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NCT Number: NCT04070612

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.

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Key information

Conditions

Cytopenia Anemia Anemia, Hemolytic Anemia, Hemolytic, Autoimmune Autoimmune Diseases Autoimmune Haemolytic Anaemia Blood Coagulation Disorders Blood Platelet Disorders Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhage Hemorrhagic Disorders Immune System Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Purpura Purpura, Thrombocytopenic Purpura, Thrombocytopenic, Idiopathic Signs and Symptoms Skin Manifestations Thrombocytopenia Thrombocytopenic Purpura, Immune Thrombotic Microangiopathies

Age range

Up to 18 year

Sex eligibility

All sexes

Study type

Observational

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

blood sample

Other

A blood sample of 2 times 2 to 5 ml additional maximum

Primary outcomes

  1. Complete sustainable remission (yes/no) for children with autoimmune haemolytic anemias

    Time frame: 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

  2. complete remission (yes/no) for children with autoimmune haemolytic anemias

    Time frame: 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

  3. partial remission (yes/no) for children with autoimmune haemolytic anemias

    Time frame: 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

  4. no response (yes/no) for children with autoimmune haemolytic anemias

    Time frame: At the screening

    Clinical Signs of Severe Anemia (Grade 3 or More) Or Hemoglobin <7 g / dl

  5. deceased patient (yes/no) for children with autoimmune haemolytic anemias

    Time frame: At the screening

    Death yes/no

  6. Complete sustainable remission (yes/no) for children with chronic immunologic thrombocytopenic purpura

    Time frame: 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

  7. complete remission (yes/no) for children with chronic immunologic thrombocytopenic purpura

    Time frame: 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

  8. partial remission (yes/no) for children with chronic immunologic thrombocytopenic purpura

    Time frame: At the screening

    Clinical Signs of Hemorrhage (Grade 1 or 2) Or platelets between 30,000 and 100,000 / mm3

  9. no response (yes/no) for children with chronic immunologic thrombocytopenic purpura

    Time frame: At the screening

    Clinical Signs of Severe Hemorrhage (Grade 3 or Greater) Or Platelets <30,000 / mm3

  10. deceased patient (yes/no) for children with chronic immunologic thrombocytopenic purpura

    Time frame: At the screening

    Death yes/no

Sponsors and collaborators

Lead sponsor

University Hospital, Bordeaux

Other

Registry information

Official study title

National Prospective Cohort for Monitoring Children With Severe Autoimmune Cytopenia

Acronym: BIOCEREVANCE

Important dates

Study start
2007
Primary completion
2012
Study completion
2012
First posted
Aug 28, 2019
Registry last updated
Jun 18, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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