European Achondroplasia Forum Practical Considerations for Following Adults with Achondroplasia.

Achondroplasia is a lifelong condition requiring lifelong management. There is consensus that infants and children with achondroplasia should be managed by a multidisciplinary team experienced in the condition. However, many people are lost to follow-up after the transition from paediatric to adult care, and there is no standardised approach for management in adults, despite the recent availability of international consensus guidelines. To address this, the European Achondroplasia Forum has developed a patient-held checklist to support adults with achondroplasia in managing their health. The checklist highlights key symptoms of spinal stenosis and obstructive sleep apnoea, both among the most frequent and potentially severe medical complications in adults with achondroplasia. The checklist acts as a framework to support individuals and their primary care provider in completing a routine review. General advice on issues such as blood pressure, pain, hearing, weight, adaptive aids, and psychosocial aspects are also included. The checklist provides key symptoms to be aware of, in addition to action points so that people can approach their primary care provider and be directed to the appropriate specialist, if needed. Additionally, the European Achondroplasia Forum offers some ideas on implementing the checklist during the transition from paediatric to adult care, thus ensuring the existing multidisciplinary team model in place during childhood can support in engaging individuals and empowering them to take responsibility for their own care as they move into adulthood.

European Achondroplasia Forum guiding principles for the detection and management of foramen magnum stenosis.

Foramen magnum stenosis is a serious, and potentially life-threatening complication of achondroplasia. The foramen magnum is smaller in infants with achondroplasia, compared with the general population, and both restricted growth in the first 2 years and premature closure of skull plate synchondroses can contribute to narrowing. Narrowing of the foramen magnum can lead to compression of the brainstem and spinal cord, and result in sleep apnoea and sudden death. There is a lack of clarity in the literature on the timing of regular monitoring for foramen magnum stenosis, which assessments should be carried out and when regular screening should be ceased. The European Achondroplasia Forum (EAF) is a group of clinicians and patient advocates, representative of the achondroplasia community. Members of the EAF Steering Committee were invited to submit suggestions for guiding principles for the detection and management of foramen magnum stenosis, which were collated and discussed at an open workshop. Each principle was scrutinised for content and wording, and anonymous voting held to pass the principle and vote on the level of agreement. A total of six guiding principles were developed which incorporate routine clinical monitoring of infants and young children, timing of routine MRI screening, referral of suspected foramen magnum stenosis to a neurosurgeon, the combination of assessments to inform the decision to decompress the foramen magnum, joint decision making to proceed with decompression, and management of older children in whom previously undetected foramen magnum stenosis is identified. All principles achieved the ≥ 75% majority needed to pass (range 89-100%), with high levels of agreement (range 7.6-8.9). By developing guiding principles for the detection and management of foramen magnum stenosis, the EAF aim to enable infants and young children to receive optimal monitoring for this potentially life-threatening complication.

Spinal cord compression in patients with mucopolysaccharidosis.

PURPOSE: Spinal abnormalities frequently occur in patients with mucopolysaccharidosis (MPS) types I, II, IV, and VI. The symptoms are manifold, which sometimes prolongs the diagnostic process and delays therapy. Spinal stenosis (SS) with spinal cord compression due to bone malformations and an accumulation of storage material in soft tissue are serious complications of MPS disease. Data on optimal perioperative therapeutic care of SS is limited. METHODS: A retrospective chart analysis of patients with MPS and SS for the time period 01/1998 to 03/2021 was performed. Demographics, clinical data, neurological status, diagnostic evaluations (radiography, MRI, electrophysiology), and treatment modalities were documented. The extent of the SS and spinal canal diameter were analyzed. A Cox regression analysis was performed to identify prognostic factors for neurological outcomes. RESULTS: Out of 209 MPS patients, 15 were included in this study. The most dominant type of MPS was I (-H) (n = 7; 46.7%). Preoperative neurological deterioration was the most frequent indication for further diagnostics (n = 12; 80%). The surgical procedure of choice was dorsal instrumentation with microsurgical decompression (n = 14; 93.3%). A univariate Cox regression analysis showed MPS type I (-H) to be associated with favorable neurological outcomes. CONCLUSION: Early detection of spinal stenosis is highly relevant in patients with MPS. Detailed neurological assessment during follow-up is crucial for timeous detection of patients at risk. The surgical intervention of choice is dorsal instrumentation with microsurgical decompression and resection of thickened intraspinal tissue. Patients with MPS type I (-H) demonstrated the best neurological course.

Vertebral column growth in children with early onset scoliosis treated with magnetically controlled growing rods - Effects of distraction on vertebral and disc morphology.

OBJECTIVES: To report changes in vertebral and disc morphology following treatment of early onset scoliosis (EOS) with magnetically controlled growing rods (MCGR). METHODS: 30 Patients, 21 girls and 9 boys, treated with MCGR for EOS were compared to a matched control group of 19 patients (12 girls, 7 boys) treated with bracing or observation. Age at surgery in the MCGR group was 8.75 (range: 4.6-11) years compared to 7.75 (range: 3.5-10.3) years in the control group at the time of onset of treatment. Mean follow-up was 45 (range: 24-65) months in the MCGR group vs 42 (range: 24-65) months in the control group. Calibrated radiographs were used to digitally measure disc height, vertebral body height, depth and width after surgery and at latest follow-up. Height, width and depth of lumbar and thoracic vertebrae and discs under distraction were compared to the control group and to vertebrae below instrumentation. T1-T12, T1-S1 length and Cobb angles were also measured. RESULTS: (1) There is a significant increase of lumbar vertebral height under distraction. (2) Lumbar disc height as well as lumbar vertebral width within distraction is significantly reduced. (3) Depth of lumbar vertebrae is not significantly affected by distraction. (4) Morphology of thoracic vertebrae and adjacent discs is not significantly changed with distraction. (5) T1-T12 distance did not show any significant changes between surgical and control group, while T1-S1 growth was significantly reduced compared to the control group. CONCLUSIONS: Significant changes of morphology of lumbar vertebra and discs are observed under distraction with MCGR compared to segments below instrumentation and the control group. LEVEL OF EVIDENCE: Level III - retrospective comparative series.

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.

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.

Vertical Expandable Rib-based Distraction Device for Correction of Congenital Scoliosis in Children of 3 Years of Age or Younger: A Preliminary Report.

BACKGROUND: This study was designed to evaluate the treatment outcome of very young children with congenital scoliosis aged 3 years or under after surgery with a vertical expandable prosthetic titanium rib (VEPTR)-based distraction device. METHODS: A retrospective study of 13 children undergoing implantation of a vertical expandable rib-based distraction device. From September 2007 to June 2018, 13 children (7 male and 6 female patients) with congenital scoliosis were followed after treatment with a VEPTR. The outcome parameters were complications, thoracic height, kyphosis, lordosis, and coronal major scoliosis curve. In addition, the American Society of Anaesthesiologists (ASA) score, assisted ventilation rating, and hemoglobin and body mass index were analyzed. Data were examined separately by 2 investigators. RESULTS: The mean age at initial surgical treatment was 24.4±10.6 months, follow-up was 91.5±23.1 months. The mean number of surgical procedures per patient was 14±3.9 (total 182 operations). Apart from planned operations every 6 months, 5 unplanned operations (2.7%) were performed. The major scoliosis curve improved significantly from 55.2±21.9 degrees to 40.5±18.7 degrees, thoracic spine height T1-T12 significantly from 111±12 mm to 137±23 mm, T1-S1 height significantly from 211±13 mm to 252±36 mm and space available for the lung from 80.9%±11.3% to 84.4%±8.8% (preoperatively and at 5-y follow-up, respectively). Kyphosis increased significantly from 30±21.4 degrees to 42.6±23.5 degrees and lordosis from 32.4±20.9 degrees to 37.6±10.4 degrees. Hemoglobin levels were virtually unchanged. Weight increased from 10.6±2.8 kg to 20.2±5.0 kg at final follow-up, but body mass index decreased from 16.6±3.7 kg/m² to 15.7±2.9 kg/m². CONCLUSIONS: The expandable rib-based distraction device is an effective method to treat severe congenital scoliosis in very young children. It can control the deformity and promotes spinal and thoracic growth. LEVEL OF EVIDENCE: Level IV.

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.

Pedicle screw instrumentation with or without pelvic fixation in neuromuscular scoliosis: Outcome and complications in a series of 37 patients with a minimum 2-year follow-up.

OBJECTIVE: Neuromuscular scoliosis (NMS) is often associated with rapid progressive spinal deformities. Indications, when to extend the instrumentation to the pelvis for pelvic obliquity are not generally accepted at this time. This study reports on the indications, surgical technique and results using pedicle screw instrumentation exclusively with or without pelvic fixation for spine fusion in patients with NMS. PATIENTS AND METHODS: Thirty-seven NMS patients were treated with pedicle screw instrumentation (PSI) between 2007 and 2013 with a minimum follow-up of 24 months. The mean age at the time of operation was 14.91 ± 2.03 years (range, 11.17-18.58). Posterior spine fusion (PSF) was conducted in 33 patients; 4 patients underwent a combined anterior spinal release followed by PSF during one-stage surgery. Pelvic fixation was achieved by ilium screws combined with S-1 screws in 4 cases and by sacral-alar-iliac (SAI) technique by Sponseller in 9 cases, respectively. RESULTS: The mean primary Cobb angle was 65.5° (range, 14-103°) and improved significantly to 19.8° (range, 1-50°, p < 0.001) after surgery and 20.5° (range, 3-57, p = 0.47) at 2 years FU, respectively. Besides, an improvement of pelvic obliquity and T1 tilt angle could be detected. Major complications occurred in 19% and minor complications in 32%. CONCLUSION: Pedicle screw fixation only for spine fusion in patients with NMS can be applied safely with reasonable complication rates. An excellent correction in all planes, a significant improvement of the pelvic obliquity and almost no loss of correction at 2 years FU were observed.

Intrathecal baclofen therapy for treatment of spasticity in infants and small children under 6 years of age.

PURPOSE: The aim of this study is to prove the efficacy and safety of intrathecal baclofen therapy in infants and children below 6 years of age by retrospective analysis of our pediatric cohort of 135 primary pump implantations. METHODS: Between 2007 and 2018, 17 patients with pump implantations were below 6 years of age. Data were acquired retrospectively with a follow-up of 12 months to 11 years regarding complications. RESULTS: The youngest infant was 11 months at implantation with a bodyweight of 6, 4 kg, and 63 cm length. Surgical complications were comparable to published literature and mainly involved the catheter (2 catheter dislocations and 1 catheter transection) and one pump infection resulting in 4 revision surgeries in 3 patients. One baclofen-related apnea during titration and an overdose after refill were treated conservatively. Using a subfascial implantation technique, we observed neither skin ulceration nor pump infection since 2007. In a growing child, catheter slides are common and related to growth, scoliosis, spine surgery, and surgical failure. CONCLUSION: Intrathecal baclofen therapy in infants and small children is as safe and effective as published for older pediatric patients; therefore, intrathecal baclofen can be considered in all infants as long as an 8-cm incision fits into the triangle of the anterior superior iliac spine, costal margin of the 10th rib, and navel. We suggest the utilization of subfascial surgical technique for implantation pump and catheter. Titration of intrathecal baclofen should be performed slowly to avoid bradycardia in infants. This is a retrospective study (level of evidence 4).

Nerve transfer from the median to musculocutaneous nerve to induce active elbow flexion in selected cases of arthrogryposis multiplex congenita.

BACKGROUND: Arthrogryposis multiplex congenita (AMC) is a rare disease which affects mainly upper and lower extremities. Affected patients are not able to eat unassisted due to elbow contracture and nonexistent active elbow flexion. In traumatic brachial plexus palsies, a nerve transfer from either median or ulnar nerve to the musculocutaneous nerve has proved to induce active elbow flexion, and we report our results of such a procedure in a nontraumatic condition, that is, arthrogryposis. METHODS: We selected four patients with AMC type 1 (6 extremities, 2 males, 2 females) diagnosed with AMC presenting to our institution shortly after birth from 2014 to 2016 to perform a nerve transfer from the median nerve to the musculocutaneous nerve in order to induce active elbow flexion. The indication of application of this surgical procedure was based on active finger and wrist flexion, limited contracture of elbow joints and evidence of flexing muscle fibers detected by sonography. RESULTS: Five nerve transfers were conducted with a follow up of 2-5 years. Two extremities reached active elbow flexion motorgrade M4, two M3, and one M1 at latest follow up. One patient developed a postoperative suture granuloma. One nerve transfer was abandoned due to neuroanatomic variation. One extremity was treated with botulinum toxin in triceps muscle in addition to the nerve transfer. CONCLUSIONS: In this series of selected cases of AMC Type 1 we were able to induce active elbow flexion using a nerve transfer technique developed for traumatic and obstetric brachial plexus palsies. In four extremities the procedure achieved independent hand-to-mouth active elbow flexion. Level of evidence four.

Treatment of pediatric spinal deformity with use of recombinant human bone morphogenetic protein-2.

In pediatric spine surgery nonunion is a challenging issue. Instability may cause neurological impairment and lead to numerous surgeries in order to achieve fusion. The use of rhBMP-2 for pediatric spinal fusion has not been widely reported. In this study, a series of 13 children (14 procedures) that underwent spinal rhBMP-2 application were analyzed in order to measure clinical and radiographic outcome. Therefore, patient data, diagnosis, construct of instrumentation, type of bone graft, quantity of BMP used, and fusion outcome were reviewed. The study cohort included four female and nine male patients with a mean age of 11.2 years (range 2.6-19.2 years) at the time of rhBMP-2 application. Rh-BMP-2 was used in both primary (n = 6) and revision surgery (n = 8) in patients with a high risk for the development of nonunion. The mean follow-up was 51 months (range 12-108 months). Fusion occurred in 11 patients. Complications that may be due to application of rhBMP-2 were seen after four operations. Three patients had an increased body temperature and in one case prolonged wound secretion was evident, treated by local wound care or observation. In one of these patients an extensive postoperative hematoma occurred, necessitating surgical treatment. In conclusion, we could detect high fusion rates following the use of rhBMP-2 in pediatric spine surgery without an increased complication rate attributable to its application. Therefore we consider recombinant human BMP-2 to be an option in selected pediatric spinal procedures, especially in cases with compromised bone healing due to congenital, systemic, or local conditions.

Strategies for Treating Scoliosis in Early Childhood.

BACKGROUND: Scoliosis in early childhood is defined as abnormal curvature of the spine of any etiology that arises before age 10. The affected children are at high risk of developing restrictive pulmonary dysfunction. The treatment presents major challenges because of the complexity and high morbidity of the disease. METHODS: This article is based on pertinent articles retrieved by a selective literature search, and on the results of a retrospective study by the authors. RESULTS: In addition to conservative treatment methods including physiotherapy, casts, and corsets, progressive scoliosis usually requires early surgical intervention. In recent years, many different so-called non-fusion techniques have been developed for the surgical treatment of early childhood scoliosis. The goal of this new strategy is to avoid early fusion procedures and to enable further growth of the rib cage, lungs, and spine in addition to correcting the scoliosis. The authors also present their own intermediate-term results with a novel growth-preserving spinal operation that exploits magnet technology. CONCLUSION: Because of the low prevalence and heterogeneous etiology of early childhood scoliosis, the literature to date contains no randomized controlled therapeutic trials concerning this small group of high-risk patients. For the treatment to succeed, it is essential for specialists from all of the involved medical disciplines to work closely together. Conservative measures such as physiotherapy, casts, and corsets can delay the (frequent) need for surgery or even make surgery unneces - sary, particularly in the idiopathic types of early childhood scoliosis. The new non-fusion techniques enable continued growth of the spine, rib cage, and lung in addition to correcting the scoliosis.

Posterior Only Vertebral Column Resection for the Treatment of Severe Spinal Deformities in Pediatric Patients: A Retrospective Case Series.

OBJECTIVE: The treatment of severe spinal deformities in pediatric patients is very challenging. Posterior only vertebral column resection (PVCR) allows for correcting of severe deformities of the vertebral column via a posterior only procedure. We analyzed radiologic outcome of PVCR performed on a series of pediatric patients with severe congenital and acquired spinal deformities. METHODS: A case series of 11 pediatric patients with severe spinal deformity who were treated by PVCR between 2009 and 2013 were retrospectively analyzed. All patients had posterior instrumentation and reconstruction of the anterior column with titanium cages filled with autologous bone. Seven patients had pure kyphosis or kyphoscoliosis, whereas 4 patients were treated because of scoliotic deformities. The patient records were reviewed for demographic and general clinical data. Complications and adverse events, transfusion rates, and surgical time were recorded. Radiologic analysis included Cobb angles and percentage of correction, analysis of sagittal profile, time to fusion, and possible complications related to instrumentation. RESULTS: Average preoperative scoliosis of 61° was corrected to 32°, resulting in a 50% correction at final follow-up. Coronal imbalance was improved to 36% at the most recent follow-up. Mean preoperative kyphotic deformity was 90° and was corrected to 43° at the last follow-up evaluation. Intraoperative complications included loss of the neuromonitoring signals in 2 cases and pleural laceration in 1 case. CONCLUSIONS: PVCR for children is an effective and safe technique providing a successful correction of complex pediatric spinal deformities. Nevertheless, it remains a technically highly demanding procedure, implying the possibility of severe complications.

Preliminary Results of Magnetically Controlled Growing Rods for Early Onset Scoliosis.

BACKGROUND: Growth-sparing techniques for the treatment of early onset scoliosis (EOS) have developed significantly over the last years. Traditional growing rods (GRs) require repeated surgical lengthening under anesthesia. Since June 2011 we have been using the magnetically controlled growing rods (MCGR) to treat patients with progressive EOS. METHODS: Thirty-five patients with EOS of different etiologies underwent treatment with MCGR. We record about our preliminary results of 24 patients who fulfilled the inclusion criteria of a minimum follow-up (FU) of 12 month and >3 lengthenings. The mean age at surgery was 8.9±2.5 years. Correction of the primary curve after the index surgery and after lengthenings was measured on standing radiographs using the Cobb technique. T1-T12 and T1-S1 spinal length were also measured. Intraoperative and postoperative complications were recorded. RESULTS: The mean FU was 21.1±7.3 months. All patients had a minimum of 3 outpatient lengthenings [mean, 4.6±1.5 (range, 3 to 8)]. The mean primary curve was 63±15 degrees (range, 40 to 96 degrees) and improved to 29±11 degrees (range, 11 to 53 degrees; P<0.001) after MCGR. The mean major curve after most recent lengthening was 26 degrees (range, 8 to 60 degrees; P<0.07). The T1-T12 as well as the T1-S1 length increased significantly (P<0.001). The mean preoperative thoracic kyphosis decreased from 43±24 degrees (range, -32 to 86 degrees) to 27±12 degrees (range, 9 to 50 degrees; P<0.001) after surgery, respectively, and measured 32±12 degrees (range, 12 to 64 degrees; P<0.05) at last FU. In 1 patient a loss of distraction occurred making rod exchange necessary. Three patients developed a proximal junctional kyphosis and in another patient a screw pull out occurred that required revision surgery. CONCLUSIONS: Our results demonstrate that MCGR is a safe and effective nonfusion technique in the treatment of progressive EOS avoiding repeated surgical lengthening procedures. It provides adequate distraction similar to standard GR. The magnetically induced transcutaneous lengthening allows noninvasive distraction achieving spinal growth comparable to conventional GR techniques. LEVEL OF EVIDENCE: Level IV-retrospective nonrandomized objective study.

Prenatal latex sensitization in patients with spina bifida: a pilot study.

OBJECT: Patients with spina bifida are particularly vulnerable to developing immunoglobulin E (IgE)-mediated latex sensitization. Even though many risk factors leading to latex allergy in these patients have been described, it is still unclear whether the increased prevalence of latex sensitization is disease associated or due to the procedures used to treat spina bifida. The aim of this study was to assess prenatal latex sensitization in patients with spina bifida by examining IgE levels in umbilical cord blood. METHODS: Patients with spina bifida and matched healthy infants were recruited from the University Medical Center Hamburg-Eppendorf and Children's Hospital Altona. Latex-specific and total IgE were assessed in umbilical cord blood using ImmunoCAP testing to evaluate the degree of prenatal latex sensitization. RESULTS: Twenty-two subjects, 10 with spina bifida and 12 healthy individuals, were included. Subjects were selected after matching for sex, gestational age, weight, parental allergy profile, number of prenatal examinations, and utilization of latex tools during pregnancy (propensity score estimates, p = 0.36). In patients with spina bifida, latex-specific and total IgE levels were significantly higher than those in healthy individuals (p = 0.001). After normalization to total IgE, latex-specific IgE levels were higher, yet not significantly increased (p = 0.085). CONCLUSIONS: Perinatally, there is a significant augmentation of total and latex-specific IgE in patients with spina bifida. After correcting for total IgE, latex-specific IgE was increased, yet not significantly higher than in matched, healthy controls. This pilot study gives novel insights in the immunological reactions related to spina bifida. The increased latex-specific IgE levels could possibly be associated with the occurrence of a latex allergy in the future.

Denosumab: a potential new and innovative treatment option for aneurysmal bone cysts.

INTRODUCTION: Aneurysmal bone cysts (ABCs) are expansive and destructive lesions positive for osteoclast markers, resembling benign giant cell tumors (GCTs). Treatment options include surgical resection, curettage and cavity filling, embolization, injection of fibrosing agents, or radiotherapy. Particularly in children and adolescents with spinal ABCs, these options may be unsatisfactory, and innovative forms of treatment are needed. Denosumab is a human monoclonal antibody that inhibits osteoclast function by blocking the cytokine receptor activator of the nuclear factor-kappa B ligand. Satisfactory results with denosumab in treating GCTs and immunohistochemical similarities suggest that it may also have positive effects on ABCs. METHODS AND RESULTS: This report is the first description of the therapeutic use of denosumab in two patients with spinal ABCs. Two boys (aged 8 and 11) had recurrent ABCs at C5 after surgery with intralesional tumor resection. Treatment options were discussed by the interdisciplinary tumor board. Arterial embolization was attempted, but failed due to an absence of appropriate afferent arteries. After the families had received extensive information and provided written consent, denosumab therapy was initiated as an individualized treatment, despite the absence as yet of scientific evidence. After the start of denosumab therapy, both patients recovered from pain and neurologic symptoms significantly and are now in a healthy condition with no severe side effects. Magnetic resonance imaging check-ups after 2 or 4 months of denosumab treatment, respectively, showed tumor regression in both patients. DISCUSSION: Longer follow-up and clinical studies are warranted to establish the value of denosumab in the treatment of ABCs.

Hemivertebra resection in children, results after single posterior approach and after combined anterior and posterior approach: a comparative study.

PURPOSE: To compare the results after hemivertebra resection through a single posterior approach and through a combined anterior and posterior approach. METHODS: This is a retrospective study on patients treated by hemivertebra resection with monosegmental instrumentation for congenital scoliosis at a single institution. The patients were divided into two groups according to the surgical approach. Both groups were compared for curve correction, complication rate and perioperative data. Paired samples T test was used for statistical evaluation. RESULTS: Twenty-five consecutive patients were included. In 12 cases the hemivertebra resection was performed through a single posterior approach (SPA) and in 13 via a combined anterior and posterior approach (CAPA). Curve correction was similar in both groups (59 vs. 55%, p>0.05). Duration of surgery (272 vs. 319 min) and postoperative mechanical ventilation were shorter in the SPA group (5 vs. 30 h), but did not reach statistical significance (p>0.05). Significant blood loss necessitating blood transfusion was observed in six patients operated through an SPA and in 8 patients operated through a CAPA. The duration of the ICU management (1 vs. 3 days) and the hospital stay (12 vs. 19 days) were significantly shorter in the SPA group (p<0.05). Less surgery related general complications were observed in the SPA group (0 vs. 38%). CONCLUSIONS: Similar correction of the main and the compensatory curves can be achieved with single posterior and combined anterior and posterior hemivertebra resection. Benefits of the SPA are lower complication rate and shorter recovery period.

The use of recombinant human BMP-2 as a salvage procedure in the pediatric spine: a report on 3 cases.

The study is a retrospective case series. The objective is to review the results after off-label recombinant human BMP-2 (rhBMP-2) use in the pediatric spine after previously failed spinal fusion. Non-union in the pediatric spine is a challenging condition associated with increased morbidity due to instability, neurological impairment or multiple revision surgeries. BMP has been used with good results in the adult spine; however, information on its use in the pediatric population is still lacking. rhBMP-2 was used at our institution at revision posterior spinal surgery in three patients. Solid spinal fusion was achieved in all three cases despite underlying bone dysplasia (Hurler's disease), instability or bony substance loss. No adverse reactions due to rhBMP-2 use were observed. rhBMP-2 should be considered as potential option to achieve spinal fusion in children with compromised bone healing due to congenital, local or systemic conditions.