TY - JOUR
T1 - The Cholangiocarcinoma in the Young (CITY) Study
T2 - Tumor Biology, Treatment Patterns, and Survival Outcomes in Adolescent Young Adults With Cholangiocarcinoma
AU - Pappas, Leontios
AU - Baiev, Islam
AU - Reyes, Stephanie
AU - Bocobo, Andrea Grace
AU - Jain, Apurva
AU - Spencer, Kristen
AU - Le, Tri Minh
AU - Rahma, Osama E.
AU - Maurer, Jordan
AU - Stanton, Jen
AU - Zhang, Karen
AU - De Armas, Anaemy Danner
AU - Deleon, Thomas T.
AU - Roth, Marc
AU - Peters, Mary Linton B.
AU - Zhu, Andrew X.
AU - Boyhen, Kylie
AU - VanCott, Christine
AU - Patel, Tushar
AU - Roberts, Lewis R.
AU - Lindsey, Stacie
AU - Horick, Nora
AU - Lennerz, Jochen K.
AU - Iafrate, A. John
AU - Goff, Laura Williams
AU - Mody, Kabir
AU - Borad, Mitesh J.
AU - Shroff, Rachna T.
AU - Javle, Milind M.
AU - Kelley, R. Katie
AU - Goyal, Lipika
N1 - Publisher Copyright:
© 2023 by American Society of Clinical Oncology.
PY - 2023
Y1 - 2023
N2 - PURPOSE Increased awareness of the distinct tumor biology for adolescents and young adults (AYAs) with cancer has led to improvement in outcomes for this population. However, in cholangiocarcinoma (CCA), a paucity of data exist on the AYA population. To our knowledge, we present the largest study to date on AYA disease biology, treatment patterns, and survival outcomes in CCA. METHODS A multi-institutional cohort of patients with CCA diagnosed with intrahepatic cholangiocarcinoma (ICC) or extrahepatic cholangiocarcinoma (ECC) was used for analysis. Retrospective chart review was conducted on patients who were 50 years old and younger (young; n = 124) and older than 50 years (older; n = 723). RESULTS Among 1, 039 patients screened, 847 patients met eligibility (72% ICC, 28% ECC). Young patients had a larger median tumor size at resection compared with older patients (4.2 v 3.6 cm; P = .048), more commonly had N1 disease (65% v 43%; P = .040), and were more likely to receive adjuvant therapy (odds ratio, 4.0; 95% CI, 1.64 to 9.74). Tumors of young patients were more likely to harbor an FGFR2 fusion, BRAF mutation, or ATM mutation (P, .05 for each). Young patients were more likely to receive palliative systemic therapy (96% v 69%; P, .001), targeted therapy (23% v 8%; P, .001), and treatment on a clinical trial (31% v 19%; P = .004). Among patients who presented with advanced disease, young patients had a higher median overall survival compared with their older counterparts (17.7 v 13.5 months; 95% CI, 12.6 to 22.6 v 11.4 to 14.8; P = .049). CONCLUSION Young patients with CCA had more advanced disease at resection, more commonly received both adjuvant and palliative therapies, and demonstrated improved survival compared with older patients. Given the low clinical trial enrollment and poor outcomes among some AYA cancer populations, data to the contrary in CCA are highly encouraging.
AB - PURPOSE Increased awareness of the distinct tumor biology for adolescents and young adults (AYAs) with cancer has led to improvement in outcomes for this population. However, in cholangiocarcinoma (CCA), a paucity of data exist on the AYA population. To our knowledge, we present the largest study to date on AYA disease biology, treatment patterns, and survival outcomes in CCA. METHODS A multi-institutional cohort of patients with CCA diagnosed with intrahepatic cholangiocarcinoma (ICC) or extrahepatic cholangiocarcinoma (ECC) was used for analysis. Retrospective chart review was conducted on patients who were 50 years old and younger (young; n = 124) and older than 50 years (older; n = 723). RESULTS Among 1, 039 patients screened, 847 patients met eligibility (72% ICC, 28% ECC). Young patients had a larger median tumor size at resection compared with older patients (4.2 v 3.6 cm; P = .048), more commonly had N1 disease (65% v 43%; P = .040), and were more likely to receive adjuvant therapy (odds ratio, 4.0; 95% CI, 1.64 to 9.74). Tumors of young patients were more likely to harbor an FGFR2 fusion, BRAF mutation, or ATM mutation (P, .05 for each). Young patients were more likely to receive palliative systemic therapy (96% v 69%; P, .001), targeted therapy (23% v 8%; P, .001), and treatment on a clinical trial (31% v 19%; P = .004). Among patients who presented with advanced disease, young patients had a higher median overall survival compared with their older counterparts (17.7 v 13.5 months; 95% CI, 12.6 to 22.6 v 11.4 to 14.8; P = .049). CONCLUSION Young patients with CCA had more advanced disease at resection, more commonly received both adjuvant and palliative therapies, and demonstrated improved survival compared with older patients. Given the low clinical trial enrollment and poor outcomes among some AYA cancer populations, data to the contrary in CCA are highly encouraging.
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U2 - 10.1200/PO.22.00594
DO - 10.1200/PO.22.00594
M3 - Article
C2 - 37561981
AN - SCOPUS:85190369349
SN - 2473-4284
VL - 7
JO - JCO Precision Oncology
JF - JCO Precision Oncology
M1 - e2200594
ER -