International Clinical Trial Results in Promising New Treatment Option for Refractory Polymyalgia Rheumatica Patients
HSS rheumatologist plays a leadership role in pivotal clinical trial
(New York, New York) – The New England Journal of Medicine published positive results from a Phase 3 international clinical trial demonstrating the efficacy of sarilumab (Kevzara®) in adult patients with refractory polymyalgia rheumatica (PMR). Robert Spiera, MD, rheumatologist and Director of the Scleroderma, Vasculitis and Myositis Center at Hospital for Special Surgery (HSS), served as primary investigator for the trial, which led to the U.S. Food and Drug Administration (FDA) approval of a new treatment option for patients with PMR who have difficulty tapering off steroids. Steroid related side effects are common in this group of patients and can often result in complications including osteoporosis, diabetes, myopathy, glaucoma, bruising, bleeding and sleep disturbances.
Polymyalgia rheumatica (PMR) among the most common inflammatory musculoskeletal diseases affecting individuals over the age of 50, causes severe pain, stiffness, fatigue, and at times low grade fever. Traditionally, treatment has relied heavily on steroids, leading to prolonged therapy and severe side effects, particularly in older patients. However, the recent trial, led by Dr. Spiera, validates the efficacy of a new treatment option for select patients suffering from the disease.
The trial, sponsored by and supported by Sanofi and Regeneron, focused on a drug called sarilumab, an interleukin-6 receptor blocker, and involved patients with refractory PMR – those who experienced flares despite having been previously treated with a substantial amount of corticosteroids. Patients were randomly assigned to receive either a very rapid 14-week steroid taper with sarilumab or a one-year steroid taper with placebo. The study demonstrated that patients on sarilumab were three times more likely to achieve sustained remission, reduce their overall steroid use, and experience significant improvements in their quality of life compared to the placebo group.
"Prior to the FDA approving sarilumab earlier this year as the first and only biologic to treat select patients with PMR, patients and physicians were limited in their treatment options,” Dr. Spiera said. “This trial represents a crucial advance in the management of PMR. It offers new hope to those who have struggled with the condition, especially older patients who are particularly susceptible to serious side effects associated with long-term steroid use.”
The approval of sarilumab for refractory PMR patients marks a monumental achievement in the treatment landscape for PMR patients, underscoring HSS’s commitment to providing specialized care and innovative treatments for patients with rheumatic conditions.
Dr. Spiera continued, “Whether there is a role for sarilumab as first line therapy in PMR, particularly in patients in whom steroid complications has not been addressed in the SAPHYR study; hopefully future clinical trials will address that question.”
HSS is the world’s leading academic medical center focused on musculoskeletal health. At its core is Hospital for Special Surgery, nationally ranked No. 1 in orthopedics (for the 14th consecutive year), No. 2 in rheumatology by U.S. News & World Report (2023-2024), and the best pediatric orthopedic hospital in NY, NJ and CT by U.S. News & World Report “Best Children’s Hospitals” list (2023-2024). In a survey of medical professionals in more than 20 countries by Newsweek, HSS is ranked world #1 in orthopedics for a fourth consecutive year (2023). Founded in 1863, the Hospital has the lowest readmission rates in the nation for orthopedics, and among the lowest infection and complication rates. HSS was the first in New York State to receive Magnet Recognition for Excellence in Nursing Service from the American Nurses Credentialing Center five consecutive times. An affiliate of Weill Cornell Medical College, HSS has a main campus in New York City and facilities in New Jersey, Connecticut and in the Long Island and Westchester County regions of New York State, as well as in Florida. In addition to patient care, HSS leads the field in research, innovation and education. The HSS Research Institute comprises 20 laboratories and 300 staff members focused on leading the advancement of musculoskeletal health through prevention of degeneration, tissue repair and tissue regeneration. In addition, more than 200 HSS clinical investigators are working to improve patient outcomes through better ways to prevent, diagnose, and treat orthopedic, rheumatic and musculoskeletal diseases. The HSS Innovation Institute works to realize the potential of new drugs, therapeutics and devices. The HSS Education Institute is a trusted leader in advancing musculoskeletal knowledge and research for physicians, nurses, allied health professionals, academic trainees, and consumers in more than 165 countries. The institution is collaborating with medical centers and other organizations to advance the quality and value of musculoskeletal care and to make world-class HSS care more widely accessible nationally and internationally. www.hss.edu.