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Background <p>High-risk features (e.g., vascular invasion, lymph node involvement, multifocality, elevated carbohydrate antigen 19-9 [CA19-9], tumor size &gt;5 cm) in intrahepatic cholangiocarcinoma (iCCA) are associated with poor prognosis, leading to increased use of neoadjuvant therapy (NAT).</p> Methods <p>Patients (<i>n</i> = 425) treated in 2000–2024 at Mayo Clinic Rochester were categorized into four groups: (1) high-risk iCCA with NAT (chemotherapy- or chemoimmunotherapy-based regimens)&#xa0;followed by surgery, (2) high-risk iCCA with upfront resection, (3) low-risk iCCA with upfront resection, and (4) high-risk iCCA receiving NAT without surgery (“dropouts”). Outcomes included overall (OS) and recurrence-free survival (RFS). An external cohort of 203 iCCA patients from MD Anderson Cancer Center also was analyzed using identical criteria.</p> Results <p>Of 425 patients, 342 (80.5%) were at high risk and 83 (19.5%) were at low risk (all upfront resection). Among the high-risk patients, 172 (50.3%) received NAT and 96 (55.8%) underwent upfront resection, whereas 76 (44.2%) dropped out. The NAT regimens included doublet gemcitabine-cisplatin (GC, <i>n</i> = 56) and triplet chemo(immuno)therapy with gemcitabine-cisplatin–nab-paclitaxel (GAP, <i>n</i> = 30) or gemcitabine-cisplatin–durvalumab (GCD, <i>n</i> = 10). The NAT patients who proceeded to resection showed significantly longer OS than those who had upfront resection (68.6 vs. 34.9 months; <i>p</i> = 0.007), with no significant difference from low-risk patients (106.2 months, <i>p</i> = 0.132). Intensified regimens (GAP/GCD) yielded superior OS, whereas RFS did not significantly differ across groups. Comparable survival trends were observed in the external cohort.</p> Conclusion <p>Neoadjuvant therapy followed by curative-intent resection was associated with improved OS, reflecting selection of systemic therapy-responsive high-risk patients with more favorable tumor biology, achieving outcomes similar to those of low-risk groups. Conversely, 45% failed to proceed to surgery due to progression or clinical deterioration, experiencing OS comparable with palliative care.</p>

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Neoadjuvant Therapy as a Selection Strategy for Curative Resection in High-Risk Intrahepatic Cholangiocarcinoma

  • 期刊论文

  • 发表日期：

    2026年5月4日

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  • Yawen Dong,
  • Kever A. Lewis,
  • David Pereyra,
  • Zhihao Li,
  • Vanja Podrascanin,
  • Galen C. Gist,
  • Jonas Santol,
  • Markus Ammann,
  • Lionel A. Kankeu Fonkoua ,
  • Rondell P. Graham,
  • Caitlin B. Conboy,
  • Nguyen H. Tran,
  • Susanne G. Warner,
  • Jean-Nicolas Vauthey,
  • Timothy E. Newhook,
  • Hop S. Tran Cao,
  • Rory L. Smoot,
  • Patrick Starlinger

摘要

Background

High-risk features (e.g., vascular invasion, lymph node involvement, multifocality, elevated carbohydrate antigen 19-9 [CA19-9], tumor size >5 cm) in intrahepatic cholangiocarcinoma (iCCA) are associated with poor prognosis, leading to increased use of neoadjuvant therapy (NAT).

Methods

Patients (n = 425) treated in 2000–2024 at Mayo Clinic Rochester were categorized into four groups: (1) high-risk iCCA with NAT (chemotherapy- or chemoimmunotherapy-based regimens) followed by surgery, (2) high-risk iCCA with upfront resection, (3) low-risk iCCA with upfront resection, and (4) high-risk iCCA receiving NAT without surgery (“dropouts”). Outcomes included overall (OS) and recurrence-free survival (RFS). An external cohort of 203 iCCA patients from MD Anderson Cancer Center also was analyzed using identical criteria.

Results

Of 425 patients, 342 (80.5%) were at high risk and 83 (19.5%) were at low risk (all upfront resection). Among the high-risk patients, 172 (50.3%) received NAT and 96 (55.8%) underwent upfront resection, whereas 76 (44.2%) dropped out. The NAT regimens included doublet gemcitabine-cisplatin (GC, n = 56) and triplet chemo(immuno)therapy with gemcitabine-cisplatin–nab-paclitaxel (GAP, n = 30) or gemcitabine-cisplatin–durvalumab (GCD, n = 10). The NAT patients who proceeded to resection showed significantly longer OS than those who had upfront resection (68.6 vs. 34.9 months; p = 0.007), with no significant difference from low-risk patients (106.2 months, p = 0.132). Intensified regimens (GAP/GCD) yielded superior OS, whereas RFS did not significantly differ across groups. Comparable survival trends were observed in the external cohort.

Conclusion

Neoadjuvant therapy followed by curative-intent resection was associated with improved OS, reflecting selection of systemic therapy-responsive high-risk patients with more favorable tumor biology, achieving outcomes similar to those of low-risk groups. Conversely, 45% failed to proceed to surgery due to progression or clinical deterioration, experiencing OS comparable with palliative care.