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与中国脊髓性肌萎缩症延迟诊断相关的因素及诊断延迟的变化。

Factors associated with delayed diagnosis of spinal muscular atrophy in China and changes in diagnostic delay.

机构信息

Department of Medical Genetics, Capital Institute of Pediatrics, Beijing, China; Institute of Basic Medical Sciences and School of Basic Medicine, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China.

Department of Medical Genetics, Capital Institute of Pediatrics, Beijing, China.

出版信息

Neuromuscul Disord. 2021 Jun;31(6):519-527. doi: 10.1016/j.nmd.2021.03.002. Epub 2021 Mar 6.

Abstract

Spinal muscular atrophy (SMA) is a rare neuromuscular disease, which often occurs in childhood. Early SMA treatment may be highly beneficial to SMA patients, their families, and society. However, delayed diagnosis is common. To identify the factors that affect the SMA diagnostic time window, we analyzed disease characteristics, family factors, and medical factors of 205 SMA families. We compared the data with those of our previous cohort to explore the dynamic changes in the diagnostic time window. The median diagnostic time windows for SMA types I, II, and III were 3.38 [interquartile range (IQR): 2.01-4.98], 4.08 (IQR: 2.07-8.17), and 11.37 (IQR: 4.92-24.07) months, respectively. The diagnostic time window in patients who were clinically diagnosed with SMA at their first hospital visit was 49.42% shorter than that in other patients. Type I/II patients visited approximately 2.56 doctors before diagnosis, while type III patients visited approximately 3.94 doctors before diagnosis. The diagnostic time windows for types II and III were 54.67 and 62.10% shorter, respectively, than those in the previous cohort, which is mainly due to improvements in medical capacity. Therefore, with public awareness, increased medical personnel understanding, and increased neonatal screening, the SMA diagnostic time window is expected to further reduce.

摘要

脊髓性肌萎缩症(SMA)是一种罕见的神经肌肉疾病,通常在儿童时期发病。早期 SMA 治疗可能对 SMA 患者、其家庭和社会高度有益。然而,延迟诊断很常见。为了确定影响 SMA 诊断时间窗的因素,我们分析了 205 个 SMA 家庭的疾病特征、家庭因素和医疗因素。我们将这些数据与我们之前的队列进行了比较,以探讨诊断时间窗的动态变化。SMA 类型 I、II 和 III 的中位诊断时间窗分别为 3.38 [四分位距(IQR):2.01-4.98]、4.08(IQR:2.07-8.17)和 11.37(IQR:4.92-24.07)个月。在首次就诊时即临床诊断为 SMA 的患者中,诊断时间窗比其他患者短 49.42%。I/II 型患者在确诊前平均就诊约 2.56 位医生,III 型患者在确诊前平均就诊约 3.94 位医生。II 型和 III 型的诊断时间窗分别比之前的队列缩短了 54.67%和 62.10%,这主要是由于医疗能力的提高。因此,随着公众意识的提高、医疗人员的理解增加和新生儿筛查的增加,SMA 的诊断时间窗有望进一步缩短。

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