A pilot study for hematopoietic stem cell transplantation with post-transplantation cyclophosphamide and anti-thymoglobuli
- Conditions
- Bone marrow failure, immunodeficiency, metabolic error
- Registration Number
- JPRN-jRCTs031180397
- Lead Sponsor
- Kato Motohiro
- Brief Summary
Hematopoietic cell transplantation with PTCy and low-dose ATG from HLA-mismatched related donors was feasible to control GVHD for non-malignant diseases in the children.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete
- Sex
- All
- Target Recruitment
- 6
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
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.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- 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, engraftment at day 30, mortality at day 100, incidence of infectious disease at week 8, time from transplantation to onset of a/cGVHD, time from transplantation to death, adverse event