Clinical trial NCT04057703: Balance Benefit / Risk of Immunomodulatory Treatments at the Child and Adolescent for Autoimmune Cytopenia.
Official 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.
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Immune Thrombocytopenic Purpura
Autoimmune Hemolytic Anemia
Evan Syndrome
Immunotherapy
Keywords: CEREVANCE
Pediatrics
Cohort
Lead sponsor: University Hospital, Bordeaux
Sponsor class: OTHER
First posted: 2019-08-15
Enrollment: 454
Sex: ALL
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Children or adolescents who have received one or more treatments of interest for a Chronic Immune-Thrombocytopenic Purpura, Autoimmune Hemolytic Anemia , or EVANS syndrome, in one of the 30 centers of the CEREVANCE network, will be identified and selected by the team CEREVANCE, in the OBS'CEREVANCE computer database created since 2004. The first second-line treatment they received will be analyzed.

Brief summary: 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.

Detailed description: 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.

Eligibility criteria: 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.

Primary outcomes:
measure: Haemorrhagic sign - Grade ≥ 3 (Buchanan score) Presence of a haemorrhagic sign ≥grade 3
timeFrame: At 6 months
measure: Thrombocytopenia Presence of thrombocytopenia <30 G / L
timeFrame: At 6 months
measure: Need of a transfusion ?
timeFrame: At 6 months
measure: Immunoglobulin IV Immunoglobulin IV
timeFrame: At 6 months
measure: Corticoids push
timeFrame: At 6 months
measure: Need a splenectomy ?
timeFrame: At 6 months
measure: Clinical signs of anemia
timeFrame: At 6 months
measure: Anemia
timeFrame: At 6 months
measure: Introduction of a new immunomodulatory treatment
timeFrame: At 6 months
measure: Stop treatment or not ?
timeFrame: At 6 months
Secondary outcomes:
Other outcomes:
Locations:
Study officials:
affiliation: University Hospital, Bordeaux
name: Nathalie ALADJIDI, Dr
role: PRINCIPAL_INVESTIGATOR
References: