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Department of Dermatology, Division of Internal Medicine, University of Texas MD Anderson Cancer Center, Houston, TexasDepartment of Head and Neck Surgery, Division of Surgery, University of Texas MD Anderson Cancer Center, Houston, Texas
Department of Medical Oncology, Institut Roi Albert II, Cliniques universitaires Saint-Luc, UCLouvain, Brussels, BelgiumInstitute for Experimental and Clinical Research, Université catholique de Louvain, Brussels, Belgium
Patients with locally advanced (laBCC) or metastatic BCC (mBCC) require treatment options other than traditional surgery and radiation, which can produce scarring and disfigurement that cause psychological stress.
Assessments of QoL in patients with laBCC and mBCC were conducted during the Basal Cell Carcinoma Outcomes with LDE225 Treatment (BOLT) trial for sonidegib.
Basal Cell Carcinoma Outcomes with LDE225 Treatment was a multicenter, randomized, double-blind, phase 2 study evaluating efficacy and safety of sonidegib (200 and 800 mg) in patients with laBCC or mBCC not amenable to surgery or radiation therapy and has been described.
All patients were invited to complete the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (QLQ-C30) and associated Head and Neck Cancer Module 35 (H&N35) at baseline, Weeks 9 and 17, every 8 weeks (first year), and then every 12 weeks until end of treatment. QLQ-C30 and H&N35 subscales most applicable to patients with advanced BCC were evaluated, including physical functioning, social functioning, pain, and fatigue (QLQ-C30), and trouble with social contact, weight loss, and head and neck pain (H&N35). Higher functioning subscale scores indicated positive (improved) change with high function, while higher symptom subscale scores indicated negative (worsened) change with high levels of symptoms. Analyses included mean change from baseline in subscale scores over time.
This study was conducted according to the ethical principles of the Declaration of Helsinki with approvals from local ethics committees or institutional review boards. All patients provided informed consent.
Sixty-six patients with laBCC and 13 with mBCC received the approved sonidegib 200 mg daily dose. Median duration of exposure was 8.9 months; 91% of patients received treatment for ≥4 months. Overall, 88.7% (QLQ-C30) and 90.0% (H&N35) of all patients had baseline and ≥1 postbaseline questionnaire assessment. Mean changes from baseline scores for QLQ-C30 and H&N35 subscales were generally modest, and positive and negative fluctuations appeared evenly distributed for most subscales (Figs 1 and 2; Supplementary Table I and II, available via Mendeley at https://doi.org/10.17632/mm3xxgy6rm.1).
Treatment with sonidegib 200 mg daily appeared to maintain or improve QoL from baseline in most patients through 73 weeks. Observed worsening in weight loss scores may be expected due to dysgeusia.
As end-of-treatment values were recorded at different timepoints, these scores often varied from the general trends, especially for European Organisation for Research and Treatment of Cancer QLQ-C30 subscales. Countertrends at end of treatment may also have resulted from decreased drug exposure from treatment holidays due to adverse events in the period leading to discontinuation. Our findings suggest that patients may have perceived that QoL benefits in most analyzed subscales outweighed occurrent adverse effects of treatment. Maintenance or improvement of QoL in patients with advanced BCC—a difficult-to-treat population—provides additional support for use of sonidegib as a viable treatment for these patients.
Conflicts of interest
Migden has participated on advisory boards and received honoraria from Genentech; Novartis; Sun Pharmaceutical Industries, Inc; and Regeneron. Loquai acted as a speaker for, participated in an advisory board for, and received honoraria from Bristol-Myers Squibb, Roche, Novartis, and Merck Sharp & Dohme. Robert has received consulting fees from Amgen, Array BioPharma, Bristol-Myers Squibb, Merck, Merck-Serono, Novartis, Pierre-Fabre, and Roche. Baurain reports that his institution has received fees for advisory boards from Bristol-Myer Squibb, Novartis, Pierre-Fabre, Sun Pharmaceutical, Sanofi-Regeneron, Amgen, Merck-Serono, and Merck Sharp & Dohme. Squittieri is an employee of Sun Pharmaceutical Industries, Inc. Arntz and Dierlamm are employees of Sun Pharmaceutical Industries, (Europe) B.V. Dréno has been a consultant or advisor for Bristol-Myers Squibb, GlaxoSmithKline, Roche, and Novartis; has served on speakers’ bureaus for Bristol-Myers Squibb, GlaxoSmithKline, and Roche.
Medical writing and editorial assistance were provided by Jennifer Masucci, VMD, of AlphaBioCom, LLC, under the direction of the authors.
Trends in basal cell carcinoma incidence rates: a 16-year retrospective study of a population in central Poland.
Dr Loquai is currently affiliated with the Department of Dermatology, Klinikum Bremen-Ost, Gesundheitnord gGmbH, Bremen, Germany.
Dr Dréno is currently affiliated with the Nantes Université, INSERM, Immunology and New Concepts in ImmunoTherapy, INCIT, UMR 1302, F-44000 Nantes, France.
Funding sources: This study was sponsored and funded by Novartis . All publication fees were supported by Sun Pharmaceutical Industries, Inc. Dréno has received research funding from Amgen, Bristol-Myers Squibb, and GlaxoSmithKline; and has received travel support from Bristol-Myers Squibb and Roche.
IRB approval status: This study was conducted according to the ethical principles of the Declaration of Helsinki with approvals from local ethics committees or institutional review boards.
Patient consent form statement: All patient consent forms are on file.