title: Movement disorder in ataxia-telangiectasia: treatment with amantadine sulfate.
abstract: Ataxia-telangiectasia is a cerebellar neurodegenerative disorder presenting with ataxia, chorea, myoclonus, and bradykinesia. Literature on treatment of movement disorders is scarce. We treated 17 children (aged 11.2 ± 3.9 years) for 8 weeks with the dopaminergic and anti-N-methyl-d-aspartate (NMDA) agent amantadine sulfate 6.3 ± 0.87 mg/kg/d. Ataxia was assessed by using the International Cooperative Ataxia Scale, parkinsonism by the Unified Parkinson Disease Rating Scale, and chorea/myoclonus by the Abnormal Involuntary Movement Scale. Responders were considered those patients who had at least 20% improvement in the summation of the 3 scales. Overall, 76.5% of patients were responders, with a mean 29.3% improvement. Ataxia, involuntary movements, and parkinsonism improved significantly (25.3%, 32.5%, and 29.5%, respectively); (P < .001, t test). Side effects were mild and transient, and they did not lead to drug discontinuation. Amantadine is a well-tolerated and effective treatment for motor symptoms in ataxia telangiectasia. Assessment of long-term effects and a double-blind study should follow.
authors.affiliation: The National A-T Clinic, Edmond and Lilly Safra Children's Hospital, Sheba Medical Center, Ramat Gan, Israel. Andreea.Nissenkorn@sheba.health.gov.il
authors.forename: Andreea
authors.initials: A
authors.lastname: Nissenkorn
authors.forename: Sharon
authors.initials: S
authors.lastname: Hassin-Baer
authors.forename: Sheera F
authors.initials: SF
authors.lastname: Lerman
authors.forename: Yonit Banet
authors.initials: YB
authors.lastname: Levi
authors.forename: Michal
authors.initials: M
authors.lastname: Tzadok
authors.forename: Bruria
authors.initials: B
authors.lastname: Ben-Zeev
mesh_terms: D000293:Adolescent; D000547:Amantadine / Q000627:therapeutic use*; D000978:Antiparkinson Agents / Q000627:therapeutic use*; D001260:Ataxia Telangiectasia / Q000150:complications*; D002648:Child; D002675:Child, Preschool; D004185:Disability Evaluation; D006801:Humans; D009069:Movement Disorders / Q000188:drug therapy* / Q000209:etiology*; D009460:Neurologic Examination; D011446:Prospective Studies; D012720:Severity of Illness Index
chemical_list: D000978:Antiparkinson Agents; D000547:Amantadine