一例Schwartz-Jampel综合征患者的他汀类药物诱导的肌病。

IF 1 Q4 PHARMACOLOGY & PHARMACY
Journal of pharmacy practice Pub Date : 2024-10-01 Epub Date: 2023-11-06 DOI:10.1177/08971900231213694
Mandeep K Rajpal, Farah K Sedki, Tony J Eid
{"title":"一例Schwartz-Jampel综合征患者的他汀类药物诱导的肌病。","authors":"Mandeep K Rajpal, Farah K Sedki, Tony J Eid","doi":"10.1177/08971900231213694","DOIUrl":null,"url":null,"abstract":"<p><p>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.</p>","PeriodicalId":16818,"journal":{"name":"Journal of pharmacy practice","volume":" ","pages":"1197-1198"},"PeriodicalIF":1.0000,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Statin-Induced Myopathy in a Patient with Schwartz-Jampel Syndrome.\",\"authors\":\"Mandeep K Rajpal, Farah K Sedki, Tony J Eid\",\"doi\":\"10.1177/08971900231213694\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>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.</p>\",\"PeriodicalId\":16818,\"journal\":{\"name\":\"Journal of pharmacy practice\",\"volume\":\" \",\"pages\":\"1197-1198\"},\"PeriodicalIF\":1.0000,\"publicationDate\":\"2024-10-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Journal of pharmacy practice\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1177/08971900231213694\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2023/11/6 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"Q4\",\"JCRName\":\"PHARMACOLOGY & PHARMACY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of pharmacy practice","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1177/08971900231213694","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2023/11/6 0:00:00","PubModel":"Epub","JCR":"Q4","JCRName":"PHARMACOLOGY & PHARMACY","Score":null,"Total":0}
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。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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.

求助全文
通过发布文献求助,成功后即可免费获取论文全文。 去求助
来源期刊
Journal of pharmacy practice
Journal of pharmacy practice PHARMACOLOGY & PHARMACY-
CiteScore
3.20
自引率
7.70%
发文量
184
期刊介绍: The Journal of Pharmacy Practice offers the practicing pharmacist topical, important, and useful information to support pharmacy practice and pharmaceutical care and expand the pharmacist"s professional horizons. The journal is presented in a single-topic, scholarly review format. Guest editors are selected for expertise in the subject area, who then recruit contributors from that practice or topic area.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信