Aplastic anemia
Conditions
Interventions
None listed
Sponsors
Kanazawa University
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Participants must meet all of the following criteria: 1. Patients with aplastic anemia aged 2 years or older at the time of enrollment 2. Platelet count <100,000/uL 3. Have undergone bone marrow examination (smear, cytogenetic analysis, and biopsy) within 6 months prior to enrollment 4. Evidence of bone marrow hypoplasia, defined as bone marrow cellularity <30% on biopsy or reduced hematopoietic activity on MRI
Exclusion criteria
Exclusion criteria: Participants meeting any of the following criteria will be excluded: 1. Prior treatment with anti-thymocyte globulin (ATG) 2. Ongoing treatment for >=6 months prior to enrollment with immunosuppressive agents (e.g., cyclosporine) or thrombopoietin receptor agonists 3. History of chemotherapy or radiotherapy within the past 5 years 4. Presence of cytogenetic abnormalities specific to hematologic malignancies, including complex karyotype, -7, 7q-, or der(1;7), on bone marrow cytogenetic analysis 5. Increased blasts, defined as persistent >=1% in peripheral blood or >=5% in bone marrow 6. Normocellular to hypercellular megakaryopoiesis observed in either bone marrow smear or biopsy 7. No elevation of serum thrombopoietin levels (only in patients in whom this was measured) 8. History of cytopenia for >=2 years prior to enrollment (defined as neutrophil count <1,500/uL [or white blood cell count <3,000/uL], hemoglobin <11 g/dL, or platelet count <150,000/uL), unless attributable to a clearly benign cause such as iron deficiency anemia 9. Secondary bone marrow failure syndromes (e.g., bone marrow carcinomatosis, myelofibrosis, hemophagocytic syndrome, systemic lupus erythematosus, drug-induced agranulocytosis) 10. Inherited bone marrow failure syndromes (including suspected cases) 11. Concomitant hematologic malignancies (hypoplastic myelodysplastic syndrome is not excluded unless the above criteria are met) 12. Concomitant solid tumors or autoimmune diseases requiring systemic treatment 13. Uncontrolled infections refractory to antimicrobial therapy 14. Life expectancy of <1 year due to comorbidities other than aplastic anemia 15. Inability to attend regular follow-up visits at a hematology outpatient clinic
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Longitudinal changes in the proportions of HLA class I allele-deficient blood cells and PNH-type blood cells | — |
Secondary
| Measure | Time frame |
|---|---|
| Complete response rate at 1 year, overall response rate at 1 year, time to complete response, time to overall response, overall survival, relapse, clonal evolution to MDS/AML, and PNH development | — |
Countries
Japan
Contacts
Public ContactYoshitaka Zaimoku
Kanazawa University Hospital Innovative Clinical Research Center
Outcome results
None listed