Amazing news! Emapalumab can help treat Macrophage Activation Syndrome (MAS)!
The brand name for emapalumab is Gamifant. It’s FDA-approved to treat hemophagocytic lymphohistiocytosis (HLH), which is pretty similar to MAS in many respects.
The following paragraph is from a summary on Contemporary Pediatrics:
Emapalumab, a fully human anti-interferon-γ (IFNγ), demonstrated efficacy for inducing remission of macrophage activation syndrome (MAS) secondary to systemic juvenile idiopathic arthritis (sJIA) or adult-onset Still’s disease (AOSD) in patients who failed standard care with high-dose glucocorticoids, with or without anakinra (Kineret; Sobi) and/or cyclosporin, according to a recent study.
This is amazing news. MAS is a terrifying secondary condition. It is most common in SJIA and AOSD, but can pop up in lupus and other conditions. If you want to learn more about MAS itself, I covered a session on the condition at the 2017 ACR annual meeting. You can learn more about the immunology on Frontiers.
Normal treatment involves a ton of gluticosteroids and often Anakinra/Kineret. That doesn’t do it for enough patients, though. It’s important to note that there was a small N (or participant number) of 14. However, by week 8, 13 of the 14 patients had achieved remission. That’s not shabby at all.
Most importantly, no deaths were reported during the trial and the long-term follow-up. That’s amazing for a condition that has a mortality rate between 20-53%.
This was a phase II, open-label, single-arm trial conducted in the US, UK, Spain, Italy, and France. I was also happy to see that Dr. Alexei Grom, who I interviewed several years ago, was a part of this study.
Read more of the summary on Contemporary Pediatrics. Or, you can read the full journal article, Efficacy and safety of emapalumab in macrophage activation syndrome, here.