ALGS News Briefs

Baby taking oral medication

Maralixibat Study Now Recruiting Patients With ALGS Under 12 Months of Age

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…

Cardiovascular system

Some Vascular Problems Can Affect Survival After Pediatric Liver Transplantation

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…

Infant in crib

FDA Approves Use of Maralixibat in Patients With ALGS Starting at 3 Months of Age

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…

laparoscopic surgery

Sign May Help Distinguish Biliary Atresia From Other Causes of Hepatic Cholestasis

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)…

FDA-Priority-Review

Odevixibat Indication for Treating ALGS Granted FDA Priority Review

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…

surgery

Risk Factors May Predispose Patients to ECMO Following Repair of PPAS

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…

group of people

Case Series: Relatives With ALGS Exhibit Diverse Phenotypes and Disease Severity

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…

liver transplant

Liver From SARS-CoV-2 RNA-Positive Donor Successfully Transplanted in Child With ALGS

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…

COVID19

Case Report: Liver Transplantation in a Pediatric Patient With ALGS and COVID-19

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…

Calculator and stethoscope

Researchers Develop Health State Vignettes for Alagille Syndrome

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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