Kansas City Bone & Joint Clinic, Overland Park, KS, United States.
Department of Neurology and Neurosurgery, McGill University, Montreal, QC, Canada.
JMIR Public Health Surveill. 2020 Dec 7;6(4):e17928. doi: 10.2196/17928.
Patient- and caregiver-reported data are lacking on the burden of spasticity, and the impact of botulinum neurotoxin type A (BoNT-A) treatment for this condition, on patients' daily lives. As recommended in recent guidance from the US Food and Drug Administration, online patient communities can represent a platform from which to gather specific information outside of a clinical trial setting on the burden of conditions experienced by patients and caregivers and their views on treatment options in order to inform evidence-based medicine and drug development.
The objective of our study is to characterize spasticity symptoms and their associated burdens on Western European and US patients and caregivers in the realms of work, daily activities, quality of life (QoL), as well as the positive and negative impacts of treatment with BoNT-A (cost, time, QoL) using Carenity, an international online community for people with chronic health conditions.
We performed a noninterventional, multinational survey. Eligible participants were 18 years old or older and had, or had cared for, someone with spasticity who had been treated with BoNT-A for at least 1 year. Patients and caregivers were asked to complete an internet-based survey via Carenity; caregivers reported their own answers and answered on behalf of their patients. Questions included the burden of spasticity on the ability to work, functioning, daily-living activities, and QoL, the impact of BoNT A therapy on patients' lives, and the potential benefits of fewer injections.
There were 615 respondents (427 patients and 188 caregivers). The mean age of patients and caregivers was 41.7 years and 38.6 years, respectively, and the most commonly reported cause of spasticity was multiple sclerosis. Caregivers were most often the parents (76/188, 40%) or another family member (51/188, 27%) of their patients. Spasticity had a clear impact on patients' and caregivers' lives, including the ability to work and injection costs. For patients, spasticity caused difficulties with activities of daily living and reduced QoL indices. The median number of BoNT-A injections was 4 times per year, and 92% (393/427) of patients reported that treatment improved their overall satisfaction with life. Regarding the BoNT-A injection burden, the greatest patient-reported challenges were the cost and availability of timely appointments. Overall, 86% (368/427) of patients believed that a reduced injection frequency would be beneficial. Caregivers answering for their patients gave largely similar responses to those reported by patients.
Spasticity has a negative impact on both patients' and caregivers' lives. All respondents reported that BoNT A treatment improved their lives, despite the associated challenges. Patients believed that reducing the frequency of BoNT-A injections could alleviate practical issues associated with treatment, implying that a longer-acting BoNT-A injection would be well received.
缺乏患者和照护者报告的关于痉挛负担的信息，以及肉毒毒素 A 型（BoNT-A）治疗对患者日常生活的影响。根据美国食品和药物管理局最近的指南建议，在线患者社区可以作为一种平台，在临床试验环境之外收集患者和照护者所经历的疾病的具体信息，以及他们对治疗方案的看法，以便为循证医学和药物开发提供信息。
本研究的目的是使用国际在线社区 Carenity，描述西欧和美国患者和照护者的痉挛症状及其相关负担，包括在工作、日常活动、生活质量（QoL）方面的负担，以及 BoNT-A（成本、时间、QoL）治疗的积极和消极影响。
我们进行了一项非干预性、多国调查。符合条件的参与者年龄在 18 岁或以上，并且有或照顾过接受 BoNT-A 治疗至少 1 年的痉挛患者。患者和照护者通过 Carenity 完成在线调查；照护者报告自己的答案，并代表患者回答。问题包括痉挛对工作能力、功能、日常生活活动和生活质量的负担、BoNT-A 治疗对患者生活的影响以及减少注射次数的潜在益处。
共有 615 名受访者（427 名患者和 188 名照护者）。患者和照护者的平均年龄分别为 41.7 岁和 38.6 岁，痉挛最常见的病因是多发性硬化症。照护者最常是患者的父母（76/188，40%）或其他家庭成员（51/188，27%）。痉挛对患者和照护者的生活有明显影响，包括工作能力和注射费用。对于患者，痉挛导致日常生活活动困难和降低生活质量指数。BoNT-A 注射的中位数次数为每年 4 次，92%（393/427）的患者报告治疗改善了他们对生活的总体满意度。关于 BoNT-A 注射负担，患者报告的最大挑战是成本和及时预约的可用性。总体而言，86%（368/427）的患者认为减少注射频率将是有益的。代表患者回答的照护者的回答与患者报告的回答基本相似。
痉挛对患者和照护者的生活都有负面影响。所有受访者都报告说 BoNT-A 治疗改善了他们的生活，尽管存在相关挑战。患者认为减少 BoNT-A 注射的频率可以缓解治疗相关的实际问题，这意味着一种作用时间更长的 BoNT-A 注射将受到欢迎。
