Biomedical Ethics Research Program, Mayo Clinic, Rochester, Minnesota, USA; University of North Carolina School of Medicine, Chapel Hill, North Carolina, USA.
Department of Biological Sciences, College of Science, University of Notre Dame, Notre Dame, Indiana, USA.
Cytotherapy. 2021 Apr;23(4):348-356. doi: 10.1016/j.jcyt.2020.12.011. Epub 2021 Feb 6.
Premature commercialization of unproven stem cell interventions (SCIs) has received significant attention within the regenerative medicine community. Patients considering SCIs may encounter misinformation and seek out guidance from their physicians who are trusted brokers of health information. However, little is known about the perspectives of academic physician specialists toward the SCI industry. The purpose of this study was to capture the attitudes of physician specialists with experience addressing patient questions about unproven SCIs.
The authors undertook 25 semi-structured interviews with academic physicians in cardiology, ophthalmology, orthopedics, pulmonology and neurology primarily from one academic center.
The authors identified two major themes: concerns and mediators of appropriateness of offering SCIs as therapies to patients. Specialists were generally aware of the industry and reported scientific and commercial concerns, including the scientific uncertainty of SCIs, medical harms to patients, misleading marketing and its impact on patient informed consent and economic harms due to large out-of-pocket costs for patients. All specialists outside of orthopedics voiced that it was inappropriate to be offering SCIs to patients today. These views were informed by previously expressed concerns surrounding safety and properly informing patients, levels of evidence needed prior to offering SCIs therapeutically and desired qualifications for clinicians. Among the specialties, orthopedists reported that under certain conditions, SCIs may be appropriate for patients with limited clinical options but only when safety is adequate, expectations are managed and patients are well informed about the risks and chances of benefit. Most participants expressed a desire for phase 3 studies and Food and Drug Administration approval prior to marketing SCIs, but some also shared the challenges associated with upholding these thresholds of evidence, especially when caring for out-of-option patients.
The authors' results suggest that medical specialists are aware of the industry and express several concerns surrounding SCIs but differ in their views on the appropriateness and clinical evidence necessary for offering SCIs currently to patients. Additional educational tools may help physicians with patient engagement and expectation management surrounding SCIs.
未经证实的干细胞干预措施（SCsI）的过早商业化已引起再生医学领域的广泛关注。考虑接受SCsI 的患者可能会遇到错误信息，并寻求他们信任的医生的指导，因为医生是健康信息的可靠经纪人。但是，人们对具有处理患者对未经证实的SCsI 提出的问题经验的学术医师专家的观点知之甚少。本研究旨在了解具有处理患者对未经证实的SCsI 提出的问题经验的学术医师专家的态度。
作者对来自一个学术中心的心脏病学、眼科学、骨科、肺病学和神经病学的学术医师进行了 25 次半结构化访谈。
作者确定了两个主要主题：对将SCsI 作为治疗方法提供给患者的适当性的关注和调节因素。专家们通常了解该行业，并报告了一些科学和商业方面的担忧，包括SCsI 的科学不确定性、对患者的医疗伤害、误导性的营销及其对患者知情同意的影响，以及由于患者需支付高额自付费用而造成的经济伤害。除骨科外的所有专家都表示，目前向患者提供SCsI 是不合适的。这些观点是基于他们对安全和适当告知患者、提供治疗性SCsI 之前需要的证据水平以及临床医生的理想资格的先前表达的担忧。在各个专业中，骨科医生报告说，在某些情况下，SCsI 可能适合临床选择有限的患者，但前提是安全性足够、预期得到管理且患者充分了解风险和获益机会。大多数参与者表示希望在进行市场营销之前开展 3 期研究和食品和药物管理局批准，但有些人也分享了与坚持这些证据门槛相关的挑战，尤其是在照顾没有选择的患者时。
作者的研究结果表明，医学专家了解该行业，并对SCsI 存在一些担忧，但在提供SCsI 给患者目前的适当性和临床证据方面存在分歧。额外的教育工具可能有助于医生在围绕SCsI 的患者参与和期望管理方面。
