Details

Autor(en) / Beteiligte

• Dummer, Reinhard, MD
• Guminski, Alexander, MD, PhD
• Gutzmer, Ralf, MD
• Dirix, Luc, MD
• Lewis, Karl D., MD
• Combemale, Patrick, MD
• Herd, Robert M., MD
• Kaatz, Martin, MD
• Loquai, Carmen, MD
• Stratigos, Alexander J., MD
• Schulze, Hans-Joachim, MD
• Plummer, Ruth, MD
• Gogov, Sven, MD
• Pallaud, Celine, PhD
• Yi, Tingting, PhD
• Mone, Manisha, PhD
• Chang, Anne Lynn S., MD
• Cornélis, Frank, MD
• Kudchadkar, Ragini, MD
• Trefzer, Uwe, MD
• Lear, John T., MD
• Sellami, Dalila, MD
• Migden, Michael R., MD

Titel

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

Ist Teil von

• Journal of the American Academy of Dermatology, 2016-07, Vol.75 (1), p.113-125.e5

Ort / Verlag

United States: Elsevier Inc

Erscheinungsjahr

2016

Quelle

MEDLINE

Beschreibungen/Notizen

• 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.