{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,20]],"date-time":"2026-03-20T08:35:16Z","timestamp":1773995716255,"version":"3.50.1"},"reference-count":38,"publisher":"MDPI AG","issue":"6","license":[{"start":{"date-parts":[[2026,3,18]],"date-time":"2026-03-18T00:00:00Z","timestamp":1773792000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JCM"],"abstract":"<jats:p>Background\/Objectives: Eliglustat is an oral therapy for Gaucher disease type 1 (GD1) that may reduce infusion-related logistical burden, particularly in resource-constrained settings. Post-approval evidence from routine clinical practice in China remains limited. This study evaluated its real-world effectiveness and safety in Chinese adults with GD1. Methods: This retrospective, multicenter study included adults with GD1 receiving eliglustat monotherapy for \u22656 months. Outcomes included plasma glucosylsphingosine (lyso-Gb1), hemoglobin (HGB), platelet count (PLT), liver and spleen volumes, and adverse events (AEs). Depending on distribution, paired changes were analyzed using paired t tests or Wilcoxon signed-rank tests. p &lt; 0.05 was considered statistically significant. Results: Nineteen patients were included in the effectiveness analysis, with a median follow-up of 7 months (range, 6\u20139). Lyso-Gb1 decreased from 468 to 210 ng\/mL (p &lt; 0.0001). HGB increased from 123 to 131 g\/L (p = 0.147); among six patients with baseline anemia, 83.3% improved and 33.3% normalized. PLT increased from 109 to 132 \u00d7 109\/L (p = 0.019); among 12 patients with baseline thrombocytopenia, 58.3% improved. Liver volume decreased from 1808 to 1747 mL (p = 0.016) (1.22 to 1.01 multiples of normal; p &lt; 0.001). Spleen volume decreased from 473 to 452 mL (p = 0.016) (4.69 to 5.17 multiples of normal; p = 0.015). Lyso-Gb1 reduction was greater in patients without prior enzyme replacement therapy (ERT) exposure than in those with prior ERT exposure (\u221255.1% vs. \u221243.1%; p = 0.049). In the safety analysis group (n = 90), suspected drug-related AEs occurred in 27.8% of patients, mainly gastrointestinal or skin-related, and were limited to grade I\/II. No serious AE or treatment discontinuation occurred. Conclusions: In routine clinical practice in China, eliglustat was associated with rapid substantial reductions in plasma lyso-Gb1, early improvements in hematologic and visceral parameters, and favorable short-term tolerability in adults with GD1.<\/jats:p>","DOI":"10.3390\/jcm15062323","type":"journal-article","created":{"date-parts":[[2026,3,18]],"date-time":"2026-03-18T11:14:14Z","timestamp":1773832454000},"page":"2323","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Real-World Effectiveness and Safety of Eliglustat in Adult Patients with Gaucher Disease Type 1: A Multicenter Retrospective Study in China"],"prefix":"10.3390","volume":"15","author":[{"ORCID":"https:\/\/orcid.org\/0009-0006-2949-419X","authenticated-orcid":false,"given":"Yongxin","family":"Zhou","sequence":"first","affiliation":[{"name":"Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China"},{"name":"Eight-Year Medical Doctor Program, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Zijian","family":"Hao","sequence":"additional","affiliation":[{"name":"Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China"},{"name":"Eight-Year Medical Doctor Program, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Qilin","family":"Zhuang","sequence":"additional","affiliation":[{"name":"Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China"},{"name":"Eight-Year Medical Doctor Program, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9970-0733","authenticated-orcid":false,"given":"Bing","family":"Han","sequence":"additional","affiliation":[{"name":"Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"1968","published-online":{"date-parts":[[2026,3,18]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","unstructured":"Castillon, G., Chang, S.C., and Moride, Y. 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