Childhood liver transplantation
Liver involvement in homozygous AATM (PI-ZZ) is highly individual, although the majority of affected children and adolescents fortunately show no or only very mild liver involvement.
Nevertheless, in larger centers, it must be assumed that in approximately 5-101 children and adolescents with TP3T (with PI-ZZ), there is a certain probability that the liver will eventually be damaged by the accumulation of alpha-1-antitrypsin to such an extent that a liver transplant (LTx) becomes necessary. However, precise figures are not available, and it is likely that many children are not even referred to specialized centers.
Unfortunately, AAT deficiency is often not diagnosed at all or is diagnosed too late, and therefore I strongly advocate that the serum concentration of AAT should be determined in every newborn with prolonged jaundice (prolonged yellowing of the skin and sclera). This test should also be performed in every child or adolescent if elevated liver enzymes (AST and ALT) are detected. Since both of these tests are often not carried out consistently, early diagnosis is frequently missed.
All parents of affected children must be professionally informed and educated about the topic of "liver transplantation," and it is extremely important that this information is not solely obtained from the internet, as this only increases the already considerable uncertainty surrounding the subject! Please feel free to use our specially set up [website/service/information - context needed]. Website alpha1bonn.de and get to know the specialist team at the University Hospital Bonn.
It is very important to me to inform you here about the most important aspects of LTX:
- Compared to all other transplantation modalities, liver transplantation is by far the best option for infants, children, and adolescents! Approximately 951% of those affected lead completely normal lives.
- In Germany (and other European countries), children receive a comparatively low level of immunosuppression, so we hardly see any relevant side effects.
- With early planning, it is possible for every affected person to receive a high-quality organ at the appropriate time, and in the case of small children, living liver donation can also be discussed.
- Even children listed as "inactive" accumulate 2 points per quarter on the waiting list and relatively quickly reach the maximum of 40 points. If they are then activated on the waiting list, they can expect a very good supply of organs. From the age of 16, children are treated the same as adults, and the prioritization process for organ allocation is lifted.
- In children with chronic liver disease, a deterioration in liver function is often only clinically apparent in a very late stage! There are children with liver cirrhosis who show little to no visible signs. Therefore, laboratory values and ultrasound findings are of particular importance for assessing the overall situation and determining further treatment.
- Children and young people with relevant liver involvement In my opinion, they should also be presented to a transplant center for a second opinion at certain intervals. Safety first!
- In the future, I expect clinical trials for children/adolescents, analogous to those for adults with AATM, which could lead to corresponding alternative therapy options to liver transplantation.
Prof. Dr. Rainer Ganschow
University Hospital Bonn
May 2021
