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

Myelodysplastic Syndromes Clinical Trial

— AIHA ITP CIN  

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.

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05931718
• Other study ID #: CYTOPAN
• Status: Recruiting
• Start date: June 1, 2021
• Est. completion date: June 2030

Study information

• Verified date: May 2023
• Source: Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 200
• Est. completion date: June 2030
• Est. primary completion date: September 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

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

Related Conditions & MeSH terms

• Anemia
• Anemia, Hemolytic
• Anemia, Hemolytic, Autoimmune
• Autoimmune Hemolytic Anemia
• Autoimmune Neutropenia
• Cold Agglutinin Disease
• Cytopenia
• Hemolysis
• Immune Thrombocytopenia
• Myelodysplastic Syndromes
• Neutropenia
• Preleukemia
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia

Intervention

Biological:
• 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

Drug:
• 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

Locations

Country	Name	City	State
Italy	Fondazione Irccs Ca' Granda Ospedale Maggiore Policlinico	Milano

Sponsors (1)

Lead Sponsor	Collaborator
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

Country where clinical trial is conducted

Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	sensitivity of autoantibody testing in autoimmune cytopenias	to define the sensitivity of autoantibody testing in autoimmune cytopenias	2021-2026
Primary	specificity of autoantibody testing in autoimmune cytopenias	to define the specificity of autoantibody testing in autoimmune cytopenias	2021-2026
Primary	sensitivity of bone marrow trephine in autoimmune cytopenias	to define the sensitivity of bone marrow trephine in autoimmune cytopenias	2021-2026
Secondary	overall response rate	to define response rates to treatment of autoimmune cytopenias and myelodysplastic syndromes	2021-2026
Secondary	Evaluation of somatic mutations	to define somatic mutations in autoimmune cytopenias and myelodysplastic syndromes by NGS	2021-2026
Secondary	Evaluation of pyruvate kinase activity	to define pyruvate kinase activity in myelodysplastic syndromes	2021-2026
Secondary	Evaluation of microbiome	to define microbiome composition in autoimmune cytopenias and myelodysplastic syndromes	2021-2026
Secondary	Single cell RNA expression	to define bone marrow composition by single cell analysis in autoimmune cytopenias and myelodysplastic syndromes	2021-2026
Secondary	Evaluation of cytokine levels	to define cytokine levels in autoimmune cytopenias and myelodysplastic syndromes by ELISA	2021-2026