aplastic anemia
Conditions
Interventions
1. The administration of rATG
2.5 mg/kg, iv. daily, day 1 - day 5 + CsA
5 mg/kg, po. bid (before breakfast and before dinner) +adrenalcorticosteroid (the dose is mentioned after) is started. CsA used is Neoral or generic drug emulsified in the same way with Neoral. A bloo
75 mg, po. daily (before sleep, requires to pass more than at least 2 hours after dinner) is started from day 6.
3. The dose of steroid is as follows:
Day 1 - day 5: methylprednisolone 2 mg/kg/day
Day
Sponsors
Ishiyama Ken
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) Aged >=18 years and <80 years (at time point of the signature of ICF). 2) Patients who understand the informed consent and can sign the informed consent form by his/her free will. 3) Patients who obey the protocol visit and other rules. 4) Good PS (0, 1 and 2) 5) Patients with non-severe or sever aplastic anemia who meet the following both A and B. A) Meet the following 2 or more and requires or estimated to require periodical transfusion a) neutrophil count < 1,000/mm3 b) platelet count < 50,000/ mm3 c) reticulocyte count < 60,000/ mm3 B) The percentage of cellular component in a bone marrow biopsy specimen is <25% and no fibrosis and no hematological malignancies. 6) Not pregnant woman 7) Agree to contraception during the study period.
Exclusion criteria
Exclusion criteria: 1) Treatment history of rATG, CsA, EPAG, anabolic steroid and allogeneic hematopoietic stem cell transplantation. 2) Fulminant form characterized by the neutrophil count of 0/mm3 at enrollment and that no response to G-CSF treatment for more than or equal to 1 week. 3) Patients with chromosomal abnormalities related to MDS that were defined by WHO 2008 diagnostic. Patients with del (13q) alone and -Y alone positive are eligible. 4) Patients with >= 10% of dysplasia in >= 1 series of category A and B defined in Classification of dysplasia edited by Working Group for preparation of morphologic diagnostic criteria of refractory anemia (myelodysplastic syndromes) 5) Congenital aplastic anemia including Fanconi anemia. 6) Patients who received chemotherapy or radiotherapy or patients who developed a cancer within 5 years of entry. 7) Patients with uncontrollable infections or diabetes mellitus. 8) HBs antigen positive or HBV DNA-positive. 9)Patients with grade 2 or more hepatic disorder, cardiac disorder, or renal dysfunction according to CTCAE v5.0 10)Any other Patients who were judged to have difficulty participating in this study by investigators.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| CR or PR achievement rate at week 12 after protocol treatment (hematological response rate by Camitta criteria) | — |
Secondary
| Measure | Time frame |
|---|---|
| 1.Type and frequency of chromosomal abnormalities detected at protocol treatment initiation 2.Type and frequency of somatic gene mutations detected at protocol treatment initiation 3.Hematological response rate at week 26 after protocol treatment 4.Correlation between hematological response rate at week 2, 4, 8, 12 and 26 after protocol treatment, and rATG blood level and the following markers: PNH-type blood cells, HLA class I allele-lacking cells, plasma thrombopoietin levels 5.Hematological response rate at week 52 after protocol treatment and treatment contents at each time point 6.Response duration and recurrence rate in patients with hematological response 7.Appearance of chromosomal abnormalities at week 26 and 52 after protocol treatment or increase in chromosomal abnormalities detected by then 8.Appearance of somatic mutated clone at week 12, 26 and 52 after protocol treatment or increase in mutated clone detected by then 9.Frequency of >= grade 3 adverse events, serious adverse events and death associated with the protocol treatment | — |
Contacts
Public ContactKen Ishiyama
Center Hospital of the National Center for Global Health and Medicine
Outcome results
None listed