National Prospective Cohort for Monitoring Children With Severe Autoimmune Cytopenia.

Study Overview
=================
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.

Official Title
-----------------
National Prospective Cohort for Monitoring Children With Severe Autoimmune Cytopenia

Conditions
-----------------
Cytopenia, Autoimmune Haemolytic Anaemia, Thrombocytopenic Purpura, Immune

Intervention / Treatment
-----------------
* Other: Blood sample


Participation Criteria
=================
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

Ages Eligible for Study
-----------------
Maximum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| children with autoimmune haemolytic anemia<br>A blood sample of 2 times 2 to 5 ml additional maximum | Other: Blood sample<br>* A blood sample of 2 times 2 to 5 ml additional maximum<br>|
| Children with Evans syndrome<br>A blood sample of 2 times 2 to 5 ml additional maximum | Other: Blood sample<br>* A blood sample of 2 times 2 to 5 ml additional maximum<br>|
| Children with Immune thrombocytopenic purpura<br>A blood sample of 2 times 2 to 5 ml additional maximum | Other: Blood sample<br>* A blood sample of 2 times 2 to 5 ml additional maximum<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Complete sustainable remission (yes/no) for children with autoimmune haemolytic anemias | Absence of clinical signs of anemia (grade 0) And Hemoglobin > 11 g / dl And reticulocytes <120,000 / mm3 And haptoglobin> 10 mg / dl And bilirubin <10 mg / l or 17 μmol / l And no specific treatment for at least 12 months | At the screening | 
| complete remission (yes/no) for children with autoimmune haemolytic anemias | Absence of clinical signs of anemia (grade 0) And Hemoglobin> 11 g / dl And reticulocytes <120,000 / mm3 Regardless of the level of haptoglobin or bilirubin And specific treatment in progress or interrupted for less than 12 months | At the screening | 
| partial remission (yes/no) for children with autoimmune haemolytic anemias | Clinical Signs of Anemia (Grade 1 or 2) Or Hemoglobin from 7 to 11 g / dl Or reticulocytes> 120,000 / mm3 Regardless of the level of haptoglobin or bilirubin | At the screening | 
| no response (yes/no) for children with autoimmune haemolytic anemias | Clinical Signs of Severe Anemia (Grade 3 or More) Or Hemoglobin <7 g / dl | At the screening | 
| deceased patient (yes/no) for children with autoimmune haemolytic anemias | Death yes/no | At the screening | 
| Complete sustainable remission (yes/no) for children with chronic immunologic thrombocytopenic purpura | Absence of clinical signs of haemorrhage (grade 0) And platelets> 100,000 / mm3 And no specific treatment for at least 12 months | At the screening | 
| complete remission (yes/no) for children with chronic immunologic thrombocytopenic purpura | Absence of clinical signs of haemorrhage (grade 0) And platelets> 100,000 / mm3 And specific treatment in progress or interrupted for less than 12 months | At the screening | 
| partial remission (yes/no) for children with chronic immunologic thrombocytopenic purpura | Clinical Signs of Hemorrhage (Grade 1 or 2) Or platelets between 30,000 and 100,000 / mm3 | At the screening | 
| no response (yes/no) for children with chronic immunologic thrombocytopenic purpura | Clinical Signs of Severe Hemorrhage (Grade 3 or Greater) Or Platelets <30,000 / mm3 | At the screening | 
| deceased patient (yes/no) for children with chronic immunologic thrombocytopenic purpura | Death yes/no | At the screening | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
Children