PMID- 28963614
OWN - NLM
STAT- MEDLINE
DCOM- 20190212
LR  - 20200225
IS  - 1532-2807 (Electronic)
IS  - 1219-4956 (Linking)
VI  - 25
IP  - 1
DP  - 2019 Jan
TI  - Efficacy of Vemurafenib Treatment in 43 Metastatic Melanoma Patients with BRAF 
      Mutation. Single-Institute Retrospective Analysis, Early Real-Life Survival Data.
PG  - 45-50
LID - 10.1007/s12253-017-0324-1 [doi]
AB  - BRAF inhibitor vemurafenib achieved improved overall survival over chemotherapy 
      and have been approved by the FDA and EMA for the treatment of BRAF-mutated 
      metastatic melanoma. The aim of our retrospective analysis was to determine the 
      efficacy and safety of vemurafenib therapy for BRAF mutated metastatic melanoma 
      and subsequently to prove the clinical benefit for the studied 43 patients, based 
      on real-life data. From November 2012 to October 2015 we have selected 43 BRAF 
      mutated, metastatic melanoma patients, treated with vemurafenib. The median 
      follow-up time was 15.9 months. We evaluated progression free survival (PFS), 
      overall survival (OS) and toxicities. According to the AJCC staging system 70% of 
      the patients had stage M1c metastasis, including 6 with stable brain metastasis. 
      Objective responses were noted in 51.1%, the disease control rate was achieved in 
      79% of the patients. Complete responses were attained by 5 patients (11.6%). 
      Median PFS was 6.48 (95% CI:4.8-15.0) months, median OS was 11.47 (95% 
      CI:8.08-NA) months. We found significant association between LDH level and OS in 
      univariate (p = 0.000613) and multivariate analysis (p = 0.0168). The most common 
      adverse events (AEs) included follicular hyperkeratosis, rash, arthralgia and 
      photosensitivity. Grade 3 AEs, such as cutaneous squamous-cell carcinoma, QTcB 
      interval prolongation, rash, arthralgia were reported in 7 patients (17%). We had 
      no Grade 4 side effects. Similar to the previously published data our analysis 
      confirms the improved survival with vemurafenib treatment (11.47 months) in 
      patients with BRAF V600 mutation. Vemurafenib therapy was well tolerated, the AE 
      profile was almost consistent with the previously reported data of randomised 
      clinical trials.
FAU - Czirbesz, Kata
AU  - Czirbesz K
AUID- ORCID: 0000-0003-0708-1547
AD  - Department of Dermatooncology, National Institute of Oncology, H-1122, Rath 
      Gyorgy utca 7-9, Budapest, Hungary. czirbikatu@gmail.com.
FAU - Gorka, Eszter
AU  - Gorka E
AD  - Department of Dermatooncology, National Institute of Oncology, H-1122, Rath 
      Gyorgy utca 7-9, Budapest, Hungary.
FAU - Balatoni, Timea
AU  - Balatoni T
AD  - Department of Dermatooncology, National Institute of Oncology, H-1122, Rath 
      Gyorgy utca 7-9, Budapest, Hungary.
FAU - Panczel, Gitta
AU  - Panczel G
AD  - Department of Dermatooncology, National Institute of Oncology, H-1122, Rath 
      Gyorgy utca 7-9, Budapest, Hungary.
FAU - Melegh, Krisztina
AU  - Melegh K
AD  - Department of Dermatooncology, National Institute of Oncology, H-1122, Rath 
      Gyorgy utca 7-9, Budapest, Hungary.
FAU - Kovacs, Peter
AU  - Kovacs P
AD  - Department of Dermatooncology, National Institute of Oncology, H-1122, Rath 
      Gyorgy utca 7-9, Budapest, Hungary.
FAU - Gezsi, Andras
AU  - Gezsi A
AD  - Department of Genetics, Cell- and Immunbiology, Semmelweis University, H-1089, 
      Nagyvarad ter 4, Budapest, Hungary.
FAU - Liszkay, Gabriella
AU  - Liszkay G
AD  - Department of Dermatooncology, National Institute of Oncology, H-1122, Rath 
      Gyorgy utca 7-9, Budapest, Hungary.
LA  - eng
PT  - Clinical Trial
PT  - Journal Article
DEP - 20170929
PL  - Switzerland
TA  - Pathol Oncol Res
JT  - Pathology oncology research : POR
JID - 9706087
RN  - 0 (Protein Kinase Inhibitors)
RN  - 207SMY3FQT (Vemurafenib)
RN  - EC 2.7.11.1 (BRAF protein, human)
RN  - EC 2.7.11.1 (Proto-Oncogene Proteins B-raf)
SB  - IM
MH  - Adult
MH  - Aged
MH  - Brain Neoplasms/*drug therapy/genetics/secondary
MH  - Female
MH  - Follow-Up Studies
MH  - Humans
MH  - Lymphatic Metastasis
MH  - Male
MH  - Melanoma/*drug therapy/genetics/pathology
MH  - Middle Aged
MH  - *Mutation
MH  - Neoplasm Invasiveness
MH  - Neoplasm Staging
MH  - Protein Kinase Inhibitors/therapeutic use
MH  - Proto-Oncogene Proteins B-raf/*genetics
MH  - Retrospective Studies
MH  - Skin Neoplasms/*drug therapy/genetics/pathology
MH  - Survival Rate
MH  - Treatment Outcome
MH  - Vemurafenib/*therapeutic use
OTO - NOTNLM
OT  - LDH level
OT  - Melanoma
OT  - Survival
OT  - Targeted therapy
OT  - Treatment
OT  - Vemurafenib
EDAT- 2017/10/01 06:00
MHDA- 2019/02/13 06:00
CRDT- 2017/10/01 06:00
PHST- 2017/03/01 00:00 [received]
PHST- 2017/09/21 00:00 [accepted]
PHST- 2017/10/01 06:00 [pubmed]
PHST- 2019/02/13 06:00 [medline]
PHST- 2017/10/01 06:00 [entrez]
AID - 10.1007/s12253-017-0324-1 [pii]
AID - 10.1007/s12253-017-0324-1 [doi]
PST - ppublish
SO  - Pathol Oncol Res. 2019 Jan;25(1):45-50. doi: 10.1007/s12253-017-0324-1. Epub 2017 
      Sep 29.