Trial Title:
Prospective Evaluation of Diagnosis and Treatment of Patients With Autoimmune Cytopenias Including Autoimmune Hemolytic Anemia, Immune Thrombocytopenia, and Chronic Idiopathic/Autoimmune Neutropenia
NCT ID:
NCT05931718
Condition:
Autoimmune Hemolytic Anemia
Immune Thrombocytopenia
Chronic Idiopathic Neutropenia
Autoimmune Neutropenia
Myelodysplastic Syndromes
Cold Agglutinin Disease
Conditions: Official terms:
Preleukemia
Anemia
Myelodysplastic Syndromes
Thrombocytopenia
Neutropenia
Purpura, Thrombocytopenic, Idiopathic
Cytopenia
Anemia, Hemolytic
Anemia, Hemolytic, Autoimmune
Hemolysis
Epoetin Alfa
Luspatercept
Conditions: Keywords:
autoimmune hemolytic anemia
immune thrombocytopenia
chronic idiopathic neutropenia
autoimmune neutropenia
cold agglutinin disease
myelodysplastic syndromes
rituximab
thrombopoietin receptor agonists
erythropoietin
steroids
splenectomy
red cell metabolism
cytokines
luspatercept
microbiome
single cell analysis
next generation sequencing
Study type:
Observational
Overall status:
Recruiting
Study design:
Time perspective:
Prospective
Intervention:
Intervention type:
Biological
Intervention name:
cytokine essays
Description:
evaluation of immunomodulatory cytokines by ELISA kits on peripheral blood samples
Arm group label:
Autoimmune hemolytic anemia
Arm group label:
Chronic idiopathic neutropenia/Autoimmune neutropenia
Arm group label:
Immune thrombocytopenia
Arm group label:
Myelodysplastic syndromes
Intervention type:
Biological
Intervention name:
NGS
Description:
evaluation of somatic mutations commonly associated with myeloid neoplasm and
immunodeficiencies by next generation sequencing on peripheral blood samples
Arm group label:
Autoimmune hemolytic anemia
Arm group label:
Chronic idiopathic neutropenia/Autoimmune neutropenia
Arm group label:
Immune thrombocytopenia
Arm group label:
Myelodysplastic syndromes
Intervention type:
Biological
Intervention name:
Fecal microbiome
Description:
evaluation of fecal microbiome on fecal samples
Arm group label:
Autoimmune hemolytic anemia
Arm group label:
Chronic idiopathic neutropenia/Autoimmune neutropenia
Arm group label:
Immune thrombocytopenia
Arm group label:
Myelodysplastic syndromes
Intervention type:
Drug
Intervention name:
Erythropoietin
Description:
evaluation of recombinant erythropoietin use, safety and efficacy in patients with
autoimmune hemolytic anemia according to clinical practice
Arm group label:
Autoimmune hemolytic anemia
Intervention type:
Drug
Intervention name:
Luspatercept
Description:
evaluation of cytokine levels, molecular profile and bone marrow microenvironment by
single cell analysis in patients treated with luspatercept according to clinical practice
Arm group label:
Myelodysplastic syndromes
Intervention type:
Drug
Intervention name:
Thrombopoietin Receptor Agonist
Description:
evaluation of TPO-RA use, safety and efficacy in patients with ITP according to clinical
practice
Arm group label:
Immune thrombocytopenia
Intervention type:
Drug
Intervention name:
G-CSF
Description:
evaluation of G-CSF use, safety and efficacy in patients with CIN/AIN according to
clinical practice
Arm group label:
Chronic idiopathic neutropenia/Autoimmune neutropenia
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.
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.
Criteria for eligibility:
Study pop:
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.
Sampling method:
Probability Sample
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
Gender:
All
Minimum age:
18 Years
Maximum age:
N/A
Healthy volunteers:
No
Locations:
Facility:
Name:
Fondazione Irccs Ca' Granda Ospedale Maggiore Policlinico
Address:
City:
Milano
Zip:
20100
Country:
Italy
Status:
Recruiting
Contact:
Last name:
Bruno Fattizzo, MD
Phone:
+390255033477
Email:
brunofattizzo@gmail.com
Start date:
June 1, 2019
Completion date:
June 2035
Lead sponsor:
Agency:
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Agency class:
Other
Source:
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Record processing date:
ClinicalTrials.gov processed this data on November 12, 2024
Source: ClinicalTrials.gov page:
https://clinicaltrials.gov/ct2/show/NCT05931718
