Avatrombopag Maleate

To evaluate the safety and efficacy of avatrombopag in the treatment of pediatric primary immune thrombocytopenia in patients who have been treated with eltrombopag before and switched to avatrobopag because of poor efficacy, excessive platelet fluctuation or intolerance, or patient preference, economic reasons, and other reasons.

Title: Efficacy and safety of Avatrombopag VS Avatrombopag combined with rhTPO in patients with severe aplastic anemia: a single-center, controlled study Observation group: Patients with severe aplastic anemia who did not respond to initial treatment (unconditional HSCT or ATG) or other treatments (except HSCT) Objective: To evaluate the efficacy and safety of Avatrombopag and rhTPO in the treatment of patients with severe aplastic anemia, to provide more treatment options for patients with severe aplastic anemia who are unable to undergo transplantation /ATG or have failed previous treatment, and to provide evidence-based evidence for the use of Avatrombopag or combined with rhTPO to promote hematopoietic recovery Experimental design: Single center, controlled study Total number of cases: 30 cases/group, 2 groups
Case selection criteria:
Inclusion criteria:
Age: > 18 years old, gender is not limited;
Patients clinically diagnosed with severe aplastic anemia (diagnostic criteria: ① myelocyte hyperplasia < 25% of normal; If ≥ 25% of normal but < 50%, the remaining hematopoietic cells should be < 30%. ② Blood routine must have two of the following three items: ANC < 0.5×109 /L; The absolute value of reticulocyte was < 20×109 /L; PLT < 20×109 /L). ③ If ANC < 0.2×109 /L, the diagnosis is very severe aplastic anemia), including patients who are newly diagnosed or have failed other treatments;
Patients currently undergoing hematopoietic stem cell transplantation or ATG without conditions;
Eastern Cancer Collaboration Group (ECOG) score 0-2;
Informed consent must be signed before participating in the study.
Exclusion criteria:
One of the following circumstances is not eligible for inclusion:
Patients with severe bleeding and/or infection that cannot be controlled after standard treatment;
Diagnosis of congenital hematopoietic failure (such as Fanconi anemia, congenital dyskeratosis, etc.);
Other causes of pancytopenia and bone marrow hypoproliferative diseases (such as hemolytic PNH, hypoproliferative MDS/AML, autoantibody-mediated pancytopenia, etc.);
All laboratory or clinically confirmed HIV infection, hepatitis C infection, chronic hepatitis B infection, or evidence of active hepatitis during screening;
Cytogenetic evidence of bone marrow abnormalities in clonal blood diseases;
History of thromboembolism or current use of anticoagulants within the past 6 months;
Accompanied by any one or more malignant diseases;
Treatment with another investigational agent within 30 days prior to the first dose of Avatrombopag;
Patients who cannot understand or are unwilling to sign an informed consent form (ICF);
Pregnant or lactating women;
The female patient or the female spouse of the male patient is unable to take effective contraceptive measures;
The Investigator considers that there are any other circumstances that may cause the subject to be unable to complete the study or that pose a significant risk to the subject.
Exit criteria:
The subject or his legal guardian voluntarily requests withdrawal;
Violation of inclusion/exclusion criteria;
Poor medication compliance;
The subject's condition requires treatment with drugs prohibited by the study;
Adverse events occur that cause subjects to be unable to continue the study;
Other unexplained severe comorbidities;
Pregnancy occurs during treatment;
Subjects deemed unsuitable for further study by the investigator.
Test termination:
For safety reasons, the study sponsor proposes to stop the study;
The Ethics committee decides to stop the study;
The lead researcher decides to stop the study. Investigational drug: Avatrombopag: tablet, specification: 20 mg/ tablet. rhTPO: 15000 units /1 ml. Treatment plan: This clinical trial is planned to carry out a 3-month drug study.
A: The Avatrombopag group was given Avatrombopag: 40 mg/ time, once a day, orally, for 3 months.
B: Avatrombopag +rhTPO group, Avatrombopag: 40 mg/ time, once a day, orally; rhTPO: 15000U/ time, once a day, subcutaneous injection; Both were 3 months.
Efficacy index:
Main therapeutic indicators:
Overall response rate at 3 months (OR);
Secondary efficacy measures:
Complete response rate at 3 months (CR); The time of the first occurrence of PR and CR within 3 months of medication; The proportion of subjects who were off platelet transfusion at 3 months; Hemorrhage score records of patients within 3 months of medication; Health-related quality of life score (SF-36 scale).
Efficacy criteria:
Complete response (CR) : HGB > 100 g/L; ANC > 1.5 × 109 /L; PLT > 100×109/L; Partial response (PR) : disengagement from component blood transfusion and no longer meeting the diagnostic criteria for SAA; Invalid (NR) : SAA diagnostic criteria are still met.