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

The First Affiliated Hospital of Zhejiang Chinese Medical University

Status: Not yet enrolling

Conditions: Transfusion-dependent Non-severe Aplastic Anemia

Treatments: Drug: Luspatercept; Drug: Luspatercept combine with Deferasirox

Study type: Interventional

Funder types: Other

Identifiers

NCT06964971

2025-KLS-046-02

Details and patient eligibility

About

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.

Enrollment: 90 estimated patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

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).

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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90 participants in 2 patient groups

Treatment Group1

Experimental group

Description:

basic treatment regimen + Luspatercept alone

Treatment:

Drug: Luspatercept

Treatment Group2

Experimental group

Description:

basic treatment regimen + Luspatercept combine with Deferasirox

Treatment:

Drug: Luspatercept combine with Deferasirox