Safety and effectiveness in an uncontrolled setting of glucagon-like-peptide-1 receptor agonists in patients with familial partial lipodystrophy: Real-life experience from a national reference network

IF 5.7 2区医学 Q1 ENDOCRINOLOGY & METABOLISM Diabetes, Obesity & Metabolism Pub Date : 2025-01-20 DOI:10.1111/dom.16175

Sophie Lamothe MD, Ines Belalem MS, Marie-Christine Vantyghem MD, Estelle Nobecourt MD, Héléna Mosbah MD, Sophie Béliard MD, Brigitte Delemer MD, Hippolyte Dupuis MD, Paul Vandenbroere MD, Nicolas Scheyer MD, Chloé Amouyal MD, Samy Hadjadj MD, Sonja Janmaat PhD, Corinne Vigouroux MD, Camille Vatier MD

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Abstract

Aim

To describe the effects of Glucagon-like peptide-1 receptor agonists (GLP-1RA) in patients with familial partial lipodystrophy (FPLD) assessed in a real-life setting in a national reference network.

Patients and Methods

We retrospectively collected clinical and metabolic parameters in patients with FPLD in the French lipodystrophy reference network, who initiated GLP-1RA. Data were recorded before, at one-year (12 ± 6 months) and at the latest follow-up on GLP-1RA therapy (≥18 months).

Results

Seventy-six patients (89.4% of women), diagnosed with LMNA-related FPLD2 (n = 57), PPARG-related FPLD3 (n = 4), PLIN1-related FPLD4 (n = 5) or FPLD1 (n = 10) initiated GLP-1RA therapy between 2008 and 2024. Patients were aged a median (IQR) 48 years (34.5–57), body mass index (BMI) was 26.0 kg/m² (23.9–29.5), HbA1c 8.3% (7.5–9.3), triglycerides 2.31 mmol/L (1.62–3.88). GLP-1RA were used in addition to previously used antidiabetics, 50% of patients being insulin-treated. After one year with GLP-1RA therapy, BMI, HbA1c and triglycerides significantly decreased to 25.6 kg/m² (22.7–29.1), 7.3% (6.6–8.3) and 1.97 mmol/L (1.5–3.2) respectively (p < 0.001, p < 0.001 and p < 0.01, respectively), without significant changes in other antidiabetic and lipid-lowering drugs. Gamma-glutamyl-transferase and alanine-aminotransferase levels also significantly decreased. Effects on HbA1c, BMI and triglycerides persisted in the long term. One case of acute pancreatitis occurred during follow-up, associated with severe hypertriglyceridemia in a non-observant patient. Gastrointestinal symptoms affected 34% of patients, leading to GLP-1RA withdrawal in six patients.

Conclusion

GLP-1RA significantly improved BMI, HbA1c and triglycerides in a large majority of patients with FPLD. Larger and prospective controlled studies are warranted for identification of predictive factors and safety.

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家族性部分脂肪营养不良患者在不受控制的情况下使用胰高血糖素样肽-1受体激动剂的安全性和有效性：来自国家参考网络的真实经验

目的：描述胰高血糖素样肽-1受体激动剂（GLP-1RA）在家族性部分脂肪营养不良（FPLD）患者中的作用，在国家参考网络的现实环境中进行评估。患者和方法：我们回顾性地收集了法国脂肪营养不良参考网络中启动GLP-1RA的FPLD患者的临床和代谢参数。记录GLP-1RA治疗前、1年（12±6个月）和最新随访时（≥18个月）的数据。结果：76例（89.4%的女性），诊断为lmna相关FPLD2 (n = 57)， pparg相关FPLD3 (n = 4)， plin1相关FPLD4 （n = 5）或FPLD1 （n = 10）在2008年至2024年间开始了GLP-1RA治疗。患者年龄中位数（IQR）为48岁（34.5-57岁），体重指数（BMI）为26.0 kg/m2(23.9-29.5)，糖化血红蛋白为8.3%(7.5-9.3)，甘油三酯为2.31 mmol/L（1.62-3.88）。GLP-1RA是在先前使用的抗糖尿病药物之外使用的，50%的患者接受胰岛素治疗。GLP-1RA治疗一年后，BMI、HbA1c和甘油三酯分别显著降低至25.6 kg/m2（22.7-29.1）、7.3%（6.6-8.3）和1.97 mmol/L (1.5-3.2) (p结论：GLP-1RA显著改善了绝大多数FPLD患者的BMI、HbA1c和甘油三酯。为了确定预测因素和安全性，需要进行更大规模的前瞻性对照研究。

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来源期刊

Diabetes, Obesity & Metabolism 医学-内分泌学与代谢

CiteScore

10.90

自引率

6.90%

发文量

319

审稿时长

3-8 weeks

期刊介绍： Diabetes, Obesity and Metabolism is primarily a journal of clinical and experimental pharmacology and therapeutics covering the interrelated areas of diabetes, obesity and metabolism. The journal prioritises high-quality original research that reports on the effects of new or existing therapies, including dietary, exercise and lifestyle (non-pharmacological) interventions, in any aspect of metabolic and endocrine disease, either in humans or animal and cellular systems. ‘Metabolism’ may relate to lipids, bone and drug metabolism, or broader aspects of endocrine dysfunction. Preclinical pharmacology, pharmacokinetic studies, meta-analyses and those addressing drug safety and tolerability are also highly suitable for publication in this journal. Original research may be published as a main paper or as a research letter.