title: Acetazolamide for the prophylaxis of migraine in CADASIL: a preliminary experience.
abstract: Cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy (CADASIL) is an inherited microangiopathy caused by NOTCH3 mutations. It is characterized by migraine, with or without aura, ischemic events, psychiatric and cognitive disturbances. There is no approved treatment for migraine prophylaxis in CADASIL, but acetazolamide has been anecdotally reported to be effective. We retrospectively reviewed our database of patients with a genetic diagnosis of CADASIL to identify how many of them were treated with acetazolamide for the prophylaxis of migraine. The efficacy and the tolerability of this treatment were checked looking at the clinic reports. Acetazolamide was prescribed in seven patients; the mean duration of treatment was 6 months, and the daily dose ranged from 125 to 500 mg. Three patients had a total and sustained remission, while in two patients a reduction in attacks and an improvement of the headache intensity were recorded. In one of these, acetazolamide was deliberately taken only during the migraine attack and the beneficial effect started 1 h after administration. In two patients, the drug did not produce any beneficial effect. Mild side effects were recorded in two patients. Our preliminary experience expands previous reports and confirms the possible efficacy of acetazolamide in CADASIL migraine. Based on these data, a randomized controlled trial seems worthy to be carried out to test the efficacy and safety of this drug.
authors.affiliation: Department of Neurological and Psychiatric Sciences, University of Florence, Largo Brambilla 3, Florence, Italy.
authors.forename: Ida
authors.initials: I
authors.lastname: Donnini
authors.forename: Serena
authors.initials: S
authors.lastname: Nannucci
authors.forename: Raffaella
authors.initials: R
authors.lastname: Valenti
authors.forename: Francesca
authors.initials: F
authors.lastname: Pescini
authors.forename: Silvia
authors.initials: S
authors.lastname: Bianchi
authors.forename: Domenico
authors.initials: D
authors.lastname: Inzitari
authors.forename: Leonardo
authors.initials: L
authors.lastname: Pantoni
mesh_terms: D000086:Acetazolamide / Q000627:therapeutic use*; D000328:Adult; D000368:Aged; D000927:Anticonvulsants / Q000627:therapeutic use*; D046589:CADASIL / Q000150:complications*; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D008881:Migraine Disorders / Q000209:etiology* / Q000517:prevention & control*; D012189:Retrospective Studies
chemical_list: D000927:Anticonvulsants; D000086:Acetazolamide