Personalized Soliris could lead to better outcomes, cost savings: Case report
Doses lowered for man, 60, with aHUS based on his clinical improvements
A 60-year-old man with atypical hemolytic uremic syndrome (aHUS) was successfully treated with Soliris (eculizumab) using a personalized dosing regimen based on clinical responses, according to a recent case study.
Clinicians lowered the dose of the medication in parallel with clinical improvements and eventually discontinued treatment instead of maintaining the recommended 1,200 mg maintenance dose every other week.
“This approach not only addresses the clinical needs but also aligns with the economic and safety concerns, ultimately leading to a more tailored and effective treatment strategy,” researchers wrote.
The case study, “Individualised therapeutic approach to the patient with atypical haemolytic-uraemic syndrome,” was published in the journal Clinical Medicine.
aHUS is a type of thrombotic microangiopathy (TMA), a group of diseases marked by the formation of blood clots in small blood vessels. Blood clots form in aHUS due to the abnormal activity of the complement cascade, a part of the immune system that normally helps clear microbes from the body. These clots may cause damage to organs, especially the kidneys, leading to symptoms of acute kidney failure.
Early treatment with Soliris can improve outcomes, prevent kidney failure
Early treatment with Soliris, an approved antibody-based therapy designed to block complement activation, is essential to improve outcomes and prevent kidney failure. Still, lifelong treatment with Soliris is associated with an increased risk of infections and comes at a high cost.
Researchers in Croatia hypothesized that “a personalised [Soliris] dosing strategy tailored to individual patient responses could optimise therapy, reduce costs and improve safety.”
To support the feasibility of personalized dosing strategies, the team described the case of an aHUS patient in whom Soliris treatment was administered based on clinical response.
A 60-year-old man with a history of type 2 diabetes and controlled high blood pressure was admitted to the urology department five days after a prostate biopsy. He had fever, lethargy, and jaundice, or yellowing of the skin and eyes. Blood tests revealed altered levels of white blood cells and platelets, and markers consistent with inflammation and poor kidney function.
When his kidneys stopped producing urine, he began dialysis and was moved to the intensive care unit, where he was placed on a mechanical ventilator for breathing support. At this point, he was unconscious, with further blood tests suggesting TMA. Additional tests ruled out other types of TMA, ultimately supporting the diagnosis of aHUS.
On day 50 of his hospital stay, after recovering from an infection, he was placed on preventive antibiotics and vaccinated against Neisseria meningitidis, a bacteria that can cause a serious life-threatening infection that has been reported in patients treated with complement inhibitors.
After four standard 900 mg doses of Soliris given in seven-day intervals, the patient regained consciousness on day 68 (about two months and one week). While he still needed transfusions, signs of red blood cell destruction diminished. The first two maintenance doses (1,200 mg) of Soliris were administered two weeks apart, and by day 100 (about three months and 10 days), he improved further, showing no signs of active TMA. He still required dialysis twice weekly, however, due to chronic kidney failure.
Soliris doses gradually reduced based on patient’s clinical condition
He was kept on Soliris maintenance treatment, but the doses were gradually reduced, from two 900 mg doses to two 600 mg doses, and then to two 300 mg doses, all given two weeks apart. His condition consistently improved in parallel, platelet levels normalized, and there were no signs of TMA. He received his final dose on day 163 (about five months and two weeks).
Genetic testing was conducted to look for the presence of mutations associated with aHUS, but results were inconclusive.
After 209 days (about seven months) in the intensive care unit, he was discharged from the hospital and continued dialysis three times per week. Two years later, he was generally in good condition, and showed no signs of TMA. He continued dialysis while waiting for a kidney transplant.
“Considering the complexity of aHUS biology, it seems that an individualised approach to treatment, monitoring and follow-up would be necessary to achieve better outcomes,” the researchers wrote. This would help “keep otherwise considerable treatment costs of lifetime therapy at a reasonable level.”
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