O-13: Teplizumab (TPZ) in pediatric type 1 diabetes (T1D): Real-World screening, treatment, and disease management – TEPLI-REAL study
M Tuffaha, J Felton, T Graham, K Simmons, A Tirosh, M Bonnemaire, B Mukherjee, C B Manzano-Salgado, T Dex, F Kleinjung, J M Gwinnutt, F Negreiro, J Zaccai, S GitelmanAbstract
Introduction
TPZ is an anti-CD3 monoclonal antibody approved to delay stage 3 T1D in individuals aged ≥8 years (y) with stage 2 T1D.
Objectives
To characterize screening, treatment (Tx) patterns, and explore disease progression in children who received TPZ.
Methods
TEPLI-REAL (NCT06892002) was a retrospective study in the US and Israel. Eligible individuals received ≥1 day of TPZ and were followed until data abstraction; analysis included participants aged <18y.
Results
Of 81 minors (74% of enrolled individuals), 54% were female, 81% were white (race collected for US only, n=75), and median age at TPZ initiation was 12y (range 8–17). A family history of T1D in first-degree relatives was common (siblings 31%, fathers 12% and mothers 9%); 14 (17%) had ≥1 comorbidity at TPZ initiation, most commonly Hashimoto’s disease (n=4) and celiac disease (n=3). Islet autoantibody (IA) testing was most requested by a pediatric endocrinologist (n=38; 47%) and performed by Autoimmunity Screening for Kids/TrialNet (n=27; 33%). Positive IAs were found for GAD65 (98%), ZnT8 (86%), IA-2 (76%), and IAA (59%); the mean was 3.1 IA/individual. At screening, 73 (91%) had HbA1c tests (median 5.8%; range 4.8–11.5), 36 (45%) had oral glucose tolerance tests, and 7 (9%) had continuous glucose monitoring data. Dysglycemia was identified before IA positivity in 40%, after in 37%, and simultaneously in 23%. The median (range) time between stage 2 confirmation and TPZ initiation was 3.6 (0.6–119.6) mo. Time from TPZ initiation to stage 3 T1D in children identified by medical review as stage 2 (exploratory endpoint) was estimated. TPZ Tx occurred mainly in hospitals (48%) or infusion centers (38%); 95% completed the 14-day course and 91% followed standard dosing. During follow-up, 26% initiated insulin, of whom 10% for reasons other than rising blood glucose.
Conclusions
This study demonstrates high completion rates of TPZ Tx in children and provides insights into its use in clinical practice.