Severe Combined Immunodeficiency Disease Clinical Trial
Official title:
Multi-center Clinical Study of Cord Blood Stem Cell Transplantation for Severe Combined Immunodeficiency Disease
Severe combined immunodeficiency (SCID) is a rare disease caused by a group of genetic disorders that leads to early death from recurrent infections in affected children.The only curative therapy for SCID is allogeneic hematopoietic stem cell transplantation.Unrelated umbilical cord blood(UCB) is increasingly used as an alternative to bone marrow.
Severe combined immunodeficiency (SCID) is a rare disease caused by a group of genetic
disorders that leads to early death from recurrent infections in affected children.The only
curative therapy for SCID is allogeneic hematopoietic stem cell transplantation.Unrelated
umbilical cord blood(UCB) is increasingly used as an alternative to bone marrow.The
development of NBS in Europe and America has enabled more SCID patients to be diagnosed
before symptoms appear, and to enter the normative assessment and intervention procedures as
soon as possible. In China SCID has not been included in the NBS program,and there are few
units that can diagnose SCID.Patients often delay diagnosis and miss the best period of
transplantation. Department of Hematology, Children's Hospital of Fudan University, has used
UCBT for the treatment of SCID patients with reduced intensity conditioning(RIC)since 2014.6
of 8 cases were treated with disease-free survival. The encouraging efficacy of these
patients suggests that RIC of UCBT may be an effective treatment for SCID patients to
urgently hematopoietic stem cell transplantation. The aim of this study is to investigate the
efficacy of UCBT in the treatment of SCID, including engraftment rate, disease-free survival
rate,overall survival rate and immune reconstitution, and to evaluate transplant-related
mortality and complications. All the selected cases are diagnosed as severe combined
immunodeficiency disease by immunological function and gene detection. These patients have no
matched sibling donors. Their organs function should be normal. The guardian of the patient
has the desire and requirement for UCBT and signs the informed consent before treatment. Cord
blood stem cell selection:HLA high-resolution detection of patients before transplantation,
searching through cord blood stem cell bank, selecting cord blood stem cells that meet the
following criteria: HLA-A, B, C, DRB1 high-resolution (genotype) > 6/8matching,total number
of nuclear cells >5x10^7/kg.Conditioning:busulfan+ cyclophosphamide.GVHD prevention:
tacrolimus (FK506) or cyclosporine A. Infection prevention: Micafungin/caspofungin before
engraftment, voriconazole after engraftment to prevent fungi. Ganciclovir is used from the
beginning of conditioning to the infusion of cord blood stem cells, and acyclovir is used to
prevent virus infection. SMZ prevents Pneumocystis carinii infection after engraftment until
half a year after the withdrawal of immunosuppressive agents.
This is a multicenter prospective observational study. Procedure/Surgery: Cord Blood Stem
Cell Transplantation Unrelated cord blood stem cell selection: HLA high-resolution detection
is performed before transplantation. High-resolution (genotype) matching of HLA-A, B, C and
DRB1 was selected. The total number of nuclear cells is more than 5*107/kg.
Reduced intensity conditioning : Busulphan 3.2mg/kg per day in divided doses for 3 days(total
dose 9.6 mg/kg) and cyclophosphamide 50 mg/kg for 2 days (total dose 100 mg/kg).
GVHD prevention: tacrolimus (FK506) 0.1 mg/kg/day, started 4 days before transplantation, is
taken orally twice, and the blood concentration is monitored to maintain the concentration at
5-10 ng/ml. If the patient does not have a GVHD transplant +100 days after the slow
reduction, until the transplant 6 months after the withdrawal.
Infection prevention: Micafungin/caspofungin before engraftment, voriconazole after
engraftment to prevent fungi. Ganciclovir is used from the beginning of pretreatment to the
beginning of reinfusion, and acyclovir is used to prevent virus infection until
immunosuppressive agents are discontinued after reinfusion. SMZ prevents Pneumocystis carinii
infection after engraftment.
;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Terminated |
NCT00579137 -
Allogeneic SCT Of Pts With SCID And Other Primary Immunodeficiency Disorders
|
Phase 1/Phase 2 |