INTEREST IN CD2, a global patient-centred study of long-term cervical dystonia treatment with botulinum toxin

Vijay P Misra,Tae Mo Chung,Tae Mo Chung,Savary Om,David Charles,Pascal Maisonobe,Carlo Colosimo,Interest In Cd2 Study Group

doi:10.1007/s00415-017-8698-2

Abstract

BackgroundLongitudinal cohort studies provide important information about the clinical effectiveness of an intervention in the routine clinical setting, and are an opportunity to understand how a population presents for treatment and is managed.MethodsINTEREST IN CD2 (NCT01753349) is a prospective, international, 3-year, longitudinal, observational study following the course of adult idiopathic cervical dystonia (CD) treated with botulinum neurotoxin type A (BoNT-A). The primary objective is to document long-term patient satisfaction with BoNT-A treatment. Here we report baseline data.ResultsThis analysis includes 1036 subjects (67.4% of subjects were female; mean age was 54.7 years old; mean TWSTRS Total score was 31.7). BoNT-A injections were usually given in line with BoNT-A prescribing information. The most commonly injected muscles were splenius capitis (87.3%), sternocleidomastoid (82.6%), trapezius (64.3%), levator scapulae (40.9%) and semispinalis capitis (26.9%); 35.5% of subjects were injected using a guidance technique. Most subjects (87.8%) had been previously treated with BoNT-A (median interval between last pre-study injection and study baseline was 4 months); of these 84.8% reported satisfaction with BoNT-A treatment at peak effect during their previous treatment cycle and 51.5% remained satisfied at the end of the treatment. Analyses by geographical region revealed heterogeneity in the clinical characteristics and BoNT-A injection practice of CD subjects presenting for routine treatment.ConclusionsThese baseline analyses provide sizeable data regarding the epidemiology and clinical presentation of CD, and demonstrate an international heterogeneity of clinical practice. Future longitudinal analyses of the full 3-year study will explore how these factors impact treatment satisfaction.

Highlights

Cervical dystonia (CD) is the most common form of focal dystonia in adults and is primarily characterised by twisting or turning of the neck causing an abnormal head position [1,2,3]
The study was conducted in compliance with the International Society for Pharmacoepidemiology (ISPE) Guidelines for Good Pharmacoepidemiology Practices (GPP) [16]; it began on 10 December 2012 and last visit of the last patient occurred on 25 September 2017
Participating centres needed to be familiar with Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) and Tsui scales in their clinical practice

Summary

Introduction

Cervical dystonia (CD) is the most common form of focal dystonia in adults and is primarily characterised by twisting or turning of the neck causing an abnormal head position [1,2,3]. It is increasingly recognised that while placebo-controlled trials remain the gold standard in assessing response to a therapeutic intervention, they do not provide adequate information of clinical effectiveness and safety of an intervention in the typical clinical setting. Such trials typically employ narrow inclusion and exclusion criteria, and assess only a restricted selection of endpoints. Longitudinal cohort studies provide important information about the clinical effectiveness of an intervention in the routine clinical setting, and are an opportunity to understand how a population presents for treatment and is managed. Future longitudinal analyses of the full 3-year study will explore how these factors impact treatment satisfaction

Methods

Results

Conclusion