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according to investigators.
These findings suggest that LSM may serve as a noninvasive tool for risk stratification and treatment planning in this population, reported lead author Jean P. Molleston, MD, of Indiana University School of Medicine, Indianapolis, and colleagues.
“Biliary atresia is frequently complicated by hepatic fibrosis with progression to cirrhosis and portal hypertension manifested by ascites, hepatopulmonary syndrome, and variceal bleeding,” the investigators wrote in Gastroenterology. “The ability to predict these outcomes can inform clinical decision-making.”
To this end, VCTE has been gaining increasing support in the pediatric setting.
“Advantages of VCTE over liver biopsy include convenience, cost, sampling bias, and risk,” the investigators wrote. “VCTE potentially allows (1) fibrosis estimation, (2) prediction of portal hypertension complications/survival, and (3) ability to noninvasively monitor liver stiffness as a fibrosis surrogate.”
The present multicenter study aimed to gauge the prognostic utility of VCTE among 254 patients, aged 21 years or younger, with biliary atresia. All patients had a valid baseline LSM, plus longitudinal clinical and laboratory data drawn from studies by the Childhood Liver Disease Research Network (ChiLDReN). Liver stiffness was assessed noninvasively with FibroScan devices, adhering to protocols that required at least 10 valid measurements and a variability of less than 30%.
The primary outcomes were survival with native liver (SNL), defined as the time to liver transplantation or death, and a composite measure of liver-related events, including the first occurrence of transplantation, death, ascites, variceal bleeding, or hepatopulmonary syndrome. Secondary outcomes focused on the trajectory of platelet decline, a marker of disease progression. The study also explored the relationship between baseline LSM and conventional biomarkers, including platelet count, albumin, and bilirubin.
LSM was a strong predictor of long-term outcomes. Specifically, Kaplan-Meier analysis showed significant differences in 5-year SNL across LSM strata (P < .001). Children with LSM values less than 10 kPa had excellent 5-year SNL rates (LSM 10 to < 15 kPa, 88.9%; 95% CI, 75.1-95.3%), while those with LSM of at least 15 kPa exhibited substantially lower 5-year SNL (58.9%; 95% CI, 46.0-69.7%).
Similarly, event-free survival (EFS) rates declined as LSM values increased (P < .001). Participants with LSM less than 10 kPa had a 5-year EFS rate of 92.2% versus with 61.2% for those with LSM of at least 15 kPa.
LSM also predicted platelet decline. For every twofold increase in baseline LSM, platelet counts declined by an additional 4,000/mm3 per year (P < .001). This association was illustrated through predicted trajectories for participants with LSM values of 4, 7, 12, 18, and 42 kPa, corresponding to different percentiles of disease severity.
Cox proportional hazards analysis indicated that a two-fold increase in LSM was associated with a hazard ratio of 3.3 (P < .001) for liver transplant or death. While LSM had good discrimination on its own (C statistic = 0.83), it did not significantly improve predictive accuracy when added to models based on platelet count, albumin, and bilirubin.
“This noninvasive measurement could potentially be used to predict natural history, stratify patients for clinical trials, plan interventions, and provide anticipatory guidance,” Molleston and colleagues concluded. This study was supported by grants from the National Institute of Diabetes, Digestive and Kidney Diseases; National Institutes of Health; Childhood Liver Disease Research Network; and others. The investigators disclosed no conflicts of interest.
Grading liver stiffness using elastography is a widely utilized tool in adult populations, and its application is expanding in pediatric hepatology clinics. Clinicians incorporate liver stiffness measurements (LSM) alongside clinical findings and biochemical markers to noninvasively assess the degree of hepatic fibrosis and cirrhosis. Molleston and colleagues leveraged the robust data from the National Institute of Diabetes and Digestive and Kidney Diseases–supported network ChiLDReN and found that LSM in children with biliary atresia (BA) correlate with the progression to complications associated with portal hypertension and liver transplantation. While these findings are not unexpected, this compelling investigation accomplishes the important function of validating the utility of elastography in this cohort.
Prognosticating the timeline of complications stemming from biliary atresia is a central tenet of pediatric hepatology. Helping families understand what the future may hold for their child is critical in fostering long-term relationships between clinicians and caregivers. Furthermore, establishing clear expectations regarding follow-up care and monitoring is beneficial for both providers and patients. Of particular importance is minimizing the need for invasive procedures, such as liver biopsy, which, while relatively safe, remains burdensome and is rarely used to assess fibrosis in BA.
Pediatric hepatologists already consider multiple factors — including age at hepatoportoenterostomy, subsequent clearance of cholestasis, exam findings such as splenomegaly, and platelet count — to predict the clinical course of infants with BA. The addition of a data-driven approach to interpreting liver stiffness measurements represents a valuable new tool in this expanding repertoire, offering an encouraging prospect for both providers and families navigating the complexities of pediatric liver disease.
Aaron Bennett, MD, is a fellow in the Division of Gastroenterology, Hepatology and Nutrition at Children’s Hospital of Philadelphia in Pennsylvania. Elizabeth B. Rand, MD, is the medical director of the Liver Transplant Program, director of the Gastroenterology Fellowship Program, and director of the Advanced Transplant Hepatology Program at Children’s Hospital of Philadelphia.
Grading liver stiffness using elastography is a widely utilized tool in adult populations, and its application is expanding in pediatric hepatology clinics. Clinicians incorporate liver stiffness measurements (LSM) alongside clinical findings and biochemical markers to noninvasively assess the degree of hepatic fibrosis and cirrhosis. Molleston and colleagues leveraged the robust data from the National Institute of Diabetes and Digestive and Kidney Diseases–supported network ChiLDReN and found that LSM in children with biliary atresia (BA) correlate with the progression to complications associated with portal hypertension and liver transplantation. While these findings are not unexpected, this compelling investigation accomplishes the important function of validating the utility of elastography in this cohort.
Prognosticating the timeline of complications stemming from biliary atresia is a central tenet of pediatric hepatology. Helping families understand what the future may hold for their child is critical in fostering long-term relationships between clinicians and caregivers. Furthermore, establishing clear expectations regarding follow-up care and monitoring is beneficial for both providers and patients. Of particular importance is minimizing the need for invasive procedures, such as liver biopsy, which, while relatively safe, remains burdensome and is rarely used to assess fibrosis in BA.
Pediatric hepatologists already consider multiple factors — including age at hepatoportoenterostomy, subsequent clearance of cholestasis, exam findings such as splenomegaly, and platelet count — to predict the clinical course of infants with BA. The addition of a data-driven approach to interpreting liver stiffness measurements represents a valuable new tool in this expanding repertoire, offering an encouraging prospect for both providers and families navigating the complexities of pediatric liver disease.
Aaron Bennett, MD, is a fellow in the Division of Gastroenterology, Hepatology and Nutrition at Children’s Hospital of Philadelphia in Pennsylvania. Elizabeth B. Rand, MD, is the medical director of the Liver Transplant Program, director of the Gastroenterology Fellowship Program, and director of the Advanced Transplant Hepatology Program at Children’s Hospital of Philadelphia.
Grading liver stiffness using elastography is a widely utilized tool in adult populations, and its application is expanding in pediatric hepatology clinics. Clinicians incorporate liver stiffness measurements (LSM) alongside clinical findings and biochemical markers to noninvasively assess the degree of hepatic fibrosis and cirrhosis. Molleston and colleagues leveraged the robust data from the National Institute of Diabetes and Digestive and Kidney Diseases–supported network ChiLDReN and found that LSM in children with biliary atresia (BA) correlate with the progression to complications associated with portal hypertension and liver transplantation. While these findings are not unexpected, this compelling investigation accomplishes the important function of validating the utility of elastography in this cohort.
Prognosticating the timeline of complications stemming from biliary atresia is a central tenet of pediatric hepatology. Helping families understand what the future may hold for their child is critical in fostering long-term relationships between clinicians and caregivers. Furthermore, establishing clear expectations regarding follow-up care and monitoring is beneficial for both providers and patients. Of particular importance is minimizing the need for invasive procedures, such as liver biopsy, which, while relatively safe, remains burdensome and is rarely used to assess fibrosis in BA.
Pediatric hepatologists already consider multiple factors — including age at hepatoportoenterostomy, subsequent clearance of cholestasis, exam findings such as splenomegaly, and platelet count — to predict the clinical course of infants with BA. The addition of a data-driven approach to interpreting liver stiffness measurements represents a valuable new tool in this expanding repertoire, offering an encouraging prospect for both providers and families navigating the complexities of pediatric liver disease.
Aaron Bennett, MD, is a fellow in the Division of Gastroenterology, Hepatology and Nutrition at Children’s Hospital of Philadelphia in Pennsylvania. Elizabeth B. Rand, MD, is the medical director of the Liver Transplant Program, director of the Gastroenterology Fellowship Program, and director of the Advanced Transplant Hepatology Program at Children’s Hospital of Philadelphia.
according to investigators.
These findings suggest that LSM may serve as a noninvasive tool for risk stratification and treatment planning in this population, reported lead author Jean P. Molleston, MD, of Indiana University School of Medicine, Indianapolis, and colleagues.
“Biliary atresia is frequently complicated by hepatic fibrosis with progression to cirrhosis and portal hypertension manifested by ascites, hepatopulmonary syndrome, and variceal bleeding,” the investigators wrote in Gastroenterology. “The ability to predict these outcomes can inform clinical decision-making.”
To this end, VCTE has been gaining increasing support in the pediatric setting.
“Advantages of VCTE over liver biopsy include convenience, cost, sampling bias, and risk,” the investigators wrote. “VCTE potentially allows (1) fibrosis estimation, (2) prediction of portal hypertension complications/survival, and (3) ability to noninvasively monitor liver stiffness as a fibrosis surrogate.”
The present multicenter study aimed to gauge the prognostic utility of VCTE among 254 patients, aged 21 years or younger, with biliary atresia. All patients had a valid baseline LSM, plus longitudinal clinical and laboratory data drawn from studies by the Childhood Liver Disease Research Network (ChiLDReN). Liver stiffness was assessed noninvasively with FibroScan devices, adhering to protocols that required at least 10 valid measurements and a variability of less than 30%.
The primary outcomes were survival with native liver (SNL), defined as the time to liver transplantation or death, and a composite measure of liver-related events, including the first occurrence of transplantation, death, ascites, variceal bleeding, or hepatopulmonary syndrome. Secondary outcomes focused on the trajectory of platelet decline, a marker of disease progression. The study also explored the relationship between baseline LSM and conventional biomarkers, including platelet count, albumin, and bilirubin.
LSM was a strong predictor of long-term outcomes. Specifically, Kaplan-Meier analysis showed significant differences in 5-year SNL across LSM strata (P < .001). Children with LSM values less than 10 kPa had excellent 5-year SNL rates (LSM 10 to < 15 kPa, 88.9%; 95% CI, 75.1-95.3%), while those with LSM of at least 15 kPa exhibited substantially lower 5-year SNL (58.9%; 95% CI, 46.0-69.7%).
Similarly, event-free survival (EFS) rates declined as LSM values increased (P < .001). Participants with LSM less than 10 kPa had a 5-year EFS rate of 92.2% versus with 61.2% for those with LSM of at least 15 kPa.
LSM also predicted platelet decline. For every twofold increase in baseline LSM, platelet counts declined by an additional 4,000/mm3 per year (P < .001). This association was illustrated through predicted trajectories for participants with LSM values of 4, 7, 12, 18, and 42 kPa, corresponding to different percentiles of disease severity.
Cox proportional hazards analysis indicated that a two-fold increase in LSM was associated with a hazard ratio of 3.3 (P < .001) for liver transplant or death. While LSM had good discrimination on its own (C statistic = 0.83), it did not significantly improve predictive accuracy when added to models based on platelet count, albumin, and bilirubin.
“This noninvasive measurement could potentially be used to predict natural history, stratify patients for clinical trials, plan interventions, and provide anticipatory guidance,” Molleston and colleagues concluded. This study was supported by grants from the National Institute of Diabetes, Digestive and Kidney Diseases; National Institutes of Health; Childhood Liver Disease Research Network; and others. The investigators disclosed no conflicts of interest.
according to investigators.
These findings suggest that LSM may serve as a noninvasive tool for risk stratification and treatment planning in this population, reported lead author Jean P. Molleston, MD, of Indiana University School of Medicine, Indianapolis, and colleagues.
“Biliary atresia is frequently complicated by hepatic fibrosis with progression to cirrhosis and portal hypertension manifested by ascites, hepatopulmonary syndrome, and variceal bleeding,” the investigators wrote in Gastroenterology. “The ability to predict these outcomes can inform clinical decision-making.”
To this end, VCTE has been gaining increasing support in the pediatric setting.
“Advantages of VCTE over liver biopsy include convenience, cost, sampling bias, and risk,” the investigators wrote. “VCTE potentially allows (1) fibrosis estimation, (2) prediction of portal hypertension complications/survival, and (3) ability to noninvasively monitor liver stiffness as a fibrosis surrogate.”
The present multicenter study aimed to gauge the prognostic utility of VCTE among 254 patients, aged 21 years or younger, with biliary atresia. All patients had a valid baseline LSM, plus longitudinal clinical and laboratory data drawn from studies by the Childhood Liver Disease Research Network (ChiLDReN). Liver stiffness was assessed noninvasively with FibroScan devices, adhering to protocols that required at least 10 valid measurements and a variability of less than 30%.
The primary outcomes were survival with native liver (SNL), defined as the time to liver transplantation or death, and a composite measure of liver-related events, including the first occurrence of transplantation, death, ascites, variceal bleeding, or hepatopulmonary syndrome. Secondary outcomes focused on the trajectory of platelet decline, a marker of disease progression. The study also explored the relationship between baseline LSM and conventional biomarkers, including platelet count, albumin, and bilirubin.
LSM was a strong predictor of long-term outcomes. Specifically, Kaplan-Meier analysis showed significant differences in 5-year SNL across LSM strata (P < .001). Children with LSM values less than 10 kPa had excellent 5-year SNL rates (LSM 10 to < 15 kPa, 88.9%; 95% CI, 75.1-95.3%), while those with LSM of at least 15 kPa exhibited substantially lower 5-year SNL (58.9%; 95% CI, 46.0-69.7%).
Similarly, event-free survival (EFS) rates declined as LSM values increased (P < .001). Participants with LSM less than 10 kPa had a 5-year EFS rate of 92.2% versus with 61.2% for those with LSM of at least 15 kPa.
LSM also predicted platelet decline. For every twofold increase in baseline LSM, platelet counts declined by an additional 4,000/mm3 per year (P < .001). This association was illustrated through predicted trajectories for participants with LSM values of 4, 7, 12, 18, and 42 kPa, corresponding to different percentiles of disease severity.
Cox proportional hazards analysis indicated that a two-fold increase in LSM was associated with a hazard ratio of 3.3 (P < .001) for liver transplant or death. While LSM had good discrimination on its own (C statistic = 0.83), it did not significantly improve predictive accuracy when added to models based on platelet count, albumin, and bilirubin.
“This noninvasive measurement could potentially be used to predict natural history, stratify patients for clinical trials, plan interventions, and provide anticipatory guidance,” Molleston and colleagues concluded. This study was supported by grants from the National Institute of Diabetes, Digestive and Kidney Diseases; National Institutes of Health; Childhood Liver Disease Research Network; and others. The investigators disclosed no conflicts of interest.
FROM GASTROENTEROLOGY