Credulity of exploratory trials for cerebral cavernous malformations

Abstract

Cerebral cavernous malformations are vascular lesions that affect approximately 0·3% of the population and can cause haemorrhage, seizures, and neurological deficits. Familial cases with autosomal dominant disease, cases of symptomatic cerebral cavernous malformation, and cases with recent haemorrhage meet rare disease criteria according to regulatory agencies in the USA and EU. To date, the only treatment for people with cerebral cavernous malformations is resection of a symptomatic lesion or radiosurgery, but there is scant evidence of the effectiveness of these approaches, which can be associated with substantial morbidity.