Prospective Evaluation of Diagnosis and Treatment of Patients With Autoimmune Cytopenias Including Autoimmune Hemolytic Anemia, Immune Thrombocytopenia, and Chronic Idiopathic/Autoimmune Neutropenia
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: cytokine essays, NGS, Fecal microbiome, Erythropoietin.
- Who it may be relevant to
- Registry conditions: Autoimmune Hemolytic Anemia, Immune Thrombocytopenia, Chronic Idiopathic Neutropenia, Autoimmune Neutropenia. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Italy
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official 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.
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.
Detailed 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.
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
Primary outcome measures
- sensitivity of autoantibody testing in autoimmune cytopenias [Time frame: 2021-2026]
- specificity of autoantibody testing in autoimmune cytopenias [Time frame: 2021-2026]
- sensitivity of bone marrow trephine in autoimmune cytopenias [Time frame: 2021-2026]
Secondary outcome measures (6)
- overall response rate [Time frame: 2021-2026]
- Evaluation of somatic mutations [Time frame: 2021-2026]
- Evaluation of pyruvate kinase activity [Time frame: 2021-2026]
- Evaluation of microbiome [Time frame: 2021-2026]
- Single cell RNA expression [Time frame: 2021-2026]
- Evaluation of cytokine levels [Time frame: 2021-2026]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
Italy · 1 center
- Fondazione Irccs Ca' Granda Ospedale Maggiore Policlinico — Milan
Publications
- Fattizzo B, Pedone GL, Brambilla C, Pettine L, Zaninoni A, Passamonti F, Barcellini W. Recombinant erythropoietin in autoimmune hemolytic anemia with inadequate bone marrow response: a prospective analysis. Blood Adv. 2024 Mar 12;8(5):1322-1327. doi: 10.1182/bloodadvances.2023011798. PMID 38029356
- Versino F, Revelli N, Villa S, Pettine L, Zaninoni A, Prati D, Passamonti F, Barcellini W, Fattizzo B. Transfusions in autoimmune hemolytic anemias: Frequency and clinical significance of alloimmunization. J Intern Med. 2024 Mar;295(3):369-374. doi: 10.1111/joim.13753. Epub 2023 Nov 27. PMID 38013593
Identifiers
NCT: NCT05931718 · CYTOPAN