影响颈部肌张力障碍患者健康相关生活质量的因素及阿扑肉毒毒素A（Dysport）治疗的影响：一项随机、双盲、安慰剂对照研究的结果
Factors affecting the health-related quality of life of patients with cervical dystonia and impact of treatment with abobotulinumtoxinA (Dysport): results from a randomised, double-blind, placebo-controlled study.
作者信息
Mordin Margaret, Masaquel Catherine, Abbott Chandra, Copley-Merriman Catherine
机构信息
Market Access and Outcomes Strategy, RTI Health Solutions, Ann Arbor, Michigan, USA.
Neurology Medical Affairs, Ipsen Biopharmaceuticals Inc, Basking Ridge, New Jersey, USA.
出版信息
BMJ Open. 2014 Oct 16;4(10):e005150. doi: 10.1136/bmjopen-2014-005150.
OBJECTIVE
To describe the health-related quality of life (HRQOL) burden of cervical dystonia (CD) and report on the HRQOL and patient perception of treatment benefits of abobotulinumtoxinA (Dysport).
DESIGN
The safety and efficacy of a single injection of abobotulinumtoxinA for CD treatment were evaluated in a previously reported international, multicenter, double-blind, randomised trial. HRQOL measures were assessed in the trial and have not been previously reported.
SETTING
Movement disorder clinics in the USA and Russia.
PARTICIPANTS
Patients had to have a diagnosis of CD with symptoms for at least 18 months, as well as a total Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) score of at least 30; a Severity domain score of at least 15; and a Disability domain score of at least 3. Key exclusion criteria included treatment with botulinum toxin type A (BoNT-A) or botulinum toxin type B (BoNT-B) within 16 weeks of enrolment.
INTERVENTIONS
Patients were randomised to receive either 500 U abobotulinumtoxinA (n=55) or placebo (n=61).
PRIMARY AND SECONDARY OUTCOME MEASURES
Efficacy assessments included TWSTRS total (primary end point) and subscale scores at weeks 0, 4, 8, 12; a pain visual analogue scale at weeks 0 and 4; and HRQOL assessed by the SF-36 Health Survey (SF-36; secondary end point) at weeks 0 and 8.
RESULTS
Patients with CD reported significantly greater impairment for all SF-36 domains relative to US norms. Patients treated with abobotulinumtoxinA reported significantly greater improvements in Physical Functioning, Role Physical, Bodily Pain, General Health and Role Emotional domains than placebo patients (p≤0.03 for all). The TWSTRS was significantly correlated with Physical Functioning, Role Physical and Bodily Pain scores, for those on active treatment.
CONCLUSIONS
CD has a marked impact on HRQOL. Treatment with a single abobotulinumtoxinA injection results in significant improvement in patients' HRQOL.
TRIAL REGISTRATION NUMBER
The trial is registered at ClinicalTrials.gov, numbers NCT00257660 and NCT00288509.
目的
描述颈部肌张力障碍（CD）与健康相关的生活质量（HRQOL）负担，并报告HRQOL以及患者对阿泊肉毒毒素A（Dysport）治疗益处的认知情况。
设计
在一项先前报道的国际多中心双盲随机试验中，评估单次注射阿泊肉毒毒素A治疗CD的安全性和有效性。在该试验中对HRQOL指标进行了评估，此前未作报道。
地点
美国和俄罗斯的运动障碍诊所。
参与者
患者必须确诊为CD且症状持续至少18个月，同时多伦多西部痉挛性斜颈评定量表（TWSTRS）总分至少为30分；严重程度领域得分至少为15分；残疾领域得分至少为3分。主要排除标准包括入组前16周内接受过A型肉毒毒素（BoNT - A）或B型肉毒毒素（BoNT - B）治疗。
干预措施
患者被随机分为接受500 U阿泊肉毒毒素A治疗组（n = 55）或安慰剂组（n = 61）。
主要和次要结局指标
疗效评估包括第0、4、8、12周时的TWSTRS总分（主要终点）及各子量表得分；第0周和第4周时的疼痛视觉模拟量表得分；以及第0周和第8周时通过SF - 36健康调查（SF - 36；次要终点）评估的HRQOL。
结果
与美国常模相比，CD患者在所有SF - 36领域的损伤均显著更严重。与安慰剂组患者相比，接受阿泊肉毒毒素A治疗的患者在身体功能、身体角色、身体疼痛、总体健康和情感角色领域的改善显著更大（所有p值均≤0.03）。对于接受积极治疗的患者，TWSTRS与身体功能、身体角色和身体疼痛得分显著相关。
结论
CD对HRQOL有显著影响。单次注射阿泊肉毒毒素A治疗可使患者的HRQOL得到显著改善。
试验注册号
该试验已在ClinicalTrials.gov注册，注册号为NCT0……
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