The investigators design a large sample size study in the real-world to explore whether targeted therapy and immunotherapy can improve the survival, quality of life and drug safety of patients with advanced hepatobiliary tumors, and analyze the correlation between genetic variation and the effectiveness of therapy.
This trial is a multicenter, non-random, open and observational real-world study. It is estimated that 2000 patients with advanced hepatobiliary tumors will be enrolled in about 20 research centers. And it is planned to complete the enrollment within 2 years and it is expected that all enrolled subjects will reach the observation end point in 5 years. After primary screening of all the patients, the investigators will further collect next-generation sequence(NGS) data and immunohistochemical data from the subjects, and enroll patients with any type of the following three treatment program: 1. Monotherapy or combination therapy with the targeted drug related to genetic variation of the subject; 2. Treatment with pan-target anti-angiogenic drugs, such as sorafenib, regorafenib, lenvatinib, apatinib, etc; 3. Immunotherapy or immunotherapy combined with targeted therapy. The investigators will collect subjects' data from assessment centers each month and the efficacy, quality of life, and safety of treatment will be evaluated. This trial plans to conduct an interim analysis and final analysis after collecting 500 cases, 1000 cases, and 2000 subjects, 3000 subjects,and explore the clinical application value of targeted therapy and immunotherapy in advanced hepatobiliary tumors in real world.
Study Type
OBSERVATIONAL
Enrollment
3,000
Therapeutic regimens are based on the clinicopathological features, genomic alterations, advices from multidisciplinary tumor board and willings from patients.
Chinese Academy of Medical Sciences & Peking Union Medical College Hospital
Beijing, Beijing Municipality, China
RECRUITINGOverall Survival (OS)
Time from the date of initial treatment to death for any cause.
Time frame: two years
Progression-free Survival (PFS)
A duration from the date of initial treatment with apatinib to disease progression (as defined by RECIST) or death.
Time frame: six months
Objective Response Rate (ORR)
Percentage of subjects with complete response and partial response.
Time frame: one year
Disease Control Rate (DCR)
Percentage of subjects with complete remission, partial remission, or stable disease during treatment or within 30 days of termination of test drug.
Time frame: six months
Incidence of Treatment-Emergent Adverse Event
Percentage of subjects who develop any adverse events (CTCAE 4.0).
Time frame: one year
Quality of Life (QoL) after treatment
The life quality of every subject will be assessed every 3 months according to the 45-item FACT-Hep questionnaire, which assesses generic HRQL concerns and disease-specific issues.
Time frame: one year
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