Effectiveness of dabrafenib in the treatment of patients with BRAF V600-mutated metastatic melanoma in a Named Patient Program
Salvador Martin-Algarra (1) , Rebecca Hinshelwood (2) , Soizick Mesnage (3) , Jonathan Cebon (4) , Pier Francesco Ferrucci (5) , Massimo Aglietta (6) , Bart Neyns (7) , Vanna Chiarion-Sileni (8) , Colin R Lindsay (9), Michele Del Vecchio (10) , Helen Linardou (11) , Barbara Merelli (12) , Giuseppe Tonini (13) , Victoria Atkinson (14) , Klaus Freivogel (15) , Dara Stein (16) , Lindi Dalland (17) , Mike Lau (18) , Philippe Legenne (18) , Paola Queirolo (19) , Michael Millward (20)
(1) Department of Medical Oncology, Clínica Universidad de Navarra, University of Navarra, Pamplona, Spain.
(2) Department of Medical Oncology, The Crown Princess Mary Cancer Centre, Westmead Hospital, Westmead, NSW.
(3) Auckland City Hospital, Auckland, New Zealand.
(4) Olivia Newton-John Cancer Research Institute at Austin Hospital, Heidelberg, VIC.
(5) Melanoma Unit, European Institute of Oncology, Milano.
(6) Department of Medical Oncology, Candiolo Cancer Institute, University of Torino, Torino.
(7) Department of Medical Oncology, Universitair Ziekenhuis Brussel, Brussels, Belgium.
(8) Melanoma and Esophageal Cancer Unit, Istituto Oncologico Veneto, Istituto Di Ricovero e Cura a Carattere Scientifico, Padova.
(9) Beatson West of Scotland Cancer Centre, NHS Greater Glasgow and Clyde, and Institute of Cancer Sciences, University of Glasgow, Glasgow, UK.
(10) Department of Medical Oncology, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan.
(11) First Department of Oncology, Metropolitan Hospital, Athens, Greece.
(12) Department of Medical Oncology, ASST Papa Giovanni XXIII, Bergamo.
(13) Department of Medical Oncology, Campus Bio-Medico University of Rome, Rome.
(14) Gallipoli Medical Research Foundation, University of Queensland and Princess Alexandra Hospital, Brisbane, QLD.
(15) UBC, Lörrach, Germany.
(16) UBC, Montreal, QC, Canada.
(17) Novartis Norge AS, Oslo, Norway.
(18) Novartis Pharma AG, Basel, Switzerland.
(19) UOC Oncologia Medica, IRCCS San Martino-IST, Genova, Italy.
(20) School of Medicine and Pharmacology, University of Western Australia, Perth, WA, Australia.
Given the approval of dabrafenib in patients with BRAF-mutant metastatic melanoma, a better understanding of treatment patterns and clinical outcomes with dabrafenib in a clinical setting is warranted.
We performed a retrospective chart review of patients who received dabrafenib in a compassionate use setting through the Named Patient Program (DESCRIBE I study) during December 2010-August 2013 in Europe, New Zealand and Australia.
Of the 331 Named Patient Program patients included, the majority (95.8%) had stage IV disease at dabrafenib initiation and 39.9% had brain metastases (BMs). Dabrafenib was used first line in 67.7% of patients, and median treatment duration was 6.4 months. Dabrafenib was well tolerated. Common grade 2/3 adverse events were hyperkeratosis (7.6%), pyrexia/fever (6.6%), fatigue (5.1%), hand-foot syndrome (5.4%) and nausea (3.6%). Overall response rate was 45.9%, median progression-free survival was 5.2 months (95% confidence interval, 4.2-6.1 months), and median overall survival was 12.4 months (95% confidence interval, 10.2-15.0 months).
In patients with known brain metastases (n = 132) versus patients without (n = 199), overall response rate was 42.4% versus 48.2%, progression-free survival was 3.9 months (95% confidence interval, 3.8-5.5 months) versus 5.9 months (95% confidence interval, 4.8-7.8 months) and overall survival was 9.5 months (95% confidence interval, 6.7-12.4 months) versus 15 months (95% confidence interval, 11.1-20.5 months), respectively. Safety and effectiveness of dabrafenib in patients with unresectable advanced BRAF V600-mutant melanoma treated in an Named Patient Program was similar to the clinical trial experience, demonstrating effectiveness of dabrafenib in a nontrial setting.
CITA DEL ARTÍCULO Melanoma Res. 2019 Oct;29(5):527-532. doi: 10.1097/CMR.0000000000000608