泌尿道感染是許多女性朋友常見的困擾。一般早期的泌尿道感染，患者常會有小便灼熱、頻尿、急尿等症狀，較嚴重一點的則可能伴隨尿失禁、血尿或發燒的情形。女性無分年齡、種族、教育或社經地位都有很高的比例受此疾病影響。根據統計女性終其一生有高達60%的機會可能遭受泌尿道感染之苦，而這些受害者中又有約20~40%的人還會有再次泌尿道感染的機會。這個問題雖然大部分不至於危及性命但卻會對個人及整個社會造成不小的成本損失。

反覆的泌尿道感染是某些女性揮之不去的夢魘，如果半年內有兩次以上的泌尿道感染或是一年內有三次以上的泌尿道感染就可視為反覆的泌尿道感染。造成反覆的泌尿道感染原因相當複雜，涉及生物性、行為、病原性等多個面向。傳統上反覆性泌尿道感染若無明顯致病原因，預防方法則為多喝水、少憋尿或調整避孕方式等，但部分病人縱然使用上述方式仍無法減少反覆性泌尿道感染的發生。口服戊聚醣多硫酸酯能修復泌尿道感染後受損的膀胱黏膜保護層 (Glycosaminoglycan, GAG, layer)，以期減少致病菌對受損泌尿上皮的入侵及附著，可能是一個新興預防反覆性泌尿道感染的有效方法。本文為第二期的臨床試驗來評估使用口服戊聚醣多硫酸酯(Pentosan polysulfate sodium)對反覆性泌尿道感染預防的功效及安全性。

此第二期臨床試驗於台灣多個大型醫學中心執行，採前瞻性開放式隨機對照模式進行，於反覆性泌尿道感染女性患者中比較使用口服戊聚醣多硫酸酯及對照組之後泌尿道感染發生的機率。執行期間總共招募26位受試者，其中12位使用為期16週口服戊聚醣多硫酸酯的受試者於試驗期間均未發生泌尿道感染(0%)，而對照組中的14位受試者則9位有泌尿道感染復發的情形(64%)。使用口服戊聚醣多硫酸酯的組別明顯較對照組不會有泌尿道感染復發(p值=0.0004)。執行期間有一位受試者因不良反應而退出臨床試驗，該不良反應與使用口服戊聚醣多硫酸酯無關。本研究受限於試驗樣本較少。

由以上試驗發現，曾有反覆性泌尿道感染的女性使用16週口服戊聚醣多硫酸酯與對照組相比能顯著減少泌尿道感染復發，若往後第大型的三期臨床試驗仍能同樣證實戊聚醣多硫酸酯對預防泌尿道感染的療效與安全性，此將能大大造福更多受苦的患者。

Background/purpose: Pentosan polysulfate sodium (PPS), a semi-synthetic polysaccharide that adheres to bladder mucosa, is effective in treating interstitial cystitis. We evaluated the clinical benefit of PPS for the prevention of recurrent urinary tract infection (UTI) in women.

Methods: We conducted a multicenter, open-label, prospective, phase II, randomized controlled trial enrolling women with recurrent UTI ≥ 2 times in the past 6 months or ≥ 3 times in the past 12 months. Patients received oral PPS monotherapy for 16 weeks in treatment group. All patients were followed every 28 days until UTI recurrence or up to 112 days. The primary endpoint was the UTI recurrence-free survival. Adverse events were recorded as secondary endpoint.

Results: A total of 26 women were eligible for analysis. In the PPS group, none (0%) of the 12 patients had UTI recurrence during the study period. However, 9 (64%) of 14 patients had UTI recurrence in the control group. The UTI recurrence-free survival was significantly higher in the PPS group than in the control group (log-rank test p = 0.0004). One adverse event which led to discontinuation of the trial regimen was regarded as irrelevance of PPS treatment. The limitation was the small number of cases.

Conclusion: Among women with recurrent UTI, 16-week PPS monotherapy significantly reduced UTI recurrence when compared with the control group.

Keywords: Cystitis; Pentosan polysulfate; Prophylaxis; Recurrent urinary tract infections.