The effect of pentosan polysulfate sodium for improving dyslipidaemia and knee pain in people with knee osteoarthritis: A pilot study

OBJECTIVE: To evaluate the efficacy and safety of pentosan polysulfate sodium (PPS, Elmiron®) for dyslipidaemia and knee osteoarthritis (OA) related symptoms. METHOD: This was a single-arm, open-label, prospective, non-randomised pilot study. People with painful knee OA and a history of primary hypercholesterolemia were included. PPS was taken orally in a dosage of 10 ​mg/kg once every 4 days for 5 weeks for two cycles. There was 5 weeks of no medication between the cycles. The main outcomes included the change in lipidemia levels, the change in knee OA-related symptoms assessed by pain numerical rating scale (NRS) and Knee Osteoarthritis Outcome Score (KOOS), and knee MRI semi-quantitative score. The changes were analysed using paired t-tests. RESULTS: 38 participants were included, with a mean age of 62.2 years. We found a statistically significant decrease in total cholesterol (from 6.23 ​± ​0.74 to 5.95 ​± ​0.77 ​mmol/L; P ​= ​0.01) and low-density lipoprotein (from 4.03 ​± ​0.61 to 3.82 ​± ​0.61 ​mmol/L; P ​= ​0.009) from baseline to week 16. Knee pain NRS was significantly reduced at weeks 6, 16 and 26 from 6.39 ​± ​1.33 to 4.18 ​± ​1.99, 3.63 ​± ​2.28 and 4.38 ​± ​2.55, respectively (P ​< ​0.001). However, there was no significant difference in terms of the primary outcome of triglyceride levels before and after treatment. The most common AEs were positive faecal occult blood tests, followed by headache and diarrhoea. CONCLUSION: The findings suggest that PPS has promising effects on improving dyslipidaemia and symptomatic pain relief in people with knee OA.