Effectiveness Not Established
Research Evidence Summaries
Hunter, A., Mahendra, P., Wilson, K., Fields, P., Cook, G., Peniket, A., … Marcus, R. (2009). Treatment of oral mucositis after peripheral blood SCT with ATL-104 mouthwash: Results from a randomized, double-blind, placebo-controlled trial. Bone Marrow Transplantation, 43, 563–569.doi: 10.1038/bmt.2008.363
To assess the tolerability of three ATL-104 doses and to assess the efficacy and safety of ATL-104 on mucositis in patients requiring high-dose melphalan or BEAM (carmustine, etoposide, cytarabine, and melphalan) before peripheral blood SCT (PBSCT)
Intervention Characteristics/Basic Study Process:
This study included two parts. In the first part, patients were randomized to receive placebo or ATL-104 in doses of 50, 100, or 150 mg orally by mouthwash as a 15 mL total volume swilled in the mouth for 15-30 seconds and then swallowed. ATL-104 is a plant lectin associated with mitogenic activity and epithelial cell growth. Following harvest of PBSCs, patients received study treatment for three days before beginning a course of chemotherapy. For patients receiving BEAM, chemotherapy lasted for six days, whereas patients receiving high-dose melphalan had one day of treatment. PBSCT was administered 1–2 days after completion of chemotherapy, and patients then received three once daily doses of ATL-104. In the second part, patients were randomized to placebo or to one of the three ATL-104 doses.
- The study reported on 63 patients with a mean age of 53.3 years.
- The sample had 17 females and 46 males.
- The majority of patients (60.3%) received high-dose melphalan chemotherapy for multiple myeloma, and 39.7% received BEAM chemotherapy for lymphoma.
The study was conducted in an inpatient setting in the United Kingdom.
- Part 1 was a randomized, double-blind, placebo-controlled phase IIa dose escalation study.
- Part 2 was a randomized, double-blind, placebo-controlled parallel group study.
- The extent and severity of mucositis was assessed daily from screening until the final assessment, which was performed 28 days after the last dose of ATL-104 or at withdrawal.
- World Health Organization (WHO) and Western Consortium for Cancer Nursing Research (WCCNR) toxicity scales were used.
Treatment with ATL-104 substantially reduced the median duration of severe oral mucositis compared with placebo (median duration: ATL-104 for two to three days, placebo 10.5 days) (p < 0.001). The effect of ATL-104 on the incidence of severe oral mucositis was inconclusive. Adverse events (AEs) were mild to moderate in intensity with gastrointestinal (GI) AEs being most common.
ALT-104 substantially reduced the duration of severe mucositis and was well tolerated.
This study had inconclusive results regarding the incidence of severe mucositis. Further studies with a larger number of patients are needed to establish the effects of ATL-104 on the duration and severity of mucositis. Limited range of ATL-104 doses may be responsible for the lack of dose-response.
Mouthwashes are easy to administer; however, adherence is a concern when used preventatively or when the patient has oral discomfort or fatigue. The hospital setting allows for easier monitoring and assistance in promoting adherence. If further studies show a benefit to patients, cost and availability will be influencing factors toward good outcomes.