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New App Helps Predict Pediatric Transplant Need 

New Tool for Predicting the Need for a Transplant in Pediatric Acute Liver Failure 

Physicians from the Children’s Hospital Los Angeles have created a web-based app to predict whether children in acute liver failure will need a liver transplant. The Children’s Hospital Los Angeles Acute Liver Failure (CHALF) score effectively assesses whether a child’s medical condition warrants a liver transplant. This helps provide parents with valuable insight in their decision-making process when determining the need for a liver transplant for their child.

 

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Promising Results in Pediatric Autoimmune Hepatitis with a Combination Drug Therapy

Reports from a recent meta-analysis suggest a promising therapeutic regimen combining corticosteroids and immunomodulators. This treatment approach allows for a patient’s native liver to remain functional, even when they have acute liver failure. Further research is needed to evaluate situations when this approach will be suitable to expand treatment options beyond transplant.

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Alagille Medication Shows Promise in PFIC Type 2 Treatment 

Maralixibat, an FDA-approved medication for alleviating pruritus in Alagille syndrome, is now showing promising potential for addressing pruritus in progressive familial intrahepatic cholestasis (PFIC). Reports from the Phase 3 MARCH trial made during the North American Society for Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN) 2023 suggest that Maralixibat may emerge as a compelling option for treatment of PFIC Type 2.

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Rivfloza Approved for in Primary Hyperoxaluria Treatment  

The U.S. FDA has granted approval for Novo Nordisk’s therapeutic agent, Rivfloza, intended for individuals aged 9 and older, as well as adults afflicted with primary hyperoxaluria. This mechanism effectively mitigates the production of urinary oxalate originating in the liver. It is expected to be available for patients in early 2024

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For more information about the Pediatric and Rare Liver Diseases Council or to learn more about joining, please visit our webpage or email pedsrare@globalliver.org.