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The Lived Experience of Pediatric Gene Therapy: A Scoping Review

Kimberly, Laura; Hunt, Cara; Beaverson, Katherine; James, Emma; Bateman-House, Alison; McGowan, Richard; DeSante-Bertkau, Jennifer
Little is known about patients' and families' lived experiences of participating in pediatric gene therapy (GT) clinical trials. Currently, pediatric GT research targets a broad range of indications--including rare and ultra-rare diseases--which vary in severity and in the availability of alternative therapies. Pediatric GT differs meaningfully from adult GT because the decision to participate involves a dyad of both the child and parent or caregiver/s. It is critical to understand patients' and caregivers' perceptions and experiences of social, emotional, physical, and logistical burdens or benefits of participating in such trials, and how they weigh and prioritize these factors when deciding whether to participate. We conducted a scoping review of the current literature in this subject area with objectives to (1) provide an overview of existing literature, (2) identify gaps and areas for further research, and (3) better understand the lived impact of pediatric GT research on patients and their parents/caregivers. Four themes emerged, including (1) weighing risks and benefits (2) timing of GT trial participation, (3) value of clear communication, and (4) potential impact on quality of life. Notably, our sample surfaced articles about how patients/parents/caregivers were thinking about GT-their understanding of its safety, efficacy, and risks-rather than accounts of their experiences, which was our initial intention. Nevertheless, our findings offer useful insights to improve the informed consent process and promote a more patient- and family-centered approach. Moreover, our findings can contribute to patient advocacy organizations' efforts to develop educational materials tailored to patients' and families' expressed informational needs and perspectives, and can inform more patient- and family-centered policies from GT clinical trial sponsors.
PMID: 37964764
ISSN: 1557-7422
CID: 5607822

Environmental influences on ophthalmic conditions: A scoping review

Heilen, Noah; Hu, Galen; Lamrani, Ryan; Prasad, Jaideep; Ogunsola, Titilola; Iskander, Mina; Elgin, Cansu Yuksel; McGowan, Richard; Vieira, Dorice; Al-Aswad, Lama A
BACKGROUND:Environmental factors have been implicated in various eye pathologies. The purpose of this review is to synthesise the published research on environmental effects on eye disease. METHODS:Four databases were searched for terms relating to environmental exposures and ophthalmic disease. Titles and abstracts were screened followed by full-text review. Data was extracted from 118 included studies. Quality assessment was conducted for each study. RESULTS:Air pollutants, including nitrogen dioxide, nitrites, sulphur dioxide, particulate matter, carbon monoxide, ozone and hydrocarbons are associated with ocular conditions ranging from corneal damage to various retinopathies, including central retinal artery occlusion. Certain chemicals and metals, such as cadmium, are associated with increased risk of age-related macular degeneration. Climate factors, such as sun exposure, have been associated with the development of cataracts. Living in rural areas was associated with various age-related eye diseases whereas people living in urban settings had higher risk for dry eye disease and uveitis. CONCLUSION/CONCLUSIONS:Environmental exposures in every domain are associated with various ophthalmic conditions. These findings underscore the importance of continued research on the interplay between the environment and eye health.
PMID: 37309709
ISSN: 1442-9071
CID: 5505112

Health Literacy and Ophthalmology: A scoping review

Iskander, Mina; Hu, Galen; Coulon, Sara; Seixas, Azizi A; McGowan, Richard; Al-Aswad, Lama A
As of 2020, it is estimated that 43.3 million people are blind, and an additional 553 million have mild to severe vision impairment.50 At least 1 billion worldwide have a vision impairment that could have been prevented or has yet to be addressed.54 Poor health literacy may be a significant contributor to the prevalence of eye disease. With implications on disease burden, progression, and health outcomes, a greater understanding of the role health literacy plays in ophthalmology is needed. This is the first scoping review to assess the impact of health literacy on eye health outcomes and blindness, including ocular screening rates/follow-up rates, treatment adherence, and self-care practices. PubMed, Embase, and CINAHL databases were searched systematically through November 12, 2021, and we evaluated the association between health literacy and ophthalmic outcomes in 4 domains: clinical outcomes, treatment adherence rates, screening/follow-up rates, and self-care practices. There is evidence to suggest that health literacy is associated with ophthalmic outcomes in all these domains. To better understand how health literacy impacts eye health, further longitudinal studies examining the effect of health literacy (using standardized health literacy measures) on ophthalmic outcomes are needed. We believe a specific ophthalmic health literacy survey could help achieve this goal and help target interventions to ultimately improve outcomes among ophthalmology patients.
PMID: 35995252
ISSN: 1879-3304
CID: 5338152

Outcomes of Primary Root Canal Therapy: An updated Systematic Review of Longitudinal Clinical Studies Published between 2003 and 2020

Burns, L E; Kim, J; Wu, Y; Alzwaideh, R; McGowan, R; Sigurdsson, A
BACKGROUND:A comprehensive effort to evaluate outcomes of primary root canal treatment (RCT) between 1966 and 2002 was published by Ng et al. (2007, 2008). Changes in endodontic materials and treatment methods warrants an updated analysis of outcomes. OBJECTIVES/OBJECTIVE:This study aimed to 1) quantify the success rates of primary RCT published between 2003 and 2020; and 2) investigate the influence of some characteristics known/ suspected to be associated with treatment outcomes. METHODS:An electronic search was performed in the following databases (01-01-2003 to 12-31-2020): Pubmed, Embase, CINHAL, Cochrane and Web of Science. Included study designs were longitudinal clinical studies (randomized control trials, cohort studies, retrospective observational studies). Studies with at least twelve-months of post-operative review and success rates based on clinical and radiographic criteria were analyzed. The terms 'strict' (complete resolution of periapical lesion) or 'loose' (reduction in size of existing periapical lesion) were used to describe the outcome criteria. Weighted, pooled success rates were calculated. Random effects meta-regression models were used to investigate potential sources of statistical heterogeneity. The Grading of Recommendations, Assessment, Development and Evaluation (GRADE) approach was used to evaluate for quality assessment of the included studies. RESULTS:Forty-two studies were included in the review. Meta-analyses showed that the weighted pooled success rates were estimated to be 92.6% (95% CI: 90.5-94.8%) under 'loose criteria' and 82.0% (95% CI: 79.3-84.8%) under 'strict' criteria. The most significant areas of study heterogeneity were year of publication and qualification of operator. The majority (64.29%) of studies were considered to be of low quality of evidence. DISCUSSION/CONCLUSIONS:Biological factors continue to have the most significant impact on RCT outcomes. The technological method of instrumentation had no significant effect. The quality of evidence, was based primarily on study design and only randomized control trials were considered to be "high" quality of evidence. CONCLUSIONS:The reported success rates show improvement over time. Weighted success rates for studies with a minimum of four-year follow-up had better outcomes, compared to those with less than four years, when 'strict criteria' were used.
PMID: 35334111
ISSN: 1365-2591
CID: 5187812

What is the societal economic cost of poor oral health among older adults in the United States? A scoping review

Huang, Shulamite S; Veitz-Keenan, Analia; McGowan, Richard; Niederman, Richard
OBJECTIVE:To assess the state of the literature in the United States quantifying the societal economic cost of poor oral health among older adults. BACKGROUND:Proponents of a Medicare dental benefit have argued that addressing the growing need for dental care among the US older adult population will decrease costs from systemic disease and other economic costs due to oral disease. However, it is unclear what the current economic burden of poor oral health among older adults is in the United States. METHODS:We conducted a scoping review examining the cost of poor oral health among older adults and identified cost components that were included in relevant studies. RESULTS:Other than oral cancer, no studies were found examining the economic costs of poor oral health among older adults (untreated tooth decay, gum disease, tooth loss and chronic disease/s). Only two studies examining the costs of oral cancer were found, but these studies did not assess the full economic cost of oral cancer from patient, insurer and societal perspectives. CONCLUSIONS:Future work is needed to assess the full economic burden of poor oral health among older adults in the United States, and should leverage novel linkages between medical claims data, dental claims data and oral health outcomes data.
PMID: 33719086
ISSN: 1741-2358
CID: 4817392

Virtual Reality and Augmented Reality in Ophthalmology: A Contemporary Prospective

Iskander, Mina; Ogunsola, Titilola; Ramachandran, Rithambara; McGowan, Richard; Al-Aswad, Lama A
PURPOSE/OBJECTIVE:Most published systematic reviews have focused on the use of virtual reality (VR)/augmented reality (AR) technology in ophthalmology as it relates to surgical training. To date, this is the first review that investigates the current state of VR/AR technology applied more broadly to the entire field of ophthalmology. METHODS:PubMed, Embase, and CINAHL databases were searched systematically from January 2014 through December 1, 2020. Studies that discussed VR and/or AR as it relates to the field of ophthalmology and provided information on the technology used were considered. Abstracts, non-peer-reviewed literature, review articles, studies that reported only qualitative data, and studies without English translations were excluded. RESULTS:A total of 77 studies were included in this review. Of these, 28 evaluated the use of VR/AR in ophthalmic surgical training/assessment and guidance, 7 in clinical training, 23 in diagnosis/screening, and 19 in treatment/therapy. 15 studies used AR, 61 used VR, and 1 used both. Most studies focused on the validity and usability of novel technologies. CONCLUSIONS:Ophthalmology is a field of medicine that is well suited for the use of VR/AR. However, further longitudinal studies examining the practical feasibility, efficacy, and safety of such novel technologies, the cost-effectiveness, and medical/legal considerations are still needed. We believe that time will indeed foster further technological advances and lead to widespread use of VR/AR in routine ophthalmic practice.
PMID: 34383716
ISSN: 2162-0989
CID: 4994032

Anti- IL-6 therapies for neuromyelitis optica spectrum disorders: A systematic review of safety and efficacy

Lotan, Itay; McGowan, Richard; Levy, Michael
BACKGROUND:Neuromyelitis Optica Spectrum Disorder (NMOSD) is a chronic autoimmune disease of the central nervous system that causes recurrent attacks of optic neuritis, myelitis, and brainstem symptoms, resulting in severe neurological disability. Preventive treatment with immunosuppressive agents reduces relapse rate and improves long-term prognosis. In recent years, the potential therapeutical effect of new agents have been investigated. Two of these, the antiinterleukin 6 (IL-6) agents tocilizumab and satralizumab, have been studied in active NMOSD. OBJECTIVE:To systematically review the current data regarding the efficacy and safety of anti-IL-6 agents in NMOSD. RESULTS:Fourteen case reports and 5 case series of intravenous tocilizumab have shown beneficial clinical and paraclinical effects compared to commonly used therapies, and another case series of subcutaneous tocilizumab has shown it is as effective as the IV formulation. A phase 2 comparative trial has shown tocilizumab IV to be more effective than azathioprine for relapse prevention. A phase 3 trial ofsubcutaneoussatralizumab versus placebo, hasshown a lower risk of relapse in the sartralizumabtreated group, both as add-on therapy to stable immunosuppressant and as monotherapy. Tocilizumab also reduced pain severity in two trials and fatigue scores in one trial, but satralizumab did not significantly improve pain and fatigue. Adverse events with both agents were relatively mild and comparable to placebo and azathioprine. CONCLUSIONS:The anti-Il-6 agents tocilizumab and satralizumab show promising results in active NMOSD. Further randomized, larger-scale trials are needed to better define the role of these agents in the growing arsenal of NMOSD treatments.
PMID: 32348222
ISSN: 1875-6190
CID: 4418242

Patient Extratherapeutic Interpersonal Problems and Response to Psychotherapy for Depression

Chen, Cory K; Nehrig, Nicole; Chou, Leetyng Jennifer; McGowan, Richard; Guyton, Angel F; Mustafiz, Fayel; Bailey, Robert W
OBJECTIVES/UNASSIGNED:This paper aimed to synthesize empirical findings of patient extratherapeutic interpersonal variables associated with individual psychotherapy treatment outcomes in adult outpatients with depression. METHODS/UNASSIGNED:A systematic search strategy was used to identify relevant studies. Thematic analysis was used to identify recurring themes in the findings. RESULTS/UNASSIGNED:Forty studies met search criteria. Three themes of patient extratherapeutic interpersonal variables were identified: capacity to engage with others, capacity to navigate relationships, and capacity to achieve intimacy, progressing from basic to advanced levels of interpersonal interaction. Interpersonal variables such as interpersonal distress and style, attachment orientation, and quality of object relations were particularly useful in predicting treatment outcomes, whereas access to social support and marital status provided mixed results, likely because they do not account for relationship quality. CONCLUSIONS/UNASSIGNED:Recognizing variables associated with treatment response can help clinicians identify patients at risk for nonresponse and guide efforts for adapting existing therapies and developing new ones.
PMID: 31813229
ISSN: 0002-9564
CID: 4238632

Quality Appraisal of Child Oral Health-Related Quality of Life Measures: A Scoping Review

Yang, C; Crystal, Y O; Ruff, R R; Veitz-Keenan, A; McGowan, R C; Niederman, R
BACKGROUND/UNASSIGNED:Children's oral health-related quality of life (COHQoL) measures are well known and widely used. However, rigorous systematic reviews of these measures and analyses of their quality are in absence. OBJECTIVES/UNASSIGNED:To systematically review and quantitatively assess the quality of COHQoL measures through a scoping review. DATA SOURCES/UNASSIGNED:Systematic literature search of PubMed, CINAHL (Cumulative Index to Nursing and Allied Health Literature), EMBASE (Excerpta Medica database), HaPI (Health and Psychosocial Instruments), and DOSS (Dentistry and Oral Sciences Source). STUDY ELIGIBILITY/UNASSIGNED:The measure's focus was COHQoL; the child age ranged from 5 to 14 years; the publication was either a research article or a systematic review and related to caries; and it was written in English or had an English abstract. Two authors independently selected the studies. Disagreements were reconciled by group discussions with a third author. APPRAISAL/UNASSIGNED:The International Society for Quality of Life Research minimum standards for patient-reported outcome measures were used for quality appraisal. SYNTHESIS/UNASSIGNED:Descriptive analysis. RESULTS/UNASSIGNED:We identified 18 measures. Their quality scores ranged from 9.5 to 15.0 on a scale of 16. The quality appeared to bear no relationship to the citation and use of these measures. However, elements of these measures might be more useful than others, depending on the age-specific use and primary quality concerns. LIMITATIONS/UNASSIGNED:Some of the information on the minimum standards of the 18 measures cannot be found in the existing literature. Measures published without English abstract were not searched. CONCLUSIONS/UNASSIGNED:The quality of these measures is suboptimal. Researchers and practitioners in this field should exercise caution when choosing and using these measures. Efforts at improving the quality of the COHQoL measures, such as refining existing ones or developing new measures, are warranted. KNOWLEDGE TRANSFER STATEMENT/UNASSIGNED:Researchers, clinician scientists, and clinicians can use the results of this study when deciding which oral health-related quality of life measure they wish to use in children.
PMID: 31238010
ISSN: 2380-0852
CID: 3962742

A systematic review on incidental findings in cone-beam computed tomography (CBCT) Scans

Dief, Sandy; Veitz-Keenan, Analia; Amintavakoli, Niloufar; McGowan, Richard
OBJECTIVES/UNASSIGNED:Cone-beam computed tomography's use (CBCT) in dentistry is increasing. Incidental findings (IFs: discoveries unrelated to the original purpose of the scan), are frequently found as a result of a large field of view. The aim of the systematic review is to analyze present literature on IFs using CBCT. METHODS AND MATERIALS/UNASSIGNED:The authors searched online databases of studies and assessed the prevalence of IFs among patients undergoing head and neck CBCT scans. STROBE criteria was used to evaluate the quality of the studies. RESULTS/UNASSIGNED:The original search retrieved 509 abstracts of which only 10 articles met the inclusion criteria. The sample size varied between 90 - 1000 participants. The frequency of IFs of the selected articles were 24.6 - 94.3 %. The most common non-threatening IFs were found in the airway, such as mucous retention cyst (55.1%) and sinusitis (41.7%). Other non-threatening IFs were soft tissue calcifications such as calcified stylohyloid ligament (26.7%), calcified pineal gland (19.2%), and tonsillolith (14.3%). Threatening IFs were rare findings (1.4%). Three articles reported incidental carotid artery calcifications with a prevalence of 5.7 - 11.6 %. Pathological findings were not common between the articles, but still relevant (2.6%). The studies had a risk of bias varying from moderate to low. CONCLUSIONS/UNASSIGNED:There is a high frequency of IFs, yet not all of them require immediate medical attention. The low prevalence of threatening IFs emphasizes that CBCT should not be considered a substitution for conventional radiographs, but when used, the scans should be evaluated by a maxillofacial radiologist.
PMID: 31216179
ISSN: 0250-832x
CID: 3938762