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.