Dabrafenib plus trametinib for compassionate use in metastatic melanoma: A STROBE-compliant retrospective observational postauthorization study

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 e...

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Detalles Bibliográficos
Autores: Martin Algarra, Salvador, Soriano, Virtudes, Fernandez-Morales, Luis, Berciano-Guerrero, Miguel-Angel, Mujika, Karmele, Luis Manzano, Jose, Puertolas, Teresa, Soria, Ainara, Rodriguez-Abreu, Delvys, Espinosa, Enrique, Medina Martinez, Javier, Marquez-Rodas, Ivan, Rubio-Casadevall, Jordi, Ortega, M. E., Jurado Garcia, Jose Miguel, Lecumberri Biurrun, Maria Jose, Palacio, Isabel, Rodriguez de la Borbolla Artacho, Maria, Perez Altozano, Javier, Castellon Rubio, Victoria Eugenia, Garcia, Almudena, Luna Fra, Pablo, Ballesteros, Ana, Fernandez, Ovidio, Lopez Martin, Jose Antonio, Berrocal, Alfonso, Arance, Ana
Tipo de recurso: artículo
Fecha de publicación:2017
País:España
Institución:Conselleria de Salut i Consum del Govern de les Illes Balears
Repositorio:Docusalut
Idioma:inglés
OAI Identifier:oai:docusalut.com:20.500.13003/17357
Acceso en línea:https://hdl.handle.net/20.500.13003/17357
Access Level:acceso abierto
Palabra clave:Neoplasm Metastasis
Compassionate Use Trials
Retrospective Studies
Aged, 80 and over
Aged
Oximes
Spain
Adult
Pyridones
Humans
Antineoplastic Combined Chemotherapy Protocols
Melanoma
Middle Aged
Male
Pyrimidinones
Imidazoles
Survival Analysis
Female
España
Oximas
Análisis de Supervivencia
Femenino
Metástasis de la Neoplasia
Masculino
Piridonas
Humanos
Persona de Mediana Edad
Pirimidinonas
Protocolos de Quimioterapia Combinada Antineoplásica
Anciano
Anciano de 80 o más Años
Ensayos de Uso Compasivo
Estudios Retrospectivos
Adulto
BRAF
BRAF inhibitors
compassionate use
dabrafenib
metastatic melanoma
trametinib
Descripción
Sumario: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.

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