Real-world experiences of adult individuals or caregivers of children who received teplizumab treatment in stage 2 type 1 diabetes

Aims: This 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 Methods: This 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. Results: A 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. Conclusions: Individuals 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.