Pilot study explores ketogenic diet effects on symptoms in adults with generalized myasthenia gravis

OBJECTIVE: Myasthenia gravis (MG) is an autoimmune neuromuscular disorder characterized by fluctuating weakness and disabling fatigue. This pilot study assessed the feasibility and explored potential clinical and immunological effects of a ketogenic diet (KD) in generalized MG. METHODS: In a randomized, controlled, open-label 12-week pilot study, adults with generalized MG were assigned to a KD (n = 20) or to continue their habitual diet (n = 21). The primary endpoint was change in Myasthenia Gravis-Activities of Daily Living (MG-ADL). Secondary outcomes included Quantitative Myasthenia Gravis score (QMG), MGFA class, Fatigue Severity Scale (FSS), Myasthenia Gravis Quality of Life (MG-QoL15r), Patient Acceptable Symptom State (PASS), serum calprotectin (sCLP), serum neurofilament light chain (sNfL), and T-cell subsets. Analyses were conducted descriptively using effect sizes and 95% confidence intervals. RESULTS: Numerical improvements were observed in the KD group compared to controls. MG-ADL decreased (median Δ -2; moderate effect size, 95% CI including zero). QMG improved (median Δ -3; effect size 1.29, 95% CI 0.55-2.02), and fatigue decreased (median Δ -7; effect size 1.19, 95% CI 0.45-1.91). MG-QoL15r and PASS remained stable in the intervention group but worsened in controls. sCLP showed a numerical decrease, while sNfL remained unchanged. Adherence, defined as study completion, was 75%. CONCLUSION: A ketogenic diet is feasible and safe. Numerical improvements in clinical outcomes, and exploratory immunological changes were observed. Given the exploratory design findings should be interpreted with caution and require confirmation in larger, blinded studies.