Real-world experience of maralixibat in Alagille syndrome: Novel findings outside of clinical trials.
J Pediatr Gastroenterol Nutr
; 78(3): 506-513, 2024 Mar.
Article
em En
| MEDLINE
| ID: mdl-38334237
ABSTRACT
OBJECTIVE:
Maralixibat, an ileal bile acid transporter inhibitor, is the first drug approved by the U.S. Food and Drug Administration for the treatment of cholestatic pruritus in patients aged ≥3 months with Alagille syndrome (ALGS). Approval was based on reductions in pruritus from the pivotal ICONIC trial, information from two additional trials (ITCH and IMAGO), and long-term extension studies. Although participants in these trials met strict inclusion and exclusion criteria, patients have received maralixibat under broader circumstances as part of an expanded access program or commercially. The expanded access and postapproval settings inform a real-world understanding of effectiveness and safety. The objective was to report on the use of maralixibat in the real-world setting in eight patients who otherwise would not have met entrance criteria for the clinical trials, providing unique insights into its effectiveness in the management of ALGS.METHODS:
We reviewed records of patients with ALGS who received maralixibat but would have been excluded from trials due to surgical biliary diversion, reduction of antipruritic/cholestatic concomitant medications, administration of medication through a gastrostomy or nasogastric tube, or use in patients under consideration for transplantation.RESULTS:
Maralixibat appeared to be effective with reductions in pruritus compared to baseline. Consistent with clinical trials, maralixibat was well tolerated without appreciable gastrointestinal complications. Liver enzyme elevations were observed but were interpreted as consistent with normal fluctuations observed in ALGS, with no increases in bilirubin.CONCLUSION:
Maralixibat may be effective and well tolerated in patients with ALGS in broader clinical contexts than previously reported.Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Benzotiepinas
/
Colestase
/
Síndrome de Alagille
Tipo de estudo:
Diagnostic_studies
Limite:
Humans
/
Infant
Idioma:
En
Revista:
J Pediatr Gastroenterol Nutr
Ano de publicação:
2024
Tipo de documento:
Article
País de afiliação:
Estados Unidos
País de publicação:
Estados Unidos