Autoimmune Hemolytic Anemia, Evan Syndrome, Immune Thrombocytopenic Purpura, Immunotherapy
Conditions
Keywords
CEREVANCE, Pediatrics, Cohort
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.
Interventions
None listed
Sponsors
University Hospital, Bordeaux
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
No minimum to 18 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Haemorrhagic sign - Grade ≥ 3 (Buchanan score) Presence of a haemorrhagic sign ≥grade 3 | At 6 months | Presence of a haemorrhagic sign ≥ grade 3 = moderate bleeding (Buchanan score : from 0 to 5) |
| Thrombocytopenia Presence of thrombocytopenia <30 G / L | At 6 months | Presence of thrombocytopenia \<30 G / L |
| Need of a transfusion ? | At 6 months | Performing a transfusion : yes or no ? |
| Immunoglobulin IV Immunoglobulin IV | At 6 months | Immunoglobulin IV push |
| Corticoids push | At 6 months | Corticoid push (no reduction of their number during treatment follow-up versus non-treatment period) |
| Need a splenectomy ? | At 6 months | Performing a splenectomy : yes or no ? |
| Clinical signs of anemia | 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) |
| Anemia | At 6 months | Presence of anemia \<7g / dl Presence of anemia \<7g / dl |
| Introduction of a new immunomodulatory treatment | At 6 months | Introduction of a new immunomodulatory treatment : yes or no ? |
| Stop treatment or not ? | At 6 months | Stop treatment because of failure, intolerance or non-compliance |
Outcome results
None listed