Symptoms and General Pathology

All Conditions

Neoplasms

Blood and Lymph Conditions

Infections

Respiratory Tract (Lung and Bronchial) Diseases

Immune System Diseases

Heart and Blood Diseases

Rare Diseases

Syndrome

Preleukemia

Myelodysplastic Syndromes

Cytopenia

Anemia

Thrombocytopenia

Respiratory Tract Infections

Neutropenia

Purpura, Thrombocytopenic, Idiopathic

Hemolysis

Anemia, Hemolytic

Anemia, Hemolytic, Autoimmune

Purpura

Purpura, Thrombocytopenic

Hematologic Diseases

Bone Marrow Diseases

Precancerous Conditions

Blood Platelet Disorders

Agranulocytosis

Leukopenia

Leukocyte Disorders

Hemostatic Disorders

Blood Coagulation Disorders

Thrombotic Microangiopathies

Hemorrhagic Disorders

Autoimmune Diseases

Hemorrhage

Skin Manifestations

Chronic Graft Versus Host Disease

Granulocytopenia

Immune Thrombocytopenia

Idiopathic Thrombocytopenic Purpura

Autoimmune Hemolytic Anemia

Cold Agglutinin Disease

Antineoplastic Agents

Antirheumatic Agents

All Drugs and Chemicals

Hematinics

Coagulants

Rituximab

Luspatercept

Epoetin Alfa

Agglutinins

AIHA patients will be enrolled at diagnosis and stratified according to AIHA type (i.e. warm, cold, mixed, and atypical forms), sampled for peripheral blood for cytokine and NGS studies, and for feces for microbiome studies. If clinically indicated, bone marrow evaluation will be performed and a sample for single cell analysis will be collected. Patients will be followed up to collect treatments, responses, relapses, and complications (particularly thromboses and infections).

ITP patients will be enrolled at diagnosis and sampled for peripheral blood for cytokine and NGS studies, and for feces for microbiome studies. If clinically indicated, bone marrow evaluation will be performed and a sample for single cell analysis will be collected.Patients will be followed up to collect treatments, responses, relapses, and complications (particularly thromboses and infections).

CIN/AIN patients will be enrolled at diagnosis and sampled for peripheral blood for cytokine and NGS studies, and for feces for microbiome studies. If clinically indicated, bone marrow evaluation will be performed and a sample for single cell analysis will be collected.Patients will be followed up to collect treatments, responses, relapses, and complications (particularly infections).

MDS patients will be enrolled at diagnosis and sampled for peripheral blood for cytokine and NGS studies, and to evaluate red cell metabolism. If clinically indicated, bone marrow evaluation will be performed and a sample for single cell analysis will be collected. Patients will be followed up to collect treatments, responses, relapses, and outcome.

evaluation of immunomodulatory cytokines by ELISA kits on peripheral blood samples

cytokine essays

evaluation of somatic mutations commonly associated with myeloid neoplasm and immunodeficiencies by next generation sequencing on peripheral blood samples

evaluation of fecal microbiome on fecal samples

Fecal microbiome

evaluation of recombinant erythropoietin use, safety and efficacy in patients with autoimmune hemolytic anemia according to clinical practice

Erythropoietin

evaluation of cytokine levels, molecular profile and bone marrow microenvironment by single cell analysis in patients treated with luspatercept according to clinical practice

evaluation of TPO-RA use, safety and efficacy in patients with ITP according to clinical practice

Thrombopoietin Receptor Agonist

evaluation of G-CSF use, safety and efficacy in patients with CIN/AIN according to clinical practice

G-CSF

Bruno Fattizzo, MD

The goal of this observational study is to characterize the diagnostic and therapeutic management of autoimmune cytopenias including autoimmune hemolytic anemia, immune thrombocytopenia, and chronic idiopathic/autoimmune neutropenia.

The main aims to answer are:

* evaluation of traditional and novel diagnostic tools including immunohematology, cytokine essays, bone marrow studies, molecular findings, and fecal microbiome.
* evaluation of type and sequence of the therapies administered, the response rates, and the adverse events.
* evaluation of clinical and laboratory (immunologic, molecular, and morphologic) predictors of outcome.
* evolution of autoimmune cytopenias into myelodysplastic syndromes.
* a subgroup of patients with myelodysplastic syndromes will be included to evaluate the presence of immunologic events, autoimmune activation, and red cell metabolism.

Participants will receive a clinical/laboratory diagnostic workup as per current clinical practice. Furthermore They will be sampled at baseline (peripheral blood and feces for microbiome) and followed up for at least 3 years to evaluate their clinical course, therapeutic management and outcome.

This observational study will characterize the diagnostic and therapeutic management of autoimmune cytopenias including autoimmune hemolytic anemia, immune thrombocytopenia, and chronic idiopathic/autoimmune neutropenia to evaluate predictors of outcome. Additionally, a subgroup of patients with myelodysplastic syndromes (diagnosed according to current WHO 5th edition 2022) will be included to evaluate the presence of autoimmune activation, and red cell metabolism.

peripheral blood for cytokines and next generation sequencing, bone marrow sample for single cell analysis, fecal sample for microbiome

Prospective Evaluation of Diagnosis and Treatment of Patients With Autoimmune Cytopenias Including Autoimmune Hemolytic Anemia, Immune Thrombocytopenia, and Chronic Idiopathic/Autoimmune Neutropenia

Evaluation of the Diagnostic/Therapeutic Course of Patients With Autoimmune Cytopenias (Autoimmune Hemolytic Anemia AIHA, Immune Thrombocytopenia ITP, Chronic Idiopathic/Autoimmune Neutropenia CIN/AIN) and Identification of Predictive and Prognostic Markers.

to define the sensitivity of autoantibody testing in autoimmune cytopenias

to define the specificity of autoantibody testing in autoimmune cytopenias

to define the sensitivity of bone marrow trephine in autoimmune cytopenias

to define response rates to treatment of autoimmune cytopenias and myelodysplastic syndromes

to define somatic mutations in autoimmune cytopenias and myelodysplastic syndromes by NGS

to define pyruvate kinase activity in myelodysplastic syndromes

to define microbiome composition in autoimmune cytopenias and myelodysplastic syndromes

to define bone marrow composition by single cell analysis in autoimmune cytopenias and myelodysplastic syndromes

to define cytokine levels in autoimmune cytopenias and myelodysplastic syndromes by ELISA

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
Prospective Evaluation of Diagnosis and Treatment of Patients With Autoimmune Cytopenias Including Autoimmune Hemolytic Anemia, Immune Thrombocytopenia, and Chronic Idiopathic/Autoimmune Neutropenia	NCT05931718	Autoimmune Hemo... Immune Thromboc... Chronic Idiopat... Autoimmune Neut... Myelodysplastic... Cold Agglutinin...	cytokine essays NGS Fecal microbiom... Erythropoietin Luspatercept Thrombopoietin ... G-CSF	18 Years -	Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Prospective Evaluation of Diagnosis and Treatment of Patients With Autoimmune Cytopenias Including Autoimmune Hemolytic Anemia, Immune Thrombocytopenia, and Chronic Idiopathic/Autoimmune Neutropenia	NCT05931718	Autoimmune Hemo... Immune Thromboc... Chronic Idiopat... Autoimmune Neut... Myelodysplastic... Cold Agglutinin...	cytokine essays NGS Fecal microbiom... Erythropoietin Luspatercept Thrombopoietin ... G-CSF	18 Years -	Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

Spots Global Cancer Trial Database for cold agglutinin disease