D83.1 C91.5
Conditions
Interventions
Group 1: Observational group: Enrolled patients will be evaluated and treated according to local institutional protocols. They will receive comparable baseline and yearly follow-up evaluations across
Sponsors
Universitätsklinikum FreiburgCentrum für Chronische Immundefizienz (CCI)
Eligibility
Sex/Gender
All
Age
1 Years to 16 Years
Inclusion criteria
Inclusion criteria: • written informed consent •T cell criteria (two of the following: CD4+ or CD8+ T cell number, proliferation, thymic function, increased gamma/sigma T cells) AND •At least one major infection criteria (viral, bacterial, opportunistic) OR •At least one major immune dysregulation criteria (granulomas, lymphoproliferative disease, unexplained interstitial lung disease, inflammatory bowel disease, autoantibody mediated disease, vasculitis) OR •At least one malignancy criteria (lymphoid malignancies and virally induced malignancies) •Age >= 1yr and <= 16yr at study inclusion
Exclusion criteria
Exclusion criteria: •No written informed consent of patient or parents in case of minors available or no assent of minor if applicable •Patients with a clinical diagnosis of SCID or Omenn syndrome within the first year of life •P-CID Patients for whom decision for SCT is taken at age <1yr •Patients with Wiskott-Aldrich syndrome and CD40 Ligand Deficiency, because disease-specific prognosis and treatment data are available •Patients undergoing gene therapy or ADA enzyme replacement will be followed using the same parameters, but will not be included in the analysis
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| For all patients: Overall survival determined after year 1 and year 5. The event analysed is death from any cause. For patients who will have a HSCT (hematopoetic stemm cell transplantation): primary endpoint is the time point when immune reconstitution is achieved | — |
Secondary
| Measure | Time frame |
|---|---|
| The time point of HSCT | — |
Countries
Austria, Canada, Czechia, France, Germany, Israel, Italy, Kuwait, Netherlands, Poland, Slovenia, Spain, Switzerland, United Kingdom, United States
Contacts
Public ContactCarsten Speckmann
Universitätsklinikum FreiburgCentrum für Chronische Immundefizienz (CCI)
Outcome results
None listed