#United States #Target RWE #Liver Disease #Pediatric Health #Durham, NC

Innovative Research on Liver Disease Terminology in Children

In a pivotal study released by Target RWE, a leader in real-world evidence (RWE) solutions, remarkable findings have been established that validate the newly introduced terminology for liver diseases affecting pediatric patients. This research, published in Hepatology Communications, is widely regarded as one of the first of its kind to draw connections between historical and contemporary nomenclature concerning fatty liver disease among children and adolescents.

The study utilized data from the TARGET-NASH cohort, enabling researchers to demonstrate a strikingly high level of agreement between the old classification of non-alcoholic fatty liver disease (NAFLD) and the updated terminology of metabolic dysfunction-associated steatotic liver disease (MASLD).

Key Findings from the Study

The study uncovered several significant insights:

• - An overwhelming 99% of pediatric participants in the TARGET-NASH cohort met the recently established MASLD diagnostic criteria.
• - Every participant diagnosed with NAFLD cirrhosis also satisfied the new diagnostic requirements, indicating a 100% concordance rate.
• - A noteworthy 89% concordance was observed between Target RWE's clinical criteria for metabolic dysfunction-associated steatohepatitis (MASH) and diagnoses confirmed through histological examination conducted by expert pathologists.

Dr. Miriam B. Vos, a prominent figure associated with the research and Director of the Pediatric Program at Emory University's Georgia Center for Clinical and Translational Science Alliance, remarked on the study's significance. She emphasized that this is the first detailed investigation into how the old and new nomenclature correlates within the realm of pediatric metabolic dysfunction-associated steatohepatitis, further asserting the relevance of past research conducted under the legacy NAFLD classification.

Dr. Vos noted, “The findings provide crucial validation for the applicability of the new MASLD/MASH terminology in children and adolescents, ensuring that previous research is not rendered obsolete.” This analysis supports the utilization of accessible non-invasive clinical data in diagnosing MASH among younger patients.

Study Methodology and Scope

The research was thorough, drawing data from 828 pediatric patients enrolled in the Target RWE MASLD cohort across the United States. Besides validating the new terminology, the study evaluated the effectiveness of clinical criteria used to diagnose MASH compared to diagnoses achieved through histological evaluations conducted by a centralized pathology team. This approach offers a non-invasive alternative to conventional methods of diagnosing MASLD in pediatric populations.

Michael W. Fried, MD, and Chief Medical Officer at Target RWE, stressed the implications of these findings for the clinical community. He remarked that the strong agreement between clinical diagnoses of MASH and histological confirmations provides a unique opportunity for further exploration and research into MASH within pediatric populations. This backs the recent consensus recommendations that established new nomenclature reflective of the underlying pathophysiology of liver diseases.

Prior to this research, Target RWE had already published findings in the American Journal of Gastroenterology, highlighting similar high concordance rates of the new MASLD terminology among adult patients registered in the TARGET-NASH registry.

The introduction of this enhanced nomenclature underscores the importance of recognizing underlying metabolic conditions and their implications on liver health in the pediatric demographic.

To explore more about Target RWE and their broad spectrum of research initiatives, you can visit their official site at targetrwe.com/research/publications/.

This innovative study not only clarifies the evolving landscape of liver disease terminology but also emphasizes the importance of aligning clinical practices with the latest scientific developments for accurate pediatric diagnosis.

About Target RWE

Target RWE is committed to generating real-world evidence that supports strategic decisions throughout the lifecycle of drug development. Their expertise combines clinical insights, analytical proficiency, and technical capabilities to provide a comprehensive understanding derived from patient journeys, marked by exceptional scale and accuracy.