CsA+EPAG/HPAG+Romiplostim N01 in Newly-diagnosed SAA/TD-NSAA
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Cyclosporine (CsA), thrombopoietin receptor agonist (TPO-RA), Romiplostim N01.
- Who it may be relevant to
- Registry conditions: Severe Aplastic Anemia (SAA), Transfusion-dependent Non-severe Aplastic Anemia. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Center list to be confirmed — check the primary protocol.
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
Cyclosporine + Eltrombopag/Hetrombopag + Romiplostim N01 in the Treatment of Newly-diagnosed Transfusion-dependent Non-severe Aplastic Anemia/ Severe Aplastic Anemia
Overview
This study aimed to explore the efficacy and safety of cyclosporine (CsA) +eltrombopag (EPAG)/hetrombopag (HPAG)+romiplostim N01 in the treatment of newly-diagnosed transfusion-dependent aplastic anemia (TD-NSAA) and severe aplastic anemia (SAA)
Interventions
- Drug Cyclosporine (CsA)
CsA 3-5mg/kg/d, trough concentration 100-200ng/ml - Drug thrombopoietin receptor agonist (TPO-RA)
Eltrombopag 50mg/d, increased by 25mg every two weeks Hetrombopag 7.5mg/d, increased by 2.5mg every two weeks - Drug Romiplostim N01
Romiplostim N01 20µg/kg, subcutaneously, once a week
Primary outcome measures
- Overall response rate (ORR) [Time frame: 6-month]
Secondary outcome measures (3)
- ORR [Time frame: 3-month, 12-month]
- red blood cell (RBC)/platelet (PLT) transfusion independent rate [Time frame: 3-month, 6-month, 12-month]
- AE rate [Time frame: through study completion, an average of 1 year]
Eligibility criteria
Inclusion criteria
- Age ≥ 18 years old;
- Diagnosed with aplastic anemia (AA) through routine blood tests, bone marrow puncture, bone marrow biopsy, and exclusion tests, and determined as transfusion-dependent non-severe aplastic anemia (TD-NSAA) or severe aplastic anemia (SAA) according to the Camitta criteria; Platelet < 30×10\^9/L;
- Had no HLA-matched donors or was not suitable for first-line allogeneic hematopoietic stem cell transplantation (HSCT);
- Not suitable for ATG, due to reasons such as age, complications, and the patient's own wishes;
- With baseline liver and kidney functions <2 ULN;
- ECOG score ≤ 2;
- Signed the informed consent;
Exclusion criteria
- Had other primary or secondary bone marrow failure (BMF) diseases, such as Fanconi anemia, congenital keratinization disorder, etc.;
- With evidence of clonal hematological bone marrow diseases (MDS, AML) in cytogenetics;
- PNH clone ≥ 50%;
- Received HSCT before enrollment;
- Previously used immunosuppressive treatments such as ATG, CsA, TPO receptor agonists (TPO-RAs);
- Allergic or intolerant to romiplostim N01, eltrombopag, hetrombopag, or CsA;
- Pregnant or lactating patients;
- Severe bleeding or infection that cannot be controlled by standard treatment;
- History of arterial or venous thrombosis;
- Complicated with malignant tumors;
- Participated in other clinical trials within 3 months;
- Patients considered not suitable to participate in this study by the investigator.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
Center list to be confirmed — check the primary protocol.
Identifiers
NCT: NCT07558434 · K10230