生长友好型器械治疗脊髓性肌萎缩症后患者报告结局测量的肺功能改善。

Improvement of Pulmonary Function Measured by Patient-reported Outcomes in Patients With Spinal Muscular Atrophy After Growth-friendly Instrumentation.

机构信息

Department of Orthopedic Surgery, Columbia University Medical Center, New York, NY.

Children's Spine Foundation, Valley Forge.

出版信息

J Pediatr Orthop. 2021 Jan;41(1):1-5. doi: 10.1097/BPO.0000000000001656.

DOI:10.1097/BPO.0000000000001656

PMID:32804864

Abstract

BACKGROUND

Children with spinal muscular atrophy (SMA) sustain a progressive reduction in pulmonary function (PF) related to both muscular weakness and the concomitant effects of spinal deformity on the thorax. Growth-friendly instrumentation is commonly utilized for younger patients with scoliosis and SMA to halt the progression of spinal curvature, but its effect on PF in these patients has not previously been investigated. Using the change in Early Onset Scoliosis 24-Item Questionnaire (EOSQ-24) PF subdomain scores, the authors will investigate whether PF improves in patients with SMA after a growth-friendly intervention.

METHODS

This was a multicenter retrospective cohort study from 2 international registries of patients with SMA undergoing spinal deformity surgery from 2005 to 2015. Data collected were age, sex, degree of major coronal curve, type of growth-friendly construct, forced vital capacity (FVC), and EOSQ-24 scores at the patient's preoperative, 1-year postoperative, and 2-year postoperative visits. Differences in EOSQ-24 PF scores and FVC between baseline and postoperative assessment were examined by paired tests.

RESULTS

A total of 74 patients were identified (mean age, 7.6±2.3 y, major curve 68.1±22.4 degrees, 51.4% female individuals). The mean EOSQ-24 PF scores improved significantly from 70.6 preoperatively to 83.6 at 1 year (P=0.092) and 86.5 at 2 years postoperatively (P=0.020). The scores in patients with rib-based constructs showed steeper increases at 1-year assessments than those in patients with spine-based constructs. The mean paired FVC value decreased from 63.9% predicted preoperatively, to 57.6% predicted at 1 year postoperatively (P=0.035), and 61.9% predicted preoperatively, to 56.3% predicted at 2 years postoperatively (P=0.178).

CONCLUSIONS

Patients with SMA who received growth-friendly instrumentation did experience improvements in PF as measured by EOSQ-24 assessing the caregivers' perception. Given the uncertain reliability of PFTs in this young population, EOSQ-24 is an important tool for measuring improvements in health-related quality of life.

LEVEL OF EVIDENCE

Level III-retrospective study.

摘要

背景

脊髓性肌萎缩症（SMA）患儿的肺功能（PF）逐渐下降，这与肌肉无力以及脊柱畸形对胸廓的继发影响有关。脊柱侧凸患儿通常采用生长友好型器械进行治疗，以阻止脊柱弯曲的进展，但之前尚未研究过该器械对这些患者 PF 的影响。本研究作者将通过早期发病脊柱侧凸 24 项问卷（EOSQ-24）PF 亚量表评分的变化，探讨 SMA 患者在接受生长友好型干预后 PF 是否得到改善。

方法

这是一项来自 2 个国际 SMA 脊柱畸形手术患者登记处的多中心回顾性队列研究，研究对象为 2005 年至 2015 年期间接受脊柱畸形手术的 SMA 患者。收集的数据包括年龄、性别、主冠状面曲线度数、生长友好型器械类型、用力肺活量（FVC）和术前、术后 1 年及术后 2 年的 EOSQ-24 评分。通过配对检验比较 EOSQ-24 PF 评分和 FVC 在基线和术后评估之间的差异。

结果

共纳入 74 例患者（平均年龄 7.6±2.3 岁，主曲线 68.1±22.4 度，女性占 51.4%）。EOSQ-24 PF 评分从术前的 70.6 分显著改善至术后 1 年的 83.6 分（P=0.092）和术后 2 年的 86.5 分（P=0.020）。肋骨基构建构患者的评分在 1 年评估时的增高幅度大于脊柱基构建构患者。FVC 的平均配对值从术前的预计值的 63.9%降至术后 1 年的 57.6%（P=0.035），和术前的预计值的 61.9%降至术后 2 年的 56.3%（P=0.178）。