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Nomogram for short-term outcome assessment in AChR subtype generalized myasthenia gravis.

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机构: [1]Department of Neurology, Huashan Hospital Fudan University, No.12 Middle Wulumuqi Road, Shanghai 200040, China. [2]Department of Pharmacy, Huashan Hospital Fudan University, Shanghai, China. [3]Radiation Oncology Center, Huashan Hospital, Fudan University, Shanghai, China. [4]Department of Neurology, Xuanwu Hospital, Capital Medical University, Beijing, China. [5]Department of Neurology, Tangdu Hospital, the Fourth Military Medical University, Xi’an, China. [6]Department of Neurology, Wuhan No.1 Hospital, Wuhan, China. [7]Department of Neurology, Xiangya Hospital, Central South University, Changsha, China.
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关键词: Generalized myasthenia gravis Anti-acetylcholine receptor antibody Minimal symptom expression Nomogram

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An accurate prediction for prognosis can help in guiding the therapeutic options and optimizing the trial design for generalized myasthenia gravis (gMG). We aimed to develop and validate a predictive nomogram to assess the short-term outcome in patients with the anti-acetylcholine receptor (AChR) subtype gMG.We retrospectively reviewed 165 patients with AChR subtype gMG who were immunotherapy naïve at the first visit from five tertiary centers in China. The short-term clinical outcome is defined as the achievement of minimal symptom expression (MSE) at 12 months. Of them, 120 gMG patients from Huashan Hospital were enrolled to form a derivation cohort (n = 96) and a temporal validation cohort (n = 24) for the nomogram. Then, this nomogram was externally validated using 45 immunotherapy naïve AChR subtype gMG from the other four hospitals. Multivariate logistic regression was used to screen independent factors and construct the nomogram.MSE was achieved in 70 (72.9%), 20 (83.3%), and 33 (73.3%) patients in the training, temporal validation, and external validation cohort, respectively. The duration ≤ 12 months (p = 0.021), ocular score ≤ 2 (p = 0.006), QMG score > 13 (p = 0.008), and gross motor score ≤ 9 (p = 0.006) were statistically associated with MSE in AChR subtype gMG. The nomogram has good performance in predicting MSE as the concordance indexes are 0.81 (95% CI, 0.72-0.90) in the development cohort, 0.944 (95% CI, 0.83-1.00) in the temporal validation cohort, and 0.773 (95% CI, 0.63-0.92) in the external validation cohort.The nomogram achieved an optimal prediction of MSE in AChR subtype gMG patients using the baseline clinical characters.

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出版当年[2020]版:
大类 | 2 区 医学
小类 | 3 区 医学:研究与实验
最新[2023]版:
大类 | 2 区 医学
小类 | 2 区 医学:研究与实验
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出版当年[2019]版:
Q2 MEDICINE, RESEARCH & EXPERIMENTAL
最新[2023]版:
Q1 MEDICINE, RESEARCH & EXPERIMENTAL

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第一作者机构: [1]Department of Neurology, Huashan Hospital Fudan University, No.12 Middle Wulumuqi Road, Shanghai 200040, China.
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