aplastic anemia
Conditions
Interventions
Group 1:Avatrombopag treatment
Sponsors
Tianjin Medical University, General Hospitial
Eligibility
Sex/Gender
All
Age
16 Years to 75 Years
Inclusion criteria
Inclusion criteria: 1. Subject has given written informed consent. Subjects aged 16 years or older and less than 75 years old at the time of informed consent; 2. Diagnosis of moderate (stage II) or higher aplastic anemia (AA), platelet count <30x10^9/L; 3. Newly diagnosed and relapsed, refractory AA can be selected; 4. Screening and day 1 organ functions were defined as follows: alanine aminotransferase (ALT) and aspartate aminotransferase (AST) 1 to 5 ULN; total bilirubin and Alkaline phosphatase (ALP) 1 to 3 ULN. with or without viral hepatitis; 5. Eastern Cooperative Oncology Group (ECOG) Performance Status (PS): 0 or 1; 6. QT < 450 milliseconds (msec).
Exclusion criteria
Exclusion criteria: 1. Normal liver function; 2. Congenital aplastic anemia (eg, Fanconi anemia, parakeratosis congenita); 3. Paroxysmal nocturnal hemoglobinuria (PNH) granulocyte clone size >= 50% by flow cytometry; 4. Presence of chromosomal aberrations (-7/7q- detected by fluorescence in situ hybridization (FISH), or complex karyotype; 5. Past history of thromboembolism or current history of using anticoagulants; 6. Past or present history of malignancy. Note: Subjects with a history of completely resected malignancy and disease free for 5 years are eligible; 7. Liver failure; 8. Subjects with uncontrollable serious infections (such as sepsis) at the same time; 10. Cardiac disease (New York Heart Association (NYHA) functional class II/III/IV congestive heart disease) or arrhythmia with risk of thrombosis (eg, atrial fibrillation). Note: Subjects with NYHA class II due to cardiac disease should not be recruited, but subjects with NYHA class II due to aplastic anemia (AA); 11. Abuse of alcohol or drugs; 12. Pregnant women (serum or urine pregnancy test positive within 7 days before the first dose of study treatment drug) or lactating women. Note: Nursing female subjects are eligible to participate if they interrupt breastfeeding prior to their first dose of study treatment and do not breastfeed within 5 days of the end of treatment; 13. Past history of immediate or delayed hypersensitivity to compounds chemically similar to avatrombopag or its activator; 14. Treatment with another investigational product within 30 days prior to the first dose of avatrombopag or a time 5 times longer than the half-life of the investigational product; 15. Prior treatment with eltrombopag, romiprostim, or any other thrombopoietin (TPO) receptor agonist. Note: Patients with hepatic impairment taking Eltrombopag are eligible to participate. Recommended drug replacement regimen: 25 mg of eltrombopag replaced by 20 mg of avatrombopag, 50 mg of eltrombopag replaced by 40 mg of avatrombopag, and 75 mg of eltrombopag or higher replaced by 60 mg of avatrombopag; 16. Patients with serum sodium<=130mEq/L; 17. Patients with known bleeding disorders or platelet dysfunction; (1) Baseline prothrombin time (PT) is more than twice the upper limit of normal; (2) Activated partial thromboplastin time (aPTT) greater than 3 times the upper limit of normal; 18. Patients with known hereditary prothrombotic disease (factor V Leiden, prothrombin 20210A, antithrombin deficiency or protein C or S deficiency) or history of antiphospholipid syndrome; 19. Patients who received anticoagulation or NSAID treatment within 7 days after enrollment; 20. The patient has known portal vein thrombosis, mesenteric thrombosis or spleen thrombosis; 21. Use of prohibited concomitant drugs.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Hematological Response; | — |
Countries
China
Contacts
Public ContactFu Rong
Tianjin Medical University General Hospital
Outcome results
None listed