Many Outstanding Questions Still Exist for Vasculitis Patients: The Massachusetts General Hospital Rheumatology Vasculitis Program Hopes to Find Some of the Answers

Vasculitis tends to present in a systemic manner, often affecting multiple organ systems. Clinicians should consider a broad differential (possible conditions that share the same symptoms) and think about the entire person—not just at the time of diagnosis but throughout their life as they face periods of remission and relapse. The Massachusetts General Hospital (MGH) Rheumatology Vasculitis Program works in close collaboration with specialists from a variety of areas. Patients can expect to receive expert care from a multidisciplinary team who has advanced training and experience managing patients with different forms of vasculitis.

The Rheumatology Vasculitis Program at MGH sees approximately 100 patients per month including those with newly diagnosed and established vasculitis. Sebastian Unizony, MD, who is co-director of the Rheumatology Vasculitis Program, explains that the program manages the entire spectrum of vasculitis and has four main goals:

• to provide personalized, cutting-edge clinical care for patients with any systemic type of vasculitis
• to conduct high-quality clinical, translational, and basic research that helps advance the frontiers of vasculitis knowledge and ultimately improve patient care
• to educate trainees and peers in diagnosing and treating vasculitis
• to advocate for patients with vasculitis

“We care for patients with any type of vasculitis in one setting and can evaluate and manage patients in Boston as well as Waltham, Massachusetts,” said Zach Wallace, MD, MSc, who is co-director of the Rheumatology Vasculitis Program. “We have strong collaborations, both for patient care and research with our colleagues in nephrology (including those at the Vasculitis & Glomerulonephritis Center at MGH), ENT, neurology, dermatology, vascular medicine, vascular surgery, ophthalmology, pulmonology, cardiology, infectious disease, and allergy.”

To assist with diagnosing, monitoring, and managing patients with vasculitis, the Rheumatology Vasculitis Program also has critical relationships with experts from radiology and pathology who are fundamental to what is done every day at the center. “We’re lucky to be able to sit down with our colleagues to review complex cases regularly and get the input of so many experts at the same place,” Dr. Wallace added.

In addition to a comprehensive clinical care program, the Rheumatology Vasculitis Program is excited to be advancing vasculitis research by taking the lead in clinical trials, observational cohort studies, as well as translational and fundamental basic science research. Currently, there are clinical trials in giant cell arteritis (GCA) and ongoing observational cohort studies in anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis evaluating strategies to improve long-term outcomes. Patients can participate in survey-based research and biobanking studies in which they can provide blood and other specimens.

“We have an ongoing study evaluating the burden of heart disease in patients with ANCA-associated vasculitis, a frequent complication of disease and treatment,” Dr. Wallace said. “We also have a research program dedicated to studying the effects of COVID-19 infections in patients with rheumatic disease, particularly patients with vasculitis.” Additional clinical trials will be opening soon, which include investigating a mind-body intervention to improve quality of life for patients with vasculitis and treatment strategies for ANCA-associated vasculitis. The center prides itself on being a part of a research community that spans from the “bench to bedside to community and population” at MGH, which is the largest hospital-based research enterprise in the United States.

Dr. Unizony added that one of the main research objectives of the Rheumatology Vasculitis Program has been to discover treatments that can help minimize the use of glucocorticoids (e.g., prednisone) in patients with vasculitis. “As such, investigators at the Rheumatology Vasculitis Program have pioneered the use of rituximab for the treatment of ANCA-associated vasculitis and the use of tocilizumab for the treatment of GCA. Studies conducted by investigators of the Rheumatology Vasculitis Program led to the FDA approval of these therapies for these conditions. These treatments are now recommended by the 2021 American College of Rheumatology/Vasculitis Foundation (VF) guidelines for the management of vasculitis.”

“We also have experience managing patients who travel to see us from around the country and the world,” Dr. Wallace explained. “We’re happy to work closely with local clinicians and rheumatologists to assist with the management of patients and serve as a resource for those who may not be able to receive more regular care with us because of where they live.” If needed, the center makes itself available to its patients on an urgent basis and has an outstanding administrative support team that can help patients get access to treatments including prior authorizations of specific medications, arrange referrals, and facilitate care.

The Rheumatology Vasculitis Program is also excited about the opportunity to work with the VF to connect with patients and advocates about vasculitis. “We are planning to host an in-person gathering with patients and advocates in early 2023 so we can introduce ourselves to the broader community, connect patients with other vasculitis experts, patients, and advocates, and share the work we are doing and educate the community on vasculitis.”

The management of vasculitis including the approaches to diagnosis, classification and treatment are constantly evolving and the experts at the Rheumatology Vasculitis Program find that it’s an exciting time to be caring for patients living with this disease. So many outstanding questions still exist for patients with vasculitis but the clinicians at the MGH Rheumatology Vasculitis Program “hope to contribute to finding some of the answers,” Dr. Unizony concluded.

Current Clinical Trials

• A Proof-of-Concept Study of Guselkumab in the Treatment of Subjects with New-onset or Relapsing Giant Cell Arteritis (ClinicalTrials.gov Identifier: NCT04633447). Study to evaluate the efficacy and safety of guselkumab (IL-23 antagonist) subjects with giant cell arteritis
• A randomized, parallel-group, double-blind, placebo-controlled, multicenter phase III trial to investigate the efficacy and safety of secukinumab 300 mg administered subcutaneously versus placebo, in combination with a glucocorticoid taper regimen, in patients with giant cell arteritis (ClinicalTrials.gov Identifier: NCT04930094). Study to evaluate the efficacy and safety of secukinumab (IL-17 antagonist) subjects with giant cell arteritis

Completed clinical trials

• A Randomized, Double-blind, Placebo-controlled Multicenter Study of Tocilizumab (anti IL-6 receptor antibody) for the Treatment of giant cell arteritis (GiACTA) (ClinicalTrials.gov Identifier: NCT01791153).
• A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Sirukumab in the Treatment of Patients with giant cell arteritis (ClinicalTrials.gov Identifier: NCT02531633).
• Ustekinumab for the treatment of Giant Cell Arteritis (ClinicalTrials.gov Identifier: NCT02955147)
• A Phase 2, randomized, double-blind placebo-controlled study to test the efficacy and safety of KPL-301 in giant cell arteritis (ClinicalTrials.gov Identifier: NCT03827018)
• A Phase 3, randomized, double-blind placebo-controlled study to test the efficacy and safety of sarilumab in giant cell arteritis (ClinicalTrials.gov Identifier: NCT03600805)
• A Phase 3, randomized, double-blind placebo-controlled study to test the efficacy and safety of sarilumab in polymyalgia rheumatica (ClinicalTrials.gov Identifier: NCT03600818)
• Tocilizumab plus a short prednisone taper for giant cell arteritis (ClinicalTrials.gov Identifier: NCT03726749).