Browsing by Subject "Follow-Up Studies"
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Item Open Access A clinical prediction model for long-term functional outcome after traumatic spinal cord injury based on acute clinical and imaging factors.(Journal of neurotrauma, 2012-09) Wilson, Jefferson R; Grossman, Robert G; Frankowski, Ralph F; Kiss, Alexander; Davis, Aileen M; Kulkarni, Abhaya V; Harrop, James S; Aarabi, Bizhan; Vaccaro, Alexander; Tator, Charles H; Dvorak, Marcel; Shaffrey, Christopher I; Harkema, Susan; Guest, James D; Fehlings, Michael GTo improve clinicians' ability to predict outcome after spinal cord injury (SCI) and to help classify patients within clinical trials, we have created a novel prediction model relating acute clinical and imaging information to functional outcome at 1 year. Data were obtained from two large prospective SCI datasets. Functional independence measure (FIM) motor score at 1 year follow-up was the primary outcome, and functional independence (score ≥ 6 for each FIM motor item) was the secondary outcome. A linear regression model was created with the primary outcome modeled relative to clinical and imaging predictors obtained within 3 days of injury. A logistic model was then created using the dichotomized secondary outcome and the same predictor variables. Model validation was performed using a bootstrap resampling procedure. Of 729 patients, 376 met the inclusion criteria. The mean FIM motor score at 1 year was 62.9 (±28.6). Better functional status was predicted by less severe initial American Spinal Injury Association (ASIA) Impairment Scale grade, and by an ASIA motor score >50 at admission. In contrast, older age and magnetic resonance imaging (MRI) signal characteristics consistent with spinal cord edema or hemorrhage predicted worse functional outcome. The linear model predicting FIM motor score demonstrated an R-square of 0.52 in the original dataset, and 0.52 (95% CI 0.52,0.53) across the 200 bootstraps. Functional independence was achieved by 148 patients (39.4%). For the logistic model, the area under the curve was 0.93 in the original dataset, and 0.92 (95% CI 0.92,0.93) across the bootstraps, indicating excellent predictive discrimination. These models will have important clinical impact to guide decision making and to counsel patients and families.Item Open Access A community-based intervention increases physical activity and reduces obesity in school-age children in North Carolina.(Child Obes, 2015-06) Benjamin Neelon, Sara E; Namenek Brouwer, Rebecca J; Østbye, Truls; Evenson, Kelly R; Neelon, Brian; Martinie, Annie; Bennett, GaryBACKGROUND: Community-based interventions are promising approaches to obesity prevention, but few studies have prospectively evaluated them. The aim of this study was to evaluate a natural experiment—a community intervention designed to promote active living and decrease obesity within a small southern town. METHODS: In 2011, community leaders implemented the Mebane on the Move intervention—a community-wide effort to promote physical activity (PA) and decrease obesity among residents of Mebane, North Carolina. We measured child PA and BMI before and after the intervention, using a nearby town not implementing an intervention as the comparison. In total, we assessed 64 children from Mebane and 40 from the comparison community 6 months before, as well as 34 and 18 children 6 months after the intervention. We assessed PA with accelerometers worn for 7 days and calculated BMI z-scores using children's height and weight. We conducted multivariable linear regressions examining pre- to postintervention change in minutes of PA and BMI z-score, adjusting for confounders. RESULTS: At follow-up, children in Mebane modestly increased their moderate-to-vigorous PA (1.3 minutes per hour; 95% confidence interval (CI): 0.2, 2.3; p=0.03) and vigorous activity (0.8 minutes per hour; 95% CI: 0.1, 1.5; p=0.04) more than comparison children. In intervention children, BMI z-scores decreased 0.5 units (kg/m(2); 95% CI: -0.9, -0.02; p=0.045), compared to children in the comparison community. CONCLUSIONS: We observed positive effects on PA level and weight status of children in Mebane, despite high rates of attrition, suggesting that the community-based intervention may have been successful.Item Open Access A comparative analysis of the prevalence and characteristics of cervical malalignment in adults presenting with thoracolumbar spine deformity based on variations in treatment approach over 2 years.(European spine journal : official publication of the European Spine Society, the European Spinal Deformity Society, and the European Section of the Cervical Spine Research Society, 2016-08) Jalai, Cyrus M; Passias, Peter G; Lafage, Virginie; Smith, Justin S; Lafage, Renaud; Poorman, Gregory W; Diebo, Bassel; Liabaud, Barthélemy; Neuman, Brian J; Scheer, Justin K; Shaffrey, Christopher I; Bess, Shay; Schwab, Frank; Ames, Christopher P; International Spine Study Group (ISSG)Purpose
Characteristics specific to cervical deformity (CD) concomitant with adult thoracolumbar deformity (TLD) remains uncertain, particularly regarding treatment. This study identifies cervical malalignment prevalence following surgical and conservative TLD treatment through 2 years.Methods
Retrospective analysis of a prospective, multicenter adult spinal deformity (ASD) database. CD was defined in operative and non-operative ASD patients according to the following criteria: T1 Slope minus Cervical Lordosis (T1S-CL) ≥20°, C2-C7 Cervical Sagittal Vertical Axis (cSVA) ≥40 mm, C2-C7 kyphosis >10°. Differences in rates, demographics, health-related quality of life (HRQoL) scores for Oswestry Disability Index (ODI) and Scoliosis Research Society Questionnaire (SRS-22r), and radiographic variables were assessed between treatment groups (Op vs. Non-Op) and follow-up periods (baseline, 1-year, 2-year).Results
Three hundred and nineteen (200 Op, 199 Non-Op) ASD patients were analyzed. Op patients' CD rates at 1 and 2 years were 78.9, and 63.0 %, respectively. Non-Op CD rates were 21.1 and 37.0 % at 1 and 2 years, respectively. T1S-CL mismatch and cSVA malalignment characterized Op CD at 1 and 2 years (p < 0.05). Op and Non-Op CD groups had similar cervical/global alignment at 1 year (p > 0.05 for all), but at 2 years, Op CD patients had worse thoracic kyphosis (TK), T1S-CL, CL, cSVA, C2-T3 SVA, and global SVA compared to Non-Ops (p < 0.05). Op CD patients had worse ODI, and SRS Activity at 1 and 2 years post-operative (p < 0.05), but had greater 2-year SRS Satisfaction scores (p = 0.019).Conclusions
In the first study to compare cervical malalignment at extended follow-up between ASD treatments, CD rates rose overall through 2 years. TLD surgery, resulting in higher CD rates characterized by T1S-CL and cSVA malalignment, produced poorer HRQoL. This information can aid in treatment method decision-making when cervical deformity is present concomitant with TLD.Item Open Access A neural biomarker of psychological vulnerability to future life stress.(Neuron, 2015-02-04) Swartz, J; Knodt, A; Radtke, S; Hariri, AWe all experience a host of common life stressors such as the death of a family member, medical illness, and financial uncertainty. While most of us are resilient to such stressors, continuing to function normally, for a subset of individuals, experiencing these stressors increases the likelihood of developing treatment-resistant, chronic psychological problems, including depression and anxiety. It is thus paramount to identify predictive markers of risk, particularly those reflecting fundamental biological processes that can be targets for intervention and prevention. Using data from a longitudinal study of 340 healthy young adults, we demonstrate that individual differences in threat-related amygdala reactivity predict psychological vulnerability to life stress occurring as much as 1 to 4 years later. These results highlight a readily assayed biomarker, threat-related amygdala reactivity, which predicts psychological vulnerability to commonly experienced stressors and represents a discrete target for intervention and prevention.Item Open Access A Novel Tool for Deformity Surgery Planning: Determining the Magnitude of Lordotic Correction Required to Achieve a Desired Sagittal Vertical Axis.(World neurosurgery, 2017-08) Goldschmidt, Ezequiel; Angriman, Federico; Agarwal, Nitin; Zhou, James; Chen, Katherine; Tempel, Zachary J; Gerszten, Peter C; Kanter, Adam S; Okonkwo, David O; Passias, Peter; Scheer, Justin; Protopsaltis, Themistocles; Lafage, Virginie; Lafage, Renaud; Schwab, Frank; Bess, Shay; Ames, Christopher; Smith, Justin S; Shaffrey, Christopher I; Miller, Emily; Jain, Amit; Neuman, Brian; Sciubba, Daniel M; Burton, Douglas; Hamilton, D Kojo; International Spine Study GroupObjective
We sought to create a model capable of predicting the magnitude of pelvic incidence-lumbar lordosis (PI-LL) correction necessary to achieve a desired change in sagittal vertical axis (SVA).Methods
A retrospective review was conducted of a prospectively maintained multicenter adult spinal deformity database collected by the International Spine Study Group between 2009 and 2014. The independent variable of interest was the degree of correction achieved in the PI-LL mismatch 6 weeks after surgery. Primary outcome was the change in global sagittal alignment 6 weeks and 1 year after surgery. We used a linear mixed-effects model to determine the extent to which corrections in the PI-LL relationship affected postoperative changes in SVA.Results
A total of 1053 adult patients were identified. Of these patients, 590 were managed surgically. Eighty-seven surgically managed patients were excluded because of incomplete or missing PI-LL measurements on follow-up; the remaining 503 patients were selected for inclusion. For each degree of improvement in the PI-LL mismatch at 6 weeks, the SVA decreased by 2.18 mm (95% confidence interval, -2.56, -1.79; P < 0.01) and 1.67 mm (95% confidence interval, -2.07, -1.27; P < 0.01) at 6 weeks and 12 months, respectively. A high SVA measurement (>50 mm) 1 year after surgery was negatively associated with health-related quality of life as measured by the Scoliosis Research Society 22 outcomes assessment.Conclusions
We describe a novel model that shows how surgical correction of the PI-LL relationship affects postoperative changes in SVA. This model may enable surgeons to determine preoperatively the amount of LL necessary to achieve a desired change in SVA.Item Open Access A Phase II Randomized Clinical Trial of the Safety and Efficacy of Intravenous Umbilical Cord Blood Infusion for Treatment of Children with Autism Spectrum Disorder.(The Journal of pediatrics, 2020-07) Dawson, Geraldine; Sun, Jessica M; Baker, Jennifer; Carpenter, Kimberly; Compton, Scott; Deaver, Megan; Franz, Lauren; Heilbron, Nicole; Herold, Brianna; Horrigan, Joseph; Howard, Jill; Kosinski, Andrzej; Major, Samantha; Murias, Michael; Page, Kristin; Prasad, Vinod K; Sabatos-DeVito, Maura; Sanfilippo, Fred; Sikich, Linmarie; Simmons, Ryan; Song, Allen; Vermeer, Saritha; Waters-Pick, Barbara; Troy, Jesse; Kurtzberg, JoanneObjective
To evaluate whether umbilical cord blood (CB) infusion is safe and associated with improved social and communication abilities in children with autism spectrum disorder (ASD).Study design
This prospective, randomized, placebo-controlled, double-blind study included 180 children with ASD, aged 2-7 years, who received a single intravenous autologous (n = 56) or allogeneic (n = 63) CB infusion vs placebo (n = 61) and were evaluated at 6 months postinfusion.Results
CB infusion was safe and well tolerated. Analysis of the entire sample showed no evidence that CB was associated with improvements in the primary outcome, social communication (Vineland Adaptive Behavior Scales-3 [VABS-3] Socialization Domain), or the secondary outcomes, autism symptoms (Pervasive Developmental Disorder Behavior Inventory) and vocabulary (Expressive One-Word Picture Vocabulary Test). There was also no overall evidence of differential effects by type of CB infused. In a subanalysis of children without intellectual disability (ID), allogeneic, but not autologous, CB was associated with improvement in a larger percentage of children on the clinician-rated Clinical Global Impression-Improvement scale, but the OR for improvement was not significant. Children without ID treated with CB showed significant improvements in communication skills (VABS-3 Communication Domain), and exploratory measures including attention to toys and sustained attention (eye-tracking) and increased alpha and beta electroencephalographic power.Conclusions
Overall, a single infusion of CB was not associated with improved socialization skills or reduced autism symptoms. More research is warranted to determine whether CB infusion is an effective treatment for some children with ASD.Item Open Access A pilot study on mindfulness based stress reduction for smokers.(BMC complementary and alternative medicine, 2007-01-25) Davis, James M; Fleming, Michael F; Bonus, Katherine A; Baker, Timothy BBackground
Mindfulness means paying attention in the present moment, non-judgmentally, without commentary or decision-making. We report results of a pilot study designed to test the feasibility of using Mindfulness Based Stress Reduction (MBSR) (with minor modifications) as a smoking intervention.Methods
MBSR instructors provided instructions in mindfulness in eight weekly group sessions. Subjects attempted smoking cessation during week seven without pharmacotherapy. Smoking abstinence was tested six weeks after the smoking quit day with carbon monoxide breath test and 7-day smoking calendars. Questionnaires were administered to evaluate changes in stress and affective distress.Results
18 subjects enrolled in the intervention with an average smoking history of 19.9 cigarettes per day for 26.4 years. At the 6-week post-quit visit, 10 of 18 subjects (56%) achieved biologically confirmed 7-day point-prevalent smoking abstinence. Compliance with meditation was positively associated with smoking abstinence and decreases in stress and affective distress.Discussions and conclusion
The results of this study suggest that mindfulness training may show promise for smoking cessation and warrants additional study in a larger comparative trial.Item Open Access A Radiographic Analysis of Lumbar Fusion Status and Instrumentation Failure After Complex Adult Spinal Deformity Surgery With Spinopelvic Fixation: Two-Year Follow-up From the Scoli-Risk-1 Prospective Database.(Clinical spine surgery, 2020-12) Shimizu, Takayoshi; Lenke, Lawrence G; Cerpa, Meghan; Beauchamp, Eduardo C; Carreon, Leah Y; Shaffrey, Christopher I; Cheung, Kenneth MC; Fehlings, Michael GStudy design
A retrospective review of prospectively collected data.Objective
The objective of this study was to investigate the fusion status of the lumbar spine and lumbosacral junction at 2 years postoperatively after complex adult spinal deformity (ASD) surgery.Summary of background data
Achieving fusion is crucial for maintaining optimal alignment in ASD surgery. However, prospective data assessing fusion status using large patient populations are lacking in this patient population.Materials and methods
Postoperative radiographs of 162 patients from the Scoli-Risk-1 database, who underwent complex ASD surgery with fusion to the sacrum, were evaluated by 3 independent spine surgeons at 6-week, 6-month, and 2-year follow-up. The fusion rate of the lumbar spine segments at a 2-year follow-up was determined by using previously published radiographic grading criteria. We also assessed the prevalence of instrumentation failures.Results
The interrater reliabilities for grading the fusion status were overall fair at each level evaluated (Fleiss κ, 0.337-0.439). Overall, 70.3% (114/162) demonstrated the solid fusion of the entire lumbar spine at a 2-year follow-up. The fusion rates of each segment were L1/L2: 87.0%, L2/L3: 82.0%, L3/L4: 83.9%, L4/L5: 89.5%, and L5/S1: 89.5%. Pedicle screw loosening was the most frequent implant failure throughout the observation period (9.2%, 11.6%, and 11.0% at 6-wk, 6-mo, and 2-y follow-up, respectively). No rod breakage was observed at 6 weeks, increasing to 9.8% at 2-year follow-up. The prevalence of postoperative proximal junctional kyphosis was 5.5% at 6 weeks, showing no difference at 2 years postoperative.Conclusions
In this series of complex ASD surgeries often requiring 3-column osteotomies, 70.3% showed solid fusion of the entire lumbar spine, including the lumbosacral junction. The lumbosacral segments showed a relatively high fusion rate at a 2-year follow-up likely due to the frequent use of anterior column support and graft. The prevalence of rod breakage increased as follow-up proceeded to 9.8%, which was most commonly observed at the lumbosacral junction.Level of evidence
Level IV.Item Open Access A TGF-β1 genetic variant at the miRNA187 binding site significantly modifies risk of HPV16-associated oropharyngeal cancer.(International journal of cancer, 2018-09) Tao, Ye; Sturgis, Erich M; Huang, Zhigang; Sun, Yan; Dahlstrom, Kristina R; Wei, Qingyi; Li, GuojunTGF-β1rs1982073 polymorphism at the miRNA-187 binding site may alter TGF-β1 expression and function, and thereby this polymorphism (genotype CT/CC) increases cancer susceptibility. HPV16 L1 seropositivity is associated with the risk of oral squamous cell carcinoma (OSCC), including oropharyngeal squamous cell carcinoma (OPSCC) and oral cavity squamous cell carcinoma (OCSCC). Thus, we hypothesized that TGF-β1rs1982073 polymorphism at the miRNA-187 binding site combined with HPV16 L1 seropositivity may have a joint effect on OSCC susceptibility. We determined the genotypes of TGF-β1rs1982073 and HPV16 status in 325 OSCC subjects and 335 cancer-free controls in the non-Hispanic white population, and used logistic regression models to evaluate the joint effects on OSCC susceptibility. TGF-β1rs1982073 polymorphism (CT/CC genotype) combined with HPV16 L1 seropositivity increased the risk of OSCC via joint effects, particularly in OPSCC subjects who were never-smokers (OR, 165.9; 95% CI, 28.6-960.4) or never-drinkers (OR, 196.0; 95% CI, 28.2-1,000.0), respectively. Younger subjects had a higher risk of OPSCC than older subjects (OR, 23.5; 95% CI, 6.3-87.0 vs. OR, 6.0; 95% CI, 1.7-17.9, respectively). The significant associations between this polymorphism and HPV16-associated OSCC and OPSCC were also observed. However, OCSCC subjects did not have similar results. Our findings suggest that the joint effects of TGF-β1rs1982073 and HPV16 L1 seropositivity can increase risk of HPV16-associated oral cancer, particularly in OPSCC subjects who are never-smokers, never-drinkers and young. This result may help us understand the tumorigenesis process and improve early detection, which are critical for prevention and intervention strategies. However, larger studies are needed to validate our findings.Item Open Access Adult Scoliosis Deformity Surgery: Comparison of Outcomes Between One Versus Two Attending Surgeons.(Spine, 2017-07) Gomez, Jaime A; Lafage, Virginie; Sciubba, Daniel M; Bess, Shay; Mundis, Gregory M; Liabaud, Barthelemy; Hanstein, Regina; Shaffrey, Christopher; Kelly, Michael; Ames, Christopher; Smith, Justin S; Passias, Peter G; Errico, Thomas; Schwab, Frank; International Spine Study GroupStudy design
Retrospective review of prospectively collected data.Objective
Assess outcomes of adult spinal deformity (ASD) surgery performed by one versus two attending surgeons.Summary of background data
ASD centers have developed two attending teams to improve efficiency; their effects on complications and outcomes have not been reported.Methods
Patients with ASD with five or more levels fused and more than 2-year follow-up were included. Estimated blood loss (EBL), length of stay (LOS), operating room (OR) time, complications, quality of life (Health Related Quality of Life), and x-rays were analyzed. Outcomes were compared between one-surgeon (1S) and two-surgeon (2S) centers. A deformity-matched cohort was analyzed.Results
A total of 188 patients in 1S and 77 in 2S group were included. 2S group patients were older and had worse deformity based on the Scoliosis Research Society-Schwab classification (P < 0.05). There were no significant differences in levels fused (P = 0.57), LOS (8.7 vs 8.9 days), OR time (445.9 vs 453.2 min), or EBL (2008 vs 1898 cm; P > 0.05). 2S patients had more three-column osteotomies (3CO; P < 0.001) and used less bone morphogenetic protein 2 (BMP-2; 79.9% vs 15.6%; P < 0.001). The 2S group had fewer intraoperative complications (1.3% vs 11.1%; P = 0.006). Postoperative (6 wk to 2 yr) complications were more frequent in the 2S group (4.8% vs 15.6%; P < 0.002). After matching for deformity, there were no differences in (9.1 vs 10.1 days), OR time (467.8 vs 508.4 min), or EBL (3045 vs 2247 cm; P = 0.217). 2S group used less BMP-2 (20.6% vs 84.8%; P < 0.001), had fewer intraoperative complications (P = 0.015) but postoperative complications due to instrumentation failure/pseudarthrosis were more frequent (P < 0.01).Conclusion
No significant differences were found in LOS, OR time, or EBL between the 1S and 2S groups, even when matching for severity of deformity. 2S group had less BMP-2 use, fewer intraoperative complications but more postoperative complications.Level of evidence
2.Item Open Access Adult Spinal Deformity Surgery Is Associated with Increased Productivity and Decreased Absenteeism From Work and School.(Spine, 2022-02) Durand, Wesley M; Babu, Jacob M; Hamilton, David K; Passias, Peter G; Kim, Han Jo; Protopsaltis, Themistocles; Lafage, Virginie; Lafage, Renaud; Smith, Justin S; Shaffrey, Christopher; Gupta, Munish; Kelly, Michael P; Klineberg, Eric O; Schwab, Frank; Gum, Jeffrey L; Mundis, Gregory; Eastlack, Robert; Kebaish, Khaled; Soroceanu, Alex; Hostin, Richard A; Burton, Doug; Bess, Shay; Ames, Christopher; Hart, Robert A; Daniels, Alan H; International Spine Study Group (ISSG)Study design
Retrospective cohort study.Objective
We hypothesized that adult spinal deformity (ASD) surgery would be associated with improved work- and school-related productivity, as well as decreased rates of absenteeism.Summary of background data
ASD patients experience markedly decreased health-related quality of life along many dimensions.Methods
Only patients eligible for 2-year follow-up were included, and those with a history of previous spinal fusion were excluded. The primary outcome measures in this study were Scoliosis Research Society-22r score (SRS-22r) questions 9 and 17. A repeated measures mixed linear regression was used to analyze responses over time among patients managed operatively (OP) versus nonoperatively (NON-OP).Results
In total, 1188 patients were analyzed. 66.6% were managed operatively. At baseline, the mean percentage of activity at work/school was 56.4% (standard deviation [SD] 35.4%), and the mean days off from work/school over the past 90 days was 1.6 (SD 1.8). Patients undergoing ASD surgery exhibited an 18.1% absolute increase in work/school productivity at 2-year follow-up versus baseline (P < 0.0001), while no significant change was observed for the nonoperative cohort (P > 0.5). Similarly, the OP cohort experienced 1.1 fewer absent days over the past 90 days at 2 years versus baseline (P < 0.0001), while the NON-OP cohort showed no such difference (P > 0.3). These differences were largely preserved after stratifying by baseline employment status, age group, sagittal vertical axis (SVA), pelvic incidence minus lumbar lordosis (PI-LL), and deformity curve type.Conclusion
ASD patients managed operatively exhibited an average increase in work/school productivity of 18.1% and decreased absenteeism of 1.1 per 90 days at 2-year follow-up, while patients managed nonoperatively did not exhibit change from baseline. Given the age distribution of patients in this study, these findings should be interpreted as pertaining primarily to obligations at work or within the home. Further study of the direct and indirect economic benefits of ASD surgery to patients is warranted.Level of Evidence: 3.Item Open Access Age of first drunkenness and risks for all-cause mortality: A 27-year follow-up from the epidemiologic catchment area study.(Drug and alcohol dependence, 2017-07) Hu, Hui; Eaton, William W; Anthony, James C; Wu, Li-Tzy; Cottler, Linda BEarly-onset drunkenness is associated with an increased risk of developing an alcohol use disorder (AUD), which predicts excess mortality risk. Here, we estimated mortality risk for drinkers with and without early drunkenness.For 14,848 adult participants interviewed about drinking, drunken episodes, and AUD in 1981-83 for the Epidemiologic Catchment Area in New Haven (Connecticut), Baltimore (Maryland), St. Louis (Missouri), and Durham (North Carolina), we linked National Death Index records through 2007.Cox regression modeling estimates showed excess mortality for drinkers with age of first drunkenness earlier than 15 years old (hazard ratio, HR: 1.47, 95% CI: 1.25, 1.72) and when first drunkenness occurred at or after age 15 (HR: 1.20, 95% CI: 1.11, 1.29), as compared with adults who had never been drunk. Consistent results were observed, irrespective of AUD history. That is, early drunkenness signaled excess mortality risk even in absence of AUD.In a large community sample from four cities in the US, early age of onset of drunkenness predicts mortality risk. We discuss experiments to investigate the possible causal significance of this predictive association.Item Open Access Allogeneic hematopoietic SCT for alpha-mannosidosis: an analysis of 17 patients.(Bone marrow transplantation, 2012-03) Mynarek, M; Tolar, J; Albert, MH; Escolar, ML; Boelens, JJ; Cowan, MJ; Finnegan, N; Glomstein, A; Jacobsohn, DA; Kühl, JS; Yabe, H; Kurtzberg, J; Malm, D; Orchard, PJ; Klein, C; Lücke, T; Sykora, K-WAlpha-mannosidosis is a rare lysosomal storage disease. Hematopoietic SCT (HSCT) is usually recommended as a therapeutic option though reports are anecdotal to date. This retrospective multi institutional analysis describes 17 patients that were diagnosed at a median of 2.5 (1.1-23) years and underwent HSCT at a median of 3.6 (1.3-23.1) years. In all, 15 patients are alive (88%) after a median follow-up of 5.5 (2.1-12.6) years. Two patients died within the first 5 months after HSCT. Of the survivors, two developed severe acute GvHD (>=grade II) and six developed chronic GvHD. Three patients required re-transplantation because of graft failure. All 15 showed stable engraftment. The extent of the patients' developmental delay before HSCT varied over a wide range. After HSCT, patients made developmental progress, although normal development was not achieved. Hearing ability improved in some, but not in all patients. We conclude that HSCT is a feasible therapeutic option that may promote mental development in alpha-mannosidosis.Item Open Access An analysis from the Quality Outcomes Database, Part 1. Disability, quality of life, and pain outcomes following lumbar spine surgery: predicting likely individual patient outcomes for shared decision-making.(Journal of neurosurgery. Spine, 2017-10) McGirt, Matthew J; Bydon, Mohamad; Archer, Kristin R; Devin, Clinton J; Chotai, Silky; Parker, Scott L; Nian, Hui; Harrell, Frank E; Speroff, Theodore; Dittus, Robert S; Philips, Sharon E; Shaffrey, Christopher I; Foley, Kevin T; Asher, Anthony LOBJECTIVE Quality and outcomes registry platforms lie at the center of many emerging evidence-driven reform models. Specifically, clinical registry data are progressively informing health care decision-making. In this analysis, the authors used data from a national prospective outcomes registry (the Quality Outcomes Database) to develop a predictive model for 12-month postoperative pain, disability, and quality of life (QOL) in patients undergoing elective lumbar spine surgery. METHODS Included in this analysis were 7618 patients who had completed 12 months of follow-up. The authors prospectively assessed baseline and 12-month patient-reported outcomes (PROs) via telephone interviews. The PROs assessed were those ascertained using the Oswestry Disability Index (ODI), EQ-5D, and numeric rating scale (NRS) for back pain (BP) and leg pain (LP). Variables analyzed for the predictive model included age, gender, body mass index, race, education level, history of prior surgery, smoking status, comorbid conditions, American Society of Anesthesiologists (ASA) score, symptom duration, indication for surgery, number of levels surgically treated, history of fusion surgery, surgical approach, receipt of workers' compensation, liability insurance, insurance status, and ambulatory ability. To create a predictive model, each 12-month PRO was treated as an ordinal dependent variable and a separate proportional-odds ordinal logistic regression model was fitted for each PRO. RESULTS There was a significant improvement in all PROs (p < 0.0001) at 12 months following lumbar spine surgery. The most important predictors of overall disability, QOL, and pain outcomes following lumbar spine surgery were employment status, baseline NRS-BP scores, psychological distress, baseline ODI scores, level of education, workers' compensation status, symptom duration, race, baseline NRS-LP scores, ASA score, age, predominant symptom, smoking status, and insurance status. The prediction discrimination of the 4 separate novel predictive models was good, with a c-index of 0.69 for ODI, 0.69 for EQ-5D, 0.67 for NRS-BP, and 0.64 for NRS-LP (i.e., good concordance between predicted outcomes and observed outcomes). CONCLUSIONS This study found that preoperative patient-specific factors derived from a prospective national outcomes registry significantly influence PRO measures of treatment effectiveness at 12 months after lumbar surgery. Novel predictive models constructed with these data hold the potential to improve surgical effectiveness and the overall value of spine surgery by optimizing patient selection and identifying important modifiable factors before a surgery even takes place. Furthermore, these models can advance patient-focused care when used as shared decision-making tools during preoperative patient counseling.Item Open Access An analysis from the Quality Outcomes Database, Part 2. Predictive model for return to work after elective surgery for lumbar degenerative disease.(Journal of neurosurgery. Spine, 2017-10) Asher, Anthony L; Devin, Clinton J; Archer, Kristin R; Chotai, Silky; Parker, Scott L; Bydon, Mohamad; Nian, Hui; Harrell, Frank E; Speroff, Theodore; Dittus, Robert S; Philips, Sharon E; Shaffrey, Christopher I; Foley, Kevin T; McGirt, Matthew JOBJECTIVE Current costs associated with spine care are unsustainable. Productivity loss and time away from work for patients who were once gainfully employed contributes greatly to the financial burden experienced by individuals and, more broadly, society. Therefore, it is vital to identify the factors associated with return to work (RTW) after lumbar spine surgery. In this analysis, the authors used data from a national prospective outcomes registry to create a predictive model of patients' ability to RTW after undergoing lumbar spine surgery for degenerative spine disease. METHODS Data from 4694 patients who underwent elective spine surgery for degenerative lumbar disease, who had been employed preoperatively, and who had completed a 3-month follow-up evaluation, were entered into a prospective, multicenter registry. Patient-reported outcomes-Oswestry Disability Index (ODI), numeric rating scale (NRS) for back pain (BP) and leg pain (LP), and EQ-5D scores-were recorded at baseline and at 3 months postoperatively. The time to RTW was defined as the period between operation and date of returning to work. A multivariable Cox proportional hazards regression model, including an array of preoperative factors, was fitted for RTW. The model performance was measured using the concordance index (c-index). RESULTS Eighty-two percent of patients (n = 3855) returned to work within 3 months postoperatively. The risk-adjusted predictors of a lower likelihood of RTW were being preoperatively employed but not working at the time of presentation, manual labor as an occupation, worker's compensation, liability insurance for disability, higher preoperative ODI score, higher preoperative NRS-BP score, and demographic factors such as female sex, African American race, history of diabetes, and higher American Society of Anesthesiologists score. The likelihood of a RTW within 3 months was higher in patients with higher education level than in those with less than high school-level education. The c-index of the model's performance was 0.71. CONCLUSIONS This study presents a novel predictive model for the probability of returning to work after lumbar spine surgery. Spine care providers can use this model to educate patients and encourage them in shared decision-making regarding the RTW outcome. This evidence-based decision support will result in better communication between patients and clinicians and improve postoperative recovery expectations, which will ultimately increase the likelihood of a positive RTW trajectory.Item Open Access An Analysis of the Incidence and Outcomes of Major Versus Minor Neurological Decline After Complex Adult Spinal Deformity Surgery: A Subanalysis of Scoli-RISK-1 Study.(Spine, 2018-07) Kato, So; Fehlings, Michael G; Lewis, Stephen J; Lenke, Lawrence G; Shaffrey, Christopher I; Cheung, Kenneth MC; Carreon, Leah Y; Dekutoski, Mark B; Schwab, Frank J; Boachie-Adjei, Oheneba; Kebaish, Khaled M; Ames, Christopher P; Qiu, Yong; Matsuyama, Yukihiro; Dahl, Benny T; Mehdian, Hossein; Pellisé, Ferran; Berven, Sigurd HStudy design
A subanalysis from a prospective, multicenter, international cohort study in 15 sites (Scoli-RISK-1).Objective
To report detailed information regarding the severity of neurological decline related to complex adult spine deformity (ASD) surgery and to examine outcomes based on severity.Summary of background data
Postoperative neurological decline after ASD surgeries can occur due to nerve root(s) or spinal cord dysfunction. The impact of decline and the pattern of recovery may be related to the anatomic location and the severity of the injury.Methods
An investigation of 272 prospectively enrolled complex ASD surgical patients with neurological status measured by American Spinal Injury Association Lower Extremity Motor Scores (LEMS) was undertaken. Postoperative neurological decline was categorized into "major" (≥5 points loss) versus "minor" (<5 points loss) deficits. Timing and extent of recovery in LEMS were investigated for each group.Results
Among the 265 patients with LEMS available at discharge, 61 patients (23%) had neurological decline, with 20 (33%) experiencing major decline. Of note, 90% of the patients with major decline had deficits in three or more myotomes. Full recovery was seen in 24% at 6 weeks and increased to 65% at 6 months. However, 34% continued to experience some neurological decline at 24 months, with 6% demonstrating no improvement. Of 41 patients (67%) with minor decline, 73% had deficits in one or two myotomes. Full recovery was seen in 49% at 6 weeks and increased to 70% at 6 months. Of note, 26% had persistence of some neurological deficit at 24 months, with 18% demonstrating no recovery.Conclusion
In patients undergoing complex ASD correction, a rate of postoperative neurological decline of 23% was noted with 33% of these being "major." Although most patients showed substantial recovery by 6 months, approximately one-third continued to experience neurological dysfunction.Level of evidence
2.Item Open Access Analysis of Successful Versus Failed Radiographic Outcomes After Cervical Deformity Surgery.(Spine, 2018-07) Protopsaltis, Themistocles S; Ramchandran, Subaraman; Hamilton, D Kojo; Sciubba, Daniel; Passias, Peter G; Lafage, Virginie; Lafage, Renaud; Smith, Justin S; Hart, Robert A; Gupta, Munish; Burton, Douglas; Bess, Shay; Shaffrey, Christopher; Ames, Christopher P; International Spine Study Group (ISSG)Study design
Prospective multicenter cohort study with consecutive enrollment.Objective
To evaluate preoperative alignment and surgical factors associated with suboptimal early postoperative radiographic outcomes after surgery for cervical deformity.Summary of background data
Recent studies have demonstrated correlation between cervical sagittal alignment and patient-reported outcomes. Few studies have explored cervical deformity correction prospectively, and the factors that result in successful versus failed cervical alignment corrections remain unclear.Methods
Patients with adult cervical deformity (ACD) included with either cervical kyphosis more than 10°, C2-C7 sagittal vertical axis (cSVA) of more than 4 cm, or chin-brow vertical angle of more than 25°. Patients were categorized into failed outcomes group if cSVA of more than 4 cm or T1 slope and cervical lordosis (TS-CL) of more than 20° at 6 months postoperatively.Results
A total of 71 patients with ACD (mean age 62 yr, 56% women, 41% revisions) were included. Fourty-five had primary cervical deformities and 26 at the cervico-thoracic junction. Thirty-three (46.4%) had failed radiographic outcomes by cSVA and 46 (64.7%) by TS-CL. Failure to restore cSVA was associated with worse preoperative C2 pelvic tilt angle (CPT: 64.4° vs. 47.8°, P = 0.01), worse postoperative C2 slope (35.0° vs. 23.8°, P = 0.004), TS-CL (35.2° vs. 24.9°, P = 0.01), CPT (47.9° vs. 28.2°, P < 0.001), "+" Schwab modifiers (P = 0.007), revision surgery (P = 0.05), and failure to address the secondary, thoracolumbar driver of the deformity (P = 0.02). Failure to correct TS-CL was associated with worse preoperative cervical kyphosis (10.4° vs. -2.1°, P = 0.03), CPT (52.6° vs. 39.1°, P = 0.04), worse postoperative C2 slope (30.2° vs. 13.3°, P < 0.001), cervical lordosis (-3.6° vs. -15.1°, P = 0.01), and CPT (37.7° vs. 24.0°, P < 0.001). Multivariate analysis revealed postoperative distal junctional kyphosis associated with suboptimal outcomes by cSVA (odds ratio 0.06, confidence interval 0.01-0.4, P = 0.004) and TS-CL (odds ratio 0.15, confidence interval 0.02-0.97, P = 0.05).Conclusion
Factors associated with failure to correct the cSVA included revision surgery, worse preoperative CPT, and concurrent thoracolumbar deformity. Failure to correct the TS-CL mismatch was associated with worse preoperative cervical kyphosis and CPT. Occurrence of early postoperative distal junctional kyphosis significantly affects postoperative radiographic outcomes.Level of evidence
3.Item Open Access Anterior versus posterior surgical approaches to treat cervical spondylotic myelopathy: outcomes of the prospective multicenter AOSpine North America CSM study in 264 patients.(Spine, 2013-12) Fehlings, Michael G; Barry, Sean; Kopjar, Branko; Yoon, Sangwook Tim; Arnold, Paul; Massicotte, Eric M; Vaccaro, Alexander; Brodke, Darrel S; Shaffrey, Christopher; Smith, Justin S; Woodard, Eric; Banco, Robert J; Chapman, Jens; Janssen, Michael; Bono, Christopher; Sasso, Rick; Dekutoski, Mark; Gokaslan, Ziya LStudy design
A prospective observational multicenter study.Objective
To help solve the debate regarding whether the anterior or posterior surgical approach is optimal for patients with cervical spondylotic myelopathy (CSM).Summary of background data
The optimal surgical approach to treat CSM remains debated with varying opinions favoring anterior versus posterior surgical approaches. We present an analysis of a prospective observational multicenter study examining outcomes of surgical treatment for CSM.Methods
A total of 278 subjects from 12 sites in North America received anterior/posterior or combined surgery at the discretion of the surgeon. This study focused on subjects who had either anterior or posterior surgery (n = 264, follow-up rate, 87%). Outcome measures included the modified Japanese Orthopedic Assessment scale, the Nurick scale, the Neck Disability Index, and the Short-Form 36 (SF-36) Health Survey version 2 Physical and Mental Component Scores.Results
One hundred and sixty-nine patients were treated anteriorly and 95 underwent posterior surgery. Anterior surgical cases were younger and had less severe myelopathy as assessed by mJOA and Nurick scores. There were no baseline differences in Neck Disability Index or SF-36 between the anterior and posterior cases. Improvement in the mJOA was significantly lower in the anterior group than posterior group (2.47 vs. 3.62, respectively, P < 0.01), although the groups started at different levels of baseline impairment. The extent of improvement in the Nurick Scale, Neck Disability Index, SF-36 version 2 Physical Component Score, and SF-36 version 2 Mental Component Score did not differ between the groups.Conclusion
Patients with CSM show significant improvements in several health-related outcome measures with either anterior or posterior surgery. Importantly, patients treated with anterior techniques were younger, with less severe impairment and more focal pathology. We demonstrate for the first time that, when patient and disease factors are controlled for, anterior and posterior surgical techniques have equivalent efficacy in the treatment of CSM.Level of evidence
3.Item Open Access Assessing risk of breast cancer in an ethnically South-East Asia population (results of a multiple ethnic groups study).(BMC cancer, 2012-11-19) Gao, Fei; Machin, David; Chow, Khuan-Yew; Sim, Yu-Fan; Duffy, Stephen W; Matchar, David B; Goh, Chien-Hui; Chia, Kee-SengBackground
Gail and others developed a model (GAIL) using age-at-menarche, age-at-birth of first live child, number of previous benign breast biopsy examinations, and number of first-degree-relatives with breast cancer as well as baseline age-specific breast cancer risks for predicting the 5-year risk of invasive breast cancer for Caucasian women. However, the validity of the model for projecting risk in South-East Asian women is uncertain. We evaluated GAIL and attempted to improve its performance for Singapore women of Chinese, Malay and Indian origins.Methods
Data from the Singapore Breast Screening Programme (SBSP) are used. Motivated by lower breast cancer incidence in many Asian countries, we utilised race-specific invasive breast cancer and other cause mortality rates for Singapore women to produce GAIL-SBSP. By using risk factor information from a nested case-control study within SBSP, alternative models incorporating fewer then additional risk factors were determined. Their accuracy was assessed by comparing the expected cases (E) with the observed (O) by the ratio (E/O) and 95% confidence interval (CI) and the respective concordance statistics estimated.Results
From 28,883 women, GAIL-SBSP predicted 241.83 cases during the 5-year follow-up while 241 were reported (E/O=1.00, CI=0.88 to 1.14). Except for women who had two or more first-degree-relatives with breast cancer, satisfactory prediction was present in almost all risk categories. This agreement was reflected in Chinese and Malay, but not in Indian women. We also found that a simplified model (S-GAIL-SBSP) including only age-at-menarche, age-at-birth of first live child and number of first-degree-relatives performed similarly with associated concordance statistics of 0.5997. Taking account of body mass index and parity did not improve the calibration of S-GAIL-SBSP.Conclusions
GAIL can be refined by using national race-specific invasive breast cancer rates and mortality rates for causes other than breast cancer. A revised model containing only three variables (S-GAIL-SBSP) provides a simpler approach for projecting absolute risk of invasive breast cancer in South-East Asia women. Nevertheless its role in counseling the individual women regarding their risk of breast cancer remains problematical and needs to be validated in independent data.Item Open Access Assessment of a Novel Adult Cervical Deformity Frailty Index as a Component of Preoperative Risk Stratification.(World neurosurgery, 2018-01) Miller, Emily K; Ailon, Tamir; Neuman, Brian J; Klineberg, Eric O; Mundis, Gregory M; Sciubba, Daniel M; Kebaish, Khaled M; Lafage, Virginie; Scheer, Justin K; Smith, Justin S; Hamilton, D Kojo; Bess, Shay; Shaffrey, Christopher I; Ames, Christopher P; International Spine Study GroupTo determine the value of a novel adult cervical deformity frailty index (CD-FI) in preoperative risk stratification.We reviewed a prospective, multicenter database of adults with cervical spine deformity. We selected 40 variables to construct the CD-FI using a validated method. Patients were categorized as not frail (NF) (<0.2), frail (0.2-0.4), or severely frail (SF) (>0.4) according to CD-FI score. We performed multivariate logistic regression to determine the relationships between CD-FI score and incidence of complications, length of hospital stay, and discharge disposition.Of 61 patients enrolled from 2009 to 2015 with at least 1 year of follow-up, the mean CD-FI score was 0.26 (range 0.25-0.59). Seventeen patients were categorized as NF, 34 as frail, and 10 as SF. The incidence of major complications increased with greater frailty, with a gamma correlation coefficient of 0.25 (asymptotic standard error, 0.22). The odds of having a major complication were greater for frail patients (odds ratio 4.4; 95% confidence interval 0.6-32) and SF patients (odds ratio 43; 95% confidence interval 2.7-684) compared with NF patients. Greater frailty was associated with a greater incidence of medical complications and had a gamma correlation coefficient of 0.30 (asymptotic standard error, 0.26). Surgical complications, discharge disposition, and length of hospital stay did not correlate significantly with frailty.Greater frailty was associated with greater risk of major complications for patients undergoing cervical spine deformity surgery. The CD-FI may be used to improve the accuracy of preoperative risk stratification and allow for adequate patient counseling.