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

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.