Adult patients with aHUS associated with HE and severe kidney involvement (needing dialysis or serum creatinine ≥ 354μM)
Conditions
Brief summary
Response to therapy, defined by the absence of any of the following events: i) at 6-month follow-up: persistent renal replacement therapy, eGFR<15ml/mn/1,73m2, or patient death; ii) at W2 : persistent hemolysis despite well conducted antihypertensive therapy. Any Eculizumab rescue in the control group will be considered as a failure.
Detailed description
Prognosis role of repeat complement biomarkers (W1, W4, W13), Frequency of Complement genetic rare variants, Renal replacement therapy rates at months 3 and 12 follow-up, Frequency of severe infections (defined by the need for hospitalization), Time to resolution of haemolysis, Time to resolution of renal replacement therapy, Frequency and prognostic role of kidney lesions (glomerular and arteriolar microthromboses), glomerulosclerosis and kidney fibrosis if kidney biopsy is performed, Costs relating to renal replacement therapy (or lack of), Eculizumab and other antihypertensive treatments, hospitalizations
Interventions
DRUGECULIZUMAB
Sponsors
Assistance Publique Hopitaux De Paris
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Response to therapy, defined by the absence of any of the following events: i) at 6-month follow-up: persistent renal replacement therapy, eGFR<15ml/mn/1,73m2, or patient death; ii) at W2 : persistent hemolysis despite well conducted antihypertensive therapy. Any Eculizumab rescue in the control group will be considered as a failure. | — |
Secondary
| Measure | Time frame |
|---|---|
| Prognosis role of repeat complement biomarkers (W1, W4, W13), Frequency of Complement genetic rare variants, Renal replacement therapy rates at months 3 and 12 follow-up, Frequency of severe infections (defined by the need for hospitalization), Time to resolution of haemolysis, Time to resolution of renal replacement therapy, Frequency and prognostic role of kidney lesions (glomerular and arteriolar microthromboses), glomerulosclerosis and kidney fibrosis if kidney biopsy is performed, Costs relating to renal replacement therapy (or lack of), Eculizumab and other antihypertensive treatments, hospitalizations | — |
Countries
France
Outcome results
None listed