The aim is rapid collection of real-life data on the epidemiology, treatment and disease course in patients with VEXAS syndrome during routine clinical practice and collect biomaterials to evaluate genotype-phenotype associations, determine optimal treatment schedule, identify diagnostic features and biomarkers
see brief summary
Study Type
OBSERVATIONAL
Enrollment
500
Universitätsklinikum Aachen
Aachen, Germany
RECRUITINGKlinikum Chemnitz gGmbH
Chemnitz, Germany
RECRUITINGCollection of epidemiological data on VEXAS
incidence, age and sex distribution in adults in Germany
Time frame: enrollment
Collection and monitoring of initial disease manifestations
* spectrum and frequency of inflammatory manifestations * Association with hematological (pre)malignancies (MDS, Myeloma, MGUS, CHIP) * Monitor cardiovascular risk/thromboembolic events
Time frame: 5 years
Documentation of the treatment approaches and therapy sequences
Documentation of the treatment approaches and therapy sequences
Time frame: 5 years
Analysis of clinical-relevant clinical endpoints
complete remission (CR), hematological and clinical remission, molecular remissions, treatment-free remission (TFR)
Time frame: 5 years
Describe disease cluster
* Phenotype cluster? * Genotype-phenotype associations? * Identification of biomarker?
Time frame: 5 years
Laboratory diagnostics
* Establish Diagnostic FACS signature * Establish biomarker for disease monitoring * Molecular characterization of the key regulatory gene UBA1, but also accompanying molecular mutations * Correlation with HLA polymorphisms * Coagulation Diagnostics
Time frame: 5 years
Correlation clinical endpoints with treatment approaches
Correlation clinical endpoints with treatment approaches
Time frame: 5 years
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Universitätsklinikum Carl Gustav Carus
Dresden, Germany
RECRUITINGUniversitätsklinikum Düsseldorf
Düsseldorf, Germany
RECRUITINGEvang. Kliniken Essen-Mitte
Essen, Germany
RECRUITINGUniversitätsklinikum Hamburg-Eppendorf
Hamburg, Germany
RECRUITINGUniversitätsklinikum Leipzig AöR
Leipzig, Germany
RECRUITINGUniversitätsmedizin Mannheim
Mannheim, Germany
RECRUITINGKlinikum rechts der Isar TUM
München, Germany
RECRUITINGKlinikum Osnabrück GmbH
Osnabrück, Germany
RECRUITING...and 3 more locations
Quality of life data (QoL), fatigue questionnaire (Facit-F)
Quality of life data (QoL), fatigue questionnaire (Facit-F)
Time frame: 5 years
Accompanying translational research
* Metabolic signature * Inflammatory signature * Microbiome * Identify disease trigger /risc factors for disease onset
Time frame: 5 years
Collection of Biospecimens
Collection of Biospecimens
Time frame: 5 years