A randomized, multi-national, non-inferiority, phase III trial to evaluate the safety and efficacy of BF-200 ALA gel versus MAL cream in the treatment of non-aggressive basal cell carcinoma with photodynamic therapy (PDT)

Morton, C.A. et al. (2018) A randomized, multi-national, non-inferiority, phase III trial to evaluate the safety and efficacy of BF-200 ALA gel versus MAL cream in the treatment of non-aggressive basal cell carcinoma with photodynamic therapy (PDT). British Journal of Dermatology, 179(2), pp. 309-319. (doi: 10.1111/bjd.16441) (PMID:29432644)

Abstract

Background: Basal cell carcinoma (BCC) represents the most common non-melanoma skin cancer worldwide affecting mainly adult, fair-skinned individuals. The WHO distinguishes aggressive and non-aggressive forms of which prototypical variants of the latter are primary nodular and superficial BCC. Objectives: To demonstrate non-inferiority of BF-200 ALA (a nanoemulsion gel containing 5-aminolaevulinic acid) compared to MAL (a cream containing methyl-aminolevulinate) in the treatment of non-aggressive BCC with photodynamic therapy (PDT). Non-inferiority of the primary efficacy variable (overall patient complete response 12 weeks after last PDT) would be declared if the mean response for BF-200 ALA was no worse than that for MAL, within a statistical margin of Δ = -15%. Patients/Methods: The study was a randomized, phase III trial performed in Germany and the UK with ongoing 5-year follow-up. Of 281 randomized patients, 138 were treated with BF-200 ALA, 143 with MAL. Patients received two PDT sessions one week apart. Remaining lesions 12 weeks after the second PDT were retreated. Illumination was performed with a red light source (635 nm, 37 J/cm2). Results shown include clinical endpoints as well as patients’ reassessment 12 months after the last PDT. Results: Of the BF-200 ALA-treated patients, 93.4% were complete responders compared to 91.8% in the MAL group. The difference of means was 1.6 with a one-sided 97.5% CI of -6.5, establishing non-inferiority (p<0.0001). Results for secondary efficacy parameters were in line with the primary outcome. Recurrence rates 12 months after the last treatment were ≤ 10%. Conclusions: Treatment of non-aggressive BCC with BF-200 ALA-PDT is highly effective and well tolerated with proven non-inferiority to MAL-PDT and demonstrates low recurrence rates after 1-year follow-up.

• Item Type: Articles
• Status: Published
• Refereed: Yes
• Glasgow Author(s) Enlighten ID: Gupta, Dr Girish
• Authors: Morton, C.A., Dominicus, R., Radny, P., Dirschka, T., Hauschild, A., Reinhold, U., Aschoff, R., Ulrich, M., Keohane, S., Ekanayake-Bohlig, S., Ibbotson, S., Ostendorf, R., Berking, C., Gröne, D., Schulze, H.J., Ockenfels, H.M., Jasnoch, V., Kurzen, H., Sebastian, M., Stege, H., Staubach-Renz, P., Gupta, G., Hübinger, F., Ziabreva, I., Schmitz, B., Gertzmann, A., Lübbert, H., and Szeimies, R.-M.
• College/School: College of Medical Veterinary and Life Sciences > School of Medicine, Dentistry & Nursing
• Journal Name: British Journal of Dermatology
• Publisher: Wiley
• ISSN: 0007-0963
• ISSN (Online): 1365-2133
• Published Online: 12 February 2018
• Copyright Holders: Copyright © 2018 British Association of Dermatologists
• First Published: First published in British Journal of Dermatology 179(2): 309-319
• Publisher Policy: Reproduced in accordance with the publisher copyright policy