OBJECTIVES: Myasthenia Gravis (MG) is a progressive neuromuscular disorder that manifests as fatiguable muscle weakness. A proportion of MG patients who are non-responsive to traditional treatments are referred as refractory. With uncontrolled disease activity, these patients experience more severe symptoms, requiring greater medical attention. This study aims to describe healthcare resource utilisation (HCRU) for refractory vs. non-refractory MG patients in England.

METHODS: We used linked primary (Clinical Practice Research Datalink) and secondary care (Hospital Episode Statistics) medical records from 01-April-1997 to 31-December-2016 to identify MG patients. An algorithm, based on current and past treatment, was adapted from a US study (Engel-Nitz, 2018) for the English setting with input from a UK Neurologist to determine refractory status. Rates (total count averaged per-person) of HCRU (inpatient hospitalisations, physician, emergency room (ER), and other outpatient visits) were compared between refractory and non-refractory. The average length of hospital stay (LOS in days) and average intravenous immunoglobin (IVIg) total doses administered (in all settings) per patient for total follow-up were also compared.

RESULTS: Among 1,149 patients with MG, 87 (7.6%) were refractory. Mean age (refractory vs. non-refractory: 59.6 vs 63.9 years, p=0.09) and proportion of females (52.9 vs 46.5, p=0.25) was similar in both groups. Refractory patients had higher rates of overnight hospitalisations (1.6 vs. 0.8 per-person year, p<0.001), physician reviews (36.2 vs. 26.0, p<0.001), ER attendance (0.6 vs. 0.3, p<0.001), and other outpatient visits (7.7 vs. 5.1, p<0.001) when compared to the non-refractory cohort. Mean LOS per patient was higher in refractory patients (57.6 vs. 32.8 days, p<0.001) but the IVIg use (5.0 vs. 2.7, p=0.07) was similar in both groups.

CONCLUSIONS: Refractory patients experience more frequent healthcare interactions and longer durations of hospital stays than non-refractory patients, emphasising the significant unmet need for a therapy to control refractory disease activity.