Alexei Grom, MD: Exploring Safer Treatment Options for Refractory Macrophage Activation Syndrome

Over 80% of patients with macrophage activation syndrome (MAS) in the NI-0501-06 (NCT03311854) and NI-0501-14 (EMERALD; NCT05001737) studies responded to emapalumab, which neutralized IFNg in all participants.

These participants, who had MAS in Still’s disease who had an inadequate response to high-dose glucocorticoids, were enrolled in the first and only controlled study for MAS evaluating a potential intervention therapy.

Findings from the studies were presented by Alexei Grom, MD, Research Director, Division of Rheumatology, and Professor, Department of Pediatrics, Cincinnati Children’s, at the American College of Rheumatology (ACR) Convergence 2024, held November 14-19 in Washington, DC.

“MAS is a life-threatening complication of many rheumatic diseases, but it's by far most common in the systemic form of juvenile idiopathic arthritis… it's a life threatening complication, and it certainly remains the one of the major sources of mortality in our field, in general. And there is a clear need for new therapeutic targets, as well as new medications to help these kids,” Grom told HCPLive® during the meeting.

In this clip, Grom discussed the study design, outcomes, and current findings, although the study is still ongoing. He emphasized the importance of providing more treatment options for patients with MAS whose disease did not respond to high-dose glucocorticoids instead of therapies that can have serious complications, like etoposide. He also noted that patients whose MAS can be treated with high-dose glucocorticoids may also benefit from emapalumab, as it could allow lower doses of steroids and faster tapering.

Relevant disclosures for Grom include Kiniksa, Novartis, SJIA Foundation, Sobi, and Up-to-Date.

REFERENCE

Grom A, Ullman U, Mahmood A, et al. Efficacy and Safety of Emapalumab in Children and Adults with Macrophage Activation Syndrome (MAS) in Still’s Disease: Results from a Phase 3 Study and a Pooled Analysis of Two Prospective Trials. Presented at: ACR Convergence 2024; November 14-19; Washington, DC. Abstract L19.