@article{3101042,
    title = "The 12-month analysis from Basal Cell Carcinoma Outcomes with LDE225 Treatment (BOLT): A phase II, randomized, double-blind study of sonidegib in patients with advanced basal cell carcinoma",
    author = "Dummer, R. and Guminski, A. and Gutzmer, R. and Dirix, L. and Lewis, K.D. and Combemale, P. and Herd, R.M. and Kaatz, M. and Loquai, C. and Stratigos, A.J. and Schulze, H.-J. and Plummer, R. and Gogov, S. and Pallaud, C. and Yi, T. and Mone, M. and Chang, A.L.S. and Cornélis, F. and Kudchadkar, R. and Trefzer, U. and Lear, J.T. and Sellami, D. and Migden, M.R.",
    journal = "Journal of the American Academy of Dermatology",
    year = "2016",
    volume = "75",
    number = "1",
    pages = "113-125.e5",
    publisher = "Mosby Year Book Inc",
    issn = "0190-9622, 1097-6787",
    doi = "10.1016/j.jaad.2016.02.1226",
    keywords = "creatine kinase;  sonidegib;  vismodegib;  antineoplastic agent;  biphenyl derivative;  pyridine derivative;  Smoothened protein;  sonidegib, adult;  advanced cancer;  alopecia;  Article;  basal cell carcinoma;  cancer patient;  controlled study;  creatine kinase blood level;  decreased appetite;  diarrhea;  double blind procedure;  drug efficacy;  drug safety;  drug withdrawal;  dysgeusia;  fatigue;  female;  follow up;  human;  major clinical study;  male;  multicenter study;  muscle spasm;  myalgia;  nausea;  outcome assessment;  phase 1 clinical trial;  phase 2 clinical trial;  priority journal;  randomized controlled trial;  rhabdomyolysis;  side effect;  treatment response;  vomiting;  weight reduction;  aged;  antagonists and inhibitors;  Carcinoma, Basal Cell;  clinical trial;  disease course;  middle aged;  pathology;  secondary;  Skin Neoplasms;  survival rate;  very elderly;  young adult, Adult;  Aged;  Aged, 80 and over;  Antineoplastic Agents;  Biphenyl Compounds;  Carcinoma, Basal Cell;  Disease Progression;  Double-Blind Method;  Female;  Humans;  Male;  Middle Aged;  Pyridines;  Skin Neoplasms;  Smoothened Receptor;  Survival Rate;  Young Adult",
    abstract = "Background The hedgehog pathway inhibitor sonidegib demonstrated meaningful tumor shrinkage in more than 90% of patients with locally advanced basal cell carcinoma (BCC) or metastatic BCC in the BCC Outcomes with LDE225 Treatment study. Objective This report provides long-term follow-up data collected up to 12 months after the last patient was randomized. Methods In this multicenter, randomized, double-blind phase II study, patients were randomized 1:2 to sonidegib 200 or 800 mg. The primary end point was objective response rate assessed by central review. Results Objective response rates in the 200- and 800-mg arms were 57.6% and 43.8% in locally advanced BCC and 7.7% and 17.4% in metastatic BCC, respectively. Among the 94 patients with locally advanced BCC who responded, only 18 progressed or died and more than 50% had responses lasting longer than 6 months. In addition, 4 of 5 responders with metastatic BCC maintained an objective response. Grade 3/4 adverse events and those leading to discontinuation were less frequent with sonidegib 200 versus 800 mg (38.0% vs 59.3%; 27.8% vs 37.3%, respectively). Limitations No placebo or comparator arms were used because sonidegib demonstrated efficacy in advanced BCC in a phase I study, and the hedgehog pathway inhibitor vismodegib was not yet approved. Conclusion With longer follow-up, sonidegib demonstrated sustained tumor responses in patients with advanced BCC. © 2016 American Academy of Dermatology, Inc."
}