Neonatal cholestasis underpowered to detect portal hypertension in kids with AAT deficiency

While the need for liver transplantation is not urgent and the risk of death is low, portal hypertension nevertheless disrupts growth measures in children with alpha-1-antitrypsin (AAT) deficiency, a recent study has shown.

Moreover, having a history of neonatal cholestasis appears to be a weak predictor of portal hypertension, suggesting that there is a need for continuous monitoring and guidance of young AAT patients, regardless of such a disease history.

Of the 350 participants (median age, 4.2 years; 60 percent male), 21 percent had definite or possible, clinically evident portal hypertension at entry. Of the remaining 278 children without such condition at baseline, 18 eventually developed it over a median 2.5 years of follow-up. The resulting incidence rate was 2.1 per 100 person-years of follow-up.

Thirty-two patients either needed liver transplantation or died over 1,077 person-years of follow-up, yielding an annual incidence rate of 3.0 transplants per 100 person-years. Almost all the transplanted patients entered the study with definite or possible portal hypertension.

For all participants, the 2-year probability of developing definite or possible portal hypertension is 5 percent. Over the same time scale, the incidence of death or liver transplantation increased slowly, reaching a peak probability of 8 percent.    

Kaplan-Meier curves for portal hypertension-free survival further showed that the likelihood of such an event was comparable between children who did vs did not have a history of neonatal cholestasis. The same was true for liver transplant-free survival rates.