Development of the Patient- and Observer-Reported PRUCISION Instruments to Assess Pruritus and Sleep Disturbance in Pediatric Patients with Cholestatic Liver Diseases

Gwaltney, Chad J.; Bean, Stephanie E.; Venerus, Meredith; Karlsson, Lisa; Warholic, Natalie; Kjems, Lise; Horn, Patrick T.

doi:10.1007/s12325-022-02261-8

Cited by 3 publications

(1 citation statement)

References 43 publications

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“…The ASSERT phase 3 trial [42] (NCT0467461) is currently evaluating the efficacy and safety of the use of odevixibat in patients with Alagille syndrome daily for 24 weeks. However, the itch measurement is different (an Albireo Observerreported scratching score called PRUCISION [43]), which may make the results of efficacy comparison across clinical trials different. Discrepancies in endpoints were present in the current study and are expected in ongoing trials.…”

Section: Strengths and Limitationsmentioning

confidence: 99%

Ileal Bile Acid Transporter Blockers for Cholestatic Liver Disease in Pediatric Patients with Alagille Syndrome: A Systematic Review and Meta-Analysis

Muntaha

Munir

Sajid

et al. 2022

JCM

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Alagille syndrome (ALGS) is a rare, debilitating inheritable disease that is associated with refractory pruritus due to chronic cholestasis. The following systemic review and meta-analysis presents the latest evidence for ileal bile acid transport (IBAT) blockers in AGLS patients in order to improve their efficacy. This study adhered to PRISMA 2020 Statement guidelines. A systematic search of PubMed/MEDLINE, Web of Science, Scopus, and the Cochrane library was conducted from inception until 23 October 2022. A combination of the following keywords was used: Alagille syndrome, therapeutics, treatment, therapy. Meta-analytical outcomes included effect directions of end-line changes in serum bile acids (sBAs), Itch Scale scores (ItchRO), Multidimensional Fatigue Scale scores, pediatric quality of life (QL), alanine aminotransferase (ALT), and total bilirubin. A total of 94 patients across four trials were enrolled and received maralixibat, odevixibat, or a placebo. There was a significant reduction in ItchRO scores by 1.8 points, as well as in sBAs by 75.8 μmol/L. Both the Multidimensional Fatigue Scale and Pediatric QL scale were also improved by 11.4 and 8.3 points, respectively. However, ALT levels were raised by 40 U/L. The efficacy of IBAT inhibitors across current trials was noted. Future trials may focus on the optimization of dosing regimens, considering gastrointestinal side effects and drug-induced ALT elevation in AGLS patients.

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Section: Strengths and Limitationsmentioning

confidence: 99%

Ileal Bile Acid Transporter Blockers for Cholestatic Liver Disease in Pediatric Patients with Alagille Syndrome: A Systematic Review and Meta-Analysis

Muntaha

Munir

Sajid

et al. 2022

JCM

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Exploring odevixibat’s efficacy in alagille syndrome: insights from recent clinical trials and IBAT inhibitor experiences

Jarasvaraparn,

Rodrigo,

Hartley

et al. 2024

Expert Opinion on Pharmacotherapy

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IBAT inhibitors in pediatric cholestatic liver diseases: Transformation on the horizon?

Sutton,

Sokol,

Kamath

2024

Hepatology

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Historically, the therapeutic options available to hepatologists managing cholestasis have been limited. Apart from bile acid–-binding resins and the choleretic ursodeoxycholic acid, the medical management of cholestasis in children has been predominately focused on managing the complications of cholestasis, namely pruritus, malnutrition, fat-soluble vitamin deficiencies, and portal hypertension. As such, invasive surgical procedures such as biliary diversion and liver transplantation may become the only options for progressive and unremitting cases of cholestasis. Particularly in the pediatric population, where debilitating pruritus is a common indication for a liver transplant, effective anti-cholestatic medications have the potential to prolong native liver survival without the need for biliary diversion. Ileal bile acid transporter (IBAT) inhibitors are a relatively new class of drugs which that target the ileal re-uptake of bile acids, thus interrupting the enterohepatic circulation and reducing the total bile acid pool size and exposure of the liver. Oral, minimally absorbed IBAT inhibitors have been demonstrated to reduce serum bile acid levels and pruritus with a minimal side effect profile in clinical trials in Alagille Ssyndrome and progressive familial intrahepatic cholestasis, leading to FDA and EMA approval. The indications for IBAT inhibitors will likely expand in the coming years as clinical trials in other adult and pediatric cholestatic conditions are ongoing. This review will summarize the published clinical and pre-clinical data on IBAT inhibitors and offer providers guidance on their practical use.

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Development of the Patient- and Observer-Reported PRUCISION Instruments to Assess Pruritus and Sleep Disturbance in Pediatric Patients with Cholestatic Liver Diseases

Cited by 3 publications

References 43 publications

Ileal Bile Acid Transporter Blockers for Cholestatic Liver Disease in Pediatric Patients with Alagille Syndrome: A Systematic Review and Meta-Analysis

Ileal Bile Acid Transporter Blockers for Cholestatic Liver Disease in Pediatric Patients with Alagille Syndrome: A Systematic Review and Meta-Analysis

Exploring odevixibat’s efficacy in alagille syndrome: insights from recent clinical trials and IBAT inhibitor experiences

IBAT inhibitors in pediatric cholestatic liver diseases: Transformation on the horizon?

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