TY - JOUR
T1 - Retrospective Chart Review of Dabrafenib Plus Trametinib in Patients with Metastatic BRAF V600-Mutant Melanoma Treated in the Individual Patient Program (DESCRIBE Italy)
AU - Aglietta, Massimo
AU - Chiarion-Sileni, Vanna
AU - Fava, Paolo
AU - Guidoboni, Massimo
AU - Depenni, Roberta
AU - Minisini, Alessandro
AU - Consoli, Francesca
AU - Ascierto, Paolo
AU - Rinaldi, Gaetana
AU - Banzi, Maria
AU - Marconcini, Riccardo
AU - Gueli, Rossana
AU - Ferraresi, Virginia
AU - Tucci, Marco
AU - Tonini, Giuseppe
AU - Lo Re, Giovanni
AU - Guida, Michele
AU - Del Vecchio, Michele
AU - Marcon, Ilaria Gioia
AU - Queirolo, Paola
N1 - Funding Information:
This study was sponsored by Novartis Farma S.p.A. As of 2 March 2015, dabrafenib and trametinib have become assets of Novartis AG. Financial support for medical editorial assistance was provided by Novartis Farma S.p.A.
Funding Information:
Vanna Chiarion-Sileni reports participation as a consultant for Bristol Myers Squibb (BMS), Merck Serono, Novartis, and Pierre Fabre; participation as an invited speaker for Merck Serono, Merck Sharp & Dohme (MSD), Novartis, Pierre Fabre, and Sanofi; and travel and accommodation support from BMS and Pierre Fabre outside the submitted work. Massimo Guidoboni received personal fees for participation in advisory boards from BMS and Novartis; travel support and consultation fees from Pierre Fabre; and a grant from MSD outside the submitted work. Roberta Depenni received grants from BMS, MSD, Novartis, and Sanofi outside the submitted work. Alessandro Minisini reports personal fees from Merck, MSD, Novartis, Pierre Fabre, Sanofi, and Sun Pharma outside the submitted work. Francesca Consoli reports personal fees for advisory board and consultancy from BMS, MSD, Novartis, and Pierre Fabre outside the submitted work. Paolo Ascierto received grants/research funds from Array, BMS, Roche-Genentech, and Sanofi; personal fees for a consultant/advisory role from Alkermes, Array, AstraZeneca, BMS, Boehringer Ingelheim, Daiichi Sankyo, Eisai, Idera, Immunocore, Incyte, Italfarmaco, Lunaphore, MedImmune, Merck, MSD, Nektar, Nouscom, Novartis, Oncosec, Pfizer, Pierre Fabre, Regeneron, Roche-Genentech, Sandoz, Sanofi, Seagen, Sun Pharma, Syndax, Takis, Ultimovacs, and 4SC; and travel support from MSD outside the submitted work. Riccardo Marconcini reports consulting fees from Incyte, La Roche, MSD, Novartis, and Pierre Fabre; honoraria from BMS, Ipsen, La Roche, MSD, Novartis, and Pierre Fabre; travel support from BMS, Ipsen, La Roche, MSD, Novartis, and Pierre Fabre; and participation in advisory boards for BMS, Ipsen, MSD, Novartis, and Pierre Fabre outside the submitted work. Michele Guida reports an advisory role for BMS, MSD, Novartis, and Pierre Fabre outside the submitted work. Michele Del Vecchio reports an advisory and consultant role for BMS, MSD, Novartis, Pierre Fabre, and Sanofi. Ilaria Gioia Marcon is an employee of Novartis Farma S.p.A. Paola Queirolo reports participation in advisory boards of BMS, Merck, MSD, Novartis, Pierre Fabre, Roche, Sanofi, and Sun Pharma. Massimo Aglietta, Paolo Fava, Gaetana Rinaldi, Maria Banzi, Rossana Gueli, Virginia Ferraresi, Marco Tucci, Giuseppe Tonini, and Giovanni Lo Re have declared no conflicts of interest.
Publisher Copyright:
© 2021, The Author(s).
PY - 2021/11
Y1 - 2021/11
N2 - Background: Real-world data on extended follow-up of patients with BRAF V600-mutant metastatic melanoma are limited. We investigated dabrafenib plus trametinib (dab + tram) outside of a clinical trial setting (Individual Patient Program; DESCRIBE Italy). Objective: To describe the baseline features, treatment patterns, efficacy, and safety outcomes in patients with BRAF V600-mutant unresectable or metastatic melanoma who had received dab + tram as part of the Managed Access Program (MAP) in Italy. Patients and methods: An observational, retrospective chart review was conducted in Italian patients with BRAF V600-mutant unresectable stage III/IV melanoma receiving dab + tram as part of the MAP. Baseline features, treatment patterns, efficacy, and safety outcomes were evaluated. Results: Overall, 499 patients were included in this analysis. BRAF V600E mutation was seen in 81.4% of patients. Overall response rate achieved in 243 of the 390 evaluable patients was 62.3% (95% CI 57.5–67.1). Median progression-free survival (PFS) was 9.3 months (95% CI 8.6–10.6). Subgroup analyses revealed that patients with normal lactate dehydrogenase (LDH) and ≤ three metastatic sites without brain metastases at baseline had better outcomes. With normal LDH at baseline, median PFS for patients with one or two metastatic sites other than cerebral was 18 months. No new safety signals were observed. Treatment was permanently discontinued because of treatment-emergent adverse events (TEAEs) in 9.2% of patients, and pyrexia (27.3%) was the most common TEAE, with a lower incidence than that in the phase 3 studies of dab + tram. Conclusion: Treatment of BRAF V600E-mutant metastatic melanoma with dab + tram in the real-world setting was effective and safe, including the unselected population with several patients having a high tumor burden – concordant with the results of the pivotal phase 3 studies of dab + tram.
AB - Background: Real-world data on extended follow-up of patients with BRAF V600-mutant metastatic melanoma are limited. We investigated dabrafenib plus trametinib (dab + tram) outside of a clinical trial setting (Individual Patient Program; DESCRIBE Italy). Objective: To describe the baseline features, treatment patterns, efficacy, and safety outcomes in patients with BRAF V600-mutant unresectable or metastatic melanoma who had received dab + tram as part of the Managed Access Program (MAP) in Italy. Patients and methods: An observational, retrospective chart review was conducted in Italian patients with BRAF V600-mutant unresectable stage III/IV melanoma receiving dab + tram as part of the MAP. Baseline features, treatment patterns, efficacy, and safety outcomes were evaluated. Results: Overall, 499 patients were included in this analysis. BRAF V600E mutation was seen in 81.4% of patients. Overall response rate achieved in 243 of the 390 evaluable patients was 62.3% (95% CI 57.5–67.1). Median progression-free survival (PFS) was 9.3 months (95% CI 8.6–10.6). Subgroup analyses revealed that patients with normal lactate dehydrogenase (LDH) and ≤ three metastatic sites without brain metastases at baseline had better outcomes. With normal LDH at baseline, median PFS for patients with one or two metastatic sites other than cerebral was 18 months. No new safety signals were observed. Treatment was permanently discontinued because of treatment-emergent adverse events (TEAEs) in 9.2% of patients, and pyrexia (27.3%) was the most common TEAE, with a lower incidence than that in the phase 3 studies of dab + tram. Conclusion: Treatment of BRAF V600E-mutant metastatic melanoma with dab + tram in the real-world setting was effective and safe, including the unselected population with several patients having a high tumor burden – concordant with the results of the pivotal phase 3 studies of dab + tram.
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U2 - 10.1007/s11523-021-00850-1
DO - 10.1007/s11523-021-00850-1
M3 - Article
AN - SCOPUS:85118776546
VL - 16
SP - 789
EP - 799
JO - Targeted Oncology
JF - Targeted Oncology
SN - 1776-2596
IS - 6
ER -