A pilot study for hematopoietic stem cell transplantation with post-transplantation cyclophosphamide and anti-thymoglobulin (Haplo-NM)
Completed
- Conditions
- Bone marrow failure, immunodeficiency, metabolic error
Recruitment & Eligibility
- Status
- Complete
- Sex
- All
- Target Recruitment
- 9
Inclusion Criteria
- Diagnosed as bone marrow failure, immunodeficiency, or inborn errors of metabolism
- Fanconi anemia is excluded for bone marrow failure.
- No HLA 5/6 (A, B, DR) or more matched related donor
- No HLA 6/6 (A, B, DR) matched unrelated donor
- Transplantation from 2- or more-antigen mismatched related donor
- Age at transplantation <25 years
- ECOG Performance status: 0-2
- 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 - Obtained informed consent from a guardian of the patient
Exclusion Criteria
- Effusion with grade 2 or worse at CTCAE ver 4.0
- Uncontrolled infection
- Severe psychological disorders
- Pregnant or suspected pregnancy
- Not eligible for this study at the discretion of the investigator.
Study & Design
- Study Type
- Interventional
- Study Design
- single assignment
- Primary Outcome Measures
Name Time Method - Incidence of events (acute GVHD grade III or more, graft failure, death) by day 30
- Secondary Outcome Measures
Name Time Method GVHD-free survival at day 100 day 100 GVHD-free survival
mortality at day 100 day 100 mortality
time from transplantation to onset of a/cGVHD time from transplantation to onset of a/cGVHD
adverse event adverse event
incidence of infectious disease at week 8 week 8 incidence of infectious disease
time from transplantation to death time from transplantation to death
engraftment at day 30 day 30 engraftment