Diabetes Mellitus, Type 1, Immunologic Deficiency Syndromes
Conditions
Keywords
Islets of Langerhans Transplantation, Cord Blood Stem Cell Transplantation
Brief summary
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.
Detailed description
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.
Interventions
PROCEDURESequential transplantation
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.
Sponsors
Children's Hospital of Fudan University
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
No minimum to 18 Years
Healthy volunteers
No
Inclusion criteria
1.Type 1 diabetes mellitus children with genetic immunodeficiency 1. Meet the diagnostic criteria of type 1 diabetes mellitus: clinical manifestations of typical diabetes mellitus include polyphagia, polyuria, weight loss, or diabetic ketoacidosis, confirmed by blood sugar level, islet function and autoimmune antibody. 2. Existence of extrapancreatic organ damage: (1) inflammatory bowel disease, (2) impairment of renal function, (3) repeated infection of mouth, skin, anus or whole body, (4) immune hepatitis, (5) persistent chronic immune iridocyclitis, (6) immune adrenalinitis leading to adrenocortical dysfunction, (7) pituitary inflammation leading to hypophysis, (8) rheumatoid disease, (9) immune vasculitis, (10) systemic lupus erythematosus, (11) other organs besides thyroid function damage. Suffering from one or more of above diseases. Recurrence after receiving regular clinical treatment, including symptomatic treatment of organ protective drugs. 3. Gene mutation was found according to gene diagnosis: gene mutation was found by gene sequencing. Literature searches at home and abroad confirmed that the defect of the gene resulted in autoimmune or immune dysfunction, resulting in multiple organ dysfunction and poor prognosis.
Exclusion criteria
1. Mature and effective treatment methods are available. 2. HIV, HBV and HCV were positive. 3. A the active period of infection. 4. At the active stage of malignant tumors. 5. Combination of other fatal diseases. 6. Existence of mental and psychological diseases.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Concentration of serum C-peptide | from the completion of treatment to 3 months | Islet function (concentration of serum C-peptide) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Concentration of blood immunoglobulin | from the completion of treatment to 3 months | Concentration of blood immunoglobulin |
| Concentration of T lymphocyte subsets | from the completion of treatment to 3 months | Concentration of T lymphocyte subsets |
| Concentraion of interleukin-2 | from the completion of treatment to 3 months | Cytokines (concentraion of interleukin-2) |
| Body height | from the completion of treatment to 3 months | Body height |
| Body weight | from the completion of treatment to 3 months | Body weight |
| Tanner stage | from the completion of treatment to 3 months | Puberty change (Tanner stage) |
| Occurrence of graft versus host disease | from the completion of treatment to 3 months | Occurrence of graft versus host disease |
| Concentration of serum C-peptide | from the completion of treatment to 6 months | Islet function (concentration of serum C-peptide) |
| Concentration of serum insulin | from the completion of treatment to 3 months | Concentration of serum insulin |
| Fast blood glucose level | from the completion of treatment to 3 months | Fast blood glucose level |
| occurrence of infection (number of infections) | from the completion of treatment to 3 months | occurrence of infection (number of infections) |
| HbA1c level | from the completion of treatment to 3 months | HbA1c level |
Countries
China
Contacts
CONTACTLuo Feihong
CONTACTXu Zhenran
STUDY_CHAIRLuo Feihong
Children's Hospital of Fudan University
Outcome results
None listed