Pediatric Endocrine Society Statement on Considerations for Use of Teplizumab (Tzield™) in Clinical Practice

Affiliations

¹ Division of Pediatric Endocrinology, Department of Pediatrics, New York Medical College, Valhalla, New York, USA.
² Division of Endocrinology, Diabetes, and Metabolism, Children's Hospital Los Angeles, Los Angeles, California, USA.
³ Division of Pediatric Endocrinology and Diabetes, The Children's Hospital of Philadelphia, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
⁴ Division of Pediatric Endocrinology and Diabetes, Hassenfeld Children's Hospital, New York University School of Medicine, New York, New York, USA.
⁵ Division of Pediatric Diabetes and Endocrinology, UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, USA.
⁶ Division of Pediatric Endocrinology, University of Florida, Gainesville, Florida, USA.
⁷ Division of Pediatrics, Barbara Davis Center for Diabetes, University of Colorado School of Medicine, Aurora, Colorado, USA.
⁸ Division of Pediatric Endocrinology and Diabetes, Department of Pediatrics, Riley Hospital for Children, Indiana University School of Medicine, Indianapolis, Indiana, USA.
⁹ Department of Pediatrics, Diabetes Center, University of California at San Francisco, San Francisco, California, USA.
¹⁰ Sanford Health, Sioux Falls, SD and Department of Pediatrics, Sanford School of Medicine, University of South Dakota, Sioux Falls, South Dakota, USA.
¹¹ Division of Pediatric Endocrinology, Diabetes and Metabolism, Rainbow Babies and Children's Hospital, CWRU School of Medicine, Cleveland, Ohio, USA.

PMID: 38663372
PMCID: PMC12416869
DOI: 10.1159/000538775

Practice Guideline

Pediatric Endocrine Society Statement on Considerations for Use of Teplizumab (Tzield™) in Clinical Practice

Ksenia N Tonyushkina et al. Horm Res Paediatr. 2025.

. 2025;98(5):597-608.

doi: 10.1159/000538775. Epub 2024 Apr 30.

Authors

Affiliations

¹ Division of Pediatric Endocrinology, Department of Pediatrics, New York Medical College, Valhalla, New York, USA.
² Division of Endocrinology, Diabetes, and Metabolism, Children's Hospital Los Angeles, Los Angeles, California, USA.
³ Division of Pediatric Endocrinology and Diabetes, The Children's Hospital of Philadelphia, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
⁴ Division of Pediatric Endocrinology and Diabetes, Hassenfeld Children's Hospital, New York University School of Medicine, New York, New York, USA.
⁵ Division of Pediatric Diabetes and Endocrinology, UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, USA.
⁶ Division of Pediatric Endocrinology, University of Florida, Gainesville, Florida, USA.
⁷ Division of Pediatrics, Barbara Davis Center for Diabetes, University of Colorado School of Medicine, Aurora, Colorado, USA.
⁸ Division of Pediatric Endocrinology and Diabetes, Department of Pediatrics, Riley Hospital for Children, Indiana University School of Medicine, Indianapolis, Indiana, USA.
⁹ Department of Pediatrics, Diabetes Center, University of California at San Francisco, San Francisco, California, USA.
¹⁰ Sanford Health, Sioux Falls, SD and Department of Pediatrics, Sanford School of Medicine, University of South Dakota, Sioux Falls, South Dakota, USA.
¹¹ Division of Pediatric Endocrinology, Diabetes and Metabolism, Rainbow Babies and Children's Hospital, CWRU School of Medicine, Cleveland, Ohio, USA.

PMID: 38663372
PMCID: PMC12416869
DOI: 10.1159/000538775

Abstract

Teplizumab (TzieldTM, Provention Bio), a monoclonal antibody directed at T-cell marker CD3, is the first medication approved by the FDA to delay progression from stage 2 to stage 3 type 1 diabetes. To date, the overwhelming majority of pediatric endocrinologists do not have experience using immunotherapeutics and seek guidance on the use of teplizumab in clinical practice. To address this need, the Pediatric Endocrine Society (PES) Diabetes Special Interest Group (Diabetes SIG) and Drug and Therapeutics Committee assembled a task force to review clinical trial data and solicit expert recommendations on the approach to teplizumab infusions. We present considerations on all aspects of teplizumab administration, utilizing evidence where possible and providing a spectrum of expert opinions on unknown aspects. We discuss patient selection and prescreening, highlighting the safety and considerations for monitoring and treatment of side effects. We propose a schedule of events, a protocol for administration, and discuss practice management aspects. We advocate for the need for further long-term systematic surveillance studies to continue evaluating the efficacy and safety of teplizumab.

Keywords: Beta cell function; Immune therapy; Stage 2 type 1 diabetes; Stage 3 type 1 diabetes prevention; Teplizumab.

PubMed Disclaimer

Conflict of interest statement

A.R.-L. has been a consultant for Ascendis, BioMarin, BridgeBio. A.R.-L. has been a grant recipient and site-principal investigator for Ultragenyx, Amgen, Ascendis, Takeda. M.J.H. has been a consultant for Sanofi and MannKind and is on the scientific advisory board for SAB Biotherapeutics. E.K.S. has received compensation for educational lectures on diabetes screening from Medscape, the American Diabetes Association, Sanofi, and Health Matters CME, for serving as the Chair of the Steering Committee for Clinical Advances in T1D: Screening, Staging, and Treatment, serving on the Sanofi Drug Agnostic T1D Screening Committee, and consulting for DRI Healthcare and Sanofi. L.A.D. has served on advisory boards for Abata and Vertex and has had clinical trials support to her institution from Dompe, Lilly, Provention Bio, Sanofi, Zealand, and NIH. S.E.G. has served on advisory boards for Abata, Avotres, Genentech, GentiBio, Provention Bio, SAB Biotherapeutics, Sana Biotechnology, and Sanofi. He has also received clinical trial support from Provention Bio and NIH. He serves on data and safety monitoring boards for Diamyd, Juvenile Diabetes Research Foundation, and INNODIA. K.J.G. served as a site PI for commercial clinical trials with teplizumab (Provention Bio and Precigen). K.N.T. served as a site PI for PROTECT clinical trial with teplizumab (Provention Bio).

References

1. Prescribing information. FDA; 2022. https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/761183s000lbl.pdf.
1. Herold KC, Bundy BN, Krischer JP; Type 1 Diabetes TrialNet Study Group . Teplizumab in relatives at risk for type 1 diabetes. Reply. N Engl J Med. 2019;381(19):1880–1. - PubMed
1. Bisikirska B, Colgan J, Luban J, Bluestone JA, Herold KC. TCR stimulation with modified anti-CD3 mAb expands CD8+ T cell population and induces CD8+CD25+ Tregs. J Clin Invest. 2005;115(10):2904–13. - PMC - PubMed
1. Ablamunits V, Bisikirska B, Herold KC. Acquisition of regulatory function by human CD8(+) T cells treated with anti-CD3 antibody requires TNF. Eur J Immunol. 2010;40(10):2891–901. - PMC - PubMed
1. Lebastchi J, Deng S, Lebastchi AH, Beshar I, Gitelman S, Willi S, et al. Immune therapy and β-cell death in type 1 diabetes. Diabetes. 2013;62(5):1676–80. - PMC - PubMed

Publication types

Actions

MeSH terms

Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions

Substances

Actions
Actions

LinkOut - more resources

Full Text Sources
Medical
- MedlinePlus Health Information

Save citation to file

Email citation

Add to Collections

Add to My Bibliography

Your saved search

Create a file for external citation management software

Your RSS Feed

Pediatric Endocrine Society Statement on Considerations for Use of Teplizumab (Tzield™) in Clinical Practice

Affiliations

Pediatric Endocrine Society Statement on Considerations for Use of Teplizumab (Tzield™) in Clinical Practice

Authors

Affiliations

Abstract

Conflict of interest statement

References

Publication types

MeSH terms

Substances

LinkOut - more resources

Full Text Sources

Medical