On 7 April, at the 2025 American Academy of Neurology (AAN) meeting, a poster presentation by the Peking Union Medical Hospital, the Chinese Academy of Medical Sciences, and the Peking Union Medical College presented results from a single-centre cohort study on the long-term outcomes of steroid dosing regimens and withdrawal in myasthenia gravis (MG). The group aimed to investigate the impact of steroid dosing regimens and steroid withdrawal in patients on steroid monotherapy. Corticosteroids are the first-line immunotherapy for MG, but the optimal steroid dosing regimen and effects of discontinuation remain unclear.
The research presented by Yangyu Huang and colleagues was a cohort study based on a single-centre prospective MG registry. There were 209 participants included in the study, each of whom were patients with sustained minimal manifestations (MM) or better on steroid monotherapy. Huang and colleagues used group-based trajectory modelling to identify three distinct steroid dosing regimens: regimen 1 was defined as a high starting steroid dose and a fast taper, regimen 2 was defined as a low starting dose and a slow taper, and regimen 3 was defined as a moderate starting dose and a gradual taper. The outcome measure was the rate of MG relapse, and the group used a Cox proportional hazards (CoxPH) model to assess relapse risk factors. It was found that regimen 1 had the highest risk of relapse, followed by regimen 3 and regimen 2. Furthermore, a maximum dose greater than 0.83mg/kg was associated with an increased risk of relapse. One final statistical analysis used in the study was Propensity Score Matching (PSM) to compare steroid maintenance and withdrawal groups. PSM matched 44 patients in each of the maintenance and withdrawal groups based on age, gender, cumulative steroid dose, and time to taper to first maintenance dose. The CoxPH model showed a significantly higher risk of relapse in the withdrawal group. Based on their findings, the group concluded that patients who required high-dose steroids to achieve sustained MM or better have a significantly higher relapse risk on steroid monotherapy and should receive additional immunotherapy early in treatment. Furthermore, the group found that discontinuing immunotherapy increases relapse risk compared to low-dose maintenance, requiring caution when stopping steroids in monotherapy. The data presented by Huang and colleagues could pose an important step towards standardising steroid dosing regimens and optimising patient outcomes on this form of immunotherapy. Key opinion leaders (KOLs) previously interviewed by leading data and analytics company GlobalData have identified a lack of standardised treatment guidelines as a key unmet need in the MG treatment landscape. Currently, there are many off-label treatments used in MG and as a result, there is a lack of international consensus on treatment regimens, including steroid dosing and tapering protocols, which contributes to suboptimal patient outcomes. Corticosteroid use is widespread and inconsistent practices increase relapse risks and corticosteroid-related comorbidities (eg, diabetes and osteoporosis). Moreover, KOLs have noted that despite their effectiveness in treating MG, the prolonged use of corticosteroids is associated with severe side effects, and that their use must be carefully managed in order to attain a therapeutic effect without impacting a patient’s quality of life. KOLs mentioned that, as a result, the optimal steroid dosing regimen and the effects of steroid discontinuation remain unclear. Therefore, the data presented by Huang and colleagues will play a key role in developing optimal treatment regimens in an indication characterised by off-label drug use and inconsistent patient outcomes.
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The findings presented by Huang and colleagues provide valuable insights into the management of MG with corticosteroid monotherapy and could contribute significantly to addressing the lack of standardised treatment guidelines in MG, paving the way for improved patient outcomes. However, further research across diverse patient populations remains essential to refine and standardise treatment protocols globally.
