Long-term outcome of 424 childhood-onset myasthenia gravis patients
Long-term outcome of 424 childhood-onset myasthenia gravis patients
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DOI：
10.1007/s00415-015-7638-2
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发表时间：
2015-04-01
影响因子：
6
通讯作者：
Bu, Bitao
中科院分区：
文献类型：
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作者：
Gui, Mengcui;Luo, Xuan;Bu, Bitao
本研究的目的是描述中国儿童期重症肌无力（CMG）患者的临床特征、预后和可能影响预后的因素。我们对同济医院424例CMG患者进行了至少5年的随访。随访结束时，根据MGFA干预后状态测量所有患者的预后。在本研究中，患者随访9.8 +/- 5.4年。平均发病年龄为5.4±3.6岁。眼部重症肌无力（OMG）是发病后2年内最主要的CMG类型（95%）。116例胸腺增生，6例胸腺瘤。69.5%的患者乙酰胆碱受体抗体升高。所有患者均接受常规治疗。34例（8.0%）患者行胸腺切除术。随访结束时，显著改善71例（16.7%），无变化66例（15.6%），恶化53例（12.5%），加重234例（55.2%）。重要的是，50例OMG患者（12.4%）在发病后2年内转变为全身性重症肌无力（GMG）。胸腺切除术不能有效减少OMG向GMG的转变。然而，GMG病例明显受益于手术。本研究表明，自身免疫性CMG病例占中国MG人群的50%以上。长期随访发现，CMG患者改善的比例较低，恶化和加重的比例较高。在患者完全稳定缓解后，不应过早停止治疗。需要更多的研究来更好地控制CMG症状。
The objective of this study was to describe the clinical characteristics, outcome and factors that may affect the outcome of childhood-onset myasthenia gravis (CMG) patients in China. We have followed up 424 patients with CMG for at least 5 years at Tongji Hospital. At the end of follow-up, the outcome of all the patients was measured according to MGFA Post-intervention Status. In this study, the patients have been followed up for 9.8 +/- A 5.4 years. The mean onset age was 5.4 +/- A 3.6 years. Ocular myasthenia gravis (OMG) was the major type of CMG within 2 years after onset (95 %). Thymic hyperplasia was found in 116 patients, and thymoma was confirmed in 6 patients. Acetylcholine receptor antibodies were elevated in 69.5 % of the patients. All the patients were routinely treated. Thymectomy was performed in 34 patients (8.0 %). At the end of follow-up, seventy-one patients (16.7 %) were significantly improved, 66 patients (15.6 %) remained unchanged, 53 patients (12.5 %) were worsened, and 234 patients (55.2 %) were exacerbated. Importantly, fifty OMG patients (12.4 %) had transformed into generalized myasthenia gravis (GMG) over 2 years after onset. Thymectomy did not effectively reduce the transformation from OMG to GMG. However, GMG cases significantly benefited from the surgery. This study indicated that the cases with autoimmune CMG account for over 50 % in Chinese MG population. The long-term follow-up discloses that CMG patients have a low percentage of improvement, and a high percentage of worsening and exacerbation. The treatment should not be withdrawn too early after the patients obtain complete stable remission. More studies are needed to gain better control of CMG symptoms.