TY - JOUR
T1 - Effectiveness of dabrafenib in the treatment of patients with braf v600-mutated metastatic melanoma in a named patient program
T2 - Melanoma Research
AU - Martin-Algarra, S.
AU - Hinshelwood, R.
AU - Mesnage, S.
AU - Cebon, J.
AU - Ferrucci, P.F.
AU - Aglietta, M.
AU - Neyns, B.
AU - Chiarion-Sileni, V.
AU - Lindsay, C.R.
AU - Del Vecchio, M.
AU - Linardou, H.
AU - Merelli, B.
AU - Tonini, G.
AU - Atkinson, V.
AU - Freivogel, K.
AU - Stein, D.
AU - Dalland, L.
AU - Lau, M.
AU - Legenne, P.
AU - Queirolo, P.
AU - Millward, M.
N1 - Cited By :1
Export Date: 28 February 2020
CODEN: MREEE
Correspondence Address: Martin-Algarra, S.; Department of Medical Oncology, Clínica Universidad de Navarra, University of Navarra, Avda Pío XII, 36, Spain; email: smalgarra@unav.es
References: Fitzmaurice, C., Dicker, D., Pain, A., Hamavid, H., Moradi-Lakeh, M., The global burden of cancer 2013 (2015) JAMA Oncol, 1, pp. 505-527; SEER Stat Fact Sheets: Melanoma of the Skin, , http://seer.cancer.gov/statfacts/html/melan.html; Singh, B.P., Salama, A.K., Updates in therapy for advanced melanoma (2016) Cancers (Basel), 8, p. E17; Sullivan, R.J., Flaherty, K.T., New strategies in melanoma: Entering the era of combinatorial therapy (2015) Clin Cancer Res, 21, pp. 2424-2435; Ascierto, P.A., Minor, D., Ribas, A., Lebbe, C., O'Hagan, A., Arya, N., Phase II trial (BREAK-2) of the BRAF inhibitor dabrafenib (GSK2118436) in patients with metastatic melanoma (2013) J Clin Oncol, 31, pp. 3205-3211; Hauschild, A., Grob, J.J., Demidov, L.V., Jouary, T., Gutzmer, R., Millward, M., Dabrafenib in BRAF-mutated metastatic melanoma: A multicentre, open-label, phase 3 randomised controlled trial (2012) Lancet, 380, pp. 358-365; Long, G.V., Trefzer, U., Davies, M.A., Kefford, R.F., Ascierto, P.A., Chapman, P.B., Dabrafenib in patients with val600glu or val600lys BRAF-mutant melanoma metastatic to the brain (BREAK-MB): A multicentre, open-label, phase 2 trial (2012) Lancet Oncol, 13, pp. 1087-1095; Long, G.V., Stroyakovskiy, D., Gogas, H., Levchenko, E., De Braud, F., Larkin, J., Combined BRAF and MEK inhibition versus BRAF inhibition alone in melanoma (2014) N Engl J Med, 371, pp. 1877-1888; Robert, C., Karaszewska, B., Schachter, J., Rutkowski, P., Mackiewicz, A., Stroiakovski, D., Improved overall survival in melanoma with combined dabrafenib and trametinib (2015) N Engl J Med, 372, pp. 30-39; Long, G.V., Stroyakovskiy, D., Gogas, H., Levchenko, E., De Braud, F., Larkin, J., Dabrafenib and trametinib versus dabrafenib and placebo for val600 BRAF-mutant melanoma: A multicentre, double-blind, phase 3 randomised controlled trial (2015) Lancet, 386, pp. 444-451; Tafinlar Package Insert, , https://www.pharma.us.novartis.com/sites/www.pharma.us.novartis.com/files/tafinlar.pdf; Lau, D.K., Andrews, M.C., Turner, N., Azad, A.A., Davis, I.D., Cebon, J.S., A single-centre experience of patients with metastatic melanoma enrolled in a dabrafenib named patient programme (2014) Melanoma Res, 24, pp. 144-149; Ascierto, P.A., Minor, D.R., Ribas, A., Lebbe, C., O'Hagan, A., Swann, R.S., Long-Term safety and overall survival update for BREAK-2, a phase 2, single-Arm, open-label study of dabrafenib in previously treated metastatic melanoma (NCT01153763) [abstract #9034] (2014) J Clin Oncol, 32. , Suppl; Hauschild, A., Grob, J.J., Demidov, L.V., Jouary, T., Gutzmer, R., Millward, M., An update on BREAK-3, a phase III, randomized trial: Dabrafenib (DAB) versus dacarbazine (DTIC) in patients with BRAF V600E-positive mutation metastatic melanoma (MM) [abstract #9013] (2013) J Clin Oncol, 31. , Suppl; (2017) National Comprehensive Cancer Network-Melanoma Volume V1. National Comprehensive Cancer Network, , www.nccn.org/professionals/physician_gls/pdf/melanoma.pdf; Flaherty, K.T., Infante, J.R., Daud, A., Gonzalez, R., Kefford, R.F., Sosman, J., Combined BRAF and MEK inhibition in melanoma with BRAF V600 mutations (2012) N Engl J Med, 367, pp. 1694-1703; Robert, C., Karaszewska, B., Schachter, J., Rutkowski, P., Mackiewicz, A., Stroyakovskiy, D., Three-year estimate of overall survival in COMBI-v, a randomized phase 3 study evaluating first-line dabrafenib (D) + trametinib (T) in patients (pts) with unresectable or metastatic BRAF V600E/K-mutant cutaneous melanoma [abstract #LBA40] (2016) Ann Oncol, 27. , Suppl 6; Atkinson, V., Van Thienen, J., McArthur, G., Hospers, G., Long, G., Carnevale-Schianca, F., Safety and effectiveness analysis of V600 BRAF-mutated metastatic melanoma patients from the dabrafenib plus trametinib named patient programme (NPP)-DESCRIBE II study (2015) Eur J Cancer, 51, pp. S677-S678. , Suppl 3
PY - 2019
Y1 - 2019
N2 - 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. © 2019 Wolters Kluwer Health, Inc. All rights reserved.
AB - 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. © 2019 Wolters Kluwer Health, Inc. All rights reserved.
KW - BRAF
KW - dabrafenib
KW - melanoma
KW - retrospective studies
KW - trametinib
U2 - 10.1097/CMR.0000000000000608
DO - 10.1097/CMR.0000000000000608
M3 - Article
VL - 29
SP - 527
EP - 532
JO - Melanoma Res.
JF - Melanoma Res.
SN - 0960-8931
IS - 5
ER -