I recently attended an online lecture which reviewed Alagille Syndrome and the emergence of an IBAT inhibitor for the management of cholestatic pruritus. Selected slides from Mirum Pharmaceutical Lecture: “Updates in the Treatment of Cholestatic Pruritus in Patients With Alagille Syndrome.” *I have no financial disclosures or conflict of interests in this medication or company.

• The severe itching which is seen in most patients with Alagille is often quite detrimental to quality of life. It impacts sleep, causes irritability, skin damage, and physical distcomfort
• Typically, the first week of receiving the medication, it is started at1/2 the maintenance dose.
• Monitoring response can be done with the ItchCheck App, Itch score or Clinical scratch score
• Monitoring hepatic blood tests and periodic monitoring of fat soluble vitamins is recommended

My take: Though Alagille syndrome is a multisystem disease, improvement in pruritus due to cholestasis with an oral daily medication is an important advance/option. There is little systemic absorption and thus far a reassuring safety profile.

Related blog posts:

• NASPGHAN Alagille Syndrome Webinar
• GALA: Alagille Study
• Liver Briefs: HLH in Infancy, Maralixibat for Alagille Syndrome, Liver Disease Due to Inborn Errors of Immunity
• Intracranial Hypertension & Papilledema with Alagille Syndrome
• Explaining Differences in Disease Severity for Alagille Syndrome