Aplastic anemia (AA)
Conditions
Interventions
Group 2: Immunosuppressive Therapy (IST)
Group 1:Eltrombopag + Immunosuppressive Therapy (IST)
Sponsors
The First Affiliated Hospital of Zhejiang Medical University
Eligibility
Sex/Gender
All
Age
16 Years to 80 Years
Inclusion criteria
Inclusion criteria: 1. Before participating in the study, a written informed consent form and informed consent (if applicable) signed by the subject, parent or guardian must be obtained. 2. Sign written informed consent and informed consent (if applicable), aged >= 16 years. 3. Diagnosed as SAA/VSAA, which is characterized by: the degree of proliferation of bone marrow cells is <25%, or 25%-50% but the residual hematopoietic cells are <30%; with pancytopenia and at least two of the following peripheral blood parameters: (1) The absolute value of neutrophils is less than 0.5x10^9/L; (2) Platelet count <20x10^9/L; (3) The absolute value of reticulocytes is less than 20 x 10^9/L, and if ANC is less than 0.2 x 10^9/L, it is considered extremely severe AA. 4. Not suitable for HSCT or HSCT is not an available treatment option (as determined by local practice or national guidelines), or the subject has refused HSCT.
Exclusion criteria
Exclusion criteria: 1. Previously received IST containing any ATG/ALG, alemtuzumab, high-dose cyclophosphamide (>=45 mg/kg/day), or previously used a thrombopoietin receptor agonist. 2. Known HIV seropositive. 3. Symptomatic paroxysmal nocturnal hemoglobinuria (PNH) and/or polymorphonuclear neutrophils (PMN) or PNH clones in RBC >50% at enrollment. 4. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >3 times the upper limit of normal (ULN). 5. Creatinine >= 2.5 times the upper limit of normal ULN. 6. Hepatitis B virus HBV DNA test positive. 7. Participate in other studies at the same time or the researchers think that they are not suitable for inclusion. 8. Any clinical history of hepatitis C infection, chronic hepatitis B infection; or any evidence of active hepatitis at the time of subject screening. Laboratory tests showed positive hepatitis C or hepatitis B (HB) serology, defined as a positive HBsAg test. In addition, if HBsAg negative but HBcAb positive (regardless of HBsAb status), HB DNA testing will be performed, and if positive, the subject will be excluded. 9. Cardiac disease (with reference to II/III/IV of the modified ROSS children's heart failure classification) should not be included in the group; arrhythmia (such as atrial fibrillation) with risk of thrombosis, pulmonary hypertension or uncontrolled hypertension (>180/100 mmHg).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Hematologic response rate; | — |
Countries
China
Contacts
Public ContactShen Jianping
The First Affiliated Hospital of Zhejiang Medical University
Outcome results
None listed