Department of Neurology, Xiangya Hospital, Central South University, Xiangya Road, Kaifu District, Changsha, 410008, China.
Department of Neurology, Changsha Central Hospital, Changsha, China.
Orphanet J Rare Dis. 2024 Jan 19;19(1):19. doi: 10.1186/s13023-024-03025-z.
Oral prednisone has been recognized as the first-line therapy for the treatment of ocular myasthenia gravis (OMG). However, its long-term use is complicated by numerous adverse effects and is ineffective for some OMG patients in reaching remission. This study aimed to evaluate the effectiveness and safety of intravenous methylprednisolone (IVMP) and tacrolimus monotherapy for OMG patients with unsatisfactory responses to conventional prednisone therapy.
We retrospectively reviewed 57 OMG patients who had not achieved satisfactory improvement after prednisone therapy and thereby received IVMP or tacrolimus monotherapy for at least 6 months. Ocular symptoms were evaluated by the ocular-quantitative MG (QMG) score at each time point. A ≥ 2-point fall in ocular QMG score was defined as the cut-off point to indicate clinical improvement. Logistic regression analysis was performed to identify factors associated with the efficacy of IVMP at discharge. Adverse events were recorded.
Both IVMP and tacrolimus monotherapy demonstrated significant clinical efficacy, with no statistical differences observed at the study endpoint. The proportions of patients who reached the cut-off point for efficacy evaluation were higher in the IVMP group than in the tacrolimus group (1, 3, and 6 months: 51.7% (15/29) vs 12.0% (3/25), p = 0.002; 69.0% (20/29) vs 40.0% (10/25), p = 0.033; 69.0% (20/29) vs 46.4% (13/28), p = 0.085, respectively). Multivariate logistics analysis showed that high ocular QMG scores at baseline indicated favourable responses to IVMP treatment (OR = 1.781; 95% CI 1.066-2.975; p = 0.028). All the adverse events were transient and tolerable.
Our findings suggest that both IVMP and tacrolimus monotherapy hold promise as viable treatment options for OMG patients with unsatisfactory responses to oral prednisone. The study supports the safety and effectiveness of both therapies, with IVMP exhibiting faster improvement and favourable efficacy in patients with high ocular QMG scores.
口服泼尼松已被认为是治疗眼肌型重症肌无力（OMG）的一线治疗方法。然而，其长期使用会引起许多不良反应，并且对一些 OMG 患者无效，无法使其达到缓解。本研究旨在评估静脉注射甲基泼尼松龙（IVMP）和他克莫司单药治疗对常规泼尼松治疗反应不佳的 OMG 患者的疗效和安全性。
我们回顾性分析了 57 例 OMG 患者，这些患者在接受泼尼松治疗后未获得满意改善，因此接受了至少 6 个月的 IVMP 或他克莫司单药治疗。在每个时间点通过眼肌定量 MG（QMG）评分评估眼部症状。眼 QMG 评分下降≥2 分定义为临床改善的截止点。进行逻辑回归分析以确定与出院时 IVMP 疗效相关的因素。记录不良反应。
IVMP 和他克莫司单药治疗均显示出显著的临床疗效，在研究终点时无统计学差异。IVMP 组达到疗效评估截止点的患者比例高于他克莫司组（1、3 和 6 个月：51.7%（15/29）比 12.0%（3/25），p=0.002；69.0%（20/29）比 40.0%（10/25），p=0.033；69.0%（20/29）比 46.4%（13/28），p=0.085）。多变量逻辑分析显示，基线时高眼 QMG 评分预示着 IVMP 治疗有良好反应（OR=1.781；95%CI 1.066-2.975；p=0.028）。所有不良反应均为一过性且可耐受。
我们的研究结果表明，IVMP 和他克莫司单药治疗对口服泼尼松治疗反应不佳的 OMG 患者均有希望成为可行的治疗选择。本研究支持这两种治疗方法的安全性和有效性，IVMP 治疗在眼 QMG 评分较高的患者中具有更快的改善和良好的疗效。
