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Prognostic marker for liver disease due to alpha1-antitrypsin deficiency
D C Pferdmenges1, U Baumann, A Müller-Heine
1Department of Paediatric Gastroenterology and Hepatology, Hannover Medical School, Hannover, Germany.
Insights
Predicting liver disease progression in Alpha1-antitrypsin deficiency (A1ATD) PiZZ patients is challenging. Certain laboratory markers, not early symptoms, help determine prognosis and the need for liver transplantation in children with A1ATD.
Area of Science:
- Hepatology
- Genetics
- Pediatrics
Background:
- Alpha1-antitrypsin deficiency (A1ATD) PiZZ genotype presents a risk for liver disease, including cirrhosis and portal hypertension, in a subset of patients.
- Understanding the disease course and identifying prognostic factors in pediatric A1ATD is crucial for timely intervention.
Purpose of the Study:
- To investigate the clinical course of liver disease in children with A1ATD PiZZ genotype.
- To identify prognostic factors associated with liver disease severity and outcomes in this pediatric population.
Main Methods:
- Retrospective review of clinical and laboratory data from 53 pediatric patients with A1ATD PiZZ genotype.
- Patients were categorized into 'good prognosis' (living with own liver) and 'bad prognosis' (liver transplant or deceased) groups.
Main Results:
- Neonatal cholestasis and other anamnesis parameters lacked prognostic significance.
- Laboratory parameters including thrombocytes, bilirubin, prothrombin time, choline-sterase, gamma-GT, and GOT significantly correlated with adverse outcomes, such as liver transplantation or death.
Conclusions:
- Early prediction of liver disease outcome in A1ATD PiZZ is difficult based on initial presentation.
- Specific laboratory markers are valuable for predicting prognosis and guiding management in children with A1ATD.
- Regular patient follow-up is essential for monitoring disease progression and outcomes.
Background:
Only some Alpha1-antitrypsin deficiency (A1ATD) PiZZ patients develop liver cirrhosis and portal hypertension. Aim of the study was to investigate the course of liver disease associated with PiZZ A1ATD and to determine prognostic factors.
Patients:
We retrospectively reviewed the clinical and laboratory data of all PiZZ children up to 18 years of age admitted to our centre since 1978. 53 patients (age at first visit 2 days to 12 years) met our criteria.
Methods:
The children were divided into 2 groups: group 1 'bad prognosis', meaning the patients which were on the waiting list for liver transplantation (LTx), had a liver transplantation or had died, and group 2 'good prognosis', containing the patients they were living with their own liver. We analysed family history including smoking, gestational age, maternal age at delivery, date of birth, sex, neonatal history, breast-feeding, symptoms at presentation, clinical and laboratory data and date of LTx and/or death.
Results:
Various anamnesis parameters such as manifestation of neonatal cholestasis showed no prognostic significance. In contrast the laboratory parameters thrombocytes (p=0.008), bilirubin (p<0.001), prothrombin time (p<0.001), choline-sterase (p<0.001), gamma-GT (p=0.001) and GOT (p=0.002) showed a correlation with a liver transplantation and/or death.
Conclusion:
Prognosis is difficult to determine at an early stage of this disease, but various laboratory parameters can help to predict an outcome. Therefore a regular follow-up is necessary for the children.
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