Clin Pediatr Hematol Oncol.  2022 Apr;29(1):12-16. 10.15264/cpho.2022.29.1.12.

Off Label Use of Eltrombopag and Recombinant Activated Factor VII in Wiskott-Aldrich Syndrome. A Case Report and Review of Literature

• Adramerina A
• Chainoglou N
• Theodoridou S
• Teli A
• Economou M

• KMID: 2529685
• DOI: http://doi.org/10.15264/cpho.2022.29.1.12

Abstract

Wiskott-Aldrich syndrome (WAS) is characterized by eczema, recurrent infections and microthrombocytopenia, with the latter being the most consistent finding in the course of the disease. The present case report describes a pediatric patient with WAS and severe bleeding phenotype resulting from a very low platelet count. In order to limit platelet transfusions and related risk of allo-immunization, use of recombinant activated factor VII (rFVIIa) for treatment of bleeding was decided. In addition, prophylactic treatment with eltrombopag was attempted, in order to increase platelet count and limit bleeding episodes. Management of the patient with the above mentioned agents led to a significant improvement in his clinical course and overall quality of life. This is the first report on off-label use of rFVIIa in a WAS patient. With regards to the off-label use of thrombopoietic agents in pediatric WAS patients, limited data is available and is reviewed in the report.

Keyword

Wiskott-Aldrich syndrome; Thrombocytopenia; Thrombopoietin receptor agonists; Eltrombopag; Recombinant activated factor VII