Eltrombopag for the Treatment of Thrombocytopenia Due to Low- and Intermediate Risk Myelodysplastic Syndromes
Status:
Unknown status
Trial end date:
1969-12-31
Target enrollment:
Participant gender:
Summary
Myelodysplastic syndromes (MDS) prevail in older age and are characterized by ineffective
erythropoiesis and peripheral cytopenias. Supportive therapy is the main therapeutic option
for most patients. Quality of Life (QoL) is mainly deteriorated by anemia and by the
limitations associated with thrombocytopenia, neutropenia and transfusion dependence. The
only available treatment for severe thrombocytopenia, in the presence of bleeding, is
platelet transfusion.
Eltrombopag is an orally bioavailable agonist of the thrombopoietin receptor. In adult
patients with chronic immune thrombocytopenia (ITP), Eltrombopag rapidly increases platelet
counts and significantly reduces bleeding episodes during treatment. Eltrombopag is well
tolerated. In 2007, Eltrombopag has received the Orphan Drug Designation for the treatment of
ITP (EMEA/OD/031/07), and in 2008 the Food and Drug Association approved Eltrombopag for the
treatment of ITP refractory or resistant. It has been shown that in patients affected by MDS
and by acute myeloid leukemia, Eltrombopag neither increases the proliferation, nor the
clonogenic growth capacity of bone marrow blasts. Furthermore, Eltrombopag induces an
increase in the megakaryocytic differentiation and in the formation of normal megakaryocytic
colonies. These results provide the rationale for pursuing further research on Eltrombopag
for the treatment of thrombocytopenia in case of MDS.
The study is open to adult patients with myelodysplastic syndrome (MDS) with thrombocytopenia
and low- or intermediate-1 IPSS risk (Index Prognostic Score System).
Severe thrombocytopenia associated with MDS may lead to death from hemorrhage, even in low
prognostic risk patients. The benefit of platelet transfusion is short-termed. Patients
become refractory in the long term. The availability of a treatment that induces the increase
of platelet count is extremely important, either in terms of quality of life, and in overall
survival.