Exploratory Phase II Study of Atractylodes lancea (Thunb.) DC. in Advanced-Stage Intrahepatic Cholangiocarcinoma

Objective: The objective of the study was to evaluate the clinical efficacy, safety, immunomodulatory activity, and effects on quality of life (QoL) of a capsule formulation of Atractylodes lancea (Thunb.) DC. Chemistry Manufacturing and Control capsule formulation of standardized extract of A. lancea (CMC-AL) in patients with advanced stage of intrahepatic cholangiocarcinoma (iCCA). Patients and Methods: A total of eligible 48 patients were randomly assigned to one of three groups. Group 1 received a once-daily dose of 1000 mg of CMC-AL in combination with palliative care for 3 months ( n = 16). Group 2 received escalating doses of 1000, 1500, and 2000 mg of CMC-AL, along with palliative care for 3 months ( n = 16). The control group received palliative care alone ( n = 16). All patients were followed up for 4 months. Serum cytokine levels were measured using a bead array, and safety and treatment responses were evaluated according to standard criteria. Results: CMC-AL at both dosage levels was well tolerated. The high-dose regimen (Group 2) significantly improved the patients’ clinical responses and QoL. Furthermore, there was a notable improvement in median total QoL scores compared to the baseline. Serum interleukin-6 levels on day 14 in both Groups 1 and 2 significantly decreased compared to baseline measurements. Conclusions: Results showed a promising role for CMC-AL in the treatment and management of iCCA. The high-dose CMC-AL showed advantages over the low-dose formulation and palliative care alone in enhancing clinical response, minimizing disease progression, improving QoL, and exhibiting immune-stimulatory effects. Therefore, the high-dose regimen could serve as an alternative treatment for iCCA patients who decline chemotherapy.