Karpen Stephen R, Mellor Richard, DiMeglio Linda A, Senior Peter, Sherr Jennifer L, Bussberg Cooper, Mansfield Carol, Marinac Marjana, Myers Kelley
Breakthrough T1D, Washington, District of Columbia, USA.
RTI Health Solutions, Durham, NC, USA.
Diabetes Ther. 2025 Sep 4. doi: 10.1007/s13300-025-01783-y.
Novel therapies, including disease-modifying and cell replacement therapies, may preserve or replace beta cells in people with type 1 diabetes. This study sought to understand how people living with type 1 diabetes or caring for someone with type 1 diabetes perceive the benefits and risks of novel therapies.
Semistructured qualitative interviews were conducted with 26 participants in the United States: four adolescents and 12 adults with type 1 diabetes, and 10 caregivers of children with type 1 diabetes. A description of the benefits and risks of disease-modifying and cell replacement therapies, developed with a steering committee of patients and clinicians, was presented during interviews to facilitate discussion among people living with type 1 diabetes and caregivers. A qualitative directed content analysis was conducted.
Participants reported that type 1 diabetes and insulin therapy regimens impacted many life areas, with some participants reporting diabetes burnout. Most participants expressed that they would have considered trying disease-modifying therapies, most frequently citing perceived benefits such as reduced insulin reliance and an extended post-diagnosis "honeymoon period" providing time to prepare for life with diabetes. Cancer risk was the most frequently reported risk of concern for disease-modifying therapies. All participants expressed willingness to consider cell replacement therapies, with insulin independence and restored pancreatic function perceived to offer greater normalcy and freedom from the constant demands of diabetes. Participants reported concerns about the use of immunosuppressants and the risks and drawbacks of the cell replacement surgical procedure.
Despite concerns about the risks and drawbacks of novel therapies, most participants reported that they would consider trying disease-modifying and cell replacement therapies. There is no substitute for consulting people living with or caring for someone with type 1 diabetes when considering new therapies with novel risks and benefits.
包括疾病改善疗法和细胞替代疗法在内的新型疗法，可能会保留或替代1型糖尿病患者的β细胞。本研究旨在了解1型糖尿病患者或照顾1型糖尿病患者的人如何看待新型疗法的益处和风险。
对美国的26名参与者进行了半结构化定性访谈：4名青少年和12名成年1型糖尿病患者，以及10名1型糖尿病儿童的照顾者。在访谈过程中，展示了由患者和临床医生指导委员会制定的疾病改善疗法和细胞替代疗法的益处和风险描述，以促进1型糖尿病患者和照顾者之间的讨论。进行了定性定向内容分析。
参与者报告称，1型糖尿病和胰岛素治疗方案影响了许多生活领域，一些参与者报告有糖尿病倦怠感。大多数参与者表示，他们会考虑尝试疾病改善疗法，最常提到的益处包括减少对胰岛素的依赖，以及诊断后的“蜜月期”延长，为应对糖尿病生活提供准备时间。癌症风险是疾病改善疗法最常被提及的风险。所有参与者都表示愿意考虑细胞替代疗法，认为胰岛素独立和胰腺功能恢复能带来更大的正常生活状态，并摆脱糖尿病的持续困扰。参与者报告了对使用免疫抑制剂以及细胞替代手术风险和缺点的担忧。
尽管对新型疗法的风险和缺点存在担忧，但大多数参与者表示他们会考虑尝试疾病改善疗法和细胞替代疗法。在考虑具有新风险和益处的新疗法时，咨询1型糖尿病患者或其照顾者是无可替代的。
