Pediatric neurology
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Pediatric neurology · Apr 2020
Sleep Parameters and Overnight Urinary Melatonin Production in Children With Persistent Post-concussion Symptoms.
Sleep disturbance is common after a mild traumatic brain injury (mTBI) in children, yet its biology is poorly understood. We aimed to explore sleep-related problems (SRPs), sleep-activity patterns, and endogenous melatonin production in children with different recovery trajectories following mTBI. We hypothesized that children with delayed recovery would have more SRPs and abnormal sleep-activity patterns, which would correlate with lower overnight melatonin production. ⋯ Although children with persistent post-concussion symptoms reported more SRPs, this was not related to actigraphy sleep parameters or melatonin production. Further research is warranted to understand the pathophysiology of post-traumatic sleep disturbance.
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Pediatric neurology · Mar 2020
Intranasal Ketamine for Abortive Migraine Therapy in Pediatric Patients: A Single-Center Review.
Ketamine has recently emerged as a promising therapeutic alternative for abortive migraine therapy, likely secondary to N-methyl-d-aspartate antagonism. Most reports examine adults and the intravenous route. Fewer utilize intranasal administration or pediatric populations. Given the limited evidence for intranasal ketamine in pediatric migraine populations, we retrospectively reviewed our experience to further characterize safety and efficacy of intranasal ketamine in this population. ⋯ Our experience with intranasal ketamine has promising outcomes in both pain relief and side effect minimization. When other therapeutic options are unavailable, practitioners should consider intranasal ketamine.
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Pediatric neurology · Feb 2020
Case ReportsBortezomib-Responsive Refractory Anti-N-Methyl-d-Aspartate Receptor Encephalitis.
Anti-N-methyl-d-aspartate receptor encephalitis is a central nervous system inflammatory autoimmune disease affecting adults and children. The use of first- and second-line immunotherapies is supported. Recent reports suggest the efficacy of bortezomib in severe anti-N-methyl-d-aspartate encephalitis in adult patients not responsive to second-line treatment; there are no data about pediatric patients. ⋯ This is the first report of the use of bortezomib in children with anti-N-methyl-d-aspartate encephalitis; it could be a useful therapeutic option in children with refractory anti-N-methyl-d-aspartate encephalitis.
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Pediatric neurology · Nov 2019
ReviewFrom Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1.
Spinal muscular atrophy is a devastating neurodegenerative autosomal recessive disease that results from survival of motor neuron 1 (SMN1) gene mutation or deletion. Patients with spinal muscular atrophy type 1 utilizing supportive care, which focuses on symptom management, never sit unassisted, and 75% die or require permanent ventilation by age 13.6 months. Onasemnogene abeparvovec (Zolgensma, formerly AVXS-101) is a gene replacement therapy comprising an adeno-associated viral vector containing the human SMN gene under control of the chicken beta-actin promoter. ⋯ The only treatment-related adverse event observed was transient asymptomatic transaminasemia that resolved with a short course of prednisolone treatment. This review discusses the biological rationale underlying gene replacement therapy for spinal muscular atrophy, describes the onasemnogene abeparvovec clinical trial experience, and provides expert recommendations as a reference for the real-world use of onasemnogene abeparvovec in clinical practice. As of May 24, 2019, the Food and Drug Administration approved onasemnogene abeparvovec, the first gene therapy approved to treat children younger than two years with spinal muscular atrophy.
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Pediatric neurology · Oct 2019
Review Case ReportsTolosa-Hunt Syndrome: Clinical Manifestations in Children.
Tolosa-Hunt syndrome (THS) is a rare condition in children characterized by painful ophthalmoplegia caused by inflammation of unknown etiology in the cavernous sinus, superior orbital fissure, or orbital apex. Our main purpose was to report two pediatric cases of THS, a typical one and another extremely rare one preceded by facial palsy. ⋯ THS is a rare condition in pediatric population that requires an extensive evaluation before the final diagnosis is made. Seventh nerve palsy preceding the diagnosis of THS is particularly rare in children. This patient represents only the second reported example of seventh nerve involvement in a child with THS.