Sagittal spinopelvic alignment in ambulatory persons with cerebral palsy.

PURPOSE: This study aimed to describe the spinopelvic alignment of a cohort of young ambulatory individuals with cerebral palsy (CP) and compare it to published spinopelvic alignment data for the typically developing adolescents. METHODS: Thirty-seven adolescents (18 females) with CP at GMFCS I-III were included in this retrospective case series. Lumbar lordosis and pelvic incidence were measured, and their mismatch was calculated. A model that calculates predicted lumbar lordosis based on pelvic incidence in normative data was utilized to calculate a predicted lumbar lordosis in this cohort with cerebral palsy. RESULTS: At imaging, ages were mean and standard deviation 13.5 ± 3.0 years. Pelvic incidence was 46.2° ± 12.9°, pelvic tilt was 2.8° ± 9.4°, sacral slope was 43.6° ± 10.8°, and measured lumbar lordosis was 59.4° ± 11.6°. There were no differences in pelvic incidence or lumbar lordosis among the GMFCS levels; however, pelvic incidence was higher in females. Pelvic incidence-lumbar lordosis mismatch greater than 10° was found in 67% of the cohort. Mean predicted lumbar lordosis based on the model was 54.7° ± 8.5°, averaging 8° less than measured lordosis. CONCLUSION: PI-LL mismatch was identified in 67% of this cohort of ambulatory adolescents with CP, in part due to greater lordosis than predicted by a model based on data from adolescents without CP. The implications of this finding, such as the correlation between sagittal spinopelvic alignment and quality of life in this population, should be assessed further in ambulatory patients with cerebral palsy. LEVEL OF EVIDENCE: Level IV-retrospective cohort study and literature comparison.

Degree of Postoperative Curve Correction Decreases Risks of Postoperative Pneumonia in Patients Undergoing Both Fusion and Growth-friendly Surgical Treatment of Neuromuscular Scoliosis.

INTRODUCTION: Due to a combination of poor respiratory muscle control and mechanical lung compression secondary to spine and chest wall deformities, patients with neuromuscular (NM) early-onset scoliosis (EOS) are at a high risk for pulmonary complications including pneumonia. The purpose of this study is to examine the effect of surgical intervention on the prevalence and risk of postoperative pneumonia in patients with NM EOS. METHODS: In this retrospective cohort study, pediatric (18 y old and below) patients with NM EOS undergoing index fusion or growth-friendly instrumentation from 2000 to 2018 were identified. Patients were then categorized into 2 groups: those with ≥50% curve correction and those with <50% curve correction of the coronal deformity at the first postoperative visit. The primary outcome of interest was postoperative pneumonia occurring between 3 weeks and 2 years postoperatively. Manual chart review was supplemented with phone call surveys to ensure all occurrences of preoperative/postoperative pneumonia (ie, in-institution and out-of-institution visits) were accounted for. RESULTS: A total of 35 patients (31% female, age at surgery: 10.3±4.3 y) with NM EOS met inclusion criteria. Twenty-three (66%) patients experienced at least 1 case of preoperative pneumonia. Twenty-six (74%) patients had ≥50% and 9 (26%) patients had <50% immediate postoperative curve correction. In total, 12 (34%) patients experienced at least 1 case of postoperative pneumonia (7 in-institution, 5 out-of-institution). Seven (27%) patients with ≥50% curve correction versus 5 (56%) with <50% curve correction experienced postoperative pneumonia. Relative risk regression demonstrated that patients with <50% curve correction had increased risk of postoperative pneumonia by 2.1 times compared with patients with ≥50% curve correction (95% confidence interval: 0.9; 4.9, P =0.099). CONCLUSION: The prevalence of preoperative and postoperative pneumonia is high in patients with NM EOS. This study presents preliminary evidence suggesting that percent curve correction is associated with the occurrence of postoperative pneumonia in patients with NM EOS undergoing surgical correction.

Development and Validation of a Health-Related Quality-of-Life Measure in Older Children and Adolescents with Early-Onset Scoliosis: Early-Onset Scoliosis Self-Report Questionnaire (EOSQ-SELF).

BACKGROUND: The 24-question Early-Onset Scoliosis Questionnaire (EOSQ-24) is a proxy measure assessing health-related quality of life (HRQoL) among patients with early-onset scoliosis (EOS). There exists an increasing need to assess HRQoL through a child's own perspective, particularly for older children and adolescents with EOS. The purpose of this study was to develop and validate a self-reported questionnaire, the Early-Onset Scoliosis Self-Report Questionnaire (EOSQ-SELF), to assess HRQoL in older children and adolescents with EOS. METHODS: A literature review, an expert focus group, and patient interviews were used to generate a preliminary survey of appropriate domains and question items. This survey was provided to English-speaking patients with EOS who were 8 to 18 years of age and capable of answering survey questions. Content validity was assessed for clarity and relevance of questions. Confirmatory factors analysis was performed to reduce the number of items and determine domains that fit items. Reliability was evaluated by measuring the internal consistency of items and test-retest reliability. Construct validity was evaluated by convergent, discriminant, and known-group validity. RESULTS: The literature review, expert focus group, and patient interviews identified 59 questions in 14 domains. Psychometric analysis reduced these to 30 questions across 12 domains: General Health, Pain/Discomfort, Pulmonary Function, Transfer, Physical Function/Daily Living, Participation, Fatigue/Energy Level, Sleep, Appearance, Relationships, Emotion, and Satisfaction. The final questionnaire was found to have good content and construct validity and adequate reliability. CONCLUSIONS: The EOSQ-SELF is a valid and reliable instrument for measuring self-reported HRQoL among older children and adolescents with EOS (ages 8 to 18 years). This will serve as an important research outcome measure and enhance clinical care by providing a better understanding of HRQoL for these patients. LEVEL OF EVIDENCE: Diagnostic Level II . See Instructions for Authors for a complete description of levels of evidence.

Comparing health-related quality of life and burden of care between early-onset scoliosis patients treated with magnetically controlled growing rods and traditional growing rods: a multicenter study.

STUDY DESIGN: Multicenter retrospective cohort study. OBJECTIVES: To compare pre-operative and post-operative EOSQ-24 scores in magnetically controlled growing rods (MCGR) and traditional growing rod (TGR) patients. Since the introduction of MCGR, early-onset scoliosis patients have been afforded a reduction in the number of surgeries compared to the TGR technique. However, little is known about (health-related quality of life) and burden of care outcomes between these surgical techniques. METHODS: This is a retrospective cohort study using a multicenter registry on patients with EOS undergoing MCGR or TGR between 2008 and 2017. The EOSQ-24 was administered at preoperative and postoperative 2-year assessments. The EOSQ-24 scores were compared between MCGR and TGR as well as preoperatively and postoperatively within each procedure. RESULTS: 110 patients were analyzed in this study (TGR, N = 32; MCGR, N = 78). There were no significant differences in preoperative age, gender, etiology, main coronal curve or maximum kyphosis between TGR and MCGR groups. Patients with TGR had averaged 3.9 surgical lengthenings and MCGR had averaged 7.7 non-invasive lengthenings by the 2-year follow-up. When changes in preoperative to postoperative scores were compared, MCGR had more improvements in pain, emotion, child satisfaction and parent satisfaction than TGR although there were no statistical significance. When analyzed separately, MCGR cohort had improvement in scores for all four domains and four sub-domains; while, TGR cohort only had improvement in financial burden domain and pulmonary function sub-domain. CONCLUSION: Although there was no statistical significance, the improvement in pain, emotion and satisfaction scores was larger in MCGR than TGR. Since these areas can be influenced more by mental well-being than other sub-domains, the results may prove our hypothesis that compared to TGR, MCGR with reduced number of surgeries have better psychosocial effects. LEVEL OF EVIDENCE: III.

Serial casting for early onset scoliosis and its effects on health-related quality of life during and after discontinuation of treatment.

INTRODUCTION: Serial body casting plays an important role in the treatment of early onset scoliosis (EOS), serving as a safer method compared to surgical intervention. There is no published evidence documenting the impact of casting on health-related quality of life (HRQoL) of patients and their caregivers. The purpose of this study was to utilize the 24-Item Early Onset Scoliosis Questionnaire (EOSQ-24) to compare the HRQoL of patients with EOS and the burden of care for their caregivers before, during, and after treatment with Mehta casting. METHODS: In this multicenter retrospective cohort study, two EOS databases were queried for patients with EOS who underwent serial casting from 2005 to 2016. Patients who had treatment prior to their initial cast application, including bracing or surgical intervention, were excluded from the study. Patients were stratified into two subgroups and analyzed separately: those with idiopathic etiology, and those with non-idiopathic etiology. HRQoL and burden of care were assessed using the EOSQ-24 completed before, during, and after cast treatment. RESULTS: 91 patients were identified in this study (mean age at the index casting: 2.1 ± 1.2 years, mean age at final cast removal: 4.1 ± 1.3 years). 59 (64.8%) had EOS of idiopathic etiology, while 32 (35.2%) had EOS of non-idiopathic etiology, including 10 congenital, 6 neuromuscular, and 16 syndromic. Idiopathic patients and non-idiopathic patients experienced an improvement in the coronal deformity from 45° pre-cast to 26° post-cast, and from 59° pre-cast to 34° post-cast, respectively. Patients with idiopathic scoliosis experienced a decrease from baseline in nearly all EOSQ-24 sub-domain scores except for general health. Furthermore, even after the removal of the cast, patients with idiopathic etiology suffered residual negative effects of casting on emotion and transfer sub-domains. Among patients with non-idiopathic etiology, decreased scores from baseline were observed only in transfer and emotion sub-domains during the casting intervention. After brace removal, patients with non-idiopathic etiology had increased scores compared to their baseline scores in most sub-domains. DISCUSSION: Serial body casting can prevent curve correction in patients with EOS. However, the psychosocial stresses secondary to this non-operative intervention can have significant negative impacts on HRQoL for both idiopathic and non-idiopathic patients during the course of treatment. Although non-idiopathic patients experience improved HRQoL following treatment, idiopathic patients do not seem to exhibit much improvement in HRQoL from baseline even years after the final cast removal.

The contribution of human/non-human animal chimeras to stem cell research.

Chimeric animals are made up of cells from two separate zygotes. Human/non-human animal chimeras have been used for a number of research purposes, including human disease modeling. Pluripotent stem cell (PSC) research has relied upon the chimera approach to examine the developmental potential of stem cells, to determine the efficacy of cell replacement therapies, and to establish a means of producing human organs. Based on ethical issues, this work has faced pushback from various sources including funding agencies. We discuss here the essential role these studies have played, from gaining a better understanding of human biology to providing a stepping stone to human disease treatments. We also consider the major ethical issues, as well as the current status of support for this work in the United States.