Aplastic anemia
Conditions
Interventions
Treatment group 1:Treatment of primary disease+Luspatercept
Treatment group 2:Treatment of primary disease+Luspatercept+Luspatercept
Control group:Primary disease treatment (immunosuppressive agents, androgens, alone or in combination)
Sponsors
The First Affiliated Hospital of Zhejiang Chinese Medical University
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Inclusion 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
Exclusion criteria: 1. Severe hepatic dysfunction (ALT or AST = 3 × ULN); 2.Severe renal impairment (eGFR 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).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Hemoglobin; | — |
Secondary
| Measure | Time frame |
|---|---|
| Ferritin;Frequency of blood transfusions; | — |
Countries
China
Contacts
Public ContactDijiong Wu
The First Affiliated Hospital of Zhejiang Chinese Medical University
Outcome results
None listed