A new open-label, phase 2 study set to evaluate the safety and tolerability of maralixibat in infants with Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis is currently recruiting infants under 12 months of age. Following the US Food and Drug Administration (FDA) approval of maralixibat for the treatment of cholestatic pruritus in patients with ALGS…
Pediatric liver transplantation is performed for a variety of indications, including Alagille syndrome (ALGS), biliary atresia, hepatoblastoma, and metabolic liver disease, researchers wrote in a retrospective study published in the journal Digestive and Liver Diseases. They sought to examine data on how posttransplant complications affected long-term survival and quality of life among children who had…
The US Food and Drug Administration (FDA) has approved maralixibat (Livmarli®) for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) aged 3 months or older, Mirum Pharmaceuticals announced. The medication was previously approved for the treatment of patients aged 1 year or older. The reduction in age is based on positive safety…
The intraoperative hepatic subcapsular spider-like telangiectasia (HSST) sign may help in differentiating biliary atresia (BA) from other causes of hepatic cholestasis, including Alagille syndrome (ALGS), according to a study published in BMC Pediatrics. “We initially thought that HSST sign might be related to liver fibrosis, but later observed that young patients (younger than 20 days)…
Odevixibat (Bylvay®) is a step closer to becoming an option for treating patients with Alagille syndrome (ALGS), after the US Food and Drug Administration (FDA) accepted Albireo Pharma’s supplemental New Drug Application and issued a Prescription Drug User Fee Act action date of June 15, 2023. “With the phase 3 randomized, placebo-controlled ASSERT data and…
Surgical repair of peripheral pulmonary artery stenosis (PPAS) may be followed by acute lung injury, with severe cases requiring support with extracorporeal membrane oxygenation (ECMO), according to findings from a retrospective study published in JTCVS Open. It is presumed that such acute lung injury (ie, lung reperfusion injury) is caused by an acute postsurgical increase…
A highly variable phenotype and differing disease severity has been reported in 4 family members with Allagile syndrome (ALGS). The case series was published in the International Journal of Pediatrics and Child Health. The proband, the third-born child in the family, presented with a globular abdomen, loss of appetite, and poor growth at 18 months…
A pediatric patient with Alagille syndrome (ALGS) underwent a successful liver transplantation from a deceased asymptomatic severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) RNA-positive pediatric donor without evidence of viral transmission. In addition to this patient, 6 other patients with different diagnoses received organs from the same donor or a second pediatric donor who also…
Liver transplantation was successfully performed in a pediatric patient with Alagille syndrome (ALGS) and a concomitant severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection, according to a case report recently published in Hepatology Research. The patient, a 3-year-old girl with liver cirrhosis, was admitted to a hospital for deceased-donor liver transplantation. As the patient’s liver…
Researchers reported that patients with Alagille syndrome and their caregivers have a wide range of concerns regarding the disease and its treatment and that health state vignettes may be useful in estimating the cost-effectiveness of future treatments. The study was published in Value in Health. Alagille syndrome is a rare cholestatic liver disorder that imposes…
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