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

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


Lead Sponsor: University Hospital, Bordeaux

Source University Hospital, Bordeaux
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.

Overall Status Completed
Start Date January 18, 2016
Completion Date December 4, 2016
Primary Completion Date December 4, 2016
Study Type Observational
Primary Outcome
Measure Time Frame
Haemorrhagic sign - Grade ≥ 3 (Buchanan score) Presence of a haemorrhagic sign ≥grade 3 At 6 months
Thrombocytopenia Presence of thrombocytopenia <30 G / L At 6 months
Need of a transfusion ? At 6 months
Immunoglobulin IV Immunoglobulin IV At 6 months
Corticoids push At 6 months
Need a splenectomy ? At 6 months
Clinical signs of anemia At 6 months
Anemia At 6 months
Introduction of a new immunomodulatory treatment At 6 months
Stop treatment or not ? At 6 months
Enrollment 454

Sampling Method: Non-Probability Sample


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.

Gender: All

Minimum Age: N/A

Maximum Age: 18 Years

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
Nathalie ALADJIDI, Dr Principal Investigator University Hospital, Bordeaux
Verification Date

August 2019

Responsible Party

Type: Sponsor

Has Expanded Access No
Condition Browse
Patient Data No
Study Design Info

Observational Model: Cohort

Time Perspective: Prospective