The record Primary purpose is to assess the efficacy of eculizumab in adult patients with Atypical Hemolytic- Uremic Syndrome (aHUS) to control Thrombotic Microangiopathy (TMA) as characterized by thrombocytopenia, hemolysis and renal impairment.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
44
All patients received open-label eculizumab administered intravenously on the following dose schedule: Induction dose - 900 mg per week for four weeks and a dose of 1200 mg one week later; Maintenance dose - 1200 mg every two weeks. Patients who received plasma exchange or infusion during the eculizumab treatment period received a supplemental dose of 600 mg within one hour before plasma infusion or within one hour after the completion of each plasma exchange.
Unnamed facility
Burlington, Massachusetts, United States
Unnamed facility
Hackensack, New Jersey, United States
Unnamed facility
Percentage of Patients With Complete TMA Response
Proportion of Patients with Complete TMA response was determined and defined by normalization of hematological parameters (platelet count and LDH) and preservation of renal function (defined as \< 25% increase in serum creatinine from baseline) which were sustained for at least two consecutive measurements obtained at least four weeks apart.
Time frame: Through 26 weeks
Percentage of Patients With Modified Complete TMA Response
Proportion of Patients with Modified Complete TMA response through 26 weeks of treatment was determined and defined by normalization of hematological parameters (platelet count and LDH) and improvement in renal function (defined as ≥ 25% reduction from the baseline value in serum creatinine, which were sustained for at least two consecutive measurements obtained at least four weeks apart.
Time frame: Through 26 weeks
Percentage of Patients With Complete Hematologic Response
Proportion of Patients with Complete Hematologic response through end of study was determined and defined by normalization of platelet count and LDH sustained for at least two consecutive measurements obtained at least four weeks apart.
Time frame: Through 26 weeks
Percentage of Patients With Platelet Count Normalization
Proportion of Patients with Platelet Count Normalization through 26 weeks of treatment was determined and defined as the platelet count observed to be ≥ 150 x 109/L on at least two consecutive measurements which span a period of at least four weeks
Time frame: Through 26 weeks
Percentage of Patients With Estimated Glomerular Filtration Rate (eGFR) Improvement
Proportion of Patients with Estimated Glomerular Filtration Rate (eGFR) Improvement was determined and defined as an increase in eGFR by ≥ 15 mL/min/1.73m\^2 from baseline, sustained for at least two consecutive measurements obtained at least four weeks apart.
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
New York, New York, United States
Unnamed facility
Columbus, Ohio, United States
Unnamed facility
Houston, Texas, United States
Unnamed facility
Liège, Belgium
Unnamed facility
Caen, France
Unnamed facility
Lille, France
Unnamed facility
Nantes, France
Unnamed facility
Nice, France
...and 13 more locations
Time frame: Through 26 weeks
Platelet Count Change From Baseline to 26 Weeks
Time frame: Through 26 weeks
Percentage of Patients With Complete TMA Response
Proportion of Patients with Complete TMA response through end of study was determined and defined by normalization of hematological parameters (platelet count and LDH) and preservation of renal function (defined as \< 25% increase in serum creatinine from baseline) which were sustained for at least two consecutive measurements obtained at least four weeks apart.
Time frame: Through End of Study, Median Exposure 52 Weeks
Percentage of Patients With Modified Complete TMA Response
Proportion of Patients with Modified Complete TMA response through end of study was determined and defined by normalization of hematological parameters (platelet count and LDH) and improvement in renal function (defined as ≥ 25% reduction from the baseline value in serum creatinine, which were sustained for at least two consecutive measurements obtained at least four weeks apart.
Time frame: Through End of Study, Median Exposure 52 Weeks
Percentage of Patients With Complete Hematologic Response
Proportion of Patients with Complete Hematologic response through end of study of treatment was determined and defined by normalization of platelet count and LDH sustained for at least two consecutive measurements obtained at least four weeks apart.
Time frame: Through End of Study, Median Exposure 52 Weeks
Percentage of Patients With Platelet Count Normalization
Proportion of Patients with Platelet Count Normalization through end of study of treatment was determined and defined as the platelet count observed to be ≥ 150 x 109/L on at least two consecutive measurements which span a period of at least four weeks
Time frame: Through End of Study, Median Exposure 52 Weeks
Percentage of Patients With Estimated Glomerular Filtration Rate (eGFR) Improvement
Proportion of Patients with Estimated Glomerular Filtration Rate (eGFR) Improvement was determined and defined as an increase in eGFR by ≥ 15 mL/min/1.73m\^2 from baseline sustained for at least two consecutive measurements obtained at least four weeks apart
Time frame: Through End of Study, Median Exposure 52 Weeks
Platelet Count Change From Baseline to 52 Weeks
Time frame: Through 52 Weeks