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

Immunotherapy Clinical Trial

— VIGICAIRE  

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.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04057703
• Other study ID #: CHU BX 2013/24
• Status: Completed
• Start date: January 18, 2016
• Est. completion date: December 4, 2016

Study information

• Verified date: August 2019
• Source: University Hospital, Bordeaux
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 454
• Est. completion date: December 4, 2016
• Est. primary completion date: December 4, 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 18 Years

Eligibility

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.

Related Conditions & MeSH terms

• Anemia, Hemolytic
• Anemia, Hemolytic, Autoimmune
• Autoimmune Hemolytic Anemia
• Evan Syndrome
• Immune Thrombocytopenic Purpura
• Immunotherapy
• Purpura
• Purpura, Thrombocytopenic
• Purpura, Thrombocytopenic, Idiopathic

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
University Hospital, Bordeaux

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Haemorrhagic sign - Grade = 3 (Buchanan score) Presence of a haemorrhagic sign =grade 3	Presence of a haemorrhagic sign = grade 3 = moderate bleeding (Buchanan score : from 0 to 5)	At 6 months
Primary	Thrombocytopenia Presence of thrombocytopenia <30 G / L	Presence of thrombocytopenia <30 G / L	At 6 months
Primary	Need of a transfusion ?	Performing a transfusion : yes or no ?	At 6 months
Primary	Immunoglobulin IV Immunoglobulin IV	Immunoglobulin IV push	At 6 months
Primary	Corticoids push	Corticoid push (no reduction of their number during treatment follow-up versus non-treatment period)	At 6 months
Primary	Need a splenectomy ?	Performing a splenectomy : yes or no ?	At 6 months
Primary	Clinical signs of anemia	Presence of clinical signs of anemia = grade 2 = Subject bedridden less than 50% of the day (WHO score : from 0 to 4)	At 6 months
Primary	Anemia	Presence of anemia <7g / dl; Presence of anemia <7g / dl	At 6 months
Primary	Introduction of a new immunomodulatory treatment	Introduction of a new immunomodulatory treatment : yes or no ?	At 6 months
Primary	Stop treatment or not ?	Stop treatment because of failure, intolerance or non-compliance	At 6 months