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. 2025 Jul 8;105(1):e213736.
doi: 10.1212/WNL.0000000000213736. Epub 2025 Jun 9.

Treatment Patterns and Disease Burden of Juvenile Myasthenia Gravis in the United States: A Cohort Study Using Health Care Claims Databases

Jiachen Zhou  1 , Sigrid Nilius  2 , Olga Pilipczuk  3 , Anna Scowcroft  4 , Thaïs Tarancón  5 , Frank Tennigkeit  2 , Piotr Zaremba  3 , Nishtha Chandra  6 , Nancy Kuntz  7 , Jonathan Strober  8 , John Brandsema  9
Affiliations

Treatment Patterns and Disease Burden of Juvenile Myasthenia Gravis in the United States: A Cohort Study Using Health Care Claims Databases

Jiachen Zhou et al. Neurology. .

Abstract

Background and objectives: Juvenile myasthenia gravis (JMG) is a rare disorder defined as MG in patients younger than 18 years. Generalized JMG is more common in postpubertal than prepubertal patients. There are no formal international JMG treatment guidelines, and knowledge on treatment patterns and disease burden is limited. The aim of this study was to describe treatment patterns and health care resource utilization (HCRU) for patients with JMG and explore differences in disease presentation between prepubertal-onset (younger than 12 years) and postpubertal-onset (12-17 years) patients.

Methods: Patients with JMG, newly diagnosed from 2008 to 2021, were identified from the US Merative MarketScan® Research Databases. Patients were followed from the first JMG claim (diagnosis/treatment with acetylcholinesterase inhibitors, immunoglobulin [Ig], or plasma exchange [PLEX]). The primary outcome was JMG-related treatment changes during follow-up, assessed descriptively. Rates of MG exacerbation, thymectomy, and acute intravenous immunoglobulin/PLEX treatment were assessed. HCRU was evaluated.

Results: A total of 630 patients (64.1% female; mean [SD] age 9.07 [5.73] years; 57.6% prepubertal onset) were followed for a median (range) of 2.4 (0-13) years. Corticosteroids were started at a median (range) of 1.28 (0-37.02) and 3.19 (0-87.68) months from diagnosis for postpubertal-onset and prepubertal-onset patients, respectively. The rate of thymectomy was highest during treatment with maintenance Ig/PLEX (incidence rate [IR]; [95% CI] per 100 patient-years: 34.62 [14.41-83.17] for postpubertal-onset and 24.24 [9.10-64.60] for prepubertal-onset patients). MG exacerbations were most frequent during the first year of follow-up in both subgroups (34.1% and 30.3%). In postpubertal-onset patients, exacerbation was highest during treatment with maintenance Ig/PLEX and nonsteroid immunosuppressant therapy ([NSIST], mostly polytherapy) (IR [95% CI] 105.81 [68.99-162.29] and 91.22 [65.80-126.47]). For prepubertal-onset patients, exacerbation was most frequent during NSIST (polytherapy) and biologic treatment (IR [95% CI] 140.44 [115.45-170.85] and 142.95 [46.10-443.23]). JMG-related hospitalizations occurred in 36.0% and 30.0% of postpubertal-onset and prepubertal-onset patients, in the first year of follow-up.

Discussion: Patients with JMG escalated rapidly through the treatment hierarchy. Postpubertal-onset patients escalated more quickly to later-line treatments than prepubertal-onset patients. However, some patients continued to experience high HCRU, highlighting the need for new JMG treatments to provide rapid disease control. A limitation is that treatment escalation reasons were not evaluated.

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Conflict of interest statement

J. Zhou and S. Nilius are employees and shareholders of UCB. O. Pilipczuk is an employee of UCB. A. Scowcroft, T. Tarancón, F. Tennigkeit, and P. Zaremba are employees and shareholders of UCB. N. Chandra is an employee of Ogilvy Health, who provided medical writing support funded by UCB. N. Kuntz served on a scientific advisory board for Genentech and received institutional clinical trial research support from Biogen, Genentech, and Novartis Gene Therapies. J. Strober is a consultant for Momenta/Janssen Pharmaceuticals, UCB, Pfizer, Sarepta, Biogen, argenx, and Scholar Rock; is a speaker for Biogen and NS Pharma; and is a site investigator for PTC Therapeutics, FibroGen, Biohaven, Genentech/Roche, Janssen Pharmaceuticals, and Alexion Pharmaceuticals. J. Brandsema is a consultant for Alexion, Audentes (now Astellas), AveXis/Novartis, Biogen, Cytokinetics, Dyne, Edgewise, FibroGen, Genentech/Roche, Janssen Pharmaceuticals, Marathon, Momenta (now Johnson & Johnson), NS Pharma, PTC Therapeutics, Sarepta, Scholar Rock, Takeda, UCB, and WaVe; is a speaker for AveXis/Novartis and Biogen; is a medical advisory council member for Cure SMA; and is a site investigator for clinical trials with Alexion, Astellas, AveXis/Novartis, Biogen, Catabasis (Astria Therapeutics), CSL Behring, Cytokinetics, FibroGen, Genentech/Roche, Ionis, Pfizer, PTC Therapeutics, Sarepta, Scholar Rock, Summit, UCB, and WaVe. Go to Neurology.org/N for full disclosures.

Figures

Figure 1
Figure 1. Treatment Escalation Patterns in (A) Postpubertal-Onset Patients, (B) Prepubertal-Onset Patients, and (C) the Overall Population
Figure shows the movement of patients between treatments at any time during the period under analysis. To be counted in the “no treatment” cohort, patients must have remained without treatment for the whole of the first 3 months. Of the 630 patients initially identified, 1 did not receive any treatment but was followed for less than 3 months and so did not qualify for the “no treatment” cohort. This patient was, therefore, excluded, and all analyses involving treatment cohorts were based on 629 patients. AChEI = acetylcholinesterase inhibitor; CS = corticosteroid; Ig = immunoglobulin; JMG = juvenile myasthenia gravis; NSIST = nonsteroid immunosuppressant therapy; PLEX = plasma exchange; Tx = treatment.
Figure 2
Figure 2. (A) Time to Treatment Segment and (B) Time in Each Treatment Cohort From Index Date by Age at Onset of JMG and in the Overall Population
Of the 630 patients initially identified, 1 patient was followed for <3 months and was, therefore, excluded from these analyses. *Rituximab or eculizumab. AChEI = acetylcholinesterase inhibitor; CS = corticosteroid; Ig = immunoglobulin; IQR = interquartile range; JMG = juvenile myasthenia gravis; NSIST = nonsteroid immunosuppressant therapy; PLEX = plasma exchange; Tx = treatment.
Figure 3
Figure 3. Incidence of Thymectomy in Each Treatment Cohort by Age at Onset of JMG and in the Overall Population
Of the 630 patients initially identified, 1 patient was followed for <3 months and was, therefore, excluded from these analyses. *Rituximab or eculizumab. AChEI = acetylcholinesterase inhibitor; CS = corticosteroid; Ig = immunoglobulin; JMG = juvenile myasthenia gravis; NSIST = nonsteroid immunosuppressant therapy; PLEX = plasma exchange; PY = patient-year; Tx = treatment.
Figure 4
Figure 4. (A) JMG-Related Exacerbation and (B) Myasthenic Crises in Each Treatment Cohort by Age at Onset of JMG and in the Overall Population
Of the 630 patients initially identified, 1 patient was followed for <3 months and was, therefore, excluded from these analyses. *Rituximab or eculizumab. AChEI = acetylcholinesterase inhibitor; CS = corticosteroid; Ig = immunoglobulin; IR = incidence rate; JMG = juvenile myasthenia gravis; NSIST = nonsteroid immunosuppressant therapy; PLEX = plasma exchange; PY = patient-year; Tx = treatment.
Figure 5
Figure 5. (A) IVIg and (B) PLEX Use as Rescue Therapy in Each Treatment Cohort by Age at Onset of JMG and in the Overall Population
Of the 630 patients initially identified, 1 patient was followed for <3 months and was, therefore, excluded from these analyses. *Rituximab or eculizumab. AChEI = acetylcholinesterase inhibitor; CS = corticosteroid; Ig = immunoglobulin; IR = incidence rate; IVIg = IV immunoglobulin; JMG = juvenile myasthenia gravis; NSIST = nonsteroid immunosuppressant therapy; PLEX = plasma exchange; PY = patient-year; Tx = treatment.
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