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Liver disease complicating severe haemophilia in childhood
Insights
Severe classical hemophilia patients with abnormal liver tests showed liver damage. Cryoprecipitate is recommended over factor VIII concentrates to reduce risks in children with hemophilia.
Area of Science:
- Pediatric Hematology
- Hepatology
- Medical Research
Background:
- Children with severe classical hemophilia often present with persistently abnormal liver function tests.
- The underlying causes and implications of these liver abnormalities require further investigation.
Purpose of the Study:
- To investigate the liver histology in boys with severe classical hemophilia and abnormal liver function tests.
- To explore potential etiological factors, including viral hepatitis and alpha-1-antitrypsin deficiency.
- To assess the safety and efficacy of different factor VIII containing blood products in this population.
Main Methods:
- Liver biopsies were performed on five boys aged 2–9 years with severe classical hemophilia.
- Histopathological examination of liver tissue was conducted.
- Serological tests for hepatitis A and B, and alpha-1-antitrypsin levels were assessed.
Main Results:
- All five patients exhibited abnormal liver histology, with four showing chronic persistent hepatitis and one displaying chronic aggressive hepatitis with early cirrhosis.
- Evidence of prior hepatitis B infection was found in one patient, while three had antibodies to hepatitis A.
- Two patients had subnormal levels of alpha-1-antitrypsin.
- One patient developed haemobilia as a late complication post-biopsy.
Conclusions:
- Liver abnormalities are common in children with severe classical hemophilia and abnormal liver function tests.
- Exposure to factor VIII containing blood products may play a role in liver pathology.
- Cryoprecipitate is suggested as a safer alternative to large pool factor VIII concentrates for children with hemophilia to mitigate liver-related risks.
Abstract:
Liver biopsies were performed in 5 boys aged between 2 and 9 years with severe classical haemophilia who had persistently abnormal liver function tests. Abnormal histology was present in all; 4 had chronic persistent hepatitis and the fifth chronic aggressive hepatitis with early cirrhosis. Evidence of previous hepatitis B infection was present in one patient, 3 had antibodies to hepatitis, A, and 2 had subnormal levels of alpha-1-antitrypsin. Haemobilia occurred as a late complication of biopsy in one. The significance of these findings in young boys is discussed, as is the role of exposure to factor VIII containing blood products. It is concluded that cryoprecipitate should be used in preference to large pool factor VIII concentrates in children with haemophilia.