3,4-Diaminopyridine in Childhood Myasthenia: Double-Blind, Placebo-Controlled Trial

• Published in: Journal of child neurology Vol. 11; no. 6; pp. 458 - 461
• Main Authors: Anlar, Banu, Varli, Kubilay, Ozdirim, Emire, Ertan, Mevlut
• Format: Journal Article
• Language: English
• Published: Thousand Oaks, CA Sage Publications 01.11.1996; SAGE PUBLICATIONS, INC
• Subjects: 4-Aminopyridine - analogs & derivatives; 4-Aminopyridine - therapeutic use; Adolescent; Adult; Child; Child, Preschool; Cross-Over Studies; Double-Blind Method; Electromyography - drug effects; Female; Humans; Male; Myasthenia Gravis - congenital; Myasthenia Gravis - diagnosis; Myasthenia Gravis - drug therapy; Neurologic Examination - drug effects
• ISSN: 0883-0738; 1708-8283
• DOI: 10.1177/088307389601100608

Eleven patients with congenital and five with juvenile myasthenia gravis, aged 5 to 24 years, were given 3,4-diaminopyridine in a double-blind, placebo-controlled, crossover study. Clinical improvement was observed in 5 of 11 congenital myasthenia patients, and placebo effect, in 3 of 11. Juvenile myasthenia patients did not respond. Single-fiber electromyographic studies did not reveal any changes correlating with the clinical status of the patient. This study demonstrates the importance of double-blind and placebo-controlled studies to determine the effect of 3,4-diaminopyridine in congenital myasthenia. This drug may have different effects on various presynaptic and postsynaptic defects of neuromuscular transmission resulting in congenital myasthenia syndromes. (J Child Neurol 1996;11:458-461).