Hepatitis-associated Aplastic Anaemia in Children
Anne-Kathrin Böske1, Annette Sander2, Karl-Walter Sykora2
1Clinic for Pediatric Nephrology, Hepatology and Metabolic Disorders, Hannover Medical School, Hannover, Germany.
Insights
Children with idiopathic acute liver failure (IALF) face a high risk of bone marrow failure (BMF). Early blood count monitoring and aggressive treatments like liver transplantation (LTx), immunosuppressive therapy (IST), and hematopoietic stem cell transplantation (HSCT) are crucial for improving outcomes.
Area of Science:
- Pediatric Hematology
- Hepatology
- Transplantation Medicine
Background:
- Idiopathic acute liver failure (IALF) in children poses a significant risk for developing life-threatening bone marrow failure (BMF).
- Hepatitis-associated aplastic anemia (HAAA) is a critical complication in pediatric IALF patients.
- Understanding HAAA's development, therapy, and prognosis is vital for patient management.
Purpose of the Study:
- To describe the clinical course, treatment strategies, and outcomes of children with HAAA.
- To compare the prognosis of HAAA with isolated acquired aplastic anemia (SAA).
- To identify key factors influencing survival in pediatric HAAA.
Main Methods:
- Retrospective analysis of 18 pediatric HAAA cases (1984-2017).
- Comparison with data from patients with isolated SAA.
- Review of treatments including liver transplantation (LTx), immunosuppressive therapy (IST), and hematopoietic stem cell transplantation (HSCT).
Main Results:
- Fifteen HAAA patients met severe aplastic anemia (SAA) criteria; 11 underwent LTx, 6 recovered without it.
- BMF signs (thrombocytopenia, leucocytopenia) preceded LTx in all cases.
- Eight patients achieved hematological remission, 6 received HSCT; 7 died from infections/bleeding. Isolated SAA patients had a 73% remission rate with IST alone.
Conclusions:
- Early and regular blood count monitoring (0-22 days post-onset) is essential for IALF patients.
- Aggressive treatment combining LTx, IST, and HSCT shows promise in improving HAAA prognosis.
- The etiology of HAAA remains largely unknown despite extensive investigations.
Background:
Children with idiopathic acute liver failure (IALF) are at a high risk of developing life-threatening bone marrow failure (BMF). The aim of the study was to describe the development, therapy and prognosis of this hepatitis-associated aplastic anaemia (HAAA) in comparison to isolated acquired aplastic anaemia.
Results:
We retrospectively found 18 patients (9 female) of HAAA between 1984 and 2017 with an age of 1.4-16.4 years. Fifteen of them fulfilled the SAA criteria, 3 had a bone marrow hypoplasia. Eleven of these children received liver transplantation (LTx) (these were 11 of 42 (26%) children receiving LTx for IALF), 6 patients recovered without LTx. The first signs of BMF, thrombocytopaenia and leucocytopaenia, occurred before LTx in all cases. During the follow-up period 8 patients reached haematological remission, 6 received haematopoietic stem cell transplantation (HSCT). Seven children died in a median of 304 days after the first symptoms mostly because of bleedings and infections. To date, extensive investigations failed to detect a genetically, viral or immunological aetiology. No AA was diagnosed in the 41 patients receiving liver transplants during the same period for ALF of known aetiology. As a comparison group, we collected the data of patients with isolated SAA. 73% achieved a remission after Immunosuppressive therapy (IST) without HSCT, and none of them died during the follow-up period.
Conclusion:
Blood counts should be examined early and regularly (0-22 days after onset) in patients with IALF. Aggressive treatment with LTx, IST and HSCT appears to improve the prognosis.
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