Standardizing Clinically Meaningful Outcome Measures Beyond HbA1c for Type 1 Diabetes: A Consensus Report of the American Association of Clinical Endocrinologists, the American Association of Diabetes Educators, the American Diabetes Association, the Endocrine Society, JDRF International, The Leona M. and Harry B. Helmsley Charitable Trust, the Pediatric Endocrine Society, and the T1D Exchange
Standardizing Clinically Meaningful Outcome Measures Beyond HbA1c for Type 1 Diabetes: A Consensus Report of the American Association of Clinical Endocrinologists, the American Association of Diabetes Educators, the American Diabetes Association, the Endocrine Society, JDRF International, The Leona M. and Harry B. Helmsley Charitable Trust, the Pediatric Endocrine Society, and the T1D Exchange
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DOI：
10.2337/dc17-1624
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发表时间：
2017-12-01
期刊：
影响因子：
16.2
通讯作者：
Weinzimer, Stuart A.
中科院分区：
文献类型：
--
作者：
Agiostratidou, Gina;Anhalt, Henry;Weinzimer, Stuart A.
目的根据对证据的回顾、临床专家的共识以及来自研究人员、1型糖尿病患者和产业界的意见，确定和定义超出血红蛋白A(1c)(HbA(1c))的1型糖尿病的临床意义结果。优先结果包括低血糖、高血糖、住院时间、糖尿病酮症酸中毒(DKA)和患者报告的结果(PRO)。虽然1型糖尿病和2型糖尿病的优先结果可能会重叠，但1型糖尿病是这项工作的重点。研究和方法指导委员会--由来自美国临床内分泌学家协会、美国糖尿病教育工作者协会、美国糖尿病协会、内分泌学会、JDRF国际基金会、Leona M.和Harry B.Helmsley慈善信托基金、儿科内分泌学会和T1D交易所的代表组成--是1型糖尿病结果计划的决策机构。他们的工作由研究人员、行业和糖尿病患者通过代表每个利益相关者群体的咨询委员会提供的意见提供信息。利益攸关方调查被用来确定优先结果。在调查中优先考虑的结果是低血糖、高血糖、在范围内的时间、DKA和PROS。为了就这些结果的定义达成共识，指导委员会依靠已发表的证据、他们的临床专业知识和咨询委员会的反馈。结果指导委员会制定了1型糖尿病患者的低血糖、高血糖、范围内时间和DKA的定义。这些定义反映了他们对结果对1型糖尿病患者的短期和长期临床影响的评估。确定了未来研究需要解决的知识差距。指导委员会讨论了优点，并得出结论，需要进一步针对1型糖尿病进行特定的开发。结论指导委员会建议在1型糖尿病治疗的研究、开发和评估中使用HBA(1c)以外的明确的临床有意义的结果。
OBJECTIVETo identify and define clinically meaningful type 1 diabetes outcomes beyond hemoglobin A(1c) (HbA(1c)) based upon a review of the evidence, consensus from clinical experts, and input from researchers, people with type 1 diabetes, and industry. Priority outcomes include hypoglycemia, hyperglycemia, time in range, diabetic ketoacidosis (DKA), and patient-reported outcomes (PROs). While priority outcomes for type 1 and type 2 diabetes may overlap, type 1 diabetes was the focus of this work.RESEARCH AND METHODSSteering Committee-comprising representatives from the American Association of Clinical Endocrinologists, the American Association of Diabetes Educators, the American Diabetes Association, the Endocrine Society, JDRF International, The Leona M. and Harry B. Helmsley Charitable Trust, the Pediatric Endocrine Society, and the T1D Exchangewas the decision-making body for the Type 1 Diabetes Outcomes Program. Their work was informed by input from researchers, industry, and people with diabetes through Advisory Committees representing each stakeholder group. Stakeholder surveys were used to identify priority outcomes. The outcomes prioritized in the surveys were hypoglycemia, hyperglycemia, time in range, DKA, and PROs. To develop consensus on the definitions of these outcomes, the Steering Committee relied on published evidence, their clinical expertise, and feedback from the Advisory Committees.RESULTSThe Steering Committee developed definitions for hypoglycemia, hyperglycemia, time in range, and DKA in type 1 diabetes. The definitions reflect their assessment of the outcome's short- and long-term clinical impact on people with type 1 diabetes. Knowledge gaps to be addressed by future research were identified. The Steering Committee discussed PROs and concluded that further type 1 diabetes-specific development is needed.CONCLUSIONSThe Steering Committee recommends use of the defined clinically meaningful outcomes beyond HbA(1c) in the research, development, and evaluation of type 1 diabetes therapies.