National Prospective Cohort for Monitoring Children With Severe Autoimmune Cytopenia.

Cytopenia Clinical Trial

— BIOCEREVANCE  

Official title:

National Prospective Cohort for Monitoring Children With Severe Autoimmune Cytopenia

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04070612
• Other study ID #: CHUBX 2005/18
• Status: Completed
• Start date: April 4, 2007
• Est. completion date: June 8, 2012

Study information

• Verified date: August 2019
• Source: University Hospital, Bordeaux
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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 information / eligibility

• Status: Completed
• Enrollment: 122
• Est. completion date: June 8, 2012
• Est. primary completion date: June 8, 2012
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 18 Years

Eligibility

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

Related Conditions & MeSH terms

• Anemia, Hemolytic, Autoimmune
• Autoimmune Haemolytic Anaemia
• Cytopenia
• Purpura
• Purpura, Thrombocytopenic
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenic Purpura, Immune

Intervention

Other:
• Blood sample
A blood sample of 2 times 2 to 5 ml additional maximum

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
University Hospital, Bordeaux

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Primary	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
Primary	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
Primary	deceased patient (yes/no) for children with autoimmune haemolytic anemias	Death yes/no	At the screening
Primary	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
Primary	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
Primary	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
Primary	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
Primary	deceased patient (yes/no) for children with chronic immunologic thrombocytopenic purpura	Death yes/no	At the screening