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The goal of this clinical trial is to learn whether Luspatercept alone or in combination with Deferasirox can promote hematopoietic function in patients with transfusion-dependent non-severe aplastic anemia, as well as to assess the safety and efficacy of this treatment approach.
The main questions it aims to answer is:
whether the combination therapy of Luspatercept and Deferasirox can improve hemoglobin levels in these patients.
Participants will receive Luspatercept every 3 to 5 weeks based on hemoglobin response, undergo complete blood counts every 1 to 3 weeks, and receive other necessary evaluations as required.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment Group1 | Experimental | basic treatment regimen + Luspatercept alone |
|
| Treatment Group2 | Experimental | basic treatment regimen + Luspatercept combine with Deferasirox |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Luspatercept | Drug | The recommended starting dose of luspatercept is 1.5 mg/kg administered subcutaneously (SC) once every 3 weeks. Treatment response should be evaluated and dosage adjusted after at least 3 treatment cycles (9 weeks). |
| Measure | Description | Time Frame |
|---|---|---|
| Hemoglobin | Mild anemia: Defined as a hemoglobin level <120 g/L in adult males or <110 g/L in adult females, but >90 g/L. Moderate anemia: Hemoglobin level between 60-90 g/L. Severe anemia: Hemoglobin level between 30-60 g/L. Extremely severe anemia: Hemoglobin level <30 g/L. | Baseline (1 month before treatment), treatment phase: 12 weeks (twice weekly in weeks 1 / 2, once weekly in week 3, once every 3 weeks ), mid-treatment phase: 12 weeks (once every 3 weeks), follow-up period: 48 weeks (once every 12 weeks). |
| Measure | Description | Time Frame |
|---|---|---|
| platelets | Baseline (30 days before treatment), treatment phase: 12 weeks (twice weekly in weeks 1 / 2, once weekly in week 3, once every 3 weeks ), mid-treatment phase: 12 weeks (once every 3 weeks). | |
| Ferritin | Baseline (30 days before treatment), treatment phase: 12 weeks ( once every 3 weeks ), mid-treatment phase: 12 weeks (once every 3 weeks) |
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Inclusion Criteria:
1.Age >= 18 years; 2.According to the "Chinese Guidelines for the Diagnosis and Treatment of Aplastic Anemia (2022 Edition)", the patient must be diagnosed with transfusion-dependent non-severe aplastic anemia (TD-NSAA) and meet the requirement of erythroid hyperplasia in bone marrow aspiration from the posterior iliac crest and/or sternum being more than 15%; 3.If not newly diagnosed with TD-NSAA, and there are combined primary disease maintenance medications, the following conditions must be met:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Dijiong Wu, Ph.D. | Contact | +86 13989463963 | wudijiong@zcmu.edu.cn |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The First Affiliated Hospital of Zhejiang Chinese Medical University | Hangzhou | Zhejiang | 310000 | China |
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Control Group: Patients will continue with their original basic treatment regimen for the disease (immunosuppressants, androgens(either alone or in combination)) under observation.
Treatment Group: In addition to the original basic treatment for the disease, patients will be further divided based on whether iron chelation therapy is used, into two subgroups: Luspatercept monotherapy group and Luspatercept combined with deferasirox group.
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| Luspatercept combine with Deferasirox | Drug | The recommended starting dose of luspatercept is 1.5 mg/kg administered subcutaneously (SC) once every 3 weeks. Treatment response should be evaluated and dosage adjusted after at least 3 treatment cycles (9 weeks). For patient with platelet counts < 50×10⁹/L: Deferasirox dosage: 8-10 mg/kg/day, orally. For platelet counts ≥ 50×10⁹/L: Deferasirox dosage: 20 mg/kg/day, orally. Discontinue deferasirox if serum ferritin drops below 500 μg/L. |
|
| Proportion of patients transfusion-free for ≥8 weeks and ≥12 weeks | treatment phase: 12 weeks (twice weekly in weeks 1 / 2, once weekly in week 3, once every 3 weeks ), mid-treatment phase: 12 weeks (once every 3 weeks) |
| Overall response rate (ORR; CR + PR) at Week 12 and Week 24 of treatment | ORR: Overall Response Rate (defined as the proportion of patients achieving complete response [CR] or partial response [PR]). | Week 12 and Week 24 |
| ID | Term |
|---|---|
| C000621232 | luspatercept |
| D000077588 | Deferasirox |
| ID | Term |
|---|---|
| D001565 | Benzoates |
| D000146 | Acids, Carbocyclic |
| D002264 | Carboxylic Acids |
| D009930 | Organic Chemicals |
| D001555 | Benzene Derivatives |
| D006841 | Hydrocarbons, Aromatic |
| D006844 | Hydrocarbons, Cyclic |
| D006838 | Hydrocarbons |
| D014230 | Triazoles |
| D001393 | Azoles |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
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