Anna Kostera-Pruszczyk, MD, PhD, of the Medical University of Warsaw in Poland, and colleagues conducted a phase 2\/3 trial to evaluate the efficacy and safety of efgartigimod in pediatric patients with anti-acetylcholine receptor antibody\u2013positive (AChR-Ab+) generalized MG.\u00a0 The researchers presented the interim study findings at the 2026 European Academy of Neurology Congress.<\/p>\n
The study involved two juvenile patients aged 2 to 17 years. The study used a staggered design.<\/p>\n
In part A, the researchers collected data on patient response to a single confirmatory dose for 8 weeks or longer.\u00a0 In part B, the study team also evaluated the efficacy of a multiple-dose treatment response for 18 weeks or longer.<\/p>\n
The findings showed that efgartigimod use led to a decline in both AChR-Ab and overall IgG serum levels. Other data from part B of the study indicated that the average overall Myasthenia Gravis Activities of Daily Living score decreased by -3.5 from baseline to 1 month during cycle 1 and \u22124.0 during cycle 2.<\/p>\n
Notably, 80% of the cycle 2 cohort and roughly 73% of the cycle 1 group reached minimal symptom expression.<\/p>\n
\u201cEfgartigimod treatment was well tolerated in adolescents and resulted in decreased levels of both total IgG and AChR-? Abs, similar to those observed in adult studies. Clinically meaningful improvements in MG-? ADL and QMG occurred, with many participants achieving MSE. Efgartigimod may address an unmet need in juvenile gMG therapy,\u201d the researchers wrote.<\/p>\n","protected":false},"excerpt":{"rendered":"Anna Kostera-Pruszczyk, MD, PhD, of the Medical University of Warsaw in Poland, and colleagues conducted a phase 2\/3…\n","protected":false},"author":2,"featured_media":652518,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[10],"tags":[163,85,46],"class_list":["post-652517","post","type-post","status-publish","format-standard","has-post-thumbnail","category-health","tag-health","tag-il","tag-israel"],"_links":{"self":[{"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/posts\/652517","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/comments?post=652517"}],"version-history":[{"count":0,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/posts\/652517\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/media\/652518"}],"wp:attachment":[{"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/media?parent=652517"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/categories?post=652517"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/tags?post=652517"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}