Golani Sonia, Khan Sulhera, Saeed Zara, Talat Humaira, Shah Nazish
Dermatology, Dow University of Health Sciences, Civil Hospital Karachi, Karachi, PAK.
Internal Medicine, Jinnah Postgraduate Medical Centre, Karachi, PAK.
Cureus. 2025 Jun 17;17(6):e86207. doi: 10.7759/cureus.86207. eCollection 2025 Jun.
Familial hypercholesterolemia (FH) is a genetically inherited lipid disorder characterized by markedly elevated levels of low-density lipoprotein cholesterol (LDL-C), leading to premature cardiovascular disease and distinctive cutaneous manifestations such as tendon xanthomas and corneal arcus. We present the case of a young Pakistani woman with extensive yellowish plaques over her chest, hands, feet, and periocular area, alongside progressive deformities of the fingers and toes that clinically resembled rheumatoid arthritis, which developed over the course of four years. Laboratory investigations revealed significantly elevated total and LDL cholesterol, while autoimmune markers including rheumatoid factor (RF) and anti-CCP were negative. Radiographic imaging demonstrated soft tissue swelling, reduced joint spaces, and features of acro-osteolysis in the affected digits, suggestive of xanthomatous infiltration. The diagnosis of FH was established using clinical criteria, including the Simon-Broome criteria, Dutch Lipid Clinic Network (DLCN), and MEDPED scores, in the absence of genetic testing due to limited access. The patient was initiated on high-dose statin therapy with dietary and lifestyle modifications, and parents were offered genetic counselling. Although Proprotein Convertase Subtilisin/Kexin Type 9 Inhibitors (PCSK9) therapy was considered, its availability and cost posed significant barriers. This case highlights the critical role of physical findings and diagnostic scoring tools in identifying FH in resource-limited settings and adds to the scarce literature by reporting a rare rheumatoid-like presentation of FH, the first of its kind from Pakistan.
家族性高胆固醇血症(FH)是一种遗传性脂质紊乱疾病,其特征是低密度脂蛋白胆固醇(LDL-C)水平显著升高,导致过早发生心血管疾病,并伴有独特的皮肤表现,如肌腱黄色瘤和角膜弓。我们报告了一例年轻的巴基斯坦女性病例,她在胸部、手部、足部和眼周区域出现广泛的淡黄色斑块,同时手指和脚趾逐渐出现畸形,临床上类似于类风湿关节炎,这种情况在四年内逐渐发展。实验室检查显示总胆固醇和LDL胆固醇显著升高,而包括类风湿因子(RF)和抗环瓜氨酸肽(anti-CCP)在内的自身免疫标志物均为阴性。影像学检查显示受影响手指软组织肿胀、关节间隙变窄以及肢端骨质溶解特征,提示有黄色瘤浸润。由于获取基因检测有限,在未进行基因检测的情况下,根据临床标准,包括西蒙-布鲁姆标准、荷兰脂质诊所网络(DLCN)和MEDPED评分,确诊为FH。患者开始接受高剂量他汀类药物治疗,并进行饮食和生活方式调整,同时为其父母提供了遗传咨询。尽管考虑了前蛋白转化酶枯草溶菌素/克新9型抑制剂(PCSK9)治疗,但其可用性和成本构成了重大障碍。本病例强调了体格检查结果和诊断评分工具在资源有限环境中识别FH的关键作用,并通过报告FH罕见的类风湿样表现,为稀缺的文献增添了内容,这是来自巴基斯坦的首例此类报告。