National Prospective Cohort for Monitoring Children With Severe Autoimmune Cytopenia. (BIOCEREVANCE)

National Prospective Cohort for Monitoring Children With Severe Autoimmune Cytopenia

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 Overview

• Status: Completed
• Conditions: Cytopenia; Autoimmune Haemolytic Anaemia; Thrombocytopenic Purpura, Immune
• Intervention / Treatment: Other: Blood sample

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.

• Study Type: Observational
• Enrollment (Actual): 122

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 18 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Number of groups / cohorts

3

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
children with autoimmune haemolytic anemia; A blood sample of 2 times 2 to 5 ml additional maximum	Other: Blood sample; A blood sample of 2 times 2 to 5 ml additional maximum
Children with Evans syndrome; A blood sample of 2 times 2 to 5 ml additional maximum	Other: Blood sample; A blood sample of 2 times 2 to 5 ml additional maximum
Children with Immune thrombocytopenic purpura; A blood sample of 2 times 2 to 5 ml additional maximum	Other: Blood sample; A blood sample of 2 times 2 to 5 ml additional maximum

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

Collaborators and Investigators

• Sponsor: University Hospital, Bordeaux
• Investigators: Principal Investigator: Yves PEREL, Pr, Bordeaux University Hsopital

Study record dates

Study Major Dates

• Study Start (Actual): April 4, 2007
• Primary Completion (Actual): June 8, 2012
• Study Completion (Actual): June 8, 2012

Study Registration Dates

• First Submitted: August 26, 2019
• First Submitted That Met QC Criteria: August 26, 2019
• First Posted (Actual): August 28, 2019

Study Record Updates

• Last Update Posted (Actual): August 28, 2019
• Last Update Submitted That Met QC Criteria: August 27, 2019
• Last Verified: August 1, 2019

More Information

Terms related to this study

• Keywords: Children
• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Autoimmune Diseases; Hematologic Diseases; Hemorrhage; Hemorrhagic Disorders; Anemia; Blood Coagulation Disorders; Skin Manifestations; Thrombocytopenia; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura; Purpura, Thrombocytopenic; Purpura, Thrombocytopenic, Idiopathic; Anemia, Hemolytic; Anemia, Hemolytic, Autoimmune
• Other Study ID Numbers: CHUBX 2005/18

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No