title: Treatment with Oral ATP decreases alternating hemiplegia of childhood with de novo ATP1A3 Mutation.
abstract: Alternating hemiplegia of childhood is an intractable neurological disorder characterized by recurrent episodes of alternating hemiplegia accompanied by other paroxysmal symptoms. Recent research has identified mutations in the ATP1A3 gene as the underlying cause. Adenosine-5'-triphosphate has a vasodilatory effect, can enhance muscle strength and physical performance, and was hypothesized to improve the symptoms of paroxysmal hemiplegia.
  
  
  
  A 7-year-old boy with alternating hemiplegia of childhood who was positive for a de novo ATP1A3 mutation was treated with adenosine- 5'- triphosphate supplementation orally as an innovative therapy for 2 years. Outcome was evaluated through the follow-up of improvement of hemiplegic episodes and psychomotor development. Side effects and safety were monitored in regularity.
  
  
  
  With the dosage of adenosine-5'-triphosphate administration increased, the patient showed significantly less frequency and shorter duration of hemiplegic episodes. Treatment with adenosine-5'-triphosphate was correlated with a marked amelioration of alternating hemiplegia of childhood episodes, and psychomotor development has improved. The maximum dose of oral administration of adenosine-5'-triphosphate reached 25 mg/kg per day. Adenosine-5'-triphosphate therapy was well tolerated without complaint of discomfort and side effects.
  
  
  
  The 2-year follow-up outcome of adenosine-5'-triphosphate therapy for alternating hemiplegia of childhood was successful.
authors.affiliation: Department of Pediatrics, Chinese PLA General Hospital, Beijing, 100853, China.|Department of Pediatrics, Beijing Chao-Yang Hospital, Chaoyang District, Beijing, 100020, China.
authors.forename: Jun
authors.initials: J
authors.lastname: Ju
authors.affiliation: Department of Pediatrics, School of Medicine, Fukuoka University, Fukuoka, Japan.
authors.forename: Shinichi
authors.initials: S
authors.lastname: Hirose
authors.affiliation: Department of Pediatrics, Chinese PLA General Hospital, Beijing, 100853, China.
authors.forename: Xiu-Yu
authors.initials: XY
authors.lastname: Shi
authors.affiliation: Department of Pediatrics, School of Medicine, Fukuoka University, Fukuoka, Japan.
authors.forename: Atsushi
authors.initials: A
authors.lastname: Ishii
authors.affiliation: Department of Pediatrics, Chinese PLA General Hospital, Beijing, 100853, China.
authors.forename: Lin-Yan
authors.initials: LY
authors.lastname: Hu
authors.affiliation: Department of Pediatrics, Chinese PLA General Hospital, Beijing, 100853, China. zouliping21@sina.com.|Center of Epilepsy, Beijing Institute for Brain Disorders, Beijing, 100069, China. zouliping21@sina.com.
authors.forename: Li-Ping
authors.initials: LP
authors.lastname: Zou
mesh_terms: D000255:Adenosine Triphosphate / Q000627:therapeutic use*; D002648:Child; D006429:Hemiplegia / Q000188:drug therapy* / Q000235:genetics*; D006801:Humans; D008297:Male; D009154:Mutation / Q000235:genetics*; D000254:Sodium-Potassium-Exchanging ATPase / Q000235:genetics*; D016896:Treatment Outcome
chemical_list: C488756:ATP1A3 protein, human; D000255:Adenosine Triphosphate; D000254:Sodium-Potassium-Exchanging ATPase
keywords: AHC; Epilepsy; Migraine