National Prospective Cohort for Monitoring Children With Severe Autoimmune Cytopenia. (BIOCEREVANCE)

University Hospital of Bordeaux

Status: Completed

Conditions: Cytopenia; Thrombocytopenic Purpura, Immune; Autoimmune Haemolytic Anaemia

Treatments: Other: Blood sample

Study type: Observational

Funder types: Other

Identifiers

NCT04070612

CHUBX 2005/18

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 122 patients

Sex: All

Ages: Under 18 years old

Volunteers: No Healthy Volunteers

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

Trial design

122 participants in 3 patient groups

children with autoimmune haemolytic anemia

Description:

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

Treatment:

Other: Blood sample

Children with Evans syndrome

Description:

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

Treatment:

Other: Blood sample

Children with Immune thrombocytopenic purpura

Description:

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

Treatment:

Other: Blood sample