Treatment outcomes of advanced hepatocellular carcinoma in real-life practice : chemotherapy versus Multikinase inhibitors
1
Issued Date
2021
Copyright Date
2021
Resource Type
Language
eng
File Type
application/pdf
No. of Pages/File Size
xvi, 142 leaves : ill.
Access Rights
open access
Rights
ผลงานนี้เป็นลิขสิทธิ์ของมหาวิทยาลัยมหิดล ขอสงวนไว้สำหรับเพื่อการศึกษาเท่านั้น ต้องอ้างอิงแหล่งที่มา ห้ามดัดแปลงเนื้อหา และห้ามนำไปใช้เพื่อการค้า
Rights Holder(s)
Mahidol University
Bibliographic Citation
Thesis (Ph.D. (Clinical Epidemiology))--Mahidol University, 2021
Suggested Citation
Songporn Oranratnachai Treatment outcomes of advanced hepatocellular carcinoma in real-life practice : chemotherapy versus Multikinase inhibitors. Thesis (Ph.D. (Clinical Epidemiology))--Mahidol University, 2021. Retrieved from: https://repository.li.mahidol.ac.th/handle/123456789/114978
Title
Treatment outcomes of advanced hepatocellular carcinoma in real-life practice : chemotherapy versus Multikinase inhibitors
Author(s)
Abstract
Multikinase inhibitors (MKIs) are the main treatment of advanced hepatocellular carcinoma (aHCC). Nevertheless, the accessibility to MKIs in developing countries is limited. Chemotherapy (CMT) which is cheaper than MKIs may be an option for the patients. Therefore, this study aimed to compare the clinical effectiveness of the CMT with MKIs in the first-line treatment of aHCC in the real-life practice. A retrospective cohort of aHCC using the real-world practice data from 3 medical schools and 1 regional hospital was conducted. The patients who received Doxorubicin, FOLFOX, or Sorafenib as first-line treatment between 2013–2019 were included in this study. Multiple imputation using the chain equation was applied to solve the missing data of covariables. The treatment effect model using Weight-adjusted-censoring inverse-probability-weighted regression adjustment (WAC-IPWRA) and IPWRA estimator was applied to time-to-event and binary outcomes, respectively. The results from the imputed data and listwise deletion were compared. A total of 673 patients were included in the estimation of relative treatment effects among Sorafenib (n=383), Doxorubicin (n=168), and FOLFOX (n=122). The treatment effect model, WAC-IPWRA estimators showed that Doxorubicin had the longest overall survival (OS) followed by Sorafenib and FOLFOX with the median OS of 23.3, 12.2, and 10.6 months, respectively. Conversely, the median progression-free survival (PFS) was the longest in Sorafenib followed by Doxorubicin and FOLFOX with the median PFS of 6.8, 5.6, and 4.8 months, respectively. Furthermore, the disease control rate was not much different in Sorafenib and Doxorubicin, but it was the lowest in FOLFOX. Finally, serious adverse events were present more in FOLFOX than Doxorubicin and Sorafenib. The results between the imputed data and listwise deletion were very closed in point estimate. Only the precision of estimation, in which the multiple imputation tended to have less standard errors when compared to the listwise deletion method. Even though the lower border of 95% confidence interval of difference of median OS crossed the non-inferior margin, the point estimate was not. Thus, we could not demonstrate the non-inferiority of CMT to Sorafenib. Therefore, further observational cohort studies with a larger sample size are needed to conclude that CMT can solve this clinical unmet need problem.
Degree Name
Doctor of Philosophy
Degree Level
Doctoral degree
Degree Department
Faculty of Medicine Ramathibodi Hospital
Degree Discipline
Clinical Epidemiology
Degree Grantor(s)
Mahidol University