Search citation statements
Paper Sections
Citation Types
Year Published
Publication Types
Relationship
Authors
Journals
BackgroundInjectable daxibotulinumtoxinA (an investigational botulinum toxin, RT002) may offer a more prolonged duration of response—and therefore less frequent dosing—than onabotulinumtoxinA.ObjectivesTo perform a phase 2, open‐label, dose‐escalation study to assess the efficacy and safety of daxibotulinumtoxinA in cervical dystonia.MethodsSubjects with moderate‐to‐severe isolated cervical dystonia were enrolled in sequential cohorts to receive a single open‐label, intramuscular dose of injectable daxibotulinumtoxinA of up to 200 U (n = 12), 200–300 U (n = 12), or 300–450 U (n = 13; https://clinicaltrials.gov identifier NCT02706795).ResultsOverall, 33/37 enrollees completed the trial. DaxibotulinumtoxinA was associated with mean reductions in Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS)‐Total score of 16.8 (38%) at week 4, 21.3 (50%) at week 6, and 12.8 (30%) at week 24. The proportion of subjects who were responders (achieved ≥ 20% reduction in TWSTRS‐Total score) was 94% at week 6 and 68% at week 24. The median duration of response (time until > 20% of the improvement in TWSTRS‐Total score achieved at week 4 was no longer retained or re‐treatment was needed) was 25.3 weeks (95% CI, 20.14–26.14 weeks). There were no serious adverse events and there was no apparent dose‐related increase in the incidence of adverse events. The most common treatment‐related adverse events were dysphagia (14%) and injection site erythema (8%).ConclusionsPreliminary assessments suggest that injectable daxibotulinumtoxinA at doses up to 450 U is well tolerated and may offer prolonged efficacy in the treatment of cervical dystonia. Further studies involving larger numbers of patients are now warranted.
BackgroundInjectable daxibotulinumtoxinA (an investigational botulinum toxin, RT002) may offer a more prolonged duration of response—and therefore less frequent dosing—than onabotulinumtoxinA.ObjectivesTo perform a phase 2, open‐label, dose‐escalation study to assess the efficacy and safety of daxibotulinumtoxinA in cervical dystonia.MethodsSubjects with moderate‐to‐severe isolated cervical dystonia were enrolled in sequential cohorts to receive a single open‐label, intramuscular dose of injectable daxibotulinumtoxinA of up to 200 U (n = 12), 200–300 U (n = 12), or 300–450 U (n = 13; https://clinicaltrials.gov identifier NCT02706795).ResultsOverall, 33/37 enrollees completed the trial. DaxibotulinumtoxinA was associated with mean reductions in Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS)‐Total score of 16.8 (38%) at week 4, 21.3 (50%) at week 6, and 12.8 (30%) at week 24. The proportion of subjects who were responders (achieved ≥ 20% reduction in TWSTRS‐Total score) was 94% at week 6 and 68% at week 24. The median duration of response (time until > 20% of the improvement in TWSTRS‐Total score achieved at week 4 was no longer retained or re‐treatment was needed) was 25.3 weeks (95% CI, 20.14–26.14 weeks). There were no serious adverse events and there was no apparent dose‐related increase in the incidence of adverse events. The most common treatment‐related adverse events were dysphagia (14%) and injection site erythema (8%).ConclusionsPreliminary assessments suggest that injectable daxibotulinumtoxinA at doses up to 450 U is well tolerated and may offer prolonged efficacy in the treatment of cervical dystonia. Further studies involving larger numbers of patients are now warranted.
Introduction The efficacy and safety of abobotulinumtoxinA in the management of cervical dystonia has been established in randomized, controlled trials that use a selected trial population. In this meta-analysis of observational data, we evaluated the real-life effectiveness of abobotulinumtoxinA as delivered in routine clinical practice. Methods Meta-analysis of patient-level data for adult patients with cervical dystonia treated with abobotulinumtoxinA from three prospective, multicenter, observational studies ( NCT01314365 , NCT00833196 and NCT01753349 ). Results We report data for patients treated with abobotulinumtoxinA over one injection cycle at 181 neurology centers in 35 countries. CD clinical features as assessed by Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) Total scores ( N = 920) significantly reduced by a mean [95%CI] of −12.9 [−13.9, −11.8] points at Week 4 ( N = 449) and −3.2 [−3.8, −2.7] points at the end of the injection cycle ( N = 890). All three TWSTRS domains (symptom severity, disability and pain) contributed to the overall improvement. Patients were generally content with symptom control at peak effect of the treatment cycle, with 86% reporting overall satisfaction. Conclusion Findings from this meta-analysis of observational studies confirm the effectiveness of abobotulinumtoxinA in routine practice. Despite inclusion of a broader population sample, the magnitude of improvements observed is consistent with that seen in the pivotal, randomized controlled trials.
ZusammenfassungIm Rahmen einer multizentrischen Patientenund Arztbefragung wurden 497 Patienten mit zervikaler Dystonie (n = 372) und Blepharospasmus (n = 125) in Bezug auf die Zufriedenheit mit der Botulinumtoxin-Therapie (BoNT) befragt. Zusätzlich wurden krankheits- und behandlungsbezogene Angaben, Einstufungen zur Lebensqualität, der beruflichen Situation, psychosozialen Variablen, Verhaltensdaten und Aussagen zur emotionalen Situation erhoben. Trotz der im Durchschnitt guten Beurteilung der BoNT-Therapie fanden sich 197 (40,8%) weniger zufriedene Patienten. Die Zufriedenheit stand in keinem Zusammenhang mit Angaben zur Erkrankung und Behandlung oder der subjektiven Beurteilung der Symptomschwere. Weniger zufriedene Patienten stuften ihre allgemeine Lebensqualität als schlecht ein, zeigten sich in verschiedenen Lebensbereichen als stark belastet und neigten dazu, soziale Situationen zu vermeiden. Außerdem gaben sie kurz vor dem Ende des Behandlungsintervalls eine noch schlechtere Beurteilung des Therapieergebnisses an als die zufriedenen Patienten. Die Ergebnisse werfen zumindest bei den weniger zufriedenen Patienten die Frage nach einer stärkeren Individualisierung der Behandlungsintervalle sowie und nach einem genaueren Monitoring des Krankheitsverhaltens und entsprechender psychoedukativer Maßnahmen auf.
scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.
hi@scite.ai
10624 S. Eastern Ave., Ste. A-614
Henderson, NV 89052, USA
Copyright © 2024 scite LLC. All rights reserved.
Made with 💙 for researchers
Part of the Research Solutions Family.