Barnett Carolina, Katzberg Hans D, Keshavjee Shaf, Bril Vera
Division of Neurology - Department of Medicine, University of Toronto and University Health Network, Toronto, Canada.
Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Canada.
Orphanet J Rare Dis. 2014 Dec 24;9:214. doi: 10.1186/s13023-014-0214-5.
The efficacy of thymectomy in patients with non-thymomatous Myasthenia Gravis (MG) is still unclear. Main limitations have been variable outcome definitions, lack of a control group and adjustment for confounding.
To study the efficacy of thymectomy in achieving remission or minimal manifestation (R/MM) status in patients with non-thymomatous MG.
Patients with generalized MG and minimum follow-up of 6 months were included. Demographic data and treatments were recorded, as well as the MGFA post-intervention status at the last visit. Propensity scores were used to create a matched cohort of treated and untreated patients. Standard and Bayesian Cox models were used to study treatment effects.
Of 395 patients included, 183(46%) had a thymectomy. Thymectomy patients were younger (p < 0.001), with more females (p < 0.001) and more patients in MGFA classes 4-5 at diagnosis (p = 0.01). A matched cohort of thymectomized patients and controls (n = 98) was created. The hazard ratio (HR) for the matched cohort was 1.9 (CI:1.6-2.3), favoring thymectomy. The predicted R/MM rate was 21% in treated and 6% in controls at 5 years (Absolute difference:15%). A Bayesian Cox model for the matched cohort had an estimated probability of thymectomy efficacy (HR > 1) of 96% using a non-informative prior, and 79% using a skeptical prior.
When controlling for potential confounders, thymectomized patients had a higher probability of achieving R/MM status through time compared to controls. This study provides class III evidence of the efficacy of thymectomy in non-thymomatous myasthenia gravis.
胸腺切除术治疗非胸腺瘤型重症肌无力(MG)患者的疗效仍不明确。主要局限性在于结局定义多变、缺乏对照组以及未对混杂因素进行调整。
研究胸腺切除术在非胸腺瘤型MG患者中实现缓解或最小表现(R/MM)状态的疗效。
纳入全身型MG且最短随访6个月的患者。记录人口统计学数据、治疗情况以及最后一次随访时的MGFA干预后状态。使用倾向评分创建治疗组和未治疗组患者的匹配队列。采用标准和贝叶斯Cox模型研究治疗效果。
纳入的395例患者中,183例(46%)接受了胸腺切除术。胸腺切除术患者更年轻(p < 0.001),女性更多(p < 0.001),诊断时MGFA 4 - 5级的患者更多(p = 0.01)。创建了胸腺切除患者与对照组的匹配队列(n = 98)。匹配队列的风险比(HR)为1.9(CI:1.6 - 2.3),支持胸腺切除术。5年时治疗组的预测R/MM率为21%,对照组为6%(绝对差异:15%)。匹配队列的贝叶斯Cox模型使用非信息先验时,胸腺切除术疗效(HR > 1)的估计概率为96%,使用怀疑先验时为79%。
在控制潜在混杂因素后,与对照组相比,胸腺切除患者随时间推移实现R/MM状态的概率更高。本研究提供了胸腺切除术治疗非胸腺瘤型重症肌无力疗效的III级证据。