The registry aims to enroll approximately 800 patients with NMOSD to evaluate the efficacy of new medications and treatments.
The first patients have been enrolled in the Synergy of Prospective Health & Experimental Research for Emerging Solutions (SPHERES) in neuromyelitis optica spectrum disorder (NMOSD) Registry, CorEvitas and The Guthy-Jackson Charitable Foundation (GJCF) announced earlier this year.1
The prospective, observational research study aims to enroll 800 patients with NMOSD through academic and private neurology practices, with a goal of improving patient outcomes by evaluating the safety and efficacy of existing treatments and recently approved medications for the disease. Investigators will log and evaluate relapses, prevalent and incident comorbidities and adverse events, and medication use patterns, while also collecting standardized patient-reported outcomes and clinician-reported outcomes.
“First patient enrollment is a milestone achievement in the SPHERES study to understand and improve clinical outcomes in the new era of approved NMOSD therapeutics,” Michael Yeaman, PhD, professor of medicine, David Geffen School of Medicine, UCLA, and chair medical advisor, GJCF, said in a statement.1 “SPHERES will also facilitate quantum leaps in the field, including the GJCF Relapse Navigator to standardize relapse adjudication and severity. In turn, these advances will create new opportunities to discover and apply clinical, therapeutic, and laboratory correlates in NMOSD to promote remission and improve patient lives.”
The SPHERES registry and data will be overseen and analyzed by CorEvitas experts in clinical research, epidemiology, and biostatistics, who will work alongside a scientific steering committee comprised of advisors from the GJCF. This effort aims to further expand previous information from the Collaborative International Research in Clinical and Longitudinal Experience for NMOSD Studies (CIRCLES) Registry.2
Originally announced in March of 2021, the SPHERES Registry is further supported by Genentech (of the Roche group) and Horizon Therapeutics. According to a statement from both organizations, any sites interested in participating can e-mail firstname.lastname@example.org for more information.
“The SPHERES Registry for NMOSD is a collaboration between CorEvitas, the leading sponsor of registries in autoimmune and inflammatory diseases and the GJCF. The SPHERES Registry is designed to study the real-world effectiveness and safety of treatments with the goal of enhancing patient outcomes in NMOSD,” CorEvitas and the GJCF told NeurologyLive in a statement. “CorEvitas is actively recruiting neurology practices to join the site network and enrollment is open to eligible patients. Sites and patients are compensated for completing questionnaires approximately every 6 months.”
Patients with NMOSD have seen FDA approvals for 3 therapeutic treatment options in recent years: eculizumab (Soliris; Alexion) in June 2019, inebilizumab (Uplinza; Horizon) in June 2020, and satralizumab (Enspryng; Genentech) in August 2020.
Experts in the field are working towards educating this patient and provider population on the new agents, as both satralizumab, an interleukin-6 receptor blocker, and inebilizumab, a B-cell depleting antibody therapy, were launched during the height of the COVID-19 pandemic, and eculizumab, a terminal complement inhibitor, was available for a limited time prior to its inception. Each treatment options have different mechanisms of action and administration for aquaporin-4 antibody (AQP4)-positive patients, as well as high rates of efficacy.
Experts have also recently commented on the importance of early diagnosis and treatment for those with NMOSD—particularly for those with optic neuritis—in order to improve patient outcomes. According to Melanie Truong-Le, DO, OD, neuro-ophthalmologist, Peter O’Donnell Jr. Brain Institute, UT Southwestern Medical Center, identification of AQP4 has helped to “drive care” within the neuro-ophthalmology space.
“It's so important to recognize the condition really early on,” Truong-Le told NeurologyLive. “Very much like in neurology and stroke, we find that time is brain, and time is vision, as well. So, if we were able to see these patients early on, identify them, and treat them, the outcome is so much better.”