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

Yıl 2016, Cilt: 3 Sayı: 3, 141 - 4, 15.03.2016

Öz

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.

Kaynakça

  • Shin SH, Lee JW. The optimal immunosuppressive therapy for aplastic anemia. Int J Hematol. 2013; 97:564- 72.
  • 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.
  • 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.
  • 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.
  • Scheinberg P. Aplastic anemia: therapeutic updates in immunosuppression and transplantation. Hematology Am Soc Hematol Educ Program. 2012; 2012:292-300.
  • Scheinberg P ,Young NS. How I treat acquired aplastic anemia. Blood. 2012; 120(6):1185-96.
  • Young NS, Calado RT, Scheinberg P. Current concepts in the pathophysiology and treatment of aplastic anemia. Blood. 2006; 108:2509-19.
  • 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.
  • Kaushansky K. Historical review: megakaryopoiesis and thrombopoiesis. Blood. 2008; 111:981-6.
  • Kuter DJ. Thrombopoietin and thrombopoietin mimetics in the treatment of thrombocytopenia. Annu Rev Med. 2009; 60:193-206.
  • Geddis AE. Congenital amegakaryocytic thrombocytopenia. Pediatr Blood Cancer. 2011; 57:199-203.
  • 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.
  • 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.
  • 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.
  • Maciejewski JP, Selleri C. Evolution of clonal cytogenetic abnormalities in aplastic anemia. Leuk Lymphoma. 2004; 45(3):433-40.

Successful treatment of severe aplastic anemia with eltrombopag

Yıl 2016, Cilt: 3 Sayı: 3, 141 - 4, 15.03.2016

Öz

Kaynakça

  • Shin SH, Lee JW. The optimal immunosuppressive therapy for aplastic anemia. Int J Hematol. 2013; 97:564- 72.
  • 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.
  • 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.
  • 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.
  • Scheinberg P. Aplastic anemia: therapeutic updates in immunosuppression and transplantation. Hematology Am Soc Hematol Educ Program. 2012; 2012:292-300.
  • Scheinberg P ,Young NS. How I treat acquired aplastic anemia. Blood. 2012; 120(6):1185-96.
  • Young NS, Calado RT, Scheinberg P. Current concepts in the pathophysiology and treatment of aplastic anemia. Blood. 2006; 108:2509-19.
  • 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.
  • Kaushansky K. Historical review: megakaryopoiesis and thrombopoiesis. Blood. 2008; 111:981-6.
  • Kuter DJ. Thrombopoietin and thrombopoietin mimetics in the treatment of thrombocytopenia. Annu Rev Med. 2009; 60:193-206.
  • Geddis AE. Congenital amegakaryocytic thrombocytopenia. Pediatr Blood Cancer. 2011; 57:199-203.
  • 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.
  • 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.
  • 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.
  • Maciejewski JP, Selleri C. Evolution of clonal cytogenetic abnormalities in aplastic anemia. Leuk Lymphoma. 2004; 45(3):433-40.
Toplam 15 adet kaynakça vardır.

Ayrıntılar

Birincil Dil İngilizce
Bölüm Vaka Sunumu
Yazarlar

Ali Eser Bu kişi benim

Funda Tanrikulu

Tayfur Toptas

Suheyla Bozkurt Bu kişi benim

Erdem Kombak Bu kişi benim

İsik Atagunduz Bu kişi benim

Osman Kara

Ayse Tulin Tuglular Bu kişi benim

Yayımlanma Tarihi 15 Mart 2016
Yayımlandığı Sayı Yıl 2016 Cilt: 3 Sayı: 3

Kaynak Göster

APA Eser, A., Tanrikulu, F., Toptas, T., Bozkurt, S., vd. (2016). Successful treatment of severe aplastic anemia with eltrombopag: a case report. Medical Science and Discovery, 3(3), 141-4. https://doi.org/10.17546/msd.74493
AMA Eser A, Tanrikulu F, Toptas T, Bozkurt S, Kombak E, Atagunduz İ, Kara O, Tuglular AT. Successful treatment of severe aplastic anemia with eltrombopag: a case report. Med Sci Discov. Mart 2016;3(3):141-4. doi:10.17546/msd.74493
Chicago Eser, Ali, Funda Tanrikulu, Tayfur Toptas, Suheyla Bozkurt, Erdem Kombak, İsik Atagunduz, Osman Kara, ve Ayse Tulin Tuglular. “Successful Treatment of Severe Aplastic Anemia With Eltrombopag: A Case Report”. Medical Science and Discovery 3, sy. 3 (Mart 2016): 141-4. https://doi.org/10.17546/msd.74493.
EndNote Eser A, Tanrikulu F, Toptas T, Bozkurt S, Kombak E, Atagunduz İ, Kara O, Tuglular AT (01 Mart 2016) Successful treatment of severe aplastic anemia with eltrombopag: a case report. Medical Science and Discovery 3 3 141–4.
IEEE A. Eser, “Successful treatment of severe aplastic anemia with eltrombopag: a case report”, Med Sci Discov, c. 3, sy. 3, ss. 141–4, 2016, doi: 10.17546/msd.74493.
ISNAD Eser, Ali vd. “Successful Treatment of Severe Aplastic Anemia With Eltrombopag: A Case Report”. Medical Science and Discovery 3/3 (Mart 2016), 141-4. https://doi.org/10.17546/msd.74493.
JAMA Eser A, Tanrikulu F, Toptas T, Bozkurt S, Kombak E, Atagunduz İ, Kara O, Tuglular AT. Successful treatment of severe aplastic anemia with eltrombopag: a case report. Med Sci Discov. 2016;3:141–4.
MLA Eser, Ali vd. “Successful Treatment of Severe Aplastic Anemia With Eltrombopag: A Case Report”. Medical Science and Discovery, c. 3, sy. 3, 2016, ss. 141-4, doi:10.17546/msd.74493.
Vancouver Eser A, Tanrikulu F, Toptas T, Bozkurt S, Kombak E, Atagunduz İ, Kara O, Tuglular AT. Successful treatment of severe aplastic anemia with eltrombopag: a case report. Med Sci Discov. 2016;3(3):141-4.