severe aplastic anemia
Conditions
Interventions
Experimental Group:immunosuppressive therapy combined with Romiplostim
Sponsors
Jiangsu Province Hospital (The First Affiliated Hospital with Nanjing Medical University)
Eligibility
Sex/Gender
All
Age
15 Years to 80 Years
Inclusion criteria
Inclusion criteria: 1. Diagnosed with severe aplastic anemia (SAA) or very severe aplastic anemia (vSAA): (1) At least two of the following criteria must be met: 1) Absolute neutrophil count < 0.5 × 10^9/L (severe) or < 0.2 × 10^9/L (very severe); 2) Platelet count < 20 × 10^9/L 3) Reticulocyte count < 60 × 10^9/L (by automated analyzer) or < 20 × 10^9/L (by manual method, now largely replaced by automated methods); (2) Accompanied by bone marrow hypocellularity (cellularity < 30%), with no evidence of fibrosis or malignant cells; 2. Male or female patients aged 15 to 80 years; 3. The written informed consent form must be signed; 4. Willing and able to comply with all protocol requirements and visit schedules; 5. Women of childbearing potential must have a negative pregnancy test result and provide written consent to use contraception throughout the study period.
Exclusion criteria
Exclusion criteria: 1. Myelofibrosis grade >= 2; 2. Previous treatment with anti-thymocyte immunoglobulin (ATG); 3. Prior allogeneic hematopoietic stem cell transplantation; 4. Discontinuation of other TPORAs for less than 5 half-lives; 5. Concurrent or history of active malignancy, except for localized tumors that have been curatively resected for more than one year; 6. Clinically significant heart disease (NYHA functional class III–IV); unstable angina; myocardial infarction within 6 months prior to enrollment; angioplasty or stent placement for heart disease within 6 months prior to enrollment; clinically significant arrhythmia; or uncontrolled hypertension; 7. History of arterial or venous thrombosis within 1 year prior to enrollment; 8. Positive test results for anti-HIV, HBV surface antigen, or HCV-RNA; 9. Thrombocytopenia due to other etiologies, such as MDS, ITP, liver cirrhosis, etc. 10. Concomitant PNH with high disease activity: LDH > 1.5 × upper limit of normal; 11. Presence of uncontrolled active infection; 12. Uncontrolled diabetes mellitus; 13. Eastern Cooperative Oncology Group (ECOG) performance status score > 2; 14. Serum aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 3 × upper limit of normal; 15. Serum creatinine >= 2.5 × upper limit of normal; 16. Pregnant or breastfeeding women; 17. Individuals considered by the study group to be unsuitable for enrollment or unable to comply with the trial for other reasons.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 3-month complete response rate; | — |
Secondary
| Measure | Time frame |
|---|---|
| Safety;3-month and 6-month good response rate;3-month and 6-month overall response rate;2-year survival rate;Time to response; | — |
Countries
China
Contacts
Public ContactYuemin Gong
Jiangsu Province Hospital (The First Affiliated Hospital with Nanjing Medical University)
Outcome results
None listed