Eltrombopag use in a patient with Wiskott–Aldrich syndrome. Issue 12 (23rd June 2017)

• Record Type: Journal Article
• Authors: Gabelli, Maria; Marzollo, Antonio; Notarangelo, Lucia Dora; Basso, Giuseppe; Putti, Maria Caterina
• Is Part Of: Pediatric blood & cancer. Volume 64:Issue 12(2017)
• Page Start: n/a
• Page End: n/a
• Publication Date: 2017-06-23
• Subjects: eltrombopag -- hematopoietic stem cell transplantation -- thrombocytopenia -- Wiskott–Aldrich syndrome; Tumors in children -- Periodicals; Blood -- Diseases -- Periodicals; Cancer in children -- Periodicals; 618.92
• DOI: 10.1002/pbc.26692
• Languages: English
• ISSNs: 1545-5009

Abstract

Abstract: Wiskott–Aldrich syndrome (WAS) is an inherited X‐linked disorder characterized by microthrombocytopenia, immunodeficiency, and eczema. Hematopoietic stem cell transplantation (HSCT) is the treatment of choice. Eltrombopag, a thrombopoietin receptor agonist, may be useful to prevent bleeding while awaiting HSCT. We present a case of a male with WAS, profound thrombocytopenia, and bleeding diathesis successfully managed with eltrombopag before HSCT. Eltrombopag was given for 32 weeks obtaining a stable platelet count without any platelet transfusion. The patient did not experience any bleeding symptom. Eltrombopag may be a suitable therapeutic option for patients with WAS and severe thrombocytopenia as "bridge" to definitive cure.