Myasthenia gravis is a rare immune-mediated disorder (affecting less than 100,000 patients in the US) in which the immune system mistakenly targets nerves or muscles. Historically, myasthenia gravis patients faced early mortality but advances in treatment and the introduction of steroid-sparing agents have significantly improved patient outcomes. Today, most patients have a normal life span, underlining the progress made in managing this condition. Despite this, steroids are still widely used as a first-line treatment, and most patients receive steroid treatment at some point.
Neelam Goyal, MD, a Clinical Associate Professor in Neurology and Neurological Sciences at Stanford, has been leading a pioneering, retrospective, real-world study exploring the use of the Steritas GTI-MD in myasthenia gravis (MG).[1] The study has made the pages of MedScape for all the right reasons.[2]
Earlier this year, Dr Goyal told us how she was inspired by a colleague, early on in her career, to be a “patient-loving” clinician rather than a “disease-hating” one. That approach to treating patients has led Dr Goyal to want to get a better understanding of the impact steroids and steroid-toxicity have on her patients with myasthenia gravis:
“As I started becoming aware of the impact of steroid-toxicity on MY patients, I also became aware of the lack of literature and understanding about how steroids and steroid-toxicity impact ALL patients. This is perhaps surprising as a majority of patients with myasthenia gravis will receive glucocorticoids as a first line of treatment.”
Dr Goyal’s study was conducted using records from the US-based Optum® electronic health record (EHR) database of myasthenia gravis patients with all the required lab values for the GTI-MD instrument (four domains).
Results presented recently at AANEM 2024 demonstrated the effectiveness of the GTI-MD in assessing steroid-toxicity in myasthenia gravis patients. The key findings were:
The results demonstrated that the GTI-MD is sensitive and effective at retrospectively detecting steroid-toxicity from EHR data and highlighted potential utility in clinical settings.
Dr Goyal’s team is also conducting a prospective study exploring the ability of the GTI instrument to track steroid-toxicity in 50 patients with myasthenia gravis. The aim of the study is to evaluate how sensitive patient steroid-toxicity (as measured by the GTI score) is to changes in prednisone dosage and how well steroid-toxicity scores correlate with MG outcomes assessed with the following validated scales:
While still ongoing, some key insights are already coming from the study:
This predictive ability could also be used to support the case for prescribing costly steroid-sparing medications by justifying their need based on a high toxicity score.
Taken together, these two argenx-funded studies highlight the utility of the STOX® Suite in quantifying steroid-toxicity and its changes over time in both large cohort studies and the clinical setting. The strong correlation reported between GTI scores, steroid dosage (cumulative and current), and patient outcomes highlights the potential use of the GTI to help refine treatment strategies. This could include more precise tapering of prednisone and earlier use of alternative therapies to reduce side effects and improve outcomes. The GTI and GTI-MD could help clinicians manage the dual burden of disease activity and steroid-related side effects more effectively, particularly in complex cases where tapering steroids risks flaring the disease.