A Systematised Literature Review of Real-World Treatment Patterns and Outcomes in Unresectable Advanced or Metastatic Biliary Tract Cancer

Peirce, Vivian; Paskow, Michael; Qin, Lei; Dadzie, Ruby; Rapoport, Maria; Prince, Samantha; Johal, Sukhvinder

doi:10.1007/s11523-023-01000-5

Targ Oncol

2023

DOI: 10.1007/s11523-023-01000-5

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A Systematised Literature Review of Real-World Treatment Patterns and Outcomes in Unresectable Advanced or Metastatic Biliary Tract Cancer

Vivian Peirce,

Michael Paskow,

Lei Qin

et al.

Abstract: Background Biliary tract cancers are rare aggressive malignancies typically diagnosed when the disease is metastatic or unresectable, precluding curative treatment. Objective We aimed to identify treatment guidelines, real-world treatment patterns, and outcomes for unresectable advanced or metastatic biliary tract cancers in adult patients. Methods Databases (MEDLINE, Embase, Cochrane Database of Systematic Reviews) were systematically searched between

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2024

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Cited by 3 publications

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Survival after targeted chemotherapy and unresectable biliary malignancies (systematic review and meta-analysis)

Tseimakh,

Mitshhenko,

Shoikhet

2024

Ann. hir. gepatol.

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Aim. To evaluate the survival rate of patients with unresectable biliary malignancies under targeted chemotherapy in order to identify the most promising adjuvant regimens.Materials and methods. The study involves publications in PubMed Central, RSCI, and Cochrane databases. Heterogeneity was assessed graphically (blobograms) and statistically (τ 2 and I2 ).Results. Meta-analysis of five-year survival revealed a greater pooled estimate of the period in the main groups treated with targeted chemotherapy – 295 ± 71 days (95% CI 144–408; p <0.001) against comparison groups – 205 ± 81 days (95% CI 81–426; p <0.001). Study heterogeneity was considered moderate (I2 = 0%, p = 0.06). No significant publication errors and biases were revealed in both meta-analyses.Conclusion. Targeted chemotherapy increases the overall survival of patients with unresectable malignancies of the bile ducts. Systemic chemotherapy based on gemcitabine and cisplatin with addition of ivosidenib, a selective inhibitor of mutant IDH1, showed the best efficacy.

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Survival after targeted chemotherapy and unresectable biliary malignancies (systematic review and meta-analysis)

Tseimakh,

Mitshhenko,

Shoikhet

2024

Ann. hir. gepatol.

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Effectiveness of palliative photodynamic therapy for unresectable biliary cancer. Systematic review and meta-analysis

Tseimakh,

Mitshenko,

Kurtukov

et al. 2024

Biomedical photonics

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A systematic review and meta-analysis was aimed to assess the effectiveness of palliative photodynamic therapy for unresectable malignant tumors of the biliary system in order to justify the feasibility of including photodynamic therapy (PDT) in the complex treatment of this category of patients. Publications in the databases PubMed Central, the bibliographic database of scientifi citations of the RSCI, and the Cochrane library were considered. Heterogeneity was assessed graphically using forest plots and statistically using tau2 and I2 statistics. A meta-analysis of 5-year survival revealed a statistically signifiantly longer pooled estimate of the survival period in groups where PDT was used – 339±161 days (95% CI 25-710; p < 0.001) compared to groups where PDT was not used – 83±16 days (95% CI 33-100; p < 0.001). Heterogeneity among studies was found to be statistically insignifiant (I2 = 29%, p = 0.23). A meta-analysis of the risk difference for adverse events revealed a statistically signifiantly lower risk (-0.2306; 95% CI -0.3917-0.0696; p = 0.005) of adverse events after PDT compared with the comparison group. Heterogeneity among studies was found to be statistically insignifiant (I2 = 0%, p = 0.35). There were no signifiant publication biases in either meta-analysis. The presented meta-analysis demonstrated that PDT may be the method of choice in the palliative complex treatment of patients with unresectable cholangicarcinomas, increasing the ˝ve-year survival of patients along with the absence of increased risks of postoperative complications in comparison with other methods of palliative surgical treatment.

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Cost-effectiveness of pembrolizumab plus chemotherapy vs. chemotherapy as first-line treatment for advanced biliary tract cancer in China and the US

Luo,

Cai,

et al. 2024

Front. Pharmacol.

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Background: Pembrolizumab is a potentially valuable treatment. However, patients, doctors, and healthcare decision-makers are uncertain about its cost-effectiveness and an appropriate pricing for this new therapy. This study aims to appraise the cost-effectiveness of pembrolizumab as a first-line treatment for advanced biliary tract cancer (BTC) patients in China and the United States (US).Methods: A Markov model was constructed from the perspectives of healthcare systems in both China and the US for pharmacoeconomic evaluation. Patient baseline characteristics and key clinical data were sourced from the KEYNOTE-966 trial (ClinicalTrials.gov, NCT04003636). Costs and utilities were collected from drug cost websites and published literature. Cumulative costs (in USD), life years (LYs), quality-adjusted life years (QALYs), and incremental cost-effectiveness ratios (ICERs) were measured and compared. Price simulations were conducted under given willingness-to-pay (WTP) thresholds to provide pricing scheme references. The model’s robustness was analyzed through one-way sensitivity analysis and probabilistic sensitivity analysis.Results: Basic data analysis illustrates that pembrolizumab ($2662.41/100 mg) in combination with chemotherapy regimen was not cost-effective relative to chemotherapy regimens at the WTP threshold of $38,201.19 in China, and the additional cost relative to chemotherapy regimens was $77,114.94 (ICER $556,689.47/QALY) while increasing 0.14 QALYs. Pembrolizumab ($54.71/1 mg) also increased efficacy by 0.14 QALYs in the US, but remained also not cost-effective at the US WTP threshold of $229,044, and the total cost increased by $160,425.24 (ICER $1,109,462.92/QALY).Conclusion: Compared with chemotherapy, pembrolizumab plus chemotherapy reduces the disease of burden. However, at its current price, it may not be a cost-effective treatment for advanced BTC in both China and the US. This study can aid decision-makers in making optimal choices.

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A Systematised Literature Review of Real-World Treatment Patterns and Outcomes in Unresectable Advanced or Metastatic Biliary Tract Cancer

Cited by 3 publications

References 98 publications

Survival after targeted chemotherapy and unresectable biliary malignancies (systematic review and meta-analysis)

Survival after targeted chemotherapy and unresectable biliary malignancies (systematic review and meta-analysis)

Effectiveness of palliative photodynamic therapy for unresectable biliary cancer. Systematic review and meta-analysis

Cost-effectiveness of pembrolizumab plus chemotherapy vs. chemotherapy as first-line treatment for advanced biliary tract cancer in China and the US

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