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.
The patient has not received and does not consider HSCT or ATG treatment for at least the next six months;
If maintaining oral immunosuppressive therapy, the course must be at least 6 months and assessed as ineffective;
If maintaining androgen therapy, the course must be at least 3 months and assessed as ineffective;
If maintaining recombinant human erythropoietin therapy, the course must be at least 3 months and assessed as ineffective;
1. Severe hepatic dysfunction (ALT or AST ≥ 3 × ULN); 2.Severe renal impairment (eGFR < 30 ml/min/1.73m² or patients with end-stage renal disease); 3.Cardiac disease, including New York Heart Association (NYHA) Class 3 or higher heart failure, or severe arrhythmia requiring treatment, or recent myocardial infarction within 6 months of randomization; 4.Patients with uncontrolled hypertension, with controlled hypertension according to NCI CTCAE version 5.0 considered as ≤ Grade 1 for this protocol; 5.Patients with a PNH clone > 1%; 6.Patients planning to become pregnant or who are pregnant; 7.Surgical or clinical conditions that may significantly alter drug absorption, distribution, metabolism, or excretion (e.g., gastritis, ulcers, history of gastrointestinal or rectal bleeding; history of major gastrointestinal surgery); 8.Patients carrying congenital bone marrow failure-related gene mutations (homozygous or heterozygous, regardless of whether they are pathogenic/benign/likely benign/ of uncertain significance).
Parallel
Luspatercept
DrugThe 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).
Luspatercept combine with Deferasirox
DrugThe 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.
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.
platelets
Ferritin
Proportion of patients transfusion-free for ≥8 weeks and ≥12 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 \[PR\]).
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This trial is not recruiting at the moment. You can still explore other options:
