Fanconi anenia severe combined immunodeficiency
Conditions
Interventions
Pre-transplant conditioning chemo-radiotherapy is administered according to disease. Additional rituximab (375mg/me2) is administered in conditioning on day -1 to reduce the risk of post transplantati
Sponsors
Iguchi Akihiro
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1)Patients with Fanconi anemia or severe combined immunodeficiency who are difficult to receive allogeneic hematopoietic stem cell transplantation (HSCT) using PTCY. 2)At time of registration, patients who require allogeneic HSCT, but HLA7/8 antigen (A, B, C, DRB1) matched or 8/8 antigen matched donor is not available. Therefore, the intended donor is one of the following. a. HLA4/8 antigen (A, B, C, DRB1) matched related donor. b. HLA5/8 antigen (A, B, C, DRB1) matched related donor. c. HLA6/8 antigen (A, B, C, DRB1) matched related donor. 3)Patients under 18 years of age at the time of HSCT. 4)Patients with an ECOG performance status (PS) score of 0-2 at enrollment. 5)Patients with adequate organ function who meet the following criteria at the time of enrollment. Laboratory findings must be within 30 days prior to enrollment. a.Serum direct bilirubin 10 years old, 45% and QTc<0.45sec 6)Patients whose written consent from legal representative have been obtained to participate in this study.
Exclusion criteria
Exclusion criteria: 1) Patients who had received prior HSCT. 2) The donor is unable to use G-CSF due to allergy or other reasons. 3) Patients who have uncontrollable infection. 4) Patients with severe mental disorder. 5) Patients with pregnant or likely to be pregnant. 6) Patients who are ineligible by physician
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| graft failure at 30 days after transplantation death up to 30 days after transplantation | — |
Secondary
| Measure | Time frame |
|---|---|
| Donor-derived platelet count of 20,000/ul or higher after transplantation Acute GVHD (grade II or higher) within 100 days after transplantation Treatment-related mortality within 100 days after transplantation Transplant-related complications up to 1 year after transplantion Occurrence of infectious disease within 100 days after transplantation Reconstitution of lymphocyte function up to 1 year after transplantation Days from transplantation to onset of chronic GVHD Days from transplantation to relapse Days from transplant to death Days from transplantation to neutrophil engraftment Development of malignancy after transplantation PTLD within 100 days after transplantation Acute reactions to rituximab administration secondary graft failure CliniMACS failure Grade 4 or higher of non-hematologic adverse events | — |
Contacts
Public ContactAkihiro Iguchi
National Center for Child Health and Development
Outcome results
None listed