Teaching Video NeuroImages: Palatal myoclonus in leukodystrophies

Martínez‐Poles, Javier; Escribano-Paredes, J B; García-Madrona, Sebastián; Nedkova-Hristova, Velina; Jiménez‐Escrig, Adriano

doi:10.1212/wnl.0000000000008795

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“…3,[5][6][7] Botulinum toxin blocks acetylcholine release, inhibiting secretion of saliva. Several trials have demonstrated efficacy and safety of intraglandular botulinum toxin injections for sialorrhea in adults [8][9][10][11][12][13][14] and children. 1,[15][16][17][18][19][20][21][22][23][24][25][26] At the time of study planning, the clinical effect of repeated botulinum toxin injections for children had not been tested, optimal doses were undefined, and no studies had fulfilled approval requirements.…”

Section: Introductionmentioning

confidence: 99%

Placebo-Controlled Clinical Trial of IncobotulinumtoxinA for Sialorrhea in Children

et al. 2021

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Background and Objectives:This prospective phase III study (SIPEXI) investigated efficacy and safety of repeated injections of incobotulinumtoxinA (incoBoNT/A) for treatment of chronic sialorrhea (drooling) associated with neurological disorders (e.g., cerebral palsy, traumatic brain injury) and/or intellectual disability in children/adolescents.Methods:The study enrolled 2-17-year-olds with sialorrhea due to neurological disorders and/or intellectual disability. Patients received body weight-dependent doses of incoBoNT/A (20 U to 75 U). A main period with 1 injection cycle (placebo-controlled, double-blind, 6-17-year-olds) was followed by an open-label extension with up to 3 further cycles. An additional cohort of 2-5-year-olds received active treatment throughout the study. Co-primary endpoints were the change in unstimulated salivary flow rate (uSFR) from baseline to week 4, and the carers’ global impression of change scale (GICS) rating at week 4. Adverse events were recorded.Results:In the main period, 220 patients aged 6-17 years were randomized and treated (148 patients in incoBoNT/A group, 72 patients in placebo group). 35 patients aged 2-5 years received incoBoNT/A (no placebo). 214 patients aged 6-17 years and 33 patients aged 2-5 years continued treatment in the open-label extension period. For the 6-17-year-olds, a significant difference between incoBoNT/A and placebo was seen in the mean uSFR decrease (difference: -0.06 g/min; p = 0.0012) and the carers’ GICS rating (difference: 0.28 points; p = 0.032) at week 4, in favor of active treatment. The secondary endpoints consistently supported these results. A sustained benefit was observed during the extension. Incidences of adverse events were comparable between incoBoNT/A and placebo and did not increase notably with repeated injections. The most common adverse events were respiratory infections. Efficacy and safety were also favorable in the uncontrolled cohort of 2-5-year-olds.Discussion:Both co-primary efficacy endpoints were reached and superiority of incoBoNT/A over placebo was confirmed. IncoBoNT/A (up to 75 U, up to 4 cycles) is an effective and well-tolerated treatment for sialorrhea associated with neurological disorders in children.Study registrations:Clinicaltrials.gov: NCT02270736 (www.clinicaltrials.gov/ct2/show/results/NCT02270736); EU Clinical Trials Register: 2013-004532-30 (www.clinicaltrialsregister.eu/ctr-search/search?query=2013-004532-30).Classification of evidence:This study provides Class I evidence that injection of incobotulinumtoxinA decreases drooling in children aged 6-17 years with neurological disorders.

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