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.
Study Type
OBSERVATIONAL
Enrollment
122
A blood sample of 2 times 2 to 5 ml additional maximum
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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: At the screening
deceased patient (yes/no) for children with autoimmune haemolytic anemias
Death yes/no
Time frame: 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
Time frame: At the screening
complete remission (yes/no) for children with chronic immunologic thrombocytopenic purpura
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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
Time frame: 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
Time frame: 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
Time frame: At the screening
deceased patient (yes/no) for children with chronic immunologic thrombocytopenic purpura
Death yes/no
Time frame: At the screening