Log In

Forgot Password?
Create New Account

Loading... please wait

Abstract Details

Characteristics, Treatment Patterns, and Disease Burden of Juvenile Myasthenia Gravis in the United States
Neuromuscular and Clinical Neurophysiology (EMG)
P4 - Poster Session 4 (11:45 AM-12:45 PM)
11-017

Myasthenia gravis (MG) is a rare, chronic, autoimmune disease characterized by dysfunction and damage at the neuromuscular junction. JMG (MG in patients <18 years old) is very rare, with limited knowledge on current treatment patterns and HCRU.

To describe treatment patterns and healthcare resource utilization (HCRU) of patients with newly diagnosed juvenile myasthenia gravis (JMG).

JMG patients, newly diagnosed between 2008 and 2021, were identified from Merative™ Marketscan® Commercial Database and Multi-State Medicaid Database. Patients were followed from the date of first JMG claim (JMG diagnosis or treatment with acetylcholinesterase inhibitors [AChEIs], maintenance intravenous [IV] or subcutaneous immunoglobulin [Ig], or plasma exchange [PLEX] therapy, whichever occurred first). JMG-related treatment changes during follow-up were assessed by descriptive statistics. Exacerbations, myasthenic crisis, thymectomy, and acute use of IVIg/PLEX during follow-up were identified and HCRU evaluated.

630 newly diagnosed JMG patients (57.6% pre-puberty-onset; <12 years old) were followed for a median of 2.4 years (range 18 days–13 years). Of 533 treated patients, 375 (70.4%) were first treated with AChEIs initiated at a median of 2 days from first JMG claim; corticosteroids were started at a median of 66 days, followed by maintenance Ig/PLEX and non-steroidal immunosuppressants (NSISTs) at 133 and 240 days, respectively. Treatments for post-puberty-onset patients (≥12 years old) were escalated faster than for pre-puberty-onset patients. Thymectomy was most frequent during maintenance Ig/PLEX (29.1 per 100 person-years) and was more frequent in post-puberty-onset patients. Exacerbation peaked during first NSIST treatment (incidence rate 122.9 per 100 person-years); patients treated with maintenance Ig/PLEX had the highest rate of MG hospitalizations (113.9 per 100 person-years).

JMG patients were escalated rapidly through the treatment hierarchy, but continued to experience exacerbations and high HCRU, suggesting current treatments do not provide adequate disease control. This indicates a need for new, more effective treatment approaches in JMG.

Authors/Disclosures
Frank Tennigkeit, PhD (UCB Biosciences GmbH)
PRESENTER
Dr. Tennigkeit has received personal compensation for serving as an employee of UCB Biosciences. Dr. Tennigkeit has stock in UCB Biosciences.
Jiachen Zhou (UCB Pharma) No disclosure on file
SIGRID Maria NILIUS (UCB Biosciences GmbH) No disclosure on file
Olga Pilipczuk (UCB) No disclosure on file
Anna Scowcroft (UCB) No disclosure on file
Thais T. Heras, Sr., MD (UCB Pharma S.A) Dr. Heras has received personal compensation for serving as an employee of UCB Pharma S.A.
Piotr Zaremba No disclosure on file
John Brandsema, MD (Children's Hospital of Philadelphia, Division of Neurology) Dr. Brandsema has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Biogen. Dr. Brandsema has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Genentech. Dr. Brandsema has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for PTC Therapeutics. Dr. Brandsema has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sarepta. Dr. Brandsema has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Novartis. Dr. Brandsema has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Argenx. Dr. Brandsema has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Janssen. Dr. Brandsema has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dyne. Dr. Brandsema has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Fibrogen. Dr. Brandsema has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Takeda. Dr. Brandsema has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Edgewise. The institution of Dr. Brandsema has received research support from Novartis. The institution of Dr. Brandsema has received research support from Biogen. The institution of Dr. Brandsema has received research support from Alexion. The institution of Dr. Brandsema has received research support from CSL Behring. The institution of Dr. Brandsema has received research support from Pfizer. The institution of Dr. Brandsema has received research support from PTC Therapeutics. The institution of Dr. Brandsema has received research support from Sarepta. The institution of an immediate family member of Dr. Brandsema has received research support from Argenx. The institution of Dr. Brandsema has received research support from Astellas. The institution of Dr. Brandsema has received research support from Fibrogen. The institution of Dr. Brandsema has received research support from Genentech. The institution of Dr. Brandsema has received research support from Janssen. The institution of Dr. Brandsema has received research support from Scholar Rock.
Jonathan B. Strober, MD (UCSF) Dr. Strober has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. Dr. Strober has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Scholar Rock. Dr. Strober has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenyx. Dr. Strober has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Biogen. Dr. Strober has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Pediatric Neurology. Dr. Strober has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for Many Law Firms. The institution of Dr. Strober has received research support from Anonymous. The institution of Dr. Strober has received research support from PTC. The institution of Dr. Strober has received research support from Fibrogen. The institution of Dr. Strober has received research support from Jansen. The institution of Dr. Strober has received research support from Biohaven.