Casas Rosaura, Tompa Andrea, Åkesson Karin, Teixeira Pedro F, Lindqvist Anton, Ludvigsson Johnny
Division of Pediatrics, Department of Biomedical and Clinical Sciences, Faculty of Medicine and Health Sciences, Linköping University, 581 83 Linköping, Sweden.
Department of Clinical Diagnostics, School of Health and Welfare, Jönköping University, 551 11 Jönköping, Sweden.
Int J Mol Sci. 2025 Jan 4;26(1):374. doi: 10.3390/ijms26010374.
Immunotherapies aimed at preserving residual beta cell function in type 1 diabetes have been successful, although the effect has been limited, or raised safety concerns. Transient effects often observed may necessitate redosing to prolong the effect, although this is not always feasible or safe. Treatment with intralymphatic GAD-alum has been shown to be tolerable and safe in persons with type 1 diabetes and has shown significant efficacy to preserve C-peptide with associated clinical benefit in individuals with the human leukocyte antigen DR3DQ2 haplotype. To further explore the feasibility and advantages of redosing with intralymphatic GAD-alum, six participants who had previously received active treatment with intralymphatic GAD-alum and carried HLA DR3-DQ2 received one additional intralymphatic dose of 4 μg GAD-alum in the pilot trial DIAGNODE-B. The participants also received 2000 U/day vitamin D (Calciferol) supplementation for two months, starting one month prior to the GAD-alum injection. During the 12-month follow-up, residual beta cell function was estimated with Mixed-Meal Tolerance Tests, and clinical and immune responses were observed. C-peptide decreased minimally, and most patients showed stable HbA1c and IDAA1c. The mean % TIR increased while the mean daily insulin dose decreased at month 12 compared to the baseline. Redosing with GAD-alum seems to be safe and tolerable, and may prolong the disease modification elicited by the original GAD-alum treatment.
旨在保留1型糖尿病患者残余β细胞功能的免疫疗法已取得成功,尽管效果有限,或引发了安全担忧。经常观察到的短暂效果可能需要重新给药以延长疗效,尽管这并不总是可行或安全的。已证明,对1型糖尿病患者进行淋巴内注射GAD-明矾治疗是可耐受且安全的,并且在携带人类白细胞抗原DR3DQ2单倍型的个体中,已显示出在保留C肽方面具有显著疗效以及相关的临床益处。为了进一步探索淋巴内注射GAD-明矾重新给药的可行性和优势,在DIAGNODE-B试点试验中,六名先前接受过淋巴内注射GAD-明矾积极治疗且携带HLA DR3-DQ2的参与者又接受了一次4μg GAD-明矾的淋巴内注射。参与者还在GAD-明矾注射前一个月开始,连续两个月每天补充2000U维生素D(骨化醇)。在12个月的随访期间,通过混合餐耐量试验评估残余β细胞功能,并观察临床和免疫反应。C肽水平下降极小,大多数患者的糖化血红蛋白(HbA1c)和免疫相关的糖化血红蛋白(IDAA1c)保持稳定。与基线相比,在第12个月时,平均血糖达标时间百分比(%TIR)增加,而平均每日胰岛素剂量减少。用GAD-明矾重新给药似乎是安全且可耐受的,并且可能会延长最初GAD-明矾治疗所引发的疾病改善效果。