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1.

Objective

The gene therapy voretigene neparvovec (VN) is the first Food and Drug Administration–approved treatment for vision loss owing to the ultra-rare RPE65-mediated inherited retinal disorders. We modeled the cost-utility of VN compared with standard of care (SoC).

Study Design

A 2-state Markov model, alive and dead, with a lifetime horizon.

Methods

Visual acuity (VA) and visual field (VF) were tracked to model quality-adjusted life-years (QALYs). VN led to an improvement in VA and VF that we assumed was maintained for 10 years followed by a 10-year waning period. The cost of VN was $850 000, and other direct medical costs for depression and trauma were included for a US healthcare system perspective. A modified societal perspective also included direct nonmedical costs and indirect costs.

Results

VN provided an additional 1.3 QALYs over the remaining lifetime of an individual. The average total lifetime direct medical cost for individuals treated with VN was $1 039 000 compared with $213 400 for SoC, leading to an incremental cost-effectiveness ratio (ICER) of $643 800/QALY from the US healthcare system perspective. Direct nonmedical costs totalled $1 070 900 for VN and $1 203 300 for SoC, and indirect costs totalled $405 400 for VN and $482 900 for SoC, leading to an ICER of $480 100/QALY from the modified societal perspective.

Conclusions

At the current price, VN was unlikely to reach traditional cost-effectiveness standards compared with SoC. VN has important implications for both development and pricing of future gene therapies; therefore clinical and economic analyses must be carefully considered.  相似文献   
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Abstract

Chronic psychological distress appears to be increasing markedly among the working population. A recent randomized controlled trial (RCT) supported the effectiveness of a three-week outpatient burnout prevention program—comprised of stress management interventions, relaxation, physical exercise, and moor baths followed by massage—in reducing perceived stress and emotional exhaustion. However, the effectiveness of treatments in the real world that were shown to be efficacious in RCTs is related to the appropriate selection of individuals who are most likely to yield sustainable gains. Therefore, factors predicting the intensity of response and nonresponse of individuals to treatment are of interest. This secondary data analysis aims to explore predictors of response to the outpatient burnout prevention program in a sample of eighty employed persons at high risk of burnout. Hierarchical linear regression was performed to identify predictors of successful response—defined by lower perceived stress at last follow up. Nutritional behavior, symptoms of eating disorder syndrome, and well-being were significant predictors of perceived stress at last follow up, when adjusted for age, sex, education level, baseline stress values, and timing of intervention. Persons with low levels of well-being, poor nutritional behavior, and higher symptoms of eating disorders should be given special care and attention to ensure that they respond well to the outpatient burnout prevention program.  相似文献   
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Journal of Clinical Immunology - A Correction to this paper has been published: https://doi.org/10.1007/s10875-021-01030-6  相似文献   
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BACKGROUND: This study investigated an operator's and pediatric patients' responses to chemo-mechanical caries removal (CMCR) versus the traditional method (TM) of caries removal using a handpiece and a round bur when treating dentinal-depth occlusal lesions with minimal enamel access in primary molars. METHODS: Data were collected from 50 children at baseline and before, during and after caries removal using CMCR or TM. The subjects in the CMCR group were on average younger than the subjects in the TM group and had more deep lesions. RESULTS: The operator rated CMCR as needing more clinical and technical effort and more total effort than TM. He was less satisfied with CMCR than with TM. Subjects in the CMCR group perceived the time needed for treatment as significantly longer than did the subjects in the TM group. Fear of the dentist decreased in subjects in the TM group from before to after the operative appointment, while it increased in subjects in the CMCR group. CONCLUSIONS: The authors found no direct advantage in using CMCR over using TM. CLINICAL IMPLICATIONS: CMCR cannot be recommended as an alternative to TM when treating dentinal depth occlusal lesions with minimal access in primary molars.  相似文献   
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Objectives: The objectives of this study is to explore the relationship between pediatric patients' orthodontic treatment need, the patients' assessments of their smile‐related quality of life (QoL), their parents' proxy assessment of their child's QoL and own assessments of their child's smile, and the patients' objectively assessed smiling patterns. Methods: Survey data were collected from 102 patients (53 boys/49 girls; age range: 9–13 years) and their parents. Orthodontic treatment need was assessed with the Index of Complexity, Outcome, and Need (ICON). Smiling patterns were determined by videotaping patients' smiles while they watched a cartoon. Thirty predetermined sections of these tapes were then assessed by two independent raters to measure the patients' smiling patterns. Results: The aesthetic component and total ICON scores correlated with the patients' smile‐related QoL (r = 0.25; P = 0.014/r = 0.23; P = 0.024), parental proxy assessments of the child's smile‐related QoL (r = 0.29; P = 0.004/r = 0.26; P = 0.009), the parents' own assessments of their child's smile (r = 0.32; P = 0.002/r = 0.29; P = 0.005), and the number of negative adjectives chosen by the parents to describe their child's smile (r = 0.32; P = 0.002/r = 0.30; P = 0.004). Although the smiling patterns were correlated with the patients' smile‐related QoL responses (height of smile: r = 0.29; P = 0.005/number of teeth shown: r = 0.30; P = 0.004), the ICON scores were not correlated with the patients' smiling patterns. Conclusions: Objectively assessed orthodontic treatment need correlates with the patients' and parents' assessments of the child's smile‐related QoL scores. However, while objective smiling patterns are related with the patients' smile‐related QoL, they are not correlated with the patients' orthodontic treatment need.  相似文献   
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BACKGROUND: This study explored which factors affected patients' decisions to pursue either surgical or non-surgical periodontal treatment. METHODS: Data were collected from 74 patients at a regularly scheduled periodontal appointment, at which each patient was told that periodontal treatment was needed, and 2 weeks following the actual treatment. The surveys assessed the patients' decisions and potential determinants of these decisions. The dental anxiety scale-revised, the state-trait anxiety inventory, and the Iowa dental control index were used to measure psychosocial factors. RESULTS: Patients who decided to have surgery did not differ from patients who decided against surgery in sociodemographic variables such as gender, age, education, and socioeconomic status, nor in their desire for control over the treatment decision. However, they had less dental fear and less general anxiety than the non-surgery patients. Although the two patient groups did not differ in their responses concerning how well the dentists had informed them about the procedure, they differed in the degree of trust and rapport with their dentists. CONCLUSIONS: The less dentally fearful and anxious patients were in general and the more they trusted their provider and felt they had good rapport, the more likely they were to accept surgical periodontal treatment. These results stress the importance of good patient-provider communication.  相似文献   
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