一例Schwartz-Jampel综合征患者的他汀类药物诱导的肌病。

IF 16.4 1区 化学 Q1 CHEMISTRY, MULTIDISCIPLINARY Accounts of Chemical Research Pub Date : 2024-10-01 Epub Date: 2023-11-06 DOI:10.1177/08971900231213694
Mandeep K Rajpal, Farah K Sedki, Tony J Eid
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引用次数: 0

摘要

Schwartz-Jampel综合征(SJS)是一种以肌强直和软骨发育不良为特征的遗传性疾病。Perlecan基因(HSPG2)编码肌肉、骨骼和软骨细胞外基质的关键成分,其突变是SJS特征性畸形的原因。临床显著的肌酸酐磷酸激酶(CPK)水平是典型的,可能与肌强直有关,肌强直是SJS患者的潜在原因。我们报告了一例独特的SJS患者使用他汀类药物的症状性不良事件,该患者在他汀类药物诱导的肌病后表现出CPK水平升高至基线。他汀类药物的停用和PCSK-9抑制剂的给药显示CPK恢复到基线水平。该病例对目前的脂质治疗算法提出了挑战,因为它适用于SJS患者。需要对这一特殊人群的治疗进行进一步调查。
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Statin-Induced Myopathy in a Patient with Schwartz-Jampel Syndrome.

Schwartz Jampel syndrome (SJS) is a genetic disorder characterized by myotonia and chondrodysplasia. Mutations of the Perlecan gene (HSPG2), which encodes a key component of the extracellular matrix of muscle, bone, and cartilage is cause for the characteristic dysmorphisms of SJS. Clinically remarkable creatinine phosphokinase (CPK) levels are typical and can be associated with myotonia as an underlying cause in SJS patients. We report a unique case of a symptomatic adverse event of statin use in a SJS patient who demonstrated heightened levels of CPK to baseline following a statin induced myopathy. Discontinuation of the statin and administration of a PCSK-9 inhibitor revealed a return to baseline CPK. This case challenges the current lipid treatment algorithm as it pertains to SJS patients. Further investigation into treatment is required in this special population.

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来源期刊
Accounts of Chemical Research
Accounts of Chemical Research 化学-化学综合
CiteScore
31.40
自引率
1.10%
发文量
312
审稿时长
2 months
期刊介绍: Accounts of Chemical Research presents short, concise and critical articles offering easy-to-read overviews of basic research and applications in all areas of chemistry and biochemistry. These short reviews focus on research from the author’s own laboratory and are designed to teach the reader about a research project. In addition, Accounts of Chemical Research publishes commentaries that give an informed opinion on a current research problem. Special Issues online are devoted to a single topic of unusual activity and significance. Accounts of Chemical Research replaces the traditional article abstract with an article "Conspectus." These entries synopsize the research affording the reader a closer look at the content and significance of an article. Through this provision of a more detailed description of the article contents, the Conspectus enhances the article's discoverability by search engines and the exposure for the research.
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