Personalized Spacing of Eculizumab Infusions Based on Therapeutic Pharmacological Monitoring: Prospective Health-economic Evaluation in Patients in Remission With Atypical Hemolytic Uraemic Syndrome Requiring Long-term Treatment.
Eculizumab is an anti-C5 monoclonal antibody approved for rare diseases including atypical haemolytic-uraemic syndrome. The maintenance phase dosing regimen is identical for all adult patients but several studies have shown a high interindividual kinetics variability. A tailored administration of eculizumab based on therapeutic drug monitoring will be compared with real-life administration in adults suffering from an atypical haemolytic uraemic syndrome. The objective is to improve efficiency of eculizumab administration.
• Adults with an atypical Hemolytic and Uremic Syndrome defined by at least 2 of the following parameters:
‣ Thrombopenia (platelet count \< 150 G/L)
⁃ Mechanical lytic anemia (Hb \< 10 g/L, Lactate dehydrogenase \> upper limit of normal, undetectable haptoglobin, presence of schistocytes on blood smear)
⁃ Acute renal failure
• Eculizumab treatment
‣ Without project of withdrawal in the next 18 months
⁃ Started since at least 6 months
⁃ Administrated with an unchanged administration schedule since at least 3 months (initial episode or relapse)
• Atypical Hemolytic and Uremic Syndrome in remission
• Patients who give informed consent.