Intravesical instillation of liposomal-formulated botulinum toxin A (lipotoxin) has shown therapeutic effects on treatment of refractory overactive bladder without needle injections. We hereby assessed lipotoxin for the treatment of refractory interstitial cystitis/bladder pain syndrome (IC/BPS).
This two-center, double-blind, randomized, placebo-controlled physician-initiated study enrolled patients with refractory IC/BPS. The patients were assigned to intravesical instillation of lipotoxin (onabotulinumtoxinA 200 U with 80mg sphingomyelin) (n=31), onabotulinumtoxinA 200 U in normal saline (n=28), or normal saline alone (n=31). The primary end-point was the average changes in O'Leary-Sant symptom scores (OSS), including IC symptom and problem indices (ICSI; ICPI), between baseline and 4 weeks after treatment. Other end-points included the average changes in 3-day voiding diary, visual analog scale (VAS) for pain and global response assessment (GRA) of patient satisfaction.
Improvements in pain scale and OSS occurred in all three groups four weeks after treatment. Lipotoxin instillation was associated with a statistically significant decrease in OSS, ICSI, ICPI, and VAS pain scale (7.38±8.75, 4.00±4.28, 3.35±5.11, and 1.64±2.52, respectively) and an increase in GRA (1.35±1.28). However, there was no difference in improvement among the three groups. No significant adverse events were found in any group.
Lipotoxin failed to demonstrate a positive proof of concept compared to OnaBoNTA or placebo, although a single intravesical instillation of lipotoxin was associated with a decrease of IC/BPS symptoms compared to baseline in patients with moderate to severe IC/BPS. The effect is likely due to a significant placebo effect.
The Journal of urology. 2017 Feb 12 [Epub ahead of print]
Yao-Chi Chuang, Hann-Chorng Kuo
Department of Urology, Kaohsiung Chang Gang Memorial Hospital, Chang Gung University, College of Medicine, Taiwan., Department of Urology, Buddhist Tzu Chi General Hospital and Tzu Chi University, Hualien, Taiwan. Electronic address: .