Stanford Center for Biomedical Ethics, Department of Medicine, Stanford University, Stanford, CA, USA.
Department of Bioethics, National Institutes of Health Clinical Center, Bethesda, MD, USA.
Genet Med. 2020 Nov;22(11):1830-1837. doi: 10.1038/s41436-020-0890-6. Epub 2020 Jun 30.
Advances in gene therapy and precision medicine have led to a growing number of novel treatments for rare genetic diseases. Patients/families may lack access to up-to-date, accurate, and relevant information about these treatments. Social media offers one potentially important resource for these communities. Our goal was to understand how patients/families with spinal muscular atrophy (SMA)-a rare genetic condition-used social media to share, consume, and evaluate information about the novel treatment nusinersen (Spinraza) following the drug's approval.
We conducted qualitative, semistructured interviews with 20 SMA patients or parents of patients, deriving themes and subthemes through content and thematic network analysis. Participants also completed a demographic survey.
Participants described leveraging social media to learn about nusinersen treatment, make informed treatment decisions, and advocate for/access treatment. They also described critically evaluating the trustworthiness of nusinersen-related information on social media and the privacy risks of social media use.
Patients/families used social media to navigate the new and dynamic landscape of nusinersen treatment for SMA, while attempting to mitigate misinformation and privacy risks. As new treatments become available, providers and patients/families may benefit from proactively discussing social media use, so as to maximize important benefits while minimizing risks.
基因治疗和精准医学的进步使得治疗罕见遗传病的新疗法越来越多。患者/家属可能无法获得有关这些疗法的最新、准确且相关的信息。社交媒体为这些群体提供了一个潜在的重要资源。我们的目标是了解脊髓性肌萎缩症（SMA）患者/家属在新型药物nusinersen（Spinraza）获得批准后，如何利用社交媒体分享、获取和评估有关该药物的信息。
我们对 20 名 SMA 患者或患者家长进行了定性、半结构化访谈，通过内容分析和主题网络分析得出主题和子主题。参与者还完成了一份人口统计调查。
参与者描述了利用社交媒体了解 nusinersen 治疗、做出明智的治疗决策以及倡导/获取治疗的过程。他们还描述了对社交媒体上有关 nusinersen 信息的可信度以及社交媒体使用的隐私风险进行批判性评估。
患者/家属利用社交媒体来应对 SMA 患者 nusinersen 治疗的新动态，同时努力减少错误信息和隐私风险。随着新疗法的出现，提供者和患者/家属可能会受益于积极讨论社交媒体的使用，以便在最大限度地利用其益处的同时最小化风险。
