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Durable response to first-line PARP inhibition in BRCA-mutated metastatic cholangiocarcinoma: case report.


ABSTRACT:

Background

Cholangiocarcinoma (CCA) is an increasingly prevalent malignancy worldwide, with poor outcomes even when diagnosed at an early stage. While recent trials have shown benefit from the addition of immunotherapy to standard-of-care chemotherapy, the improvement in overall survival is modest. Multiple novel therapies for advanced CCA targeting actionable genetic alterations have been approved in recent years; BRCA1/2 mutations are identified in up to 5% of CCA patients and may be an additional target for novel treatment approaches. While BRCA mutations have been shown in clinical trials to predict response to poly(ADP-ribose) polymerase (PARP) inhibitors in several solid tumors including breast, ovarian, prostate, and pancreas, no similar large-scale trials have

SUBMITTER: Smith JT 

PROVIDER: S-EPMC10772693 | biostudies-literature | 2023 Dec

REPOSITORIES: biostudies-literature