Long-Term Improvement in a Chinese Cohort of Glucocorticoid-Resistant Childhood-Onset Myasthenia Gravis Patients Treated With Tacrolimus.
Long-Term Improvement in a Chinese Cohort of Glucocorticoid-Resistant Childhood-Onset Myasthenia Gravis Patients Treated With Tacrolimus.
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用他克莫司治疗的中国糖皮质激素耐药儿童发病重症肌无力患者队列的长期改善。
DOI：
10.3389/fneur.2022.820205
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发表时间：
2022
影响因子：
3.4
通讯作者：
Bu B
中科院分区：
文献类型：
--
作者：
Bi Z;Cao Y;Lin J;Zhang Q;Liu C;Gui M;Bu B
评估他克莫司治疗对糖皮质激素反应不足的儿童期发病的重症肌无力 (CMG) 的长期疗效，并调查与他克莫司治疗后良好结局相关的因素。对接受足够的泼尼松治疗至少 8 周后未取得满意改善的 CMG 患者进行了一项回顾性观察性队列研究。所有患者均接受 2-3 mg 剂量的他克莫司治疗 6 个月以上。使用泼尼松剂量、定量 MG (QMG) 和 MG 日常生活活动 (ADL) 评分来评估主要疗效结果。根据QMG评分的变化将参与者分为改善组和未改善组，以调查影响他克莫司疗效的危险因素。最终共有 149 名糖皮质激素耐药 CMG 患者纳入我们的研究，其中 113 名（75.8%）对他克莫司反应良好（定义为最小表现状态或更好）。开始使用他克莫司一个月后，泼尼松剂量、QMG 和 ADL 评分明显改善，并在整个研究过程中持续改善。更重要的是，89 名患者（78.8%）最终停用了泼尼松。胸腺类型 [比值比 (OR) = 3.156，95% 置信区间 (CI) 1.427–6.978； P = 0.005]和干预前状态（OR = 0.284，95%CI 0.109-0.741；P = 0.010）是控制多重逻辑回归中的混杂因素后他克莫司疗效的独立预测因子。大多数糖皮质激素耐药的 CMG 患者在加用他克莫司后长期预后良好。胸腺类型和干预前状态可以作为影响他克莫司疗效的潜在预测因素。
To evaluate the long-term outcome of tacrolimus for childhood-onset myasthenia gravis (CMG) with an inadequate response to glucocorticoids, and investigate factors associated with favorable outcomes following tacrolimus treatment. A retrospective, observational cohort study was performed for CMG patients who had not improved satisfactorily after sufficient prednisone therapy for at least 8 weeks. All patients were given tacrolimus in doses of 2–3 mg for more than 6 months. The primary efficacy outcome was assessed using the prednisone dose, quantitative MG (QMG), and MG-activity of daily living (ADL) scores. The participants were divided into improved and unimproved groups based on changes in QMG scores to investigate the risk factors that affected tacrolimus efficacy. A total of 149 glucocorticoid resistant CMG patients were finally enrolled in our study, with 113 (75.8%) responding well to tacrolimus (defined as minimal manifestation status or better). One month after initiating tacrolimus, there was a noticeable improvement in prednisone dose, QMG, and ADL scores, which continued to improve throughout the study. More importantly, the prednisone was eventually stopped in 89 of the patients (78.8%). Thymus type [odds ratio (OR) = 3.156, 95% confidence interval (CI) 1.427–6.978; P = 0.005] and pre-intervention status (OR = 0.284, 95%CI 0.109–0.741; P = 0.010) were independent predictors of tacrolimus efficacy after controlling for confounding factors in multiple logistic regression. The majority of glucocorticoid-resistant CMG patients have a good long-term prognosis after adding tacrolimus. Thymus type and pre-intervention status can serve as potential predictors affecting the efficacy of tacrolimus.
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