CompletedNot Applicable

Autoimmune Cytopenia: Genetics and Pathophysiological Mechanism in Pediatric Evans Syndrome

France374 participantsStarted 2019-07-05

Plain-language summary

Characterization of the genetic causes, and of the immunopathological clinical and biological manifestations in children with pediatric Evans syndrome included in a prospective national observational cohort of rare diseases.

Who can participate

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 registered in the French national prospective OBS'CEREVANCE cohort * Diagnosis of pediatric Evans syndrome (PTI+AHAI) * Age strictly under 18 years at the initial onset * Child residing in metropolitan France and affiliated to a french health insurance system * Free, informed, written and signed consent Exclusion Criteria: * Evans syndrome secondary to chemotherapy, bone marrow transplantation or organ transplantation. * Refusal to participate from parents/patients

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.

1Since this study has already been completed, would my child's case be eligible to have genetic samples reviewed as part of the CEREVANCE cohort, or is there a follow-up study we could participate in?
2This trial focused on finding causal genetic mutations in children with Evans Syndrome — has genetic testing already become a standard part of how you evaluate and diagnose my child's condition, based on what studies like this have found?
3Since this was an observational study rather than a treatment trial, what does the research on genetic mutations in pediatric Evans Syndrome actually mean for how we might approach my child's treatment going forward?
4Evans Syndrome can be linked to broader immune system disorders — did studies like this one change how doctors look for underlying conditions, and should we be testing my child for any of those?
5If a genetic cause is identified in my child through testing informed by research like this, how would that change the treatment options or long-term monitoring plan you'd recommend?

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

Number of patients for whom a causal mutation has been identified (known or new)

Timeframe: after the genetic analyzes carried out on all the participants included, may 2022

The number of biological samples collected for PSE children included in the OBS'CEREVANCE cohort and their relatives will be recorded

Timeframe: every 3 months, between may 2019 and may 2022

Trial details

NCT IDNCT03912129

SponsorUniversity Hospital, Bordeaux

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2023-07-09

View on ClinicalTrials.gov More Evans Syndrome trials