随机、前瞻性、全国性的法国医学经济学胰岛移植研究在严重不稳定 1 型糖尿病患者中的应用：STABILOT 研究方案。
Randomised, prospective, medico-economic nationwide French study of islet transplantation in patients with severely unstable type 1 diabetes: the STABILOT study protocol.
机构信息
Department of Diabetology, Pôle DigiDune, Grenoble University Hospital, Grenoble Alpes University, Grenoble, France.
Grenoble Alpes University, INSERM, U1055Laboratory of Fundamental and Applied Bioenergetics, Grenoble, France.
出版信息
BMJ Open. 2017 Feb 20;7(2):e013434. doi: 10.1136/bmjopen-2016-013434.
INTRODUCTION
Islet transplantation may be an appropriate treatment option for patients with severely unstable type 1 diabetes experiencing major glucose variability with severe hypoglycaemia despite intensive insulin therapy. Few data are available on the costs associated with islet transplantation in relation to its benefits. The STABILOT study proposes to assess the economic impact of islet transplantation in comparison with the current best medical treatment defined as sensor-augmented pump (SAP) therapy.
METHODS
The trial will adopt an open-label, randomised, multicentred design. The study will include 30 patients with severely unstable type 1 diabetes. Eligible participants will be 18-65 years old, with type 1 diabetes duration >5 years, a negative basal or stimulated C-peptide, and severe instability defined by persistent, recurrent and disabling severe hypoglycaemia, despite optimised medical treatment. Participants will be randomised into two groups: one group with immediate registration for islet transplantation, and one group with delayed registration for 1 year while patients receive SAP therapy. The primary endpoint will be the incremental cost-utility ratio at 1 year between islet transplantation and SAP therapy. Perspectives of both the French Health Insurance System and the hospitals will be retained.
ETHICS AND DISSEMINATION
Ethical approval has been obtained at all sites. The trial has been approved by ClinicalTrials.gov (Trial registration ID NCT02854696). All participants will sign a free and informed consent form before randomisation. Results of the study will be communicated during national and international meetings in the field of diabetes and transplantation. A publication will be sought in journals usually read by physicians involved in diabetes care, transplantation and internal medicine.
TRIAL REGISTRATION NUMBER
NCT02854696; Pre-results.
简介
对于接受强化胰岛素治疗后仍存在严重低血糖且血糖波动较大的重度不稳定 1 型糖尿病患者，胰岛移植可能是一种合适的治疗选择。目前关于胰岛移植相关成本与获益的相关数据有限。STABILOT 研究旨在评估胰岛移植相对于当前最佳治疗（定义为传感器增强型胰岛素泵治疗）的经济影响。
方法
该试验采用开放标签、随机、多中心设计。该研究将纳入 30 例重度不稳定 1 型糖尿病患者。合格的参与者年龄在 18-65 岁之间，1 型糖尿病病程>5 年，基础或刺激后 C 肽阴性，且尽管接受了优化的药物治疗，仍存在持续性、复发性和致残性严重低血糖，定义为重度不稳定。参与者将随机分为两组：一组立即登记进行胰岛移植，一组延迟登记 1 年，同时接受 SAP 治疗。主要终点为 1 年内胰岛移植与 SAP 治疗的增量成本-效用比。将保留法国健康保险系统和医院的观点。
伦理与传播
所有站点均获得伦理批准。该试验已在 ClinicalTrials.gov 上获得批准（试验注册 ID：NCT02854696）。所有参与者在随机分组前都将签署一份自由和知情同意书。研究结果将在糖尿病和移植领域的国家和国际会议上进行交流。将在通常被参与糖尿病治疗、移植和内科的医生阅读的期刊上寻求发表。
试验注册号
NCT02854696；预结果。
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