ClinicalTrials.gov record NCT05931718

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Aug 22, 2026

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.

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeObservationalA study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.
Age18 years old and older
SexAll
LocationItaly
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This prospective observational study follows adults with autoimmune cytopenias (AIHA, ITP, chronic idiopathic/autoimmune neutropenia) and a subgroup with myelodysplastic syndromes. Participants give baseline blood and stool samples and are followed for at least three years while their diagnostic workup, treatments given in routine care, responses, and complications are recorded. Bone marrow sampling and single-cell analysis may be done if clinically indicated.

What it is studying. The study aims to characterize diagnostic tools and real-world treatment patterns for autoimmune cytopenias. It evaluates traditional and novel diagnostics (autoantibody testing, bone marrow studies, cytokine measurements, somatic mutation profiling, and the fecal microbiome), records types and sequences of therapies, documents responses and adverse events, seeks clinical and laboratory predictors of outcome, and monitors whether autoimmune cytopenias evolve into myelodysplastic syndromes. A subgroup of patients with MDS will be assessed for immune activation and red cell metabolism.

What the researchers hope to learn. Scientific goals include defining sensitivity and specificity of autoantibody testing and bone marrow trephine, describing treatment types and response rates, and identifying somatic mutations, cytokine patterns, microbiome composition, pyruvate kinase activity, and bone marrow cell composition by single-cell analysis. The study also aims to evaluate adverse events and the possible progression from autoimmune cytopenias to myelodysplastic syndromes.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • Erythropoietin
  • Luspatercept
  • Thrombopoietin Receptor Agonist
  • G-CSF
Biological
  • cytokine essays
  • NGS
  • Fecal microbiome

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

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.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants receive routine clinical and laboratory diagnostic workup as per usual care. At baseline they provide blood samples and a fecal sample for microbiome studies; targeted laboratory testing (cytokine assays, molecular/NGS) will be performed on collected samples. If clinically indicated, a bone marrow evaluation (biopsy/aspirate) may be done and a sample taken for single-cell analysis. Participants are followed for at least 3 years with regular visits or records review to collect information on treatments, responses, relapses, and complications.

How the study is structured. A prospective observational cohort: people are enrolled (typically at diagnosis), provide baseline blood and stool samples, may undergo bone marrow sampling if clinically indicated, and are followed for at least three years. No experimental treatments are assigned; treatments are provided as part of routine clinical care and the study records diagnostic results, therapies, responses, and complications.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
  • The listed sites are in Italy. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on cold agglutinin disease

This trial is also listed on this site under autoimmune hemolytic anemia.

Clinical trials6 trials listed for cold agglutinin diseaseEvery trial the registry lists for cold agglutinin disease, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in cold agglutinin disease researchLatest monthly update: September 2026. Includes: Rituximab-based therapy. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Jun 1, 2019
Primary completion
September 2030 (estimated)
Study completion
June 2035 (estimated)
Target enrollment
200 participants (estimated)
Last updated on registry
Oct 15, 2024
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

Participating sites (1)

Terms used on this page · explanations by MedicalUpdates.ai

adverse events
Any unwanted medical problem that happens during a study, whether or not it was caused by the treatment. A 'serious' adverse event is one that meets specific criteria, such as causing death, being life-threatening, requiring or prolonging hospital admission, causing significant disability, or another medically important outcome.
baseline
Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
follow up
The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
enrolment
The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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