Chronic granulomatous disease
Conditions
Interventions
Conditioning regimen with targeted-busulfan and fludarabin Total body irradiation (3Gy) at day -1 Stem cell transplantation at day 0
Sponsors
National Center for Child Health and Development
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) Diagnosed as chronic granulomatous disease 2) Planned to have stem cell transplantation 3) Donor for transplantation is one of the follows; a. HLA 5/6 (A, B, DR) or more matched related donor b. HLA 5/6 (A, B, DR) or more matched unrelated donor 4) Age at transplantation <25 years 5) ECOG Performance status: 0-2 6) 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 7) Obtained informed consent from a guardian of the patient
Exclusion criteria
Exclusion criteria: 1. Intracranial hemorrhage 2. Severe psychological disorders 3. Pregnant or suspected pregnancy 4. Not eligible for this study at the discretion of the investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Event free survival at day 100 | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Engraftment probability at day 30 2. Mortality at day 100 3. Incidence of infectious disease at week 8 4. Reconstruction of immune status 5. Time from transplantation to onset of acute GVHD 6. Reconstruction of neutrophil function | — |
Countries
Japan
Contacts
Public ContactMotohiro Kato
National Center for Child Health and Development Children's Cancer Center
Outcome results
None listed