Neelam Goyal , Cynthia Qi , John Stone , Tobias Ruck , Gil I. Wolfe , A. Gordon Smith , Deborah Gelinas , Matthew Jefferson , Edward Brauer , Tharun Balaji Suthagar , Rohit R. Menon , Mai Sato , Glenn Phillips , Pushpa Narayanaswami
{"title":"在全身性重症肌无力患者临床实践中,efgartigimod开始后口服糖皮质激素的使用减少","authors":"Neelam Goyal , Cynthia Qi , John Stone , Tobias Ruck , Gil I. Wolfe , A. Gordon Smith , Deborah Gelinas , Matthew Jefferson , Edward Brauer , Tharun Balaji Suthagar , Rohit R. Menon , Mai Sato , Glenn Phillips , Pushpa Narayanaswami","doi":"10.1016/j.jns.2025.123652","DOIUrl":null,"url":null,"abstract":"<div><div>This study evaluated changes in oral glucocorticoid (GC, prednisone equivalent) use among patients with generalized myasthenia gravis (gMG) previously using chronic GCs, who initiated and continued efgartigimod for ≥12 months, identified retrospectively from a US claims database. Myasthenia Gravis Activities of Daily Living (MG-ADL) scores were retrieved from a patient support program. The primary outcome was GC average daily dose (GC ADD) assessed during the 3 months before and 3, 6, 9, and 12 months after efgartigimod initiation. Overall, 266 patients were included (mean age: 59.8 years; 45 % female). Mean (95 % CI) GC ADD was significantly reduced at 3 (14.3 [12.6, 16.0] mg/day), 6 (13.4 [11.5, 15.4] mg/day), 9 (11.6 [10.0, 13.3] mg/day), and 12 (10.2 [8.8, 11.6] mg/day) months post-efgartigimod initiation compared to baseline (17.9 [15.7, 20.1] mg/day) (<em>p</em> < 0.001). At 12 months, 66 % (<em>n</em> = 176/266) reduced steroid dose. Among those, 68 % and 51 % achieved ≥50 % and ≥ 75 % ADD reduction, respectively. The proportion of patients using GC ADD of ≤5 mg/day increased from 13 % at baseline to 42 % at 12 months. Among 126 patients (47 %) who had MG-ADL scores available, there was a significant reduction from baseline in best follow-up mean (95 % CI) MG-ADL (from 7.9 [7.2, 8.5] to 3.1 [2.6, 3.6]), while mean GC ADD remained significantly reduced (<em>p</em> < 0.05). Thus, in patients with gMG using chronic GCs at baseline, GC use substantially reduced following efgartigimod initiation, while retaining improved MG-ADL scores, underscoring the potential steroid-sparing effect of efgartigimod in gMG treatment.</div></div>","PeriodicalId":17417,"journal":{"name":"Journal of the Neurological Sciences","volume":"477 ","pages":"Article 123652"},"PeriodicalIF":3.2000,"publicationDate":"2025-08-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Reduction in oral glucocorticoid use after efgartigimod initiation in clinical practice among patients with generalized myasthenia gravis\",\"authors\":\"Neelam Goyal , Cynthia Qi , John Stone , Tobias Ruck , Gil I. Wolfe , A. Gordon Smith , Deborah Gelinas , Matthew Jefferson , Edward Brauer , Tharun Balaji Suthagar , Rohit R. Menon , Mai Sato , Glenn Phillips , Pushpa Narayanaswami\",\"doi\":\"10.1016/j.jns.2025.123652\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><div>This study evaluated changes in oral glucocorticoid (GC, prednisone equivalent) use among patients with generalized myasthenia gravis (gMG) previously using chronic GCs, who initiated and continued efgartigimod for ≥12 months, identified retrospectively from a US claims database. Myasthenia Gravis Activities of Daily Living (MG-ADL) scores were retrieved from a patient support program. The primary outcome was GC average daily dose (GC ADD) assessed during the 3 months before and 3, 6, 9, and 12 months after efgartigimod initiation. Overall, 266 patients were included (mean age: 59.8 years; 45 % female). Mean (95 % CI) GC ADD was significantly reduced at 3 (14.3 [12.6, 16.0] mg/day), 6 (13.4 [11.5, 15.4] mg/day), 9 (11.6 [10.0, 13.3] mg/day), and 12 (10.2 [8.8, 11.6] mg/day) months post-efgartigimod initiation compared to baseline (17.9 [15.7, 20.1] mg/day) (<em>p</em> < 0.001). At 12 months, 66 % (<em>n</em> = 176/266) reduced steroid dose. Among those, 68 % and 51 % achieved ≥50 % and ≥ 75 % ADD reduction, respectively. The proportion of patients using GC ADD of ≤5 mg/day increased from 13 % at baseline to 42 % at 12 months. Among 126 patients (47 %) who had MG-ADL scores available, there was a significant reduction from baseline in best follow-up mean (95 % CI) MG-ADL (from 7.9 [7.2, 8.5] to 3.1 [2.6, 3.6]), while mean GC ADD remained significantly reduced (<em>p</em> < 0.05). Thus, in patients with gMG using chronic GCs at baseline, GC use substantially reduced following efgartigimod initiation, while retaining improved MG-ADL scores, underscoring the potential steroid-sparing effect of efgartigimod in gMG treatment.</div></div>\",\"PeriodicalId\":17417,\"journal\":{\"name\":\"Journal of the Neurological Sciences\",\"volume\":\"477 \",\"pages\":\"Article 123652\"},\"PeriodicalIF\":3.2000,\"publicationDate\":\"2025-08-11\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Journal of the Neurological Sciences\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S0022510X25002722\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"CLINICAL NEUROLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of the Neurological Sciences","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S0022510X25002722","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"CLINICAL NEUROLOGY","Score":null,"Total":0}
Reduction in oral glucocorticoid use after efgartigimod initiation in clinical practice among patients with generalized myasthenia gravis
This study evaluated changes in oral glucocorticoid (GC, prednisone equivalent) use among patients with generalized myasthenia gravis (gMG) previously using chronic GCs, who initiated and continued efgartigimod for ≥12 months, identified retrospectively from a US claims database. Myasthenia Gravis Activities of Daily Living (MG-ADL) scores were retrieved from a patient support program. The primary outcome was GC average daily dose (GC ADD) assessed during the 3 months before and 3, 6, 9, and 12 months after efgartigimod initiation. Overall, 266 patients were included (mean age: 59.8 years; 45 % female). Mean (95 % CI) GC ADD was significantly reduced at 3 (14.3 [12.6, 16.0] mg/day), 6 (13.4 [11.5, 15.4] mg/day), 9 (11.6 [10.0, 13.3] mg/day), and 12 (10.2 [8.8, 11.6] mg/day) months post-efgartigimod initiation compared to baseline (17.9 [15.7, 20.1] mg/day) (p < 0.001). At 12 months, 66 % (n = 176/266) reduced steroid dose. Among those, 68 % and 51 % achieved ≥50 % and ≥ 75 % ADD reduction, respectively. The proportion of patients using GC ADD of ≤5 mg/day increased from 13 % at baseline to 42 % at 12 months. Among 126 patients (47 %) who had MG-ADL scores available, there was a significant reduction from baseline in best follow-up mean (95 % CI) MG-ADL (from 7.9 [7.2, 8.5] to 3.1 [2.6, 3.6]), while mean GC ADD remained significantly reduced (p < 0.05). Thus, in patients with gMG using chronic GCs at baseline, GC use substantially reduced following efgartigimod initiation, while retaining improved MG-ADL scores, underscoring the potential steroid-sparing effect of efgartigimod in gMG treatment.
期刊介绍:
The Journal of the Neurological Sciences provides a medium for the prompt publication of original articles in neurology and neuroscience from around the world. JNS places special emphasis on articles that: 1) provide guidance to clinicians around the world (Best Practices, Global Neurology); 2) report cutting-edge science related to neurology (Basic and Translational Sciences); 3) educate readers about relevant and practical clinical outcomes in neurology (Outcomes Research); and 4) summarize or editorialize the current state of the literature (Reviews, Commentaries, and Editorials).
JNS accepts most types of manuscripts for consideration including original research papers, short communications, reviews, book reviews, letters to the Editor, opinions and editorials. Topics considered will be from neurology-related fields that are of interest to practicing physicians around the world. Examples include neuromuscular diseases, demyelination, atrophies, dementia, neoplasms, infections, epilepsies, disturbances of consciousness, stroke and cerebral circulation, growth and development, plasticity and intermediary metabolism.