National Prospective Cohort for Monitoring Children With Severe Autoimmune Cytopenia.
- Conditions
- Autoimmune Haemolytic AnaemiaCytopeniaThrombocytopenic Purpura, Immune
- Interventions
- Other: Blood sample
- Registration Number
- NCT04070612
- Lead Sponsor
- University Hospital, Bordeaux
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 122
- 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
- Diagnosis of constitutional haemolytic anemia
- Diagnosis of platelet constitutional disease
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Children with Immune thrombocytopenic purpura Blood sample A blood sample of 2 times 2 to 5 ml additional maximum children with autoimmune haemolytic anemia Blood sample A blood sample of 2 times 2 to 5 ml additional maximum Children with Evans syndrome Blood sample A blood sample of 2 times 2 to 5 ml additional maximum
- Primary Outcome Measures
Name Time Method 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
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
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
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
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
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
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
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
- Secondary Outcome Measures
Name Time Method