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NCT Number: NCT05931718

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

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.

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Key information

Conditions

Autoimmune Hemolytic Anemia Anemia Anemia, Hemolytic Anemia, Hemolytic, Autoimmune Autoimmune Diseases Autoimmune Neutropenia Blood Coagulation Disorders Blood Platelet Disorders Bone Marrow Diseases Chronic Idiopathic Neutropenia Cold Agglutinin Disease Cytopenia Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhage Hemorrhagic Disorders Immune System Diseases Immune Thrombocytopenia Myelodysplastic Syndromes Pathologic Processes Pathological Conditions, Signs and Symptoms Purpura Purpura, Thrombocytopenic Purpura, Thrombocytopenic, Idiopathic Signs and Symptoms Skin Manifestations Thrombocytopenia Thrombotic Microangiopathies

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Fondazione Irccs Ca' Granda Ospedale Maggiore Policlinico

Milan, 20100, Italy

Location status: Recruiting

Location contact

Bruno Fattizzo, MD

CONTACT

[email protected]

+390255033477

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

cytokine essays

Biological

evaluation of immunomodulatory cytokines by ELISA kits on peripheral blood samples

NGS

Biological

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

Fecal microbiome

Biological

evaluation of fecal microbiome on fecal samples

Erythropoietin

Drug

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

Luspatercept

Drug

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

Thrombopoietin Receptor Agonist

Drug

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

G-CSF

Drug

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

Primary outcomes

  1. sensitivity of autoantibody testing in autoimmune cytopenias

    Time frame: 2021-2026

    to define the sensitivity of autoantibody testing in autoimmune cytopenias

  2. specificity of autoantibody testing in autoimmune cytopenias

    Time frame: 2021-2026

    to define the specificity of autoantibody testing in autoimmune cytopenias

  3. sensitivity of bone marrow trephine in autoimmune cytopenias

    Time frame: 2021-2026

    to define the sensitivity of bone marrow trephine in autoimmune cytopenias

Secondary outcomes

  1. overall response rate

    Time frame: 2021-2026

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

  2. Evaluation of somatic mutations

    Time frame: 2021-2026

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

  3. Evaluation of pyruvate kinase activity

    Time frame: 2021-2026

    to define pyruvate kinase activity in myelodysplastic syndromes

  4. Evaluation of microbiome

    Time frame: 2021-2026

    to define microbiome composition in autoimmune cytopenias and myelodysplastic syndromes

  5. Single cell RNA expression

    Time frame: 2021-2026

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

  6. Evaluation of cytokine levels

    Time frame: 2021-2026

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

Sponsors and collaborators

Lead sponsor

Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

Other

Registry information

Official study title

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.

Acronym: AIHA ITP CIN

Important dates

Study start
2019
Primary completion
2030
Study completion
2035
First posted
Jul 5, 2023
Registry last updated
Oct 15, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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