This study evaluates the efficacy of sequential transplantation of umbilical cord blood stem cells and islet cells in children with monogenic immunodeficiency type 1 diabetes mellitus. Umbilical cord blood stem cell transplantation will be performed first. Children with stable immune reconstruction will than receive islet cell transplantation.
Monogenic immunodeficiency type 1 diabetes mellitus (T1DM) usually onsets in early age and has a long course of treatment. Because of T cell deficiency, patients are prone to recurrent infection, hemorrhage, sepsis, colitis or complications of diabetes mellitus, which lead to early death. New clinical treatment schemes have been explored and introduced around the world. Sequential transplantation of umbilical cord blood stem cells and islet cells is the latest treatment method for these children. Early treatment of monogenic immunodeficiency T1DM children can avoid disease-related organ toxicity, infection risk associated with chronic immunosuppression, and possible prevention of autoimmune endocrine organ damage. Thus, sequential transplantation of umbilical cord blood stem cells and islet cells is the only possible cure for those patients currently.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
50
After successful matching of umbilical cord blood stem cells, patients will receive pretreatment and chemotherapy under protective isolation, followed by thawing and reinfusion of umbilical cord blood stem cells. Immunosuppressive agents will be used for GVHD prevention and anti-infection support will be provided after reinfusion. The status of umbilical cord blood stem cell implantation, immune reconstruction and therapeutic effect will be evaluated. Islet transplantation will be performed in those who meet the conditions. The long-term prognosis will be observed by long-term follow-up.
Children's Hospital of Fudan University
Shanghai, Shanghai Municipality, China
RECRUITINGConcentration of serum C-peptide
Islet function (concentration of serum C-peptide)
Time frame: from the completion of treatment to 3 months
Concentration of serum C-peptide
Islet function (concentration of serum C-peptide)
Time frame: from the completion of treatment to 3 years
Concentration of serum C-peptide
Islet function (concentration of serum C-peptide)
Time frame: from the completion of treatment to 6 months
Concentration of serum C-peptide
Islet function (concentration of serum C-peptide)
Time frame: from the completion of treatment to 9 months
Concentration of serum C-peptide
Islet function (concentration of serum C-peptide)
Time frame: from the completion of treatment to 12 months
Concentration of serum C-peptide
Islet function (concentration of serum C-peptide)
Time frame: from the completion of treatment to 15 months
Concentration of serum C-peptide
Islet function (concentration of serum C-peptide)
Time frame: from the completion of treatment to 18 months
Concentration of serum C-peptide
Islet function (concentration of serum C-peptide)
Time frame: from the completion of treatment to 21 months
Concentration of serum C-peptide
Islet function (concentration of serum C-peptide)
Time frame: from the completion of treatment to 24 months
Concentration of serum C-peptide
Islet function (concentration of serum C-peptide)
Time frame: from the completion of treatment to 27 months
Concentration of serum C-peptide
Islet function (concentration of serum C-peptide)
Time frame: from the completion of treatment to 30 months
Concentration of serum C-peptide
Islet function (concentration of serum C-peptide)
Time frame: from the completion of treatment to 33 months
Concentration of serum insulin
Concentration of serum insulin
Time frame: from the completion of treatment to 3 months
Concentration of serum insulin
Concentration of serum insulin
Time frame: from the completion of treatment to 6 months
Concentration of serum insulin
Concentration of serum insulin
Time frame: from the completion of treatment to 9 months
Concentration of serum insulin
Concentration of serum insulin
Time frame: from the completion of treatment to 12 months
Concentration of serum insulin
Concentration of serum insulin
Time frame: from the completion of treatment to 15 months
Concentration of serum insulin
Concentration of serum insulin
Time frame: from the completion of treatment to 18 months
Concentration of serum insulin
Concentration of serum insulin
Time frame: from the completion of treatment to 21 months
Concentration of serum insulin
Concentration of serum insulin
Time frame: from the completion of treatment to 24 months
Concentration of serum insulin
Concentration of serum insulin
Time frame: from the completion of treatment to 27 months
Concentration of serum insulin
Concentration of serum insulin
Time frame: from the completion of treatment to 30 months
Concentration of serum insulin
Concentration of serum insulin
Time frame: from the completion of treatment to 33 months
Concentration of serum insulin
Concentration of serum insulin
Time frame: from the completion of treatment to 36 months
Fast blood glucose level
Fast blood glucose level
Time frame: from the completion of treatment to 3 months
Fast blood glucose level
Fast blood glucose level
Time frame: from the completion of treatment to 6 months
Fast blood glucose level
Fast blood glucose level
Time frame: from the completion of treatment to 9 months
Fast blood glucose level
Fast blood glucose level
Time frame: from the completion of treatment to 12 months
Fast blood glucose level
Fast blood glucose level
Time frame: from the completion of treatment to 15 months
Fast blood glucose level
Fast blood glucose level
Time frame: from the completion of treatment to 18 months
Fast blood glucose level
Fast blood glucose level
Time frame: from the completion of treatment to 21 months
Fast blood glucose level
Fast blood glucose level
Time frame: from the completion of treatment to 24 months
Fast blood glucose level
Fast blood glucose level
Time frame: from the completion of treatment to 27 months
Fast blood glucose level
Fast blood glucose level
Time frame: from the completion of treatment to 30 months
Fast blood glucose level
Fast blood glucose level
Time frame: from the completion of treatment to 33 months
Fast blood glucose level
Fast blood glucose level
Time frame: from the completion of treatment to 36 months
HbA1c level
HbA1c level
Time frame: from the completion of treatment to 3 months
HbA1c level
HbA1c level
Time frame: from the completion of treatment to 6 months
HbA1c level
HbA1c level
Time frame: from the completion of treatment to 9 months
HbA1c level
HbA1c level
Time frame: from the completion of treatment to 12 months
HbA1c level
HbA1c level
Time frame: from the completion of treatment to 15 months
HbA1c level
HbA1c level
Time frame: from the completion of treatment to 18 months
HbA1c level
HbA1c level
Time frame: from the completion of treatment to 21 months
HbA1c level
HbA1c level
Time frame: from the completion of treatment to 24 months
HbA1c level
HbA1c level
Time frame: from the completion of treatment to 27 months
HbA1c level
HbA1c level
Time frame: from the completion of treatment to 30 months
HbA1c level
HbA1c level
Time frame: from the completion of treatment to 33 months
HbA1c level
HbA1c level
Time frame: from the completion of treatment to 36 months
occurrence of infection (number of infections)
occurrence of infection (number of infections)
Time frame: from the completion of treatment to 3 months
occurrence of infection (number of infections)
occurrence of infection (number of infections)
Time frame: from the completion of treatment to 6 months
occurrence of infection (number of infections)
occurrence of infection (number of infections)
Time frame: from the completion of treatment to 9 months
occurrence of infection (number of infections)
occurrence of infection (number of infections)
Time frame: from the completion of treatment to 12 months
occurrence of infection (number of infections)
occurrence of infection (number of infections)
Time frame: from the completion of treatment to 15 months
occurrence of infection (number of infections)
occurrence of infection (number of infections)
Time frame: from the completion of treatment to 18 months
occurrence of infection (number of infections)
occurrence of infection (number of infections)
Time frame: from the completion of treatment to 21 months
occurrence of infection (number of infections)
occurrence of infection (number of infections)
Time frame: from the completion of treatment to 24 months
occurrence of infection (number of infections)
occurrence of infection (number of infections)
Time frame: from the completion of treatment to 27 months
occurrence of infection (number of infections)
occurrence of infection (number of infections)
Time frame: from the completion of treatment to 30 months
occurrence of infection (number of infections)
occurrence of infection (number of infections)
Time frame: from the completion of treatment to 33 months
occurrence of infection (number of infections)
occurrence of infection (number of infections)
Time frame: from the completion of treatment to 36 months
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Time frame: from the completion of treatment to 3 months
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Time frame: from the completion of treatment to 6 months
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Time frame: from the completion of treatment to 9 months
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Time frame: from the completion of treatment to 12 months
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Time frame: from the completion of treatment to 15 months
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Time frame: from the completion of treatment to 18 months
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Time frame: from the completion of treatment to 21 months
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Time frame: from the completion of treatment to 24 months
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Time frame: from the completion of treatment to 27 months
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Time frame: from the completion of treatment to 30 months
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Time frame: from the completion of treatment to 33 months
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Time frame: from the completion of treatment to 36 months
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Time frame: from the completion of treatment to 3 months
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Time frame: from the completion of treatment to 6 months
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Time frame: from the completion of treatment to 9 months
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Time frame: from the completion of treatment to 12 months
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Time frame: from the completion of treatment to 15 months
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Time frame: from the completion of treatment to 18 months
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Time frame: from the completion of treatment to 21 months
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Time frame: from the completion of treatment to 24 months
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Time frame: from the completion of treatment to 27 months
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Time frame: from the completion of treatment to 30 months
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Time frame: from the completion of treatment to 33 months
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Time frame: from the completion of treatment to 36 months
Concentraion of interleukin-2
Cytokines (concentraion of interleukin-2)
Time frame: from the completion of treatment to 3 months
Concentraion of interleukin-2
Cytokines (concentraion of interleukin-2)
Time frame: from the completion of treatment to 6 months
Concentraion of interleukin-2
Cytokines (concentraion of interleukin-2)
Time frame: from the completion of treatment to 9 months
Concentraion of interleukin-2
Cytokines (concentraion of interleukin-2)
Time frame: from the completion of treatment to 12 months
Concentraion of interleukin-2
Cytokines (concentraion of interleukin-2)
Time frame: from the completion of treatment to 15 months
Concentraion of interleukin-2
Cytokines (concentraion of interleukin-2)
Time frame: from the completion of treatment to 18 months
Concentraion of interleukin-2
Cytokines (concentraion of interleukin-2)
Time frame: from the completion of treatment to 21 months
Concentraion of interleukin-2
Cytokines (concentraion of interleukin-2)
Time frame: from the completion of treatment to 24 months
Concentraion of interleukin-2
Cytokines (concentraion of interleukin-2)
Time frame: from the completion of treatment to 27 months
Concentraion of interleukin-2
Cytokines (concentraion of interleukin-2)
Time frame: from the completion of treatment to 30 months
Concentraion of interleukin-2
Cytokines (concentraion of interleukin-2)
Time frame: from the completion of treatment to 33 months
Concentraion of interleukin-2
Cytokines (concentraion of interleukin-2)
Time frame: from the completion of treatment to 36 months
Body height
Body height
Time frame: from the completion of treatment to 3 months
Body height
Body height
Time frame: from the completion of treatment to 6 months
Body height
Body height
Time frame: from the completion of treatment to 9 months
Body height
Body height
Time frame: from the completion of treatment to 12 months
Body height
Body height
Time frame: from the completion of treatment to 15 months
Body height
Body height
Time frame: from the completion of treatment to 18 months
Body height
Body height
Time frame: from the completion of treatment to 21 months
Body height
Body height
Time frame: from the completion of treatment to 24 months
Body height
Body height
Time frame: from the completion of treatment to 27 months
Body height
Body height
Time frame: from the completion of treatment to 30 months
Body height
Body height
Time frame: from the completion of treatment to 33 months
Body height
Body height
Time frame: from the completion of treatment to 36 months
Body weight
Body weight
Time frame: from the completion of treatment to 3 months
Body weight
Body weight
Time frame: from the completion of treatment to 6 months
Body weight
Body weight
Time frame: from the completion of treatment to 9 months
Body weight
Body weight
Time frame: from the completion of treatment to 12 months
Body weight
Body weight
Time frame: from the completion of treatment to 15 months
Body weight
Body weight
Time frame: from the completion of treatment to 18 months
Body weight
Body weight
Time frame: from the completion of treatment to 21 months
Body weight
Body weight
Time frame: from the completion of treatment to 24 months
Body weight
Body weight
Time frame: from the completion of treatment to 27 months
Body weight
Body weight
Time frame: from the completion of treatment to 30 months
Body weight
Body weight
Time frame: from the completion of treatment to 33 months
Body weight
Body weight
Time frame: from the completion of treatment to 36 months
Tanner stage
Puberty change (Tanner stage)
Time frame: from the completion of treatment to 3 months
Tanner stage
Puberty change (Tanner stage)
Time frame: from the completion of treatment to 6 months
Tanner stage
Puberty change (Tanner stage)
Time frame: from the completion of treatment to 9 months
Tanner stage
Puberty change (Tanner stage)
Time frame: from the completion of treatment to 12 months
Tanner stage
Puberty change (Tanner stage)
Time frame: from the completion of treatment to 15 months
Tanner stage
Puberty change (Tanner stage)
Time frame: from the completion of treatment to 18 months
Tanner stage
Puberty change (Tanner stage)
Time frame: from the completion of treatment to 21 months
Tanner stage
Puberty change (Tanner stage)
Time frame: from the completion of treatment to 24 months
Tanner stage
Puberty change (Tanner stage)
Time frame: from the completion of treatment to 27 months
Tanner stage
Puberty change (Tanner stage)
Time frame: from the completion of treatment to 30 months
Tanner stage
Puberty change (Tanner stage)
Time frame: from the completion of treatment to 33 months
Tanner stage
Puberty change (Tanner stage)
Time frame: from the completion of treatment to 36 months
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Time frame: from the completion of treatment to 3 months
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Time frame: from the completion of treatment to 6 months
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Time frame: from the completion of treatment to 9 months
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Time frame: from the completion of treatment to 12 months
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Time frame: from the completion of treatment to 15 months
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Time frame: from the completion of treatment to 18 months
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Time frame: from the completion of treatment to 21 months
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Time frame: from the completion of treatment to 24 months
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Time frame: from the completion of treatment to 27 months
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Time frame: from the completion of treatment to 30 months
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Time frame: from the completion of treatment to 33 months
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Time frame: from the completion of treatment to 36 months
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