Severe Immune Cytopenia Registry Www.Sic-reg.Org (sic-reg)

Medical University of Graz

Status: Completed

Conditions: Evans Syndrome; Immune Thrombocytopenia; Autoimmune Hemolytic Anemia; Primary Immunodeficiency

Treatments: Diagnostic Test: potential biomarkers

Study type: Observational

Funder types: Other

Identifiers

NCT03576742

30-155 ex 17/18

Details and patient eligibility

About

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.

Full description

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).

Enrollment: 80 patients

Sex: All

Ages: 6 months to 25 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Autoimmune hemolytic anemia (AIHA)
• Evans syndrome (ES)
• Persistent or chronic immune thrombocytopenia (ITP; >6 months after first manifestation)

Exclusion criteria

• (history of) malignancies
• (history of) hematopoietic stem cell transplantation

Trial design

80 participants in 1 patient group

patients

Description:

all who fulfil inclusion criteria and consent to participation; potential biomarkers will be documented

Treatment:

Diagnostic Test: potential biomarkers