Cytopenia Clinical Trial
Official title:
National Prospective Cohort for Monitoring Children With Severe Autoimmune Cytopenia
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.
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.
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