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.