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Sustained functional benefits after a single set of injections with abobotulinumtoxinA using a 2-mL injection volume in adults with cervical dystonia: 12-week results from a randomized, double-blind, placebo-controlled phase 3b study.

ABSTRACT: Cervical dystonia (CD) is primarily treated with botulinum toxin, at intervals of ≥ 12 weeks. We present efficacy, patient-reported outcomes (PROs), and safety in adults with CD at the last available visit after a single set of abobotulinumtoxinA (aboBoNT-A) injections versus placebo using 500 U in a 2-mL injection volume. In this 12-week, randomized, double-blind trial, patients were ≥ 18 years of age with primary idiopathic CD, had a Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) total score ≥ 20, and TWSTRS-Severity subscale score > 10 at baseline. Patients (N = 134) were randomized (2:1) to aboBoNT-A (n = 89) or placebo (n = 45), with aboBoNT-A patients treated with 500 units (U) if toxin-naïve, and 250 to 500 U based on previous onabotulinumtoxinA dose if non-naïve. Endpoints included total TWSTRS, Pain Numeric Rating Scale (NRS-Pain; 24-hour), Treatment Satisfaction Questionnaire for Medication, and other PROs for pain, depression, and global health. Results are for the intent-to-treat population, with "Week 12" (Wk12) comprising the last available post-baseline assessment (end-of-study or early withdrawal). Mean TWSTRS total scores improved from 42.5 at baseline to 35.4 at Wk12 with aboBoNT-A and 42.4 to 40.4 with placebo (treatment difference: -4.8; 95% confidence interval [CI]: -8.5, -1.1; p = 0.011). At Wk12, mean (95% CI) change from baseline in NRS-Pain was -1.0 (-1.59, -0.45) for aboBoNT-A and -0.2 (-0.96, 0.65) for placebo. AboBoNT-A demonstrated numeric improvements in other PROs. More aboBoNT-A-treated patients than patients receiving placebo reported being at least "somewhat satisfied" with treatment (60.4% vs 42.2%, respectively), symptom relief (57.0% vs 40.0%), and time for treatment to work (55.8% vs 33.3%). No new adverse events were reported. Results indicate that in patients with CD, treatment with aboBoNT-A using a 2-mL injection provided sustained improvement in the TWSTRS total score and patient-perceived benefits up to 12 weeks. Trial registration: Clinicaltrials.gov Identified: NCT01753310.

SUBMITTER: Patel AT

PROVIDER: S-EPMC7850472 | biostudies-literature |

REPOSITORIES: biostudies-literature

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Project description:Background:Botulinum neurotoxins type A (BoNT-As) are commonly used treatments for cervical dystonia (CD). Clinical trials have demonstrated the benefits of them in these patients, but data from real-life clinical practice as well as comparative data on the cost and outcome of different BoNT-A formulations are limited. The aim of this study was to compare abobotulinumtoxinA (aboBoNT-A) and onabotulinumtoxinA (onaBoNT-A) on their clinical outcomes and drug costs in real-life clinical practice. Methods:This analysis included 356 adult patients with idiopathic CD treated with aboBoNT-A (n?=?253) or onaBoNT-A (n?=?103) from 38 centres across Europe and Australia (NCT00833196). The clinical outcome measures were treatment responses, changes in TWSTRS scores and changes in health utility scores from baseline to study visit 2 and 3. Health utility score was mapped from the TWSTRS total scale, using a previous publication. Costs included drug cost for France. Results:The aboBoNT-A treated group had 2.06 (95% CI: 1.15 to 3.69) times higher odds of achieving treatment response than the onaBoNT-A treated group. The adjusted mean change in TWSTRS total score from baseline to visit 3 were?-?6.42 (95% CI: -?7.52 to -?5.33) for aboBoNT-A and?-?3.94 (95% CI: -?5.68 to -?2.2) for onaBoNT-A, with a difference of -?2.48 (95% CI: -?4.57 to -?0.39). The corresponding difference in the adjusted mean change for health utility score was 0.008 (95% CI: 0.001 to 0.014). Mean treatment costs for aboBoNT-A and onaBoNT-A were 314.1 (95% CI: 299.1 to 329.0) and 346.6 (95% CI: 322.9 to 370.4) Euros, respectively. Conclusions:This comparative analysis indicated that treatment with aboBoNT-A may be less costly and lead to improved clinical outcomes when compared with onaBoNT-A, from a French healthcare system perspective. Additional comparative clinical data from larger patient cohorts, as well as more information about cost consequences of an improvement in clinical outcome would be of value to further confirm the findings.

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.

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Sustained functional benefits after a single set of injections with abobotulinumtoxinA using a 2-mL injection volume in adults with cervical dystonia: 12-week results from a randomized, double-blind, placebo-controlled phase 3b study.

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