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病例报告:亡羊补牢,为时未晚,但早做更好:两名 KCNJ11 变异致新生儿糖尿病患者由 CSII 转用磺脲类药物。

Case report: Better late than never, but sooner is better: switch from CSII to sulfonylureas in two patients with neonatal diabetes due to KCNJ11 variants.

机构信息

Division of Pediatrics, Department of Health Sciences, University of Piemonte Orientale, Novara, Italy.

Neonatal and Pediatric Intensive Care Unit, Azienda Ospedaliero Universitaria Maggiore della Carità, Novara, Italy.

出版信息

Front Endocrinol (Lausanne). 2023 May 11;14:1143736. doi: 10.3389/fendo.2023.1143736. eCollection 2023.

Abstract

Neonatal diabetes mellitus (NDM) is a rare genetic disease characterized by severe hyperglycemia requiring insulin therapy with onset mostly within the first 6 months and rarely between 6-12 months of age. The disease can be classified into transient (TNDM) or permanent neonatal diabetes mellitus (PNDM), or it can be a component of a syndrome. The most frequent genetic causes are abnormalities of the 6q24 chromosomal region and mutations of the ABCC8 or KCNJ11 genes coding for the pancreatic beta cell's potassium channel (KATP). After the acute phase, patients with ABCC8 or KCNJ11 mutations treated with insulin therapy can switch to hypoglycemic sulfonylureas (SU). These drugs close the KATP channel binding the SUR1 subunit of the potassium channel and restoring insulin secretion after a meal. The timing of this switch can be different and could affect long-term complications. We describe the different management and clinical outcome over the time of two male patients with NDM due to KCNJ11 pathogenetic variants. In both cases, continuous subcutaneous insulin infusion pumps (CSII) were used to switch therapy from insulin to SU, but at different times after the onset. The two patients kept adequate metabolic control after the introduction of glibenclamide; during the treatment, insulin secretion was evaluated with c-peptide, fructosamine, and glycated hemoglobin (HbA1c), which were within the normal range. In neonates or infants with diabetes mellitus, genetic testing is an indispensable diagnostic tool and KCNJ11 variants should be considered. A trial of oral glibenclamide must be considered, switching from insulin, the first line of NDM treatment. This therapy can improve neurological and neuropsychological outcomes, in particular in the case of earlier treatment initiation. A new modified protocol with glibenclamide administered several times daily according to continuous glucose monitoring profile indications, was used. Patients treated with glibenclamide maintain good metabolic control and prevent hypoglycemia, neurological damage, and apoptosis of beta cells during long-term administration.

摘要

新生儿糖尿病(NDM)是一种罕见的遗传性疾病,其特征为严重高血糖,需要胰岛素治疗,发病时间大多在出生后 6 个月内,极少数在 6-12 个月之间。该疾病可分为暂时性(TNDM)或永久性新生儿糖尿病(PNDM),也可能是综合征的一个组成部分。最常见的遗传原因是 6q24 染色体区域的异常和编码胰腺β细胞钾通道(KATP)的 ABCC8 或 KCNJ11 基因突变。在急性阶段后,接受胰岛素治疗的 ABCC8 或 KCNJ11 突变患者可以改用降血糖磺酰脲类药物(SU)。这些药物可关闭 KATP 通道,结合钾通道 SUR1 亚基,在餐后恢复胰岛素分泌。这种转换的时机可能不同,并可能影响长期并发症。我们描述了两名男性 NDM 患者因 KCNJ11 致病性变异而接受不同治疗管理和临床结局的情况。在两种情况下,均使用连续皮下胰岛素输注泵(CSII)将治疗从胰岛素切换为 SU,但发病后时间不同。两名患者在引入格列本脲后保持了适当的代谢控制;在治疗期间,通过 C 肽、果糖胺和糖化血红蛋白(HbA1c)评估胰岛素分泌,这些指标均在正常范围内。在患有糖尿病的新生儿或婴儿中,基因检测是不可或缺的诊断工具,应考虑 KCNJ11 变异。必须考虑试用口服格列本脲,作为 NDM 治疗的一线药物。这种治疗方法可以改善神经和神经心理学结局,尤其是在早期治疗开始时。使用了一种新的改良方案,根据持续血糖监测谱的指示,每天多次给予格列本脲。接受格列本脲治疗的患者在长期治疗期间保持良好的代谢控制,防止低血糖、神经损伤和β细胞凋亡。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5508/10211331/417a1b54bc66/fendo-14-1143736-g001.jpg

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