Congenital athymia(Complete DiGeorge anomaly)
Conditions
Interventions
Experimental group:Transplanting cultured thymus tissue "chips" into the quadriceps muscle of a patient with congenital athymia
Sponsors
Children's Hospital of Chongqing Medical University
Eligibility
Sex/Gender
All
Age
No minimum to 2 Years
Inclusion criteria
Inclusion criteria: Donors: 1). Age 0-12 months (including boundary values), gender is not limited; 2). Surgery for congenital heart disease; 3). The percentage of total T cells, CD4+ T cells, CD8+ T cells, naïve CD4+ T cells, naïve CD8+ T cells, B cells, and NK cells in the peripheral blood is within the normal range; 4). The guardian fully understands the protocol and agrees to sign the approved informed consent form; Receptor: 1) For a diagnosis of typical complete DiGeorge syndrome, the patient has one of the following characteristics: Heart defects,poparathyroidism,22q11 hemizygote,10p13 hemizygote,Coloboma, heart defect, choanal atresia, growth and development retardation, genital hypoplasia, ear anomalies/ deafness CHARGE association mutation (CHD7 deletion),PHA proliferative responses less than 20-fold above background; 2) The patient's peripheral immunological indicators meet one of the following two conditions: The number of CD3+T cells in PBMCs is less than 50/mm3, or PHA proliferative responses < 20-fold over background; If the number of CD3+T cells is greater than 50/mm3, then the number of Naïve T cells is less than 50/mm3, or the TREC by PCR has to be less than 100 per 100000 CD3+T; 3) The guardian fully understands the plan and agrees to sign the approved informed consent form.
Exclusion criteria
Exclusion criteria: Donors: 1) The donor and his/her mother have any of the following genetic diseases: Severe Combined Immunodeficiency Disease (SCID) ; X-linked agammaglobulinemia; trisomy 13 (Patau syndrome); trisomy 18 (Edwards' syndrome); trisomy 21 (Down syndrome); 5p syndrome (meow syndrome); 2) The donor and his or her mother have any of the following infectious diseases: Human immunodeficiency virus (HIV); Treponec pallidum (Syphilis); cytomegalovirus (CMV); Epstein-Barr virus (EBV); Toxoplasmosis; human T-lymphotropic virus 1/2 (HTLV1/2); Hepatitis A (HAV); hepatitis B (HBV); hepatitis C (HCV); Adenovirus; respiratory syncytial virus (RSV); Human Herpesvirus Type 6 A/B (HHV6) 3) The donor and his/her parents have any of the following autoimmune diseases and infectious diseases (past medical history): Type I diabetes mellitus; thyroid disease; common variant immunodeficiency disorders; systemic lupus erythematosus; Crohn's disease; ulcerative colitis; juvenile idiopathic arthritis or rheumatoid arthritis; Kujazkob disease (mad cow disease) 4) There are any other factors that the investigator considers unsuitable for inclusion in this study or completion of the study. Receptor: 1) Subjects who underwent cardiac surgery within 4 weeks prior to transplantation; 2) Cardiac surgery anticipated within 3 months of the proposed time of transplantation; 3) Lack of sufficient muscle tissue to accept thymus tissue "chip" transplantation, or the recipient's body weight is less than 3.5kg; 4) Human immunodeficiency virus (HIV) infection; 5) Suffering from severe kidney disease; 6) Cytomegalovirus (CMV) infection with clear organ damage; 7) EB virus (EBV) infection with clear organ damage; 8) Ventilator dependence: Subjects must discontinue the ventilator or other pressure support before enrollment; 9) Prior to enrollment, the subjects had attempted immune reconstitution procedures such as bone marrow transplantation or thymus transplantation; 10) There are any other factors that researchers consider unsuitable for inclusion or completion of this study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Survival at 1 year post-thymus tissue "chip" transplantation; | — |
Secondary
| Measure | Time frame |
|---|---|
| Survival at 2 years post-thymus tissue "chip" transplantation;The peripheral immune reconstitution efficacy in patients at 1 year post-thymus tissue "chip" transplantation;Assessment of the grade 4 or higher adverse events directly related to transplantation therapy at 1 year post-thymus tissue "chip" transplantation.; | — |
Countries
China
Contacts
Public ContactXiaodong Zhao
Children's Hospital of Chongqing Medical University
Outcome results
None listed