Immunotherapy and Targeted Therapy for Advanced Biliary Tract Cancer: Adding New Flavors to the Pizza

Queiroz, Marcello Moro; Lima, Nildevande Firmino; Castria, Tiago Biachi De

doi:10.3390/cancers15071970

Cited by 10 publications

(4 citation statements)

References 104 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…The most common gene alterations involving IDH1 are fusions, more frequent in intrahepatic cholangiocarcinoma, followed by amplification and activating mutations, found in extrahepatic tumors. Given their prevalence and the fact that there is a targeted therapeutic option for them, several clinical trials have been conducted in recent years to verify their effectiveness [20]. Ivosidenib and olutasidenib are the most widely used and known IDH1 inhibitors, ivosidenib being the first studied in cholangiocarcinoma in a phase 1 study, with encouraging results [21].…”

Section: Targetable Alterationsmentioning

confidence: 99%

Options in Targeted Therapy for Advanced Cholangiocarcinoma: A 2024 Update

Oprescu Macovei,

Venter,

Makkai

et al. 2024

Cureus

View full text Add to dashboard Cite

Bile duct carcinomas have a different prognosis and genetic profile depending on their location; intrahepatic/extrahepatic or at the level of the gallbladder. Although in recent years there have been important advances in first-line therapy, second-line therapy in cholangiocarcinoma does not currently have a standard. Therefore at this level, there is an acute need for personalized treatment. The present article is a narrative review that aims to list the newest targeted therapeutic options for this type of cancer, based on identified genetic alterations. The literature selected for analysis includes phase 2 or 3 studies with targeted therapy in this disease and original articles no older than three years that describe the prevalence of the most common gene alterations in this type of cancer. PubMed/Medline, Scopus, and Clarivate-Web of Science databases were searched and keywords such as "cholangiocarcinoma," "biliary cancer," "targeted therapy," "gene amplifications," and "mutations" were used. This narrative review was designed taking into account the SANRA (Scale for the Assessment of Narrative Review Articles) criteria. The conclusions lead to the fact that next-generation sequencing testing is of particular usefulness in cholangiocarcinoma. Bile duct cancers are rich in targetable genetic alterations, and their treatment is in constant change, although much of the current data comes from phase II studies. There is a great need for the current options to be analyzed in phase III studies. Hence, the need of the oncological community to stay informed about targeted treatment options for cholangiocarcinoma is supported by the present article.

show abstract

Section: Targetable Alterationsmentioning

confidence: 99%

Options in Targeted Therapy for Advanced Cholangiocarcinoma: A 2024 Update

Oprescu Macovei,

Venter,

Makkai

et al. 2024

Cureus

View full text Add to dashboard Cite

show abstract

“…In a phase II study, everolimus, an mTOR inhibitor, demonstrated some clinical activity as a monotherapy in patients with advanced BTCs [ 65 ]. Current evidence regarding targeted therapy for HC is limited and more work in the pre-clinical setting is necessary to isolate targetable genetic aberrations [ 59 , 66 ].…”

Section: Systemic Therapymentioning

confidence: 99%

Multi-Disciplinary Care of Hilar Cholangiocarcinoma: Review of Guidelines and Recent Advancements

Padmanaban,

Ruff,

Pawlik

2023

Cancers

View full text Add to dashboard Cite

Cholangiocarcinoma (CCA) is a rare malignancy of the intrahepatic and extrahepatic biliary ducts. CCA is primarily defined by its anatomic location: intrahepatic cholangiocarcinoma versus extrahepatic cholangiocarcinoma. Hilar cholangiocarcinoma (HC) is a subtype of extrahepatic cholangiocarcinoma that arises from the common hepatic bile duct and can extend to the right and/or left hepatic bile ducts. Upfront surgery with adjuvant capecitabine is the standard of care for patients who present with early disease and the only curative therapy. Unfortunately, most patients present with locally advanced or metastatic disease and must rely on systemic therapy as their primary treatment. However, even with current systemic therapy, survival is still poor. As such, research is focused on developing targeted therapies and multimodal strategies to improve overall prognosis. This review discusses the work-up and management of HC focused on the most up-to-date literature and ongoing clinical trials.

show abstract

“…Nonetheless, effective medical therapy remains a considerable challenge in the management of these hepatobiliary malignancies. Immunotherapy, such as immune checkpoint inhibitors (ICIs), has shown antitumor responses in a select group of patients [ 8 ]. Notably, current studies indicate that immunotherapy in the form of adoptive cell therapy represents a promising approach in solid tumor malignancies.…”

Section: Introductionmentioning

confidence: 99%

Cost-effectiveness analysis of pembrolizumab in combination with chemotherapy compared with chemotherapy alone as first-line treatment for patients with advanced biliary tract cancer in China

Zheng,

Fang,

Cai

2023

BMC Cancer

View full text Add to dashboard Cite

Objective The objective of this study is to evaluate the cost-effectiveness of adding pembrolizumab to the standard first-line therapy of advanced biliary tract cancer (BTC) with gemcitabine and cisplatin from the perspective of the Chinese healthcare system. Methods The partitioned survival model developed from clinical data obtained in The KEYNOTE-966 trial served as the basis for a simulation in the TreeAge Pro 2011 software. The objective of the research was to estimate the 10-year life expectancy and total healthcare costs of patients with BTC, utilizing primary outcomes that evaluated costs, quality-adjusted life years (QALYs), and incremental cost-effectiveness ratio (ICER). To establish the willingness-to-pay (WTP) threshold, the 2022 Chinese per capita gross domestic product (GDP) of $37304.346/QALY was adopted. Furthermore, sensitivity analysis was conducted to ascertain the study’s results under varying levels of uncertainty. Results Compared to chemotherapy alone, the addition of pembrolizumab to chemotherapy has been shown to yield an incremental gain of 0.184 quality-adjusted life years (QALY) at an additional cost of $103940.706. This translates into an incremental cost-effectiveness ratio (ICER) of $564895.141/QALY, which exceeds the willingness-to-pay (WTP) threshold in China. One-way sensitivity analyses performed on the model recognize the utility of PD, subsequent cost, and the cost of Pembrolizumab 100 mg had a major influence on the outcomes. However, no parameter elicited an ICER lower than the willingness-to-pay (WTP) threshold. Conclusions Based on the perspective of the Chinese healthcare system, the utilization of pembrolizumab in combination with chemotherapy as an first-line treatment option for BTC does not appear to be a cost-effective approach compared to chemotherapy as a standalone therapy.

show abstract

Immunotherapy and Targeted Therapy for Advanced Biliary Tract Cancer: Adding New Flavors to the Pizza

Cited by 10 publications

References 104 publications

Options in Targeted Therapy for Advanced Cholangiocarcinoma: A 2024 Update

Options in Targeted Therapy for Advanced Cholangiocarcinoma: A 2024 Update

Multi-Disciplinary Care of Hilar Cholangiocarcinoma: Review of Guidelines and Recent Advancements

Cost-effectiveness analysis of pembrolizumab in combination with chemotherapy compared with chemotherapy alone as first-line treatment for patients with advanced biliary tract cancer in China

Contact Info

Product

Resources

About