BPS symptom load during pentosane polysulfate sodium treatment in real life in France.

RATIONAL: Oral pentosane polysulfate sodium (PPS) is a symptomatic treatment for bladder pain syndrome (BPS), a rare, chronic and incurable inflammatory disease of the bladder leading to pelvic pain, frequent micturition, urgency, nocturia and reduced quality of life (QoL). As population, treatment practice and outcomes are insufficiently understood in France, data were collected in line with French Health authorities.

METHODOLOGY: ELMIRON was a national, multicenter, prospective, uncontrolled, observational study aiming to include up to 100 patients. Patients receiving PPS in clinical routine under ANSM were observed until discontinuation or study end. Endpoints included drug survival, demographics, BPS symptoms, QoL, and safety.

RESULTS

Ninety-five patients were included, i.e., up to 29% of the BPS population in France under PPS treatment. Among the patients, 92.6% were female, with an age of 20-87years. Among the patients, 83.2% reported at least one medical history. On average, 9.4±7.7years had passed since first symptoms, all but one patient had received pre-treatment, 47.4% were PPS naive. Patients started treatment with 2.9 doses of 100mg PPS, subsequently reduced to 2.4. After baseline, 58.3% reported improvement of pain, especially bladder pain, urinary frequency and of the O'Leary Sant problem score. Associated diseases and QoL changed little. Thirty-six patients (37.9%) experienced 71 new findings, one unrelated finding was assessed as serious by the investigator.

CONCLUSION

Safety of PPS was confirmed. PPS treatment was associated with improvement of BPS symptoms like urinary frequency, pain and patient perception of BPS-related problems. Monitoring every 6months was confirmed to enable timely treatment modifications.

LEVEL OF EVIDENCE: Low.