Clinical trial NCT03576742: Severe Immune Cytopenia Registry Www.Sic-reg.Org
Official title: Register für Schwere Immunzytopenien - Severe Immune Cytopenia Registry (SIC-Reg.Org)
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Immune Thrombocytopenia
Autoimmune Hemolytic Anemia
Evans Syndrome
Primary Immunodeficiency
Keywords: pancytopenia
aplastic anemia
myelodysplastic syndrome
primary immunodeficiency
immune dysregulation
autoimmune cytopenia
Lead sponsor: Medical University of Graz
Sponsor class: OTHER
First posted: 2018-07-03
Enrollment: 80
Sex: ALL
Minimum age (years): 6
Maximum age (years): 25
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: consecutive registration of pediatric, adolescent, and young adult patients with severe immune cytopenia who consent

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.

Detailed 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).

Eligibility criteria: 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

Primary outcomes:
measure: underlying disease that causes or is associated with severe immune cytopenia
timeFrame: 0-4 years
Secondary outcomes:
measure: Clinical course
timeFrame: 0-4 years
measure: Biomarkers - Blood
timeFrame: 0-4 years
measure: Biomarkers - Stool
timeFrame: 0-4 years
measure: Routine laboratory parameters
timeFrame: 0-4 years
measure: Number of participants with the diagnosis of severe immune cytopenia per participating centre and per year
timeFrame: 0-4 years
Other outcomes:
Locations:
city: Graz
country: Austria
facility: Pediatric Hematology-Oncology Outpatient Clinic
state: Styria
Study officials:
affiliation: Medical University of Graz
name: Seidel
role: PRINCIPAL_INVESTIGATOR
References: