Pentosan Polysulfate: Oral Versus Subcutaneous Injection in Mucopolysaccharidosis Type I Dogs

Simonaro CM, Tomatsu S, Sikora T, Kubaski F, Frohbergh M, Guevara JM, Wang RY, Vera M, Kang JL, Smith LJ, Schuchman EH, Haskins ME · PLoS One

A head-to-head of the two routes at matched dose in a large animal. Both cut inflammatory cytokines and tissue glycosaminoglycan storage and improved arterial pathology, but the reductions were most evident after subcutaneous dosing, and only the subcutaneous route significantly lowered cytokines in cerebrospinal fluid. No liver-enzyme or coagulation abnormalities appeared on either route over a year or more.

Population

Dogs with mucopolysaccharidosis type I, treated 12–17 months

Intervention

Daily oral PPS or twice-weekly subcutaneous PPS, both at a human-equivalent 1.6 mg/kg

Comparator

Untreated MPS I dogs

Limitations

A lysosomal storage disease model, not osteoarthritis; endpoints are arterial and biochemical; group sizes are small, as they are in most large-animal genetic-disease work.