Service d'endocrinologie, diabétologie et endocrinologie de la reproduction, Centre national de référence des pathologies rares de l'insulino-sécrétion et de l'insulino-sensibilité (PRISIS), hôpital Saint-Antoine, Assistance publique-Hôpitaux de Paris, Paris, France; INSERM UMRS_938, Centre de recherche Saint-Antoine, Institut hospitalo-universitaire de cardiométabolisme et nutrition (ICAN), Sorbonne université, Paris, France.
Service d'endocrinologie, diabétologie et endocrinologie de la reproduction, Centre national de référence des pathologies rares de l'insulino-sécrétion et de l'insulino-sensibilité (PRISIS), hôpital Saint-Antoine, Assistance publique-Hôpitaux de Paris, Paris, France; INSERM UMRS_938, Centre de recherche Saint-Antoine, Institut hospitalo-universitaire de cardiométabolisme et nutrition (ICAN), Sorbonne université, Paris, France.
Ann Endocrinol (Paris). 2024 Jun;85(3):190-194. doi: 10.1016/j.ando.2024.05.019. Epub 2024 Jun 13.
Primary diseases of adipose tissue are rare disorders resulting from impairments in the physiological functions of adipose tissue (lipid stockage and endocrine function). It mainly refers to lipodystrophy syndromes with subcutaneous adipose tissue atrophy and/or altered body distribution of adipose tissue leading to insulin resistance, diabetes, hepatic steatosis, dyslipidemia, cardiovascular complications and polycystic ovary syndrome in women. Those syndromes are congenital or acquired, and lipoatrophy is partial or generalized. The diagnosis of lipodystrophy syndromes is often unrecognized, delayed and/or inaccurate, while it is of major importance to adapt investigations to search for specific comorbidities, in particular cardiovascular involvement, and set up multidisciplinary care, and in some cases specific treatment. Physicians have to recognize the clinical and biological elements allowing to establish the diagnosis. Lipodystrophic syndromes should be considered, notably, in patients with diabetes at a young age, with a normal or low BMI, negative pancreatic autoantibodies, presenting clinical signs of lipodystrophy and insulin resistance (acanthosis nigricans, hyperandrogenism, hepatic steatosis, high insulin doses). The association of diabetes and a family history of severe and/or early cardiovascular disease (coronary atherosclerosis, cardiomyopathy with rhythm and/or conduction disorders) may reveal Dunnigan syndrome, the most frequent form of familial lipodystrophy, due to LMNA pathogenic variants. Clinical assessment is primarily done through clinical examination: acanthosis nigricans, abnormal adipose tissue distribution, lipoatrophy, muscular hypertrophy, acromegaloid or Cushingoid features, lipomas, highly visible subcutaneous veins, may be revealing signs. The amount of circulating adipokines may reflect of adipose dysfunction with low leptinemia and adiponectinemia. Other biological metabolic parameters (hypertriglyceridemia, hyperinsulinemia, increased glycemia and hepatic enzymes) may also represent markers of insulin resistance. Quantification of total body fat by impedancemetry or dual-photon X-ray absorptiometry (DEXA) reveals decreased total body mass, in correlation with adipose tissue atrophy; metabolic magnetic resonance imaging can also quantify intraperitoneal and abdominal fat and the degree of hepatic steatosis. Histological analysis of adipose tissue showing structural abnormalities should be reserved for clinical research. Acquired lipodystrophic syndromes most often lead to similar clinical phenotype as congenital syndromes with generalized or partial lipoatrophy. The most frequent causes are old anti-HIV therapy or glucocorticoid treatments. Family history, history of treatments and clinical examination, including a careful physical examination, are keys for diagnosis.
原发性脂肪组织疾病是罕见的疾病,源于脂肪组织(脂质储存和内分泌功能)的生理功能障碍。它主要是指脂肪营养不良综合征,表现为皮下脂肪组织萎缩和/或脂肪组织分布改变,导致胰岛素抵抗、糖尿病、肝脂肪变性、血脂异常、心血管并发症和多囊卵巢综合征。这些综合征有先天性和获得性,脂肪减少是部分性或全身性的。脂肪营养不良综合征的诊断往往被忽视、延迟和/或不准确,而适应调查以寻找特定的合并症,特别是心血管受累,并建立多学科护理,在某些情况下进行特定的治疗,这一点非常重要。医生必须认识到能够建立诊断的临床和生物学要素。特别是在年轻的糖尿病患者中,当 BMI 正常或较低、无胰腺自身抗体、存在脂肪营养不良和胰岛素抵抗的临床体征(黑棘皮病、高雄激素血症、肝脂肪变性、高胰岛素剂量)时,应考虑脂肪营养不良综合征。糖尿病与严重和/或早期心血管疾病(冠状动脉粥样硬化、伴有节律和/或传导障碍的心肌病)家族史的联合可能揭示最常见的家族性脂肪营养不良形式——Dunnigan 综合征,这是由于 LMNA 致病性变异引起的。临床评估主要通过临床检查进行:黑棘皮病、异常脂肪组织分布、脂肪减少、肌肉肥大、肢端肥大症或库欣样特征、脂肪瘤、高度可见的皮下静脉,这些可能是提示性的体征。循环脂联素的量可能反映了脂肪功能障碍,表现为瘦素血症和脂联素血症降低。其他生物学代谢参数(高三酰甘油血症、高胰岛素血症、血糖升高和肝酶升高)也可能是胰岛素抵抗的标志物。通过阻抗法或双能 X 射线吸收法(DEXA)定量测量全身脂肪量可发现总身体质量减少,与脂肪组织萎缩相关;代谢磁共振成像还可以定量测量腹腔内和腹部脂肪以及肝脂肪变性程度。显示结构异常的脂肪组织的组织学分析应保留用于临床研究。获得性脂肪营养不良综合征最常导致与先天性综合征相似的临床表型,表现为全身性或部分性脂肪减少。最常见的原因是旧的抗 HIV 治疗或糖皮质激素治疗。家族史、治疗史和临床检查,包括仔细的体格检查,是诊断的关键。