What question did this study set out to answer?

To assess the efficacy and safety of teplizumab for stage 3 type 1 diabetes based on randomized controlled trials.

May 25, 2026Open Access

The Efficacy and Safety of Teplizumab in the Treatment of Stage 3 Type 1 Diabetes: A Systematic Review and Meta-Analysis of Randomized Controlled Trials

Key Points

To assess the efficacy and safety of teplizumab for stage 3 type 1 diabetes based on randomized controlled trials.
Systematic review and meta-analysis of randomized controlled trials
Comparison of intravenous teplizumab monotherapy with placebo/standard care
Assessment of primary outcome C-peptide AUC and secondary outcomes including insulin dose and HbA1c.
Teplizumab improved C-peptide AUC preservation at 9–15 months (SMD=0.28, 95% CI 0.09–0.48, P=0.016)
Reduced insulin dose (MD=−0.11 U/kg/day) and HbA1c (MD=−0.22%)
Increased partial remission rate (RR=1.39) while showing increased risk of immune-related events.

Abstract

Background: After diagnosis of stage 3 type 1 diabetes, β-cell decline persists, and intensive insulin therapy alone still fails to control metabolic fluctuations and acute risks; teplizumab may delay decline, but trial findings are inconsistent, necessitating a systematic review of efficacy and safety. Objective: To evaluate the efficacy and safety of teplizumab for stage 3 type 1 diabetes from randomized controlled trials and to determine the certainty and applicability of the evidence. Methods: PubMed, Embase, Cochrane Library, and Web of Science were searched up to October 31, 2025, including parallel RCTs comparing intravenous teplizumab monotherapy with placebo/standard care. The primary outcome was preservation of MMTT C-peptide AUC at 9– 15 months (SMD), with assessment at 18– 24 months; secondary outcomes were insulin dose and HbA1c (MD), and partial remission (RR, IDAA1c≤ 9). Risk of bias was assessed using RoB 2; random-effects meta-analysis was performed, and subgroup, sensitivity, and GRADE assessments were conducted. Results: Five double-blind, placebo-controlled RCTs were included (975 analyzed), with overall low risk of bias. Teplizumab improved C-peptide AUC preservation at 9– 15 months (SMD=0.28, 95% CI 0.09– 0.48, P=0.016; exceeded MCID 0.20), and showed only a favorable trend at 18– 24 months (SMD=0.27, 95% CI − 0.02– 0.56). It also reduced insulin dose (MD=− 0.11 U/kg/day) and HbA1c (MD=− 0.22%), and increased partial remission rate (RR=1.39). For safety, immune-related events increased (lymphopenia RR=3.36, infusion-related reactions RR=2.79), whereas infection (RR=0.98) and diabetic ketoacidosis (RR=0.80) showed no apparent difference, and severe hypoglycemia decreased (RR=0.67). GRADE indicated moderate certainty for short-term outcomes, and low certainty for long-term C-peptide and some acute events. Conclusion: Teplizumab was associated with preservation of β-cell function and modest metabolic benefit, with immune-related reactions as the main risk and no increase in serious clinical events observed; long-term benefits remain to be confirmed. Keywords: teplizumab, type 1 diabetes, randomized controlled trial, meta-analysis

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