Bracing Adolescent Idiopathic Scoliosis (BASIS) study - night-time versus full-time bracing in adolescent idiopathic scoliosis: study protocol for a multicentre, randomized controlled trial.

Aims: Scoliosis is a lateral curvature of the spine with associated rotation, often causing distress due to appearance. For some curves, there is good evidence to support the use of a spinal brace, worn for 20 to 24 hours a day to minimize the curve, making it as straight as possible during growth, preventing progression. Compliance can be poor due to appearance and comfort. A night-time brace, worn for eight to 12 hours, can achieve higher levels of curve correction while patients are supine, and could be preferable for patients, but evidence of efficacy is limited. This is the protocol for a randomized controlled trial of 'full-time bracing' versus 'night-time bracing' in adolescent idiopathic scoliosis (AIS). Methods: UK paediatric spine clinics will recruit 780 participants aged ten to 15 years-old with AIS, Risser stage 0, 1, or 2, and curve size (Cobb angle) 20° to 40° with apex at or below T7. Patients are randomly allocated 1:1, to either full-time or night-time bracing. A qualitative sub-study will explore communication and experiences of families in terms of bracing and research. Patient and Public Involvement & Engagement informed study design and will assist with aspects of trial delivery and dissemination. Discussion: The primary outcome is 'treatment failure' (Cobb angle progression to 50° or more before skeletal maturity); skeletal maturity is at Risser stage 4 in females and 5 in males, or 'treatment success' (Cobb angle less than 50° at skeletal maturity). The comparison is on a non-inferiority basis (non-inferiority margin 11%). Participants are followed up every six months while in brace, and at one and two years after skeletal maturity. Secondary outcomes include the Scoliosis Research Society 22 questionnaire and measures of quality of life, psychological effects of bracing, adherence, anxiety and depression, sleep, satisfaction, and educational attainment. All data will be collected through the British Spine Registry.

Improvement in functional gait parameters following corrective thoracolumbar surgery in children affected by Mucopolysaccharidosis 1 (Hurler syndrome).

OBJECTIVE: Thoracolumbar kyphosis is a common indication for spinal surgery in children with Mucopolysaccharidosis. Functional outcome of spinal surgical intervention has never been published in patients with this rare disease. We present a cohort of patients with Mucopolysaccharidosis 1(Hurler syndrome) who underwent thoraco-lumbar spinal deformity correction and functional outcome assessed by pre-operative and post-operative gait analysis. This study represents the first attempt at presenting a functional assessment of surgical outcome in any Mucopolysaccharidosis subtype. METHODS: A retrospective analysis of prospectively collected data was carried out from 11 children diagnosed with this subtype of Mucopolysaccharidosis. All patients underwent thoracolumbar kyphosis correction between the years 2013 to 2016. Gait assessment was performed using GAITRite™ electronic walkway pre-operatively and post-operatively within 9 to 24 months from the index surgery. Walking distance, cadence and gait velocity were the three spatio-temporal parameters analysed. Wilcoxon signed rank test was used to analyse the data and P-Value ≤0.05 was deemed significant. RESULTS: There was a statistically significant improvement in walking distance in 9 out of 11 patient post-operatively with a mean increase of 232.06 cms (P = 0.05). There was marginal improvement in cadence by 6.33 steps/min post-operatively (P-value 0.79). Gait velocity also showed a marginal increase by 8.73 cms/sec post-operatively (P-value 0.32). CONCLUSION: The results of our study suggest that correction of thoracolumbar kyphosis in children with Mucopolysaccharidosis 1 resulted in a significant improvement of walking distance with a trend towards improved gait in the other parameters. Post-operative change in cadence was not statistically significant suggesting that physiological maturation of gait had minimal effect in the specified post-operative assessment timeframe. This study emphasizes that outcomes of spinal surgery in children with Mucopolysaccharidosis 1 should be determined by functional measures aiming to maintain or improve quality of life.

Treatment of thoracolumbar kyphosis in patients with mucopolysaccharidosis type I: results of an international consensus procedure.

BACKGROUND: In all patients with mucopolysaccharidosis type I (MPS I), skeletal disease (dysostosis multiplex) is a prominent, debilitating, condition related complication that may impact strongly on activities of daily living. Unfortunately, it is not alleviated by treatment with hematopoietic cell transplantation (HCT) or enzyme replacement therapy (ERT). Although early kyphosis is one of the key features of dysostosis multiplex, there is no international consensus on the optimal management. Therefore, an international consensus procedure was organized with the aim to develop the first clinical practice guideline for the management of thoracolumbar kyphosis in MPS I patients. METHODS: A literature review was conducted to identify all available information about kyphosis and related surgery in MPS I patients. Subsequently, a modified Delphi procedure was used to develop consensus statements. The expert panel included 10 spinal orthopedic surgeons, 6 pediatricians and 3 physiotherapists, all experienced in MPS I. The procedure consisted of 2 written rounds, a face-to-face meeting and a final written round. The first 2 rounds contained case histories, general questions and draft statements. During the face-to-face meeting consensus statements were developed. In the final round, the panel had the opportunity to anonymously express their opinion about the proposed statements. RESULTS: Eighteen case series and case reports were retrieved from literature reporting on different surgical approaches and timing of thoracolumbar kyphosis surgery in MPS I. During the face-to-face meeting 16 statements were discussed and revised. Consensus was reached on all statements. CONCLUSION: This international consensus procedure resulted in the first clinical practice guideline for the management of thoracolumbar kyphosis in MPS I patients, focusing on the goals and timing of surgery, as well as the optimal surgical approach, the utility of bracing and required additional assessments (e.g. radiographs). Most importantly, it was concluded that the decision for surgery depends not only on the kyphotic angle, but also on additional factors such as the progression of the deformity and its flexibility, the presence of symptoms, growth potential and comorbidities. The eventual goal of treatment is the maintenance or improvement of quality of life. Further international collaborative research related to long-term outcome of kyphosis surgery in MPS I is essential as prognostic information is lacking.

Heterotopic ossification following use of i-Factor for spinal fusion in Mucopolysaccharidosis 1: a case report.

Mucopolysaccharidosis is a rare group of genetic disorder which results in a complex of anomalies involving various systems. In Mucopolysaccharidosis 1 progressive thoracolumbar kyphosis is a common presentation which can result in instability and neurological deficit. Posterior spinal surgery is performed to correct deformity and obtain spinal fusion. Peptide enhanced bone graft substitute (i-FactorTM) is relatively a new component with proven efficacy to obtain early spinal fusion. An 8-year-old child with progressive high lumbar kyphosis due to Mucopolysaccharidosis 1 was admitted for Posterior spinal fusion with i-Factor bone graft substitute. Postoperatively patient had serous discharge from the wound which settled without intervention. A month after the surgery spinal radiographs revealed heterotopic ossification at the distal end of spinal construct in the paraspinal region. Patient remained asymptomatic and clinically well.

Thoracolumbar kyphosis in treated mucopolysaccharidosis 1 (Hurler syndrome).

STUDY DESIGN: A retrospective radiographical follow-up study of thoracolumbar deformity in 33 children with mucopolysaccharidosis 1 (Hurler syndrome). OBJECTIVE: To report the severity, natural history, risk factors for progression, and results of intervention for thoracolumbar kyphosis in children with Hurler syndrome. SUMMARY OF BACKGROUND DATA: Literature on the subject of thoracolumbar kyphosis in Hurler syndrome and its treatment is limited to small case series. The natural history and thus indications for intervention are unknown. METHODS: Patients who had been treated with bone marrow transplantation and/or enzyme replacement therapy were followed up with erect radiographs of the spine. Mean follow-up period was 3.5 years (range, 2-12 yr). Radiographs were retrieved and analyzed retrospectively. Seven patients underwent varied forms of surgical intervention for progressive deformity, the technique and principles of which are described. RESULTS: The thoracolumbar kyphosis on initial radiographs obtained at a mean age of 17 months measured 38° (95% confidence interval, 34°-42°). Fifteen of the 33 patients (45%) followed for more than 2 years developed a deformity that made a progression of more than 10°. The magnitude of the initial deformity was predictive of whether the deformity progressed (univariate analysis, P < 0.001). An initial kyphosis angle greater than 45° was predictive of progression of more than 10° with sensitivity of 67% and specificity of 88%. All patients who underwent surgical intervention had sustained improvement in the magnitude of thoracolumbar deformity. CONCLUSION: Thoracolumbar kyphosis in Hurler syndrome is of variable severity with an average deformity, in our series, of 38° at a mean age of 17 months. Forty-five percent of patients developed progression of greater than 10°. Patients with an initial deformity greater than 45° seemed to be more likely to progress. Surgical interventions in the form of anterior fusion, combined anterior and posterior surgery and use of the vertical expandable prosthetic titanium rib provided good correction. LEVEL OF EVIDENCE: N/A.

Familial relationships of normal spine shape.

Familial correlations in the spinal shape of children are explored using statistical shape analysis. Measurements of the spine on the surface of the back were taken using an optical device, and recorded for several hundred children from the Leeds area of the U.K. A portion of the spine from the distal thoracic to proximal lumbar spine was used for the analysis, and measures of the shape of the spine line were determined using Procrustes analysis and principal components (PCs) analysis. Analysis was carried out on scans repeated in triplicate and scans taken six months apart, and good repeatability was demonstrated. Four groups of children were considered in the main study: monozygotic twins, dizygotic twins, same sex siblings and different sex siblings. Intra-familial correlations were calculated for the shape measures, and also for age and sex matched unrelated controls. The second PC score (which measures possible lordosis) had significant positive correlation in the family groups. It was observed that the familial correlations were higher for the genetically closer groups. Also, the same sex siblings had higher correlation than the different sex siblings. All sibling groups showed greater correlation of sagittal profile for the second PC score than unrelated controls. A significant correlation was observed in same sex pairings for the control data. This work suggests that some elements of spinal profile may be familial but also shows correlation with sex. Both of these observations may be important in the aetiology of idiopathic scoliosis.

Internal fixation of a patella fracture using an absorbable suture.

This case report describes the use of an absorbable suture for the fixation of a patella fracture in a child. In this case the fracture was held satisfactorily by the suture until union. This technique means that there is no need for a second operation to remove the fixation material.