Clinical trial NCT04070612: National Prospective Cohort for Monitoring Children With Severe Autoimmune Cytopenia.
Official title: National Prospective Cohort for Monitoring Children With Severe Autoimmune Cytopenia
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Cytopenia
Autoimmune Haemolytic Anaemia
Thrombocytopenic Purpura, Immune
Keywords: Children
Lead sponsor: University Hospital, Bordeaux
Sponsor class: OTHER
First posted: 2019-08-28
Enrollment: 122
Sex: ALL
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no

Brief summary: 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: 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.

Eligibility criteria: 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

Primary outcomes:
measure: Complete sustainable remission (yes/no) for children with autoimmune haemolytic anemias
timeFrame: At the screening
measure: complete remission (yes/no) for children with autoimmune haemolytic anemias
timeFrame: At the screening
measure: partial remission (yes/no) for children with autoimmune haemolytic anemias
timeFrame: At the screening
measure: no response (yes/no) for children with autoimmune haemolytic anemias
timeFrame: At the screening
measure: deceased patient (yes/no) for children with autoimmune haemolytic anemias
timeFrame: At the screening
measure: Complete sustainable remission (yes/no) for children with chronic immunologic thrombocytopenic purpura
timeFrame: At the screening
measure: complete remission (yes/no) for children with chronic immunologic thrombocytopenic purpura
timeFrame: At the screening
measure: partial remission (yes/no) for children with chronic immunologic thrombocytopenic purpura
timeFrame: At the screening
measure: no response (yes/no) for children with chronic immunologic thrombocytopenic purpura
timeFrame: At the screening
measure: deceased patient (yes/no) for children with chronic immunologic thrombocytopenic purpura
timeFrame: At the screening
Secondary outcomes:
Other outcomes:
Locations:
Study officials:
affiliation: Bordeaux University Hsopital
name: Yves PEREL, Pr
role: PRINCIPAL_INVESTIGATOR
References: