Assessing functional outcomes of children with muscular dystrophy and scoliosis: the Muscular Dystrophy Spine Questionnaire.

BACKGROUND: The purpose of this study was to develop a valid and reliable scale to evaluate the symptoms and functional abilities important to children with scoliosis and muscular dystrophy and their parents. METHODS: Eighty-five items were generated from a review of the literature and interviews with clinicians, parents, and children with muscular dystrophy and scoliosis. Items were reviewed and rated in terms of "importance" and "severity" by the children and their parents. The foremost 29 items were formatted into a self-administered questionnaire. RESULTS: The questionnaire, completed 2 weeks apart, demonstrated "excellent" test-retest reliability (intraclass correlation coefficient = 0.97). Construct validity was established through correlations with forced vital capacity (r = 0.46; P = 0.02), the Activities Scale for Kids (r = 0.40; P = 0.04), and the Pediatric Outcomes Data Collection Questionnaire (r = 0.72; P = 0.00). CONCLUSIONS: In conclusion, the Muscular Dystrophy Spine Questionnaire is a valid and reliable questionnaire designed to assess the outcomes of treatment in children with muscular dystrophy and scoliosis. LEVEL OF EVIDENCE OR CLINICAL RELEVANCE: Prognostic level 2.

Treatments for paediatric femoral fractures: a randomised trial.

BACKGROUND: Treatments for femoral fractures in children vary widely and have been investigated only in case series. We did a multicentre randomised trial to compare malunion rates after external fixation and after early application of a hip spica cast for paediatric femoral shaft fractures. METHODS: All children aged 4-10 years with femoral fractures, admitted to four paediatric hospitals, were randomly assigned early application of hip spica or external fixation. The primary outcome was malunion at 2 years after the fracture. Secondary outcomes were scores on the RAND physical function child health questionnaire and the post-hospitalisation behavioural questionnaire, and parents' and children's ratings of overall satisfaction with treatment. Analysis was by intention to treat based on children who reached the 2 year evaluation. FINDINGS: Of 60 children assigned to the hip-spica group, 56 reached the 2-year assessment; of them, six (11%) required other forms of treatment because of unacceptable loss of reduction. Of 48 children assigned external fixation, 45 reached the 2-year assessment; two (4%) had refractures and five (11%) required operative adjustment of the fixator. The rate of malunion was significantly higher in the hip-spica group than in the external-fixator group (25/56 [45%] vs 7/45 [16%]; 95% CI for difference 12-46%; p=0.002). The two groups had similar mean scores for the RAND physical function health questionnaire (0.34 vs 0.45; 95% CI for difference, -0.57 to 0.34; p=0.61), for the post-hospitalisation questionnaire (106.8 vs 106.3; -4.9 to 5.9; p=0.86), and for parents' satisfaction (4.3 vs 4.2; -0.3 to 0.6; p=0.5) and children's ratings of happiness with treatment (6.9 vs 7.7; -2.2 to 0.5; p=0.21). INTERPRETATION: Early application of hip spica has a small role in the treatment of paediatric femoral fractures. Future trials need to compare external fixation with flexible intramedullary nails.

Can children with spina bifida and muscular dystrophy participate in their own health status evaluations?

BACKGROUND: The outcomes movement has emphasized the importance of the patient in evaluating treatment outcome. However, concern has been raised about the ability of children, particularly those with multisystem disease, to evaluate their disability. PURPOSE: To determine whether children with spina bifida and muscular dystrophy and their parents agree when asked about the relative difficulty of daily activities and the severity of symptoms experienced by the child. METHODS: First, a list of symptoms and activity restrictions was generated from the literature, clinicians, and interviews with families with spina bifida and muscular dystrophy. Second, another group of parents and children with spina bifida (with hip dislocation or scoliosis) and with muscular dystrophy (with scoliosis), including those before and after surgery, independently rated the severity and importance of their objective and subjective complaints. RESULTS: The correlation between parents and their children was high for both objective (median Spearman's = 0.70; standard deviation [SD] = ±0.17; range = -0.05-1.00) and subjective (median Spearman's = 0.76; SD = ±0.14; range = 0.13-1.00) complaints, with an overall excellent level of agreement (Kappa = 0.75; 95% confidence interval [CI]: 0.73, 0.76). CONCLUSION: Children with spina bifida and muscular dystrophy are capable of understanding and assessing their disability.

Patient preferences before and after total knee arthroplasty.

OBJECTIVE: Patients before total joint arthroplasty vary in the spectrum and importance of their concerns. The objectives of this study were to evaluate the psychometric properties of the Knee Patient-Specific Index (KPSI) and to determine the type and importance of patients' concerns before and after knee arthroplasty. STUDY DESIGN AND SETTING: A cohort of 119 patients scheduled for elective primary (or revision) total knee arthroplasty were interviewed at two tertiary care teaching hospitals. Patients also completed the Knee Society Scale (KSS), the Short Form 36, the Western Ontario and McMaster University Osteoarthritis Index (WOMAC), and the McMaster Toronto Arthritis Patient Preference Disability Questionnaire (MACTAR). RESULTS: Patients improved after total knee arthroplasty in all 42 symptoms and physical limitations, except crouching/kneeling and walking up and down stairs. Patients' summated concerns correlated with the WOMAC pain subscale (ranging from 0.72 to 0.79), WOMAC physical function subscale (ranging from 0.72 to 0.76), and KSS (ranging from 0.28 to 0.39). The summated responses changed after knee arthroplasty as demonstrated by the standardized response mean of 1.1. CONCLUSIONS: The KPSI captures individual patient unique preferences for patients undergoing total knee arthroplasty. Patients improved in virtually all aspects of their symptoms and function after surgery, with the exception of crouching/kneeling and knee feeling hot.

Reliability of radiographic measurement of fracture angulation in children with femoral shaft fractures.

INTRODUCTION: Clinical decisions are based on x-ray measures of radiographs. However, the reliability of assessing the angular measurement of fractured femurs in children is unknown. The purpose of this study was to determine the intra- and interrater reliability of an x-ray assessment of femoral shaft fracture angulation in children. METHODS: On 2 occasions, 3 raters evaluated 30 radiographs of children aged 4 to 10 years, 3 to 24 months after a femoral fracture. The radiographs were evaluated by an orthopedic surgeon, an orthopedic fellow and a research assistant, using a standardized methodology. Reliability was assessed with the intraclass correlation coefficient (ICC), an index of concordance that ranges from 01, where > 0.75 indicates excellent concordance. RESULTS: The ICC for the intrarater ranged from 0.75 to 0.97. The interrater reliability ICC for anterior or posterior angulation was 0.91 (95% confidence interval [CI] 0.840.95) and for varus or valgus angulation was 0.90 (95% CI 0.790.95). A change in measurement of more than 5 degrees exceeds the variation in measurement and, therefore, can be attributed to a true shift in fracture position. CONCLUSIONS: With specific standardized protocols, radiographic assessment of femoral fractures is reliable, irrespective of the examiner's level of experience.

Measuring physical function in children with spina bifida and dislocated hips: the Spina Bifida Hips Questionnaire.

The objective of this study was to develop a questionnaire to evaluate the activities of daily living that are important to children with spina bifida and dislocated hips and their families. Ninety-six items were generated from a review of the literature and interviews with clinicians, parents, and children with spina bifida and dislocated hips. Items were rated independently for "importance" and "severity" by the children and their parents. The 26 most important and severe items were formatted into a self-administered questionnaire, the Spina Bifida Hips Questionnaire (SBHQ). Parents and children completed 2 weeks apart the SBHQ, the Activities Scale for Kids (ASK), and the Pediatric Outcomes Data Collection Questionnaire (PODCQ). The SBHQ showed excellent test-retest reliability (intraclass correlation = 0.97). Construct validity was established by correlations with the ASK (r = 0.79, P < 0.01) and the PODCQ (r = 0.84, P < 0.01). In conclusion, the SBHQ is a valid and reliable questionnaire for the evaluation of treatment outcomes for children with spina bifida and dislocated hips.