title: A Prospective, Multicenter, Double-Blind, Randomized Trial of Bladder Instillation of Liposome Formulation OnabotulinumtoxinA for Interstitial Cystitis/Bladder Pain Syndrome.
abstract: Intravesical instillation of liposomal formulated botulinum toxin A (lipotoxin) has shown therapeutic effects as treatment of refractory overactive bladder without needle injections. We assessed lipotoxin to treat refractory interstitial cystitis/bladder pain syndrome.
  
  
  
  This 2-center, double-blind, randomized, placebo controlled, physician initiated study enrolled patients with refractory interstitial cystitis/bladder pain syndrome. A total of 31 patients were assigned to intravesical instillation of lipotoxin (onabotulinumtoxinA 200 U with 80 mg sphingomyelin), 28 were assigned to onabotulinumtoxinA 200 U in normal saline and 31 were assigned to normal saline alone. The primary end point was the average change in O'Leary-Sant symptom scores, including ICSI (Interstitial Cystitis Symptom Index) and ICPI (Interstitial Cystitis Problem Index) between baseline and 4 weeks after treatment. Other end points included the average changes in a 3-day voiding diary, a visual analog scale for pain and a global response assessment of patient satisfaction.
  
  
  
  Improvements in the pain scale and O'Leary-Sant symptom scores occurred in all 3 groups by 4 weeks after treatment. Lipotoxin instillation was associated with a statistically significant decrease in O'Leary-Sant symptom scores (mean ± SD 7.38 ± 8.75), ICSI (4.00 ± 4.28), ICPI (3.35 ± 5.11) and the visual analog scale pain scale (1.64 ± 2.52), and an increase in the global response assessment (1.35 ± 1.28). However, there was no difference in improvement among the 3 groups. No significant adverse events were found in any group.
  
  
  
  Lipotoxin failed to demonstrate a positive proof of concept compared to onabotulinumtoxinA or placebo. However, a single intravesical instillation of lipotoxin was associated with decreased interstitial cystitis/bladder pain syndrome symptoms compared to baseline in patients with moderate to severe interstitial cystitis/bladder pain syndrome. The effect was likely due to a significant placebo effect.
authors.affiliation: Department of Urology, Kaohsiung Chang Gang Memorial Hospital, College of Medicine, Chang Gung University, Hualien, Taiwan; Department of Urology, Buddhist Tzu Chi General Hospital and Tzu Chi University, Hualien (HCK), Taiwan.
authors.forename: Yao-Chi
authors.initials: YC
authors.lastname: Chuang
authors.affiliation: Department of Urology, Kaohsiung Chang Gang Memorial Hospital, College of Medicine, Chang Gung University, Hualien, Taiwan; Department of Urology, Buddhist Tzu Chi General Hospital and Tzu Chi University, Hualien (HCK), Taiwan. Electronic address: hck@tzuchi.com.tw.
authors.forename: Hann-Chorng
authors.initials: HC
authors.lastname: Kuo
mesh_terms: D000283:Administration, Intravesical; D000328:Adult; D000368:Aged; D019274:Botulinum Toxins, Type A / Q000008:administration & dosage*; D018856:Cystitis, Interstitial / Q000150:complications / Q000188:drug therapy*; D004311:Double-Blind Method; D005260:Female; D006801:Humans; D008081:Liposomes; D008297:Male; D008875:Middle Aged; D009465:Neuromuscular Agents / Q000008:administration & dosage*; D010147:Pain Measurement; D017060:Patient Satisfaction; D017699:Pelvic Pain / Q000188:drug therapy* / Q000209:etiology; D000075082:Proof of Concept Study; D013109:Sphingomyelins; D016896:Treatment Outcome
chemical_list: D008081:Liposomes; D009465:Neuromuscular Agents; D013109:Sphingomyelins; D019274:Botulinum Toxins, Type A; C000629279:onabotulinum toxin A
keywords: botulinum toxin; interstitial cystitis; liposome; pelvic pain; urinary bladder