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

Matsumoto, Hiroko; Mueller, John D.; Königsberg, Matthew; Ball, Jacob; Hilaire, Tricia St.; Pawelek, Jeff; Roye, David P.; Cahill, Patrick J.; Sturm, Peter; Smith, John T.; Thompson, George H.; Sponseller, Paul D.; Skaggs, David L.; Vitale, Michael G.

doi:10.1097/bpo.0000000000001656

Cited by 8 publications

(6 citation statements)

References 15 publications

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“…Redding and Mayer have shown us that lung structure and function change with early-onset scoliosis and with early-onset scoliosis treatment 1,2 . We tend to rely on radiographs and patient-reported outcomes as our measures of success, with some good evidence that there is correlation 3 . This study by Wang et al not only identifies successful improvement in children with severe early-onset scoliosis, but also goes a step further in describing the timeline of radiographic and respiratory changes.…”

Section: Commentarymentioning

confidence: 99%

Waiting for Change: Pulmonary Function Improves in a Delayed Fashion Following Dual Growing-Rod Surgery for Early-Onset Scoliosis

Hardesty

2023

Journal of Bone and Joint Surgery

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Jess Lair, PhD, said, "Children are not things to be molded, but people to be unfolded." He clearly understood the fine line that we walk when we treat children with early-onset scoliosis. We need to intervene in a way that helps the child to grow and maximizes lung capacity without doing so too early, too late, too aggressively, or not aggressively enough, with a limited fusion, but also just enough fusion to avoid progression. All the while, we wonder if these interventions really help children to live longer or better. Pulmonary function is a very objective way to evaluate our outcomes, but these data are difficult to obtain. Redding and Mayer have shown us that lung structure and function change with early-onset scoliosis and with early-onset scoliosis treatment 1,2 . We tend to rely on radiographs and patient-reported outcomes as our measures of success, with some good evidence that there is correlation 3 . This study by Wang et al. not only identifies successful improvement in children with severe early-onset scoliosis, but also goes a step further in describing the timeline of radiographic and respiratory changes.Notably, the pulmonary function at 1 year following original dual growing-rod surgery improved but was not significantly different from before the operation, although this was the time frame in which the radiographic parameters improved the most. The improvement in pulmonary function was delayed to the time between 1 year postoperatively and the final follow-up. The lungs may have taken a bit of time to grow or may have still been recovering from the surgical procedure, but improvement was ultimately seen: dual growing-rod surgery indeed resulted in improvements in lung function demonstrated by pulmonary function testing.One concern with regard to this study is the ability of children to participate in pulmonary function testing, and another concern is the flexibility of curves that improved from 104°to 38°following the index surgical procedure. Also, the cohort was heterogeneous, and these results cannot be universally applied to all children with early-onset scoliosis, as some diagnoses will not show the same response.Although we as pediatric orthopaedic surgeons do our best to let children "unfold" over their natural course, sometimes we do have to "mold" them a bit, and it is reassuring to know that we ultimately help create healthier adults with better lung function than they would have had without intervention.

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Section: Commentarymentioning

confidence: 99%

Waiting for Change: Pulmonary Function Improves in a Delayed Fashion Following Dual Growing-Rod Surgery for Early-Onset Scoliosis

Hardesty

2023

Journal of Bone and Joint Surgery

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“…134 The effect on pulmonary function metrics after spine surgery is variable; postoperative decrease in pulmonary function (e.g., from 61.9%-63.9% predicted FVC preoperatively to 56.3%-57.6% postoperatively), increase, and no change have all been reported. 22,23,30,31,74,[135][136][137][138][139][140] Postoperative reduction in the rate of decline of pulmonary function (e.g., from FVC decline of 5.31% per year preoperatively to 1.77% per year postoperatively) has also been described. Even with variable FVC outcomes, healthrelated quality of life pulmonary function subscores improve after growth-friendly instrumentation 140 (example in Figure 2).…”

Section: Chest Deformity Thoracic Insufficiency and Pulmonary Healthmentioning

confidence: 99%

“…22,23,30,31,74,[135][136][137][138][139][140] Postoperative reduction in the rate of decline of pulmonary function (e.g., from FVC decline of 5.31% per year preoperatively to 1.77% per year postoperatively) has also been described. Even with variable FVC outcomes, healthrelated quality of life pulmonary function subscores improve after growth-friendly instrumentation 140 (example in Figure 2).…”

Section: Chest Deformity Thoracic Insufficiency and Pulmonary Healthmentioning

confidence: 99%

Best Practices for the Orthopaedic Care of Children with Spinal Muscular Atrophy: A Consensus Statement from the European Neuromuscular Centre Standard of Care Orthopaedic Working Group

Vitale¹,

Roye²,

Bloom³

et al. 2022

Journal of the Pediatric Orthopaedic Society of North America

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Purpose: Spinal muscular atrophy (SMA) is a genetic disease resulting in orthopaedic problems that warrant intervention. Recent genetic therapies appear to improve the natural course of this disease, prompting care providers to rethink treatment strategies. Insufficient high-quality evidence exists to guide optimal intervention in this new landscape. To address this, the European Neuromuscular Centre (ENMC) tasked an orthopaedic subgroup to update previous recommendations. Materials and Methods: A consensus statement on orthopaedic treatment of patients with SMA undergoing genetic therapy was sought via Delphi method. After review of the orthopaedic literature, three iterative surveys were administered from 2015-2016 to worldwide experts in orthopaedic care of children with SMA. Surveys identified important topics for optimal management, solicited responses regarding management and evaluation of identified topics, and clarified decision-making strategies and indications for evaluation and management, respectively. In light of new evidence of the long-term effects of gene therapy on SMA patients, an additional review of literature was used to contextualize the consensus recommendations. Results: Thirteen experts on orthopaedic management of SMA identified treatment of spine deformity, hip instability, and joint contractures as issues of importance. Experts recommended instrumentation in spinal deformity for major curves greater than 50 degrees, while monitoring pain, respiratory function, kyphosis, pelvic obliquity, functional status, trunk imbalance, and rib deformity. Preferential use of magnetically controlled growth constructs and performance of final fusion with skipped levels to accommodate intrathecal access were also endorsed. Additionally, experts advocated for surgical correction of hip instability and/or contractures that cause pain or functional impairment. Conclusions: Practice guidelines for management of scoliosis, hip instability and joint contractures were created to aid clinical decision-making in the era of genetic therapy.

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“…deformity correction. Dual growing rods (DGRs) have obvious advantages in controlling the progress of scoliosis and maintaining the development of pulmonary function compared with a single growing rod, especially for sEOS [3][4][5][6][7] . However, the long-term correction effects of DGRs in patients with sEOS and the changes in pulmonary function have yet to be clarified.…”

mentioning

confidence: 99%

Long-Term Radiographic and Pulmonary Function Outcomes After Dual Growing-Rod Treatment for Severe Early-Onset Scoliosis

Wang

Hai

Kang

et al. 2023

Journal of Bone and Joint Surgery

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Background:We evaluated long-term outcomes (radiographic parameters and pulmonary function) at a minimum follow-up of 5 years after use of dual growing rods (DGRs) to treat severe early-onset scoliosis (sEOS).Methods:Among a total of 112 patients who were diagnosed with early-onset scoliosis (EOS) and were treated with DGRs between 2006 and 2015, 52 patients had sEOS, with a major Cobb angle of >80°. Of these patients, 39 with a minimum follow-up of 5 years had complete radiographic and pulmonary function test results and were included. The Cobb angle of the major curve, T1-S1 height, T1-T12 height, and maximum kyphosis angle in the sagittal plane were measured on radiographs. Pulmonary function test results were collected in all patients before the initial operation (preoperatively), 12 months after the initial operation (postoperatively), and at the last follow-up. The changes in pulmonary function and complications during treatment were analyzed.Results:The mean age of patients before the initial operation was 7.7 ± 1.2 years, and the mean follow-up period was 75.0 ± 14.1 months. The mean number of lengthenings was 4.5 ± 1.3, and the mean interval between lengthenings was 11.2 ± 2.1 months. The Cobb angle improved from 104.5° ± 18.2° preoperatively to 38.1° ± 10.1° after the initial surgical procedure (postoperatively) and 21.9° ± 8.6° at the final follow-up. The T1-S1 height increased from 25.1 ± 4.0 cm preoperatively to 32.4 ± 3.5 cm postoperatively and to 39.5 ± 4.0 cm at the final follow-up. However, no significant difference was detected between the increased pulmonary function parameters at 1 year and those before the operation (p > 0.05), except for residual volume, whereas pulmonary function parameters had significantly increased at the final follow-up (p < 0.05). During the treatment period, 17 complications occurred in 12 patients.Conclusions:DGRs are effective in treating sEOS in the long term. They allow longitudinal growth of the spine, and the correction of the spinal deformity can provide conditions that make improving pulmonary function possible in patients with sEOS.Level of Evidence:Therapeutic Level IV. See Instructions for Authors for a complete description of levels of evidence.

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

Cited by 8 publications

References 15 publications

Waiting for Change: Pulmonary Function Improves in a Delayed Fashion Following Dual Growing-Rod Surgery for Early-Onset Scoliosis

Waiting for Change: Pulmonary Function Improves in a Delayed Fashion Following Dual Growing-Rod Surgery for Early-Onset Scoliosis

Best Practices for the Orthopaedic Care of Children with Spinal Muscular Atrophy: A Consensus Statement from the European Neuromuscular Centre Standard of Care Orthopaedic Working Group

Long-Term Radiographic and Pulmonary Function Outcomes After Dual Growing-Rod Treatment for Severe Early-Onset Scoliosis

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