My 30‐year roadmap for pediatric myasthenia gravis in Hong Kong: Searching for international collaboration

Abstract

Pediatric myasthenia gravis (P‐MG) is an autoimmune disease affecting the neuromuscular junction (NMJ), and is rare in children. The roadmap of the research carried out for P‐MG in Hong Kong over the past 30 years was reviewed. Currently, there is a lack of epidemiological studies in comparing clinical data or the outcome for people of different ethnic origins. P‐MG is more common in Asians, especially Chinese and Japanese. Because of the lack of comparison studies for P‐MG worldwide, the author initiated collaboration with international colleagues in 2008, and set up “The International Paediatric Myasthenia Gravis Registry Focus Group (FG)” in 2009. We then established an “International Pediatric Myasthenia Gravis Registry (IPMGR)” to review the current knowledge of P‐MG in children. We designed an e‐MG questionnaire for “Myasthenia Syndromes in Children,” and launched through the website (ICNAPaedia) of the International Child Neurology Association (ICNA) in 2009–2010. As of 2014, we had collected 121 replies from clinicians–researchers in 43 countries. In 2013, we started a Delphi process for obtaining a consensus for diagnosis, management and treatment of P‐MG. Despite the lack of research funding support as a result of the rarity of P‐MG, and the lack of innovative pharmaceutical research and development, we still stress that we set up a foundation as a small step towards consolidating international collaboration to carry out epidemiological studies, genetic studies and randomized controlled trials of all the current available treatments, especially steroids for ocular P‐MG, and indications for thymectomy.

Oral corticosteroid therapy and present disease status in myasthenia gravis

“…Wong described the characteristics of pediatric MG and has attempted to collaborate with international researchers. 5 Other heterogenous aspects of MG are a result of the variety of autoantibodies present. Previous seronegative MG included patients harboring autoantibodies against muscle-specific receptor tyrosine kinase and lowdensity lipoprotein receptor-related protein 4.…”

What should we target? The beginning of a new era in the diagnosis and treatment of myasthenia gravis

“…However, there are not enough pediatric neurologists worldwide. Wong described the characteristics of pediatric MG and has attempted to collaborate with international researchers . Other heterogenous aspects of MG are a result of the variety of autoantibodies present.…”