The purpose of this focused review is to provide an overview of neuromuscular scoliosis from the perspective of the rehabilitation physician. Scoliosis is a common consequence of neuromuscular diseases, including central nervous system disorders such as cerebral palsy and spinal cord injury; motor neuron disorders, for example, spinal muscular atrophy; muscle fiber disorders, for example, Duchenne muscular dystrophy; multifactorial disorders, for example, spina bifida; and many other neuropathic and myopathic conditions. Unlike adolescent idiopathic scoliosis, which is the most common form of spinal deformity, neuromuscular scoliosis is more severe and more progressive, and is associated with more morbidity. Factors that contribute to this spinal deformity include asymmetric paraplegia, imbalance of mechanical forces, intraspinal and congenital anomalies of the spine, altered sensory feedback, and abnormal posture via central pathways. Spinal deformity combined with limitations due to an underlying neuromuscular condition lead to significant physiologic impairments that affect limb movement, cardiopulmonary function, gait, standing, sitting, balance, trunk stability, bimanual activities, activities of daily living, and pain, as well as concerns with self-image and social interactions. Evaluation and management of this population requires understanding of disease progression, pulmonary status, functional limitations, indications for conservative and surgical interventions, and social considerations.
Objective To characterize the population pharmacokinetics (PK) of oral baclofen and assess impact of patient-specific covariates in children with cerebral palsy (CP) in order to support its clinical use. Subjects design Children (2-17 years of age) with CP received a dose of titrated oral baclofen from 2.5 mg 3 times a day to a maximum tolerated dose of up to 20 mg 4 times a day. PK sampling followed titration of 10-12 weeks. Serial R- and S-baclofen plasma concentrations were measured for up to 16 hours in 49 subjects. Population PK modeling was performed using NONMEM 7.1 (ICON PLC; Ellicott City, Maryland). Results R- and S-baclofen showed identical concentration-time profiles. Both baclofen enantiomers exhibited linear and dose/kg-proportional PK, and no sex differences were observed. Average baclofen terminal half-life was 4.5 hours. A 2-compartment PK model with linear elimination and transit absorption steps adequately described concentration-time profiles of both baclofen enantiomers. The mean population estimate of apparent clearance/F was 0.273 L/h/kg with 33.4% inter-individual variability (IIV), and the apparent volume of distribution (Vss/F) was 1.16 L/kg with 43.9% IIV. Delayed absorption was expressed by a mean transit time of 0.389 hours with 83.7% IIV. Body weight, a possible genetic factor, and age were determinants of apparent clearance in these children. Conclusion The PK of oral baclofen exhibited dose-proportionality and were adequately described by a 2-compartment model. Our population PK findings suggest that baclofen dosage can be based on body weight (2 mg/kg per day) and the current baclofen dose escalation strategy is appropriate in the treatment of children with CP older than 2 years of age.
Objectives: Non-selective laryngeal reinnervation (NSLR) using the ansa cervicalis to the recurrent laryngeal nerve (RLN) is a promising treatment option for pediatric unilateral neuronal vocal fold movement impairment (VFMI). The aim is to describe our clinical outcomes with this technique and to identify preoperative characteristics that may predict postoperative voice outcomes.Methods: This is a cohort study of pediatric patients with unilateral neuronal VFMI, who underwent NSLR from March 2012 to July 2018. Pre-and postoperative Pediatric Voice Related Quality of Life (PVRQOL) questionnaires, Consensus Auditory Perceptual Evaluation of Voice (CAPE-V) ratings, and objective voice measures were obtained. In addition, patients underwent preoperative laryngeal electromyography (LEMG).Results: Thirty-two patients were identified. Twenty-one had complete data sets for analysis. The mean duration of VFMI was 9.02 years (range 1.1-26.1 years). There were significant improvements in PVRQOL (P = .0005), in all CAPE-V subsets (P ≤ .0001 to .0195), mean and maximum intensities (P = .0342 and 0.0110, respectively), cepstral peak prominence (P = .0001), and cepstral spectral index of dysphonia (P ≤ .0001). A worse preoperative LEMG correlated with a greater change in maximum phonation time (P = .0162) and maximum intensity (P = .0346). Age at injury and duration of injury had no significant impact on voice outcomes; however, patients with concurrent posterior glottic insufficiency did have smaller changes in PVRQOL (P = .012).Conclusion: NSLR is an effective treatment for pediatric unilateral neuronal VFMI even many years after initial RLN injury. LEMG may help predict voice outcomes of reinnervation in pediatric patients, but further data is still needed.
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