Farmacoriflessioni
Malattie colestatiche del bambino e i nuovi farmaci IBAT inibitori
Cholestatic liver disease in children and the new ibat inhibitor drugs
Marco Sciveres1, Silvio Veraldi1, Francesco Cirillo2, Andrea Pietrobattista1, Giuseppe Maggiore1
1Epatologia e Trapianto di fegato, IRCCS Ospedale Pediatrico “Bambino Gesù”, Roma
2Unità di Gastroenterologia ed Epatologia, Ospedale Pediatrico “Santobono-Pausilipon”, Napoli
Settembre 2026 - pagg. 447 -453 | DOI: 10.53126/MEB45447
Abstract
Cholestatic diseases in children are a heterogeneous group of disorders that, with few exceptions, have no cure. For decades, off-label drugs and/or drugs with little evidence of efficacy have been used to treat pruritus or as supportive therapy. In recent years, a family of molecules known as bile acid transporter inhibitors (IBATis) has been developed, with two of these being approved for treating pruritus in progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome (ALGS). Blocking the ileal reabsorption of bile acids (BA) lowers serum levels. This contributes to reducing cholestatic pruritus. This mechanism of action opens scenarios that speculatively may also include a potential benefit in other cholestatic diseases and even on the consequences of chronic cholestasis. This is a narrative review of the literature that includes the most recent communications and summarises data on efficacy and safety of IBATis in the treatment of pruritus in PFIC and ALGS in children. It also includes a description of the latest results from their use in a real-world setting. Reports on off-label use in adults and their feedbacks are also discussed. This review aims to help physicians understand the potential and limitations of these new drugs in the treatment of cholestatic pruritus.
Riassunto
Le malattie colestatiche nei bambini rappresentano un gruppo eterogeneo di disturbi che, salvo poche eccezioni, non hanno una cura. Per decenni, sono stati utilizzati farmaci off-label e/o farmaci con scarse prove di efficacia per trattare il prurito o come terapia di supporto. Negli ultimi anni è stata sviluppata una famiglia di molecole nota come inibitori del trasportatore degli acidi biliari (IBATi), due dei quali sono stati approvati per il trattamento del prurito nella colestasi intraepatica familiare progressiva (PFIC) e nella sindrome di Alagille (ALGS). Il blocco del riassorbimento ileale degli acidi biliari (BA) riduce i loro livelli sierici, contribuendo così a diminuire il prurito colestatico. Questo meccanismo d'azione apre scenari che, a livello speculativo, potrebbero includere anche un potenziale beneficio in altre malattie colestatiche e persino sulle conseguenze della colestasi cronica. Questa è una revisione narrativa della letteratura che include le comunicazioni più recenti e riassume i dati sull'efficacia e la sicurezza degli IBATi nel trattamento del prurito nella PFIC e nella ALGS nei bambini. Include inoltre una descrizione degli ultimi risultati derivanti dal loro utilizzo in contesti reali (real-world). Vengono infine discussi i report sull'uso off-label negli adulti e i relativi riscontri. Questa revisione mira ad aiutare i medici a comprendere le potenzialità e i limiti di questi nuovi farmaci nel trattamento del prurito colestatico.
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