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Abstract Details

Factors Associated with Recurrent Clinical Events among Pediatric Generalized Myasthenia Gravis Patients in the United States
Child Neurology and Developmental Neurology
P8 - Poster Session 8 (11:45 AM-12:45 PM)
8-002

To estimate rates of exacerbation and myasthenic crisis and identify associated risk factors for crisis and repeat exacerbations among commercially-insured children with generalized myasthenia gravis (gMG) in the United States.

Clinical events in MG are costly and repeat events indicate insufficient disease management.

MG patients aged 2-17 years were identified from MarketScan® commercial insurance data (01/01/2010 to 06/30/2025) based on ≥1 inpatient or ≥2 outpatient claims (within 60-days) for MG and ≥6 months of continuous enrollment before the first qualified MG diagnosis (index date). Exacerbation and crisis rates were calculated separately for ages 2-11 and 12-17. To assess factors associated with crisis and repeat exacerbation, a nested case-control design considered patients with an event in the follow-up period as cases (corresponding event date as new case index date) and those without events as controls (date at the end of the follow-up as the new control index date). The 6-month period before the new index date was used to pre-select risk factors.

The study included 508 gMG patients (226 aged 2-11; 282 aged 12-17). Over 25% of 2-11 year-olds experienced ≥1 exacerbation and 15.5% experienced ≥1 crisis in the follow-up period; incidence rate (95% confidence interval [CI]) per 100 person-years was 18.2 (11.4–29.1) for exacerbation and 9.3 (5.1-17.1) for crisis. Among those 12-17, 34.8% and 16.7% experienced exacerbation and crisis; incidence rates 22.8 (16.9-30.1) and 7.3 (4.7-11.4). Logistic regression models found female, prior exacerbation, acetylcholinesterase inhibitors, corticosteroids, and intravenous immunoglobulin treatment as significantly associated with repeat exacerbations and crises.

Pediatric gMG patients had high rates of clinical events. Crisis and repeat exacerbation were associated with prior exacerbation and common MG treatments. This study highlights the burden of MG in this patient population and the need for improved treatments to reduce disease burden.

Authors/Disclosures
Jacqueline Pesa (Janssen)
PRESENTER
Jacqueline Pesa has received personal compensation for serving as an employee of Johnson and Johnson.
Zhiwen Liu, PhD Dr. Liu has received personal compensation for serving as an employee of Janssen Scientific Affairs, LLC,. Dr. Liu has stock in Janssen Scientific Affairs, LLC,.
Louis Jackson, PharmD (Janssen) Dr. Jackson has received personal compensation for serving as an employee of Johnson and Johnson.
Ning Lin, MD Dr. Lin has nothing to disclose.
Alicia Campbell, PharmD Dr. Campbell has received personal compensation for serving as an employee of Janssen Scientific Affairs, LLC, a Johnson & Johnson company. Dr. Campbell has stock in Johnson & Johnson.
Raghav Govindarajan, MD, FAAN (HSHS St. Elizabeth Medical Group) Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for MT pharma. Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx. Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Catalyst. Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche . Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amicus. Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB. Dr. Govindarajan has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Alexion. Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for MT pharma . Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Catalyst. Dr. Govindarajan has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Argenx. Dr. Govindarajan has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Biohaven. Dr. Govindarajan has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for UCB. Dr. Govindarajan has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Takeda. Dr. Govindarajan has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Abbvie. The institution of Dr. Govindarajan has received research support from Band of Hope . The institution of Dr. Govindarajan has received research support from Alexion. Dr. Govindarajan has received publishing royalties from a publication relating to health care.