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Observations in patients with cavernous malformations of the brain, as well as in preclinical models of this disease, suggest that β the receptor blocker propranolol may reduce the risk of intracerebral hemorrhage.
Academics from Italy aimed to assess the safety and efficacy of long-term treatment with propranolol to reduce the incidence
of symptomatic intracerebral hemorrhage or focal neurological dysfunction in patients with familial cavernous malformations.
The researchers conducted a randomized, open-label, double-blind, Phase II trial (Treat_CCM)
at six national rare disease reference centers in Italy.
Patients aged 18 years or older with symptomatic familial cavernous malformation were randomly assigned (2:1) to receive oral propranolol (20-320 mg per day) plus standard care (intervention group), or standard care alone (control group) for 24 months
.
Clinical evaluation and 3TMRI studies were performed at baseline, 12 months, and 24 months, and the primary results were symptomatic intra-brain hemorrhage or focal neurological deficit
newly developed within 24 months that could be attributed to cavernous malformations.
Between 11 April 2018 and 5 December 2019, 95 people were assessed for eligibility and 83 were selected, of whom 57 were assigned to propranolol plus standard care and 26 to standard care alone
.
The mean age of participants was 46 years (SD 15); Forty-eight (58%) were female and 35 (42%) were male
.
The incidence of symptomatic intracerebral haemorrhage or focal neurologic deficit was 1.
7 per 100 person-years (95% CI 1.
4 to 2.
0) in the standard care group and 3.
9 per 100 person-years (3.
1 to 4.
7) in the standard care group alone (univariate HR = 0.
43, 80% CI 0.
18 to 0.
98).
According to the predetermined criteria, the results suggest that propranolol shows efficacy
.
There was no difference
in the incidence of hospitalization between groups.
In summary, propranolol is safe in this population and well tolerated
.
Propranolol may be beneficial in reducing the rate of
clinical events in patients with symptomatic familial cavernous malformations.
References:
Safety and efficacy of propranolol for treatment of familial cerebral cavernous malformations (Treat_CCM): a randomised, open-label, blinded-endpoint, phase 2 pilot trial