Duchenne Parent Project Netherlands, the Netherlands; Department of Human Genetics, Leiden University Medical Center, the Netherlands.
Duchenne UK, UK.
Neuromuscul Disord. 2019 Aug;29(8):576-584. doi: 10.1016/j.nmd.2019.06.004. Epub 2019 Jun 12.
The number of clinical trials for Duchenne muscular dystrophy is increasing. Many trials require muscle biopsies, which involve an invasive surgical procedure. Little is known about short- and long-term impacts of muscle biopsies as perceived by patients and caregivers. Therefore a survey was held among patients and their caregivers who participated in trials involving muscle biopsies, in seven countries. Seventy-eight responses were received. Analysis revealed that many patients and parents had significant anxiety before the biopsy. The main concern of caregivers was the required general anaesthesia. In most cases biopsies caused pain and temporarily hampered daily activities. The main long-term impact was scarring, although large variation in size was reported. Seventy-nine percent of caregivers were little bothered and 21% were moderately or severely bothered by the scar. Willingness to consider another biopsy in future protocols was higher for open-label studies than for placebo-controlled trials. Caregivers stressed the importance of knowing the results of biopsy analyses; only a minority actually received this information. Recommendations are made on the informed consent procedure regarding risks and consequences of muscle biopsies, and communication of results. Furthermore, efforts should be made to minimise the impact of biopsies through pain management and by considering plastic surgery.
杜氏肌营养不良症的临床试验数量正在增加。许多试验需要进行肌肉活检，这涉及到一种有创的外科手术。患者和护理人员对肌肉活检的短期和长期影响知之甚少。因此，在七个国家对参与涉及肌肉活检试验的患者及其护理人员进行了调查。共收到 78 份回复。分析表明，许多患者和家长在活检前有明显的焦虑。护理人员主要关注的是所需的全身麻醉。在大多数情况下，活检会引起疼痛，并暂时妨碍日常活动。主要的长期影响是疤痕，尽管报告的大小差异很大。79%的护理人员对此疤痕几乎没有困扰，21%的护理人员有中度或严重困扰。在未来的方案中，愿意考虑再次进行开放性研究而非安慰剂对照试验的活检意愿更高。护理人员强调了解活检分析结果的重要性；只有少数人实际上收到了这些信息。就肌肉活检的风险和后果的知情同意程序以及结果的沟通提出了建议。此外，应通过疼痛管理和考虑整形手术来努力减轻活检的影响。
