Jankovic Joseph, Adler Charles H, Charles David, Comella Cynthia, Stacy Mark, Schwartz Marc, Manack Adams Aubrey, Brin Mitchell F
Baylor College of Medicine, Houston, TX, USA.
Mayo Clinic Arizona, Scottsdale, AZ, USA.
J Neurol Sci. 2015 Feb 15;349(1-2):84-93. doi: 10.1016/j.jns.2014.12.030. Epub 2014 Dec 27.
The Cervical Dystonia Patient Registry for Observation of OnabotulinumtoxinA Efficacy (CD PROBE; NCT00836017) is a prospective, observational, multicenter, real-world registry designed to assess the safety, effectiveness, and treatment utilization following multiple treatments of onabotulinumtoxinA.
Subjects were naïve to botulinum toxin, new to practice, or had not received toxin in ≥ 16 weeks if in a clinical trial. Dosages and treatment intervals varied due to the real-world design. Descriptive and inferential statistics evaluated changes over 3 treatments.
1046 subjects enrolled. Subjects were 74.4% female, 63.5% toxin-naïve, mean age 58.0 ± 14.7 years. The mean dose over 2481 treatment sessions was 189. 8 ± 87.1U, with average treatment intervals of 14.6 and 15.1 weeks. The mean Toronto Western Spasmodic Torticollis Rating Scale Total score in subjects who completed all assessments (n=479) decreased from 39.2 at baseline to 27.1 at final visit (P<.0001). A high percentage of physicians reported improvement in Clinician Global Impression of Change after initial assessment; this significantly increased at final assessment (n=479, 91.2% vs 95.0%; P<.0001). Similarly, a high percentage of subjects reported improvement in Patient Global Impression of Change after initial assessment, which significantly increased at final assessment (n=470, 83.0% vs 91.7%; P<.0001). Significant reductions in all Cervical Dystonia Impact Profile-58 scores were observed (n=407). Overall, 26.2% of subjects reported adverse events, including muscular weakness (7.0%) and dysphagia (6.4%).
Results indicate robust improvement in clinical ratings and excellent tolerability following onabotulinumtoxinA treatment of CD.
用于观察A型肉毒毒素疗效的颈部肌张力障碍患者登记研究（CD PROBE；NCT00836017）是一项前瞻性、观察性、多中心、真实世界的登记研究，旨在评估多次注射A型肉毒毒素后的安全性、有效性和治疗应用情况。
受试者未曾使用过肉毒毒素、新开始接受治疗，或在临床试验中如果已有≥16周未接受毒素治疗。由于采用真实世界设计，剂量和治疗间隔有所不同。描述性和推断性统计评估了3次治疗后的变化情况。
共纳入1046名受试者。受试者中女性占74.4%，63.5%未曾使用过毒素，平均年龄为58.0±14.7岁。在2481次治疗疗程中，平均剂量为189.8±87.1U，平均治疗间隔为14.6周和15.1周。完成所有评估的受试者（n=479）的多伦多西部痉挛性斜颈评定量表总分从基线时的39.2降至末次随访时的27.1（P<0.0001）。高比例的医生报告在初始评估后临床总体印象改善；在末次评估时这一比例显著增加（n=479，91.2%对95.0%；P<0.0001）。同样，高比例的受试者报告在初始评估后患者总体印象改善，在末次评估时显著增加（n=470，83.0%对91.7%；P<0.0001）。观察到颈部肌张力障碍影响量表-58所有评分均显著降低（n=407）。总体而言，26.2%的受试者报告了不良事件，包括肌肉无力（7.0%）和吞咽困难（6.4%）。
结果表明，A型肉毒毒素治疗颈部肌张力障碍后临床评分有显著改善且耐受性良好。
