Incidence and Prevalence of Juvenile Myasthenia Gravis in the United States Between 2010 and 2020: Analysis of Two Claims Databases.

IF 3.9 3区 医学 Q1 CLINICAL NEUROLOGY
Neurology and Therapy Pub Date : 2025-06-01 Epub Date: 2025-05-03 DOI:10.1007/s40120-025-00750-w
Jiachen Zhou, Anna Kuba, Sigrid Nilius, Olga Pilipczuk, Thaïs Tarancón, Frank Tennigkeit
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引用次数: 0

Abstract

Introduction: Few published population-based studies report the incidence or prevalence of juvenile myasthenia gravis (JMG) due to the rarity of the disorder. Despite recent progress in new targeted treatments and ongoing developments, there remains a critical need for novel and effective therapies specifically for JMG. Most treatments used for JMG are only approved for adult patients with MG. Thus, a thorough evaluation and understanding of the basic epidemiology of JMG is needed.

Methods: We conducted a population-based retrospective study to estimate the annual incidence and prevalence of JMG in the US from 2010 to 2020 by analyzing the Merative™ MarketScan® Commercial Claims and Encounters Database (CCAE) and Multi-State Medicaid Database (MDCD).

Results: The incidence of JMG in 2020 was 5.9 [95% confidence interval (CI) 3.3-9.7] per million person-years in CCAE and 8.7 (95% CI 6.0-12.3) per million person-years in MDCD, with considerable variation across the study period. The prevalence of diagnosed JMG remained fairly consistent, with 25.3 (95% CI 19.9-32.2) per million population in CCAE and 37.6 (95% CI 31.9-44.4) per million population in MDCD in 2020. Both databases consistently showed higher incidence and prevalence among girls compared with boys. No clear pattern was observed in incidence by age of onset over the study period, whereas prevalence generally increased with age. Both incidence and prevalence by age of onset were higher in MDCD than CCAE. Furthermore, higher incidence and prevalence were observed among Black population compared with White population. Overall, no obvious increasing or decreasing trend was observed during the study period.

Conclusion: The incidence and prevalence of JMG in the US, previously understudied, may be higher than earlier research suggested, possibly due to limited research into the epidemiology of JMG. This finding implies that the actual burden of JMG could be greater than previously estimated.

2010年至2020年美国青少年重症肌无力的发病率和患病率:两个索赔数据库的分析
由于青少年重症肌无力(JMG)的罕见性,很少有基于人群的研究报道其发病率或患病率。尽管最近在新的靶向治疗和持续发展方面取得了进展,但仍然迫切需要针对JMG的新颖有效的治疗方法。大多数用于JMG的治疗方法仅被批准用于成年MG患者。因此,有必要对JMG的基本流行病学进行全面的评价和了解。方法:我们进行了一项基于人群的回顾性研究,通过分析Merative™MarketScan®商业索赔和遭遇数据库(CCAE)和多州医疗补助数据库(MDCD),估计2010年至2020年美国JMG的年发病率和患病率。结果:2020年,CCAE的JMG发病率为5.9[95%置信区间(CI) 3.3-9.7] /百万人年,MDCD的JMG发病率为8.7 (95% CI 6.0-12.3) /百万人年,在整个研究期间变化很大。诊断为JMG的患病率保持相当一致,2020年CCAE为25.3 (95% CI 19.9-32.2) /百万人,MDCD为37.6 (95% CI 31.9-44.4) /百万人。这两个数据库一致显示,女孩的发病率和患病率高于男孩。在研究期间,没有观察到发病年龄之间发病率的明确模式,而患病率通常随着年龄的增长而增加。MDCD的发病率和发病年龄患病率均高于CCAE。此外,黑人的发病率和患病率高于白人。总体而言,研究期间未见明显的上升或下降趋势。结论:由于对JMG流行病学的研究有限,JMG在美国的发病率和患病率可能高于之前的研究结果。这一发现表明,JMG的实际负担可能比以前估计的要大。
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来源期刊
Neurology and Therapy
Neurology and Therapy CLINICAL NEUROLOGY-
CiteScore
5.40
自引率
8.10%
发文量
103
审稿时长
6 weeks
期刊介绍: Aims and Scope Neurology and Therapy aims to provide reliable and inclusive, rapid publication for all therapy related research for neurological indications, supporting the timely dissemination of research with a global reach, to help advance scientific discovery and support clinical practice. Neurology and Therapy is an international, open access, peer reviewed, rapid publication journal dedicated to the publication of high-quality clinical (all phases), observational, real-world and health outcomes research around the discovery, development, and use of neurological and psychiatric therapies, (also covering surgery and devices). Studies relating to diagnosis, pharmacoeconomics, public health, quality of life, and patient care, management, and education are also welcomed. The journal is of interest to a broad audience of healthcare professionals and publishes original research, reviews, case reports, trial designs, communications and letters. The journal is read by a global audience and receives submissions from all over the world. Neurology and Therapy will consider all scientifically sound research be it positive, confirmatory or negative data. Submissions are welcomed whether they relate to an international and/or a country-specific audience, something that is crucially important when researchers are trying to target more specific patient populations. This inclusive approach allows the journal to assist in the dissemination of all scientifically and ethically sound research. Rapid Publication The journal’s rapid publication timelines aim for a peer review decision within 2 weeks of submission. If an article is accepted, it will be published online 3-4 weeks from acceptance. These rapid timelines are achieved through the combination of a dedicated in-house editorial team, who closely manage article workflow, and an extensive Editorial and Advisory Board who assist with rapid peer review. This allows the journal to support the rapid dissemination of research, whilst still providing robust peer review. Combined with the journal’s open access model, this allows for the rapid and efficient communication of the latest research and reviews to support scientific discovery and clinical practice. Open Access All articles published by Neurology and Therapy are open access. Personal Service The journal’s dedicated in-house editorial team offer a personal “concierge service” meaning that authors will always have a personal point of contact able to update them on the status of their manuscript. The editorial team check all manuscripts to ensure that articles conform to the most recent COPE and ICMJE publishing guidelines. This supports the publication of ethically sound and transparent research. We also encourage pre-submission enquiries and are always happy to provide a confidential assessment of manuscripts. Digital Features and Plain Language Summaries Neurology and Therapy offers a range of additional features designed to increase the visibility, readership and educational value of the journal’s content. Each article is accompanied by key summary points, giving a time-efficient overview of the content to a wide readership. Articles may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand the scientific content and overall implications of the article. The journal also provides the option to include various types of digital features including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations. All additional features are peer reviewed to the same high standard as the article itself. If you consider that your paper would benefit from the inclusion of a digital feature, please let us know. Our editorial team are able to create high-quality slide decks and infographics in-house, and video abstracts through our partner Research Square, and would be happy to assist in any way we can. For further information about digital features, please contact the journal editor (see ‘Contact the Journal’ for email address), and see the ‘Guidelines for digital features and plain language summaries’ document under ‘Submission guidelines’. For examples of digital features please visit our showcase page https://springerhealthcare.com/expertise/publishing-digital-features/ Publication Fees Upon acceptance of an article, authors will be required to pay the mandatory Rapid Service Fee of €5250/$6000/£4300. The journal will consider fee discounts and waivers for developing countries and this is decided on a case-by-case basis. Peer Review Process Upon submission, manuscripts are assessed by the editorial team to ensure they fit within the aims and scope of the journal and are also checked for plagiarism. All suitable submissions are then subject to a comprehensive single-blind peer review. Reviewers are selected based on their relevant expertise and publication history in the subject area. The journal has an extensive pool of editorial and advisory board members who have been selected to assist with peer review based on the afore-mentioned criteria. At least two extensive reviews are required to make the editorial decision, with the exception of some article types such as Commentaries, Editorials and Letters which are generally reviewed by one member of the Editorial Board. Where reviews conflict, an Editorial Board Member will be contacted for further advice and a presiding decision. Manuscripts are then either accepted, rejected or authors are required to make major or minor revisions (both reviewer comments and editorial comments may need to be addressed. Once a revised manuscript is re-submitted, it is assessed along with the responses to reviewer comments and if it has been adequately revised, it will be accepted for publication. Accepted manuscripts are then copyedited and typeset by the production team before online publication. Appeals against decisions following peer review are considered on a case-by-case basis and should be sent to the journal editor, and authors are welcome to make rebuttals against individual reviewer comments, if appropriate. Preprints We encourage posting of preprints of primary research manuscripts on preprint servers, authors'' or institutional websites, and open communications between researchers whether on community preprint servers or preprint commenting platforms. Posting of preprints is not considered prior publication and will not jeopardize consideration in our journals. Please see here for further information on preprint sharing: https://www.springer.com/gp/authors-editors/journal-author/journal-author-helpdesk/submission/1302#c16721550 Copyright Neurology and Therapy is published under the Creative Commons Attribution-Noncommercial License, which allows users to read, copy, distribute, and make derivative works for non-commercial purposes from the material, as long as the author of the original work is cited. The author assigns the exclusive right to any commercial use of the article to Springer. For more information about the Creative Commons Attribution-Noncommercial License, click here: http://creativecommons.org/licenses/by-nc/4.0. Contact For more information about the journal, including pre-submission enquiries, please contact managing editor Lydia Alborn at lydia.alborn@springer.com.
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