Hip Instability in Children With Spinal Muscular Atrophy: A Retrospective Study.

PURPOSE: Hip subluxation and dislocation are known problems for children with spinal muscular atrophy (SMA). Medical complexity of these children typically results in absence of monitoring and intervention for pathologic hips. Patterns of hip migration and acetabular morphology in SMA have not been described. This study examines the natural progression of hip instability across all types of SMA in a pediatric population. METHODS: Following institutional review board approval, a retrospective chart review of all children with SMA at our institution was performed. All x-rays taken before the age of 18 years containing adequate projections of the pelvis were measured for Reimer migration index (MI), acetabular depth ratio (ADR), and acetabular index (AI). Demographics and clinical data were collected including use of nusinersen, ambulatory status, contractures, and hip pain. Linear mixed effects model was fit to serial MI measures of individual hips with fixed effects consisting of SMA type, age at x-ray, and their interaction. ADR and AI measures were similarly modeled following conversion of raw values to z-scores based on the model developed by Novais et al Slope indicated rate of measure change as a function of age. RESULTS: Forty-five children (22 males) with SMA types 1 to 3 were included in this retrospective study. Six children were classified as type 1, 25 were type 2, and 14 were type 3. The interaction of age by SMA type was statistically significant (P=0.01), indicating a difference in the rate of hip subluxation between the 3 SMA types as measured by MI. By age 4, MI values were different from one another across all 3 groups (P<0.01). ADR decreased with age across all SMA types. The slopes of ADR regression lines were negative and statistically significant between the 3 groups (P=0.002). AI values were higher for all types of SMA, which is the opposite of expected in normal hips. CONCLUSIONS: Hip subluxation occurs across all SMA types, most rapidly in SMA type 1. Regression lines of ADR and AI compared with those seen in unaffected populations suggest hips in children with SMA demonstrate a difference in morphology of the acetabulum and do not follow normal adaptive remodeling. As treatments advance and the population of SMA patients becomes more mobile, there is an increased need to monitor hip instability in children with SMA, thus making orthopaedic management an important consideration.

A consensus statement on the use of botulinum toxin in pediatric patients.

Botulinum toxin has been used in medicine for the past 30 years. However, there continues to be controversy about the appropriate uses and dosing, especially in the pediatric population. A panel of nine pediatric physiatrists from different regions and previous training programs in the United States were nominated based on institutional reputation and botulinum toxin (BoNT) experience. Based on a review of the current literature, the goal was to provide the rationale for recommendations on the administration of BoNT in the pediatric population. The goal was not only to review safety, dosing, and injection techniques but also to develop a consensus on the appropriate uses in the pediatric population. In addition to upper and lower limb spasticity, the consensus also provides recommendations for congenital muscular torticollis, cervical dystonia, sialorrhea, and brachial plexus palsies.

Out of the spasticity box: Off-label uses of botulinum toxin in children.

Botulinum toxin is frequently used as a therapeutic for a variety of non-FDA approved indications in children. This narrative literature review explores three off-label uses of botulinum toxin in pediatric conditions including congenital muscular torticollis, spastic trismus, and neonatal brachial plexus palsy. While more research is needed to establish treatment, dosing and localization guidelines for the use of botulinum toxin in these commonly treated conditions, available evidence is discussed.

Pediatric spinal cord tumors and masses.

BACKGROUND/OBJECTIVE: Spinal cord tumors are a relatively rare diagnosis, accounting for 1% to 10% of all pediatric central nervous system tumors. Understanding the etiology and clinical outcomes of these tumors is therefore very important. This study presents detailed information regarding clinical presentation, histological findings, outcomes, functional assessment, and management of a series of patients with this diagnosis. METHOD: Retrospective, descriptive study. SUBJECTS: Thirty-five children with a final diagnosis of spinal cord tumor or mass, excluding dysraphism. RESULTS: Neurodevelopmental tumors (dermoid tumors, epidermoid tumors, and teratomas) were the most common tumor type (31%), followed by astrocytomas (29%) and neuroblastomas (14%). Other types included schwannomas, meningiomas, giant cell tumors, extradural cystic masses, leukemic-related masses, and masses related to neurofibromatosis. Mean age at diagnosis was 6.6 years (SD = 5.5 y) and did not vary significantly by tumor type except for children with neuroblastoma (mean = 0.4 y, SD = 0.5 y). More boys (57%) were identified in the series than girls (43%); however, there was no association between tumor type and sex. Presenting complaints of pain were noted in 57% and were localized to the back, neck, or extremities. Extremity weakness was reported as an initial presenting symptom in 46%. Three children had scoliosis as a presenting issue and 14 had gait abnormalities. Regardless of treatment modality, mobility was retained in 83% of children with or without gait aids. Neurogenic bowel and/or bladder were present in 23% of the population. CONCLUSIONS: This study corroborates other studies indicating that intramedullary tumors are the predominant form of pediatric spinal cord tumor. This population, however, presented with an unusually large number of developmental tumors, contrary to several published studies. The disparity may be the result of this institution acting as a regional referral center, thus increasing the number of this type of patient. The population is too small to make any other conjecture. The predominance of astrocytomas and neuroblastomas among those patients with poor outcomes and the prevalence of developmental tumors suggest the need for broader investigation. Although, in general, spinal cord tumors are relatively rare, this preliminary study supports the need to further evaluate associations between tumor type, presenting symptoms, treatment, and functional outcome in children with spinal cord tumors.

Botulinum toxin type a in the treatment of children with congenital muscular torticollis.

This is a retrospective case series describing the use of botulinum toxin type A in the treatment of children with congenital muscular torticollis who fail to progress with conservative management. A total of 27 children with congenital muscular torticollis, 6-18 mos of age, received 30 botulinum toxin type A injections into their sternocleidomastoid or upper trapezius muscle, or both, at a pediatric tertiary care center between 1995 and 2001. Three children received repeat injections. Twenty of 27 children (74%) had improved cervical rotation or head tilt after the injections, and 2 of 27 (7%) experienced transient adverse events, specifically, mild dysphagia and neck weakness. This series suggests that botulinum toxin type A may be a safe and effective treatment option for children with congenital muscular torticollis who are unresponsive to a traditional regimen of physical therapy and a home program. A prospective, randomized controlled trial is necessary to definitively assess the role of botulinum toxin type A in this population.