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J Am Acad Dermatol. 2016 Jul;75(1):113-125.e5. doi: 10.1016/j.jaad.2016.02.1226. Epub 2016 Apr 7.

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 information

1
UniversitätsSpital Zürich-Skin Cancer Center, University Hospital, Zürich, Switzerland. Electronic address: reinhard.dummer@usz.ch.
2
Royal North Shore Hospital, Sydney, Australia.
3
Medizinische Hochschule Hannover, Hannover, Germany.
4
Sint-Augustinus Ziekenhuis, Antwerp, Belgium.
5
Division of Medical Oncology, School of Medicine, University of Colorado, Aurora, Colorado.
6
Anti Cancer Institute, Léon Bérard, Lyon, France.
7
Glasgow Royal Infirmary, Glasgow, United Kingdom.
8
University Hospital Jena, Jena, Germany; SRH Wald-Klinikum Gera GmbH, Gera, Germany.
9
University Medical Center Mainz, Mainz, Germany.
10
Department of Dermatology, Andreas Sygros Hospital, University of Athens, Athens, Greece.
11
Fachklinik Hornheide, Münster, Germany.
12
Northern Centre for Cancer Care, Freeman Hospital, Newcastle upon Tyne, United Kingdom.
13
Novartis Pharma AG, Oncology Global Development, Basel, Switzerland.
14
Novartis Pharmaceuticals Corporation, East Hanover, New Jersey.
15
Stanford University School of Medicine, Redwood City, California.
16
Cliniques Universitaires Saint-Luc, Université Catholique de Louvain, Brussels, Belgium.
17
Winship Cancer Institute at Emory University, Atlanta, Georgia.
18
Dermatologikum Berlin, Berlin, Germany.
19
Manchester Academic Health Science Centre, University of Manchester, Manchester, United Kingdom.
20
Departments of Dermatology and Head and Neck Surgery, University of Texas MD Anderson Cancer Center, Houston, Texas.

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.

KEYWORDS:

Basal Cell Carcinoma Outcomes with LDE225 Treatment study; advanced basal cell carcinoma; hedgehog pathway inhibitor; locally advanced basal cell carcinoma; metastatic basal cell carcinoma; sonidegib

PMID:
27067394
DOI:
10.1016/j.jaad.2016.02.1226
[Indexed for MEDLINE]
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