Successful treatment of severe aplastic anemia with eltrombopag: a case report

Abstract

Introduction: Aplastic anemia is a clinical syndrome characterized by peripheral pancytopenia and deficiency of hematopoietic precursors in the bone marrow. Allogeneic hematopoietic stem cell transplantation (AHSCT) should be considered as first line treatment for young patients with an available donor. However,alternative therapy options are scant in patients who are not candidates for transplantation. Here, we report the efficacy of eltrombopag in a case of severe aplastic anemia.

Case Presentation: Twenty-two years old female patient was admitted to the Marmara University Hospital because of severe aplastic anemia. There was not an available HLA-matched sibling donor and immunosuppressive treatment with horse-derived ATG (40 mg per day for 5 days) and cyclosporine (5 mg/kg per day) was started. At the sixth month of therapy she was still in need of transfusion. Eltrombopag was prescribed at a dose of 50 mg and the dose was increased up to 150 mg per day in 2 months. Successful response was noted within 2 weeks of 150 mg dosage and this response was sustained at the 4th month following  discontinuation of drug.

Conclusions: Eltrombopag provides good and permanent clinical response in refractory severe aplastic anemia.

Keywords

• Eltrombopag, Aplastic anemia

Successful treatment of severe aplastic anemia with eltrombopag

Öz

References

1. Shin SH, Lee JW. The optimal immunosuppressive therapy for aplastic anemia. Int J Hematol. 2013; 97:564- 72.
2. Maciejewski J, Selleri C, Anderson S, Young NS. Fas antigen expression on CD34+ human marrow cells is induced by interferon gamma and tumor necrosis factor alpha and potentiates cytokine-mediated hematopoietic suppression in vitro. Blood. 1995; 85(11):3183-90.
3. Viollier R, Passweg J, Gregor M, Favre G, Kühne T, Nissen C, et al. Quality-adjusted survival analysis shows differences in outcome after immunosuppression or bone marrow transplantation in aplastic anemia. Ann Hematol. 2005; 84(1): 47-55.
4. Scheinberg P, Nunez O, Weinstein B, Scheinberg P, Biancotto A, Wu CO, et al. Horse versus rabbit antithymocyte globulin in acquired aplastic anemia. N Engl J Med. 2011; 365(5):430-8.
5. Scheinberg P. Aplastic anemia: therapeutic updates in immunosuppression and transplantation. Hematology Am Soc Hematol Educ Program. 2012; 2012:292-300.
6. Scheinberg P ,Young NS. How I treat acquired aplastic anemia. Blood. 2012; 120(6):1185-96.
7. Young NS, Calado RT, Scheinberg P. Current concepts in the pathophysiology and treatment of aplastic anemia. Blood. 2006; 108:2509-19.
8. Di Bona E, Rodeghiero F, Bruno B, Gabbas A, Foa P, Locasciulli A, et al. Rabbit antithymocyte globulin (r-ATG) plus cyclosporine and granulocyte colony stimulating factor is an effective treatment for aplastic anaemia patients unresponsive to a first course of intensive immunosuppressive therapy. Br J Haematol. 1999;107:330-4.

9. Kaushansky K. Historical review: megakaryopoiesis and thrombopoiesis. Blood. 2008; 111:981-6.
10. Kuter DJ. Thrombopoietin and thrombopoietin mimetics in the treatment of thrombocytopenia. Annu Rev Med. 2009; 60:193-206.
11. Geddis AE. Congenital amegakaryocytic thrombocytopenia. Pediatr Blood Cancer. 2011; 57:199-203.
12. Olnes MJ, Scheinberg P, Calvo KR, Desmond R, Tang Y, Dumitriu B, et al. Eltrombopag and improved hematopoiesis in refractory aplastic anemia. N Engl J Med. 2012; 367(1):11-19.
13. Desmond R, Townsley DM, Dumitriu B, Olnes MJ, Scheinberg P, Bevans M, et al. Eltrombopag restores trilineage hematopoiesis in refractory severe aplastic anemia that can be sustained on discontinuation of drug. Blood. 2014; 123(12):1818-25.
14. Saleh MN, Bussel JB, Cheng G, Meyer O, Bailey CK, Arning M, et al. Safety and efficacy of eltrombopag for treatment of chronic immune thrombocytopenia: results of the long-term, open-label EXTEND study. Blood. 2013; 121(3):537-45.
15. Maciejewski JP, Selleri C. Evolution of clonal cytogenetic abnormalities in aplastic anemia. Leuk Lymphoma. 2004; 45(3):433-40.