Paediatric Neurology Department and Neuromuscular Reference center; Hôpital Universitaire des Enfants Reine Fabiola (HUDERF), Université Libre de Bruxelles (ULB), Brussels, Belgium.
Neuromuscular Reference Center (NMRC) Universitair Ziekenhuis (UZ Gent), Ghent, Belgium.
J Neuromuscul Dis. 2024;11(4):839-853. doi: 10.3233/JND-240017.
Spinal muscular atrophy (SMA), a genetic neuromuscular disease caused by lack of survival of motor neuron (SMN) protein, is characterized by muscular atrophy and respiratory and bulbar dysfunction. While swallowing disorders are common, they remain poorly studied.
Our study aimed to explore 1) intraoral pressure measurements with the Iowa Oral Performance Instrument system and the reliability of a Swallowing Function Assessment Questionnaire (SFAQ) in healthy controls, and 2) evaluate their use as swallowing function biomarkers and the evolution of swallowing function over time in children with SMA.
We recruited 53 healthy children and 27 SMA patients all treated with SMN gene modulator therapy. Participants completed the SFAQ and underwent at least one measurement of maximal oral pressures (lingual, labial, and masseter).
Mean oral normalized pressure index were lower (all sites p < 0.001) and mean SFAQ scores were higher (p < 0.001) in patients compared with healthy controls. Pressure evolution over 1 year in SMA patients for all three oral sites did not show significant differences. SFAQ scores correlated negatively with oral pressures at all three sites in patients.
Both tools provided new insights on the oral and pharyngeal phase of swallowing in SMA patients. In SMA patients, muscle strength in certain crucial anatomical regions during swallowing is weaker than in healthy children.
脊髓性肌萎缩症(SMA)是一种由运动神经元生存蛋白缺失引起的遗传性神经肌肉疾病,其特征为肌肉萎缩和呼吸及延髓功能障碍。吞咽障碍虽较为常见,但研究仍不完善。
本研究旨在探讨 1)使用爱荷华口腔表现仪器系统(Iowa Oral Performance Instrument system)进行口腔内压力测量和吞咽功能评估问卷(Swallowing Function Assessment Questionnaire,SFAQ)在健康对照组中的可靠性,2)评估其作为吞咽功能生物标志物的作用,以及 SMA 患儿随时间推移吞咽功能的演变。
我们招募了 53 名健康儿童和 27 名接受 SMN 基因调节剂治疗的 SMA 患者。参与者完成了 SFAQ,并至少进行了一次最大口腔压力(舌部、唇部和咬肌)测量。
与健康对照组相比,患者的口腔压力标准化指数均值较低(所有部位 p 值均<0.001),SFAQ 评分均值较高(p 值均<0.001)。SMA 患者在 1 年内所有三个口腔部位的压力变化均无显著差异。SFAQ 评分与患者三个口腔部位的口腔压力均呈负相关。
这两种工具均为 SMA 患者的口腔和咽期吞咽提供了新的见解。在 SMA 患者中,吞咽时某些关键解剖区域的肌肉力量比健康儿童弱。