Propranolol (beta-blocker), is successfully used for the treatment of infantile hemangiomas,
the most common vascular tumor of newborns. The mechanism is related to its anti-angiogenetic
and pro-apoptotic effects. Recently, in vitro studies demonstrated that propranolol decreased
the expression of target genes of the HIF (hypoxia-inducible factor, of which the VHL gene is
the main regulator) pathway in hemangioblastoma cells and affected their viability. The
efficacy of propranolol (stabilization of all HB and decrease in serum VEGF levels) was
demonstrated in a phase III study, but only in retinal BHs . The only study that evaluated
the effect of propranolol on CNS HB was retrospective and involved a limited number of
patients. Nevertheless, it showed a decrease in the growth rate of HBs. The investigator
therefore propose to carry out a randomized controlled trial to study the effect of
propranolol on the growth of CNS HB in patients with VHL disease (von Hippel-Lindau).
The hypothesis of the present work is the following: the use of propranolol in VHL patients
with CNS HB allows to decrease and/or slow down the tumor growth.
Phase:
N/A
Details
Lead Sponsor:
Assistance Publique - Hôpitaux de Paris
Collaborator:
Agence Générale des Equipements et Produits de Santé