Background and aims: A previously published trial demonstrated that odevixibat is effective in the treatment of cholestatic pruritus of children with progressive familial intrahepatic cholestasis (PFIC). Real-world experience is necessary to confirm the results of registration trials having selective eligibility criteria. We present our “real life experience” on odevixibat effectiveness and safety in patients with different PFIC subtypes. Methods: multicenter prospective study of patients with PFIC treated with odevixibat (40 or escalated to 120 µg/kg/day). Pruritus was assessed by “Physician Global Impression of Symptom” at baseline and monthly up to 6 months. Serum bile acids (sBA) responders were patients who achieved a reduction in sBA levels ≥70% from baseline (or a value <70 µmol/L) after 6 months; pruritus responders were patients who reported improvement in pruritus score. Results: 24 patients [median age 6.6 years (3.7-12.1), M/F=11/13] were enrolled; 16 (67%) had classic PFIC types (PFIC-1=2; PFIC-2=11; PFIC-3=3), 8 (33%) had rarer forms (PFIC-4=5, PFIC-5=1; PFIC-6=1; PFIC-9=1). All had high sBA levels and 22/24 (92%) had pruritus. 4 (17%) had associated comorbidities. After 6 months of treatment, sBA decreased from a median of 317.1 µmol/L (range 82.3- 369.0) to 45.6 µmol/L (range 7.2-120; p<0.001); mean change of pruritus score was -1.7. Overall, 75% of patients were sBA responders, 73% were pruritus responders; 30% required dose escalation. Reduced pruritus correlated significantly with reduced sBA (p<0.05). A cutoff value of sBA >333.5 µmol/L increased the risk of no response to odevixibat by 17 folds (p < 0.001). No serious adverse events were recorded.
Real-word experience with Odevixibat in children with Progressive Familial Intrahepatic Cholestasis
Mandato, Claudia;
2024-01-01
Abstract
Background and aims: A previously published trial demonstrated that odevixibat is effective in the treatment of cholestatic pruritus of children with progressive familial intrahepatic cholestasis (PFIC). Real-world experience is necessary to confirm the results of registration trials having selective eligibility criteria. We present our “real life experience” on odevixibat effectiveness and safety in patients with different PFIC subtypes. Methods: multicenter prospective study of patients with PFIC treated with odevixibat (40 or escalated to 120 µg/kg/day). Pruritus was assessed by “Physician Global Impression of Symptom” at baseline and monthly up to 6 months. Serum bile acids (sBA) responders were patients who achieved a reduction in sBA levels ≥70% from baseline (or a value <70 µmol/L) after 6 months; pruritus responders were patients who reported improvement in pruritus score. Results: 24 patients [median age 6.6 years (3.7-12.1), M/F=11/13] were enrolled; 16 (67%) had classic PFIC types (PFIC-1=2; PFIC-2=11; PFIC-3=3), 8 (33%) had rarer forms (PFIC-4=5, PFIC-5=1; PFIC-6=1; PFIC-9=1). All had high sBA levels and 22/24 (92%) had pruritus. 4 (17%) had associated comorbidities. After 6 months of treatment, sBA decreased from a median of 317.1 µmol/L (range 82.3- 369.0) to 45.6 µmol/L (range 7.2-120; p<0.001); mean change of pruritus score was -1.7. Overall, 75% of patients were sBA responders, 73% were pruritus responders; 30% required dose escalation. Reduced pruritus correlated significantly with reduced sBA (p<0.05). A cutoff value of sBA >333.5 µmol/L increased the risk of no response to odevixibat by 17 folds (p < 0.001). No serious adverse events were recorded.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
