STILPAD Observational Study
Brief Summary in Lay Language
Common variable immunodeficiency (CVID) is the most frequent clinically relevant immunodeficiency in humans. As patients show a lack of antibodies they frequently suffer from recurrent bacterial infections of the respiratory system. About 10-20% of the patients are additionally affected by inflammatory changes of the lung tissue, called granulomatous lymphocytic interstitial lung disease. These changes can influence the lung function, the exchange of oxygen in the lung and subsequently impact quality of life and the course of the disease. Up to now the cause and exact significance of this interstitial lung disease is poorly understood. According to the current state of knowledge, the standard immunoglobulin replacement therapy has no detectable therapeutic or preventive effect on the progression of the disease in most patients with interstitial lung involvement. Thus patients are often put on additional treatment with corticosteroids or other immunosuppressive therapies. However there is currently no evidence that an immunosuppressive therapy is effective and further clinical investigations are essential to explore the cause, impact and optimal treatment of interstitial lung disease in patients with CVID. Therefore STILPAD was conceived as an observational trial. It is planned to include a total of 200 international patients to describe the natural course of the disease with and without treatment recording the past. Additionally the natural course of the disease will be observed for further 5 years into the future. As STILPAD is a purely observational trial, there will be no influence on the treatment strategy for individual patients. To further investigate the cause of the disease and to identify markers to improve diagnostics, innovative parameters in blood and tissue samples will be analysed in addition to routine diagnostics. For this purpose samples that have been already obtained in the past or will be obtained during the next 5 years.
Brief Summary in Scientific Language
Common variable immunodeficiency (CVID) is the most frequent primary immunodeficiency disease in humans. About 10-20% of the patients are additionally affected by a granulomatous lymphocytic interstitial lung disease which leads to a significantly reduced life expectancy compared to CVID patients without. In most patients with interstitial lung disease no causative infectious agent can be isolated and probably immune dysregulation contributes to the inflammatory changes found by CT scan. The standard immunoglobulin replacement therapy has no detectable therapeutic or preventive effect. Therefore other, mainly immunosuppressive strategies have been employed to improve outcome. As the inflammatory damage is only partially reversible, early diagnosis and preventive measures is desirable. Currently there is no sufficient evidence that immunosuppressive therapy is effective and safe and current practice varies between experts. Therefore this observational trial was conceived to address the course of the disease with and without therapeutic intervention and to provide a better insight into the pathogenesis and biomarkers of interstitial lung disease in CVID by immunological and microbiological examinations. This trial serves as a basis for subsequent interventional trials in order to define optimal clinical management for CVID patients with interstitial lung disease.
Cooperation & Funding
The Medical Center - University of Freiburg - Center for Chronic Immonodeficiency (CCI) is member of the European Reference Network for Primary Immonodeficiencies, Autoinflammatory Disorders, Autoimmune Diseases, Paediatric Rheumatic Diseases (ERN RITA). In this context it cooperates with other members from different states in carrying out this study.
MEDICAL CENTER - UNIVERSITY OF FREIBURG
Center for Chronic Immunodeficiency (CCI)
at Center for Translational Cell Research (ZTZ)
Breisacher Str. 115
D-79106 Freiburg
Prof. Dr. med. Klaus Warnatz
klaus.warnatz
@uniklinik-freiburg.de
Dr. med. Sigune Goldacker
sigune.goldacker
@uniklinik-freiburg.de
Dr. rer. nat. Annette Uhlmann