XCiDaBLE: A Phase 4, Observational, Prospective Trial Evaluating Xeomin (IncobotulinumtoxinA) for Cervical Dystonia or Blepharospasm in the United States - Preliminary Baseline Disease Severity and Quality of Life Data (P01.240)

Abstract

Objective: Report initial baseline data from XCiDaBLE: a prospective, observational study of incobotulinumtoxinA for blepharospasm (BEB) and cervical dystonia (CD). Background There is a lack of prospectively acquired data on clinicians9 “real world9 experience using botulinum toxins to treat BEB or CD, especially with newer agents. Design/Methods: XCiDaBLE is an open-label, prospective, observational study of subjects with CD or BEB. Subjects are followed for 2 treatments and monitored via IVRS/Web Response. Investigators rate the Clinical Global Impression (CGI)-Severity at baseline. Subject-reported scales include the Cervical Dystonia Impact Profile (CDIP-58) [CD], Jankovic Rating Scale (JRS) [BEB], Signs and Symptoms Scale and Subject Global Impression-Severity and Improvement; Work Productivity and Quality of Life (QoL) were assessed by means of the SF-12v2, an employment questionnaire and work history. Results: To date, 130 CD/184 BEB subjects were enrolled (76% female). The mean enrollment age was 62 years (92.0% Caucasian). The mean estimated duration of disease was 12.2 yrs and 91.7% were previously treated with botulinum toxin. For CD CGI-Severity at baseline was: 20.2% normal-to-mild, 48.1% moderate, 20.9% marked, 9.3% severe and 1.6% extreme. For CD subjects, the mean baseline CDIP score was 159 and subscale scores were: 47.7-Symptom Scale Total; 45.9-Daily Activities Total; and 66.0-Psychosocial Functioning. The SF-12v2 mean scores for CD were 44.0-Mental QoL and 38.1-Physical QoL. The CGI-Severity at baseline for BEB subjects was 32.8% normal-to-mild, 35.5% moderate, 22.4% marked and 9.3% severe. For BEB subjects, the mean baseline JRS score was 5.0. The SF-12v2 mean scores for BEB were 49.1-Mental QoL and 43.1-Physical QoL. Conclusions: CD and BEB can have a significant adverse impact on QoL. This observational study promises to provide valuable information about disease severity and QoL for patients with the often disabling conditions of CD and BEB treated with botulinum toxins. Supported by: Merz Pharmaceuticals. Disclosure: Dr. LeDoux has received personal compensation for activities with Boehringer-Ingelheim and Teva Pharmaceuticals as a speaker.Dr. LeDoux has received research support from MERZ, Xenoport, Boehringer-Ingelheim, and HP Therapeutics Foundation. Dr. Lin has received personal compensation for activities with Merz Pharmaceuticals as a consultant. Dr. Jankovic has received personal compensation for activities with Allergan, Inc., Chelsea Therapeutics, Serono Inc., Merz Pharma, Lundbeck Research USA, Inc, Teva Neuroscience as a consultant. Dr. Jankovic has received personal compensation in an editorial capacity for Medlink: Neurology in Clinical Practice. Dr. Jankovic has received research support from Allergan, Inc, Allon Therapeutics, Ceregene, Inc., Chelsea Therapeutics; Diana Helis Henry Medical Research Foundation, Serono Inc., Huntington9s Disease Society of America, Huntington Study Group, Impax Pharmaceuticals, Ipsen Limited, Lundbeck Research USA, Inc. Dr. Pappert has received personal compensation for activities with Merz Pharmaceuticals as an employee.Dr. Pappert has received research support from Adamas and Teva. Mr. Verma has received personal compensation for activities with Merz Pharma. Dr. Sethi has received personal compensation for activities with Abbott, Synosia, Veloxis, Merz, Teva, and Ipsen. Dr. Sethi has received research support from GlaxoSmithKline, Teva, Acadia, Allergan, and Impax. Dr. Fernandez has received personal compensation for activities with USF CME, Cleveland Clinic CME, Medical Communications Media, Health Professions Conferencing, Ipsen, Merz Pharmaceuticals, and US World Meds Dr. Fernandez has received personal compensation in an editorial capacity for the Movement Disorders Society Website. Dr. Fernandez has received royalty payments from Demos Publishing, Manson Publishing, and Springer Publishing. Dr. Fernandez has received research support from Abbott, Acadia, Biotie Therapeutics, EMD-Serono, Huntington Study Group, Ipsen, Merz Pharmaceuticals, Michael J. Fox Foundation, Movement Disorders Society, National Parkinson Foundation, NIH/NINDS, Novartis, Parkinson Study Group, Teva, but has no owner interest in any pharmaceutical company.