Severe Immune Cytopenia Registry Www.Sic-reg.Org

NCT03576742 | Completed

• 1 other identifier: 30-155 ex 17/18
• Study Type: observational
• Target: 80
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

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

Keywords

pancytopenia; aplastic anemia; myelodysplastic syndrome; primary immunodeficiency; immune dysregulation; autoimmune cytopenia

Outcome Measures

Primary Outcomes (1)

• underlying disease that causes or is associated with severe immune cytopenia

  identify the underlying condition or other disease, e.g., primary immunodeficiency or bone marrow failure syndrome by diagnostic procedures according to a standardized algorithm

  0-4 years

Secondary Outcomes (5)

• Clinical course

  0-4 years

• Biomarkers - Blood

  0-4 years

• Biomarkers - Stool

  0-4 years

• Routine laboratory parameters

  0-4 years

• Number of participants with the diagnosis of severe immune cytopenia per participating centre and per year

  0-4 years

Study Arms (1)

patients

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

Diagnostic Test: potential biomarkers

Interventions

potential biomarkers DIAGNOSTIC_TEST

facs analysis, microbiome analysis

patients

Eligibility Criteria

• Age: 6 Months - 25 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

consecutive registration of pediatric, adolescent, and young adult patients with severe immune cytopenia who consent

You may qualify if:

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

You may not qualify if:

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

Sponsors & Collaborators

• Medical University of Graz: lead

Study Sites (1)

Pediatric Hematology-Oncology Outpatient Clinic

Graz, Styria, 8036, Austria

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

stool, peripheral blood mononuclear cells

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic; Anemia, Hemolytic, Autoimmune; Evans Syndrome; Primary Immunodeficiency Diseases; Pancytopenia; Anemia, Aplastic; Myelodysplastic Syndromes

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms; Anemia, Hemolytic; Anemia; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Immunologic Deficiency Syndromes; Bone Marrow Failure Disorders; Bone Marrow Diseases

Study Officials

• Seidel

  Medical University of Graz

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 4 Years
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 8, 2018
• First Posted: July 3, 2018
• Study Start: March 9, 2018
• Primary Completion: December 31, 2023
• Study Completion: December 31, 2024
• Last Updated: November 21, 2025

Record last verified: 2025-10

Locations

AU (1)