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

PURPOSE: 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). METHODS: 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. RESULTS: 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). CONCLUSION: 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.

Current trends in surgical magnitude of neuromuscular scoliosis curves: a study of 489 operative patients with non-ambulatory cerebral palsy.

PURPOSE: A curve magnitude at which posterior spinal fusion (PSF) is indicated for children with cerebral palsy (CP) scoliosis is not defined. We sought to evaluate whether agreement exists for a curve magnitude at which PSF is undertaken for CP scoliosis and to evaluate outcomes by quartile of curve magnitude and flexibility at time of fusion. METHODS: A prospective multicenter pediatric spine database was queried for patients with a Gross Motor Function Classification Scale (GMFCS) IV or V who underwent PSF for CP scoliosis. Demographics, surgical indications, and correlations between curve magnitude, postoperative radiographic outcomes, and Caregiver's Priorities and Child Health Index of Life and Disabilities (CPCHILD) scores were evaluated for patients with at least 2 years of follow-up. RESULTS: 489 patients from 15 sites were analyzed. Median major Cobb angle at time of PSF was 87° and significantly varied by site (p < 0.001). Median Cobb angle on flexibility studies was 55° and median percent correction on flexibility studies was 36.3%. Severity of the curve at surgery correlated significantly with lower overall quality of life and CPCHILD score (p < 0.05). Larger residual curves correlated with larger operative curves (p < 0.001) and decreased flexibility on preoperative flexibility studies (p < 0.001), although postoperative CPCHILD scores did not differ by curve size or flexibility at time of fusion or by size of residual curve (p > 0.05). CONCLUSION: The median curve magnitude is large and there is substantial variability in curve size of CP scoliosis at time of fusion, although clinical outcomes are not negatively influenced by larger operative magnitudes. Further study should aim to narrow surgical indications by defining unacceptable radiographic outcomes. LEVEL OF EVIDENCE: Level III.

Zoledronate Increases Bone Mineral Density in Nonambulant Children With Cerebral Palsy: A Randomized Controlled Trial.

CONTEXT: Zoledronate appears to reduce fracture rates in children with cerebral palsy (CP), but no previous randomized, controlled trial has been performed to compare the effect of zoledronate to placebo in children with CP. OBJECTIVE: To investigate the effect of zoledronate on bone mineral density (BMD) Z-scores in children with nonambulant CP in a randomized, controlled, double-blind trial. METHODS: Nonambulant children with CP (5 to 16 years of age) were randomized 1:1 to receive 2 doses of zoledronate or placebo at a 6-month interval. BMD Z-score changes at the lumbar spine and the lateral distal femur (LDF) were calculated from dual-energy x-ray absorptiometry scans. Monitoring included weight, bone age, pubertal staging, knee-heel length, adverse events, biochemical markers, and questionnaires. RESULTS: Twenty-four participants were randomized and all completed the study. Fourteen were assigned to zoledronate. The mean lumbar spine BMD Z-score increased 0.8 SD (95% CI: 0.4; 1.2) in the zoledronate group, which was significant when compared to 0.0 SD (95% CI: -0.3; 0.3) in the placebo group. Similarly, the LDF BMD Z-scores increased more in the zoledronate group. Severe acute phase symptoms affected 50% of the patients in the zoledronate group but were reported exclusively after the first dose. Growth parameters were similar in both groups. CONCLUSION: Zoledronate for 12 months increased BMD Z-scores significantly without affecting growth, but first-dose side effects were common and considerable. Studies into lower first doses and long-term outcomes are needed.

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

Purpose: The 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 Methods: A 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. Results: Test-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. Conclusion: The 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.

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 rehabilitation Valid 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.

Intrathecal baclofen versus selective dorsal rhizotomy for children with cerebral palsy who are nonambulant: a systematic review.

OBJECTIVE: Cerebral palsy (CP) is the most common childhood physical disability. Historically, children with hypertonia who are nonambulatory (Gross Motor Function Classification System [GMFCS] level IV or V) were considered candidates for intrathecal baclofen (ITB) therapy to facilitate care and mitigate discomfort. Selective dorsal rhizotomy (SDR) was often reserved for ambulant children to improve gait. Recently, case series have suggested SDR as an alternative to ITB in selected children functioning at GMFCS level IV/V. The objective for this study was to systematically review the evidence for ITB and SDR in GMFCS level IV or V children. METHODS: Medline, Embase, Web of Science, and Cochrane databases were systematically searched. Articles were screened using the following inclusion criteria: 1) peer-reviewed articles reporting outcomes after SDR or ITB; 2) outcomes reported using a quantifiable scale or standardized outcome measure; 3) patients were < 19 years old at the time of operation; 4) patients had a diagnosis of CP; 5) patients were GMFCS level IV/V or results were reported based on GMFCS status and included some GMFCS level IV/V patients; 6) article and/or abstract in English; and 7) primary indication for surgery was hypertonia. Included studies were assessed with the Risk of Bias in Non-Randomized Studies - of Interventions (ROBINS-I) tool. RESULTS: Twenty-seven studies met inclusion criteria. The most commonly reported outcomes were spasticity (on the Mean Ashworth Scale) and gross motor function (using the Gross Motor Function Measure), although other outcomes including frequency of orthopedic procedures and complications were also reported. There is evidence from case series that suggests that both ITB and SDR can lower spasticity and improve gross motor function in this nonambulatory population. Complication rates are decidedly higher after ITB due in part to the ongoing risk of device-related complications. The heterogeneity among study design, patient selection, outcome selection, and follow-up periods was extremely high, preventing meta-analysis. There are no comparative studies, and meaningful health-related quality of life outcomes such as care and comfort are lacking. This review is limited by the high risk of bias among included studies. Studies of SDR or ITB that did not clearly describe patients as being GMFCS level IV/V or nonambulatory were excluded. CONCLUSIONS: There is a lack of evidence comparing the outcomes of ITB and SDR in the nonambulatory CP population. This could be overcome with standardized prospective studies using more robust methodology and relevant outcome measures.

Does hip displacement influence health-related quality of life in children with cerebral palsy?

OBJECTIVE: To evaluate the association of hip lateralisation with health-related quality of life (HRQL) in children with cerebral palsy (CP) using the Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD(®)) questionnaire. METHODS: We assessed n = 34 patients (mean age: 10.2 years, SD: 4.7 years; female: n = 16) with bilateral CP and Gross Motor Function Classification System (GMFCS) Level III-V using the CPCHILD(®) questionnaire. Hip lateralisation was measured by Reimer`s migration percentage (MP). RESULTS: There was an association between both, MP and GMFCS with CPCHILD(®) total score. Stratified analyses did not suggest interaction of the association between MP and CPCHILD(®) total score by GMFCS level. After adjustment for GMFCS level, we found a significant linear decrease of CPCHILD(®) total score of -0.188 points by 1% increment in MP. CONCLUSIONS: There was an association between MP and HRQL, which could not be explained by the GMFCS level.