Real‐world experiences of adult individuals or caregivers of children who received teplizumab treatment in stage 2 type 1 diabetes
Authors: O'Donnell Holly K. , Simmons Kimber M. , Gitelman Stephen E. , Dex Terry , Hill Robert , Wieloch Mattias , Zaccai Julia , D'Souza Jennifer , Ginchereau Sowell France , Turnbull James , Hood Korey K. 
Published: February 2025

AbstractAimsThis study surveyed individuals and caregivers of children who received teplizumab at stage 2 type 1 diabetes (T1D) to garner real‐world experiences and their health outlook for the future following treatment with the first approved disease‐modifying immune therapy for delaying the onset of Stage 3 T1D.Materials and MethodsThis was a cross‐sectional, observational, online survey (conducted September–October 2024) of adults (≥18 years) and caregivers of children (8–17 years) who received teplizumab while participating in the US COMPASS patient support program. Questions pertained to demographics, health history, T1D screening, the decision to take teplizumab, treatment, post‐treatment experience, outlook on prognosis and self‐reported health status. All data were summarized using descriptive statistics.ResultsA total of 47/116 invited individuals responded (30 adults, 17 caregivers of children). Almost half of respondents had a family history of T1D, and 36% reported autoimmune comorbidities. The top reason for both screening for T1D and receiving teplizumab was for a chance at delaying Stage 3 T1D. Although respondents expressed continued concern over diabetes progression, 87% felt grateful to receive teplizumab, 72% felt it would help slow down the disease, 60% felt it would make T1D easier to manage and most (>80%) would recommend treatment/make the same decision for another family member.ConclusionsIndividuals living with or caring for someone who received teplizumab felt grateful for the opportunity to delay disease or make it easier to manage following teplizumab, with most agreeing they would recommend teplizumab and make the same decision for family in their situation.Plain Language SummaryType 1 diabetes (T1D) happens when the body destroys cells in the pancreas that make insulin. This ultimately causes high blood sugar. People with T1D often worry about the long‐term effects of the disease. Teplizumab is the first FDA‐approved treatment that delays the onset of high blood sugar in people in the early stages of T1D—when there are signs of cell damage and mild increases in blood sugar. We surveyed adults (N = 30) and caregivers (N = 17) of children treated with teplizumab about their journey before and after treatment. More than half of adults were initially told they might have a different type of diabetes, usually type 2, before being accurately diagnosed with early‐stage T1D. Doctors specializing in diabetes were most often the ones who recommended screening for T1D. The most common reason to screen was a chance for more time before the onset of high blood sugar. Greater than 80% of people were grateful they or their child had the opportunity to receive teplizumab, and most would recommend it to others in a similar situation. After receiving teplizumab, most people still worried about their blood sugar and measured it frequently. Most thought that teplizumab would delay the disease progression to symptoms, and more than half thought teplizumab would make T1D easier to manage. These findings are the first to describe the journey of adults and caregivers of children treated with teplizumab, their reasons for screening, and their beliefs about the future.