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Economic burden of spinal muscular atrophy in the United States: a contemporary assessment.

美国脊髓性肌萎缩的经济负担: 当代评估。

  • 影响因子:1.81
  • DOI:10.1080/13696998.2019.1646263
  • 作者列表:"Droege M","Sproule D","Arjunji R","Gauthier-Loiselle M","Cloutier M","Dabbous O
  • 发表时间:2020-01-01
Abstract

:Aims: To estimate healthcare resource utilization (HRU) and costs among patients with spinal muscular atrophy (SMA) type 1 (SMA1) in real-world practice, overall and among patients treated with nusinersen. As a secondary objective, HRU and costs were estimated among patients with other SMA types (i.e. 2, 3, or 4 combined), overall and among patients treated with nusinersen.Materials and methods: Patients with SMA were identified from the Symphony Health's Integrated Dataverse (IDV) open claims database (September 1, 2016-August 31, 2018) and were classified into four cohorts based on SMA type and nusinersen treatment (i.e. SMA1, SMA1 nusinersen, other SMA, and other SMA nusinersen cohorts). The index date was the date of the first SMA diagnosis after December 23, 2016 or, for nusinersen cohorts, the date of nusinersen initiation. The study period spanned from the index date to the earlier among the end of clinical activity or data availability.Results: Patients in the SMA1 (n = 349) and SMA1 nusinersen (n = 45) cohorts experienced an average of 59.4 and 56.6 days with medical visits per-patient-per-year (PPPY), respectively, including 14.1 and 4.6 inpatient days. Excluding nusinersen-related costs, total mean healthcare costs were $137,627 and $92,618 PPPY in the SMA1 and SMA1 nusinersen cohorts, respectively. Mean nusinersen-related costs were $191,909 per-patient-per-month (PPPM) for the first 3 months post-initiation (i.e. loading phase) and $36,882 PPPM thereafter (i.e. maintenance phase). HRU and costs were also substantial among patients in the other SMA (n = 5,728) and other SMA nusinersen (n = 404) cohorts, with an average of 44.5 and 63.7 days with medical visits PPPY and total mean healthcare costs (excluding nusinersen-related costs) of $49,175 and $76,371 PPPY, respectively.Limitations: The database may contain inaccuracies or omissions in diagnoses, procedures, or costs, and does not capture medical services outside of the IDV network.Conclusions: HRU and healthcare costs were substantial in patients with SMA, including in nusinersen-treated patients.

摘要

目的: 在真实世界实践中估计脊髓性肌萎缩症 (SMA) 1 型 (SMA 1) 患者的医疗资源利用 (HRU) 和成本,总体和接受nusinersen治疗的患者中。作为次要目标,在其他SMA类型的患者中估计HRU和成本 (2 、 3 或 4 联合),总体和接受nusinersen治疗的患者中。材料和方法: 从Symphony Health的Integrated Dataverse (IDV) 开放索赔数据库 (2 0 1 6 年 9 月 1 日-2 0 1 8 年 8 月 3 日) 中确定SMA患者并根据SMA类型和nusinersen治疗分为 4 个队列 (i。e.SMA1 、SMA1 nusinersen、其他SMA和其他SMA nusinersen队列)。索引日期是 2016 年 12 月 23 日后首次SMA诊断的日期,或者对于nusinersen队列,是nusinersen启动的日期。研究期从索引日期跨越到临床活动结束或数据可用性之间的较早。结果: SMA1 (n = 349) 和SMA1 nusinersen (n = 45) 患者队列分别经历了平均 59.4 天和 56.6 天,每个患者每年就诊 (PPPY),包括 14.1 天和 4.6 住院日。排除nusinersen相关费用,SMA1 和SMA1 nusinersen队列的总平均医疗费用分别为 137,627 美元和 92,618 美元PPPY。开始后的前 3 个月,平均nusinersen相关费用为每位患者每月 191,909 美元 (PPPM) (即加载阶段) 和此后的 $36,882 PPPM (即维护阶段)。HRU和费用在其他SMA (n = 5,728) 和其他SMA nusinersen (n = 404) 队列中的患者中也很大,平均 44.5 天和 63.7 天,就诊PPPY和总平均医疗费用 (不包括nusinersen相关费用) 分别为 49,175 美元和 76,371 美元PPPY。限制:数据库可能包含诊断、程序或成本方面的不准确或遗漏,并且不捕获IDV网络之外的医疗服务。结论: SMA患者的HRU和医疗费用相当高,包括nusinersen治疗的患者。

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影响因子:4.22
发表时间:2020-01-01
DOI:10.1016/j.ajog.2019.07.015
作者列表:["Tarasoff LA","Ravindran S","Malik H","Salaeva D","Brown HK"]

METHODS:BACKGROUND:Women with disabilities are increasingly becoming pregnant, and growing evidence suggests maternal disability may be associated with increased risk for perinatal complications. OBJECTIVE:A systematic review and meta-analysis were undertaken to examine the association between maternal disabilities and risk for perinatal complications. STUDY DESIGN:Medline, CINAHL, EMBASE, and PsycINFO were searched from inception to July 2018 for full-text publications in English on pregnancy, delivery, and postpartum complications in women with any disability and those with physical, sensory, and intellectual and developmental disabilities specifically. Searches were limited to quantitative studies with a comparison group of women without disabilities. Reviewers used standardized instruments to extract data from and assess the quality of included studies. Pooled odds ratios and 95% confidence intervals were generated using DerSimonian and Laird random effects models for outcomes with data available from ≥3 studies. RESULTS:The review included 23 studies, representing 8,514,356 women in 19 cohorts. Women with sensory (pooled unadjusted odds ratio, 2.85, 95% confidence interval, 0.79-10.31) and intellectual and developmental disabilities (pooled unadjusted odds ratio, 1.10, 95% confidence interval, 0.76-1.58) had elevated but not statistically significant risk for gestational diabetes. Women with any disability (pooled unadjusted odds ratio, 1.45, 95% confidence interval, 1.16-1.82) and intellectual and developmental disabilities (pooled unadjusted odds ratio, 1.77, 95% confidence interval, 1.21-2.60) had increased risk for hypertensive disorders of pregnancy; risk was elevated but not statistically significant for women with sensory disabilities (pooled unadjusted odds ratio, 2.84, 95% confidence interval, 0.85-9.43). Women with any (pooled unadjusted odds ratio, 1.31, 95% confidence interval, 1.02-1.68), physical (pooled unadjusted odds ratio, 1.60, 95% confidence interval, 1.21-2.13), and intellectual and developmental disabilities (pooled unadjusted odds ratio, 1.29, 95% confidence interval, 1.02-1.63) had increased risk for cesarean delivery; risk among women with sensory disabilities was elevated but not statistically significant (pooled unadjusted odds ratio, 1.28, 95% confidence interval, 0.84-1.93). There was heterogeneity in all analyses, and 13 studies had weak-quality ratings, with lack of control for confounding being the most common limitation. CONCLUSION:Evidence that maternal disability is associated with increased risk for perinatal complications demonstrates that more high-quality research is needed to examine the reasons for this risk and to determine what interventions could be implemented to support women with disabilities during the perinatal period.

影响因子:1.81
发表时间:2020-01-01
DOI:10.1080/13696998.2019.1646263
作者列表:["Droege M","Sproule D","Arjunji R","Gauthier-Loiselle M","Cloutier M","Dabbous O"]

METHODS::Aims: To estimate healthcare resource utilization (HRU) and costs among patients with spinal muscular atrophy (SMA) type 1 (SMA1) in real-world practice, overall and among patients treated with nusinersen. As a secondary objective, HRU and costs were estimated among patients with other SMA types (i.e. 2, 3, or 4 combined), overall and among patients treated with nusinersen.Materials and methods: Patients with SMA were identified from the Symphony Health's Integrated Dataverse (IDV) open claims database (September 1, 2016-August 31, 2018) and were classified into four cohorts based on SMA type and nusinersen treatment (i.e. SMA1, SMA1 nusinersen, other SMA, and other SMA nusinersen cohorts). The index date was the date of the first SMA diagnosis after December 23, 2016 or, for nusinersen cohorts, the date of nusinersen initiation. The study period spanned from the index date to the earlier among the end of clinical activity or data availability.Results: Patients in the SMA1 (n = 349) and SMA1 nusinersen (n = 45) cohorts experienced an average of 59.4 and 56.6 days with medical visits per-patient-per-year (PPPY), respectively, including 14.1 and 4.6 inpatient days. Excluding nusinersen-related costs, total mean healthcare costs were $137,627 and $92,618 PPPY in the SMA1 and SMA1 nusinersen cohorts, respectively. Mean nusinersen-related costs were $191,909 per-patient-per-month (PPPM) for the first 3 months post-initiation (i.e. loading phase) and $36,882 PPPM thereafter (i.e. maintenance phase). HRU and costs were also substantial among patients in the other SMA (n = 5,728) and other SMA nusinersen (n = 404) cohorts, with an average of 44.5 and 63.7 days with medical visits PPPY and total mean healthcare costs (excluding nusinersen-related costs) of $49,175 and $76,371 PPPY, respectively.Limitations: The database may contain inaccuracies or omissions in diagnoses, procedures, or costs, and does not capture medical services outside of the IDV network.Conclusions: HRU and healthcare costs were substantial in patients with SMA, including in nusinersen-treated patients.

影响因子:4.08
发表时间:2020-01-01
来源期刊:European radiology
DOI:10.1007/s00330-019-06352-z
作者列表:["Bernabéu-Sanz Á","Mollá-Torró JV","López-Celada S","Moreno López P","Fernández-Jover E"]

METHODS:OBJECTIVES:To investigate the effect of cervical spondylosis (CS) in the brain with a combination of advanced neuroimaging techniques. METHODS:Twenty-seven patients with CS and 24 age- and gender-matched healthy controls were studied. Disease severity was quantified using the Modified Japanese Orthopaedic Association Scoring System (mJOHA). Magnetic resonance (MR) imaging of the brain and spinal cord, functional MR imaging (fMRI) with a bilateral rest/finger-tapping paradigm, brain diffusion tensor imaging (DTI), voxel-based morphometry (VBM), and MR spectroscopy of the sensorimotor cortex were performed. RESULTS:A total of 92.3% of patients had more than one herniated disc. In the MRI, 33.33% presented signs of myelopathy. The mJOHA score was 13.03 ± 2.83. Compared with controls, DTI results showed significant lower FA values in Corpus callosum, both corticospinal tracts and middle cerebellar peduncles (p < 0.05 corrected). Only in CS patients fMRI results showed activation in both globus pallidi, caudate nucleus, and left thalamus (p < 0.001). Subject-specific activation of the BOLD signal showed in CS patients lower activation in the sensorimotor cortex and increased activation in both cerebellum hemispheres (p < 0.05 corrected). VBM showed bilateral clusters of gray matter loss in the sensorimotor cortex and pulvinar nucleus (p < 0.05 corrected) of CS patients. NAA/Cr was reduced in the sensorimotor cortex of CS patients (p < 0.05). Linear discriminant and support vector machine analyses were able to classify > 97% of CS patients with parameters obtained from the fMRI, DTI, and MRS results. CONCLUSION:CS may lead to distal brain damage affecting the white and gray matter of the sensorimotor cortex causing brain atrophy and functional adaptive changes. KEY POINTS:• This study suggests that patients with cervical spondylosis may present anatomical and functional adaptive changes in the brain. • Cervical spondylosis may lead to white matter damage, gray matter volume loss, and functional adaptive changes in the sensorimotor cortex. • The results reported in this work may be of value to better understand the effect of prolonged cervical spine compression in the brain.

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脊髓疾病方向

包括脊髓肿瘤、脊髓非肿瘤样病变和脊髓发育异常。脊髓病变可引起脊髓压迫症。脊髓压迫症是由各种性质的病变引起脊髓、脊神经根及其血管受压的一组病症,可导致患者瘫痪等。

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