Severe Immune Cytopenia Registry Www.Sic-reg.Org

Overview

Prospective registry study for children and young adults with severe immune cytopenias (persisting/chronic immune thrombocytopenia, autoimmune hemolytic anemia, and Evans syndrome) to improve the management, facilitate the differential diagnostic work-up, and document the clinical course under various treatments. Time points: at inclusion, after 6 months, after 12 months, then yearly up to 4 years after inclusion. No intervention, mere observation and documentation. Guided pre-inclusion (differential) diagnostic work-up.

The study aims to improve the management and care of patients with severe immune cytopenias, to identify underlying causes of severe immune cytopenias and to develop a strategy for early treatment stratification based on a standardized diagnostic algorithm, potentially supported by biomarker analyses and (off study) genetic analyses, where clinically indicated. Primary Goal: Rapid detection of underlying causes of severe immune cytopenias with the aid of a structured diagnostic approach and access to a clinical care network of the participating centers, allowing early treatment stratification Secondary Goals: * Collection of data about epidemiology of rare diseases * Systemic documentation of response rates to various treatments * Identification of biomarkers and modifiers of immune tolerance * Collection of data about the usage of novel/experimental therapeutic agents * Documentation of physician-reported outcome measures/performance scores * Consultation of the caring physicians through a regular SIC-Reg board There will be no additional venous punctures or investigational time points. At clinical visits around planned study time points, additional blood parameters and stool specimen will be obtained. The current clinical management follows international guidelines, which are summarized in the study documents but do not represent part of the study (no diagnostic or therapeutic investigational arm).

Conditions