Autoimmune Haemolytic Anaemia, Cytopenia, Thrombocytopenic Purpura, Immune
Conditions
Keywords
Children
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.
Interventions
OTHERBlood sample
A blood sample of 2 times 2 to 5 ml additional maximum
Sponsors
University Hospital, Bordeaux
Eligibility
Sex/Gender
ALL
Age
No minimum to 18 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Complete sustainable remission (yes/no) for children with autoimmune haemolytic anemias | At the screening | 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 |
| complete remission (yes/no) for children with autoimmune haemolytic anemias | At the screening | 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 |
| partial remission (yes/no) for children with autoimmune haemolytic anemias | At the screening | 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 |
| no response (yes/no) for children with autoimmune haemolytic anemias | At the screening | Clinical Signs of Severe Anemia (Grade 3 or More) Or Hemoglobin \<7 g / dl |
| deceased patient (yes/no) for children with autoimmune haemolytic anemias | At the screening | Death yes/no |
| Complete sustainable remission (yes/no) for children with chronic immunologic thrombocytopenic purpura | At the screening | Absence of clinical signs of haemorrhage (grade 0) And platelets\> 100,000 / mm3 And no specific treatment for at least 12 months |
| complete remission (yes/no) for children with chronic immunologic thrombocytopenic purpura | At the screening | 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 |
| partial remission (yes/no) for children with chronic immunologic thrombocytopenic purpura | At the screening | Clinical Signs of Hemorrhage (Grade 1 or 2) Or platelets between 30,000 and 100,000 / mm3 |
| no response (yes/no) for children with chronic immunologic thrombocytopenic purpura | At the screening | Clinical Signs of Severe Hemorrhage (Grade 3 or Greater) Or Platelets \<30,000 / mm3 |
| deceased patient (yes/no) for children with chronic immunologic thrombocytopenic purpura | At the screening | Death yes/no |
Contacts
PRINCIPAL_INVESTIGATORYves PEREL, Pr
Bordeaux University Hsopital
Outcome results
None listed