Eltrombopag for the treatment of aplastic anemia: current perspectives
نویسندگان
چکیده
Aplastic anemia (AA) is a potential life-threatening hematopoietic stem cell (HSC) disorder resulting in cytopenia. The mainstays of treatment for AA are definitive therapy to restore HSCs and supportive measures to ameliorate cytopenia-related complications. The standard definitive therapy is HSC transplantation for young and medically fit patients with suitable donors and immunosuppressive therapy (IST) with antithymocyte globulin and cyclosporine for the remaining patients. A significant proportion of patients are refractory to IST or relapse after IST. Various strategies have been explored in these patients, including second course of antithymocyte globulin, high-dose cyclophosphamide, and alemtuzumab. Eltrombopag, a thrombopoietin mimetic, has recently emerged as an encouraging and promising agent for patients with refractory AA. It has demonstrated efficacy in restoring trilineage hematopoiesis, and this positive effect continues after discontinuation of the drug. There are ongoing clinical trials exploring the role of eltrombopag as a first-line therapy in moderate to severe AA and a combination of eltrombopag with IST in severe AA.
منابع مشابه
Nationwide survey on the use of eltrombopag in patients with severe aplastic anemia: a report on behalf of the French Reference Center for Aplastic Anemia
Few therapeutic options are available for patients with aplastic anemia who are ineligible for transplantation or refractory to immunosuppressive therapy. Eltrombopag was recently shown to produce trilineage responses in refractory patients. However, the effects of real-life use of this drug remain unknown. This retrospective study (2012-2016) was conducted by the French Reference Center for Ap...
متن کاملEltrombopag restores trilineage hematopoiesis in refractory severe aplastic anemia that can be sustained on discontinuation of drug.
About a quarter of patients with severe aplastic anemia remain pancytopenic despite immunosuppressive therapy. We have previously demonstrated that eltrombopag has efficacy in this setting with 44% (11/25) of patients having clinically significant hematologic responses. We now report safety and efficacy data on a further 18 patients and long-term follow-up on the entire cohort of 43 patients. T...
متن کاملAplastic Anemia and Eltrombopag
Aplastic anemia which was once considered as rare and invariably fatal disease. Over the years the understanding of its pathophysiology, its relationship with constitutional bone marrow failure syndrome and evolution to myelodysplastic syndrome and leukemia has improved. Evolution of standard immunotherapy and bone marrow transplantation has dramatically improved the survival of patients over t...
متن کاملThe Impact of Eltrombopag Administration on the Clinical Course of Severe Refractory Fatal Acquired Aplastic Anemia
Severe aplastic anemia (SAA) has an aggressive clinical course and represents a “difficult-to-treat” situation with current medications [1]. Eltrombopag is a c-mpl receptor agonist oral thrombopoietin-mimetic drug, mainly active in immune thrombocytopenic purpura (ITP) [2]. Single-agent oral eltrombopag produced hematological responses in 11 of 25 cases of aplastic pancytopenia, with trilineage...
متن کاملRecent advances in treatment of aplastic anemia
Recent advances in the treatment of aplastic anemia (AA) made most of patients to expect to achieve a long-term survival. Allogeneic stem cell transplantation (SCT) from HLA-matched sibling donor (MSD-SCT) is a preferred first-line treatment option for younger patients with severe or very severe AA, whereas immunosuppressive treatment (IST) is an alternative option for others. Horse anti-thymoc...
متن کامل