Effectiveness of dabrafenib in the treatment of patients with BRAF V600-mutated metastatic melanoma in a Named Patient Program

S. Martin-Algarra, R. Hinshelwood, S. Mesnage, J. Cebon, P.F. Ferrucci, M. Aglietta, B. Neyns, V. Chiarion-Sileni, C.R. Lindsay, M. Del Vecchio, H. Linardou, B. Merelli, G. Tonini, V. Atkinson, K. Freivogel, D. Stein, L. Dalland, M. Lau, P. Legenne, P. QueiroloM. Millward

Research output: Contribution to journalArticle

Abstract

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.
Original languageEnglish
Pages (from-to)527-532
Number of pages6
JournalMelanoma Research
Volume29
Issue number5
DOIs
Publication statusPublished - 2019

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Melanoma
Confidence Intervals
Therapeutics
Disease-Free Survival
Fever
Compassionate Use Trials
dabrafenib
Hand-Foot Syndrome
Neoplasm Metastasis
Survival
Brain
New Zealand
Nausea
Fatigue
Clinical Trials
Safety

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Effectiveness of dabrafenib in the treatment of patients with BRAF V600-mutated metastatic melanoma in a Named Patient Program. / Martin-Algarra, S.; Hinshelwood, R.; Mesnage, S.; Cebon, J.; Ferrucci, P.F.; Aglietta, M.; Neyns, B.; Chiarion-Sileni, V.; Lindsay, C.R.; Del Vecchio, M.; Linardou, H.; Merelli, B.; Tonini, G.; Atkinson, V.; Freivogel, K.; Stein, D.; Dalland, L.; Lau, M.; Legenne, P.; Queirolo, P.; Millward, M.

In: Melanoma Research, Vol. 29, No. 5, 2019, p. 527-532.

Research output: Contribution to journalArticle

Martin-Algarra, S, Hinshelwood, R, Mesnage, S, Cebon, J, Ferrucci, PF, Aglietta, M, Neyns, B, Chiarion-Sileni, V, Lindsay, CR, Del Vecchio, M, Linardou, H, Merelli, B, Tonini, G, Atkinson, V, Freivogel, K, Stein, D, Dalland, L, Lau, M, Legenne, P, Queirolo, P & Millward, M 2019, 'Effectiveness of dabrafenib in the treatment of patients with BRAF V600-mutated metastatic melanoma in a Named Patient Program', Melanoma Research, vol. 29, no. 5, pp. 527-532. https://doi.org/10.1097/CMR.0000000000000608
Martin-Algarra, S. ; Hinshelwood, R. ; Mesnage, S. ; Cebon, J. ; Ferrucci, P.F. ; Aglietta, M. ; Neyns, B. ; Chiarion-Sileni, V. ; Lindsay, C.R. ; Del Vecchio, M. ; Linardou, H. ; Merelli, B. ; Tonini, G. ; Atkinson, V. ; Freivogel, K. ; Stein, D. ; Dalland, L. ; Lau, M. ; Legenne, P. ; Queirolo, P. ; Millward, M. / Effectiveness of dabrafenib in the treatment of patients with BRAF V600-mutated metastatic melanoma in a Named Patient Program. In: Melanoma Research. 2019 ; Vol. 29, No. 5. pp. 527-532.
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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.

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