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

Summary

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.

Conditions

• Autoimmune Hemolytic Anemia
• Immune Thrombocytopenia
• Chronic Idiopathic Neutropenia
• Autoimmune Neutropenia
• Myelodysplastic Syndromes
• Cold Agglutinin Disease

Interventions

BIOLOGICAL

cytokine essays

evaluation of immunomodulatory cytokines by ELISA kits on peripheral blood samples

BIOLOGICAL

NGS

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

BIOLOGICAL

Fecal microbiome

evaluation of fecal microbiome on fecal samples

DRUG

Erythropoietin

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

DRUG

Luspatercept

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

DRUG

Thrombopoietin Receptor Agonist

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

DRUG

G-CSF

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

Sponsors & Collaborators

• Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

  lead OTHER

Eligibility

Min Age

18 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2019-06-01

Primary Completion

2030-09-30

Completion

2035-06-30

Countries

• Italy

Study Locations