Long-term Soliris safe, effective in PNH: Real-world Korean study

Treatment reduced symptom severity in patients with high disease burden

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by Steve Bryson PhD |

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A person sits on a hospital bed whil receiving an intravenous infusion, which hangs on a pole nearby.

Long-term treatment with Soliris (eculizumab) safely and effectively reduced the severity of paroxysmal nocturnal hemoglobinuria (PNH) in Korean adults with high disease activity, according to a real-world study.

Soliris was found to ease PNH symptoms, including fatigue, anemia — low levels of hemoglobin, the protein in red blood cells that carries oxygen — and shortness of breath. It also reduced PNH-related complications such as kidney failure, muscle spasms, and blood clot-related events, data showed.

The study spanned more than a decade; Soliris was tolerated well, and no adverse events led to treatment discontinuation during that time.

“These data provided evidence for the long-term efficacy and safety of [Soliris] in Korean PNH patients with high disease burdens,” the researchers wrote.

The study, “Long-Term Efficacy and Safety of Eculizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria and High Disease Burden: Real-World Data From Korea,” was published in the Journal of Korean Medical Science.

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Soliris prescribed in Korea since 2009 for patients with severe PNH

PNH is a rare disease characterized by overactivation of the body’s immune complement cascade. It ultimately leads to the destruction of red blood cells, called hemolysis.

The disease also is marked by activation of platelets — cell fragments involved in blood clotting — which contribute to an increased risk of blood clot-related, or thromboembolic, events.

An antibody-based therapy, Soliris works by suppressing the complement cascade, and was the first disease-specific treatment approved for PNH. Administered via intravenous or into-the-vein infusions, the therapy has been shown to ease PNH symptoms and related complications, and to extend survival.

Soliris “was introduced in Korea in 2009 and has been the standard treatment option for PNH,” the researchers wrote.

In the country, Soliris reimbursement is limited to patients with high disease burden, including signs of hemolysis and at least one PNH-related complication.

Now, researchers from across Korea conducted a real-world study to evaluate Soliris’ long-term safety and efficacy in PNH patients with a high disease burden and severe disease-related complications.

The team looked back at data from 80 adults with PNH, who started Soliris treatment from 2009 to 2020. Their median age was 51.5, and 62.5% of participants were men.

Kidney failure was the most common PNH-related complication leading to Soliris use, followed by muscle spasms, thromboembolic events, and pulmonary hypertension, or high blood pressure in the blood vessels that supply the lungs.

Median treatment duration was about four years, ranging from one month to about 10 years. More than two-thirds of patients (67.5%) continued Soliris treatment, while 32.5% discontinued, primarily due to participation in clinical trials.

“There were no cases of treatment discontinuation due to inadequate responses or loss of responses to [Soliris] in this study,” the team wrote.

Blood tests revealed that Soliris led to a 6.5 times greater mean decrease in the levels of lactate dehydrogenase, or LDH, a hemolysis marker. LDH levels fell below 1.5 times the upper limit of normal in nearly all patients (95%) and were normalized in more than half (61.3%).

Treatment also lessened anemia, as indicated by a significant increase in the mean level of hemoglobin (9.5 to 10.4 g per deciliter), and reduced the number of patients needing red blood cell transfusions, from 75 to 52.

In addition to anemia, Soliris reduced other clinical symptoms of PNH, including fatigue and shortness of breath. It also successfully resolved thromboembolic episodes in 70% of the 20 patients experiencing such events.

The researchers noted all cases of deep vein thrombosis — marked by a blood clot within the deep veins, usually in the leg — were resolved, while other thromboembolic events became less frequent after Soliris treatment. Also, six patients were able to discontinue anti-blood clotting therapy without any new thromboembolic events.

There were no cases of treatment discontinuation due to inadequate responses or loss of responses to [Soliris] in this study.

Muscle spasms were eliminated in all but one patient with this complication (95.8%), while pulmonary hypertension was resolved in four of 15 patients (26.7%). A total of 16 of 36 patients (44.4%) no longer had kidney failure.

According to clinician-based assessments, 77 patients (96.3%) were considered to have improved with Soliris.

In addition, 96.2% of patients were alive after five years of Soliris treatment.

The therapy generally was well-tolerated, with no treatment discontinuations due to adverse events. Infection was reported in 22.5% of patients, none of which was deemed related to Soliris, according to the researchers.

Two deaths occurred within three months after Soliris discontinuation: one due to bladder cancer and one associated with a severe fungal infection. Neithere were linked to the therapy.

Although an increased risk of life-threatening meningococcal infections has been reported in Soliris-treated patients, “no such infections were observed in this study,” even without use of preventive antibiotics, the team wrote.

“Collectively, the data confer the long-term efficacy of eculizumab in patients with PNH who have high disease activity,” the researchers wrote.