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Differential Diagnostic of Immune ThrombocytoPenia (ITP) and Myelodysplastic Syndrome (MDS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03469661
Recruitment Status : Completed
First Posted : March 19, 2018
Last Update Posted : August 18, 2021
Information provided by (Responsible Party):
Fundacion CRIS de Investigación para Vencer el Cáncer

Brief Summary:

Current diagnostic criteria for Immune ThrombocytoPenia (ITP) are mainly based on the presence of low numbers of platelets, excluding other multiple causes of thrombocytopenia, including immunodeficiencies, constitutional or acquired thrombocytopenia, hypersplenism and clonal hematological disorders such as MDS, disorders lymphoproliferative and acute myeloid leukemia (AML), among others. The analysis complementary tests for the diagnosis of ITP include studies basic systematic hematology, together with autoimmune assays and microbiological tests, while the evaluation of bone marrow is limited to elderly patients and/or patients resistant to treatment. Previous research has described the development of Myelodysplastic Syndrome (MDS) in patients with a previous diagnosis of ITP, and even the presence of MDS associated with genetic background. Therefore, it is conceivable fact that a percentage of cases with clinical signs of ITP in the moment of appearance may actually correspond to the first stages of MDS development in which bone marrow cells are not systematically evaluated in the initial presentation.

The anomalous immunophenotypic patterns between multiple compartments of bone marrow cells and peripherally blood (PB) platelets have been characterized through flow cytometry. The flow cytometry currently represents an important complementary tool for diagnosis of MDS that has shown great effectiveness and applicability in the differential diagnosis of non-clonal cytopenias against early MDS and for the detection of stages prior to MDS. Besides, the flow cytometry has made it possible to detect the presence of coexisting features related to MDS in patients with other malignancies hematologic conditions such as multiple myeloma, AML, and lymphocytic leukemia chronic. Therefore, the immunophenotypic analysis of the cells of the bone marrow of patients with ITP at the time of appearance would help to identify the cases that underlie clonal hematopoiesis MDS type. In the present study it is planned a broad characterization immunophenotyping of multiple compartments of bone marrow cells and PB platelets from patients with recently diagnosed ITP and investigate their morphological antecedents, in order to identify those patients who show compatible clonal hematopoietic patterns with MDS evident (or at risk of development), as candidates to receive most appropriate therapeutic methods.

Condition or disease
Immune Thrombocytopenia Myelodysplastic Syndromes

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Study Type : Observational
Actual Enrollment : 63 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Differential Diagnostic of ITP and MDS: a Prospective Study by Next-Generation Flow Cytometry and Cytomorphological Approaches
Actual Study Start Date : April 25, 2018
Actual Primary Completion Date : April 19, 2021
Actual Study Completion Date : April 19, 2021

Immune Thrombocytopenia Diagnosis
Myelodysplastic Syndrome Diagnosis

Primary Outcome Measures :
  1. Morphological profile [ Time Frame: Baseline ]
    Bone marrow cell compartment profiles

  2. Morphological profile [ Time Frame: Baseline ]
    Peripheral blood platelets profiles

  3. Immunological profile [ Time Frame: Baseline ]
    Bone marrow cell compartment profiles

  4. Immunological profile [ Time Frame: Baseline ]
    Peripheral blood platelets profiles

Secondary Outcome Measures :
  1. Immunophenotypic abnormalities [ Time Frame: Baseline ]
    Evaluation of abnormal immunophenotypic profiles

  2. Morphological abnormalities [ Time Frame: Baseline ]
    Evaluation of abnormal morphological profiles.

Biospecimen Retention:   Samples With DNA
Given a diagnosis of ITP or low-risk MDS, EDTA-anticoagulated bone marrow and peripheral blood aspirates will be obtained per case. A biopsy sample and two unstained blood smears should also be included

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
It is estimated that approximately 60 patients will be included in the study, 30 newly diagnosed ITP patients and 30 newly diagnosed MDS cases.

Inclusion Criteria:

  • Patients aged ≥ 18 years old at diagnosis
  • Informed consent in writing
  • Newly diagnosed primary ITP patients, or
  • Newly diagnosed MDS patients

Exclusion Criteria:

  • Patients who participated in a interventional thrombopoietin receptor agonists (TPO-RA) clinical trial since TPO-RA treatment initiation
  • Patients with secondary immune thrombopenia

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03469661

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Complejo Hospitalario de A Coruña
A Coruña, Spain
Hospital de Burgos
Burgos, Spain
Hospital Universitario Insular de Gran Canaria
Las Palmas De Gran Canaria, Spain
Hospital Ramon y Cajal
Madrid, Spain
Hospital Regional de Málaga
Málaga, Spain
Hospital Virgen de la Victoria
Málaga, Spain
Sponsors and Collaborators
Fundacion CRIS de Investigación para Vencer el Cáncer
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Principal Investigator: Tomás González Hospital de Burgos
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Responsible Party: Fundacion CRIS de Investigación para Vencer el Cáncer Identifier: NCT03469661    
Other Study ID Numbers: FCR-PTI-2017-01
First Posted: March 19, 2018    Key Record Dates
Last Update Posted: August 18, 2021
Last Verified: August 2021

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Myelodysplastic Syndromes
Purpura, Thrombocytopenic, Idiopathic
Pathologic Processes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Blood Platelet Disorders
Purpura, Thrombocytopenic
Blood Coagulation Disorders
Thrombotic Microangiopathies
Hemorrhagic Disorders
Autoimmune Diseases
Immune System Diseases
Skin Manifestations