Efficacy of cyclosporine therapy in the treatment of non-severe aplastic anemia

Not Applicable

• Conditions: aplastic anemia

• Registration Number: JPRN-UMIN000030453
• Lead Sponsor: Cooperative study between the West Japan Hematology Study Group and Clinical Research Support Center Kyushu

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2017-12-18
• Study Completion: 2021-06-30
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: Complete: follow-up continuing
• Sex: All
• Target Recruitment: 34

Inclusion Criteria

Not provided

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.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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.