Catteruccia Michela, Vuillerot Carole, Vaugier Isabelle, Leclair Danielle, Azzi Viviane, Viollet Louis, Estournet Brigitte, Bertini Enrico, Quijano-Roy Susana
Unit of Neuromuscular and Neurodegenerative Disorders, Laboratory of molecular Medicine, Department of Neurosciences and Neurorehabilitation, Bambino Gesù Children's Research Hospital, IRCCS, Rome, Italy.
Hospices Civils de Lyon,Hôpital Femme-Mère-Enfant, L'Escale, Service de Médecine Physique et de Réadaptation Pédiatrique, Bron, F-69500, France, Université de Lyon, Lyon, F-69000, France, CNRS UMR 5558, Laboratoire de Biométrie et Biologie Evolutive, Equipe Biostatistique Santé, Pierre-Bénite, F-69310, France.
J Neuromuscul Dis. 2015 Nov 21;2(4):453-462. doi: 10.3233/JND-150084.
Scoliosis is the most debilitating issue in SMA type 2 patients. No evidence confirms the efficacy of Garches braces (GB) to delay definitive spinal fusion.
Compare orthopedic and pulmonary outcomes in children with SMA type 2 function to management.
We carried out a monocentric retrospective study on 29 SMA type 2 children who had spinal fusion between 1999 and 2009. Patients were divided in 3 groups: group 1-French patients (12 children) with a preventive use of GB; group 2-French patients (10 children) with use of GB after the beginning of the scoliosis curve; and group 3-Italian patients (7 children) with use of GB after the beginning of the scoliosis curve referred to our centre to perform orthopedic preoperative management.
Mean preoperative and postoperative Cobb angle were significantly lower in the group 1 of proactively braced than in group 2 or 3 (Anova p = 0.03; Kruskal Wallis test p = 0.05). Better surgical results were observed in patients with a minor preoperative Cobb angle (r = 0.92 p < 0.0001). Fewer patients in the group 1 proactively braced required trunk casts and/or halo traction and an additional anterior fusion in comparison with patients in the group 2 and 3. Moreover, major complications tend to be less in the group 1 proactively braced. No significant differences were found between groups in pulmonary outcome measures.
A proactive orthotic management may improve orthopedic outcome in SMA type 2. Further prospective studies comparing SMA management are needed to confirm these results.
Therapeutic Level III. See Instructions to Authors on jbjs.org for a complete description of levels of evidence (Retrospective comparative study).
脊柱侧弯是2型脊髓性肌萎缩症（SMA）患者最严重的问题。尚无证据证实加歇支具（GB）在延迟确定性脊柱融合方面的疗效。
比较2型SMA功能儿童在矫形和肺部方面的治疗结果。
我们对1999年至2009年间接受脊柱融合手术的29例2型SMA儿童进行了单中心回顾性研究。患者分为3组：第1组为法国患者（12名儿童），预防性使用GB；第2组为法国患者（10名儿童），脊柱侧弯曲线开始后使用GB；第3组为意大利患者（7名儿童），脊柱侧弯曲线开始后使用GB，转诊至我们中心进行矫形术前管理。
积极使用支具的第1组术前和术后平均Cobb角显著低于第2组或第3组（方差分析p = 0.03；Kruskal Wallis检验p = 0.05）。术前Cobb角较小的患者手术效果更好（r = 0.92，p < 0.0001）。与第2组和第3组患者相比，积极使用支具的第1组需要躯干石膏固定和/或头环牵引以及额外前路融合的患者更少。此外，积极使用支具的第1组严重并发症往往更少。各组在肺部治疗结果指标方面未发现显著差异。
积极的矫形器管理可能改善2型SMA的矫形效果。需要进一步进行比较SMA治疗的前瞻性研究来证实这些结果。
治疗性III级。有关证据水平的完整描述，请参阅jbjs.org上的作者指南（回顾性比较研究）。
