Mordin Margaret, Masaquel Catherine, Abbott Chandra, Copley-Merriman Catherine
Market Access and Outcomes Strategy, RTI Health Solutions, Ann Arbor, Michigan, USA.
Neurology Medical Affairs, Ipsen Biopharmaceuticals Inc, Basking Ridge, New Jersey, USA.
BMJ Open. 2014 Oct 16;4(10):e005150. doi: 10.1136/bmjopen-2014-005150.
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).
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.
Movement disorder clinics in the USA and Russia.
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.
Patients were randomised to receive either 500 U abobotulinumtoxinA (n=55) or placebo (n=61).
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.
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.
CD has a marked impact on HRQOL. Treatment with a single abobotulinumtoxinA injection results in significant improvement in patients' HRQOL.
The trial is registered at ClinicalTrials.gov, numbers NCT00257660 and NCT00288509.
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。
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。
美国和俄罗斯的运动障碍诊所。
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。
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）。
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。
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。
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。
该试验已在ClinicalTrials.gov注册，注册号为NCT0……
