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Eculizumab inhibited thrombotic microangiopathy in hemolytic-uremic syndrome
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Eculizumab inhibited complement-mediated thrombotic microangiopathy and led to significant improvements in renal function in patients aged 12 years and older with atypical hemolytic-uremic syndrome, according to results of two phase 2 trials.
The prospective, open-label trials included a combined 37 patients.
The first trial included 17 patients with low platelet counts and renal damage. The second trial included 20 patients with renal damage but no decrease in platelet counts of more than 25% for at least 2 months during infusion.
All patients received 900 mg eculizumab (Soliris, Alexion) intravenously per week for 4 weeks, a 1,200 mg dose 1 week later, and a maintenance dose of 1,200 mg every 2 weeks. Patients who received plasma exchange or infusion during the treatment period received a 600 mg supplemental dose before plasma infusion or within 1 hour after the completion of each plasma exchange.
The primary outcome measure of the first trial was change in platelet count.
Median treatment was 64 weeks. Results showed eculizumab was associated with increased platelet counts from baseline to week 26 (P<.001).
Dialysis was discontinued in four out of five patients who required dialysis at the time of eculizumab initiation. Those patients remained dialysis free for the duration of eculizumab treatment, researchers said.
The primary outcome of the second trial was thrombotic microangiopathy event-free status, defined as no decrease in platelet counts of more than 25%, no plasma exchange and no dialysis.
Median treatment was 62 weeks. Results showed 80% of patients had thrombotic microangiopathy event-free status.
Overall, eculizumab was associated with continuous, time-dependent increases in estimated glomerular filtration rate (GFR). Earlier treatment led to significant improvements in estimated GFR. Eculizumab also was associated with improvements in health-related quality of life, and no serious adverse events were observed throughout the trial and extension periods.
“These two clinical studies suggest that long-term eculizumab treatment is effective in patients with atypical hemolytic–uremic syndrome, with earlier intervention associated with a greater clinical benefit,” the researchers wrote. “The data indicate that terminal complement inhibition with eculizumab inhibits complement-mediated thrombotic microangiopathy, decreases the need for thrombotic microangiopathy-related intervention, significantly improves the platelet count and renal function across patient groups, and is associated with substantial kidney recovery and improved clinical outcomes in patients with atypical hemolytic-uremic syndrome.”
Disclosure: The research was supported by Alexion Parmaceuticals.
Perspective
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Harry S. Jacob, MD, FRCPath(Hon)
Another example of an out-of-sight cost of a medication. This rare syndrome is also treatable by simple plasma infusion that, to be sure, may intermittently need to be repeated in order to bolster a usually congenital deficient complement-inhibitory protein. Thus, economically stressed patients may not routinely need this wildly expensive material.
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