Background, purpose and methods: This observational study assessed the effect of continuous intraventricular infusion of pentosan polysulphate (PPS) in seven patients at different clinical centres in the UK.
Results: Complications of intraventricular catheterization were frequent. PPS was well-tolerated over a wide dose range (11–110 μg/kg/day) during the 6-month study. Four patients were assessed for the entire study period: one remained stable, two showed minimal deterioration and one progressed significantly.
Conclusion: Mean survival of all patients was longer than reported values for natural history of specific prion disorders. Possible reasons for these findings are explored.