Treatment outcomes of advanced hepatocellular carcinoma in real-life practice: Chemotherapy versus multikinase inhibitors.

BACKGROUND: Multikinase inhibitors (MKIs) represent the main treatment options for advanced hepatocellular carcinoma (aHCC). However, accessibility in developing countries is limited. A chemotherapy, Fluorouracil and Oxaliplatin (FOLFOX), offers a less expensive treatment. Therefore, this study sought to compare the clinical effectiveness of FOLFOX with Sorafenib as a first-line treatment for aHCC in real-life practice. METHODS: A retrospective aHCC cohort from four Thai hospitals was investigated for patients who received FOLFOX or Sorafenib between 2013-2019. Multiple imputation by chained equations addressed missing covariate data in a treatment effect model using Weight-adjusted-censoring inverse-probability-weighted regression adjustment; overall survival (OS) and progression-free survival (PFS) were estimated. RESULTS: A total of 504 patients were included, (Sorafenib [n = 382] and FOLFOX [n = 122]). The treatment effect model estimated a median OS for Sorafenib and FOLFOX of 11.38 and 8.22 months, representing a significantly shorter OS (95% confidence interval) of -3.16 (-6.21, -0.11) months for FOLFOX, p = 0.042. A significant shorter median PFS of FOLFOX to Sorafenib of -2.13 (-3.03, -1.24) months, p < 0.001, was reported. CONCLUSION: Despite significantly shorter median OS and PFS than Sorafenib, FOLFOX still extended OS by 8.22 months. This evidence may offer clinical utility to physicians considering treatment options for aHCC in low resource settings.

Efficacy and tolerability of BP-C1 in metastatic breast cancer: a Phase II, randomized, double-blind, and placebo-controlled Thai multi-center study.

AIMS: The aim of this study was to compare the efficacy and tolerability of BP-C1 vs equal-looking placebo in metastatic breast cancer. MATERIALS AND METHODS: A randomized, double-blind, placebo-controlled multi-center study with a semicross-over design was performed. Sixteen patients received daily intramuscular injection of 0.035 mg/kg bodyweight of BP-C1 and 15 patients received equal-looking placebo for 32 days. After 32 days, the placebo patients crossed to BP-C1 with the last observation in the placebo period as baseline. The status of receptors including estrogen receptor (ER), progesterone receptor (PtR), and human EGF receptor 2 (HER2) was analyzed prior to inclusion in the study. Thoracoabdominal CT scan was blindly analyzed by the same independent radiologist in accordance with the RECIST criteria 1.1. Toxicity was assessed according to the NCI Bethesda Version 2.0 (CTC-NCI), and the quality of life (QOL) was assessed according to European Organization for the Research and Treatment of Cancer QOL-C30 and QOL-BR23. RESULTS: The sum of target lesion diameters (sum lesions) after 32 days of treatment increased by 8.9% (P=0.08) in the BP-C1 arm compared to 37.6% (P<0.001) in placebo patients. Twelve of the 15 placebo patients subsequently had BP-C1 treatment. The increase in sum lesions was 3.5% in these patients. The sum of CTC-NCI was increased 18.7% in the BP-C1 arm (P=0.38) compared to 50.9% (P=0.04) in placebo patients. Four mild/moderate adverse events (AEs) present in BP-C1. Two mild/moderate AEs and one severe AE present in placebo. The QOL benchmarks "breast cancer problems last week", "sexual interest and activity last 4 weeks", and "breast cancer-related pain and discomfort last week" were stable in the BP-C1 arm but deteriorated in placebo patients. The sum lesions increased significantly in ER+ (P=0.02) and PtR+ (P=0.03) but not in HER2+. The increase in sum lesions significantly decreased (P=0.02) with an increasing number of negative receptors. CONCLUSION: A total of 32 days of BP-C1 treatment inhibited cancer growth and was well tolerated with few and mainly mild AEs. The efficacy of BP-C1 was superior in receptor-negative patients. CLINICALTRIALSGOV IDENTIFIER: NCT03603197.

CA 19-9 in combination with abdominal CT scan for the diagnosis of mass-forming intrahepatic cholangiocarcinoma.

AIM: Cholangiocarcinoma (CCA) is a common form of cancer in Thailand. The diagnosis of CCA by pathology is widely accepted. Unfortunately, tissue diagnosis of mass-forming intrahepatic cholangiocarcinoma is difficult to perform due to the risk of the procedure and accessibility. The aim of this study is to assess the diagnostic utility of CA19-9 in combination with CT scan for the diagnosis of mass-forming cholangiocarcinoma. MATERIALS & METHODS: The medical records of patients with a diagnosis of CCA and hepatocellular carcinoma (HCC) during January 2005-December 2009 were reviewed. Only cases with pathology and CT scan reports from Maharaj Nakorn Chiang Mai Hospital were included. Demographic data, clinical manifestations and laboratory results, including CA19-9 and CT scans of the liver, were carefully examined in order to establish their diagnostic utility in mass-forming CCA without a pathological diagnosis. RESULTS: A total of 79 CCA patients and 66 HCC patients were included in CA19-9 cut-off level analyses. A total of 31 CCA patients and 44 HCC patients were included in the CT scan evaluation and scored according to the Chiang Mai CT score for CCA. The specificity of a CA19-9 value of 500 U/ml for the diagnosis of CCA was 95.5% with a sensitivity of 50.6%, positive predictive value of 91.8% and likelihood ratio of positive (LR+) of 11.24. A CT score greater than 2 demonstrates a positive predictive value of more than 90% in diagnosis of CCA. The CA19-9 level of 140 U/ml in combination with a CT score of 2 demonstrated LR+ as high as 57.09. A CA19-9 level of 280 U/ml in combination with a CT score of 1 demonstrated LR+ of 58.4. CONCLUSION: A CA19-9 level combined with the Chiang Mai CT score for CCA are good diagnostic tools for the diagnosis of mass-forming cholangiocarcinoma with high specificity, high positive predictive value and high likelihood ratio of positive.