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

NCT04057703 | Completed

• 1 other identifier: CHU BX 2013/24
• Study Type: observational
• Target: 454
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Immune Thrombocytopenic Purpura; Autoimmune Hemolytic Anemia; Evan Syndrome; Immunotherapy

Keywords

CEREVANCE; Pediatrics; Cohort

Outcome Measures

Primary Outcomes (10)

• 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

• Thrombocytopenia Presence of thrombocytopenia <30 G / L

  Presence of thrombocytopenia \<30 G / L

  At 6 months

• Need of a transfusion ?

  Performing a transfusion : yes or no ?

  At 6 months

• Immunoglobulin IV Immunoglobulin IV

  Immunoglobulin IV push

  At 6 months

• Corticoids push

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

  At 6 months

• Need a splenectomy ?

  Performing a splenectomy : yes or no ?

  At 6 months

• 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

• Anemia

  Presence of anemia \<7g / dl Presence of anemia \<7g / dl

  At 6 months

• Introduction of a new immunomodulatory treatment

  Introduction of a new immunomodulatory treatment : yes or no ?

  At 6 months

• Stop treatment or not ?

  Stop treatment because of failure, intolerance or non-compliance

  At 6 months

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• 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.

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• University Hospital, Bordeaux: lead

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic; Anemia, Hemolytic, Autoimmune; Evans Syndrome

Condition Hierarchy (Ancestors)

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

Study Officials

• Nathalie ALADJIDI, Dr

  University Hospital, Bordeaux

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 12, 2019
• First Posted: August 15, 2019
• Study Start: January 18, 2016
• Primary Completion: December 4, 2016
• Study Completion: December 4, 2016
• Last Updated: August 15, 2019

Record last verified: 2019-08

Data Sharing

• IPD Sharing: Will not share