Le Minh V, Harrison Leonard C, Spelman Tim, Wentworth John M
Department of Diabetes and Endocrinology, Royal Melbourne Hospital, Parkville, Victoria, Australia.
Coeliac Research Laboratory, Walter and Eliza Hall Institute of Medical Research, Parkville, Victoria, Australia.
Diabetes Care. 2025 Aug 1;48(8):1352-1355. doi: 10.2337/dc24-2794.
Approval of teplizumab as disease-modifying therapy for type 1 diabetes heralds a new therapeutic era. To facilitate prevention trials, we determined if the M120 risk score could enrich for type 1 diabetes risk and define early treatment effects.
M120, based on age, sex, BMI, IA-2 antibody status, HbA1c, blood glucose, and C-peptide 120 min after oral glucose, was determined in TrialNet participants with multiple islet autoantibodies and those who joined the teplizumab prevention trial.
Compared with the oral glucose tolerance test, M120 identified 26% more children at high risk of progression. When applied to data from the teplizumab trial, M120 improved after teplizumab (P = 0.0362) and deteriorated after placebo (P = 0.0489) to reveal a significant treatment effect after 6 months (P < 0.001).
M120 improves risk stratification and identifies early effects of immunotherapy. It could be applied to increase prevention trial efficiency and guide treatment decisions in the clinic.
替普珠单抗获批作为1型糖尿病的疾病改善疗法开创了一个新的治疗时代。为推动预防试验，我们确定了M120风险评分是否能富集1型糖尿病风险并确定早期治疗效果。
在TrialNet中具有多种胰岛自身抗体的参与者以及参加替普珠单抗预防试验的参与者中，根据年龄、性别、体重指数、胰岛抗原2抗体状态、糖化血红蛋白、血糖和口服葡萄糖后120分钟的C肽水平来确定M120。
与口服葡萄糖耐量试验相比，M120识别出进展高危儿童的比例高出26%。将M120应用于替普珠单抗试验的数据时，替普珠单抗治疗后M120有所改善（P = 0.0362），安慰剂治疗后M120恶化（P = 0.0489），6个月后显示出显著的治疗效果（P < 0.001）。
M120改善了风险分层并识别出免疫治疗的早期效果。它可用于提高预防试验效率并指导临床治疗决策。
