Type 1 diabetes (T1D) is a chronic autoimmune disease often diagnosed in childhood. Advances in staging and screening have enabled identification of presymptomatic T1D, creating new opportunities for early intervention. We aim to review the clinical rationale, evidence, safety profile, and practical considerations for the use of teplizumab in delaying the onset of clinical T1D in children. This narrative review synthesizes current literature, guidelines, and clinical trial data on islet autoantibody screening, referral pathways, and teplizumab’s mechanism, efficacy, and safety in pediatric populations. Teplizumab delays progression from stage 2 to stage 3 T1D by a median of 2 years in both children and adults with multiple islet autoantibodies and early dysglycemia. It is most effective when initiated before significant β-cell loss. Adverse effects are generally mild and transient. Early screening and referral are essential for identifying eligible patients. Integration of immunotherapy with existing diabetes technologies offers a more comprehensive approach to care. Conclusion: Teplizumab represents a significant advance in pediatric diabetes care, enabling early immunomodulation to delay disease onset. Pediatricians play a crucial role in risk-based screening, timely referral, and supporting shared decision-making around disease-modifying therapy.<Table Float="No" ID="Taba"> <tgroup cols="2"> <colspec align="left" colname="c1" colnum="1" /> <colspec align="left" colname="c2" colnum="2" /> <tbody> <row> <entry align="left" nameend="c2" namest="c1"> What is Known: • Type 1 diabetes develops through defined autoimmune stages before symptoms appear. • Technologies like CGMs and AID systems improve diabetes management but do not delay disease onset. </entry> </row> <row> <entry align="left" nameend="c2" namest="c1"> What is New: • Teplizumab is the first therapy to delay the onset of type 1 diabetes by targeting autoimmunity before symptoms arise. • This review provides/suggests a practical roadmap for pediatricians to identify, refer, and counsel at-risk children for timely immunotherapy. </entry> </row> </tbody> </tgroup> </Table>

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

期刊论文
发表日期：

2025年10月8日

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Doaa Zaitoon,
Hussein Zaitoon

摘要

Type 1 diabetes (T1D) is a chronic autoimmune disease often diagnosed in childhood. Advances in staging and screening have enabled identification of presymptomatic T1D, creating new opportunities for early intervention. We aim to review the clinical rationale, evidence, safety profile, and practical considerations for the use of teplizumab in delaying the onset of clinical T1D in children. This narrative review synthesizes current literature, guidelines, and clinical trial data on islet autoantibody screening, referral pathways, and teplizumab’s mechanism, efficacy, and safety in pediatric populations. Teplizumab delays progression from stage 2 to stage 3 T1D by a median of 2 years in both children and adults with multiple islet autoantibodies and early dysglycemia. It is most effective when initiated before significant β-cell loss. Adverse effects are generally mild and transient. Early screening and referral are essential for identifying eligible patients. Integration of immunotherapy with existing diabetes technologies offers a more comprehensive approach to care. Conclusion: Teplizumab represents a significant advance in pediatric diabetes care, enabling early immunomodulation to delay disease onset. Pediatricians play a crucial role in risk-based screening, timely referral, and supporting shared decision-making around disease-modifying therapy.

What is Known:

• Type 1 diabetes develops through defined autoimmune stages before symptoms appear.

• Technologies like CGMs and AID systems improve diabetes management but do not delay disease onset.

What is New:

• Teplizumab is the first therapy to delay the onset of type 1 diabetes by targeting autoimmunity before symptoms arise.

• This review provides/suggests a practical roadmap for pediatricians to identify, refer, and counsel at-risk children for timely immunotherapy.