[Growth-preserving instrumentation for early onset scoliosis]. / Mitwachsende Korrektursysteme bei Early-Onset-Skoliosen.

OBJECTIVE: Early onset scoliosis is defined as a spinal deformity originating in the first 10 years of life. Growth-preserving spinal instrumentation has therefore been designed to preserve growth of spine and chest wall and lungs to avoid serious pulmonary complications after early spine fusion. Indications, surgical technique and results of the vertical expandable prosthetic titanium rib (VEPTR) technique, traditional growing rods (TGR), and magnetically controlled growing rods (MCGR) will be described. INDICATIONS: Indications for VEPTR are so-called mixed congenital deformities (type 3) associated with vertebral malformations in association with chest wall deformities, especially fused ribs. There are also indications for neuromuscular or syndromic early onset scoliosis with bilateral rib-to-ilium constructs. However, most of those deformities are currently treated with either GR or MCGR in most centers. GR and MCGR are currently the treatment of choice for the majority of early onset scoliosis. CONTRAINDICATIONS: There is no indication for growth-preserving strategies if the patients are mature or there is only little growth remaining. In these cases, final fusion should be performed. SURGICAL TECHNIQUE: While the VEPTR technique involves an extensive approach with muscular dissections to the thoracic cage including rib osteotomies and thoracotomies, treatment with TGR or MCGR is minimally invasive, only exposing proximal and distal anchor points, leaving most of the spine including the apex undisturbed. POSTOPERATIVE MANAGEMENT: Early mobilization is usually possible after 24-48 h. Braces may have to be prescribed for patients with osteopenia, noncompliance, or a risk to fall. RESULTS: Since 2005, more than 200 patients were treated with the VEPTR technique, more than 200 patients with the MCGR technique, and about 30 patients with the TGR technique in our department. Complication rates are high with all techniques including the law of diminishing returns, autofusion, bone anchor-related complications like loosening or migration of implants, failure to distract and proximal junctional kyphosis. In our own series of 13 patients below age 3 years, VEPTR proved to be effective for mixed deformities. In other studies, we were able to show that physiological growth with MCGR can be maintained for 2-3 years but spinal growth declines after that period with acceptable complications. Complication rates in most studies are lower with MCGR compared to TGR and VEPTR. Therefore, it is currently the treatment of choice for most early onset scoliosis patients.

Medium-term Results After Femoral Head Resection and Subtrochanteric Valgus Osteotomy in Children and Adolescents With Neuromuscular Disorders.

BACKGROUND: Various salvage surgical procedures for painful hip dislocation in adolescent patients with cerebral palsy exist. To date, no significant differences among these surgical techniques have been described. In our institution the McHale procedure is the standard of care for painful chronically dislocated hips with or without deformity of the femoral head in patients with cerebral palsy. This study focuses on mid-term results after surgical treatment. METHODS: Surgical reports and patient charts were analyzed retrospectively. All x-rays were evaluated and migration of the proximal femur and heterotopic ossification according to Brooker were recorded. In addition, we conducted a telephone interview with the caretakers with special reference to pain preoperatively and postoperatively and after implant removal, sitting tolerance, range of motion, mobility, quality of life, and personal hygiene. RESULTS: Fifty-two patients (65 hips) with a mean age of 13.5 ± 3.6 years (range: 4 to -20 y) were included. Mean surgery time was 178.4 ± 63.4 minutes (range: 45 to 380 min) and mean follow-up was 45.17 ± 30.6 months (range: 12 to 204 mo). A significant difference between preoperative and postoperative pain levels was found, P <0.001. Personal hygiene ( P =0.02) and quality of life ( P =0.013) improved significantly. Eighty-five percent of the caregivers would have the surgery performed on their child again and 81% of the caregivers would recommend the surgery to others. The removal of implants leads to a significant improvement in pain ( P =0.011). A total of 22 complications in 65 McHale procedures (33.9%) were related to the procedures. CONCLUSIONS: A significant reduction in pain and a significant improvement of hygiene as well as quality of life can be achieved with the McHale procedure in painful chronically dislocated hips in patients with cerebral palsy. Overall, the procedure is predominantly experienced as helpful by the caregivers and recommended to others. The removal of the implants improves pain significantly, but complications may occur in one third of the patients. LEVEL OF EVIDENCE: IV.

Anterior Vertebral Body Tethering for Skeletally Immature Patients with AIS: Indication for Spinal Fusion at Skeletal Maturity Is Not Obviated in 60% of Cases.

The role of anterior vertebral body tethering (aVBT) in obviating the need for spinal fusion in patients with AIS remains unclear, and a large amount of variation exists in the data among different studies. The present study aims to investigate and analyze what factors have a potential influence on aVBT outcome. Skeletally immature patients with AIS who underwent aVBT for scoliosis correction were followed up until skeletal maturity. The mean age at the time of surgery was 13.4 ± 1.1, and the mean follow-up time was 2.5 ± 0.5 years. The Cobb angle of the main curve was 46.6 ± 9° at the time of surgery and was significantly corrected to 17.7 ± 10.4° (p < 0.001) immediately postoperatively. A significant loss of correction was observed during the latest follow-up (Cobb angle 33.8 ± 18.7°; p < 0.001). An indication for spinal fusion at skeletal maturity was not obviated in 60% of the patients. The factors identified as having an influence on the outcome were preoperative bone age and the magnitude of the major curve. Patients with advanced bone age and larger curves were more likely to reach an indication for spinal fusion at skeletal maturity. In conclusion, no general recommendation for aVBT can be made for AIS patients. The method can be discussed as a treatment option in skeletally very immature preadolescent patients (Sanders Stadium ≤ 2) with a moderate Cobb angle (≤50°) who failed previous brace therapy.

Radiolucent zones of biodegradable magnesium-based screws in children and adolescents-a radiographic analysis.

INTRODUCTION: Albeit the implantation of magnesium-based biodegradable implants can avoid a second surgery for implant removal, the postoperative occurrence of radiolucent zones around these implants based on corrosion processes has not been previously investigated in children and adolescents. We sought to characterize the distinct temporal and spatial dynamics for magnesium-based implants based on standard clinical routine radiographs. MATERIALS AND METHODS: We retrospectively analyzed 29 patients, treated with magnesium-based compression screws (MAGNEZIX® CS 2.7 mm, CS 3.2 mm, CSC 4.8 mm; Syntellix AG) for fracture fixation, osteotomy, or osteochondral refixation. During a follow-up examination, the clinical and functional status was evaluated. Based on digital radiographs, the ratio of the area of the radiolucent zone and that of the screw was evaluated to assess implant degradation at two follow-up visits (i.e., after 6-8 weeks and 12-24 weeks). RESULTS: In 29 patients (16/29 females, 14.03 ± 2.13 years), a total of 57 implants were evaluated that were used for osteotomy (n = 13, screws n = 26), fracture fixation (n = 9, screws n = 18), or osteochondral refixation (n = 7, screws n = 13). All patients healed without complications and regained full function. Radiolucent zones were observed in 27/29 patients at the first follow-up, with significantly decreased ratios at the second follow-up (2.10 ± 0.55 vs 1.64 ± 0.60, p = 0.0006). Regression analyses were performed to assess the temporal dynamics of radiolucent zones and revealed significant logarithmic developments for the 2.7 mm and 3.2 mm screws, marked by a strong ratio decrease during the first weeks and an almost complete disappearance after approximately 100 days and 200 days, respectively. In contrast, the ceramic-coated 4.8 mm screws presented a significant linear and slower decrease of radiolucent zones. CONCLUSION: Radiolucent zones are a common phenomenon in the course of implant degradation. However, they represent a self-limiting phenomenon. Remarkably, neither implant failure nor affected implant function is noted in this context. Yet, the microstructural changes accompanying the presence of radiolucent zones remain to be analyzed by three-dimensional high-resolution imaging.

Treatment of Unspecific Back Pain in Children and Adolescents: Results of an Evidence-Based Interdisciplinary Guideline.

Using a structured approach and expert consensus, we developed an evidence-based guideline on the treatment and prevention of non-specific back pain in children and adolescents. A comprehensive and systematic literature search identified relevant guidelines and studies. Based on the findings of this literature search, recommendations on treatment and prevention were formulated and voted on by experts in a structured consensus-building process. Physical therapy (particularly physical activity) and psychotherapy (particularly cognitive behavioral therapy) are recommended for treating pediatric non-specific back pain. Intensive interdisciplinary treatment programs should be provided for chronic and severe pain. Drug therapy should not be applied in children and adolescents. Further research on non-specific back pain in childhood and adolescence is strongly needed to reduce the imbalance between the high burden of non-specific back pain in childhood and adolescence and the low research activity in this field.

Etiology, Risk Factors, and Diagnosis of Back Pain in Children and Adolescents: Evidence- and Consensus-Based Interdisciplinary Recommendations.

Using a structured approach and expert consensus, we developed an evidence-based guideline on the diagnosis of back pain and the treatment of non-specific back pain in children and adolescents. The first part comprises etiology, risk factors, and diagnosis. The second part, published in the same issue, includes treatment and prevention. A comprehensive and systematic literature search was conducted to identify relevant guidelines and studies. Based on the findings of this literature search, recommendations on risk factors and diagnosis were formulated and voted on by experts in a structured consensus-building process. Notable red flags for specific back pain and evidence-based risk factors for non-specific back pain in children and adolescents were identified. Only three evidence-based recommendations could be formulated for causes, red flags, and risk factors for back pain, while two recommendations are based on expert consensus. Regarding diagnostics, eight expert consensus recommendations and one evidence-based recommendation could be provided. Despite the importance of adequate diagnosis for the treatment of back pain in children and adolescents, results of this work confirm the deficit in research investment in this area.

22q11.2 Deletion Syndrome as a Human Model for Idiopathic Scoliosis.

To better understand the etiology of idiopathic scoliosis, prospective research into the pre-scoliotic state is required, but this research is practically impossible to carry out in the general population. The use of 'models', such as idiopathic-like scoliosis established in genetically modified animals, may elucidate certain elements, but their translatability to the human situation is questionable. The 22q11.2 deletion syndrome (22q11.2DS), with a 20-fold increased risk of developing scoliosis, may be a valuable and more relevant alternative and serve as a human 'model' for idiopathic scoliosis. This multicenter study investigates the morphology, dynamic behavior, and presence of intraspinal anomalies in patients with 22q11.2DS and scoliosis compared to idiopathic scoliosis. Scoliosis patients with 22q11.2DS and spinal radiography (n = 185) or MRI (n = 38) were included (mean age 11.6 ± 4.2; median Cobb angle 16°) and compared to idiopathic scoliosis patients from recent literature. Radiographic analysis revealed that 98.4% of 22q11.2DS patients with scoliosis had a curve morphology following predefined criteria for idiopathic curves: eight or fewer vertebrae, an S-shape and no inclusion of the lowest lumbar vertebrae. Furthermore, curve progression was present in 54.2%, with a mean progression rate of 2.5°/year, similar to reports on idiopathic scoliosis with 49% and 2.2-9.6°/year. The prevalence of intraspinal anomalies on MRI was 10.5% in 22q11.2DS, which is also comparable to 11.4% reported for idiopathic scoliosis. This indicates that 22q11.2DS may be a good model for prospective studies to better understand the etiology of idiopathic scoliosis.

Does Juvenile Idiopathic Arthritis Affect the Course of Legg-Calvé-Perthes Disease? A Case-Control Study with a Mean Follow-Up of 8 Years.

BACKGROUND: This study aimed to determine the clinical and radiological course in children who had Legg-Calvé-Perthes disease (LCPD) associated with juvenile idiopathic arthritis (JIA). METHODS: In a retrospective chart review between 2007 and 2019, eight consecutive JIA patients diagnosed with concomitant LCPD were identified and compared with a case-control group of 10 children with LCPD only. RESULTS: LCPD was diagnosed at a mean age of 8.1 years (3.0-14.7) in children with JIA as compared to 6.1 years (2.9-10.0) in controls. According to the modified Harris Hip Score (mHHS), four children with JIA and all controls had an excellent result. Regarding the fragmentation severity and the duration of each stage, we found no differences using the lateral pillar and modified Elizabethtown classification. Five hips were classified as Stulberg I/II, two hips as Stulberg III, and one hip as Stulberg V with no evidence of hip dysplasia or severe overcoverage in either group. CONCLUSIONS: The radiological outcome of LCPD did not differ between both groups, while the clinical outcome was slightly better in controls. Physicians should be aware that children with LCPD may have JIA too. In suspicious cases, further investigations are recommended, and patients should be referred to pediatric rheumatologists.

Postnatal Repair of Open Neural Tube Defects: A Single Center with 90-Month Interdisciplinary Follow-Up.

After publication of the Management of Myelomeningocele Study (MOMS) there is confusion regarding which treatment of open neural tube defects (NTD) is best. We report our results of postnatally repaired open NTDs born between 2007-2018 (n = 36) in critical reflection of the MOMS study. Neurosurgical, orthopedic, and urologic data were assessed. We also introduce a new entity: "status post prenatal repair". FU ranged from 29 to 161 months (mean: 89.1 m) in 7 cases of myeloschisis and 24 myelomeningoceles in the final collective n = 31. The shunt rate was 41.9%, and the endoscopic third ventriculostomy rate was 16.1%. Hydrocephalus requiring treatment was not associated with the anatomical level, but with premature birth (p = 0.048). Myeloschisis was associated with shunt placement (p = 0.008). ROC analysis revealed birth <38.5th week predicts the necessity for hydrocephalus treatment (sensitivity: 89%; specificity: 77%; AUC= 0.71; p = 0.055). Eight (25.8%), patients are wheelchair-bound, 2 (6.5%) ambulate with a posterior walker, 10 (32.3%) with orthosis and 11 (35.5%) independently. One (3.2%) patient underwent detethering at 5.5 years. A total of three patients underwent five Chiari decompressions (9.6%). Further, nineteen orthopedic procedures were performed in nine patients (29.0%). A total of 17 (54.8%) patients self-catheterize, which was associated with an anatomical lesion at L3 or below (p = 0.032) and 23 (74.2%) take anticholinergic medication. In conclusion, shunt dependency is associated with myeloschisis, not with the anatomical defect level. Hydrocephalus treatment is associated with premature birth. In this postnatal cohort with significantly longer follow-up data than the MOMs study, the ambulation rate is better, the shunt rate lower and the secondary tethered cord rate better compared to the MOMS study.

Low SARS-CoV-2 seroprevalence but high perception of risk among healthcare workers at children's hospital before second pandemic wave in Germany.

BACKGROUND: Healthcare workers are considered a particularly high-risk group during the coronavirus disease 2019 (COVID-19) pandemic. Healthcare workers in paediatrics are a unique subgroup: they come into frequent contact with children, who often experience few or no symptoms when infected with severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) and, therefore, may transmit the disease to unprotected staff. In Germany, no studies exist evaluating the risk of COVID-19 to healthcare workers in paediatric institutions. METHODS: We tested the staff at a large children's hospital in Germany for immunoglobulin (Ig) G antibodies against the nucleocapsid protein of SARS-CoV-2 in a period between the first and second epidemic wave in Germany. We used a questionnaire to assess each individual's exposure risk and his/her own perception of having already been infected with SARS-CoV-2. RESULTS: We recruited 619 participants from all sectors, clinical and non-clinical, constituting 70% of the entire staff. The seroprevalence of SARS-CoV-2 antibodies was 0.325% (95% confidence interval 0.039-1.168). Self-perceived risk of a previous SARS-CoV-2 infection decreased with age (odds ratio, 0.81; 95% confidence interval, 0.70-0.93). Having experienced symptoms more than doubled the odds of a high self-perceived risk (odds ratio, 2.18; 95% confidence interval, 1.59-3.00). There was no significant difference in self-perceived risk between men and women. CONCLUSIONS: Seroprevalence was low among healthcare workers at a large children's hospital in Germany before the second epidemic wave, and it was far from a level that confers herd immunity. Self-perceived risk of infection is often overestimated.

[Spinal deformity in Duchenne muscular dystrophy]. / Wirbelsäulendeformität bei Muskeldystrophie Typ Duchenne.

Boys with Duchenne muscular dystrophy develop scoliosis in 95% of cases. Conservative management to avoid progression of the deformity is not successful. However, treatment with corticosteroids may avoid the onset of scoliosis in some patients. Early surgical treatment and spine fusion is usually recommended before severe cardiopulmonary symptoms have developed. With modern surgical techniques, the sitting ability and quality of life can be preserved with a moderate complication rate. An interdisciplinary approach is mandatory to achieve these goals.

Safety and performance of biodegradable magnesium-based implants in children and adolescents.

AIMS: Biodegradable magnesium-based alloy implants represent a promising option in orthopedic surgery, as the clinical outcomes have been reported to be comparable to those of titanium implants and no surgical interventions are required for removal. To date, little is known about the results of the use of these implants in children and adolescents. Therefore, the aim of the present study was to analyze the safety and performance of these implants in children and adolescents. PATIENTS AND METHODS: Eighty-nine patients treated with magnesium-based implants for fracture stabilization, osteotomy and osteochondral refixation were analyzed; 38 were treated by osteosynthesis; 18, osteotomy; and 33, osteochondral refixation. The mean follow-up duration was 8.2 months (range, 1.5-30 months). Clinical and radiographical follow-up examinations were performed at 4-8 weeks and 3-6 months, respectively, to evaluate implant performance and osseous consolidation. RESULTS: Clinical outcomes were rated as good to very good in all patients. Radiolucent zones were apparent after surgery in all patients but were noted to decrease in size during the follow-up period. Revision surgery was necessary in 1 of 89 patients who had a highly unstable osteochondritis dissecans lesion of the knee. None of the magnesium-based implants required surgical removal. CONCLUSION: Magnesium-based implants in children and adolescents results in good clinical outcomes when used for fracture stabilization, osteotomy and osteochondral defect refixation. Future studies are needed to further analyze the significance of the transient appearance and temporal development of radiolucent zones in the growing skeleton as well as the long-term performance of these implants.

Does eight-plate epiphysiodesis of the proximal tibia in treating angular deformity create intra-articular deformity?

AIMS: Eight-plates are used to correct varus-valgus deformity (VVD) or limb-length discrepancy (LLD) in children and adolescents. It was reported that these implants might create a bony deformity within the knee joint by change of the roof angle (RA) after epiphysiodesis of the proximal tibia following a radiological assessment limited to anteroposterior (AP) radiographs. The aim of this study was to analyze the RA, complemented with lateral knee radiographs, with focus on the tibial slope (TS) and the degree of deformity correction. METHODS: A retrospective, single-centre study was conducted. The treatment group (n = 64 knees in 44 patients) was subclassified according to the implant location in two groups: 1) medial hemiepiphysiodesis; and 2) lateral hemiepiphysiodesis. A third control group consisted of 25 untreated knees. The limb axes and RA were measured on long standing AP leg radiographs. Lateral radiographs of 40 knees were available for TS analysis. The mean age of the patients was 10.6 years (4 to 15) in the treatment group and 8.4 years (4 to 14) in the control group. Implants were removed after a mean 1.2 years (0.5 to 3). RESULTS: No significant differences in RA (p = 0.174) and TS (p = 0.787) were observed. The limb axes were significantly corrected in patients with VVD (p < 0.001). The change in tibial slope (∆TS) did not correlate (r = -0.026; p = 0.885) to the plate's position on the physis when assessed by lateral radiographs. CONCLUSION: We were not able to confirm the reported change in the bony morphology of the proximal tibia on AP radiographs in our patient population. In addition, no significant change in TS was detected on the lateral radiographs. A significant correction of the VVD in the lower limb axes was evident. Position of the implant did not correlate with TS change. Therefore, eight-plate epiphysiodesis is a safe and effective procedure for correcting VVD in children without disturbing the knee joint morphology. Cite this article: Bone Joint J 2020;102-B(10):1412-1418.

Management of spinal deformities and tibial pseudarthrosis in children with neurofibromatosis type 1 (NF-1).

The skeletal system is affected in up to 60% of patients with neurofibromatosis type 1. The most commonly observed entities are spinal deformities and tibial dysplasia. Early recognition of radiologic osseous dystrophy signs is of utmost importance because worsening of the deformities without treatment is commonly observed and surgical intervention is often necessary. Due to the relative rarity and the heterogenic presentation of the disease, evidence regarding the best surgical strategy is still lacking. PURPOSE: To report our experience with the treatment of skeletal manifestations in pediatric patients with (neurofibromatosis type 1) NF-1 and to present the results with our treatment protocols. MATERIALS AND METHODS: This is a retrospective, single expert center study on children with spinal deformities and tibial dysplasia associated with NF-1 treated between 2006 and 2020 in a tertiary referral institution. RESULTS: Spinal deformity: Thirty-three patients (n = 33) were included. Mean age at index surgery was 9.8 years. In 30 patients (91%), the deformity was localized in the thoracic and/or lumbar spine, and in 3 patients (9%), there was isolated involvement of the cervical spine. Eleven patients (33%) received definitive spinal fusion as an index procedure and 22 (67%) were treated by means of "growth-preserving" spinal surgery. Halo-gravity traction before index surgery was applied in 11 patients (33%). Progression of deformity was stopped in all patients and a mean curve correction of 60% (range 23-98%) was achieved. Mechanical problems with instrumentation requiring revision surgery were observed in 55% of the patients treated by growth-preserving techniques and in none of the patients treated by definitive fusion. One patient (3%) developed a late incomplete paraplegia due to a progressive kyphotic deformity. Tibial dysplasia: The study group comprised of 14 patients. In 5 of them (36%) pathological fractures were present on initial presentation. In the remaining 9 patients (64%), anterior tibial bowing without fracture was observed initially. Four of them (n = 4, 28%) subsequently developed a pathologic fracture despite brace treatment. Surgical treatment was indicated in 89% of the children with pathological fractures. This involved resection of the pseudarthrosis, autologous bone grafting, and intramedullary nailing combined with external fixation in some of the cases. In 50% of the patients, bone morphogenic protein was used "off-label" in order to promote union. Healing of the pseudarthrosis was achieved in all of the cases and occurred between 5 to 13 months after the index surgical intervention. Four of the patients treated surgically needed more than one surgical intervention in order to achieve union; one patient had a re-fracture. All patients had a good functional result at last follow-up. CONCLUSION: Early surgical intervention is recommended for the treatment dystrophic spinal deformity in children with NF-1. Good and sustainable curve correction without relevant thoracic growth inhibition can be achieved with growth-preserving techniques alone or in combination with short spinal fusion at the apex of the curve. Preoperative halo-gravity traction is a safe and very effective tool for the correction of severe and rigid deformity in order to avoid neurologic injury. Fracture union in tibial dysplasia with satisfactory functional results can be obtained in over 80% of the children by means of surgical resection of the pseudarthrosis, intramedullary nailing, and bone grafting. Wearing a brace until skeletal maturity is achieved is mandatory in order to minimize the risk of re-fracture.

Posterior fusion of the craniocervical junction in the pediatric spine: Wright's translaminar C2 screw technique provides for more safety and effectiveness.

PURPOSE: Posterior fusion of the craniocervical junction (CCJ) has always been challenging in children with rare congenital diseases and malformations. At our institution, the introduction of the translaminar C2 screw technique led to a significant improvement in the quality of treatment. METHODS: Retrospective analysis of a pediatric cohort at a single institution who underwent CCJ posterior fusion between 2007 and 2018. Patients were divided into group 1 (other posterior fusion techniques, n = 12) and group 2 (translaminar axis screw placement, n = 19). Diagnosis, sex, age at surgery, surgical technique, immobilization, revisions, fusion, reduction, and complications were assessed. RESULTS: Follow-up ranged from 12 to 145 months (mean 50.7). The initial fusion rate detected at 3 months by CT differed significantly (66, 7% in group 1 vs. 100% in group 2, p = 0.018). Full reduction of C1/C2 malalignments was achieved in 41, 6% of group 1 versus 84, 2% of group 2 (p = 0.007). Immobilization was applied in 83, 3% of group 1 versus 26, 3% of group 2 (p = 0.0032). Ten complications were treated conservatively, and 15 events required revision surgery (80% in group 1 vs. 20% in group 2). Eight complications were related to immobilization. CONCLUSIONS: The implementation of the translaminar C2 technique resulted in significantly more safety and efficiency regarding pediatric posterior fusion CCJ surgery at our institution, with significantly higher rates of rigid fixation, full reduction, and fusion, and significantly lower rates of complications and immobilization. These slides can be retrieved under Electronic Supplementary Material.

Serial casting in early onset scoliosis: syndromic scoliosis is no contraindication.

BACKGROUND: Serial casting is a treatment for early onset scoliosis (EOS) in young children to achieve curve correction before bracing or to postpone initial surgical treatment until the patient is older. Good results have been reported for patients with idiopathic early onset scoliosis (IS). However, there are few reports of results in non-idiopathic cases, and the benefits of non-surgical methods in the syndromic-associated early onset scoliosis subgroup are unknown. METHODS: Retrospective single-institution study of patient charts and X-rays of all cases of sustained serial casting for EOS. Staged correction was obtained by applying three consecutive casts under general anaesthesia. These were changed every 4 weeks, followed by the implementation of a custom-made full-time Chêneau brace. Correction was measured by Cobb angle (CA) and rib-vertebra angle difference (RVAD) on whole spine anterior-posterior radiographs. Statistical analysis was performed via ANOVA. RESULTS: The study group consisted of 6 patiens with IS and 10 with non-idiopathic scoliosis (NIS) - exclusively syndromic-associated. The mean age at onset of treatment was 35 months (±15). The mean follow up was 21 months (±15). In IS patients average CA/RVAD before treatment was 46°(±8)/20°(±12). In NIS patients average CA/RVAD before treatment was 55°(±15)/24°(±14). After application of the third cast, the CA/RVAD was reduced to 20°(±11)/11°(±10) in IS patients. Whereas in NIS patients average CA/RVAD after the thrid cast was 28°(±12)/18°(±13). At latest follow-up the CA/RVAD was 16°(±7)/9°(±8) in IS patients and 31°(±11)/17° (±15) in NIS patients. CONCLUSION: Syndromic etiology is not a contraindication for serial casting in EOS. Our results show a curve correction, measured in CA, of 65% in IS patients and 44% in NIS patients. Significant reduction in the morphologic deformity, measured in RVAD, was achieved in the IS cohort, but not in the NIS cohort. In all cases surgical treatment could be delayed.

Growth charts for patients with Sanfilippo syndrome (Mucopolysaccharidosis type III).

BACKGROUND: Mucopolysaccharidosis (MPS) type III (Sanfilippo syndrome) comprises a group of rare, lysosomal storage diseases caused by the deficiency of one of four enzymes involved in the degradation of heparan sulfate. The clinical hallmark of the disease is severe neurological deterioration leading to dementia and death in the second decade of life. Adult MPS patients are generally of short stature. To date there is no clear description of the physical development of MPS III patients. The aim of this study was to document growth reference data for MPS III patients. We collected growth data of 182 German MPS III patients and were able to develop growth charts for this cohort. Growth curves for height, weight, head circumference, and body mass index were calculated and compared to German reference charts. RESULTS: Birth height, weight and head circumference were within the physiological ranges. Both genders were significantly taller than healthy children at 2 years of age, while only male patients were taller at the age of four. Growth velocity decelerated after the ages of 4.5 and 5 years for female and male patients, respectively. Both genders were significantly shorter than the reference group at the age of 17.5 years. Head circumference was larger compared to healthy matched controls within the first 2 years of life and remained enlarged until physical maturity. CONCLUSION: MPS III is a not yet treatable severe neuro-degenerative disease, developing new therapeutic strategies might change the course of the disease significantly. The present charts contribute to the understanding of the natural history of MPS III. Specific growth charts represent an important tool for families and physicians as the expected height at physical maturity can be estimated and therapeutic effects can be monitored.