Clinical features, treatment and prognosis of MuSK antibody-associated myasthenia gravis in Northwest China: a single-centre retrospective cohort study

Background and purpose To summarize the clinical characteristics of patients with muscle-specific kinase antibody-associated myasthenia gravis (MuSK-MG) and to evaluate the therapeutic responses to different treatment regimes. Methods Eighteen MuSK-MG patients admitted in our department between October 2017 and September 2020 were included. Clinical parameters were collected and the responses to different immunosuppressive drugs were assessed by MGFA Postintervention Status (MGFA-PIS). Meanwhile, the correlation between QMG scores and MuSK antibody titers were analyzed and MuSK antibody (MuSK-ab) titers were compared before and after therapy based on different immunosuppressive treatment regimes. Results Female predominance (ratio of females to males, 15:3) was evident in the study population, with the average onset age of (40.28 +/- 18.57) years and the median disease course of 30.50 months (interquartile range [IQR], 17.50-44.75 months). Ocular manifestation was the most common onset symptom (11/18; 61.11%), and mild symmetrical ptosis was most frequent. Bulbar symptoms had the highest incidence of 88.89% over the entire disease course. Abnormal responses to RNS test were recorded most frequently on the musculus deltoideus (83.33%). All patients were treated with prednisone (Pred) alone or plus azathioprine (AZA), tacrolimus (TAC) or low-dose rituximab (RTX), and 17 (94.44%) of them achieved a favorable outcome defined as minimal manifestation (MM) or better. In general, an obvious positive correlation between QMG score and MuSK-ab titer (r = 0.710, P < 0.001) were found in all patients. A more significant reduction of MuSK-ab titers was observed in patients receiving TAC or RTX plus Pred than those receiving AZA plus Pred. Conclusions The prominent clinical manifestations of ocular and bulbar muscles involvements, together with abnormal RNS response mostly recorded on the musculus deltoideus and better efficacy associated with TAC or low-dose RTX plus Pred, provide a more exhaustive picture of MuSK-MG, particularly in Northwest China.

Automated summary

Patients with MuSK-MG showed a female predominance, with an average age of onset at 40.28 years and a median disease course of 30.50 months. Ocular manifestation was the most common onset symptom, and there was an obvious positive correlation between QMG score and MuSK-ab titer in all patients.