Treatment acceptability for disease‐modifying therapy for type 1 diabetes ( T1D )—Views from parents of children with presymptomatic T1D
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· 2025
· Open Access
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· DOI: https://doi.org/10.1111/dom.16639
· OA: W4412591919
The first disease-modifying therapy for type 1 diabetes (T1D), teplizumab, became available in 2022 in the US.1 Teplizumab is currently undergoing review for licensing in the UK and delays symptomatic onset and need for insulin by approximately two years in those with stage 2 presymptomatic T1D2, defined as multiple islet-specific autoantibody seropositivity and dysglycaemia on oral glucose tolerance testing.3 Teplizumab is administered by daily infusion over two weeks, as a single course of treatment. Both hospital setting and home infusion can be accommodated.1 The most common adverse event is a mild cytokine release syndrome including myalgia, fever, and rash2 which resolves with prophylactic, symptomatic management.1 Approximately 70% develop transient lymphopenia within 30 days of treatment which resolves without sequelae.2 Although appearing safe in the long-term, with more than six years surveillance data demonstrating no increased risk of infection or malignancy in this cohort of patients4, treatment comes at significant financial cost (both to healthcare systems and family) and requires absence from work and school5. Critically, the acceptability of teplizumab treatment has not been formally assessed via qualitative interviews anywhere in the world and we wanted to explore this to outline important considerations for clinicians to support shared decision-making should it be licensed in the UK. Here, we present the first qualitative data with parents/caregivers of stage 2 children, exploring views towards disease-modifying treatment.
