A prospective outcome study on patients with profound combined immunodeficiency - P-CID

niversitätsklinikum FreiburgCentrum für Chronische Immundefizienz (CCI)0 个研究点目标入组 140 人开始时间: 2011年7月26日最近更新: 2024年8月19日

适应症

试验速览

暂无简介。

•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

•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

发起方

niversitätsklinikum FreiburgCentrum für Chronische Immundefizienz (CCI)