Intraventricular pentosan polysulphate in human prion diseases: an observational study in the UK.

ABSTRACT

UNLABELLED 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 microg/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.