National Prospective Cohort for Monitoring Children With Severe Autoimmune Cytopenia. (BIOCEREVANCE)
Primary Purpose
Cytopenia, Autoimmune Haemolytic Anaemia, Thrombocytopenic Purpura, Immune
Status
Completed
Phase
Locations
Study Type
Observational
Intervention
Blood sample
Sponsored by
About this trial
This is an observational trial for Cytopenia focused on measuring Children
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
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Arm Label
children with autoimmune haemolytic anemia
Children with Evans syndrome
Children with Immune thrombocytopenic purpura
Arm Description
A blood sample of 2 times 2 to 5 ml additional maximum
A blood sample of 2 times 2 to 5 ml additional maximum
A blood sample of 2 times 2 to 5 ml additional maximum
Outcomes
Primary Outcome Measures
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
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
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
no response (yes/no) for children with autoimmune haemolytic anemias
Clinical Signs of Severe Anemia (Grade 3 or More) Or Hemoglobin <7 g / dl
deceased patient (yes/no) for children with autoimmune haemolytic anemias
Death yes/no
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
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
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
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
deceased patient (yes/no) for children with chronic immunologic thrombocytopenic purpura
Death yes/no
Secondary Outcome Measures
Full Information
NCT ID
NCT04070612
First Posted
August 26, 2019
Last Updated
August 27, 2019
Sponsor
University Hospital, Bordeaux
1. Study Identification
Unique Protocol Identification Number
NCT04070612
Brief Title
National Prospective Cohort for Monitoring Children With Severe Autoimmune Cytopenia.
Acronym
BIOCEREVANCE
Official Title
National Prospective Cohort for Monitoring Children With Severe Autoimmune Cytopenia
Study Type
Observational
2. Study Status
Record Verification Date
August 2019
Overall Recruitment Status
Completed
Study Start Date
April 4, 2007 (Actual)
Primary Completion Date
June 8, 2012 (Actual)
Study Completion Date
June 8, 2012 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University Hospital, Bordeaux
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cytopenia, Autoimmune Haemolytic Anaemia, Thrombocytopenic Purpura, Immune
Keywords
Children
7. Study Design
Enrollment
122 (Actual)
8. Arms, Groups, and Interventions
Arm Title
children with autoimmune haemolytic anemia
Arm Description
A blood sample of 2 times 2 to 5 ml additional maximum
Arm Title
Children with Evans syndrome
Arm Description
A blood sample of 2 times 2 to 5 ml additional maximum
Arm Title
Children with Immune thrombocytopenic purpura
Arm Description
A blood sample of 2 times 2 to 5 ml additional maximum
Intervention Type
Other
Intervention Name(s)
Blood sample
Intervention Description
A blood sample of 2 times 2 to 5 ml additional maximum
Primary Outcome Measure Information:
Title
Complete sustainable remission (yes/no) for children with autoimmune haemolytic anemias
Description
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
Title
complete remission (yes/no) for children with autoimmune haemolytic anemias
Description
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
Title
partial remission (yes/no) for children with autoimmune haemolytic anemias
Description
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
Title
no response (yes/no) for children with autoimmune haemolytic anemias
Description
Clinical Signs of Severe Anemia (Grade 3 or More) Or Hemoglobin <7 g / dl
Time Frame
At the screening
Title
deceased patient (yes/no) for children with autoimmune haemolytic anemias
Description
Death yes/no
Time Frame
At the screening
Title
Complete sustainable remission (yes/no) for children with chronic immunologic thrombocytopenic purpura
Description
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
Title
complete remission (yes/no) for children with chronic immunologic thrombocytopenic purpura
Description
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
Title
partial remission (yes/no) for children with chronic immunologic thrombocytopenic purpura
Description
Clinical Signs of Hemorrhage (Grade 1 or 2) Or platelets between 30,000 and 100,000 / mm3
Time Frame
At the screening
Title
no response (yes/no) for children with chronic immunologic thrombocytopenic purpura
Description
Clinical Signs of Severe Hemorrhage (Grade 3 or Greater) Or Platelets <30,000 / mm3
Time Frame
At the screening
Title
deceased patient (yes/no) for children with chronic immunologic thrombocytopenic purpura
Description
Death yes/no
Time Frame
At the screening
10. Eligibility
Sex
All
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Yves PEREL, Pr
Organizational Affiliation
Bordeaux University Hsopital
Official's Role
Principal Investigator
12. IPD Sharing Statement
Plan to Share IPD
No
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National Prospective Cohort for Monitoring Children With Severe Autoimmune Cytopenia.
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