Soliris may help relieve symptoms during acute NMOSD attacks
Effective for 5 patients who failed to respond well to standard treatments
Soliris (eculizumab) may help relieve symptoms during severe attacks in the acute phase of neuromyelitis optica spectrum disorder (NMOSD) when other treatments are ineffective, according to a case series study from Japan.
The study included five patients who had experienced severe NMOSD attacks that did not respond well to standard treatments, including intravenous (into-the-vein) steroids and plasma exchange therapy, a blood-cleaning procedure. Soliris was administered about one to two months following the onset of attacks, partially easing symptoms in all patients.
“[Soliris] might be a treatment option to ameliorate the symptoms of severe NMOSD attacks if disability improvement is insufficient or disease activity fluctuates, even after [steroid and plasma exchange] treatment,” researchers wrote.
The study, “The Efficacy of Eculizumab in the Acute Phase of Neuromyelitis Optica Spectrum Disorder: A Case Series Study,” was published in Cureus.
NMOSD is a rare autoimmune disorder that primarily affects the nervous system, including the spinal cord and optic nerves. It is characterized by relapsing inflammatory attacks, which, if not effectively treated, can cause permanent damage and lead to severe disability over time.
The disorder is typically driven by self-reactive antibodies against the water channel protein aquaporin-4 (AQP4) found on the surface of nerve-supporting cells called astrocytes, and by activation of the complement system, a component of the body’s immune system.
Soliris designed to block complement activation
Soliris is an antibody-based therapy designed to block complement activation. By inhibiting complement activity, it helps reduce inflammation and prevents further injury to the nervous system, decreasing the severity of NMOSD attacks and the risk of relapses. Since 2019, Soliris has been approved in the U.S. for adults with NMOSD who test positive for anti-AQP4 antibodies.
Although Soliris is expected to be an effective treatment during the acute phases of the disease, “clinical reports on the efficacy of [Soliris] in the acute phase of NMOSD are limited,” the researchers wrote.
In the report, researchers described the cases of five patients to whom Soliris was administered shortly after they received treatment for severe attacks, partially easing symptoms in all cases.
The five patients in the study, four women and one man, ranged in age from 50 to 93 years. All tested positive for anti-AQP4 antibodies.
The first case, a 50-year-old woman with severe spinal cord inflammation, experienced significant disability, including sensory loss and partial paralysis. Soliris was administered after several courses of intravenous methylprednisolone (IVMP) and plasma exchange failed to ease her symptoms. Her condition improved thereafter, and she was discharged from the hospital after 47 days.
Another patient, a 93-year-old man with vision problems caused by optic neuritis, was given Soliris after IVMP and plasma exchange failed to relieve his condition. Although his vision partially improved, he discontinued Soliris due to difficulties in attending regular hospital visits. The immunosuppressant tacrolimus was then prescribed to prevent future relapses.
The third case was a 54-year-old woman who had severe visual impairment due to optic neuritis in her right eye. Despite receiving multiple rounds of plasma exchange and IVMP, her vision did not improve significantly. Soliris was started 42 days after the attack, and the woman’s visual acuity improved mildly. She was discharged from the hospital after 20 days and continued treatment with Soliris in an outpatient clinic.
Soliris led to gradual improvements in visual acuity
The fourth patient, a 78-year-old woman with a 27-year history of spinal cord inflammation and optic neuritis, was severely disabled and wheelchair-bound. Soliris was administered 30 days after she experienced a relapse, leading to gradual improvements in her visual acuity. However, she discontinued Soliris after five infusions due to difficulty in attending regular follow-ups. She was also prescribed tacrolimus to prevent future relapses.
The final case was a 53-year-old woman with a 10-year history of NMOSD. Her symptoms included dizziness, double vision, and muscle weakness. After standard therapies provided only partial relief, she started on Soliris, which improved her double vision and other symptoms.
The researchers noted all five patients tolerated Soliris well, even when combined with other treatments. No adverse effects were reported during the study.
While the study emphasized Soliris’ potential benefits, it also acknowledged the need for more extensive studies to confirm its effectiveness.
“We cannot exclude that the improved symptoms were related to the delayed effects of IVMP and [plasma exchange] use before [Soliris] administration,” they wrote. “Therefore, the exact effect of [Soliris] during the acute phase should be confirmed in a case-control study with a large sample size or a randomized controlled trial.”
