TY - JOUR AU - Albiñana, Virginia AU - Escribano, Rosa María Jiménez AU - Soler, Isabel AU - Padial, Luis Rodríguez AU - Recio-Poveda, Lucia AU - Villar Gómez de las Heras, Karina AU - Botella, Luisa María PY - 2017 DA - 2017/06/29 TI - Repurposing propranolol as a drug for the treatment of retinal haemangioblastomas in von Hippel-Lindau disease JO - Orphanet Journal of Rare Diseases SP - 122 VL - 12 IS - 1 AB - Von Hippel-Lindau (VHL) disease is a rare oncological disease with an incidence of 1:36,000, and is characterized by the growth of different types of tumours. Haemangioblastomas in the central nervous system (CNS) and retina, renal carcinoma and pheochromocytomas are the most common tumours. The absence of treatment for VHL leads to the need of repeated surgeries as the only option for these patients. Targeting VHL-derived tumours with drugs with reduced side effects is urgent to avoid repeated CNS surgeries. Recent reports have demonstrated that propranolol, a β-blocker used for the treatment of hypertension and other cardiac and neurological diseases, is the best option for infantile hemangioma (IH). Propranolol could be an efficient treatment to control haemangioblastoma growth in VHL disease given its antiangiogenic effects that were recently demonstrated by us. The main objective of the present study was the assessment of the efficacy and safety of propranolol on retinal haemangioblastoma in von Hippel-Lindau disease (VHL). SN - 1750-1172 UR - https://doi.org/10.1186/s13023-017-0664-7 DO - 10.1186/s13023-017-0664-7 ID - Albiñana2017 ER -