This study was a single-arm, multicenter, phase Π clinical study. Patients admitted to the enrollment unit center with a confirmed diagnosis of TDNSAA/VSAA/SAA, treated with IST (p/r-ATG+CSA) in combination with TPO-RA (including eltrombopta or hydtrombopta) for at least 3 months with no hematologic response at 6-month follow-up, and who were not suitable or unwilling to undergo hematopoietic stem cell transplantation (HSCT), were to another novel TPO-RA avatrombopta, 40-60 mg (weight \<80 kg), in addition to maintaining the original immunosuppressive therapy ( CSA or equivalent immune potency drugs), switch to another new TPO-RA avatropa 40-60 mg (40 mg daily for weight \<80 kg; 60 mg daily for weight \>80 kg) orally once daily for at least 3 months and follow up for 3 months to determine the hematologic response and to assess the safety of the drug
This study was a single-arm, multicenter, phase Π clinical study. Patients admitted to the enrollment unit center with a confirmed diagnosis of TDNSAA/VSAA/SAA, treated with IST (p/r-ATG+CSA) in combination with TPO-RA (including eltrombopta or hydtrombopta) for at least 3 months with no hematologic response at 6-month follow-up, and who were not suitable or unwilling to undergo hematopoietic stem cell transplantation (HSCT), were to another novel TPO-RA avatrombopta, 40-60 mg (weight \<80 kg), in addition to maintaining the original immunosuppressive therapy ( CSA or equivalent immune potency drugs), switch to another new TPO-RA avatropa 40-60 mg (40 mg daily for weight \<80 kg; 60 mg daily for weight \>80 kg) orally once daily for at least 3 months and follow up for 3 months to determine the hematologic response and to assess the safety of the drug.Selection of study population Severe aplastic anemia patients with poor efficacy of IST combined with TPO-RA Patients should be judged for inclusion and exclusion criteria. Number of subjects: 35 effective cases, 39 patients should be included according to the dropout rate of 10%.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
39
Avatrombopag, 40-60 mg (body weight \< 80 kg, 40 mg per day; body weight \> 80 kg, 60 mg per day) orally once daily for at least 3 months and followed up for 3 months to determine hematological response and evaluate the drug security.
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, China
RECRUITINGThe rate of HR in patients after switching to avatrombopag.
Percentage of the total number of patients receiving treatment who received APAG
Time frame: 3 months
Incidence of Treatment-Emergent Adverse Events as assessed by information on Common Toxicity Criteria (CTC) AE grading
Incidence of Treatment-Emergent AE by CTCAE
Time frame: 3 months
Percentage of patients with transformation
Rate of patients with transformation to PNH or MDS,AML, or other disease
Time frame: 3 months
The rate of HR in patients after switching to avatrombopag.
Percentage of the total number of patients receiving treatment who received APAG
Time frame: 6months
ncidence of Treatment-Emergent Adverse Events as assessed by information on Common Toxicity Criteria (CTC) AE grading
Incidence of Treatment-Emergent AE by CTCAE
Time frame: 6months
Percentage of patients with transformation
Rate of patients with transformation to PNH or MDS,AML, or other disease
Time frame: 6months
The rate of HR in patients after switching to avatrombopag.
Percentage of the total number of patients receiving treatment who received APAG
Time frame: 12months
ncidence of Treatment-Emergent Adverse Events as assessed by information on Common Toxicity Criteria (CTC) AE grading
Incidence of Treatment-Emergent AE by CTCAE
Time frame: 12months
Percentage of patients with transformation
Rate of patients with transformation to PNH or MDS,AML, or other disease
Time frame: 12months
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