Dataset Information

[Treatment satisfaction in patients with cervical dystonia : Subgroup analysis of INTEREST-IN-CD-2].

ABSTRACT:

Background

Even though treatment guidelines recommend botulinum neurotoxin A (BoNT-A) as first line treatment for primary cervical dystonia (CD), there are only limited data on how BoNT-A-injections are administered in routine clinical practice.

Objective

This subgroup analysis evaluated patient satisfaction and symptom control under consideration of BoNT‑A treatment modalities in German and Austrian CD patients (DE/AT, n = 79) compared to the full international cohort (n = 995).

Material and methods

The INTEREST-IN-CD2 was a prospective, multicenter, longitudinal observational study. Course of treatment in adult primary CD patients under BoNT‑A treatment was assessed over a time period of 3 years. Primary outcome measure was the long-term satisfaction of patients with treatment, measured as maximum satisfaction between two consecutive injections as well as satisfaction at the time of reinjection.

Results

Treatment satisfaction at the maximum effect was stable and comparably good in both populations during the study (82.3-92.7% and 85.0-89.9%). Satisfaction decreased with decreasing BoNT‑A effect at the end of the treatment interval: it was comparable at the start of the study in both groups (54.2% vs. 51.4%), decreased numerically in the DE/AT group to 32.7% but remained stable in the total population. Analysis of Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) and Tsui scores did not reveal any substantial differences between the DE/AT subgroup and total cohort.

Conclusion

Overall, the study confirmed good clinical symptom control by BoNT‑A. The numerical differences in the current satisfaction seen in the comparison of DE/AT to the total cohort are possibly due to different proportions of BoNT‑A naïve patients in both groups, as they expressed different levels of satisfaction than previously treated patients.

SUBMITTER: Ip CW

PROVIDER: S-EPMC8648643 | biostudies-literature |

REPOSITORIES: biostudies-literature

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Project description:IntroductionThe Cervical Dystonia Patient Registry for Observation of OnabotulinumtoxinA Efficacy (CD PROBE) study (ClinicalTrials.gov identifier: NCT00836017), a multicenter, prospective, observational registry, was designed to identify real-world practices and outcomes for patients with cervical dystonia (CD) treated with onabotulinumtoxinA (onabotA). This secondary analysis from CD PROBE aims to determine the impact of presentation subtype on onabotA utilization and CD severity.Materials and methodsThe study cohort includes those who completed all 3 treatments, 4 office visits, and had data recorded for all assessments. Patient outcomes were assessed with the Cervical Dystonia Impact Profile (CDIP-58), Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS), and determination of CD severity. Treatment interval, dose, and adverse events (AEs) were also recorded. Data were stratified according to prior exposure to botulinum toxins (BoNTs) and analyzed with descriptive statistics.ResultsTorticollis was the most common presentation subtype in the study cohort (N = 350); the proportion of patients with torticollis was highest in those with severe disease. At each treatment, between 40.7 and 65.2% of those categorized as severe shifted to moderate or mild severity after treatment. Sustained improvements in CDIP-58 and TWSTRS were observed regardless of prior exposure to BoNTs. Dosing of onabotA generally increased from injection 1 to injection 3 and tended to be lower for patients naïve to BoNT. Median time interval between injections for the study cohort was 94.0 to 97.5 days. The most common AEs (dysphagia, muscular weakness) and injection intervals were similar between naïve vs. non-naïve patients; there were no serious treatment-related AEs.ConclusionsThis secondary cohort analysis from CD PROBE demonstrates that three repeat treatments with onabotA at intervals consistent with labeling attenuated disease severity and neck pain, resulting in sustained improvements in physician- and patient-reported outcomes. No new safety signals were identified.

Project description:BackgroundCervical dystonia (CD) involves painful involuntary contraction of the neck and shoulder muscles and abnormal posture in middle-aged adults. Botulinum neurotoxin type A (BoNT-A) is effective in treating CD but little is known about its associated cost-effectiveness.ObjectiveTo evaluate the cost-effectiveness of abobotulinumtoxinA for treating CD from the UK payer perspective.MethodsA Markov model was developed to evaluate the cost-effectiveness of abobotulinum-toxinA versus best supportive care (BSC) in CD, with a lifetime horizon and health states for response, nonresponse, secondary nonresponse, and BSC in patients with CD (mean age: 53 years; 37% male). Clinical improvement measured using Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) was mapped to utility using data from a randomized trial of abobotulinumtoxinA. Health care resource use, costs, and other inputs were from the British National Formulary, Personal Social Services Research Unit, published literature, or expert opinion. Costs and outcomes were discounted at 3.5% per annum.ResultsIn the base case, the incremental lifetime quality-adjusted life-years (QALYs) gained from abobotulinumtoxinA arm versus BSC was 0.253 per patient, whereas the incremental cost was £7,160, leading to an incremental cost-effectiveness ratio (ICER) of £30,468 per QALY. One-way sensitivity analyses showed that these results were sensitive to the proportion of responders to abobotulinumtoxinA at first injection, duration between injections, the number of reinjections allowed among primary nonresponders, and any difference in baseline TWSTRS value between the BSC and abobotulinumtoxinA arms. Probabilistic sensitivity analysis showed that abobotulinumtoxinA was cost-effective 46% and 49% of times at thresholds of £20,000 and £30,000 per QALY, respectively. Scenarios are considered including vial-sharing, productivity losses, secondary response/nonresponse at subsequent injections, 5-year time horizon, and alternative reinjection intervals for BoNT-As produced ICERs ranging from cost-saving to £40,777 per QALY, versus BSC.ConclusionAbobotulinumtoxinA was found to be cost-effective in treating adults with CD, at acceptable willingness-to-pay thresholds in the UK.