|
KMID : 0387820220290010012
|
|
Clinical Pediatric Hematology-Oncology
2022 Volume.29 No. 1 p.12 ~ p.16
|
|
|
Off Label Use of Eltrombopag and Recombinant Activated Factor VII in Wiskott-Aldrich Syndrome. A Case Report and Review of Literature
|
|
Adramerina1 Alkistis
Chainoglou Nancy
Theodoridou Stamatia
Teli Aikaterini
Economou Marina
|
|
|
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, pro-phylactic treatment with eltrombopag was attempted, in order to increase platelet count and limit bleeding episodes. Management of the patient with the above men-tioned agents led to a significant improvement in his clinical course and overall qual-ity 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, lim-ited data is available and is reviewed in the report.
|
|
|
KEYWORD
|
|
|
Wiskott-Aldrich syndrome, Thrombocytopenia, Thrombopoietin receptor agonists, Eltrombopag, Recombinant activated factor VII
|
|
|
FullTexts / Linksout information
|
|
|
|
|
|
Listed journal information
|
|
  
|