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

Condition(s) studied

Investigational drug(s) / intervention(s)

cytokine essays: evaluation of immunomodulatory cytokines by ELISA kits on peripheral blood samples

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

Fecal microbiome: evaluation of fecal microbiome on fecal samples

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

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

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

Study 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.

Eligibility

Primary outcome measure(s)

• sensitivity of autoantibody testing in autoimmune cytopenias — 2021-2026
  to define the sensitivity of autoantibody testing in autoimmune cytopenias
• specificity of autoantibody testing in autoimmune cytopenias — 2021-2026
  to define the specificity of autoantibody testing in autoimmune cytopenias
• sensitivity of bone marrow trephine in autoimmune cytopenias — 2021-2026
  to define the sensitivity of bone marrow trephine in autoimmune cytopenias

Trial sites (1)

More Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico trials in Italy

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