Aplastic anemia
Conditions
Interventions
Durgs Group:Elrombopag combined with Cyclosporine
Sponsors
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences
Eligibility
Sex/Gender
All
Age
18 Years to 85 Years
Inclusion criteria
Inclusion criteria: 1. Treatment-naive patients who meet the diagnostic criteria for transfusion-dependent non-severe aplastic anemia, refer to "2015, Guideline for the diagnosis and management of adult aplastic anemia"(at least two of the following(Camitta et al, 1975) HGB <100 g/L, platelet count <50 x10^9/L, neutrophil count <1.5 x 10^9/L): 1) Bone marrow aspiration and biopsy show hypocellular bone marrow; 2) The patient does not meet the diagnostic criteria for severe aplastic anemia, complete blood count shows one of the following: platelets < 20 x 10^9/L or absolute neutrophil count < 0.5 x 10^9/L or HGB <80g/L; 3) Excluding other blood system and non-blood system disorders that cause pancytopenia; 2. 18-85 years of age. 3. Subjects must complete all screening assessments listed in the trial protocol. 4. Be able to swallow or take drug orally. 5. No previous ATG treatment. 6. Those who have not been previously treated with cyclosporine, tacrolimus, androgen or steroids or have been treated for less than 3 months. 7. Informed consent form must be signed before the start of all specific study procedures. In consideration of patient's condition, if the patient's signature is not conducive to the treatment of the condition, the informed consent form shall be signed by the immediate relatives of the patient.
Exclusion criteria
Exclusion criteria: 1. Severe infectious diseases (uncured tuberculosis, pulmonary aspergillosis, viral infection, active hepatitis B/C; for positive HBsAg and HBcAg, patient is excluded if hepatitis B DNA nucleic acid test is positive, DNA negative patients can enter this clinical trial; patients with hepatitis C who have a positive hepatitis C RNA nucleic acid test are excluded). 2. HIV infection. 3. Pregnancy or lactation. 4. Known diagnosis of congenital hematopoietic failure diseases (such as Fanconi anemia) and other hematopoietic failure diseases (such as MDS, PNH, ICUS, CCUS). 5. Patients with cytogenetic clonal changes lasting 12 weeks or more (any abnormal chromosome karyotype and FISH test are excluded in this trial). 6. Uncontrolled hypertension. 7. Concurrent malignant tumor. 8. Previous history of thrombosis. 9. Obvious abnormality of cardiopulmonary function. 10. Abnormal liver and kidney function: creatinine level >= 177 µmol/l (1.5mg/dl), transaminase and bilirubin levels increased significantly (3 times or more than the upper limit of normal), and who cannot be enrolled at the discretion of clinician. 11. In moribund condition or concurrent severe liver, kidney, heart, nerve, lung, infectious or metabolic diseases, the severity of which will cause the patient to be unable to tolerate the treatment regimen, or may die within 7-10 days. 12. Potential cancer patients receiving immunosuppressive therapy. 13. Subjects who not suitable for enrollment at the discretion of investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Hematologic complete response rate at 24 weeks;Safety at 24 weeks; | — |
Secondary
| Measure | Time frame |
|---|---|
| Hematologic complete response rate at 12 weeks;overall hematologic response rate at 12 weeks;overall hematologic response rate at 24 weeks; | — |
Countries
China
Contacts
Public ContactFengkui Zhang
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences
Outcome results
None listed