PMID- 36291857 OWN - NLM STAT- PubMed-not-MEDLINE LR - 20221030 IS - 2072-6694 (Print) IS - 2072-6694 (Electronic) IS - 2072-6694 (Linking) VI - 14 IP - 20 DP - 2022 Oct 17 TI - Newest Therapies for Cholangiocarcinoma: An Updated Overview of Approved Treatments with Transplant Oncology Vision. LID - 10.3390/cancers14205074 [doi] LID - 5074 AB - A minority of cholangiocarcinoma (CCA) can be cured by surgical intervention (i.e., liver resection (LR) and liver transplantation (LT)). When modern criteria for LT are met, this intervention along with neoadjuvant treatments may achieve unprecedented survival in selected patients. Liver resection is associated with a median overall survival (OS) of 40 months, this number drastically decreases for unresectable advanced cholangiocarcinoma (CCA), which is treated with systemic therapy. The first-line chemotherapy regimen of gemcitabine and cisplatin is associated with a median overall survival of only 11.7 months. Since the Food and Drug Administration (FDA)'s approval of the isocitrate dehydrogenase (IDH) 1 inhibitor ivosidenib in August 2021, there has been increasing interest in targeted therapy for CCA patients harboring mutations in fibroblast growth factor receptor (FGFR) 2, neurotrophic receptor tyrosine kinase (NTRK), B-raf kinase (BRAF), and HER2. At the same time, immunotherapy with immune checkpoint inhibitors isalso being used in relapsed CCA. This review looks into the most recently completed and ongoing studies of targeted therapy as monotherapy or in combination with chemo- and/or immunotherapy. Whether it is resection, liver transplant, radiotherapy, chemotherapy, immunotherapy, targeted therapy, or any combination of these treatment modalities, great strides are being made to improve outcomes for this challenging disease. FAU - Zhang, Yuqi AU - Zhang Y AD - Department of Medicine, Houston Methodist Hospital, Houston, TX 77030, USA. FAU - Esmail, Abdullah AU - Esmail A AUID- ORCID: 0000-0002-2337-8351 AD - Section of GI Oncology, Department of Medical Oncology, Houston Methodist Cancer Center, Houston, TX 77030, USA. AD - Cancer Clinical Trials, Houston Methodist Research Institute, Houston, TX 77030, USA. FAU - Mazzaferro, Vincenzo AU - Mazzaferro V AD - Department of Oncology, University of Milan, 20133 Milan, Italy. AD - Gastro-Intestinal Surgery and Liver Transplantation Unit, The Instituto Nazionale Tumori (National Cancer Institute) of Milan, 20133 Milan, Italy. FAU - Abdelrahim, Maen AU - Abdelrahim M AD - Section of GI Oncology, Department of Medical Oncology, Houston Methodist Cancer Center, Houston, TX 77030, USA. AD - Weill Cornell Medical College, New York, NY 14853, USA. AD - Cockrell Center for Advanced Therapeutic Phase I program, Houston Methodist Research Institute, Houston, TX 77030, USA. LA - eng PT - Journal Article PT - Review DEP - 20221017 PL - Switzerland TA - Cancers (Basel) JT - Cancers JID - 101526829 PMC - PMC9600404 OTO - NOTNLM OT - cholangiocarcinoma OT - immunotherapy OT - targeted therapy OT - transplant oncology COIS- The authors declare no conflict of interest. EDAT- 2022/10/28 06:00 MHDA- 2022/10/28 06:01 PMCR- 2022/10/17 CRDT- 2022/10/27 01:14 PHST- 2022/08/25 00:00 [received] PHST- 2022/10/09 00:00 [revised] PHST- 2022/10/12 00:00 [accepted] PHST- 2022/10/27 01:14 [entrez] PHST- 2022/10/28 06:00 [pubmed] PHST- 2022/10/28 06:01 [medline] PHST- 2022/10/17 00:00 [pmc-release] AID - cancers14205074 [pii] AID - cancers-14-05074 [pii] AID - 10.3390/cancers14205074 [doi] PST - epublish SO - Cancers (Basel). 2022 Oct 17;14(20):5074. doi: 10.3390/cancers14205074.