aplastic anemia
Conditions
Interventions
1. hATG 40 mg/kg/day x 4 days + CsA 5 mg/kg/min2 (before breakfast and dinner) + corticosteroids (dosage to be discussed later). The blood concentration of CsA should be measured and the dosage adjust
if C2 is less than 600 ng/mL, the CsA dose should be increased accordingly.
2. Beginning on the first day of treatment, ROMI is injected subcutaneously once a week
the starting dose of ROMI is 10 ug/kg, and the dose is fixed for the first 4 weeks. If CR is met for 4 consecutive weeks after Week 14, the dose is maintained and changed to once every 2 weeks. If the
Sponsors
Yamazaki Hirohito
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) Aged >=18 years and <80 years (at time pointof the sig nature 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 sever aplastic anemia who meet 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 hATG, rATG, CsA, EPAG, anabolic steroid and allogeneic hematopoietic stem cell transplantation. 2) Fulminant form characterized by the neutrophil count of 0/uL 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 hepatic, cardiac, or renal dysfunction requiring treatment. 10) Patients who are judged by their physician to be unable to participate in this study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| CR or PR achievement rate at week 12 after protocol treatment (hematolog ical response rate by NIH criteria partially revised) | — |
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, 52 after protocol treatment 4.Correlation between hematological response rate at week 12, 26 and 52 after protocol treatm ent and the following markers: PNH-type blood cells, HLA class I allele-lacking cells, plasma thrombopoietin levels 5.Response duration and recurrence rate in patients with hematological response 6.Time to response in patients with hematologic 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 >= g rade 3 adverse events (only causally related), serious adverse events and death associated with the protocol treatment | — |
Contacts
Public ContactHirohito Yamazaki
Kanazawa University Hospital
Outcome results
None listed