Improved Quality of Life in Children With Progressive Familial Intrahepatic Cholestasis Following 24 Weeks of Treatment With Odevixibat, an Ileal Bile Acid Transporter Inhibitor: Results From the Phase 3 PEDFIC 1 Study
Author(s)
Thompson R1, Kjems L2, Hardikar W3, Lainka E4, Calvo PL5, Horn P2
1Institute of Liver Studies, King's College London, London, LON, UK, 2Albireo Pharma, Inc., Boston, MA, USA, 3Royal Children's Hospital, Melborne, VIC, Australia, 4Children’s Hospital, Department of Pediatric Gastroenterology, Hepatology, and Transplant Medicine, University Duisburg-Essen, Essen, Germany, 5Pediatric Gastroenterology Unit, Regina Margherita Children’s Hospital, Azienda Ospedaliera Città della Salute e della Scienza di Torino, Turin, Italy
OBJECTIVES Patients with progressive familial intrahepatic cholestasis (PFIC) have debilitating symptoms that can impact many aspects of their and their families’ quality of life (QoL). Key findings from PEDFIC 1 have been previously described; briefly, the ileal bile acid transporter inhibitor odevixibat improved serum bile acids, pruritus, and several sleep parameters in children with PFIC1 and PFIC2. Here, we describe the effect of odevixibat on QoL, assessed as an exploratory outcome. METHODS Randomized children (n=62) received placebo or odevixibat (40 or 120 μg/kg/day). To assess QoL, caregivers of patients ≥2 years old completed the Pediatric QoL Inventory (PedsQL) questionnaire, which outputs a total score (summarizing patient functioning in physical, emotional, social, and school domains) and a family impact (FI) total score (comprising physical, emotional, social, and cognitive functioning as well as communication, worry, daily activities, and family relationship domains). Here, mean score changes from baseline to week 24 are presented for both odevixibat doses combined versus placebo; these are summarized descriptively. Higher scores indicate improvement in QoL. RESULTS Mean PedsQL total scores improved with odevixibat versus placebo (7.8 vs 0.5, respectively). Among PedsQL domains, improvements were observed with odevixibat, while with placebo, 3 of 4 domains showed worsening (physical: 7.8 vs –5.9; emotional: 14.1 vs 13.5; social: 3.6 vs –1.0, school functioning: 2.3 vs –5.3). Larger mean improvements were observed with odevixibat versus placebo in FI total (14.5 vs 5.6, respectively) and domain scores (physical: 18.9 vs 8.1; emotional: 13.4 vs 7.9; social: 13.5 vs 8.5; cognitive: 16.4 vs 3.2; communication: 8.3 vs –4.4; worry: 12.8 vs 7.9; daily activities: 21.1 vs 9.3; family relationships: 10.9 vs 2.1). CONCLUSIONS Odevixibat improved patient and family QoL, parallel to previously described improvements in clinical signs and symptoms. Overall, odevixibat represents a potential noninvasive option to treat patients with PFIC and improve QoL.
Conference/Value in Health Info
2021-05, ISPOR 2021, Montreal, Canada
Value in Health, Volume 24, Issue 5, S1 (May 2021)
Code
PMU62
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
Multiple Diseases, Pediatrics