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.