CASE REPORT
A 9-year-old patient with severe haemophilia A complicated by factor VIII inhibitor treated with emicizumab – case report and literature review
1
Medical University, Lublin, Poland
2
Student Research Group at the Department of Hematology, Oncology and Children’s Transplantology, Medical
University, Lublin, Poland
3
Department of Transfusion Medicine, Children`s University Hospital, Lublin, Poland
Corresponding author
Justyna Małgorzata Tomasik
Medical University of Lublin, Aleje Racławickie 1, 20-059, Lublin, Poland
Medical University of Lublin, Aleje Racławickie 1, 20-059, Lublin, Poland
J Pre Clin Clin Res. 2024;18(2):128-134
KEYWORDS
TOPICS
ABSTRACT
Introduction:
The case is presented of a 10-year-old boy with severe Haemophilia A, in whom standard therapy led to the development of an inhibitor to factor VIII. Immunological tolerance induction (ITI) did not yield the expected result, and inhibitor titers were not reduced. Due to numerous recurrent bleeds, prophylaxis with activated prothrombin complex concentrate (aPCC) was initiated, with recombinant factor VIIa administered in the case of bleeding episodes. Despite second and third-line ITI treatments, desired outcomes were not achieved, leading to therapy discontinuation and initiation of emicizumab treatment. The new therapy significantly reduced bleeding occurrences.
Results:
This case clearly illustrates the greater efficacy of emicizumab compared to other drugs, confirming its application in patients with developed factor VIII inhibitors where ITI therapy was ineffective. Prophylaxis with this antibody significantly reduces bleeding frequency and improves the patient’s quality of life.
The case is presented of a 10-year-old boy with severe Haemophilia A, in whom standard therapy led to the development of an inhibitor to factor VIII. Immunological tolerance induction (ITI) did not yield the expected result, and inhibitor titers were not reduced. Due to numerous recurrent bleeds, prophylaxis with activated prothrombin complex concentrate (aPCC) was initiated, with recombinant factor VIIa administered in the case of bleeding episodes. Despite second and third-line ITI treatments, desired outcomes were not achieved, leading to therapy discontinuation and initiation of emicizumab treatment. The new therapy significantly reduced bleeding occurrences.
Results:
This case clearly illustrates the greater efficacy of emicizumab compared to other drugs, confirming its application in patients with developed factor VIII inhibitors where ITI therapy was ineffective. Prophylaxis with this antibody significantly reduces bleeding frequency and improves the patient’s quality of life.
Tomasik JM, Szuba J, Chrostowski K, Woźnica-Karczmarz I. A 9-year-old patient with severe haemophilia A complicated by factor VIII inhibitor
treated with emicizumab – case report and literature review. J Pre-Clin Clin Res. 2024; 18(2): 128–134. doi: 10.26444/jpccr/187549
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