RT Journal Article
T1 Dabrafenib plus trametinib for compassionate use in metastatic melanoma: A STROBE-compliant retrospective observational postauthorization study
A1 Martin-Algarra, Salvador
A1 Soriano, Virtudes
A1 Fernandez-Morales, Luis
A1 Berciano-Guerrero, Miguel-Angel
A1 Mujika, Karmele
A1 Manzano, Jose Luis
A1 Puertolas-Hernandez, Teresa
A1 Soria, Ainara
A1 Rodriguez-Abreu, Delvys
A1 Espinosa-Arranz, Enrique
A1 Medina-Martinez, Javier
A1 Marquez-Rodas, Ivan
A1 Rubio-Casadevall, Jordi
A1 Ortega, Maria Eugenia
A1 Jurado-Garcia, Jose Miguel
A1 Lecumberri-Biurrun, Maria Jose
A1 Palacio, Isabel
A1 Rodriguez-de-la-Borbolla-Artacho, Maria
A1 Perez-Altozano, Javier
A1 Castellon-Rubio, Victoria Eugenia
A1 Garcia, Almudena
A1 Luna, Pablo
A1 Ballesteros, Anabel
A1 Fernandez, Ovidio
A1 Lopez-Martin, Jose Antonio
A1 Berrocal, Alfonso
A1 Arance, Ana
K1 BRAF
K1 BRAF inhibitors
K1 compassionate use
K1 dabrafenib
K1 metastatic melanoma
K1 trametinib
K1 Área de Gestión Sanitaria Sur de Sevilla
AB The main objective of the study was to evaluate the efficacy and safety of dabrafenib alone or combined with trametinib for compassionate use in patients with metastatic melanoma.This retrospective, observational study involved 135 patients with unresectable stage IIIC or stage IV melanoma from an expanded-access program at 30 Spanish centers.Forty-eight patients received dabrafenib monotherapy and 87 received combination dabrafenib and trametinib; 4.4% and 95.6% of the patients had stage IIIC and IV melanoma, respectively. All patients showed BRAF mutations in their primary or metastatic lesions; 3 were positive for V600K while the remainder had V600E or V600+. A positive response to treatment was reported in 89.3% of the patients. Overall survival rates at 12 and 24 months were 59.6% (95% confidence interval [CI], 52.5-68.9%) and 36.4% (95% CI, 27.8-45%), respectively. Progression-free survival rates at 12 and 24 months were 39.3% (95% CI, 31.1-47.5%) and 21.6% (95% CI, 14.5-28.7%), respectively. Fifty-seven patients (42.2%) reported cutaneous toxicity of any type, mainly hyperkeratosis (14.8%) and rash (11.9%). The most frequent adverse events were pyrexia (27.4%), asthenia (19.3%), arthralgia (16.9%), and diarrhoea (13.2%).Our results suggest that both dabrafenib alone or in combination with trametinib are effective for compassionate use in terms of response and/or survival rates. However, differences in patients' prognostic features ought to be considered. No new findings were revealed regarding the safety profiles of either regimen. This is the first study to evaluate the efficacy of these 2 selective BRAF and mitogen-activated extracellular signal-regulated kinase inhibitors in a real-world setting in Spain.
PB Wolters Kluwer Health
SN 0025-7974
YR 2017
FD 2017-12-06
LK http://hdl.handle.net/10668/18962
UL http://hdl.handle.net/10668/18962
LA en
NO Martín Algarra S, Soriano V, Fernández-Morales L, Berciano-Guerrero MÁ, Mujika K, Manzano JL, et al. Dabrafenib plus trametinib for compassionate use in metastatic melanoma: A STROBE-compliant retrospective observational postauthorization study. Medicine (Baltimore). 2017 Dec;96(52):e9523
DS RISalud
RD Apr 17, 2025