aplastic anemia
Conditions
Interventions
Group 1:Romiplostim starting dose is 10ug/kg QW, maximum dose is 20ug/kg QW, continued for 26 weeks.
Sponsors
Tianjin Medical University General Hospital
Eligibility
Sex/Gender
All
Age
18 Years to 85 Years
Inclusion criteria
Inclusion criteria: 1) Male or female aged =18 years old; 2) For diagnosis of aplastic anemia (AA) (including SAA, NSAA), the platelet count must be <30×109/L at the time of enrollment. 3) Definition of relapse: The patient’s blood cells decline again after achieving CR after previous IST+TPO-RA treatment, and the platelet level is <30×109/L 4) Definition of refractory: failure to respond to standard IST+TPO-RA treatment after 3 months. 5) Definition of intolerance: At least one of AST, ALT, GGT, and bilirubin increases more than 2-fold after the patient uses the popag TPO-RA; or the patient complains of intolerance to the popag TPO-RA; Or the doctor in charge believes that there are factors that interfere with the metabolism of popag TPO-RA and may affect the efficacy and recommend a drug change. 6) The patient's dose of cyclosporine must be stable within 4 weeks before enrollment, and no adjustment is required during treatment. 7) The patients can sign a written informed consent form and understand and follow the requirements of the research.
Exclusion criteria
Exclusion criteria: 1) Congenital aplastic anemia (e.g., Fanconi anemia, parakeratosis congenita) 2) Flow cytometry determination of paroxysmal nocturnal hemoglobinuria (PNH) granulocyte clone size >= 50% 3) Presence of chromosomal aberrations (-7/7q- detected by fluorescence in situ hybridization (FISH), or complex karyotype. 4) Past or current history of malignancy. (Note: Subjects with a history of completely resected malignancy who have been disease-free for 5 years are eligible.) 5) Patients with uncontrollable severe infections (such as severe pneumonia, sepsis). 6) Alcohol or drug abuse. 7) Patients who have been diagnosed with arterial thrombosis (such as cerebral thrombosis, transient ischemic attack or myocardial infarction), patients with a past history or complications of venous thrombosis (such as deep vein thrombosis, pulmonary embolism), or patients taking anticoagulants or antiplatelet drugs at the time of screening initiation. 8) Have a history of severe cardiovascular disease (such as grade III/IV congestive heart failure, arrhythmia or angina that increases the risk of thromboembolic events, unstable angina, coronary stent placement, angioplasty) or coronary artery bypass grafting). 9) Patients who have been diagnosed with antiphospholipid antibody syndrome or other autoimmune diseases (such as lupus erythematosus). 10) Patients who have received romiplostim treatment in the past. 11) Pregnant or lactating patients (Note: Lactating female subjects are eligible to participate if they interrupt lactation before taking the first dose of study treatment and do not breastfeed within 5 days after the end of treatment). 12) Patients with severe drug allergic reactions. 13) Patients who are judged by the principal investigator or investigators to be unfit to participate in this trial. 14) Laboratory tests of coagulation function show that prothrombin time-international normalized ratio (PT-INR) and activated partial thromboplastin time (APTT) values ??exceed 20% of the normal reference range; or there is a history of coagulation abnormalities in the past. 15) Those who have received any Chinese herbal medicine or other treatment with the purpose of increasing platelets within 1 week before signing the informed consent form. 16) The test results for hepatitis C virus antibodies and human immunodeficiency virus antibodies are positive. Patients who are positive for hepatitis B virus surface antigen and have a copy number of hepatitis B virus DNA exceeding 1000 cps/ML. 17) People with renal insufficiency: creatinine clearance <80ml/min for men and <65ml/min for women 18) There are prohibited concomitant medications.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Hematological Response; | — |
Secondary
| Measure | Time frame |
|---|---|
| Granulofilocytes;Immune cell subpopulations;Adverse events;Serious adverse events; | — |
Countries
China
Contacts
Public ContactFu Rong
Tianjin Medical University General Hospital
Outcome results
None listed