We found no evidence of severe adverse events in our small cohort. [Orthopedics. 2024;47(1):52-56.].

Cerebralis paresisben szenvedő gyermekek egészségindexe

Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD) questionnaire is a measurement tool for assessing health status and wellbeing of disabled children, which evaluates children's quality of life from the caregiver point of view. The aim of our work was the Hungarian translation and cultural adaptation of the questionnaire and also validation of the CPCHILD on Hungarian cerebral palsy patients. Further aim was to test the eligibility of the questionnaire for superficial assessment of caregiver's psychological attitudes. Translation of the questionnaire was carried out according to the Beaton's guidelines. Test-retest, interrater reliability (ICC) and also internal consistency (Cronbach-alpha) were calculated for reliability. The importance of the questions was assessed for face validity and known group validity test was done to measure construct validity. For examining parental attitudes, the patients were divided into ambulatory and non-ambulatory groups and the 36 quality of life questions of the 7th domain were used to find relations. During test-retest reliability measurements, the ICC was 0.96 (95% CI: 0.88-0.98), and Cronbach-alpha exceeded the minimal expected value of 0.7 (0.74-0.97) except in the 5th domain (0.67), while measuring interrater reliability the ICC was 0.87 (95% CI 0.70-0.94). Face validity was above the 2.0 threshold in every question (2.6-4.5; mean: 3.4 ± 1.34) and the known group validity calculations showed significant differences between the CPCHILD scores of ambulatory and non-ambulatory groups. Examining parental attitudes, a significant difference was also shown among the parents of ambulatory and non-ambulatory children in assessing the importance of sitting in the quality of life of their children (2.89 ± 1.28 vs. 3.51 ± 0.82; p<0.01). The final outcome of our study is that CPCHILD questionnaire has become widely accessible in Hungarian language. Our result, that the answers referring to the sitting abilities and the activities should be carried out in sitting position, was significantly different among the caregivers of the ambulatory and non-ambulatory children, showing that the parents of the GMFCS IV and V category children evaluate the importance of sitting ability higher compared to those parents who care for GMFCS I, II and III category children. Furthermore, the results draw attention to the wellbeing and health of the children measurable with CPCHILD as well as that parental caregiver attitudes can be recognized which may give further help in finding the balance between expectations and possibilities during the rehabilitation of cerebral palsy children. Orv Hetil. 2023; 164(16): 610-617.

Improved Sitting Balance in Children with Cerebral Palsy: Body Functions to Activity and Participation

AbstractThe aim of this study is to evaluate the changes in activity and participation domains of International Classification of Functioning (ICF), Disability and Health, in children with cerebral palsy (CP) who received integrated botulinum toxin A (BoNT-A) treatment with intensive rehabilitation with a treatment goal of improved sitting balance. In this prospective observational study, 29 patients with CP (mean age: 6.7 ± 3.8 years) and the Gross Motor Function Classification System (GMFCS) levels of IV and V were included. The primary outcome measures were the mean changes from baseline to posttreatment weeks 10 to 12 of the Child and Adolescent Scale of Participation (CASP) and Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD). The modified Ashworth scale (MAS) and Tardieu scale (TS) results at 6 to 8 weeks of posttreatment were the secondary outcome measures. Statistically significant improvements in home and community participation of CASP (p &lt; 0.001 and 0.001), CPCHILD (p &lt; 0.01), MAS (p &lt; 0.001), and all parameters of TS (p &lt; 0.05) were recorded after treatment. The result of this prospective, observational study showed that the functional improvements in sitting balance by the integrated BoNT-A treatment provides positive effects on activity and participation levels of the patient and quality of life of patients and their families.

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.

Foam Splint versus Spica Cast-Early Mobilization after Hip Reconstructive Surgery in Children-Preliminary Data from a Prospective Randomized Clinical Trial.

Background: Surgical hip joint reconstruction may be the method of choice for children and adolescents with developmental dysplasia of the hip (DDH), as well as neurogenic dislocation of the hip (NDH) and Legg–Calvé–Perthes disease (LCPD). Following pelvic surgery, immobilization using a spica cast is considered to be the gold standard, despite the fact that casting may cause complications, such as hygienic problems, skin lesions, neurological deficits, and rigidity of the adjacent joints. An alternative for postoperative immobilization is a foam splint. The purpose of this randomized controlled trial was to compare spica cast and foam splint immobilization after hip reconstruction in children and adolescents with DDH, NDH, and LCPD. Methods: In a prospective randomized clinical trial, children and adolescents (age: 4–14 years), who received hip reconstructive surgery (osteotomy of the ilium and proximal femur, open reduction, soft tissue techniques) for DDH, NDH, and LCPD were included. Patient recruitment, group allocation, surgery, and aftercare were carried out in a department for orthopaedic surgery in Central Europe. Standardized questionnaires SF-36 (Short Form-36), EQ-5D (Euro Quality of Life 5D and CPCHILD (Caregiver Priorities and Child Health Index of Life with Disabilities) were gathered before, six, and twelve weeks after surgery from each patient. Group one received a spica cast and group two a foam splint for a period of six weeks postoperatively. There was no difference in surgical treatment. Results: Twenty-one out of thirty planned patients were enrolled in the study. One patient had to be excluded because of a lack of compliance. All quality of life (QOL) scores showed a significant reduction at the 6-week follow-up compared to the preoperative assessment. After twelve weeks, the scores came back close to the preoperative values. A significant reduction was seen in the spica cast group pre- vs. postoperatively for the variables CPCHILD (81% vs. 64%, p = 0.001), EQ-5d (65% vs. 45%, p = 0.014), and SF-36 (85% vs. 74%, p = 0.004). The corresponding values for the foam splint group also presented a reduction for all scores, but without statistical significance. Complications occurred in five cases. Conclusions: Recent retrospective studies suggest that foam splint immobilization after hip reconstruction surgery is a safe and feasible method, promising fewer complications compared to spica casting. The preliminary results of this prospective randomized clinical trial show an improvement of the scores when using a foam splint compared to the conventionally used spica cast. Benefits for the patients may be fewer adverse events and no need to undergo a second round of anaesthesia for recasting. Data suggest higher patient and caretaker satisfaction in the foam splint group.

Dynamic Standing Exercise in a Novel Assistive Device Compared with Standard Care for Children with Cerebral Palsy Who Are Non-Ambulant, with Regard to Quality of Life and Cost-Effectiveness

Children with cerebral palsy who are non-ambulant cannot stand or walk independently. In Sweden, daily static standing is part of standard care for these children even though it is time-consuming and costly, and while it may benefit the well-being of many children, it is inconvenient for others. A novel stander enables dynamic standing through walking movements. The aim of this study was to compare dynamic and static standing regarding quality of life and cost-effectiveness. Twenty children with cerebral palsy who were non-ambulant were included in this randomized controlled study with a cross-over design. Mean age of the participants was 11.6 ± 3.6 years, nine were female. They participated in four months of static and four months of dynamic standing. Quality of life was assessed with the Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD), which has been shown to be valid and reliable, and is tailored to children with severe cerebral palsy. Information about other aspects of quality of life and about cost-effectiveness was collected by study-specific questionnaires. Dynamic standing was the preferred alternative from the family perspective, as it cost less (€64, p &lt; 0.01) and had greater benefits than standard (static standing) care. From societal and healthcare provider perspectives, dynamic standing had benefits but higher costs than static standing (€290 and €354 respectively, p &lt; 0.01). These findings may contribute to the development of individualized standing recommendations.

Disease Severity and Motor Impairment Correlate With Health-Related Quality of Life in AP-4-Associated Hereditary Spastic Paraplegia.

ObjectiveAP-4-associated hereditary spastic paraplegia (AP-4-HSP) is a childhood-onset neurogenetic disease and mimic of cerebral palsy. Data on health-related quality of life (HRQoL) are lacking. To establish a metric for HRQoL and caregiver priorities, we used the Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD) questionnaire to assess HRQoL in correlation with disease severity in 64 patients with AP-4-HSP.MethodsA cross-sectional analysis of caregiver-reported HRQoL was performed using the CPCHILD questionnaire in combination with a detailed clinical characterization.ResultsHRQoL was impaired in all domains in patients with AP-4-HSP (mean score: 59.6 ± 12.6 [SD]), with no significant difference between the 4 subtypes. Age, as a surrogate for disease duration, and Spastic Paraplegia Rating Scale scores, as an indicator for corticospinal tract dysfunction and motor impairment, correlated with lower CPCHILD scores (Pearson r = −0.31, p = 0.01 and r = −0.52, p < 0.0001, respectively). Patients with tetraplegia showed lower CPCHILD scores compared with individuals with diplegia or no spasticity. Wheelchair dependence reduced HRQoL in all domains. The presence of seizures, including medically refractory epilepsy, was not associated with lower CPCHILD scores. Standardized assessment of caregiver priorities identified several areas of high importance to HRQoL.ConclusionsWe show that the CPCHILD questionnaire, developed for use in children with cerebral palsy, can be used to assess HRQoL in patients with childhood-onset complex hereditary spastic paraplegia. HRQoL is reduced in patients with AP-4-HSP and correlates with the degree of motor impairment. These results provide a framework for medical decision making and a baseline for the future development of treatment guidelines and interventional trials.

Correlation between hand functioning, cognition and quality of life of CP children: 2-year prospective randomized study

IntroductionFor treatment of CP patients multidisciplinary approach is often promoted. But it is not cost-efficient to involve all medical staff and variety of diagnostic/intervention tools for every patient.ObjectivesHaving knowledge on pivotal manifestations of CP and what are they related to would ease multidirectional approach in practice. It will shorten a search of pathologies only to those related to main manifestations related to quality of life.Methods611 children aged 2 to 18 years (mean age–6y5m) with CP(G80.0-G80.9): examined by 3 independent doctors. 56%males, 44%females. Randomized blinded assessment. Fine hand function assessed by “9-hole peg”,“Box and Blocks” tests, dynamometry. Intellectual functioning assessment was done using Raven Matrices. Quality of Life(QoL) assessment according to Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD). The intraclass correlation coefficient(ICC) was used for finding out discrepancies between observers. Inferencial statistics including 95%CI and P-value.ResultsICC coefficient between observers was highly reliable–0.93(95%CI:0.89–0.95). The mean QoL score for children GMFCS levels I and II was 58.5(SD 16.6), for GMFCS III, IV and V children–22.4(SD13.4). QoL was more related to fine hand functioning(r=0,344) than to cognition(r=0,295). There was a strong correlation bond between fine hand functioning and cognition (r=0,663). In case fine hand function improved positive changes in cognition were observed in 74% of subjects(p&lt;0,05). Correlation between grasp power and IQ was weak(r=0,184). Grasp power improvement was slightly related to QoL(r=0,102).ConclusionsFine hand functioning improves QoL even more that cognition.So training of fine motor skills should be given a priority in case of limited rehabilitation resources.DisclosureNo significant relationships.

The Impact of Oral-Gut Inflammation in Cerebral Palsy.

Background: Oral-gut inflammation has an impact on overall health, placing subjects at risk to acquire chronic conditions and infections. Due to neuromotor disturbances, and medication intake, cerebral palsy (CP) subjects present intestinal constipation, impacting their quality of life (QOL). We aimed to investigate how oral inflammatory levels predicted gut phenotypes and response to therapy.Methods: A total of 93 subjects aging from 5 to 17 years were included in the study, and assigned into one of the 4 groups: CP with constipation (G1, n = 30), CP without constipation (G2, n = 33), and controls without CP with constipation (G3, n = 07) and without CP and without constipation (G4, n = 23). In addition to characterizing subjects' clinical demographics, medication intake, disease severity levels, salivary cytokine levels [TNF-α, interleukin (IL)-1β, IL-6, IL-8, IL-10], and Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD). Statistical significance was evaluated by Shapiro-Wilks, Student's T-Test, ANOVA, and ANCOVA analysis.Results: Salivary proinflammatory cytokines were highly correlated with the severe form of gut constipation in G1 (P < 0.001), and out of all cytokines IL-1β levels demonstrated highest correlation with all gut constipation (P < 0.05). A significant relationship was found between the type of medication, in which subjects taking Gamma-Aminobutyric Acid (GABA) and GABA+ (GABA in association with other medication) were more likely to be constipated than the other groups (P < 0.01). Cleary salivary inflammatory levels and gut constipation were correlated, and impacted QOL of CP subjects. G1 presented a lower QOL mean score of CPCHILD (49.0 ± 13.1) compared to G2 (71.5 ± 16.7), when compared to G3 (88.9 ± 7.5), and G4 (95.5 ± 5.0) (P < 0.01). We accounted for gingival bleeding as a cofounder of oral inflammation, and here were no differences among groups regarding gender (P = 0.332) and age (P = 0.292).Conclusions: Collectively, the results suggest that saliva inflammatory levels were linked to gut constipation, and that the clinical impact of medications that controlled gut was reliably monitored via oral cytokine levels, providing reliable and non-invasive information in precision diagnostics.

Of Major Complication Types, Only Deep Infections After Spinal Fusion Are Associated With Worse Health-related Outcomes in Children With Cerebral Palsy.

Retrospective review. The aim of this study was to determine whether major postoperative complications ("complications") are associated with 2-year improvements in Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD) scores after scoliosis surgery, and whether complications and preoperative characteristics predict 2-year improvements in CPCHILD Total score. Spinal arthrodesis can halt the progression of spinal deformity in patients with cerebral palsy (CP)-related scoliosis. However, these patients are prone to postoperative complications. Using a multicenter CP registry, we identified 222 patients aged ≤21 years who underwent spinal fusion from 2008 to 2015 and had ≥2-year follow-up. We compared CPCHILD score improvement between 71 patients who had 1 or more complications ("complications group") versus 151 who did not ("no-complications group"). Complications were deep infections, thromboembolic events, and cardiopulmonary, gastrointestinal, and neurologic complications. Multiple linear regression was used to identify predictors of 2-year postoperative CPCHILD score improvement (alpha = 0.05). At 2-year follow-up, the complications group had similar mean improvement in CPCHILD score across all domains compared with the no-complications group (P > 0.05). When stratifying by complication type, deep infection was associated with less improvement in CPCHILD Comfort and Emotions (P = 0.02), Quality of Life (P < 0.01), and Total (P = 0.04) scores. When controlling for Gross Motor Function Classification System subcategory, age, and body mass index, only preoperative CPCHILD Total score and postoperative deep infection (F[4, 176] = 14; P < 0.0001; R = 0.24) predicted 2-year improvement in CPCHILD Total score. Higher preoperative Total score and postoperative deep infection independently predicted less improvement in Total score. Postoperative deep infection and higher preoperative CPCHILD Total score independently predicted less improvement in CPCHILD Total score. Other major postoperative complications were not associated with differences in 2-year postoperative improvements in CPCHILD scores across all domains. 3.

Early and late hospital readmissions after spine deformity surgery in children with cerebral palsy

Retrospective review of a prospectively collected multicenter registry of pediatric patients with cerebral palsy (CP) and neuromuscular scoliosis (NMS) undergoing spinal fusion. To define risk factors for unplanned readmission after elective spinal deformity surgery. Patients with CP and NMS have high rates of hospital readmission; however, risk factors for readmission are not well established. Univariate and multivariate analyses were used to compare the demographics, operative and postoperative course, radiographic characteristics, and preoperative Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD) questionnaires of patients who did not require readmission to those who required either early readmission (within 90days of the index surgery) or late readmission (readmission after 90days). Of the 218 patients identified, 19 (8.7%) required early readmission, while 16 (7.3%) required late readmission. Baseline characteristics were similar between the three cohorts. On univariate analysis, early readmission was associated with longer duration of surgery (p < 0.001) and larger magnitude of residual deformity (p = 0.003 and p = 0.029 for postoperative major and minor angles, respectively). The health score of the CPCHILD Questionnaire was lower in patients who required early readmission than in those who did not require readmission (p = 0.032). On multivariate analysis, oral feeding status was inversely related to early readmission (less likely to require readmission), while decreasing lumbar lordosis and increasing length of surgery were related to an increased likelihood of early readmission. In patients with CP and NMS, longer surgical time, larger residual major and minor Cobb angles, lumbar lordosis, feeding status, and overall health may be related to a greater likelihood for early hospital readmission after elective spinal fusion. No factors were identified that correlated with an increased need for late hospital readmission after elective spinal fusion in patients with CP. IV.

Improving Health-related Quality of Life for Patients With Nonambulatory Cerebral Palsy: Who Stands to Gain From Scoliosis Surgery?

It is unclear what factors influence health-related quality of life (HRQOL) in neuromuscular scoliosis. The aim of this study was to evaluate which factors are associated with an improvement in an HRQOL after spinal fusion surgery for nonambulatory patients with cerebral palsy (CP). A total of 157 patients with nonambulatory CP (Gross Motor Function Classification System IV and V) with a minimum of 2-year follow-up after PSF were identified from a prospective multicenter registry. Radiographs and quality of life were evaluated preoperatively and 2 years postoperatively. Quality of life was evaluated using the validated Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD) questionnaire. Patients who had an increase of 10 points or greater from baseline CPCHILD scores were considered to have meaningful improvement at 2 years postoperatively. 10 points was chosen as a threshold for meaningful improvement based on differences between Gross Motor Function Classification System IV and V patients reported during the development of the CPCHILD. Perioperative demographic, clinical, and radiographic variables were analyzed to determine predicators for meaningful improvement by univariate and multivariate regression analysis. A total of 36.3% (57/157) of the patients reported meaningful improvement in CPCHILD scores at 2 years postoperatively. Preoperative radiographic parameters, postoperative radiographic parameters, and deformity correction did not differ significantly between groups. Patients who experienced meaningful improvement from surgery had significantly lower preoperative total CHPILD scores (43.8 vs. 55.2, P<0.001). On backwards conditional binary logistic regression, only the preoperative comfort, emotions, and behavior domain of the CPCHILD was predictive of meaningful improvement after surgery (P≤0.001). Analysis of 157 CP patients revealed a meaningful improvement in an HRQOL in 36.3% of the patients. These patients tended to have lower preoperative HRQOL, suggesting more "room for improvement" from surgery. A lower score within the comfort, emotions, and behavior domain of the CPCHILD was predictive of meaningful improvement after surgery. Radiographic parameters of deformity or curve correction were not associated with meaningful improvement after surgery. Level II-retrospective review of prospectively collected data.

A Prospective, Crossover Survey Study of Child- and Proxy-Reported Quality of Life According to Spinal Muscular Atrophy Type and Medical Interventions.

Spinal muscular atrophy is an autosomal-recessive, progressive neuromuscular disease associated with extensive morbidity. Children with spinal muscular atrophy have potentially increased life spans due to improved nutrition, respiratory support, and novel pharmaceuticals. To report on the quality of life and family experience for children with spinal muscular atrophy with attentiveness to patient- and proxy-concordance and to stratify quality of life reports by spinal muscular atrophy type and medical interventions. A prospective, crossover survey study inclusive of 58 children (26 spinal muscular atrophy type I, 23 type II, 9 type III) and their family caregivers at a free-standing Midwestern children's hospital. Twenty-eight families completed the 25-item PedsQL 3.0 Neuromuscular Module. Forty-four participants completed the 36-item PedsQL Family Impact Module and 47 completed the Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD) questionnaire. The PedsQL Family Impact Module demonstrated significant differences between spinal muscular atrophy types I and II in functioning domains including physical, emotional, social, and family relations (P < .03). Child self-report and proxy report surveys demonstrated significant differences between spinal muscular atrophy types in the communication domains (P < .003). Children self-reported their quality of life higher than proxy report of child quality of life. Gastrostomy tube (P = .001) and ventilation support (P = .029) impacted proxy-reported quality of life perspectives, whereas nusinersen use did not. Spinal surgery was associated with improved parental quality of life and family impact (P < .03). The measurement and monitoring of quality of life for children with spinal muscular atrophy and their families represents an implementable priority for care teams.

Longitudinal validation of the Caregiver Priorities and Child Health Index of Life with Disabilities in a Dutch sample of nonambulatory children with severe disabilities.

The purpose of this study was to evaluate the longitudinal validity of the Dutch version of the Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD), a health-related quality of life instrument for nonambulatory children with severe motor disabilities and accompanying disorders. The effect of two interventions, Botox injections in the hip region and percutaneous endoscopic gastrostomy, was followed over time. Caregivers (n=38) of nonambulatory children (26 boys, 12 girls; mean age: 9years, 5months [4years, 9months]) with severe disabilities completed the questionnaire prior to the intervention, at 3months and 6months follow up. Seven a priori hypotheses were formulated. Longitudinal validity was analysed by a paired t test of the pre-post scores and correlation analysis between the change-scores and two external criteria: a caregivers' perceived change in health-related quality of life of the child questionnaire and a general health-related quality of life instrument. The results reported here follow completely the pattern we hypothesized for four analyses and partially in the remaining three. In the Botox group, the mean change-score at 3months was 6.9 points (p<0.05), which exceeds the minimal clinically important difference of 5.8 points. At 6months, the effect was diminished to 4.5 points, in line with the temporary effect of Botox. There were moderate positive correlations between the change-scores and an external criterion (Spearman's rho: 0.46-0.58). This study indicates that the CPCHILD Dutch version has sufficient longitudinal validity statistically and clinically in nonambulatory children with severe disabilities.

Development and content validation of the Muscular Dystrophy Child Health Index of Life with Disabilities questionnaire for children with Duchenne muscular dystrophy.

To develop a patient-reported outcome measure that comprehensively captures the health-related priorities of children with Duchenne muscular dystrophy (DMD). Children with DMD and their parents completed the iteratively revised versions of the Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD), followed by a cognitive interview to develop a pilot version of a new measure. Multidisciplinary health care professionals completed an item-by-item analysis of the measure and a 14-item sensibility questionnaire. Minimum content validity ratio for each item of the new measure and the mean score (0-7) for the items of the sensibility questionnaire were calculated. The CPCHILD underwent changes over 19 interviews with children and their parents, resulting in the pilot Muscular Dystrophy Child Health Index of Life with Disabilities (MDCHILD). The content validity ratio of each MDCHILD item ranged from 0.85 to 1 based on health care professionals' ratings. The mean score exceeded the threshold of four for all items of the sensibility questionnaire. Based on child, parent, and health care professional recommendations, 16 items were added, six eliminated, and 15 items modified from the original CPCHILD. The MDCHILD consists of 47 items over seven domains. The MDCHILD metall sensibility criteria by children with DMD, their parents, and health care professionals, and is ready for psychometric evaluation. The Muscular Dystrophy Child Health Index of Life with Disabilities (MDCHILD) is a new patient-reported outcome measure for Duchenne muscular dystrophy (DMD). The Priority Framework of Outcomes underpins the content for the MDCHILD. The MDCHILD incorporates the health-related priorities of males with DMD and their parents. The MDCHILD was deemed sensible by children, their parents, and health care professionals.

Assessing the Risk-Benefit Ratio of Scoliosis Surgery in Cerebral Palsy: Surgery Is Worth It.

The true benefits of scoliosis surgery in cerebral palsy (CP) remain uncertain. Our aims were to determine the benefits of spinal fusion according to health-related quality of life (HRQoL) improvement at long-term follow-up and to explore the effect of surgery-related complications on clinical outcomes. The cases of consecutive patients who had Gross Motor Function Classification System (GMFCS) level-IV or V cerebral palsy with 5-year follow-up from a prospective, longitudinal, multicenter database were analyzed. Caregivers completed the Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD) questionnaire and 4 Likert-type anchor questions preoperatively and at 1, 2, and 5 years of follow-up. Data on complications were collected prospectively. Preoperative CPCHILD scores were compared with postoperative scores at the 1, 2, and 5-year follow-up evaluations. Preoperative CPCHILD scores were compared with postoperative scores at the 1, 2, and 5-year follow-up evaluations using repeated-measures analysis of variance (ANOVA). Spearman correlation coefficient was used to explore the association between changes in the CPCHILD at 1, 2, and 5-year follow-up and the reported complications within the follow-up period. Similarly, a comparative analysis between the percentage distribution of the answers to the 4 anchor questions and the reported complications was also performed. Sixty-nine patients with a mean age (and standard deviation) of 13.4 ± 2.6 years at enrollment were analyzed. The major Cobb angle was a mean of 81.9° ± 26.7° preoperatively and improved to a mean of 28.7° ± 14.4° at 2 years and 30.7° ± 15.3° at 5 years postoperatively. Significant improvements in CPCHILD personal care, positioning, and comfort domains were noted at all time points. The mean increase in the total score was 7.19 (p < 0.001) at 1 year, and the score gain was maintained at 2 and 5 years postoperatively. The overall complication rate was 46.4% at 1 year, 1.4% between 1 and 2 years, and 4.3% at 2 to 5 years postoperatively, with surgical intervention required in 6 patients within 1 year and in 2 additional patients within 5 years following scoliosis surgery. There was no correlation between complications and CPCHILD scores postoperatively at all time points, with the only exception of a weak correlation (ρ = -0.450, p = 0.002) with CPCHILD comfort score at 1 year after surgery. Scoliosis surgery in patients with CP leads to a significant improvement in HRQoL, which is maintained 5 years following surgery. The substantial complication rate does not correlate with HRQoL changes postoperatively, suggesting that the benefits of surgery outweigh the risks in this fragile population. Therapeutic Level IV. See Instructions for Authors for a complete description of levels of evidence.

Psychometric evaluation of the Scandinavian version of the caregiver priorities and child health index of life with disabilities

Purpose: To examine test–retest reliability and construct validity of the Scandinavian version of the caregiver priorities and child health index of life with disabilities (CPCHILD) questionnaire for children with cerebral palsy (CP).Methods: Families were recruited in Sweden and Norway and stratified according to the gross motor function classification system levels I–V for children born 2000–2011, mean age 7.9 (SD 3.2). Construct validity based on the first questionnaire (n = 106) was evaluated for known groups, using linear regression analysis. Intraclass correlation coefficient was used to estimate test–retest reliability (n = 64), and Cronbach’s alpha was calculated as an indicator of internal consistency.Results: The questionnaire showed construct validity and the ability to discriminate between levels of gross motor function for the total score and all domain scores (p < 0.05). Test–retest reliability was high with intraclass correlation coefficient of 0.92 for the total score and of 0.72–0.92 for the domain scores. Cronbach’s alpha was 0.96 for the total score and 0.83–0.96 for the domain scores.Conclusions: The Scandinavian version of the CPCHILD for children with CP seems to be a valid and reliable proxy measure for health related quality of life.Implications for rehabilitationValid and reliable outcome measures are needed to evaluate whether follow-up programs enhance health related quality of life in different countries.The Scandinavian version of the caregiver priorities and child health index of life with disabilities (CPCHILD) was evaluated for known-groups validity and test–retest reliability.The Scandinavian version of the CPCHILD is a sound and valid measurement for evaluation and comparison of health related quality of life of children with cerebral palsy in different countries.

Improvement in Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD) scale after deep brain stimulation (DBS) in childhood

Improvement in Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD) scale after deep brain stimulation (DBS) in childhood

Recurrence of Deep Surgical Site Infection in Cerebral Palsy After Spinal Fusion Is Rare

Study DesignRetrospective review of prospective registry. ObjectivesTo assess the following in children with cerebral palsy (CP) who develop deep surgical site infection (DSSI) after spinal fusion: (1) rate of infection recurrence after treatment; (2) treatments used; (3) radiographic outcomes; and (4) differences in Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD) scores versus those of children with no infection (NI). Summary of Background DataStudies show high rates of surgical site infection in patients with CP but do not address late recurrence or quality-of-life effects. MethodsOne hundred fifty-one children with CP underwent spinal fusion surgery from 2008 through 2011 and had ≥2-year follow-up. Patients who developed DSSI were compared with patients with NI. Student t tests were used to analyze deformity; analysis of variance was used to analyze CPCHILD scores in both groups preoperatively and at final follow-up. ResultsEleven patients developed DSSI. Causative organisms were polymicrobial infection (5 cases), Escherichia coli (2 cases), and Proteus mirabilis, Staphylococcus aureus, Enterococcus faecalis, and Peptostreptococcus (1 case each). All patients underwent irrigation and debridement and received at least 6 weeks of antibiotics. Six had negative-pressure-dressing-assisted wound closure; 5 had primary closure. At mean 4-year follow-up (range, 3–5 years) no patient had recurrent infection. From immediate postoperative to final follow-up, no patient had significant loss of coronal curve (p = .77) or pelvic obliquity (p = .71) correction. However, at final follow-up, comfort and emotions, overall quality-of-life, and total CPCHILD scores in the DSSI group were significantly lower compared with the NI group (p = .005, .022, and .026, respectively). ConclusionsIn children with CP who developed DSSI after spinal fusion, there was no recurrence of infection or deformity after infection treatment. CPCHILD scores in patients with DSSI were lower compared with the NI group.