aplastic anemia
Conditions
Interventions
1. Start CsA (Neoral or generic drugs emulsified in the same way as Neoral) at a dose of 3.5 mg/kg, bid (before breakfast and before dinner). 2. Measure the blood concentration of cyclosporine at 2 h
600 ng/mL. Continue the dose of cyclosporine for 8 weeks. 3. Continue the treatment for further 44 weeks (a total of 52 weeks) when patients meet the response criteria of HI-E or HI-P at the end of
Sponsors
Cooperative study between the West Japan Hematology Study Group and Clinical Research Support Center Kyushu
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) Aged >=16 years. 2) Good PS (0, 1 and 2) 3) Meet the diagnostic criteria of aplastic anemia of stage 1 or 2, and is planned to receive CsA as a first treatment. 4) Meet both of the platelet count < 100,000/mm3 and the reticulocyte count < 60,000/ mm3, and at least one of the followings; hemoglobin level <10.0 g/dL and the neutrophil count < 1,500/ mm3. 5) The percentage of cellular component in a bone marrow biopsy specimen is <30%. 6) No treatment history with anti-thymocyte globulin (ATG), CsA, anabolic steroid, erythropoietin preparation, eltrombopag and vitamin K. 7) Not pregnant, or agree to contraception during the study period.
Exclusion criteria
Exclusion criteria: 1) Disease duration >=5 years. Patients whose disease onset is unclear are not excluded. 2) Severe (stage 3 or 4) or moderately severe (stage 3) aplastic anemia that is defined by the severity criteria by the Ministry of Health, Welfare, and Labor of Japan. 3) Patients with chromosomal abnormalities related to MDS that were defined by WHO 2008 diagnostic criteria. 4) Patients showing dysplastic signs defined as category A morphological abnormalities (1. hypo-segmented mature neutrophils (pseudo-Pelger nuclear abnormality), 2. degranulation of neutrophils, 3. micromegakaryocytes, 4. ringed sideroblasts) in "Atlas on diagnosis accuracy division and morphological diagnosis based on morphologic dysplasia of refractory anemia (myelodysplastic syndromes) ". 5) Congenital aplastic anemia including Fanconi anemia. 6) Patients who developed a cancer or received chemotherapy or radiotherapy within 5 years of entry. 7) Patients with uncontrollable infections. 8) Patients with severe impairment of the liver, heart or kidney (eGFR < 45 mL/min/1.73m2). 9) Patients who were judged to be ineligible for the study participation by investigators.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Hematologic improvement in the erythrocyte (HI-E) and platelet (HI-P) count according to IWG response criteria 2006 at 8 weeks after CsA therapy. | — |
Secondary
| Measure | Time frame |
|---|---|
| 1.Increment of reticulocyte >=20,000/mm3 at 8 weeks after the initiation of CsA. 2.HI-E or HI-P at 4 weeks, 16 weeks and 52 weeks after the initiation of CsA. 3.Increment of reticulocyte >=20,000/mm3 at 4 weeks, 16 weeks and 52 weeks after the initiation of CsA. 4.Correlation between HI-E or HI-P at 8 weeks after the initiation of CsA and the following biomarkers; presence of increased PNH-type cells, presence of cells with HLA class I allele-lacking leukocytes, and plasma thrombopoietin. 5.Correlation between HI-E or HI-P at 8 weeks after the initiation of CsA and somatic gene mutations. 6.Time to HI-E or HI-P after the initiation of CsA. 7.Incidence of somatic mutations in granulocyts. 8.Adverse events >=grade 3 associated with CsA therapy. | — |
Countries
Japan
Contacts
Public ContactKen Ishiyama
Kanazawa University Hospital Department of Hematology
Outcome results
None listed