Hetrombopag for Low/Intermediate-1 Risk MDS With Thrombocytopenia
Status:
Not yet recruiting
Trial end date:
2023-12-01
Target enrollment:
Participant gender:
Summary
Myelodysplastic syndrome (MDS) is a kind of clonal myeloid tumor. The major manifestation is
decrease of tri-lineages of blood due to ineffective and abnormal hematopoiesis, some of
which can progress to acute myeloid leukemia. According to the international prognosis
scoring system (IPSS) of MDS, about 10% low/intermediate risk-1 MDS patients have severe
thrombocytopenia (PLT < 30 × 109/ L). These patients have both decreased platelet count and
platelet dysfunction, resulting in a high risk of bleeding. In the new prognostic score, such
as IPSS-r, the degree of thrombocytopenia is regarded as a poor prognostic factor. Platelet
transfusion is mainly used in the treatment of this kind of patients. The indications of
transfusion include bleeding events or severe platelet count reduction (< 10 × 109 / L).
However, platelet transfusion can only lead to short-term platelet elevation, while repeated
transfusion increases the possibility of infection and ineffective platelet transfusion. TPO
is a newly discovered hematopoietic promoting factor, which can specifically bind to the TPO
receptor on the cell and participate in the regulation of proliferation, differentiation,
maturation and division of megakaryocyte to form functional platelet. The efficacy and safety
of the TPO receptor agonists eltrombopag and romiplostim in the treatment of thrombocytopenia
in low/intermediate risk-1 MDS patients have been successfully confirmed in foreign studies.
Hetrombopag is a new kind of a TPO receptor agonists which is highly specific platelet
stimulating factor. At present, there is no large report on the application of Hetrombopag in
such patients. The purpose of this study is to explore the short-term and long-term
therapeutic effect and safety of Hetrombopag on low/intermediate risk-1 MDS patients.