aplastic anemia
Conditions
Interventions
Study Group: Receive high-dose Ropylstim N01 monotherapy (20 µg/kg, subcutaneously once weekly).
Concurrent Control Group 1 : Receive high-dose Ropylstim N01 monotherapy (20 µg/kg, subcutaneously once weekly)
Concurrent Control Group 2:Receive traditional immunosuppressive therapy (e.g., ATG combined with CsA)
Sponsors
The First Affiliated Hospital of Zhejiang Chinese Medical University
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1.Age >=18 years, any gender; 2. Diagnosed with acquired AA according to the Chinese Guidelines for the Diagnosis and Treatment of Aplastic Anemia (2022 edition) based on blood cell count and bone marrow examination; 3. At least documented failure to respond to one immunosuppressive therapy (including ATG, CsA, etc.); or unable to use or intolerant to immunosuppressive therapy due to age, comorbidities, etc.; 4. No prior treatment with Romiplostim; patients who have failed other TPO-RAs (such as rhTPO, Eltrombopag, Hetrombopag) may be included, but must undergo a 30-day washout period; 5. No prior hematopoietic stem cell transplantation (HSCT) and considered non-HSCT candidates; 6. Completion of whole exome sequencing and AA-specific next-generation sequencing is required (samples can be collected and sent to the study sponsor); 7. Patients voluntarily sign the informed consent to participate in this study.
Exclusion criteria
Exclusion criteria: 1. Severe liver dysfunction (ALT or AST >=3×ULN); 2. Severe renal impairment (eGFR < 30 ml/min/1.73m^2 or end-stage renal disease); 3. Heart disease, New York Heart Association (NYHA) class 3 or higher heart failure, or serious arrhythmias requiring treatment, or recent myocardial infarction within 6 months after randomization; 4. Patients with uncontrolled hypertension. According to NCI CTCAE version 5.0, controlled hypertension in this protocol is considered <= grade 1; 5. Patients with uncontrolled active infection; 6. Patients who are planning pregnancy or are pregnant; 7. Previously or currently existing active malignancy, except for localized tumors that were surgically treated with curative intent more than one year ago.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Proportion of patients achieving hematological response at Week 8; | — |
Secondary
| Measure | Time frame |
|---|---|
| Proportion achieving transfusion independence or reduced requirements within 8 weeks;Time to hematological response;Sustained response rates at 3 and 6 months; | — |
Countries
China
Contacts
Public ContactWu Dijiong
The First Affiliated Hospital of Zhejiang Chinese Medical University (Zhejiang Provincial Hospital of Chinese Medicine)
Outcome results
None listed