National Prospective Cohort for Monitoring Children With Severe Autoimmune Cytopenia.

NCT ID: NCT04070612

Last Updated: 2019-08-28

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

122 participants

Study Classification

OBSERVATIONAL

Study Start Date

2007-04-04

Study Completion Date

2012-06-08

Brief Summary

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This study aims to study prospectively the clinical and paraclinical evolution and prognostic factors of autoimmune haemolytic anemias, Evans syndromes and chronic immunological thrombocytopenic purpura of children in France.

Detailed Description

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These autoimmune haematological diseases are rare diseases affecting the child, often very young, and serious and potentially life-threatening. International literature data are scarce, and include individual cases or small series.

They do not allow to determine an optimal therapeutic strategy in case of escape from the first-line treatments. Existing treatments (long-term corticosteroid therapy, immunoglobulins, splenectomy, immunosuppressants, chemotherapies, and more recently anti-CD20 antibodies) are inconsistently effective, and often associated with serious side effects.

The seriousness of these diseases, the therapeutic difficulties, and the absence of a targeted research project in France, led to the implementation of this study.

This study aims to study prospectively the clinical and paraclinical evolution and prognostic factors of autoimmune haemolytic anemias, Evans syndromes and chronic immunological thrombocytopenic purpura of children in France.

Conditions

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Cytopenia Autoimmune Haemolytic Anaemia Thrombocytopenic Purpura, Immune

Study Groups

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children with autoimmune haemolytic anemia

A blood sample of 2 times 2 to 5 ml additional maximum

Blood sample

Intervention Type OTHER

A blood sample of 2 times 2 to 5 ml additional maximum

Children with Evans syndrome

A blood sample of 2 times 2 to 5 ml additional maximum

Blood sample

Intervention Type OTHER

A blood sample of 2 times 2 to 5 ml additional maximum

Children with Immune thrombocytopenic purpura

A blood sample of 2 times 2 to 5 ml additional maximum

Blood sample

Intervention Type OTHER

A blood sample of 2 times 2 to 5 ml additional maximum

Interventions

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Blood sample

A blood sample of 2 times 2 to 5 ml additional maximum

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* Age strictly below 18 years of age at initial diagnosis
* Affiliate child or beneficiary of a social security scheme
* Child residing in metropolitan France
* Diagnosis of autoimmune haemolytic anemias, Evans syndrome and / or chronic Immune thrombocytopenic purpura
* Free, informed, written and signed consent

Exclusion Criteria

* Diagnosis of constitutional haemolytic anemia
* Diagnosis of platelet constitutional disease
Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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University Hospital, Bordeaux

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Yves PEREL, Pr

Role: PRINCIPAL_INVESTIGATOR

Bordeaux University Hsopital

Other Identifiers

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CHUBX 2005/18

Identifier Type: -

Identifier Source: org_study_id

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