Pyridostigmine Treatment for Pediatric Axonal Guillain–Barré Syndrome

INTRODUCTION/AIMS: Direct involvement of the neuromuscular junction (NMJ) in the inflammatory process of Guillain-Barré syndrome (GBS) has been described. Despite this, the NMJ very rarely serves as a target for direct medical intervention in GBS. Here, we report the use of an acetylcholinesterase inhibitor, pyridostigmine, in four pediatric patients with axonal GBS. METHODS: All patients received standard immune therapy. Pyridostigmine treatment was started 7 weeks to 5 months after disease onset, and 5-11 weeks from reaching the plateau phase. Treatment efficacy was monitored by the 6-min walk, quadriceps femoris and hamstring strength (manual muscle testing), the pediatric evaluation of disability inventory-functional skills, and the GBS disability score. All tests were performed before and during drug intervention. RESULTS: All treated patients showed marked improvement in their motor and functional abilities. After 1 month of treatment, quadriceps femoris and hamstring strength increased by at least two points, and walking distance increased by 10-272 m. The pediatric evaluation of disability inventory functional skills mobility test increased by 22.3-34.7 points. No serious side effects were documented. DISCUSSION: Pyridostigmine may be a safe and effective add-on treatment in pediatric patients with axonal GBS who show insufficient response to immune therapy, and may be effectively used even at a late stage. Additional, larger studies are needed.