Stieglbauer Karl, Pichler Robert, Topakian Raffi
Consultant Neurologist in Private Practice, Linz, Austria.
Institute of Nuclear Medicine, Neuromed Campus, Johannes Kepler University, Linz, Austria.
J Neurol Sci. 2017 Apr 15;375:241-244. doi: 10.1016/j.jns.2017.02.009. Epub 2017 Feb 4.
Rituximab (RTX) has emerged as an attractive off-label treatment option for patients with myasthenia gravis (MG) refractory to other immune therapies. However, data on long-term outcome after RTX for MG are still scarce. Here we present the 10-year outcomes [median (range) 10.1 (6.7-11.2) years] with respect to efficacy, safety, costs of inhospital care, and impact on childbearing potential in all four MG patients treated by one of the authors with RTX. In all patients, RTX led to sustained clinical improvement and eventual tapering of other immune therapies. RTX was well tolerated, and complications were not observed. After the start of RTX, annual costs for hospital admissions were markedly reduced compared to costs in the year preceding RTX. Under close clinical observation, two patients had uncomplicated pregnancies giving birth to a healthy child. With regard to its efficacy, excellent tolerance, lack of complications, low frequency of repeat infusions and pending patent expiry in many countries, RTX appears to compare favourably with other immune therapies used for MG. Multicentre trials and registries are urgently needed to further address long-term safety issues and clarify the efficacy and role of RTX in managing MG.
利妥昔单抗（RTX）已成为一种有吸引力的非标签治疗选择，用于对其他免疫疗法难治的重症肌无力（MG）患者。然而，关于RTX治疗MG后的长期结果的数据仍然很少。在此，我们展示了由本文作者之一用RTX治疗的所有4例MG患者的10年结果[中位数（范围）10.1（6.7 - 11.2）年]，涉及疗效、安全性、住院护理费用以及对生育潜力的影响。在所有患者中，RTX导致临床持续改善以及其他免疫疗法最终逐渐减少用量。RTX耐受性良好，未观察到并发症。RTX开始使用后，与RTX治疗前一年的费用相比，每年的住院费用显著降低。在密切的临床观察下，两名患者顺利怀孕并生下健康的孩子。鉴于其疗效、良好的耐受性、无并发症、重复输注频率低以及在许多国家即将专利到期，RTX似乎比用于MG的其他免疫疗法更具优势。迫切需要进行多中心试验和登记，以进一步解决长期安全性问题，并阐明RTX在治疗MG中的疗效和作用。
