Currently, intrahepatic cholangiocarcinoma (iCCA) is regarded as a contraindication for liver transplantation (LT) in most centers. However, emerging evidence supports the role of LT in selected patients with iCCA. At the 2024 Cholangiocarcinoma Foundation Annual Conference, Sudha Kodali, MD, MSPH, discussed considerations for LT in selected patients with iCCA.
Dr Kodali stated that, although surgical resection is widely considered the only curative treatment, many patients are not candidates for resection due to presentation with multifocal disease, nodal metastasis, advanced stage, or preexisting liver disease. Additionally, following resection, the 5-year overall survival is between 22% and 44%, with most patients showing tumor recurrence at a median of 26 months from resection.
Emerging data have indicated that LT was associated with better outcomes than resection for select patients with iCCA. A retrospective study showed that LT, in combination with neoadjuvant and adjuvant therapy, was superior in terms of recurrence-free survival (RFS) to combination radical bile duct resection and partial hepatectomy plus adjuvant therapy in patients with locally advanced iCCA and hilar CCA.
Predictive factors for worse RFS outcomes with LT included hilar CCA, multifocal tumors, perineural invasion, and partial hepatectomy; tumor sizes ≥5 cm did not predict poor outcomes. Another study showed that LT was associated with improved outcomes among cirrhotic patients with small incidental iCCA or combined hepatocellular CCA on explant; differentiation and tumors <5 cm were the subgroup’s most significant independent factors for recurrence. Patients with very early (single ≤2 cm) tumors had superior pooled 5-year RFS versus advanced iCCA (>2 cm).
Dr Kodali described the Methodist-MD Anderson transplant protocol, consisting of neoadjuvant chemotherapy, disease stability for at least 6 months on the given regimen, and post-transplant adjuvant therapy for 4 to 6 months. A prospective single-center case series from Dr Kodali’s group that used this protocol showed that patients selected based on tumor biological activity and who underwent liver transplantation achieved a 5-year survival of 57%.
In the same series, LT patients’ survival significantly improved compared with listed but not transplanted patients. In another series of non-LT patients with stage I/II iCCA treated with systemic therapy alone, the 5-year survival was 15%.
Dr Kodali stated that “of all the treatment outcomes available, transplantation offers better survival outcomes” but cautioned that more extensive studies are needed to gain more insights. She also shared that molecular genetic tumor profiling of circulating tumor DNA and recurrent tumors is important post-transplantation to tailor personalized therapies.
Dr Kodali concluded that LT remains a viable treatment option for patients with iCCA and that patient selection for LT should not be based on size but on biologic tumor activity, which can be achieved by pretransplant neoadjuvant therapy and molecular genetic tumor profiling.1
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