Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, London, UK.
Population, Policy and Practice Department, University College London Great Ormond Street Institute of Child Health, London, UK.
Muscle Nerve. 2024 Nov;70(5):1000-1009. doi: 10.1002/mus.28238. Epub 2024 Sep 4.
INTRODUCTION/AIMS: Available studies on scoliosis surgery in spinal muscular atrophy (SMA) have focused on the primary outcome of the procedure-the correction of the curve-whereas research focusing on secondary outcomes is scarce. We aimed to investigate postsurgical changes in respiratory function, motor function, weight, pain, and satisfaction.
We retrospectively reviewed the clinical notes of 32 disease-modifying treatment-naïve patients (26 SMA2, 6 nonambulant SMA3). We also performed investigator-developed phone interviews and conducted a focus group with families on postsurgical satisfaction.
Mean annual rate of forced vital capacity percent decline improved in SMA2: -3.2% postsurgery versus -6.9% presurgery (p < .001), with similar trajectories in SMA3. Gross motor functional scores (Hammersmith Functional Motor Scale) available in 12/32 dropped immediately after surgery: median loss of 6.5 points, with relatively spared upper limb function. Weight z-scores postsurgery dropped in 16/32, requiring food supplements (5/16); one/16 lost >5% of total weight requiring gastrostomy. Postsurgical pain was frequently reported, especially hip pain (13/32). Overall, 10/10 patients/parents participating in the phone interview rated the procedure as very successful for posture and physical appearance. Nonetheless, 7/10 reported postsurgical pain, reduced mobility, and unmet care needs. The seven patients/parents attending the focus group highlighted lack of intensive physiotherapy programs, occupational therapy assistance, and psychological support as postsurgical unmet care needs.
This study reports a positive impact of scoliosis surgery on respiratory function and overall satisfaction with posture and physical appearance. The observed negative impact on the other outcomes highlights the importance of multidisciplinary approaches to improve postoperative management.
引言/目的：现有的脊柱肌萎缩（SMA）脊柱侧弯手术研究主要集中在手术的主要结果——即曲线的矫正，而对次要结果的研究则很少。我们旨在研究术后呼吸功能、运动功能、体重、疼痛和满意度的变化。
我们回顾性地审查了 32 名未经疾病修正治疗的患者（26 名 SMA2，6 名非卧床 SMA3）的临床记录。我们还对手术后的满意度进行了调查员开发的电话访谈和焦点小组讨论。
SMA2 的用力肺活量百分比年下降率在手术后改善：-3.2%，而术前为-6.9%（p<0.001），SMA3 也有类似的趋势。在 32 名患者中有 12 名可获得总体运动功能评分（Hammersmith 运动功能量表），他们在手术后立即下降：中位数下降 6.5 分，上肢功能相对较好。手术后体重 z 评分下降 16/32，需要补充食物（5/16）；1/16 失去了超过 5%的体重，需要胃造口术。术后疼痛经常报告，尤其是髋关节疼痛（13/32）。总的来说，10/10 名参与电话访谈的患者/家长将手术评为对姿势和身体外观非常成功。尽管如此，仍有 7/10 名患者/家长报告术后疼痛、活动能力下降和未满足的护理需求。参加焦点小组的七名患者/家长强调了缺乏强化物理治疗方案、职业治疗协助和心理支持是术后未满足的护理需求。
本研究报告了脊柱侧弯手术对呼吸功能和对姿势和身体外观的整体满意度的积极影响。观察到对其他结果的负面影响突出了采用多学科方法改善术后管理的重要性。
