Patient healthcare trajectories of intrahepatic cholangiocarcinoma in France: A nationwide retrospective analysis

Neuzillet, Cindy; Émery, C.; Teissier, C.; Bouée, S.; Lièvre, Astrid

doi:10.1016/j.lanepe.2022.100324

Cited by 17 publications

(18 citation statements)

References 20 publications

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“…for biliary stenting), but at present it is difficult to quantify their impact on outcomes in the absence of specific studies. In line with other reports ( 27 ), our study further underlines the aggressiveness of biliary tract cancers, and the need for better treatment options as well as specialized management in trained centers: this could contribute to offering effective palliative measures, with the aim of increasing the chances of receiving active therapeutic options in the advanced setting. Future therapeutic combinations with novel agents and the routine introduction of individualized therapy with targeted agents has the potential to improve overall outcomes in selected subgroups of patients.…”

Section: Discussionsupporting

confidence: 89%

Survival trends over 20 years in patients with advanced cholangiocarcinoma: Results from a national retrospective analysis of 922 cases in Italy

et al. 2023

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Cholangiocarcinoma is a rare group of tumors that involve the hepatic biliary tree. Prognosis for patients with cholangiocarcinoma remains dismal. Herein, we present survival trends over a long time period spanning almost 20 years in patients with advanced cholangiocarcinoma receiving systemic chemotherapy. We retrospectively analyzed a large multicenter dataset of cholangiocarcinoma outpatients evaluated in 14 centers within the Cholangiocarcinoma Italian Group Onlus (Gruppo Italiano Colangiocarcinoma Onlus, G.I.C.O.) between 2000 and 2017 (first-line), and 2002 and 2017 (second-line). Three time periods were considered: 2000-2009, 2010-2013, and 2014-2017. A total of 922 patients (51.19% male) with cholangiocarcinoma undergoing first-line therapy were evaluated. The median durations of follow-up for progression-free survival (PFS) and overall survival (OS) were 37 and 57 months, respectively. PFS at 12 months in the three periods of starting first-line therapy was similar, ranging from 11.71% to 15.25%. OS at 12 months progressively improved (38.30%, 44.61% and 49.52%, respectively), although the differences were not statistically significant after adjusting for age, disease status, and primary tumor site. A total of 410 patients (48.5% male) underwent second-line chemotherapy. The median durations of follow-up for PFS and OS were 47.6 and 41.90 months, respectively. An OS of 24.3%, 32.3%, and 33.1% was observed in 2002-2009, 2010-2013, and 2014-2017, respectively. Despite incremental benefits across years, our clinical experience confirms that modest overall advances have been achieved with first- and second-line chemotherapy in advanced cholangiocarcinoma. Efforts should focus on the identification of patients who derive the greatest benefit from treatment.

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Section: Discussionsupporting

confidence: 89%

Survival trends over 20 years in patients with advanced cholangiocarcinoma: Results from a national retrospective analysis of 922 cases in Italy

et al. 2023

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“…In Western countries, including the United Kingdom (UK) and the United States of America (USA), the incidence of iCCA is increasing while the incidence of eCCA remains relatively stable [20][21][22]. A recent retrospective analysis of a French nationwide hospital database estimated the incidence of newly diagnosed iCCA as 1,825 patients per year (3650 new cases between 2014 and 2015) [23]. The study investigators noted this was higher than the incidence of iCCA recorded in earlier French registry studies [24].…”

Section: Epidemiology Of Biliary Tract Cancermentioning

confidence: 99%

“…For patients with advanced disease, the 5-year survival rate was < 5% [ 7 ]. The abovementioned French nationwide study underlines the aggressive nature of BTC, with one-quarter of patients dying during their first hospital stay and two-thirds of patients not having access to active treatment (receiving only BSC) [ 23 ].…”

Section: Epidemiology Of Biliary Tract Cancermentioning

confidence: 99%

“…Interestingly, the proportion of patients receiving BSC decreased between 1976 and 2005, and the proportion of patients receiving palliative chemotherapy and/or radiotherapy, as well as curative-intent resection, increased over this period [ 7 ]. However, this shift has somewhat plateaued more recently, as attested by the abovementioned French nation-wide hospital database study of 3650 newly diagnosed iCCA cases (assessed between 2014 and 2015), in which 11.2% of patients underwent surgery, 23.8% received chemotherapy, and 65.0% received BSC [ 23 ]. Patient care was mainly provided by general hospitals (59.8%), rather than university hospitals (15.0%) and private (19.2%) or cancer centers (6.0%).…”

Section: Current Treatments For Btcmentioning

confidence: 99%

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Optimizing Patient Pathways in Advanced Biliary Tract Cancers: Recent Advances and a French Perspective

et al. 2023

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Biliary tract cancers (BTCs) are a heterogeneous group of tumors that are rare in Western countries and have a poor prognosis. Three subgroups are defined by their anatomical location (intrahepatic cholangiocarcinoma, extrahepatic cholangiocarcinoma and gallbladder carcinoma) and exhibit distinct clinical, molecular, and epidemiologic characteristics. Most patients are diagnosed at an advanced disease stage and are not eligible for curative-intent resection. In addition to first-and second-line chemotherapies (CisGem and FOLFOX, respectively), biologic therapies are now available that target specific genomic alterations identified in BTC. To date, targets include alterations in the genes for isocitrate dehydrogenase (IDH) 1, fibroblast growth factor receptor (FGFR) 2, v-raf murine sarcoma viral oncogene homolog B1 (BRAF), human epidermal growth factor receptor 2 (HER2 or ERRB2), and neurotrophic tyrosine receptor kinase (NTRK), and for those leading to DNA mismatch repair deficiency. Therapies targeting these genomic alterations have demonstrated clinical benefit for patients with BTC. Despite these therapeutic advancements, genomic diagnostic modalities are not widely used in France, owing to a lack of clinician awareness, local availability of routine genomic testing, and difficulties in obtaining health insurance reimbursement. The addition of durvalumab, a monoclonal antibody targeting the immune checkpoint programmed cell death ligand-1, to CisGem in the first-line treatment of advanced BTC has shown an overall survival benefit in the TOPAZ-1 trial. Given the high mortality rates associated with BTC and the life-prolonging therapeutic options now available, it is hoped that the data presented here will support updates to the clinical management of BTC in France. Key PointsBiliary tract cancers (BTCs) are a rare heterogeneous group of aggressive epithelial cancers that, due to a lack of symptoms in the early stage of disease, are typically diagnosed at an advanced stage when the disease is unresectable or has metastasized.While treatments for this advanced disease are limited, recent advances that offer the opportunity for more personalized therapy for patients with BTC include therapies specific to the more common genetic alterations, as well as immunotherapies that mediate immune recognition of the tumors. This review comprises data from recent publications on the efficacy of treatment options, in addition to expert opinions on the current state of BTC treatment in France.

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“…A recent analysis of 3,650 iCCA cases from a French nationwide hospitalisation database revealed that the vast majority of patients (65%) were managed with the best supportive care only and over 70% were treated in low/intermediate volume centres with minimal iCCA experience. 37 Summing up, Malka highlighted that the last decade in BTC has seen the emergence of SOC across all disease settings, with the immunochemotherapy triplet regimen of durvalumab-gem-cis potentially becoming the new first-line SOC. 18,35,36 BTC, especially iCCA, is a target-rich disease, underscoring the importance of a systemic policy of early molecular profiling within or before the first line.…”

Section: Juan Vallementioning

confidence: 99%

Targeting Fibroblast Growth Factor Receptor Alterations in Cholangiocarcinoma and Other Solid Tumours: A New Frontier in Personalised Medicine

Braud¹,

Valle²,

Lamarca³

et al. 2022

EMJ Oncol

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Cholangiocarcinoma (CCA) is a cancer of the bile duct with poor prognosis and increasing incidence. Filippo de Braud gave an overview of CCA and its classification and highlighted key unmet needs in both diagnosis and treatment. Juan Valle explained that intrahepatic CCA (iCCA) accounts for 11% of the entire cancer of unknown primary (CUP) population, as well as 33% of patients with liver-inclusive disease. It is, therefore, vital that CUP with a ‘liver-dominant lesion’ is reviewed by a specialist team familiar with iCCA management. Angela Lamarca emphasised that managing CAA requires a multidisciplinary approach to both diagnosis and treatment. Precision medicine is now a reality in biliary tract carcinoma (BTC), particularly iCCA, so molecular testing is essential to ensure targeted therapy options are considered. David Malka explored existing and emerging standard-of-care (SOC) treatments for advanced BTC, which represents a target-rich disease. Updated guidelines now recommend targeted treatment for second-line iCCA in patients with appropriate alterations, underscoring the importance of systematic and early molecular profiling. A wide range of malignancies harbour FGFR alterations at varying frequencies, and the FGFR inhibitor pemigatinib is already approved for the treatment of CCA with FGFR2 fusions or rearrangements. Antoine Hollebecque highlighted the final results from the Phase II FIGHT-202 study, in which pemigatinib achieved an overall response rate (ORR) of 37% in the target population with a manageable safety profile.

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Patient healthcare trajectories of intrahepatic cholangiocarcinoma in France: A nationwide retrospective analysis

Cited by 17 publications

References 20 publications

Survival trends over 20 years in patients with advanced cholangiocarcinoma: Results from a national retrospective analysis of 922 cases in Italy

Survival trends over 20 years in patients with advanced cholangiocarcinoma: Results from a national retrospective analysis of 922 cases in Italy

Optimizing Patient Pathways in Advanced Biliary Tract Cancers: Recent Advances and a French Perspective

Targeting Fibroblast Growth Factor Receptor Alterations in Cholangiocarcinoma and Other Solid Tumours: A New Frontier in Personalised Medicine

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