Aplastic Anemia
Conditions
Interventions
Observation group:None
Sponsors
The First Affiliated Hospital of Shandong First Medical University
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Before enrollment, patients diagnosed with aplastic anemia (AA) according to the Chinese Guidelines for the Diagnosis and Treatment of Aplastic Anemia (2022 version) based on blood cell counts, bone marrow examination, and cytogenetic tests; 2. Age >=18 years; 3. Patients who have previously received other TPO-RA treatments such as eltrombopag, hetrombopag, or avatrombopag can be enrolled after a 1-month washout period; 4. Major organ functions meet the following criteria: alanine aminotransferase (ALT) and aspartate aminotransferase (AST) = 60 mL/min, serum creatinine, total bilirubin, or alkaline phosphatase <= 1.5 x ULN; 5. Eastern Cooperative Oncology Group (ECOG) performance status score <=2; 6. Patients have the ability and willingness to sign a written informed consent form and agree to participate in this study.
Exclusion criteria
Exclusion criteria: 1. Pregnant or lactating women; 2. Bone marrow reticulin fiber grade >= 2, as defined by the grading criteria in Bone Marrow Pathology (Second Edition); 3. History of allergy to Romiplostim for injection or any of its components; 4. Any significant medical condition, including active malignancy or severe psychiatric disorder; 5. Clinically significant cardiac disease, including: New York Heart Association (NYHA) Class III or IV heart failure. Unstable angina. Myocardial infarction within 6 months prior to enrollment. Angioplasty or stenting for cardiac disease within 6 months prior to enrollment. Clinically significant arrhythmia; 6. Moribund status or concurrent hepatic, renal, cardiac, neurological, pulmonary, infectious, or metabolic diseases of such severity that the patient is unable to provide consent, comply with study procedures, tolerate the protocol treatment, or is likely to die within 30 days; 7. History of arterial or venous thrombosis within 3 months prior to enrollment; 8. Positive anti-HIV antibody test at screening; 9. Women of childbearing potential who are unwilling to employ adequate contraception during the treatment period, or patients who are pregnant or lactating; 10. Considered by the investigator to be unsuitable for enrollment based on the overall assessment.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 24-week overall hematologic response rate; | — |
Secondary
| Measure | Time frame |
|---|---|
| 12 and 24-week complete hematologic response rate;The proportion of patients achieving freedom from blood component transfusion or a reduction in transfusion frequency at Weeks 12 and 24.??;Disease progression rate (transformation to SAA/MDS/AML) at 1 and 2 years.??;AE rate and category; | — |
Countries
China
Contacts
Public ContactWang Yan
The First Affiliated Hospital of Shandong First Medical University
Outcome results
None listed