Treatment of Transfusion-dependent Nonsevere Aplastic Anemia With Luspatercept: a Multicenter Prospective Clinical Study

Launched by THE FIRST AFFILIATED HOSPITAL OF ZHEJIANG CHINESE MEDICAL UNIVERSITY · May 7, 2025

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ClinConnect Summary

This clinical trial is looking at a new treatment for patients with a condition called transfusion-dependent non-severe aplastic anemia, which means their bodies are not producing enough blood cells and they need regular blood transfusions. The study aims to find out if a medication called Luspatercept, either on its own or combined with another drug called Deferasirox, can help improve blood cell production and increase hemoglobin levels, which is important for carrying oxygen in the blood.

To participate in the trial, you must be at least 18 years old and have been diagnosed with this specific type of anemia, along with other criteria related to your health and previous treatments. If eligible, you will receive Luspatercept every 3 to 5 weeks based on your hemoglobin levels and will have regular blood tests to monitor your condition. The trial is not yet recruiting, so there may be more information available soon. This could be an opportunity to potentially benefit from a new treatment approach, but it’s important to discuss any questions or concerns with your healthcare provider.

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Eligibility criteria

• 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:
• • 1. The patient has not received and does not consider HSCT or ATG treatment for at least the next six months;
• • 2. If maintaining oral immunosuppressive therapy, the course must be at least 6 months and assessed as ineffective;
• • 3. If maintaining androgen therapy, the course must be at least 3 months and assessed as ineffective;
• • 4. If maintaining recombinant human erythropoietin therapy, the course must be at least 3 months and assessed as ineffective;
• • 5. If maintaining thrombopoietin receptor agonist (TPO-RA) therapy, the duration must be \>=6 months with confirmed inefficacy, and a washout period of \>=1 month is required before study enrollment;
• • 6. If the above maintenance medication durations are not met, a washout period of at least 1 month is required; 4.Serum ferritin level \>= 1000 ng/ml; 5.Complete whole exome sequencing and MDS/AA next-generation sequencing testing are required.
• Exclusion Criteria:
• • 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).