Personalised approach in combined treatment of cholangiocarcinoma: a case report of healing from cholangiocellular carcinoma at stage IV

Bunyatov, Timur; Zhao, Alexey; Kovalenko, Yu. A.; Гурмиков, Б. Н.; Vishnevsky, V.

doi:10.21037/jgo.2019.03.05

Cited by 15 publications

(13 citation statements)

References 17 publications

(14 reference statements)

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“…Lavingia et al described two cases reaching 9 and 12 months PFS when treated with dabrafenib and trametinib [ 37 ]. Bunyatov et al showed the longest remission with the same combination: a full response after 7 months of treatment with complete remission lasting 28 months [ 38 ]. In the second case, the PDX model predicted clinical response to dabrafenib and trametinib.…”

Section: Discussionmentioning

confidence: 99%

Tumor Type Agnostic Therapy Carrying BRAF Mutation: Case Reports and Review of Literature

Bernocchi

Sirico²,

Corona

et al. 2021

Pharmaceuticals

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Background: Precision medicine is based on molecular and genotypic patient characterization to define specific target treatment. BRAF mutation is an oncogenic driver, and the Cancer Genome Atlas has identified BRAF mutations in different cancer types. Tumor type agnostic therapy is based on targeting genomic alterations, regardless of tumor origin. In this context, novel therapeutic agents including BRAF and MEK inhibitors based on the molecular landscape in solid tumors have been investigated. Case presentation, Case 1: The first case is chemotherapy-refractory, BRAF V600E mutated intrahepaticcholangiocarcinoma treated with vemurafenib and cobimetinib as third line therapy. In this setting the dual BRAF and MEK inhibition resulted in improved progression-free survival and quality of life; Case 2: The second case shows aBRAF G466A mutated Bellini duct carcinoma (BDC), treated with dabrafenib and trametinib in second line therapy. The disease remained under control for 11 months after the first relapse. Discussion: In the literature there is strong evidence that melanoma, colorectal cancer, non small cell lung cancer and anaplastic thyroid cancer with BRAF mutations are good targets for BRAF/MEK pathway inhibitors. The VE-BASKET and ROAR basket trials explored the efficacy of vemurafenib and the combination of dabrafenib/trametinib, respectively, in BRAF V600 mutation-positive cancers other than melanoma, papillary thyroid cancer, colorectal cancer and non small cell lung cancer. Within the concept of tumor type agnostic therapy, we decided to treat our BRAF-mutated tumors with the association of BRAF and MEK inhibitors. Conclusions: Our results confirm the emerging importance of molecular tumor profiling for the successful management of cancer, and the potential of BRAF-targeted therapy in the treatment of rare solid tumors with poor prognosis and no clinical benefit from systemic therapies with.

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Section: Discussionmentioning

confidence: 99%

Tumor Type Agnostic Therapy Carrying BRAF Mutation: Case Reports and Review of Literature

Bernocchi

Sirico²,

Corona

et al. 2021

Pharmaceuticals

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“…Since 2017, six cases of BTCs treated with pembrolizumab have been reported ( Table 2 ) ( 14 - 17 ). Among these cases, the responses achieved with ICI treatment were durable.…”

Section: Discussionmentioning

confidence: 99%

Microsatellite Instability-high Intrahepatic Cholangiocarcinoma with Portal Vein Tumor Thrombosis Successfully Treated with Pembrolizumab

et al. 2020

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A 60-year-old man presented with postoperative recurrence of intrahepatic cholangiocarcinoma with right portal vein tumor thrombosis (PVTT). After failure of standard chemotherapy, a liver biopsy showed that his microsatellite instability (MSI) status was high. Treatment with the immune checkpoint inhibitor (ICI) pembrolizumab was commenced, which resulted in a partial response and resolution of the PVTT. There were no significant immune-related adverse events. According to recently published reports, the frequency of MSI-high biliary tract cancer (BTC) is about 0-2.1%, which is extremely rare. However, ICIs may be effective in patients with MSI-high BTC, such as the present patient.

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“…In addition, several case series have published good tolerability and response to this combination [ 22 , 23 , 24 ] and another phase-2 trial, VE-BASKET, demonstrated single agent efficacy of the BRAF inhibitor, vemurafenib, in BRAF V600E mutant cancers. In this study, a total of 208 patients with 26 different types of cancers were enrolled, including nine CCA patients.…”

Section: Potential Clinical Applications Of Molecular Profilingmentioning

confidence: 99%

Recent Progress in the Systemic Treatment of Advanced/Metastatic Cholangiocarcinoma

Fostea

Fontana

Torga

et al. 2020

Cancers

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Cholangiocarcinomas (CCAs) comprise of a heterogeneous group of cancers arising in the biliary tract (intrahepatic or iCCA, perihilar or pCCA and distal or dCCA; the latter are known under the collective term of eCCA), each subtype having its own particularities in carcinogenesis, management and prognosis. The increasing incidence in recent decades, limited treatment options and high mortality rates, even in the early stages, have led to an imperious need for more in-depth understanding and development of tailored treatments for this type of aggressive tumour. The wide use of molecular profiling has increased the understanding of biology and identified key molecular drivers, for example, IDH1 mutations or FGFR2 fusions for iCCA, or BRAF mutations in eCCA. Most recently, the FDA approved pemigatinib, an FGFR inhibitor and ivosidenib, an IDH1 inhibitor, but even though progress has been made to better understand the mechanisms of tumorigenesis, genetic make-up, and tumour resistance to standard chemotherapy and targeted therapies, cholangiocarcinomas still represent an important challenge in the daily clinical practice of oncology. The purpose of this review is to highlight the recent progress in the systemic treatment of advanced/metastatic CCAs with a focus on targeted drugs and their biomarkers currently evaluated in early-phase clinical trials.

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Personalised approach in combined treatment of cholangiocarcinoma: a case report of healing from cholangiocellular carcinoma at stage IV

Cited by 15 publications

References 17 publications

Tumor Type Agnostic Therapy Carrying BRAF Mutation: Case Reports and Review of Literature

Tumor Type Agnostic Therapy Carrying BRAF Mutation: Case Reports and Review of Literature

Microsatellite Instability-high Intrahepatic Cholangiocarcinoma with Portal Vein Tumor Thrombosis Successfully Treated with Pembrolizumab

Recent Progress in the Systemic Treatment of Advanced/Metastatic Cholangiocarcinoma

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