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Evolving Concepts in Pathophysiology, Screening, and Prevention of Type 1 Diabetes: Report of Diabetes Mellitus Interagency Coordinating Committee Workshop
Diabetes ( IF 6.2 ) Pub Date : 2024-08-22 , DOI: 10.2337/dbi24-0020 Carla J Greenbaum 1 , Gerald T Nepom 2 , Lauren K Wood-Heickman 3 , Diane K Wherrett 4 , Linda A DiMeglio 5 , Kevan C Herold 6 , Jeffrey P Krischer 7
Diabetes ( IF 6.2 ) Pub Date : 2024-08-22 , DOI: 10.2337/dbi24-0020 Carla J Greenbaum 1 , Gerald T Nepom 2 , Lauren K Wood-Heickman 3 , Diane K Wherrett 4 , Linda A DiMeglio 5 , Kevan C Herold 6 , Jeffrey P Krischer 7
Affiliation
The approval of teplizumab to delay the onset of type 1 diabetes is an important inflection point in the decades long pursuit to treat the cause of the disease rather than its symptoms. NIDDK convened a workshop of the Diabetes Mellitus Interagency Coordinating Committee titled “Evolving Concepts in Pathophysiology, Screening, and Prevention of Type 1 Diabetes” to review this accomplishment and identify future goals. Speakers representing Diabetes TrialNet (TN) and the Immune tolerance Network (ITN) emphasized that the ability to robustly identify individuals destined to develop type 1 diabetes was essential for clinical trials. The presenter from the FDA described that regulatory approval relied upon data from TN's single clinical trial testing teplizumab for delay of clinical diagnosis, along with confirmatory evidence from studies in patients after diagnosis. The workshop reviewed the etiology of type 1 diabetes as a disease involving multiple immune pathways, highlighting the current understanding of prognostic markers, and proposing potential strategies to improve the therapeutic response of disease modifying therapies based on their mechanism of action. While celebrating these achievements funded by the congressionally appropriated Special Diabetes Program, panelists from professional organizations, non-profit advocacy/funding groups, and industry also identified significant hurdles in translating this research into clinical care.
中文翻译:
1 型糖尿病病理生理学、筛查和预防的不断发展的概念:糖尿病机构间协调委员会研讨会报告
teplizumab 被批准用于延缓 1 型糖尿病的发作,这是几十年来追求治疗疾病原因而不是症状的一个重要转折点。NIDDK 召集了糖尿病机构间协调委员会的一次研讨会,题为“1 型糖尿病病理生理学、筛查和预防的不断发展的概念”,以审查这一成就并确定未来目标。代表糖尿病试验网 (TN) 和免疫耐受网络 (ITN) 的发言人强调,强有力地识别注定会患上 1 型糖尿病的个体的能力对于临床试验至关重要。FDA 的介绍者描述说,监管批准依赖于 TN 测试 teplizumab 延迟临床诊断的单一临床试验的数据,以及诊断后对患者进行研究的确认证据。该研讨会回顾了 1 型糖尿病作为一种涉及多种免疫途径的疾病的病因,强调了当前对预后标志物的理解,并提出了根据其作用机制改善疾病修饰疗法治疗反应的潜在策略。在庆祝由国会拨款的特殊糖尿病计划资助的这些成就的同时,来自专业组织、非营利倡导/资助团体和行业的小组成员也发现了将这项研究转化为临床护理的重大障碍。
更新日期:2024-08-22
中文翻译:
1 型糖尿病病理生理学、筛查和预防的不断发展的概念:糖尿病机构间协调委员会研讨会报告
teplizumab 被批准用于延缓 1 型糖尿病的发作,这是几十年来追求治疗疾病原因而不是症状的一个重要转折点。NIDDK 召集了糖尿病机构间协调委员会的一次研讨会,题为“1 型糖尿病病理生理学、筛查和预防的不断发展的概念”,以审查这一成就并确定未来目标。代表糖尿病试验网 (TN) 和免疫耐受网络 (ITN) 的发言人强调,强有力地识别注定会患上 1 型糖尿病的个体的能力对于临床试验至关重要。FDA 的介绍者描述说,监管批准依赖于 TN 测试 teplizumab 延迟临床诊断的单一临床试验的数据,以及诊断后对患者进行研究的确认证据。该研讨会回顾了 1 型糖尿病作为一种涉及多种免疫途径的疾病的病因,强调了当前对预后标志物的理解,并提出了根据其作用机制改善疾病修饰疗法治疗反应的潜在策略。在庆祝由国会拨款的特殊糖尿病计划资助的这些成就的同时,来自专业组织、非营利倡导/资助团体和行业的小组成员也发现了将这项研究转化为临床护理的重大障碍。