{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,19]],"date-time":"2026-03-19T03:07:30Z","timestamp":1773889650430,"version":"3.50.1"},"reference-count":22,"publisher":"Georg Thieme Verlag KG","issue":"02\/03","funder":[{"name":"Natural Science Foundation of Chongqing, China","award":["CSTB2022NSCQ-MSX1207"],"award-info":[{"award-number":["CSTB2022NSCQ-MSX1207"]}]},{"name":"Chongqing University Jiangjin Hospital","award":["project No. 2022qdjfxm002"],"award-info":[{"award-number":["project No. 2022qdjfxm002"]}]},{"name":"Scientific Research cultivation project of Chongqing University\n                        Jiangjin Hospital","award":["No. 2024YCXM003 and 2023YCXM002"],"award-info":[{"award-number":["No. 2024YCXM003 and 2023YCXM002"]}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Drug Res (Stuttg)"],"published-print":{"date-parts":[[2026,3]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>Both eliglustat and miglustat are substrate reduction therapies targeting\n                        glucosylceramide synthase; yet, their safety profile has not been\n                        comprehensively analyzed. This study analyzes adverse events associated with\n                        both drugs using the U.S. Food and Drug Administration Adverse Event\n                        Reporting System to provide insights for clinical safety.<\/jats:p>\n                  <jats:p>Adverse events were classified by MedDRA System Organ Class (SOC, v26.1).\n                        Adverse event signals were mined by disproportionality analyses, including\n                        the reporting odds ratio, the proportional reporting ratio, the multi-item\n                        gamma Poisson shrinker algorithms, and the Bayesian confidence propagation\n                        neural network.<\/jats:p>\n                  <jats:p>A total of 1,223 and 980 adverse event reports were retrieved from eliglustat\n                        and miglustat, respectively, involving 27 System Organ Class categories\n                        each. Some positive signals were consistent with the drug labels, including\n                        dyspepsia identified in eliglustat and diarrhoea identified in miglustat. We\n                        also identified unexpected signals not listed on the drug labels, such as\n                        paresthesia, dry skin, and ichthyosis for eliglustat and dysphagia for\n                        miglustat. For patients treated with eliglustat and miglustat, the majority\n                        of adverse events manifested more than 1 year after the initiation of\n                        therapy. Notably, male patients treated with eliglustat have the\n                        significantly higher incidence of weight increase and dry skin. Female\n                        patients treated with miglustat have the significantly higher incidence of\n                        dysphagia and cognitive disorder.<\/jats:p>\n                  <jats:p>In the clinical administration of eliglustat and miglustat, clinicians need\n                        to monitor the effects of adverse events varied by gender and to pay more\n                        attention to new adverse event signals.<\/jats:p>","DOI":"10.1055\/a-2791-0972","type":"journal-article","created":{"date-parts":[[2026,2,5]],"date-time":"2026-02-05T23:50:40Z","timestamp":1770335440000},"page":"82-90","source":"Crossref","is-referenced-by-count":0,"title":["Adverse Event Profile Differences Between Eliglustat and Miglustat: A\n                    Pharmacovigilance Study using the U.S. Food and Drug Administration Adverse\n                    Event Reporting System"],"prefix":"10.1055","volume":"76","author":[{"given":"Ya","family":"Gan","sequence":"additional","affiliation":[{"name":"Department of Pharmacy, Guang\u2019an People\u2019s Hospital, Sichuan,\n                    China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jie","family":"Liu","sequence":"additional","affiliation":[{"name":"Department of Pharmacy, Chongqing University Jiangjin Hospital,\n                    Chongqing, China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3855-7969","authenticated-orcid":false,"given":"Chunyong","family":"Xia","sequence":"additional","affiliation":[{"name":"Department of Pharmacy, Chongqing University Jiangjin Hospital,\n                    Chongqing, China"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"194","published-online":{"date-parts":[[2026,2,5]]},"reference":[{"issue":"02","key":"ref1","doi-asserted-by":"crossref","first-page":"107","DOI":"10.1080\/17446651.2018.1445524","article-title":"Recent advances in the diagnosis and management of Gaucher disease","volume":"13","author":"S E Gary","year":"2018","journal-title":"Expert Rev Endocrinol Metab"},{"key":"ref2","doi-asserted-by":"crossref","first-page":"13","DOI":"10.1182\/asheducation.V2012.1.13.3797921","article-title":"Gaucher disease and other storage disorders","volume":"2012","author":"G A Grabowski","year":"2012","journal-title":"Hematology Am Soc Hematol Educ Program"},{"issue":"02","key":"ref3","doi-asserted-by":"crossref","first-page":"441","DOI":"10.3390\/ijms18020441","article-title":"A review of Gaucher disease pathophysiology,clinical presentation and\n                    treatments","volume":"18","author":"J Stirnemann","year":"2017","journal-title":"Int J Mol Sci"},{"issue":"01","key":"ref4","doi-asserted-by":"crossref","first-page":"85","DOI":"10.3390\/jcm12010085","article-title":"Global incidence and prevalence of Gaucher Disease: A targeted literature\n                    review","volume":"12","author":"G Castillon","year":"2022","journal-title":"J Clin Med"},{"issue":"77","key":"ref5","first-page":"273","article-title":"Gaucher disease: insights from a rare Mendelian disorder","volume":"14","author":"E Sidransky","year":"2012","journal-title":"Discov Med"},{"issue":"14","key":"ref6","doi-asserted-by":"crossref","first-page":"7699","DOI":"10.3390\/ijms22147699","article-title":"Impact of long-term enzyme replacement therapy on glucosylsphingosine (Lyso-Gb1)\n                    values in patients with Type 1 Gaucher disease: Statistical models for comparing\n                    three enzymatic formulations","volume":"22","author":"T Dinur","year":"2021","journal-title":"Int J Mol Sci"},{"issue":"02","key":"ref7","doi-asserted-by":"crossref","first-page":"275","DOI":"10.1023\/A:1010335505357","article-title":"Inhibition of substrate synthesis as a strategy for glycolipid lysosomal storage\n                    disease therapy","volume":"24","author":"F M Platt","year":"2001","journal-title":"J Inherit Metab Dis"},{"issue":"9214","key":"ref8","doi-asserted-by":"crossref","first-page":"1481","DOI":"10.1016\/S0140-6736(00)02161-9","article-title":"Novel oral treatment of Gaucher\u2019s disease with N-butyldeoxynojirimycin\n                    (OGT 918) to decrease substrate biosynthesis","volume":"355","author":"T Cox","year":"2000","journal-title":"Lancet"},{"issue":"04","key":"ref9","doi-asserted-by":"crossref","first-page":"274","DOI":"10.1016\/j.bcmd.2014.04.002","article-title":"Eliglustat, an investigational oral therapy for Gaucher disease type 1: Phase 2\n                    trial results after 4 years of treatment","volume":"53","author":"E Lukina","year":"2014","journal-title":"Blood Cells Mol Dis"},{"issue":"07","key":"ref10","doi-asserted-by":"crossref","first-page":"695","DOI":"10.1001\/jama.2015.459","article-title":"Effect of oral eliglustat on splenomegaly inpatients with Gaucher disease type\n                    1: the ENGAGE randomized clinical trial","volume":"313","author":"P K Mistry","year":"2015","journal-title":"JAMA"},{"issue":"1\/2","key":"ref11","doi-asserted-by":"crossref","first-page":"56","DOI":"10.1016\/j.bcmd.2014.02.006","article-title":"Impact of velaglucerase alfa on bone marrow burden score in adult patients with\n                    type 1 Gaucher disease: 7-year follow-up","volume":"53","author":"D Elstein","year":"2014","journal-title":"Blood Cells Mol Dis"},{"issue":"04","key":"ref12","first-page":"1012","article-title":"Long-term follow-up of 103 untreated adult patients with Type 1 Gaucher\n                    disease","volume":"9","author":"T Dinur","year":"2020","journal-title":"J Clin 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Remenova","year":"2015","journal-title":"Orphanet J Rare Dis"},{"issue":"03","key":"ref17","doi-asserted-by":"publisher","first-page":"567","DOI":"10.1002\/cpt.3701","article-title":"FDA adverse event reporting system (FAERS) essentials: A guide to understanding,\n                    applying, and interpreting adverse event data reported to FAERS","volume":"118","author":"E Potter","year":"2025","journal-title":"Clin Pharmacol Ther"},{"issue":"07","key":"ref18","first-page":"597","article-title":"Empirical estimation of under-reporting in the U.S. Food and drug administration\n                    adverse event reporting system (FAERS)","volume":"40","author":"Y M Alatawi","year":"2017","journal-title":"Drug Saf"},{"issue":"11","key":"ref19","doi-asserted-by":"crossref","first-page":"1105","DOI":"10.1016\/j.clinthera.2023.08.010","article-title":"Effectiveness and safety of eliglustat treatment in gaucher disease: Real-life\n                    unicentric 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