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Safety and Efficacy of RimabotulinumtoxinB for Treatment of Sialorrhea in Adults: A Randomized Clinical Trial.
JAMA Neurology ( IF 20.4 ) Pub Date : 2020-04-01 , DOI: 10.1001/jamaneurol.2019.4565 Stuart H Isaacson 1 , William Ondo 2, 3 , Carlayne E Jackson 4 , Richard M Trosch 5 , Eric Molho 6 , Fernando Pagan 7 , Mark Lew 8 , Khashayar Dashtipour 9 , Thomas Clinch 10 , Alberto J Espay 11 ,
JAMA Neurology ( IF 20.4 ) Pub Date : 2020-04-01 , DOI: 10.1001/jamaneurol.2019.4565 Stuart H Isaacson 1 , William Ondo 2, 3 , Carlayne E Jackson 4 , Richard M Trosch 5 , Eric Molho 6 , Fernando Pagan 7 , Mark Lew 8 , Khashayar Dashtipour 9 , Thomas Clinch 10 , Alberto J Espay 11 ,
Affiliation
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Importance
RimabotulinumtoxinB (RIMA) may be preferable as an anti-sialorrhea treatment compared with current oral anticholinergic drugs in people with neurological disorders.
Objective
To assess the safety, efficacy, and tolerability of RIMA injections for the treatment of sialorrhea in adults.
Design, Setting, and Participants
This randomized, parallel, double-blind, placebo-controlled clinical trial of RIMA 2500 U and 3500 U was conducted from November 14, 2013, to January 23, 2017. A total of 249 adult patients with troublesome sialorrhea secondary to any disorder or cause were screened. Of them, 13 refused further participation in the study or were lost to follow-up and 49 did not fulfill the criteria for participation; 187 were ultimately enrolled. Patients had to have a minimum unstimulated salivary flow rate (USFR) of 0.2 g/min and a minimum Drooling Frequency and Severity Scale score of 4.
Exposures
Patients were randomized 1:1:1 to RIMA, 2500 U (n = 63); RIMA, 3500 U (n = 64); or placebo (n = 60).
Main Outcomes and Measures
Primary outcomes were the change in USFR from baseline to week 4 and the Clinical Global Impression of Change (CGI-C) at week 4. The CGI-C scores were recorded on a 7-point scale ranging from very much improved to very much worse. Adverse events were recorded throughout the trial period.
Results
Of 187 patients enrolled (147 men [78.6%]; mean [SD] age, 63.9 [13.3] years), 122 patients had Parkinson disease (65.2%), 13 (7.0%) were stroke survivors, 12 had amyotrophic lateral sclerosis (6.4%), 6 had medication-induced sialorrhea (3.2%), 4 had adult cerebral palsy (2.1%), and 30 had sialorrhea owing to other causes (16.0%). A total of 176 completed the study. Treatment with both doses of RIMA significantly reduced USFR at week 4 vs placebo (mean treatment difference, -0.30 g/min [95% CI, -0.39 to -0.21] for both doses vs placebo, P < .001). The CGI-C scores were statistically significantly improved at week 4 for both treatment groups vs placebo (-1.21 [95% CI, -1.56 to -0.87] for 2500 U, -1.14 [95% CI, -1.49 to -0.80] for 3500 U, both P < .001). Treatment benefits were seen as early as 1 week after injection and were maintained over the treatment cycle of approximately 13 weeks. The RIMA injections were well tolerated compared with placebo. The most common adverse events were self-limited mild to moderate dry mouth, dysphagia, and dental caries.
Conclusions and Relevance
Treatment with RIMA (2500 U and 3500 U) in adults was well tolerated and reduced sialorrhea, with the onset of the effect at 1 week after the injection. These data support the clinical use of RIMA in the management of sialorrhea in adults.
Trial Registration
ClinicalTrials.gov Identifier: NCT01994109.
中文翻译：
RimabotulinumtoxinB在成人唾液中的安全性和有效性：一项随机临床试验。
与目前在神经系统疾病患者中口服抗胆碱能药物相比，重要的是利马肉毒杆菌毒素B（RIMA）作为抗流涎治疗更可取。目的评估RIMA注射液治疗成人腹泻的安全性，疗效和耐受性。设计，背景和参与者该RIMA 2500 U和3500 U的随机，平行，双盲，安慰剂对照临床试验于2013年11月14日至2017年1月23日进行。共有249名患有麻烦性腹泻的成人患者筛选出因任何疾病或原因引起的继发性疾病。其中13名拒绝进一步参加研究或失去随访，还有49名不符合参加标准；最终有187人被录取。患者的最小非刺激唾液流量（USFR）必须为0。暴露速度为2 g / min，最低流口水频率和严重度等级评分为4。暴露患者以RIMA，2500 U（n = 63）的比例1：1：1随机分组。RIMA，3500 U（n = 64）；或安慰剂（n = 60）。主要结果和指标主要结果是从基线到第4周的USFR变化，以及第4周的临床总体变化印象（CGI-C）。CGI-C评分以7分制记录，范围从非常改善更糟糕的是 在整个试验期间记录不良事件。结果入组的187例患者（147例男性[78.6％]；平均[SD]年龄为63.9 [13.3]岁），122例帕金森病（65.2％），13例（7.0％）为中风幸存者，12例为肌萎缩性侧索硬化（6.4％），6例因药物引起的腹泻（3.2％），4例患有成人脑瘫（2.1％），30例因其他原因而引起的腹泻（16.0％）。共有176人完成了研究。与安慰剂相比，两种剂量的RIMA治疗在第4周时均显着降低USFR（两种剂量相比，安慰剂的平均治疗差异为-0.30 g / min [95％CI，-0.39至-0.21]，P <.001）。与安慰剂相比，两个治疗组的CGI-C评分在第4周都有显着改善（2500 U的-1.21 [95％CI，-1.56至-0.87]，-1.14 [95％CI，-1.49至-0.80]。 3500 U，均P <.001）。早在注射后1周即可看到治疗益处，并在大约13周的治疗周期内得以维持。与安慰剂相比，RIMA注射剂耐受性好。最常见的不良事件是自限性轻度至中度口干，吞咽困难和龋齿。结论成人使用RIMA（2500 U和3500 U）的治疗和耐受性良好，并减少了腹泻，从注射后1周开始起效。这些数据支持RIMA在成人唾液管理中的临床应用。试用注册ClinicalTrials.gov标识符：NCT01994109。
更新日期：2020-04-01
中文翻译：
RimabotulinumtoxinB在成人唾液中的安全性和有效性：一项随机临床试验。
与目前在神经系统疾病患者中口服抗胆碱能药物相比，重要的是利马肉毒杆菌毒素B（RIMA）作为抗流涎治疗更可取。目的评估RIMA注射液治疗成人腹泻的安全性，疗效和耐受性。设计，背景和参与者该RIMA 2500 U和3500 U的随机，平行，双盲，安慰剂对照临床试验于2013年11月14日至2017年1月23日进行。共有249名患有麻烦性腹泻的成人患者筛选出因任何疾病或原因引起的继发性疾病。其中13名拒绝进一步参加研究或失去随访，还有49名不符合参加标准；最终有187人被录取。患者的最小非刺激唾液流量（USFR）必须为0。暴露速度为2 g / min，最低流口水频率和严重度等级评分为4。暴露患者以RIMA，2500 U（n = 63）的比例1：1：1随机分组。RIMA，3500 U（n = 64）；或安慰剂（n = 60）。主要结果和指标主要结果是从基线到第4周的USFR变化，以及第4周的临床总体变化印象（CGI-C）。CGI-C评分以7分制记录，范围从非常改善更糟糕的是 在整个试验期间记录不良事件。结果入组的187例患者（147例男性[78.6％]；平均[SD]年龄为63.9 [13.3]岁），122例帕金森病（65.2％），13例（7.0％）为中风幸存者，12例为肌萎缩性侧索硬化（6.4％），6例因药物引起的腹泻（3.2％），4例患有成人脑瘫（2.1％），30例因其他原因而引起的腹泻（16.0％）。共有176人完成了研究。与安慰剂相比，两种剂量的RIMA治疗在第4周时均显着降低USFR（两种剂量相比，安慰剂的平均治疗差异为-0.30 g / min [95％CI，-0.39至-0.21]，P <.001）。与安慰剂相比，两个治疗组的CGI-C评分在第4周都有显着改善（2500 U的-1.21 [95％CI，-1.56至-0.87]，-1.14 [95％CI，-1.49至-0.80]。 3500 U，均P <.001）。早在注射后1周即可看到治疗益处，并在大约13周的治疗周期内得以维持。与安慰剂相比，RIMA注射剂耐受性好。最常见的不良事件是自限性轻度至中度口干，吞咽困难和龋齿。结论成人使用RIMA（2500 U和3500 U）的治疗和耐受性良好，并减少了腹泻，从注射后1周开始起效。这些数据支持RIMA在成人唾液管理中的临床应用。试用注册ClinicalTrials.gov标识符：NCT01994109。
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