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

NCT03469661 | Completed

• 1 other identifier: FCR-PTI-2017-01
• Study Type: observational
• Target: 63
• Locations: 1 country
• Sites: 6

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.

Conditions

Immune Thrombocytopenia; Myelodysplastic Syndromes

Outcome Measures

Primary Outcomes (4)

• Morphological profile

  Bone marrow cell compartment profiles

  Baseline

• Morphological profile

  Peripheral blood platelets profiles

  Baseline

• Immunological profile

  Bone marrow cell compartment profiles

  Baseline

• Immunological profile

  Peripheral blood platelets profiles

  Baseline

Secondary Outcomes (2)

• Immunophenotypic abnormalities

  Baseline

• Morphological abnormalities

  Baseline

Study Arms (2)

Immune Thrombocytopenia Diagnosis

Myelodysplastic Syndrome Diagnosis

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

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

You may not qualify if:

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

Sponsors & Collaborators

• Fundacion CRIS de Investigación para Vencer el Cáncer: lead
• Amgen: collaborator

Study Sites (6)

Complejo Hospitalario de A Coruña

A Coruña, Spain

Location

Hospital de Burgos

Burgos, Spain

Location

Hospital Universitario Insular de Gran Canaria

Las Palmas de Gran Canaria, Spain

Location

Hospital Ramon y Cajal

Madrid, Spain

Location

Hospital Regional de Málaga

Málaga, Spain

Location

Hospital Virgen de la Victoria

Málaga, Spain

Location

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

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic; Myelodysplastic Syndromes

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms; Bone Marrow Diseases

Study Officials

• Tomás González

  Hospital de Burgos

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 2, 2018
• First Posted: March 19, 2018
• Study Start: April 25, 2018
• Primary Completion: April 19, 2021
• Study Completion: April 19, 2021
• Last Updated: August 18, 2021

Record last verified: 2021-08

Locations

ES (6)