Presented by Professor Richard J. Thompson, MD, King's College London, United Kingdom
Patients with PFIC showed significant and sustained improvements in pruritus severity, serum bile acid (sBA) levels, total bilirubin and growth following up to two years of LIVMARLI treatment. Similar improvements in pruritus and sBA were seen in patients originally randomized to placebo who received LIVMARLI in the open-label study.
Abstract 594: Maralixibat Leads to Significant Reductions in Bilirubin for Patients with Progressive Familial Intrahepatic Cholestasis: Data from the MARCH Trial
Presented by Lorenzo D'Antiga, MD, Papa Giovanni XXIII Hospital, Bergamo, Italy
Patients treated with LIVMARLI experienced significant decreases in both total and direct bilirubin compared to placebo. 40% of the patients with abnormal bilirubin at baseline treated with LIVMARLI achieved normalization versus none in the placebo group. Further, these reductions in bilirubin were consistent with reductions in sBAs.
Abstract 600: Maralixibat Impact on Concomitant Medication Use for the Treatment of Cholestatic Pruritus in Alagille Syndrome: Real-World Experience
Presented by Jolan Terner-Rosenthal, PhD, Mirum Pharmaceuticals, Inc., Foster City, California, USA
Real-world evidence from 116 patients treated with LIVMARLI for at least one year showed that more than one-third of patients were able to discontinue ≥1 concomitant antipruritic medication, and one in five patients discontinued ≥2 medications. Reductions in concomitant medication usage were seen across all medication types and suggest that LIVMARLI may reduce the polypharmacy burden within the first year of treatment.
Other presentations featured during ESPGHAN include:
Abstract 592: Maralixibat Leads to Significant Improvements in Cholestatic Pruritus for Children with Progressive Familial Intrahepatic Cholestasis Without a Genetic Diagnosis: Data from the MARCH Trial
Presented by Professor Richard J. Thompson, MD, King's College, London, United Kingdom
Abstract 599: Maralixibat Leads to Improvements in Cholestatic Pruritus for Children with Progressive Familial Intrahepatic Cholestasis Due to MDR3 Deficiency: Data From the MARCH/MARCH-ON Trials
Presented by Professor Richard J. Thompson, MD, King's College, London, United Kingdom
Abstract 595: Improvements in Pruritus with Maralixibat are Associated with Improved Quality of Life for Patients with Progressive Familial Intrahepatic Cholestasis: Data From the MARCH Trial
Presented by Douglas B. Mogul, MD, PhD, Mirum Pharmaceuticals, Inc., Foster City, California, USA
Abstract 597: Maralixibat Can Improve Cholestatic Pruritus in Children with Progressive Familial Intrahepatic Cholestasis Who Previously Underwent a Surgical Biliary Diversion: Data From the MARCH/MARCH-ON Trials