Recurrent multiple CNS hemangioblastomas with VHL disease treated with pazopanib: a case report and literature review.
Hemangioblastoma is a rare benign neoplasm, accounting for less than 2% of all primitive brain tumors. It may arise sporadically in a solitary form, or associated with Von Hippel-Lindau (VHL) disease with multiple tumors. Surgery is the mainstay treatment, but management is challenging in case of recurrent and/or multiple tumors. VHL protein is defective in both forms of hemangioblastoma, leading to the accumulation of hypoxia-inducible factor, stimulating angiogenesis via VEGF and PDGF mainly. Here, we report a 37-year-old woman's case with recurrent and rapidly progressive VHL-associated hemangioblastomas, causing severe disability. She was treated 24 months with pazopanib, a multityrosine kinase inhibitor (TKI) targeting VEGF and PDGF-β pathways. Despite moderate radiological changes, progressive improvement in her clinical condition persisting over 3 years was observed. Inhibiting angiogenesis is a therapeutic option that may improve the quality of life and the autonomy of VHL patients disabled with multiple hemangioblastomas.
Centre of Oncology, University Hospital of Geneva, Geneva, Switzerland.
Denis
D
Migliorini
Department of Radiology & Medical Informatics, University Hospital of Geneva, Geneva, Switzerland.
Sven
S
Haller
Department of Pathology & Immunology, Division of Clinical Pathology, University Hospital of Geneva, Geneva, Switzerland.
Doron
D
Merkler
Centre of Oncology, University Hospital of Geneva, Geneva, Switzerland.
Angela
A
Pugliesi-Rinaldi
Department of Neurosurgery, University Hospital of Geneva, Geneva, Switzerland.
Avinash
A
Koka
Department of Neurosurgery, University Hospital of Geneva, Geneva, Switzerland.
Karl
K
Schaller
Centre of Oncology, University Hospital of Geneva, Geneva, Switzerland.
Beatrice
B
Leemann
Centre of Oncology, University Hospital of Geneva, Geneva, Switzerland.
Pierre-Yves
PY
Dietrich
D000328:Adult; D020533:Angiogenesis Inhibitors / Q000627:therapeutic use*; D001932:Brain Neoplasms / Q000150:complications / Q000188:drug therapy* / Q000473:pathology / Q000503:physiopathology; D002574:Cervical Vertebrae; D005260:Female; D005500:Follow-Up Studies; D018325:Hemangioblastoma / Q000150:complications / Q000188:drug therapy* / Q000473:pathology / Q000503:physiopathology; D006801:Humans; D007191:Indazoles; D008279:Magnetic Resonance Imaging; D009364:Neoplasm Recurrence, Local / Q000188:drug therapy / Q000473:pathology / Q000503:physiopathology; D011743:Pyrimidines / Q000627:therapeutic use*; D013449:Sulfonamides / Q000627:therapeutic use*; D006623:von Hippel-Lindau Disease / Q000150:complications / Q000188:drug therapy* / Q000473:pathology / Q000503:physiopathology
D020533:Angiogenesis Inhibitors; D007191:Indazoles; D011743:Pyrimidines; D013449:Sulfonamides; C516667:pazopanib
Von Hippel–Lindau disease; hemangioblastoma; pazopanib