Study Outcome Measures Research Articles

Convergence Insufficiency Neuro-mechanism in Adult Population Study Randomized Clinical Trial: Clinical Outcome Results.

These data confirm the effectiveness of office-based vergence/accommodative therapy for improving the near point of convergence and positive fusional vergence in young adults with symptomatic convergence insufficiency within a double-masked longitudinal randomized clinical trial. This study aimed to report changes in clinical signs and symptoms of convergence insufficiency from a randomized clinical trial evaluating the effectiveness of office-based vergence/accommodative therapy for young adults with symptomatic convergence insufficiency. In this double-masked, randomized clinical trial, convergence insufficiency patients (n = 50; average age, 21 ± 3 years; range, 18 to 32 years) were randomized to either office-based vergence/accommodative therapy or office-based placebo therapy. Improvements in (1) near point of convergence, (2) positive fusional vergence, and (3) self-reported symptoms (Convergence Insufficiency Symptom Survey [CISS] score) were evaluated after twelve 1-hour sessions of treatment within the office comparing the results from the vergence/accommodative therapy and the placebo therapy groups. The mean near point of convergence improved by 6.0 and 3.1 cm in the vergence/accommodative and placebo therapy groups, respectively (mean difference of -2.9 cm; 95% confidence interval [CI], -4.6 to -1.0 cm; P < .01). The mean positive fusional vergence increased by 17.3 and 7.4Δ in the vergence/accommodative and placebo therapy groups, respectively (mean difference of 9.9Δ; 95% CI, 4.9 to 16.0Δ; P < .001). The mean CISS score improved by 12.4 and 10.1 points in the vergence/accommodative and placebo therapy groups, respectively (mean difference of 2.3 points; 95% CI, -8.3 to +4.6 points; P = .56). Our results demonstrate that office-based vergence/accommodative therapy is effective for improving the near point of convergence and positive fusional vergence in young adults with symptomatic convergence insufficiency. However, given that both treatment groups had a similar reduction in self-reported symptoms, we recommend that the CISS be revised if it is to be used as an outcome measure in future studies of convergence insufficiency.

Using a Composite Maternal-Infant Outcome Measure in Tuberculosis-Prevention Studies Among Pregnant Women.

Tuberculosis (TB-)-preventive therapy (TPT) among pregnant women reduces risk of TB in mothers and infants, but timing of initiation should consider potential adverse effects. We propose an analytical approach to evaluate the risk-benefit of interventions. A novel outcome measure that prioritizes maternal and infant events was developed with a 2-stage Delphi survey, where a panel of stakeholders assigned scores from 0 (best) to 100 (worst) based on perceived desirability. Using data from TB APPRISE, a trial among pregnant women living with human immunodeficiency virus (WLWH) that randomized the timing of initiation of isoniazid, antepartum versus postpartum, was evaluated. The composite outcome scoring/ranking system categorized mother-infant paired outcomes into 8 groups assigned identical median scores by stakeholders. Maternal/infant TB and nonsevere adverse pregnancy outcomes were assigned similar scores. Mean (SD) composite outcome scores were 43.7 (33.0) and 41.2 (33.7) in the antepartum and postpartum TPT initiation arms, respectively. However, a modifying effect of baseline antiretroviral regimen was detected (P = .049). When women received nevirapine, composite scores were higher (worse outcomes) in the antepartum versus postpartum arms (adjusted difference, 14.3; 95% confidence interval [CI], 2.4-26.2; P = .02), whereas when women received efavirenz there was no difference by timing of TPT (adjusted difference, .62; 95% CI, -3.2-6.2; P = .53). For TPT, when used by otherwise healthy persons, preventing adverse events is paramount from the perspective of stakeholders. Among pregnant WLWH in high-TB-burden regions, it is important to consider the antepartum antiretroviral regimen taken when deciding when to initiate TPT. Clinical Trials Registration. NCT01494038 (IMPAACT P1078).

Person Ability Scores as an Alternative to Norm-Referenced Scores as Outcome Measures in Studies of Neurodevelopmental Disorders.

Although norm-referenced scores are essential to the identification of disability, they possess several features which affect their sensitivity to change. Norm-referenced scores often decrease over time among people with neurodevelopmental disorders who exhibit slower-than-average increases in ability. Further, the reliability of norm-referenced scores is lower at the tails of the distribution, resulting in floor effects and increased measurement error for people with neurodevelopmental disorders. In contrast, the person ability scores generated during the process of constructing a standardized test with item response theory are designed to assess change. We illustrate these limitations of norm-referenced scores, and relative advantages of ability scores, using data from studies of autism spectrum disorder and creatine transporter deficiency.

Clinical outcome of patients with ST-elevation myocardial infarction and angiographic evidence of coronary artery ectasia

Abstract Background Coronary artery ectasia (CAE) is a relatively frequent finding in patient with ST-elevation myocardial infarction (STEMI) who undergo emergent coronary angiography. However, the long-term outcome of STEMI patients with CAE as compared to Non-CAE has been poorly investigated. Purpose To compare the baseline features and outcome of patients with and without CAE in the clinical setting of STEMI. Methods All patients with STEMI who underwent emergent coronary angiography from January 2012 to December 2017 at our Institution were retrospectively enrolled. Baseline demographic, clinical, instrumental, angiographic and percutaneous coronary intervention (PCI) findings were collected for patients with and without CAE. The study outcome measures were recurrent myocardial infarction (MI) and all-cause death. The propensity score weighting (PSW) technique was used to take into account for potential selection bias in treatment assignment between CAE and Non-CAE groups. Results The study included 534 patients with STEMI (mean age 62.9±12.0 years), 154 were CAE and 380 Non-CAE. The two groups were significantly different in terms of sex (90.9% in CAE vs 72,6% in Non-CAE, p&amp;lt;0.001), diabetes (11.7% vs. 25.8%; p=0.009) and smoking status (72.1% vs. 62.4%; p=0.042). The right coronary artery was more commonly treated in CAE patients (41.6% vs. 30.8%, p=0.023) and, as expected, the stent diameter (p&amp;lt;0.001) and the TIMI frame count (p&amp;lt;0.001) were significantly higher in CAE group. The myocardial blush grade was higher in Non-CAE (p&amp;lt;0.001). The Kaplan-Meyer analysis showed a comparable rate of all-cause death among the two groups (3.4/100 person/years in CAE vs. 3.5 per 100 person/years in Non-CAE, Log-Rank = 0.86). The survival free from recurrent MI was lower, although not statistically significant, in CAE vs. Non-CAE patients (3.1/100 person/years vs. 4.8/100 person/years; Log-Rank = 0.068). After PSW, an optimal balance was obtained as demonstrated by a standardized mean difference &amp;lt;0.1 for all the variables included in the model. The adjusted Cox regression analysis showed a significantly higher risk of recurrent MI in CAE vs. Non-CAE groups (HR = 1.93; p=0.009). No difference in the risk of all-cause death was observed (HR = 0.83, p=0.501). Conclusions Patient with STEMI and angiographic evidence of CAE have a different clinical profile compared to Non-CAE. In this analysis focused on STEMI patients, CAE was associated with a higher risk of recurrent MI at long-term follow-up. Funding Acknowledgement Type of funding source: None

An intervention to improve the quality of life in children of parents with serious mental illness: the Young SMILES feasibility RCT.

An intervention to improve the quality of life in children of parents with serious mental illness: the Young SMILES feasibility RCT.

FN3K expression in COPD: a potential comorbidity factor for cardiovascular disease

IntroductionCigarette smoking and oxidative stress are common risk factors for the multi-morbidities associated with chronic obstructive pulmonary disease (COPD). Elevated levels of advanced glycation endproducts (AGE) increase the risk of...

Gabapentin to reduce pain in women aged between 18 and 50 years with chronic pelvic pain: the GaPP2 RCT

BackgroundChronic pelvic pain affects 2–24% of women worldwide, and evidence for medical treatments is limited. Gabapentin is effective in treating some chronic pain conditions, but its effect on central pain processing is unknown.ObjectivesTo test the hypothesis that gabapentin can reduce pain and improve physical and emotional functioning in women with chronic pelvic pain. We investigated the mechanism of action of gabapentin in a subset of women.DesignA randomised, double-blind, placebo-controlled, multicentre trial with a brain imaging substudy.SettingThis trial took place in 39 UK hospitals.ParticipantsA target of 300 women with a history of chronic pelvic pain in whom a laparoscopy revealed no obvious pelvic pathology.InterventionWomen were randomised to receive 300 mg of gabapentin (which was escalated to a maximum of 2700 mg daily) or a matched placebo over a 4-week dose-escalation period, followed by 12 weeks on optimal dose. A mechanistic substudy was also undertaken, in which a subset of participants had a functional magnetic resonance imaging scan of their brain before and following 16 weeks of treatment.Main outcome measuresThe dual primary measure of the worst and average pelvic pain scores was assessed weekly by a numerical rating scale (0–10) in weeks 13–16 post randomisation. The secondary outcomes were patient-reported questionnaires, assessed physical functioning, fatigue, psychological health, sexual activity, work and productivity, and pain catastrophising. Health-care resource use, analgesic use and adverse events were also collected. The main outcome measure for the mechanistic study was brain activity at rest and in response to noxious stimuli.ResultsIn the main trial, 306 participants were randomised. The mean worst pain score was 7.1 (standard deviation 2.6) in the gabapentin group and 7.4 (standard deviation 2.2) in the placebo group (adjusted mean difference –0.20, 97.5% confidence interval –0.81 to 0.42;p = 0.47). The mean average pain score was 4.3 (standard deviation 2.3) in the gabapentin group and 4.5 (standard deviation 2.2) in the placebo group (adjusted mean difference –0.18, 97.5% confidence interval –0.71 to 0.35;p = 0.45). No significant between-group differences were observed for any secondary outcome. A higher proportion of women experienced a serious adverse event in the gabapentin group than in the placebo group (10/153 vs. 3/153;p = 0.04). Dizziness, drowsiness and visual disturbances were more common in the gabapentin group than in the placebo group. In the mechanistic study, 45 participants had a baseline functional magnetic resonance imaging scan of their brain, with 25 participants returning for a scan at the end of treatment. Gabapentin significantly decreased evoked activity in the anterior cingulate cortex and cuneus. Change in anterior cingulate cortex activity after treatment related to improvement on the pain interference scale, and baseline activation of this region predicted response to treatment.ConclusionsGabapentin did not reduce pain and did not improve other outcomes compared with placebo over 16 weeks. Serious adverse effects were significantly higher in the gabapentin group than in the placebo group. Gabapentin reduces evoked activity in the anterior cingulate cortex, with changes of activity in this region tracking reported pain, and baseline activity predicting response to treatment.LimitationsPrimary outcome data were unavailable in 62 and 60 women for the average and worst numerical rating scale pain scores, respectively. A sensitivity analysis using imputation methods did not change the result.Future workClinical trials to investigate other pharmacological interventions (monotherapy vs. combination therapy), physiotherapy and cognitive–behavioural therapy to treat women with chronic pelvic pain are needed.Trial registrationCurrent Controlled Trials ISRCTN77451762 and EudraCT 2014-005035-13.FundingThis project was funded by the Efficacy and Mechanism Evaluation (EME) programme, a Medical Research Council and National Institute for Health Research (NIHR) partnership. This will be published in full inEfficacy and Mechanism Evaluation; Vol. 7, No. 7. See the NIHR Journals Library website for further project information.

Evaluating the Benefits of Exercise Training in HFrEF or COPD Patients: ISO-LEVEL COMPARISON CAN ADD VALUABLE INFORMATION TO V˙o2peak.

Heart failure with reduced ejection fraction (HFrEF) and chronic obstructive pulmonary disease (COPD) are relatively common conditions with similar symptoms of exercise intolerance and dyspnea. The aim of this study was to compare exercise capacity, ventilatory response, and breathing pattern in patient groups with either advanced HFrEF or COPD before and after exercise training. An observational study was conducted with parallel groups of 25 HFrEF and 25 COPD patients who took part in 6 wk of inpatient rehabilitation with exercise training. All patients underwent cardiopulmonary exercise tests at the start and end of the training, with resting arterial blood gas measurements. The average peak oxygen uptake (V˙o2) was low at the start of the study but increased significantly after training in both groups, or by 2.2 ± 2.1 mL/kg/min in HFrEF patients and 1.2 ± 2.2 mL/kg/min in COPD patients. At ISO-V˙o2 (ie, same level of V˙o2 in pre- and post-exercise tests), carbon dioxide production (V˙co2) decreased after exercise training in both groups. Similarly, at ISO-V˙E (ie, same level of ventilation), breathing frequency (f) decreased and tidal volume (VT) increased, resulting in an improved breathing pattern (lower f/VT ratio) after training. The findings of this study show that exercise training in severely affected patient groups with HFrEF or COPD led to an increase in maximal exercise capacity, a more favorable breathing pattern, and a diminished V˙co2 during exercise. Therefore, comparisons of V˙co2 and breathing pattern at ISO-levels of V˙o2 or V˙E before and after training are valuable and underutilized outcome measures in treatment studies.

Malaria and cardiovascular disease: a systematic review

Abstract Background Myocardial tissue appears to be a favored site for the malaria parasite, and several studies have suggested that malaria may lead to myocardial dysfunction. Therefore, we conducted a systematic review of clinical studies reporting on malaria and cardiac disease. Methods We searched PubMed and Embase through January 2020. We applied strict inclusion criteria (Figure 1A) and assessed studies reporting on verified cardiac disease by paraclinical tests, ECG, cardiac biomarkers and echocardiographic findings. Two reviewers independently screened articles, extracted data and assessed the PRISMA guidelines. Results Twenty-eight articles were identified (published 1954–2020; n=19 case-reports, n=5 cohort studies, n=4 cross-sectional studies; Figure 1B), of which most were conducted in India (n=11) and Germany (n=4). The studies included a total of 2,221 malaria cases (mean age 42 years; 67% men; 41% complicated infection) and the distribution of species were: Plasmodium falciparum (n=15), vivax (n=12) and knowlesi (n=1). The most commonly reported cardiac diagnoses in case-reports were myocarditis (n=9), pericarditis (n=3) and acute myocardial infarction (n=4). Although the cohort and cross-sectional studies reported on different cardiac parameters (Figure 1C), a majority showed that malaria cases more often had elevated levels of cardiac biomarkers, ECG alterations and reduced left ventricular ejection fraction when compared to controls (P&amp;lt;0.05 for all). Conclusion Studies reporting on malaria and cardiovascular disease display considerable heterogeneity in terms of study design, severity and outcome measurements. Despite this, the included studies demonstrate a potential link between malaria and cardiac disease. This should be explored in future and larger hypothesis generating clinical studies. Figure 1 Funding Acknowledgement Type of funding source: None

To Keto or Not to Keto? A Systematic Review of Randomized Controlled Trials Assessing the Effects of Ketogenic Therapy on Alzheimer Disease

Alzheimer disease (AD) is a global health concern with the majority of pharmacotherapy choices consisting of symptomatic treatment. Recently, ketogenic therapies have been tested in randomized controlled trials (RCTs), focusing on delaying disease progression and ameliorating cognitive function. The present systematic review aimed to aggregate the results of trials examining the effects of ketogenic therapy on patients with AD/mild cognitive impairment (MCI). A systematic search was conducted on PubMed, CENTRAL, clinicaltrials.gov, and gray literature for RCTs performed on adults, published in English until 1 April, 2019, assessing the effects of ketogenic therapy on MCI and/or AD compared against placebo, usual diet, or meals lacking ketogenic agents. Two researchers independently extracted data and assessed risk of bias with the Cochrane tool. A total of 10 RCTs were identified, fulfilling the inclusion criteria. Interventions were heterogeneous, acute or long term (45-180 d), including adherence to a ketogenic diet, intake of ready-to-consume drinks, medium-chain triglyceride (MCT) powder for drinks preparation, yoghurt enriched with MCTs, MCT capsules, and ketogenic formulas/meals. The use of ketoneurotherapeutics proved effective in improving general cognition using the Alzheimer's Disease Assessment Scale-Cognitive, in interventions of either duration. In addition, long-term ketogenic therapy improved episodic and secondary memory. Psychological health, executive ability, and attention were not improved. Increases in blood ketone concentrations were unanimous and correlated to the neurocognitive battery based on various tests. Cerebral ketone uptake and utilization were improved, as indicated by the global brain cerebral metabolic rate for ketones and [11C] acetoacetate. Ketone concentrations and cognitive performance differed between APOE ε4(+) and APOE ε4(-) participants, indicating a delayed response among the former and an improved response among the latter. Although research on the subject is still in the early stages and highly heterogeneous in terms of study design, interventions, and outcome measures, ketogenic therapy appears promising in improving both acute and long-term cognition among patients with AD/MCI. This systematic review was registered at www.crd.york.ac.uk/prospero as CRD42019128311.

Baseline assessment of a potassium iodide distribution for nuclear power plant emergencies in the Canadian-United States border region.

This study sought to evaluate resident demographics and resident understanding of the proper use of potassium iodide (KI) pills as a countermeasure in the event of a nuclear power plant emergency. We also described expected behaviors of community members in the event of a nuclear accident. The study design utilized a cross-sectional survey with a validated written questionnaire. Subjects, Participants: The study subjects were Canadian residents living within the primary emergency planning zone of the Fermi 2 nuclear power reactor. There was a 77 percent participation rate (n = 180) in Amherst Point and 61 percent for Bois Blanc Island (commonly referred to as Bob-Lo Island) (n = 57). The primary study outcome measures were the number of knowledge-based questions survey respondents correctly answered about proper KI use (Ksum) and various behavior-based survey questions in the context of the extended parallel process model (EPPM). Overall, we found that residents in general had a very low overall comprehension of proper KI use. We found that most resident demographics (eg, age, gender) did not significantly impact their knowledge of proper KI use but did find that households with children under 13 years of age tended to have higher comprehension scores than households without young children. Most residents reported that if they thought they were exposed to radiation they would go to the hospital or call 911 and few residents knew their evacuation routes, few were aware of the emergency siren, none knew where the reception center was located, and most did not have an emergency kit in their home. The majority of the survey respondents did indicate that they would evacuate if told to do so by their government. Public health outreach is crucial for KI distribution programs because of the overall low pre-existing comprehension in communities. Also, hospitals and 911 must be prepared to deal with higher volume of residents seeking help should an accident occur.

Analysis of Granulomatous Lymphocytic Interstitial Lung Disease Using Two Scoring Systems for Computed Tomography Scans-A Retrospective Cohort Study.

BackgroundGranulomatous lymphocytic interstitial lung disease (GLILD) is present in about 20% of patients with common variable immunodeficiency disorders (CVID). GLILD is characterized by nodules, reticulation, and ground-glass opacities on CT scans. To date, large cohort studies that include sensitive CT outcome measures are lacking, and severity of structural lung disease remains unknown. The aim of this study was to introduce and compare two scoring methods to phenotype CT scans of GLILD patients.MethodsPatients were enrolled in the “Study of Interstitial Lung Disease in Primary Antibody Deficiency” (STILPAD) international cohort. Inclusion criteria were diagnosis of both CVID and GLILD, as defined by the treating immunologist and radiologist. Retrospectively collected CT scans were scored systematically with the Baumann and Hartmann methods.ResultsIn total, 356 CT scans from 138 patients were included. Cross-sectionally, 95% of patients met a radiological definition of GLILD using both methods. Bronchiectasis was present in 82% of patients. Inter-observer reproducibility (intraclass correlation coefficients) of GLILD and airway disease were 0.84 and 0.69 for the Hartmann method and 0.74 and 0.42 for the Baumann method.ConclusionsIn both the Hartmann and Baumann scoring method, the composite score GLILD was reproducible and therefore might be a valuable outcome measure in future studies. Overall, the reproducibility of the Hartmann method appears to be slightly better than that of the Baumann method. With a systematic analysis, we showed that GLILD patients suffer from extensive lung disease, including airway disease. Further validation of these scoring methods should be performed in a prospective cohort study involving routine collection of standardized CT scans.Clinical Trial Registration https://www.drks.de, identifier DRKS00000799.

Characterizing the face in facioscapulohumeral muscular dystrophy

ObjectiveTo evaluate facial weakness in patients with FSHD to better define clinical signs, and pilot a facial weakness severity score.Methods87 FSHD patients and 55 controls were video recorded while performing seven facial tasks. The videos were assessed by three independent examiners to compile an overview of signs of facial weakness. Next, videos were semi-quantitatively assessed using a newly developed 4-point facial weakness score (FWS). This score was evaluated and correlated to other FSHD disease characteristics.ResultsPatients had lower scores on the total FWS than controls (mean score 43 ± 28, range 4–118, vs 14 ± 9, range 0–35, p < 0.001) and on all seven individual facial tasks (all p < 0.001). 54% of patients had FWS scores outside the range of controls. Patients had more asymmetry between the left and right side of the face than controls. About 10% of the patients had very mild facial weakness. These were mostly males (89%) with longer D4Z4 repeat sizes of 7–9 units. More severe facial weakness correlated to more severe overall disease severity and shorter D4Z4 repeat size, but not to disease duration. Interobserver agreement for the FWS between three raters was low with a Fleiss Kappa of 0.437.ConclusionThis study provides an overview of the clinical spectrum of facial weakness and its relation to other disease characteristics. The 4-point scale we introduced to grade the severity of facial weakness enables correlation of facial weakness to disease characteristics, but is not suited as clinical outcome measure for longitudinal studies.

Reliability and Feasibility of the First-Episode Psychosis Services Fidelity Scale-Revised for Remote Assessment.

The authors sought to evaluate the interrater reliability and feasibility of the First-Episode Psychosis Services Fidelity Scale-Revised (FEPS-FS-R) for remote assessment of first-episode psychosis programs according to the coordinated specialty care model. The authors used the FEPS-FS-R to assess the fidelity of 36 first-episode psychosis program sites in the United States with information from three sources: administrative data, health record review, and phone interviews with staff. Four raters independently conducted fidelity assessments of five program sites by listening to each of the staff interviews and independently rating the two other data sources from each site. To calculate interrater reliability, the authors used intraclass correlation coefficients (ICCs) for each of the five sites and across the total scores for each site. Total interrater reliability was in the good to excellent range, with a mean ICC of 0.91 (95% confidence interval = 0.72-0.99, p<0.001). Two first-episode psychosis program sites (6%) achieved excellent fidelity, 25 (69%) good fidelity, and nine (25%) fair fidelity. Of the 32 distinct items on the FEPS-FS-R, 23 (72%) were used with good or excellent fidelity. Most sites achieved high fidelity on most items, but five items received ratings indicating low-fidelity use at most sites. The fidelity assessment proved feasible, and sites required on average 10.5 hours for preparing and conducting the fidelity review. The FEPS-FS-R has high interrater reliability and can differentiate high-, moderate-, and low-fidelity sites. Most sites had good overall fidelity, but the FEPS-FS-R identified some services that were challenging to implement at many sites.

F@ce: a team-based, person-centred intervention for rehabilitation after stroke supported by information and communication technology \u2013 a feasibility study

BackgroundGlobally, there is a growing use of Information and Communication Technology (ICT), including mobile phones, tablets and computers, which are being integrated into people’s daily activities. An ICT-based intervention called F@ce was developed in order to provide a structure for the process in stroke rehabilitation and facilitate change by integrating a global problem-solving strategy using SMS alerts. The aim of the study was to evaluate the feasibility of i) F@ce within in-patient and primary care rehabilitation after stroke, ii) the study design and outcome measures used, and iii) the fidelity, adherence and acceptability of the intervention.MethodsThree teams comprising occupational therapists and physiotherapists who work in neurological rehabilitation participated in a preparatory workshop on F@ce and then enrolled 10 persons with stroke to participate in the intervention. Goals were set using the Canadian Occupational Performance Measure (COPM) and the participants with stroke rated their performance and satisfaction with the activities associated with the three goals every day for 8 weeks. Data were collected at inclusion, at four and 8 weeks, using the COPM, Stroke Impact Scale, Frenchay Activities Index, Life Satisfaction Checklist, Self-Efficacy Scale, Hospital Anxiety and Depression Scale, Fatigue Severity Scale, follow-up survey, daily ratings on the web platform and logbooks.ResultsAll of the participants showed increased scores in the primary outcome (COPM) and a clinically meaningful improvement of ≥2 points was found in four participants regarding performance and in six participants regarding satisfaction. Overall fidelity to the components of F@ce was good. The response rates to the F@ce web platform were 44–100% (mean 78%). All of the participants stated that F@ce had supported their rehabilitation.ConclusionThe results indicate that the most beneficial part of F@ce was the person-centred, goal-setting process and SMS alerts. All participants were satisfied with F@ce and highlighted the benefits of receiving daily alerts about their goals. This encouraged them to be more active. The only downside mentioned was that they felt under an obligation to practice, although this was described as “a positive obligation”.

The GoAudio Quantitative Mobile Audiology Test Enhances Access to Clinical Hearing Assessments.

Purpose Hearing loss is a common impairment of the human senses with an estimated 48 million American adults reporting some trouble hearing; however, access to hearing health care is limited. Detection of hearing loss through a mobile, handheld tool can provide an important access point and potentially expedited access to the continuum of hearing health care. Here, we determined that GoAudio, a portable, automated hearing assessment tool, can be used to identify individuals who require additional hearing evaluation in a clinical workflow. Method This initial study included 24 adults, ages 18-65 years (M = 50, SD = 12), tested with GoAudio versus "gold-standard" clinical audiometry for eight frequencies to evaluate "real-world" applications. Participants utilized noise-canceling headphones combined with a tablet-based application for the GoAudio assessment. Results The primary study outcome measurements were the comparison of hearing thresholds (dB HL) from clinical audiometry and GoAudio. Results suggest that GoAudio is comparable to clinical audiometry for the identification of hearing loss at most frequencies (except 1 kHz for both ears and 2 kHz in the right ear). Upon stratifying data based on age, we identified that GoAudio is capable of identifying suspected age-related hearing loss or hearing thresholds greater than 30 dB HL at higher frequencies in both ears. Conclusion The study results support that GoAudio can be used effectively in clinical practice workflows as a reliable hearing assessment tool for the identification of hearing loss at the majority of frequencies outside a sound-treated booth and can detect characteristics of age-related hearing loss. Supplemental Material https://doi.org/10.23641/asha.13087682.

Trends in the use of patient-reported outcome measures for inguinal hernia repair: a quantitative systematic review.

To quantitatively assess the use of patient-reported outcome measures in studies involving patients undergoing inguinal hernia repair. We performed a systematic literature search in Medline and EMBASE. We included all studies published between 2000 and 2019 that involved > 5 patients receiving inguinal hernia repair and evaluated a postoperative patient-reported outcome measure. Studies were stratified in 5-year intervals. We extracted data on which patient-reported outcome measure was used, its time of administration, study design, and the size and composition of the study population. Data were presented using descriptive statistics. We included 929 studies that covered 81 different patient-reported outcome measures. Of these, the Short-Form 36 was the most commonly used generic instrument (14%), the Carolinas Comfort Scale was the most commonly used hernia-specific instrument (5%), and the Visual Analogue Scale was the most commonly used domain-specific instrument (70%). There was a proportional decrease in the use of generic instruments, from 24% of studies in 2000-2004 to only 14% of studies in 2015-2019. Conversely, there was an increase in the use of hernia-specific instruments, from 0% in 2000-2004 to 18% in 2015-2019. There is heterogeneity in the use of patient-reported outcome measures in the field of inguinal hernia research. The use of hernia-specific instruments is increasing, the use of generic instruments is decreasing, and the use of domain-specific instruments remains consistently high. This study serves as a repository of all available patient-reported outcome measures relevant to patients undergoing inguinal hernia repair.

Patient reported outcome measures in a large cohort of patients with type 1 Gaucher disease

BackgroundIt is now acknowledged that the input of patientsin health outcome assessment is vital to understanding the impact of diseases and interventions for those diseases. This study is the first report of patient-reported outcome measures (PROM) in a large cohort of patients with type 1 Gaucher disease (GD1) enabling us to study predictors of the reported outcomes.MethodThe PROM was sent via a mobile phone surveyto 405 adult patients with GD1. Demographics, clinical data, and treatment status were extracted from clinic charts. Age, sex, severity score index (SSI) at presentation and treatment status were used as variables to assess outcomes.ResultsA total of 192 patients with GD1 (111 females) responded (47.4% response rate), of whom 124 (64.5%) had received GD1-specific therapy. Around 40% of patients reported that GD had restricted their education/job and fun activities and were concerned about being emotional and financial burdens on others. Concerns regarding the risk of bone disease and Parkinson disease were also high (60%). The severity of GD1 (reflected by the need for GD1-specific therapy and a high SSI) was associated with GD1-related restrictions and concerns, fatigue, physical weakness, bone pain, and worry regarding the future.ConclusionsThe use of GD1 specific PROM highlights personal problems that are not captured by traditional outcome parameters and that need to be addressed to improve health-related quality of life. Validated PROM should be included among the outcome measures in clinical practice and future prospective studies for patients with chronic and rare diseases.

Utilization trends of an ophthalmology-specific emergency department: the Massachusetts Eye and Ear experience.

To describe the utilization trends of a dedicated ophthalmology emergency department (ED) in Boston, Massachusetts. The medical records of 500 randomly selected patients who presented at the Massachusetts Eye and Ear (MEE) Emergency Department (ED) from January 2015 to March 2016 were reviewed retrospectively. Data were analyzed using the Pearson χ2 test and multiple logistic regression. The primary study outcome measure was whether a patient's visit was emergent or nonemergent. Emergent or nonemergent conditions were classified based on the diagnosis and treatment required at follow-up appointments. Nonemergent diagnoses were classified as conditions that could have been seen as an outpatient without negative consequences for vision. Of the 500 cases, 252 were males and 248 were females. The median age was 45 years (range, 2-101 years). The most common diagnoses were posterior vitreous detachment (8.6%), corneal abrasion (8.4%), dry eye syndrome (7%), and viral conjunctivitis (5.4%). Of the total, 92.6% of patients originated from within Massachusetts. The majority of patients were self-referred (78.6%) or referred from another hospital (12.8%). Nonemergent visits accounted for 49.4% of patients seen. Compared to patients who presented with duration of symptoms for ≥1 week, patients who presented with symptoms of <1 week were more likely to present with an emergent condition (8.8% vs 41.8%). Referrals from an outside ophthalmologist or hospital were predictive of emergent patient visits (OR, resp., 1.971 [95% CI, 0.478-3.462; P = 0.01]; 1.040 [95% CI, 0.462-1.616; P < 0.001]). In our study, nonemergent patient visits comprised nearly half of all ophthalmology ED visits. Emergent visits were associated with acute symptoms and referrals from outside healthcare providers.

Studying outcome predictors of drowning at the scene: Why do we have so few answers?

Identification of predictors of outcome at the scene of drowning events could guide prevention, care and resource utilization. This review aimed to describe where, what and how scene predictive factors have been evaluated in drowning outcome studies. We reviewed studies reporting scene drowning predictors published between 2003 and 2019. Data extraction included study populations, data sources, predictor factors (victim, incident, rescue, resuscitation and hospital-related), outcome measures and type of analyses. Of 49 studies, 87.6% were from high-income countries, 57.1% used data from only one source (92.9% of these from either hospital or EMS), 73.5% included cases who received medical care and 53.1% defined outcomes as survival or death. A total of 78 different factors were studied; the most commonly studied group of factors described victim demographics, included in 42 studies (85.7%), followed by resuscitation factors, included in 30 studies (61.2%). Few studies described rescue (6.1%). The most frequent statistically significant single predictors of outcome known at the scene were submersion duration (evaluated in 19, predictor in 14) and age (evaluated in 31, predictor in 16). Only 38.7% of studies employed multivariable methods. Gaps to be addressed in drowning outcomes research include data from low- and middle-income countries, standardized definition of factors to allow evaluation across studies, inclusive study populations that can be generalized beyond those receiving medical care, study rescue and resuscitation factors, use of more meaningful outcomes (survival with good neurologic status) and advanced analyses to identify which factors are true predictors versus confounding variables.