Children with Alagille syndrome (ALGS) who responded to maralixibat (Livmarli™) treatment showed clinically meaningful improvements in health-related quality of life (HRQoL) when compared to nonresponders. The new results were presented by Mirum Pharmaceuticals, Inc. during the 2021 North American Society for Pediatric Gastroenterology Hepatology and Nutrition Annual Meeting.

“These data demonstrate that the significant improvements in pruritus seen with maralixibat at week 48 of the ICONIC study are clinically meaningful and are associated with improvements in patients’ quality of life,” the authors wrote in the poster’s conclusions.

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Most (74%) participants showed a positive response to the treatment at week 48 (ie, ≥1 point reduction in caregiver Itch-Reported Outcome [ItchRO] instrument score from baseline to week 48). ItchRO treatment response was associated with clinically meaningful improvements in all the HRQoL measures analyzed (ie, Pediatric Quality of Life (PedsQL) Generic Core Total Scale Score, Family Impact Total Scale Score, and Multidimensional Fatigue Total Scale Score).

In comparison with the results obtained for nonresponders at week 48, responders showed:

  • Almost twice the minimal clinically important difference in Pediatric Quality of Life (PedsQL) Generic Core Total Scale Score
  • More than 3 times the minimal clinically important difference in Family Impact Total Scale Score
  • More than 2 times the minimal clinically important difference in Multidimensional Fatigue Total Scale Score
  • Larger changes in 6 sleep-related items (trouble sleeping, feeling tired, sleeping a lot, difficulty sleeping through the night, feeling tired upon walking, and taking a lot of naps).

“Patients often suffer greatly due to lack of sleep caused by severe and unrelenting pruritus, and these data help elucidate the correlation between pruritus response and improvements in quality of life outcomes,” said Pamela Vig, PhD, head of research and development at Mirum.

Clinicians’ Insights on ICONIC Study Findings in Patients With ALGS

The analysis included 27 participants of the ICONIC study who had available data at week 48. The patients received 380 µg/kg per day of an oral solution of maralixibat.

Reference

LIVMARLI (maralixibat) presentations show improvements in quality of life measures in Alagille syndrome and PFIC2 at NASPGHAN Annual Meeting 2021. News release. Mirum Pharmaceuticals, Inc.; December 13, 2021

Kamath BM, Goldstein A, Howard R, et al. Response to treatment with maralixibat in Alagille syndrome is associated with improved health-related quality of life. Poster presented at: North American Society for Pediatric Gastroenterology Hepatology and Nutrition Annual Meeting: December 12-18, 2021; Virtual.