Pediatric Von Hippel-Lindau Disease: Natural History, Predictive Factors, and Long-Term Functional Outcomes of Central Nervous System Hemangioblastomas

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Conditions studied: Von Hippel-Lindau Disease, Central Nervous System Hemangioblastoma

In brief

Von Hippel-Lindau (VHL) disease is a rare hereditary cancer predisposition syndrome associated with the development of central nervous system hemangioblastomas from childhood. The natural history of these lesions in pediatric patients remains poorly characterized, particularly regarding the factors that predict progression from radiological surveillance to neurosurgical intervention. This multicenter retrospective observational study aims to identify clinical, radiological, and genetic predictors of surgical indication in children with VHL-associated CNS hemangioblastomas and to evaluate their long-term neurological and functional outcomes. The findings may contribute to optimizing surveillance strategies and improving clinical decision-making in this rare population.

Key facts

Study ID

NCT07705529

Run by

Assistance Publique - Hôpitaux de Paris

People needed

25

Starts

2026-07-15

Expected to finish

2027-07-15

Last updated by the study team

2026-07-15

Who can join

Age: any, up to 18. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Age under 18 years at diagnosis of Von Hippel-Lindau disease
• Presence of at least one central nervous system hemangioblastoma
• Available clinical, radiological and genetic data

You may not qualify if…

• Insufficient follow-up data to assess clinical or radiological progression
• Opposition from the child or his/her parents

Where it is running

• Hôpital Roger Salengro, CHU Lille — Lille, France
• Hôpital Femme Mère Enfant, HCL — Lyon, France
• Hôpital Necker — Paris, France

Full record on ClinicalTrials.gov

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