Non-malignant diseases (bone marrow failure, immunodeficiency, inborn errors of metabolism)
Conditions
Interventions
Conditioning regimen with anti-thymocyte globulin. Stem cell transplantation at day 0. Cyclophosphamide at day 3 and 4.
Sponsors
National Center for Child Health and Development
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) Diagnosed as bone marrow failure, immunodeficiency, or inborn errors of metabolism 2) Fanconi anemia is excluded for bone marrow failure. 3) No HLA 5/6 (A, B, DR) or more matched related donor 4) No HLA 6/6 (A, B, DR) matched unrelated donor 5) Transplantation from 2- or more-antigen mismatched related donor 6) Age at transplantation <25 years 7) ECOG Performance status: 0-2 8) Sufficient organ function, as follows; a. T-Bil <= 1.5 mg/dl b. Serum creatinine <= 0.8 mg/dl (age <5y), <= 1.2 mg/dl (age 5-9y), <= 1.5 mg/dl (10y or older) c. Ejection fraction 45% or better, and QTfc <0.45 sec 9) Obtained informed consent from a guardian of the patient
Exclusion criteria
Exclusion criteria: 1. Effusion with grade 2 or worse at CTCAE ver 4.0 2. Uncontrolled infection 3. Severe psychological disorders 4. Pregnant or suspected pregnancy 5. Not eligible for this study at the discretion of the investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of events (acute GVHD grade III or more, graft failure, death) by day 30 | — |
Countries
Japan
Contacts
Public ContactMotohiro Kato
National Center for Child Health and Development Children's Cancer Center
Outcome results
None listed