CompletedNot Applicable

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

454 participantsStarted 2016-01-18

Plain-language 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.

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Haemorrhagic sign - Grade ≥ 3 (Buchanan score) Presence of a haemorrhagic sign ≥grade 3

Timeframe: At 6 months

Thrombocytopenia Presence of thrombocytopenia <30 G / L

Timeframe: At 6 months

Need of a transfusion ?

Timeframe: At 6 months

Immunoglobulin IV Immunoglobulin IV

Timeframe: At 6 months

Corticoids push

Timeframe: At 6 months

Need a splenectomy ?

Timeframe: At 6 months

Clinical signs of anemia

Timeframe: At 6 months

Anemia

Timeframe: At 6 months

Trial details

NCT IDNCT04057703

SponsorUniversity Hospital, Bordeaux

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2016-12-04

View on ClinicalTrials.gov More Immune Thrombocytopenic Purpura trials

Plain-language summary

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Haemorrhagic sign - Grade ≥ 3 (Buchanan score) Presence of a haemorrhagic sign ≥grade 3

Timeframe: At 6 months

Thrombocytopenia Presence of thrombocytopenia <30 G / L

Timeframe: At 6 months

Need of a transfusion ?

Timeframe: At 6 months

Immunoglobulin IV Immunoglobulin IV

Timeframe: At 6 months

Corticoids push

Timeframe: At 6 months

Need a splenectomy ?

Timeframe: At 6 months

Clinical signs of anemia

Timeframe: At 6 months

Anemia

Timeframe: At 6 months