Real-World Treatment Patterns and Survival Outcomes in Patients With Intermediate-Stage Hepatocellular Carcinoma.

Real-World Treatment Patterns and Survival Outcomes in Patients With Intermediate-Stage Hepatocellular Carcinoma: An Observational Cohort Study.

The goal of this observational study is to learn about the different treatment options for intermediate-stage hepatocellular carcinoma. The main question it aims to answer is:

- What are the survival outcomes among participants aged 18 or older who receive treatment for intermediate-stage hepatocellular carcinoma?

Aim of work:

- To evaluate real-world treatment patterns and survival outcomes among patients with intermediate-stage hepatocellular carcinoma (BCLC-B) treated at a tertiary care center.

Study Design:

- Observational cohort study.

Study Setting:

- Single-center study at Sohag Oncology Center.

Study Period:

- 01 January 2020 - 31 December 2025.

Sample Size:

• Expected cohort: 120-300 patients.
• Adequate for survival analysis and multivariable modeling in real-world studies.

Data Collection

1. Baseline Characteristics:

   • Age, sex
   • Etiology of liver disease (HBV, HCV, MASH, …)
   • AFP level
   • Child-Pugh score
   • ALBI grade
   • Tumor burden (number, size of largest lesion, bilobar involvement)

2. Treatment Variables

   • Type of initial therapy
   • Number of TACE sessions
   • Time to TACE refractoriness
   • Use of systemic therapy (Tyrosine Kinase inhibitors, Immunotherapy, combinations)
   • Treatment sequencing

Statistical analysis:

• Statistical analysis will be performed by SPSS 24.0 software (SPSS, Inc., Chicago, IL).

• Descriptive statistics for baseline characteristics.

• Kaplan-Meier survival curves for OS and PFS.

• Log-rank test for group comparisons.

• Multivariable Cox proportional hazards regression to identify predictors of survival.

• Subgroup analyses:

  • Up-to-7 in vs out.
  • Child-Pugh A vs B.
  • TACE-only vs sequential/systemic.

Study Objectives:

1. Primary Objective

   - To estimate overall survival (OS) among patients with intermediate-stage HCC receiving real-world treatment.

2. Secondary Objectives

   • To describe real-world treatment patterns (First-line, subsequent lines, combination therapies) and time to treatment changes.

   • To estimate progression-free survival (PFS) and time to treatment failure (TTF) by treatment group.

   • To evaluate safety signals (hospitalizations for treatment-related complications) in each treatment group.

   • To identify predictors of survival, including:

     • Tumor burden.
     • Child-Pugh class.
     • Laboratory markers (AFP, Bilirubin, Albumin).

Inclusion Criteria:

• Aged ≥18 years.
• Both sexes.
• Diagnosis of hepatocellular carcinoma based on accepted radiologic or histologic criteria.
• Classified as intermediate stage (BCLC-B) at time of decision for first-line treatment.
• Child-Pugh A or B.
• ECOG performance status 0-1.

Exclusion Criteria:

• BCLC A, C, or D at diagnosis.
• Prior curative therapy (resection, transplantation, curative ablation).
• Missing essential data (imaging, liver function, treatment records).
• Mixed HCC-cholangiocarcinoma.