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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

ConditionsAutoimmune Hemolytic AnemiaImmune ThrombocytopeniaChronic Idiopathic NeutropeniaAutoimmune NeutropeniaMyelodysplastic SyndromesCold Agglutinin Disease
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18+
SponsorFondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Diagnosis of autoimmune cytopenias (AIHA/ITP/CIN/AIN)

age >/= 18 years

ability to sign informed consent

availability to undergo 3 year follow up

Disqualifiers

any condition impeding the acquisition of the informed consent

immune cytopenia diagnosis preceding >/= 6 months the enrolment

Trial population

Patients with anemia (Hb \<12 g/dL), thrombocytopenia (PLT\<100.000/mmc) and/or neutropenia (neutrophils\<1000/mmc) attending the inpatient or outpatient facility of Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy. Cytopenia diagnosis will be based on current guidelines for AIHA (evidence of hemolysis and positive direct Coombs test or negative once excluded other causes), for ITP (exclusion of other thrombocytopenia causes) and for CIN/AIN (exclusion of other neutropenia causes). Patients will be classified according to the degree of the cytopenia and, for AIHA, according to thermal characteristics (warm, cold, mixed, atypical). For the substudy of patients with MDS patients will be stratified according to WHO 2022 classification of myeloid neoplasms.

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

  • cytokine essays

    Biological/Vaccine

    evaluation of immunomodulatory cytokines by ELISA kits on peripheral blood samples

  • NGS

    Biological/Vaccine

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

  • Fecal microbiome

    Biological/Vaccine

    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

Treatment groups

200 Participants
are divided into 4 treatment groups
Group A: Autoimmune hemolytic anemia4 interventions
Group B: Immune thrombocytopenia4 interventions
Group C: Chronic idiopathic neutropenia/Autoimmune neutropenia4 interventions
Group D: Myelodysplastic syndromes4 interventions

Trial outcomes

Primary outcomes

1

sensitivity of autoantibody testing in autoimmune cytopenias

to define the sensitivity of autoantibody testing in autoimmune cytopenias

Time frame
2021-2026
2

specificity of autoantibody testing in autoimmune cytopenias

to define the specificity of autoantibody testing in autoimmune cytopenias

Time frame
2021-2026
3

sensitivity of bone marrow trephine in autoimmune cytopenias

to define the sensitivity of bone marrow trephine in autoimmune cytopenias

Time frame
2021-2026

Secondary outcomes

1

overall response rate

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

Time frame
2021-2026
2

Evaluation of somatic mutations

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

Time frame
2021-2026
3

Evaluation of pyruvate kinase activity

to define pyruvate kinase activity in myelodysplastic syndromes

Time frame
2021-2026
4

Evaluation of microbiome

to define microbiome composition in autoimmune cytopenias and myelodysplastic syndromes

Time frame
2021-2026

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

Lead sponsor

Source ClinicalTrials.gov
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