The Cholangiocarcinoma in the Young (CITY) Study: Tumor Biology, Treatment Patterns, and Survival Outcomes in Adolescent Young Adults With Cholangiocarcinoma

Author:

Pappas Leontios12ORCID,Baiev Islam1,Reyes Stephanie3,Bocobo Andrea Grace4,Jain Apurva5ORCID,Spencer Kristen6ORCID,Le Tri Minh7,Rahma Osama E.8ORCID,Maurer Jordan1,Stanton Jen1,Zhang Karen4ORCID,De Armas Anaemy Danner5ORCID,Deleon Thomas T.9,Roth Marc10ORCID,Peters Mary Linton B.11ORCID,Zhu Andrew X.1213ORCID,Boyhen Kylie14,VanCott Christine15,Patel Tushar16,Roberts Lewis R.17ORCID,Lindsey Stacie18ORCID,Horick Nora1,Lennerz Jochen K.19ORCID,Iafrate A. John19,Goff Laura Williams20ORCID,Mody Kabir21ORCID,Borad Mitesh J.9ORCID,Shroff Rachna T.22,Javle Milind M.5ORCID,Kelley R. Katie4ORCID,Goyal Lipika1223ORCID

Affiliation:

1. Department of Medicine, Massachusetts General Hospital Cancer Center, Boston, MA

2. Harvard Medical School, Boston, MA

3. Duke University School of Medicine, Durham, NC

4. Helen Diller Family Comprehensive Cancer Center, University of California San Francisco, San Francisco, CA

5. Division of Cancer Medicine, Department of Gastrointestinal Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX

6. Department of Medicine, NYU Langone Health Perlmutter Cancer Center, NYU School of Medicine, New York, NY

7. Department of Medicine, University of Virginia Comprehensive Cancer Center, Charlottesville, VA

8. Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA

9. Division of Hematology/Oncology, Mayo Clinic, Scottsdale, AZ

10. Department of Medical Oncology, St Luke's Cancer Institute, Kansas City, MO

11. Department of Medicine, Beth Israel Deaconess Medical Center, Boston, MA

12. Jiahui International Cancer Center, Jiahui Health, Shanghai, China

13. I-MAB Biopharma, Shanghai, China

14. Yale University, New Haven, CT

15. Department of Medicine, HCA Florida South Tampa Hospital, Tampa, FL

16. Department of Transplantation, Mayo Clinic, Jacksonville, FL

17. Division of Gastroenterology and Hepatology, Mayo Clinic, Rochester, MN

18. The Cholangiocarcinoma Foundation, Herriman, UT

19. Center for Integrated Diagnostics, Department of Pathology, Massachusetts General Hospital/Harvard Medical School, Boston, MA

20. Department of Medicine, Vanderbilt-Ingram Cancer Center, Nashville, TN

21. Division of Hematology/Oncology, Mayo Clinic, Jacksonville, FL

22. University of Arizona Cancer Center, University of Arizona, Tucson, AZ

23. Department of Medicine, Division of Oncology, Stanford Cancer Center, Palo Alto, CA

Abstract

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.

Publisher

American Society of Clinical Oncology (ASCO)

Subject

Cancer Research,Oncology

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