The value of Botox-A in acute radiation proctitis: results from a phase I/II study using a three-dimensional scoring system

Int J Radiat Oncol Biol Phys. 2011 Aug 1;80(5):1505-11. doi: 10.1016/j.ijrobp.2010.04.017. Epub 2010 Jul 23.

Abstract

Purpose: Acute radiation proctitis (ARP) is a common side effect of pelvic radiotherapy, and its management is challenging in daily practice. The present phase I/II study evaluates the safety and efficacy of the botulinum toxin A (BTX-A) in ARP treatment for rectal cancer patients undergoing neoadjuvant high-dose-rate endorectal brachytherapy (HDREBT).

Methods and materials: Fifteen patients, treated with neoadjuvant HDREBT, 26-Gy in 4 fractions, received the study treatment that consisted of a single injection of BTX-A into the rectal wall. The injection was performed post-HDREBT and prior to the development of ARP. The control group, 20 such patients, did not receive the BTX-A injection. Both groups had access to standard treatment with hydrocortisone rectal aerosol foam (Cortifoam) and anti-inflammatory and narcotic medication. The ARP was clinically evaluated by self-administered daily questionnaires using visual analog scores to document frequency and urgency of bowel movements, rectal burning/tenesmus, and pain symptoms before and after HDREBT.

Results: At the time of this analysis, there was no observed systemic toxicity. Patient compliance with the self-administered questionnaire was 100% from week 1 to 4, 70% during week 5, and 40% during week 6. The maximum tolerated dose was established at the 100-U dose level, and noticeable mean differences were observed in bowel frequency (p = 0.016), urgency (p = 0.007), and pain (p = 0.078).

Conclusions: This study confirms the feasibility and efficacy of BTX-A intervention at 100-U dose level for study patients compared to control patients. A phase III study with this dose level is planned to validate these results.

Publication types

  • Clinical Trial, Phase I
  • Clinical Trial, Phase II

MeSH terms

  • Anti-Inflammatory Agents / administration & dosage*
  • Botulinum Toxins, Type A / administration & dosage*
  • Brachytherapy / adverse effects*
  • Brachytherapy / methods
  • Case-Control Studies
  • Feasibility Studies
  • Female
  • Humans
  • Hydrocortisone / administration & dosage
  • Male
  • Maximum Tolerated Dose
  • Proctitis / drug therapy*
  • Radiation Injuries / drug therapy*
  • Rectal Neoplasms / radiotherapy*
  • Surveys and Questionnaires

Substances

  • Anti-Inflammatory Agents
  • Botulinum Toxins, Type A
  • Hydrocortisone