Durable response after olaparib treatment for perihilar cholangiocarcinoma with germline BRCA2 mutation Journal Article
| Authors: | Costa, B. A.; Tallón de Lara, P.; Park, W.; Keane, F.; Harding, J. J.; Khalil, D. N. |
| Article Title: | Durable response after olaparib treatment for perihilar cholangiocarcinoma with germline BRCA2 mutation |
| Abstract: | INTRODUCTION: Despite major advances in surveillance and management, advanced cholangiocarcinoma (CCA) still carries a dismal prognosis. In recent years, several actionable genomic alterations in pancreatobiliary malignancies have been identified. For instance, homologous recombination deficiency (HRD) has been considered a predictive biomarker of clinical response to platinum and poly (ADP-ribose) polymerase (PARP) inhibitors. CASE REPORT: A 53-year-old man with a stage 3 (T4N0M0) BRCA2-mutant CCA developed intolerable toxicity after 44 cycles of gemcitabine/cisplatin. In light of his HRD positivity, treatment was switched to single-agent olaparib. The patient showed a partial radiological response, which was maintained after 8 months of olaparib discontinuation (progression-free survival >36 months). CONCLUSION: Given the durable response observed, olaparib can be a valuable therapeutic tool in BRCA-mutant CCAs. Ongoing and future clinical trials are needed to confirm the role of PARP inhibition in similar patients and to define the clinicopathological and molecular profile of the individuals most likely to benefit. © 2023 S. Karger AG, Basel. |
| Keywords: | middle aged; genetics; mutation; case report; ovarian neoplasms; germ cell; pathology; brca2 protein; germ cells; ovary tumor; nicotinamide adenine dinucleotide adenosine diphosphate ribosyltransferase inhibitor; bile duct neoplasms; bile ducts, intrahepatic; cholangiocarcinoma; nicotinamide adenine dinucleotide adenosine diphosphate ribosyltransferase; biliary tract cancers; klatskin tumor; olaparib; personalized medicine; bile duct tumor; poly(adp-ribose) polymerases; intrahepatic bile duct; parp inhibitor; brca2 mutation; brca2 protein, human; humans; human; male; female; poly(adp-ribose) polymerase inhibitors |
| Journal Title: | Oncology Research and Treatment |
| Volume: | 46 |
| Issue: | 5 |
| ISSN: | 2296-5270 |
| Publisher: | S. Karger AG |
| Date Published: | 2023-05-01 |
| Start Page: | 211 |
| End Page: | 215 |
| Language: | English |
| DOI: | 10.1159/000529919 |
| PUBMED: | 36882017 |
| PROVIDER: | scopus |
| DOI/URL: | |
| Notes: | Article -- MSK corresponding author is Danny Khalil -- Source: Scopus |
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