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NCT Number: NCT04057703

Balance Benefit / Risk of Immunomodulatory Treatments at the Child and Adolescent for Autoimmune Cytopenia.

In France, a national prospective cohort for monitoring children and adolescents with autoimmune cytopenia OBS'CEREVANCE is in place since 2004. It is coordinated in Bordeaux by the Center's team. Reference Rare Diseases CEREVANCE. It has been validated by the French Data Protection Authority in 2009 (information note and written consent). It had mid 2013 more of 900 patients, and the data collected make it possible to study intentionally to treat the therapeutic management of patients with Chronic Immune-Thrombocytopenic Purpura, from Autoimmune Hemolytic Anemia, or from EVANS syndrome.

This study evaluates efficacy and tolerance at 6 months of treatment immunomodulators prescribed in France in real conditions of use, in children and adolescents under the age of 18, for a Chronic Immune-Thrombocytopenic Purpura, an Autoimmune Hemolytic Anemia or a simultaneous EVANS syndrome.

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Key information

Conditions

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

Age range

Up to 18 year

Sex eligibility

All sexes

Study type

Observational

About this study

Chronic immunological thrombocytopenic purpura and anemia hemolytic autoimmune are rare autoimmune hematologic diseases, primary or secondary, affecting the child often very young, sometimes associated simultaneously or sequentially (Evans syndrome). They can be life-threatening, they in 20 to 50% of cases of prolonged dependence on immunosuppressants. The incidence of Immune-Thrombocytopenic Purpura is 2 to 5 / 100,000 inhabitants of under 18, the Autoimmune Hemolytic Anemia 5 to 10 times lower.

For the pediatric population, the experience reported in the literature is limited to individual cases or small series often retrospective and not comparative. It does not allow to have in 2013 a reasoning based on on evidence to define the second-line therapeutic strategy, in especially for Autoimmune Hemolytic Anemia where the therapeutic data are almost nonexistent. Splenectomy remains to this day the reference treatment of the Chronic Immune-Thrombocytopenic Purpura of the adult. In children, the therapeutic goal is to avoid it or to prevent it delay it. After failure of first-line treatments (immunoglobulins or corticosteroids) used for treatment of relapses or continuously, the main immunomodulatory treatments used in second line are azathioprine, ciclosporin, hydroxychloroquine, rituximab, mycophenolate mofetil, romiplostim, eltrombopag.

The benefit / risk balance of these 7 immunomodulatory treatments prescribed to the child for autoimmune cytopenia is presumed to be favorable based on the very limited data from the pediatric literature and the experience of most adults do not benefit from a specific marketing authorization.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Patient initiating a first second-line immunomodulatory treatment (azathioprine, ciclosporine, eltrombopag, hydroxychloroquine, mycophénolate mofétil, rituximab, romiplostim), registered in the OBS'CEREVANCE database, presenting a Chronic Immune-Thrombocytopenic Purpura, Autoimmune Hemolytic Anemia or simultaneous EVANS syndrome.

Exclusion criteria

  • Patient under immunosuppressant for another immunological pathology at the initiation of the first second line treatment,
  • Patient treated with 2 second-line treatments on the same day,
  • Oral refusal of participation of the patient or his legal representatives, after reading the information note specific for the study.

Treatment and study plan

Primary outcomes

  1. Haemorrhagic sign - Grade ≥ 3 (Buchanan score) Presence of a haemorrhagic sign ≥grade 3

    Time frame: At 6 months

    Presence of a haemorrhagic sign ≥ grade 3 = moderate bleeding (Buchanan score : from 0 to 5)

  2. Thrombocytopenia Presence of thrombocytopenia <30 G / L

    Time frame: At 6 months

    Presence of thrombocytopenia <30 G / L

  3. Need of a transfusion ?

    Time frame: At 6 months

    Performing a transfusion : yes or no ?

  4. Immunoglobulin IV Immunoglobulin IV

    Time frame: At 6 months

    Immunoglobulin IV push

  5. Corticoids push

    Time frame: At 6 months

    Corticoid push (no reduction of their number during treatment follow-up versus non-treatment period)

  6. Need a splenectomy ?

    Time frame: At 6 months

    Performing a splenectomy : yes or no ?

  7. Clinical signs of anemia

    Time frame: At 6 months

    Presence of clinical signs of anemia ≥ grade 2 = Subject bedridden less than 50% of the day (WHO score : from 0 to 4)

  8. Anemia

    Time frame: At 6 months

    Presence of anemia <7g / dl

    Presence of anemia <7g / dl

  9. Introduction of a new immunomodulatory treatment

    Time frame: At 6 months

    Introduction of a new immunomodulatory treatment : yes or no ?

  10. Stop treatment or not ?

    Time frame: At 6 months

    Stop treatment because of failure, intolerance or non-compliance

Sponsors and collaborators

Lead sponsor

University Hospital, Bordeaux

Other

Registry information

Official study title

National Study Backed by a Rare Disease Cohort the Benefit / Risk Balance of Immunomodulatory Treatments Prescribed in the Child and Adolescent for Autoimmune Cytopenia.

Acronym: VIGICAIRE

Important dates

Study start
2016
Primary completion
2016
Study completion
2016
First posted
Aug 15, 2019
Registry last updated
Aug 15, 2019

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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