Sideroblastic anemia (congenital and acquired)
Conditions
Interventions
Food supplement containing 150 mg of ALA phosphate was given per day for 24 weeks. For the evaluation of efficacy and safety, medical examinations as well as urine and blood tests will be performed pr
Sponsors
Department of Hematolgy and Rheumatology, Tohoku University Graduate School of Medicine
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Patients with sideroblastic anemia (congenital and acquired), who agreed to participate in the study.
Exclusion criteria
Exclusion criteria: 1) Thrombocytopenia (less than 100,000), neutropenia (less than 1,500) 2)Patients who do not understand the study. 3) Serious complication (renal insufficiency with Ccr < 30 ml/min, liver insufficiency with total bilirubin >2 mg/dL, hypoxia requiring home oxygen therapy)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To evaluate if ALA responds to the therapy, hemoglobin levels at 12 and 24 weeks will be compared with the data at pre-treatment. Effectiveness is defined as more than 1g/dL increase in hemoglobin level (if the case undergoes regular red blood cell transfusion, the effectiveness is defined as achieving transfusion withdrawal.). | — |
Secondary
| Measure | Time frame |
|---|---|
| Onset of adverse event is evaluated at 2 weeks, 4 weeks, and thereafter every 4 weeks up to 12 months. The grade is defined according o the Common Terminology Criteria for Adverse Events (CTCAE) v4 guideline. | — |
Countries
Japan
Contacts
Public ContactFujiwara Tohru
Tohoku University Graduate School of Medicine Department of Hematolgy and Rheumatology
Outcome results
None listed