Laura M J Hochstenbach, Domino Determann, Rianne R R Fijten, Esther J Bloemen-van Gurp, Renée Verwey
Taking shared decision making for prostate cancer to the next level: Requirements for a Dutch treatment decision aid with personalized risks on side effects
Internet interventions
patients alagille syndrome cholestasisassociated clinical features include high serum bile acids severe pruritus necessitate liver transplantation aimed evaluate efficacy safety ileal bile acid transporter inhibitor odevixibat versus placebo patients alagille syndrome assert study phase 3 doubleblind randomised placebocontrolled trial enrolled patients 21 medical centres hospitals ten countries belgium france germany italy malaysia netherlands poland trkiye uk usa eligible patients genetically confirmed diagnosis alagille syndrome history significant pruritus elevated serum bile acids patients randomly assigned 21 receive oral odevixibat 120 gkg per day placebo 24 weeks block size six stratified age 10 years 10 years 18 years via webbased system patients clinicians study staff people analysing data masked treatment allocation primary efficacy endpoint change caregiverreported scratching score prucision instrument range 04 baseline weeks 2124 prespecified key secondary efficacy endpoint change serum bile acid concentration baseline average weeks 20 24 outcomes analysed patients received least one dose study drug full analysis set efficacy outcomes safety analysis set safety outcomes trial registered clinicaltrialsgov nct04674761 eudract 202000401128 completed feb 26 2021 sept 9 2022 52 patients randomly assigned receive odevixibat n35 placebo n17 included analysis sets median age 55 years iqr 32 89 27 52 52 patients male 25 48 female mean scratching score elevated baseline groups 28 sd 05 odevixibat vs 30 06 placebo mean scratching scores weeks 2124 11 09 odevixibat 22 10 placebo representing leastsquares ls mean change 17 95 ci 20 13 odevixibat 08 13 03 placebo significantly greater odevixibat placebo difference ls mean change baseline 09 95 ci 14 03 p00024 odevixibat also resulted significantly greater reductions mean serum bile acids baseline versus placebo 237 moll sd 115 odevixibat vs 246 moll 121 placebo average weeks 20 24 149 moll 102 vs 271 moll 167 ls mean change 90 moll 95 ci 133 48 odevixibat vs 22 moll 35 80 placebo difference ls mean change 113 moll 95 ci 179 47 p00012 common treatmentemergent adverse events diarrhoea ten 29 35 patients odevixibat group vs one 6 17 placebo group pyrexia eight 23 vs four 24 seven patients serious treatmentemergent adverse events treatment period five 14 odevixibat group two 12 placebo group patients discontinued treatment deaths odevixibat could efficacious nonsurgical intervention improve pruritus reduce serum bile acids enhance standard care patients alagille syndrome longerterm safety efficacy data odevixibat population awaited ongoing openlabel assertext study albireo pharma ipsen company