再给药淋巴内gad -明矾治疗1型糖尿病:诊断- b先导试验

IF 5.6 2区 生物学 International Journal of Molecular Sciences Pub Date : 2025-01-04 DOI:10.3390/ijms26010374
Rosaura Casas, Andrea Tompa, Karin Åkesson, Pedro F Teixeira, Anton Lindqvist, Johnny Ludvigsson
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引用次数: 0

摘要

旨在保留1型糖尿病患者剩余β细胞功能的免疫疗法已经取得了成功,尽管其效果有限,或者引起了安全问题。经常观察到的短暂效应可能需要重新给药以延长效果,尽管这并不总是可行或安全的。在1型糖尿病患者中,使用淋巴内gad -明矾治疗已被证明是可耐受和安全的,并且在具有人类白细胞抗原DR3DQ2单倍型的个体中,已显示出保存c肽的显著疗效,并具有相关的临床益处。为了进一步探索再给药淋巴内gad -明矾的可行性和优势,在初步试验diagnosis - b中,6名先前接受淋巴内gad -明矾积极治疗并携带HLA DR3-DQ2的参与者接受了一次额外的4 μg gad -明矾淋巴内剂量。参与者还接受了2000 U/天的维生素D(钙化醇)补充,为期两个月,从注射gad -明矾前一个月开始。在12个月的随访中,通过混合膳食耐受性试验估计剩余β细胞功能,并观察临床和免疫反应。c肽下降幅度最小,多数患者HbA1c和IDAA1c稳定。在第12个月,与基线相比,平均% TIR增加,而平均每日胰岛素剂量减少。重新给药gad -明矾似乎是安全且耐受的,并可能延长原gad -明矾治疗引起的疾病改变。
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Redosing with Intralymphatic GAD-Alum in the Treatment of Type 1 Diabetes: The DIAGNODE-B Pilot Trial.

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.

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期刊介绍: The International Journal of Molecular Sciences (ISSN 1422-0067) provides an advanced forum for chemistry, molecular physics (chemical physics and physical chemistry) and molecular biology. It publishes research articles, reviews, communications and short notes. Our aim is to encourage scientists to publish their theoretical and experimental results in as much detail as possible. Therefore, there is no restriction on the length of the papers or the number of electronics supplementary files. For articles with computational results, the full experimental details must be provided so that the results can be reproduced. Electronic files regarding the full details of the calculation and experimental procedure, if unable to be published in a normal way, can be deposited as supplementary material (including animated pictures, videos, interactive Excel sheets, software executables and others).
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