Clinical trial NCT06813508: Prognostic Factors for HCC and Liver Transplantation in Patients With MASLD/MASH
Official title: Prognostic Factors for the Development of Hepatocellular Carcinoma (HCC) and Indications for Liver Transplantation in Patients With Metabolic Liver Diseases (MASLD/MASH): The BOMASH Study
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Metabolic Dysfunction-Associated Steatotic Liver Disease
Hepatocellular Carcinoma
Metabolic Dysfunction-Associated Steatohepatitis
Liver Transplant
Lead sponsor: IRCCS Azienda Ospedaliero-Universitaria di Bologna
Sponsor class: OTHER
First posted: 2025-02-07
Enrollment: 1000
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The study will be presented to every patient with a diagnosis of MASLD/MASH, either newly diagnosed or previously diagnosed at the promoting center during follow-ups or routine diagnostic-therapeutic pathways.

Brief summary: The BOMASH study is a single-center, prospective/retrospective observational study without pharmacological interventions. It will include all patients diagnosed with Metabolic-Associated Steatotic Liver Disease (MASLD/MASH), whether newly diagnosed or previously identified at the center during follow-up or as part of routine diagnostic and therapeutic care.

The aim of the study is to identify predictive factors related to the prognosis of patients with metabolic liver disease (MASLD/MASH). Specifically, the study seeks to uncover biomarkers that can identify individuals at risk of requiring a liver transplant or developing HCC.

Detailed description: The BOMASH study is a single-center, prospective/retrospective observational study without pharmacological interventions. It will include all patients diagnosed with Metabolic-Associated Steatotic Liver Disease and Metabolic-Associated Steatohepatitis (MASLD/MASH), whether newly diagnosed or previously identified at the center during follow-ups or as part of routine diagnostic and therapeutic care. MASLD is characterized by significant variability in terms of severity and progression rates. Although a large portion of the population is at risk, only a minority develop liver-related comorbidities. Epidemiological studies reveal that patients with MASH have a higher risk of developing liver-related complications compared to those with simple steatosis (MASLD). However, the factors driving progression to MASH and its advanced stages remain unclear, and disease staging can only be accurately determined through liver biopsy. Given the large number of individuals at risk for MASLD, liver biopsy is not a feasible screening tool for widespread use. Key challenges involve understanding the biological and environmental factors that drive variability among MASLD patients and using this knowledge to develop effective methods for risk stratification, enabling targeted treatment for individuals at the highest risk.

The identification of risk factors through the combination of non-invasive tests (serum biomarkers and non-invasive techniques) can enable risk stratification for hepatocellular carcinoma (HCC) development and identify individuals who may require liver transplantation.

A study on MASLD represents a valuable tool to enhance understanding of this nosological entity and to support basic, clinical, and epidemiological research. It also benefits individuals affected by these conditions and assists national and local authorities in planning and optimizing healthcare and social services. Systematic data collection on MASLD can be instrumental in identifying previously unrecognized risk factors that may predispose individuals to more aggressive and treatment-resistant forms of the disease.

Analyzing the collected data could highlight potential common markers among patients whose MASLD diagnosis progresses to HCC and subsequently necessitates liver transplantation. These findings would provide useful prognostic factors for patient management.

In conclusion, the opportunity to longitudinally track disease progression in a large cohort of patients with chronic metabolic liver disease could pave the way for strategies that make the management of this highly prevalent condition more sustainable for national and regional healthcare systems.

Therefore, the aim of the study is to identify predictive factors related to the prognosis of patients with metabolic liver disease (MASLD/MASH). Specifically, the study seeks to uncover biomarkers that can identify individuals at risk of requiring a liver transplant or developing HCC.

Eligibility criteria: Inclusion Criteria:

* All patients with a diagnosis of MASLD, established according to the most recent published guidelines (EASL, EASD, EASO)
* Age ≥18 years

Inclusion Criteria for Biological Sample Collection:

* Patients requiring liver biopsy for diagnostic purposes, as indicated by the most recent published guidelines (EASL, EASD, EASO)

Exclusion Criteria:

* No exclusion criteria.

Primary outcomes:
measure: Number of hepatocellular carcinoma (HCC) or liver transplantation (OLT) cases and time to onset.
timeFrame: From enrollment to november 2044
Secondary outcomes:
measure: Incidence of HCC and liver transplantation (OLT)
timeFrame: From enrollment to november 2044
measure: Response rate in patients
timeFrame: From enrollment to november 2044
measure: Number of cases of liver complications
timeFrame: From enrollment to november 2044
measure: Response to dietologic and pharmacological therapies related to glycated hemoglobin levels
timeFrame: From enrollment to november 2044
measure: Response to dieto-therapeutic and pharmacological therapies related to weight
timeFrame: From enrollment to november 2044
measure: Response to dieto-therapeutic and pharmacological therapies related to biochemical parameters
timeFrame: From enrollment to november 2044
measure: Number of HCC or liver transplantation (OLT) cases categorized by MASLD and MASH
timeFrame: From enrollment to november 2044
Other outcomes:
Locations:
city: Bologna
country: Italy
facility: IRCCS Azienda Ospedaliero-Universitaria di Bologna
Study officials:
affiliation: IRCCS Azienda Ospedaliero-Universitaria di Bologna
name: Fabio Piscaglia, Prof, MD
role: PRINCIPAL_INVESTIGATOR
References: