Teplizumab for stage 2 type 1 diabetes: a new era in pediatric diabetes prevention

Area of Science:

• Immunology
• Endocrinology
• Pediatrics

Background:

• Type 1 diabetes (T1D) progresses through identifiable autoimmune stages before clinical symptoms manifest.
• Current diabetes management technologies like continuous glucose monitoring (CGM) and automated insulin delivery (AID) systems enhance glycemic control but do not prevent disease progression.
• Teplizumab offers the first therapeutic approach to delay T1D onset by targeting the underlying autoimmune process before symptom presentation.

Purpose of the Study:

• To review the clinical rationale, evidence, safety, and practical considerations for using teplizumab to delay clinical T1D onset in children.
• To synthesize current literature, guidelines, and trial data on islet autoantibody screening, referral pathways, and teplizumab's use in pediatric populations.
• To provide a roadmap for pediatricians on identifying, referring, and counseling at-risk children for immunotherapy.

Main Methods:

• Narrative review of current literature, clinical trial data, and guidelines.
• Synthesis of information on islet autoantibody screening and referral pathways.
• Analysis of teplizumab's mechanism of action, efficacy, and safety in pediatric populations.

Main Results:

• Teplizumab delays progression from stage 2 to stage 3 T1D by a median of 2 years in children and adults with multiple islet autoantibodies and early dysglycemia.
• The therapy is most effective when initiated prior to substantial beta-cell loss.
• Reported adverse effects associated with teplizumab are generally mild and transient.

Conclusions:

• Teplizumab represents a significant advancement in pediatric diabetes care, allowing for early immunomodulation to postpone T1D onset.
• Pediatricians are vital in implementing risk-based screening, facilitating timely referrals, and supporting shared decision-making regarding disease-modifying therapies.