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

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

Recruiting
18 years and older
All
Phase N/A

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Overview

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.

Description

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.

Eligibility

Inclusion Criteria:

  • Diagnosis of autoimmune cytopenias (AIHA/ITP/CIN/AIN)
  • age >/= 18 years
  • ability to sign informed consent
  • availability to undergo 3 year follow up
  • for the subgroup of patients with myelodysplastic syndrome: bone marrow evaluation showing >/= 10% dysplastic features of at least one lineage along with MDS defining cytopenia and/or MDS defining cytogenetics.

Exclusion Criteria:

  • any condition impeding the acquisition of the informed consent
  • immune cytopenia diagnosis preceding >/= 6 months the enrolment

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

NCT05931718

Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

22 March 2024

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