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.
