Glycopyrronium 320 μg/mL in children and adolescents with severe sialorrhoea and neurodisabilities: A randomized, double-blind, placebo-controlled trial.

Autor: Fayoux P; Department of Paediatric Otolaryngology Head Neck Surgery, Jeanne de Flandre Hospital, Lille, France.; ULR 2694 - METRICS: Évaluation des technologies de santé et des pratiques médicales, Université de Lille, Lille, France., Dinomais M; Department of Physical Medicine and Rehabilitation, CHU Angers-Les Capucins, Angers, France., Shaw H; Proveca Ltd, Manchester, UK., Villain F; Proveca Ltd, Manchester, UK., Schwartz D; Kappa Santé, Paris, France., Rondeau S; Department of Early Medico-Social Action (CAMSP), CHU de Rouen, Rouen, France., Letellier G; Department of Physical Medicine and Rehabilitation, ESEAN-APF, Nantes, France., Auvin S; APHP, Service de Neurologie Pédiatrique, EpiCARE ERN membre, Hôpital Robert Debré, Paris, France.; INSERM NeuroDiderot, Université Paris-Cité, Paris, France.; Institut Universitaire de France (IUF), Paris, France.
Jazyk: angličtina
Zdroj: Developmental medicine and child neurology [Dev Med Child Neurol] 2024 Jul; Vol. 66 (7), pp. 910-918. Date of Electronic Publication: 2024 Jan 12.
DOI: 10.1111/dmcn.15841
Abstrakt: Aim: To investigate the efficacy, safety, and impact on quality of life (QoL) of an oral formulation of 320 μg/mL glycopyrronium designed for children.
Method: A double-blind, placebo-controlled SALIVA (Sialanar plus orAl rehabiLitation against placebo plus oral rehabilitation for chIldren and adolescents with seVere sialorrhoeA and neurodisabilities) trial was conducted. Children (3-17 years) with neurodisabilities and severe sialorrhoea (modified Teachers Drooling Scale ≥6) were randomized to 320 μg/mL glycopyrronium or placebo, in addition to non-pharmacological standard care.
Results: Of 87 participants, 44 were aged 10 years or under and 43 had cerebral palsy. The primary endpoint, change in total Drooling Impact Scale (DIS) score from baseline to day 84, was significantly greater (improved) with 320 μg/mL glycopyrronium versus placebo (median [quartile 1, quartile 3] -29.5 [-44.5, 0] vs -1 [-16, 5]; p < 0.001), an effect also observed at day 28 (median - 25 vs -2; p < 0.01). Significant reduction in bibs/clothes used per day was seen with glycopyrronium versus placebo at day 84 (median - 2 vs 0; p < 0.01). Glycopyrronium significantly improved DIS items 9 and 10 related to the extent that drooling affects the child's and family's life (p ≤ 0.03). Adverse events were reported by 77.3% and 69.8% of children with glycopyrronium and placebo respectively; the most common treatment-related adverse event was constipation (20.5% and 16.3%).
Interpretation: The formulation of 320 μg/mL glycopyrronium significantly improved drooling and reduced its impact on QoL, with good tolerability in children with neurodisabilities.
What This Paper Adds: The formulation of 320 μg/mL glycopyrronium significantly improved Drooling Impact Scale score versus placebo at day 84. The formulation reduced the impact of drooling on the child's and family's quality of life. There were no safety or tolerability concerns with this specific formulation.
(© 2024 Proveca Ltd. Developmental Medicine & Child Neurology published by John Wiley & Sons Ltd on behalf of Mac Keith Press.)
Databáze: MEDLINE
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