This huge collaborative study with 130 patients provides a great deal of information about familial intrahepatic cholestasis type 1 (FIC1). Key findings:
Survival analysis showed an overall native liver survival (NLS) of 44% at age 18 years. NLS was comparable among FIC1-A, FIC1-B, and FIC1-C (% NLS at age 10 years: 67%, 41%, and 59%, respectively; P = 0.12)
The number of predicted protein truncating mutations did not correlate with natural history or prognosis
This practice guidance (with 276 references) is an update from similar guidelines published in 2012.
Key Points For Children:
Children with cirrhosis and ascites should be referred for evaluation for LT
Children undergoing LVP should receive 25% albumin infusion of 0.5-1.0 g/kg, or 6-8 g per liter of ascites removed.
Diagnostic paracentesis should be performed in children with ascites and fever, abdominal pain, or clinical deterioration. The risks and benefits of this procedure for use in all children with new ascites but without these symptoms have not been defined.
Two recent studies have suggested that tenofovir may be more effective for hepatitis B virus (HBV) infections.
T C-F Yip et al. Gastroenterol 2020; 158: 215-25.
J Choi et al. JAMA Oncol 2019; 5: 30-6. (Reviewed in a commentary by P Lampertico, M Colombo. Gastroenterol 2019; 157: 1682-88)
In the first retrospective study from Hong Kong, the authors analyzed 29,350 consecutive treated-patients (mean age 52.9 years). 1309 were treated with tenofovir disoproxil fumarate (TDV) and 28,041 were treated with entecavir. Key findings:
TDF-treated patients were younger (mean age 43.2 years vs. 53.4 years) and had less cirrhosis at baseline (2.9% vs. 13.6%).
After a median follow-up of 3.6 years, 8 TDF-treated patients (0.6%) and 1386 (4.9%) of entecavir-treated patients developed hepatocellular carcinoma (HCC). The authors note that TDF maintained a lower rate of HCC after propensity score weighting (hazard ratio of 0.36)
The second study was a nationwide population cohort database with >24,000 patients –all with ALT >80. Key finding:
HCC was significantly lower in TDF group than in the entecavir group, the percent person-years being 0.64 compared to 1.06; though, there was not a lower mortality rate or a lower liver transplantation rate.
The commentary associated with the latter study makes the following points:
Both TDF and entecavir could prevent “the incidence and mortality of HCC …in >85% of patients who received [them] for years.”
Studies comparing TDF and entecavir have come up with conflicting results. “Three studies in Korea, the U.S, and Europe reported no differences between NAs even after patient matching by a propensity score.”
“Cumulatively, all these studies deliver the reassuring message of a robust risk reduction of liver cancer taking place in patients with chronic hepatitis B who experience prolonged virus suppression after NA therapy, but currently they fail to provide convincing evidence that one NA is superior to the other one in determining such clinical benefit.”
My take: Tenofovir may be better but the answer is not definitive; due to lack of randomization, there may still be confounding variables in which sicker patients are receiving entecavir and this could be contributing to the difference in outcomes. Also, in patients with bone disease and renal impairment, tenofovir alafenamide (TAF) or entecavir is recommended.
These images (S Sengupta, S Bose. NEJM 2019; 380: 472) show hyperpigmented macules on the lips, oral mucosa and nose in the first frame, a target sign on CT scan indicative of intussception, and a jejunal resection with polyps that triggered the intussception. Related blog post: Update for Peutz-Jegher Syndrome