Therapeutic Orientation Test in Thrombotic Microangiopathy

Study Overview
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Brief Summary
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Haemolytic and Uraemic Syndrome (HUS) is a serious disease requiring rapid diagnosis and management. The atypical HUS diagnosis has been greatly improved by anti-CS antibody (Eculizumab) wich block alternative complement pathway activation. To rise treatment success, Eculizumab introduction should be as early as possible. In some secondary HUS (infection, drugs…) complement is also involved as second-hit. To date, there is no tool to confirm complement involvement in a HUS at diagnosis stage. This study suggest to evaluate a therapeutic orientation test, in order to determine the complement implication in HUS diagnosis. The test evaluates the complement deposits on endothelial cell surface in vitro, compared to a normal human serum. In order to determine the test performance, first the positive or negative results will be compared to the HUS clinical evolution, treated or not by the clinician with Eculizumab. Second, the test results will be compared to the presence of alternative complement pathway regulation abnormalities.

Official Title
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Therapeutic Orientation Test in Thrombotic Microangiopathy

Conditions
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Thrombotic Micro-angiopathy

Intervention / Treatment
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* Diagnostic Test: Therapeutic orientation test for TMA


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: TMA with: mechanic haemolytic anemia, undetectable haptoglobin, LDH>1.5*LNS thrombopenia acute kidney injury TMA on native kidney or in post-transplantation. Exclusion Criteria: DIVC patients plasma exchange during 1 month before sample collection treatment by Eculizumab before sample collection no consent not beneficiary of a social security pregnancy or breastfeeding patient Under guardianship

Ages Eligible for Study
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Maximum Age: 90 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Diagnostic
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Patients with Thrombotic micro-angiopathy<br> | Diagnostic Test: Therapeutic orientation test for TMA<br>* Therapeutic orientation test for TMA performed on blood sample at inclusion, first visit at 1 month and last visit at 6 months.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Therapeutic orientation test sensitivity | The proportion of patients testing positive among those receiving a relevant Eculizumab treatment ( TMA resolution with treatment or presence of abnormalities in alternative complement pathway. | Through study completion, an average of 3 years. | 
| Therapeutic orientation test specificity | The proportion of patients testing negative among patients who did not receive Eculizumab treatment.(TMA resolution without treatment or therapeutic failure with Eculizumab). | Through study completion, an average of 3 years. | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Untreated test positive patients | The proportion of patients with positive test but untreated by Eculizumab, and who therefore could have benefited from treatment. | Through study completion, an average of 3 years. | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Diagnostic, Nephrology, Complement, Thrombotic micro-angiopathy, Haemolytic and Uraemic Syndrome