背景:我们设计并实现了以患者为中心的,数据驱动,整体护理模式,评估其对年轻发病2型糖尿病(T2D)患者临床结局的影响,缺乏循证实践指南.
方法:在这项为期3年的精准医学重新定义胰岛素分泌和单基因糖尿病的随机对照试验中,我们评估了综合使用信息和通信技术(亚洲糖尿病联合评估(JADE)平台)的多组分护理模式的效果,在≤40岁和≤50岁诊断为T2D的患者中,生物遗传学标志物和患者报告的结局指标。JADE-PRISM组接受了为期1年的专家领导的基于团队的管理,使用生物遗传标记(全基因组单核苷酸多态性阵列,34个单基因糖尿病基因的外显子组测序,C-肽,自身抗体)以实现多个治疗目标(糖化血红蛋白(HbA1c)<6.2%,血压<120/75mmHg,低密度脂蛋白胆固醇<1.2mmol/L,腰围<80厘米(女性)或<85厘米(男性))在糖尿病中心设置与常规护理(JADE-only)。主要结果是所有糖尿病相关并发症的发生率。
结果:在2020-2021年,884名患者(56.6%的男性,中位(IQR)糖尿病病程:7(3-12)年,当前/戒烟者:32.5%,体重指数:28.40±5.77kg/m2,HbA1c:7.52%±1.66%,胰岛素治疗:27.7%)被分配到JADE-only组(n=443)或JADE-PRISM组(n=441).全组资料包括阳性家族史(74.7%),一般肥胖(51.4%),中心性肥胖(79.2%),高血压(66.7%),血脂异常(76.4%),白蛋白尿(35.4%),估计肾小球滤过率<60毫升/分钟/1.73平方米(4.0%),视网膜病变(13.8%),动脉粥样硬化性心血管疾病(5.2%),癌症(3.1%),情绪困扰(26%-38%)和次优依从性(54%),其中5项EuroQol的生活质量指数为0.88(0.87-0.96)。总的来说,13.7%达到次要结局中定义的≥3个代谢目标。在JADE-PRISM组中,4.5%有单基因糖尿病基因的致病/可能致病变异;5%有自身抗体,8.4%有空腹C肽<0.2nmol/L。其他重大事件包括低/大出生体重(33.4%),儿童肥胖症(50.7%),精神疾病(10.3%)和以前的自杀未遂(3.6%)。在妇女中,17.3%患有多囊卵巢综合征,44.8%的人在怀孕期间需要胰岛素治疗,17.3%的人出现不良妊娠结局。
结论:年轻发病型糖尿病的特点是病因复杂,并伴有包括精神疾病和生命历程事件在内的合并症。
背景:NCT04049149。
BACKGROUND: We designed and implemented a patient-centered, data-driven, holistic care model with evaluation of its impacts on clinical outcomes in patients with young-onset type 2 diabetes (T2D) for which there is a lack of evidence-based practice guidelines.
METHODS: In this 3-year Precision Medicine to Redefine Insulin Secretion and Monogenic Diabetes-Randomized Controlled Trial, we evaluate the effects of a multicomponent care model integrating use of information and communication technology (Joint Asia Diabetes Evaluation (JADE) platform), biogenetic markers and patient-reported outcome measures in patients with T2D diagnosed at ≤40 years of age and aged ≤50 years. The JADE-PRISM group received 1 year of specialist-led team-based management using treatment algorithms guided by biogenetic markers (genome-wide single-nucleotide polymorphism arrays, exome-sequencing of 34 monogenic diabetes genes, C-peptide, autoantibodies) to achieve multiple treatment goals (glycated hemoglobin (HbA1c) <6.2%, blood pressure <120/75 mm Hg, low-density lipoprotein-cholesterol <1.2 mmol/L, waist circumference <80 cm (women) or <85 cm (men)) in a diabetes center setting versus usual care (JADE-only). The primary outcome is incidence of all diabetes-related complications.
RESULTS: In 2020-2021, 884 patients (56.6% men, median (IQR) diabetes duration: 7 (3-12) years, current/ex-smokers: 32.5%, body mass index: 28.40±5.77 kg/m2, HbA1c: 7.52%±1.66%, insulin-treated: 27.7%) were assigned to JADE-only (n=443) or JADE-PRISM group (n=441). The profiles of the whole group included positive family history (74.7%), general obesity (51.4%), central obesity (79.2%), hypertension (66.7%), dyslipidemia (76.4%), albuminuria (35.4%), estimated glomerular filtration rate <60 mL/min/1.73 m2 (4.0%), retinopathy (13.8%), atherosclerotic cardiovascular disease (5.2%), cancer (3.1%), emotional distress (26%-38%) and suboptimal adherence (54%) with 5-item EuroQol for Quality of Life index of 0.88 (0.87-0.96). Overall, 13.7% attained ≥3 metabolic targets defined in secondary outcomes. In the JADE-PRISM group, 4.5% had pathogenic/likely pathogenic variants of monogenic diabetes genes; 5% had autoantibodies and 8.4% had fasting C-peptide <0.2 nmol/L. Other significant events included low/large birth weight (33.4%), childhood obesity (50.7%), mental illness (10.3%) and previous suicide attempts (3.6%). Among the women, 17.3% had polycystic ovary syndrome, 44.8% required insulin treatment during pregnancy and 17.3% experienced adverse pregnancy outcomes.
CONCLUSIONS: Young-onset diabetes is characterized by complex etiologies with comorbidities including mental illness and lifecourse events.
BACKGROUND: NCT04049149.