Eltrombopag for secondary failure of platelet recovery post-allogeneic hematopoietic stem cell transplant in children
Corresponding Author
Salah Ali
Division of Hematology/Oncology/BMT, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada
Correspondence
Salah Ali, The Hospital for Sick Children, Toronto, ON, Canada.
Email: [email protected]
Search for more papers by this authorAdam Gassas
Division of Hematology/Oncology/BMT, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada
Search for more papers by this authorMelanie Kirby-Allen
Division of Hematology/Oncology/BMT, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada
Search for more papers by this authorJoerg Krueger
Division of Hematology/Oncology/BMT, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada
Search for more papers by this authorMuhammad Ali
Division of Hematology/Oncology/BMT, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada
Search for more papers by this authorTal Schechter
Division of Hematology/Oncology/BMT, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada
Search for more papers by this authorCorresponding Author
Salah Ali
Division of Hematology/Oncology/BMT, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada
Correspondence
Salah Ali, The Hospital for Sick Children, Toronto, ON, Canada.
Email: [email protected]
Search for more papers by this authorAdam Gassas
Division of Hematology/Oncology/BMT, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada
Search for more papers by this authorMelanie Kirby-Allen
Division of Hematology/Oncology/BMT, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada
Search for more papers by this authorJoerg Krueger
Division of Hematology/Oncology/BMT, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada
Search for more papers by this authorMuhammad Ali
Division of Hematology/Oncology/BMT, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada
Search for more papers by this authorTal Schechter
Division of Hematology/Oncology/BMT, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada
Search for more papers by this authorAbstract
Secondary failure of platelet engraftment occurs in 20% of patients undergoing allogeneic HSCT and is associated with poor outcome. Currently, there are no guidelines for treatment of late thrombocytopenia and platelet transfusion is the mainstay of treatment. Here, we describe the use of Eltrombopag to treat secondary failure of platelet recovery following HSCT in a child with severe aplastic anemia. Eltrombopag resulted in recovery of platelet count with no need for platelet transfusion support with no reported side effects. Eltrombopag may be used successfully in children with secondary failure of platelet recovery post-HSCT for SAA.
REFERENCES
- 1Bruno B, Gooley T, Sullivan KM, et al. Secondary failure of platelet recovery after hematopoietic stem cell transplantation. Biol Blood Marrow Transplant. 2001; 7: 154-162.
- 2Nash RA, Gooley T, Davis C, Appelbaum FR. The problem of thrombocytopenia after hematopoietic stem cell transplantation. Oncologist. 1996; 1: 371-380.
- 3Yamazaki R, Kuwana M, Mori T, et al. Prolonged thrombocytopenia after allogeneic hematopoietic stem cell transplantation: associations with impaired platelet production and increased platelet turnover. Bone Marrow Transplant. 2006; 38: 377-384.
- 4Tanaka T, Inamoto Y, Yamashita T, et al. Eltrombopag for treatment of thrombocytopenia after allogeneic hematopoietic cell transplantation. Biol Blood Marrow Transplant. 2016; 22: 919-924.
- 5Bussel JB, de Miguel PG, Despotovic JM, et al. Eltrombopag for the treatment of children with persistent and chronic immune thrombocytopenia (PETIT): a randomised, multicentre, placebo-controlled study. Lancet Haematol. 2015; 2: e315-e325.
- 6Olnes MJ, Scheinberg P, Calvo KR, et al. Eltrombopag and improved hematopoiesis in refractory aplastic anemia. N Engl J Med. 2012; 367: 11-19.
- 7Saleh MN, Bussel JB, Cheng G, 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: 537-545.
- 8Bussel JB, Cheng G, Saleh MN, et al. Eltrombopag for the treatment of chronic idiopathic thrombocytopenic purpura. N Engl J Med. 2007; 357: 2237-2247.
- 9Bussel JB, Provan D, Shamsi T, et al. Effect of eltrombopag on platelet counts and bleeding during treatment of chronic idiopathic thrombocytopenic purpura: a randomised, double-blind, placebo-controlled trial. Lancet. 2009; 373: 641-648.
- 10Cheng G, Saleh MN, Marcher C, et al. Eltrombopag for management of chronic immune thrombocytopenia (RAISE): a 6-month, randomised, phase 3 study. Lancet. 2011; 377: 393-402.
- 11Grainger JD, Locatelli F, Chotsampancharoen T, et al. Eltrombopag for children with chronic immune thrombocytopenia (PETIT2): a randomised, multicentre, placebo-controlled trial. Lancet. 2015; 386: 1649-1658.
- 12Neunert C, Despotovic J, Haley K, et al. Thrombopoietin receptor agonist use in children: data from the pediatric ITP consortium of North America ICON2 study: thrombopoietin receptor agonist use in children. Pediatr Blood Cancer. 2016; 63: 1407-1413.
- 13Fujimi A, Kamihara Y, Hashimoto A, et al. Identification of antithrombopoietin receptor antibody in prolonged thrombocytopenia after allogeneic hematopoietic stem cell transplantation treated successfully with Eltrombopag. Int J Hematol. 2015; 102: 471-476.
- 14Dyba J, Tinmouth A, Bredeson C, et al. Eltrombopag after allogeneic haematopoietic cell transplantation in a case of poor graft function and systematic review of the literature. Transfusion Medicine. 2016; 26: 202-207.
- 15Reid R, Bennett JM, Becker M, et al. Use of eltrombopag, a thrombopoietin receptor agonist, in post-transplantation thrombocytopenia. American Journal of Hematology. 2012; 87: 743-745.
- 16Liesveld JL, Phillips GL, Becker M, et al. A Phase 1 trial of eltrombopag in patients undergoing stem cell transplantation after total body irradiation. Biol Blood Marrow Transplant. 2013; 19: 1745-1752.
- 17Kuter DJ. Thrombopoietin and thrombopoietin mimetics in the treatment of thrombocytopenia. Annu Rev Med. 2009; 60: 193-206.
- 18Stepan DE, Sergis-Deavenport E, Kelly R, et al. Safety profile of AMG 531 in healthy volunteers and in thrombocytopenic patients with immune thrombocytopenic purpura. Blood. 2015; 106: 361a-361a.
- 19Desmond R, Townsley DM, Dumitriu B, et al. Eltrombopag restores trilineage hematopoiesis in refractory severe aplastic anemia that can be sustained on discontinuation of drug. Blood. 2014; 123: 1818-1825.
- 20Broudy VC, Lin NL. AMG531 stimulates megakaryopoiesis in vitro by binding to Mpl. Cytokine. 2004; 25: 52-60.
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