Abstract:
Eculizumab has improved recovery from ventilatory support in myasthenic crisis (MC) cases. However, the safety and efficacy profiles from prospective studies are still lacking. This study aimed to explore eculizumab\'s safety and efficacy in a prospective case series of patients with refractory MC. We followed a series of anti-acetylcholine receptor (AChR) antibody-positive myasthenia gravis (MG) patients who received eculizumab as an add-on therapy for 12 weeks during MC to facilitate the weaning process and reduced disease activity. Serum anti-AChR antibodies and peripheral immune molecules associated with the complement pathway were evaluated before and after eculizumab administration. Compared to the baseline Myasthenia Gravis Foundation of America (MGFA)-quantitative MG test (QMG) scores (22.25 ± 4.92) and MG-activities of daily living (MG-ADL; 18.25 ± 2.5) scores at crisis, improvements were observed from 4 weeks (14.5 ± 10.47 and 7.5 ± 7.59, respectively) through 12 weeks (7.5 ± 5.74 and 2.25 ± 3.86, respectively) post-treatment. Muscle strength consistently improved across ocular, bulbar, respiratory, and limb/gross domain groups. One patient died of cardiac failure at 16 weeks. Three cases remained in remission at 24 weeks, with a mean QMG score of 2.67 ± 2.89 and ADL score of 0.33 ± 0.58. No significant side effects were reported. Serum CH50 and soluble C5b-9 levels significantly declined, while there were no significant changes in serum anti-AChR antibody levels, C1q, C5a levels, or peripheral lymphocyte proportions. Eculizumab was well tolerated and showed efficacy in this case series. Large prospective cohort studies with extended follow-up periods are needed to further explore the safety and efficacy profile in real-world practice.
摘要:
Eculizumab改善了肌无力危象(MC)病例的通气支持恢复。然而,前瞻性研究的安全性和有效性仍缺乏.本研究旨在探讨依库珠单抗在难治性MC患者的前瞻性病例系列中的安全性和有效性。我们跟踪了一系列抗乙酰胆碱受体(AChR)抗体阳性的重症肌无力(MG)患者,这些患者在MC期间接受了依库珠单抗作为附加治疗12周,以促进断奶过程并降低疾病活动。在依库珠单抗给药之前和之后评估血清抗AChR抗体和与补体途径相关的外周免疫分子。与基线美国重症肌无力基金会(MGFA)-定量MG测试(QMG)得分(22.25±4.92)和MG-日常生活活动(MG-ADL;18.25±2.5)得分相比,从治疗后4周(分别为14.5±10.47和7.5±7.59)到12周(分别为7.5±5.74和2.25±3.86)观察到改善.肌肉力量持续改善整个眼睛,球杆,呼吸,和肢体/粗体域组。1例患者在16周时死于心力衰竭。3例24周时仍处于缓解状态,平均QMG评分为2.67±2.89,ADL评分为0.33±0.58。没有明显的副作用报告。血清CH50和可溶性C5b-9水平显著下降,虽然血清抗AChR抗体水平没有显著变化,C1q,C5a液位,或外周淋巴细胞比例。Eculizumab的耐受性良好,在该病例系列中显示出疗效。需要进行长期随访的大型前瞻性队列研究,以进一步探索现实实践中的安全性和有效性。