Category: Statistics

Stat Methods Med Res. 2022 Aug 30:9622802221122423. doi: 10.1177/09622802221122423. Online ahead of print.

ABSTRACT

In clinical and public health studies, it is often the case that some variables relevant to the analysis are too difficult or costly to measure for all individuals in the population of interest. Rather, a subsample of these individuals must be identified for additional data collection. A sampling scheme that incorporates readily-available information for the entire target population at the design stage can increase the statistical efficiency of the intended analysis. While there is no universally optimal sampling design, under certain principles and restrictions, a well-designed and efficient sampling strategy can be implemented. In two-phase designs, efficiency can be gained by stratifying on the outcome and/or auxiliary information that is known at phase I. Additional gains in efficiency can be obtained by determining the optimal allocation of the sample sizes across the strata, which depends on the quantity that is being estimated. In this paper, the inference is concerned with one or multiple regression parameter(s) where the study units are naturally clustered and, thus, exhibit correlation in outcomes. We propose several allocation strategies within the framework of two-phase designs for the estimation of the regression parameter(s) obtained from weighted generalized estimating equations. The proposed methods extend existing theory to address the objective of the estimating regression parameters in cluster-correlated data settings by minimizing the asymptotic variance of the estimator subject to a fixed sample size. Through a comprehensive simulation study, we show that the proposed allocation schemes have the potential to yield substantial efficiency gains over alternative strategies.

PMID:36039539 | DOI:10.1177/09622802221122423

Prog Transplant. 2022 Aug 30:15269248221122897. doi: 10.1177/15269248221122897. Online ahead of print.

ABSTRACT

Introduction: Pain following donor nephrectomy for living kidney donation is common. In Israel, non-directed, altruistic donations account for 45% of all kidney transplants. Design: This cross-sectional, retrospective survey included 2 groups of donors derived from the data of Israel Transplant, namely directed and non-directed, altruistic donors, who donated between 2015 to 2018. The degree of recalled pain memory post-surgery was assessed using the Visual Analog Scale at 5 time points: immediately post-surgery, after 1 week, 1 month and 3 months post-surgery and in the month preceding completion of the questionnaire. In addition, continued requirement for analgesics for more than one-month post-surgery, the degree of interference with daily activities in the month preceding the questionnaire and the recalled time to return to full-time employment were also noted. Results: In total, 246 (131 directed and 115 non-directed, altruistic) donors were included in the study. Non-directed, altruistic donors reported statistically significantly lower degrees of recalled pain memory at all time points, a lower requirement for prolonged analgesic use and less recalled interference with daily activities due to pain. In addition, these donors recalled returning significantly earlier to full-time employment. Finally, no significant differences in the degree of recalled pain memory were noted for directed donors according to their relation to the recipient, apart from donation to a spouse. Conclusion: These unique findings, if validated in a prospective study, could provide important information to potential non-directed, altruistic donors regarding the expected level of post-surgical pain and their return to full-time employment.

PMID:36039525 | DOI:10.1177/15269248221122897

Prog Transplant. 2022 Aug 29:15269248221122867. doi: 10.1177/15269248221122867. Online ahead of print.

ABSTRACT

Introduction: Given the negative outcomes associated with uncontrolled diabetes mellitus, non-insulin therapies with glycemic, cardiovascular, and weight-loss benefits in the general population, such as the glucagonlike peptide-1 receptor agonists (GLP1-RA) have become a more alluring therapeutic option in transplant populations. However, limited evidence exists to demonstrate its safety and efficacy in solid organ transplant. Methods: This program evaluation included adult kidney, liver, lung transplant recipients initiated on a GLP1-RA for diabetes mellitus management for a minimum of 3 months, had at least one follow-up visit after starting therapy, and had at least one hemoglobin A1c (HbA1c) level drawn between 3-12 months after GLP1-RA initiation. Outcomes were assessed at time of initiation of GLP1-RA (baseline) and 3-12 months post-initiation. Nadir values between 3-12 months were utilized to assess outcomes. Results: One-hundred eighteen patients met study inclusion criteria. Seventy-percent of patients received a kidney transplant, 19.5% received a liver transplant, and 6.8% received a lung transplant. A statistically significant difference was observed in median fasting blood glucose and HbA1c at baseline to 3-12-month nadir (P < 0.0001). A significant weight loss benefit was also observed. The rate of adverse drug reactions was low. Seven-percent of patients experienced nausea, 4.2% developed pancreatitis, and 7.1% reported having had at least one hypoglycemic event. Discussion: This is the largest study evaluating GLP1-RA in organ transplantation and demonstrates GLP1-RA is both safe and effective. Further assessment on long-term use of these agents on cardiovascular and renal outcomes is still needed.

PMID:36039519 | DOI:10.1177/15269248221122867

JMIR Public Health Surveill. 2022 Aug 29;8(8):e25735. doi: 10.2196/25735.

ABSTRACT

BACKGROUND: Effective health policy formulation requires sound information of the numerical data and causes of deaths in a population. Currently, in Bangladesh, neither births nor deaths are fully and promptly registered. Birth registration in Bangladesh is around 54% nationally. Although the legal requirements are to register within 45 days of an event, only 4.5% of births and 35.9% of deaths were reported within the required time frame in 2020. This study adopted an innovative digital notification approach to improve the coverage of registration of these events at the community level.

OBJECTIVE: Our primary objective was to assess (1) the proportion of events identified by the new notification systems (success rate) and the contribution of the different notifiers individually and in combination (completeness) and (2) the proportion of events notified within specific time limits (timeliness of notifications) after introducing the innovative approach.

METHODS: We conducted a pilot study in 2016 in 2 subdistricts of Bangladesh to understand whether accurate, timely, and complete information on births and deaths can be collected and notified by facility-based service providers; community health workers, including those who routinely visit households; local government authorities; and key informants from the community. We designed a mobile technology-based platform, an app, and a call center through which the notifications were recorded. All notifications were verified through the confirmation of events by family members during visits to the concerned households. We undertook a household survey-based assessment at the end of the notification period.

RESULTS: Our innovative system gathered 13,377 notifications for births and deaths from all channels, including duplicate reports from multiple sources. Project workers were able to verify 92% of the births and 93% of the deaths through household visits. The household survey conducted among a subsample of the project population identified 1204 births and 341 deaths. After matching the notifications with the household survey, we found that the system was able to capture over 87% of the births in the survey areas. Health assistants and family welfare assistants were the primary sources of information. Notifications from facilities were very low for both events.

CONCLUSIONS: The Global Civil Registration and Vital Statistics: Scaling Up Investment Plan 2015-2024 and the World Health Organization reiterated the importance of building an evidence base for improving civil registration and vital statistics. Our pilot innovation revealed that it is possible to coordinate with the routine health information system to note births and deaths as the first step to ensure registration. Health assistants could capture more than half of the notifications as a stand-alone source.

PMID:36036979 | DOI:10.2196/25735

Dermatol Surg. 2022 Aug 23. doi: 10.1097/DSS.0000000000003544. Online ahead of print.

ABSTRACT

BACKGROUND: Aesthetic procedures are widely performed by dermatologists and plastic surgeons, often with subjective results. In cosmetic dermatology, there is a lack of studies that use accurate instruments to assess result outcomes. Quality of life (QoL) questionnaires can objectively assess results.

OBJECTIVE: To assess the impact of nonsurgical facial cosmetic procedures on QoL.

MATERIALS AND METHODS: This was a longitudinal observational study performed with 81 patients who underwent facial cosmetic procedures at a University Hospital in Rio de Janeiro, Brazil. The Skin Rejuvenation Outcome Evaluation questionnaire, previously adapted and translated to the Portuguese language spoken in Brazil, was selected, and the results were compared with the Dermatology Life Quality Index. The impact on QoL was statistically evaluated after the interventions.

RESULTS: The Skin Rejuvenation Outcome Evaluation scores after the procedures were significantly higher than before (p < .001), independent of sociodemographic characteristics. Patients treated with botulinum toxin and dermal fillers showed greater satisfaction than those treated with laser, chemical peels or microneedling.

CONCLUSION: Minimally invasive cosmetic procedures result in improvements in QoL. The use of translated, adapted, and validated questionnaires favors the comparison of results among different populations and reinforces the confidence on the results of established cosmetic treatments.

PMID:36036975 | DOI:10.1097/DSS.0000000000003544

JMIR Public Health Surveill. 2022 Aug 29;8(8):e37656. doi: 10.2196/37656.

ABSTRACT

BACKGROUND: The human papillomavirus (HPV) vaccine is recommended for adolescents and young adults to prevent HPV-related cancers and genital warts. However, HPV vaccine uptake among the target age groups is suboptimal.

OBJECTIVE: The aim of this infodemiology study was to examine public online searches in the United States related to the HPV vaccine from January 2010 to December 2021.

METHODS: Google Trends (GT) was used to explore online searches related to the HPV vaccine from January 1, 2010, to December 31, 2021. Online searches and queries on the HPV vaccine were investigated using relative search volumes (RSVs). Analysis of variance was performed to investigate quarterly differences in HPV vaccine searches in each year from 2010 to 2021. A joinpoint regression was used to identify statistically significant changes over time; the α level was set to .05.

RESULTS: The year-wise online search volume related to the HPV vaccine increased from 2010 to 2021, often following federal changes related to vaccine administration. Joinpoint regression analysis showed that HPV vaccine searches significantly increased on average by 8.6% (95% CI 5.9%-11.4%) across each year from 2010 to 2021. Moreover, HPV vaccine searches demonstrated a similar pattern across years, with search interest increasing through August nearly every year. At the state level, the highest 12-year mean RSV was observed in California (59.9, SD 14.3) and the lowest was observed in Wyoming (17.4, SD 8.5) during the period of 2010-2021.

CONCLUSIONS: Online searches related to the HPV vaccine increased by an average of 8.6% across each year from 2010 to 2021, with noticeable spikes corresponding to key changes in vaccine recommendations. We identified patterns across years and differences at the state level in the online search interest related to the HPV vaccine. Public health organizations can use GT as a tool to characterize the public interest in and promote the HPV vaccine in the United States.

PMID:36036972 | DOI:10.2196/37656

JAMA Pediatr. 2022 Aug 29. doi: 10.1001/jamapediatrics.2022.2535. Online ahead of print.

ABSTRACT

IMPORTANCE: Inappropriate use of antibiotics for diarrheal illness can result in adverse effects and increase in antimicrobial resistance.

OBJECTIVE: To determine whether the diarrheal etiology prediction (DEP) algorithm, which uses patient-specific and location-specific features to estimate the probability that diarrhea etiology is exclusively viral, impacts antibiotic prescriptions in patients with acute diarrhea.

DESIGN, SETTING, AND PARTICIPANTS: A randomized crossover study was conducted to evaluate the DEP incorporated into a smartphone-based electronic clinical decision-support (eCDS) tool. The DEP calculated the probability of viral etiology of diarrhea, based on dynamic patient-specific and location-specific features. Physicians were randomized in the first 4-week study period to the intervention arm (eCDS with the DEP) or control arm (eCDS without the DEP), followed by a 1-week washout period before a subsequent 4-week crossover period. The study was conducted at 3 sites in Bangladesh from November 17, 2021, to January 21, 2021, and at 4 sites in Mali from January 6, 2021, to March 5, 2021. Eligible physicians were those who treated children with diarrhea. Eligible patients were children between ages 2 and 59 months with acute diarrhea and household access to a cell phone for follow-up.

INTERVENTIONS: Use of the eCDS with the DEP (intervention arm) vs use of the eCDS without the DEP (control arm).

MAIN OUTCOMES AND MEASURES: The primary outcome was the proportion of children prescribed an antibiotic.

RESULTS: A total of 30 physician participants and 941 patient participants (57.1% male; median [IQR] age, 12 [8-18] months) were enrolled. There was no evidence of a difference in the proportion of children prescribed antibiotics by physicians using the DEP (risk difference [RD], -4.2%; 95% CI, -10.7% to 1.0%). In a post hoc analysis that accounted for the predicted probability of a viral-only etiology, there was a statistically significant difference in risk of antibiotic prescription between the DEP and control arms (RD, -0.056; 95% CI, -0.128 to -0.01). No known adverse effects of the DEP were detected at 10-day postdischarge.

CONCLUSIONS AND RELEVANCE: Use of a tool that provides an estimate of etiological likelihood did not result in a significant change in overall antibiotic prescriptions. Post hoc analysis suggests that a higher predicted probability of viral etiology was linked to reductions in antibiotic use.

TRIAL REGISTRATION: Clinicaltrials.gov Identifier: NCT04602676.

PMID:36036920 | DOI:10.1001/jamapediatrics.2022.2535

JAMA. 2022 Aug 29. doi: 10.1001/jama.2022.15071. Online ahead of print.

ABSTRACT

IMPORTANCE: During the COVID-19 pandemic, the US federal government required that skilled nursing facilities (SNFs) close to visitors and eliminate communal activities. Although these policies were intended to protect residents, they may have had unintended negative effects.

OBJECTIVE: To assess health outcomes among SNFs with and without known COVID-19 cases.

DESIGN, SETTING, AND PARTICIPANTS: This retrospective observational study used US Medicare claims and Minimum Data Set 3.0 for January through November in each year beginning in 2018 and ending in 2020 including 15 477 US SNFs with 2 985 864 resident-years.

EXPOSURES: January through November of calendar years 2018, 2019, and 2020. COVID-19 diagnoses were used to assign SNFs into 2 mutually exclusive groups with varying membership by month in 2020: active COVID-19 (≥1 COVID-19 diagnosis in the current or past month) or no-known COVID-19 (no observed diagnosis by that month).

MAIN OUTCOMES AND MEASURES: Monthly rates of mortality, hospitalization, emergency department (ED) visits, and monthly changes in activities of daily living (ADLs), body weight, and depressive symptoms. Each SNF in 2018 and 2019 served as its own control for 2020.

RESULTS: In 2018-2019, mean monthly mortality was 2.2%, hospitalization 3.0%, and ED visit rate 2.9% overall. In 2020, among active COVID-19 SNFs compared with their own 2018-2019 baseline, mortality increased by 1.60% (95% CI, 1.58% to 1.62%), hospitalizations decreased by 0.10% (95% CI, -0.12% to -0.09%), and ED visit rates decreased by 0.57% (95% CI, -0.59% to -0.55%). Among no-known COVID-19 SNFs, mortality decreased by 0.15% (95% CI, -0.16% to -0.13%), hospitalizations by 0.83% (95% CI, -0.85% to -0.81%), and ED visits by 0.79% (95% CI, -0.81% to -0.77%). All changes were statistically significant. In 2018-2019, across all SNFs, residents required assistance with an additional 0.89 ADLs between January and November, and lost 1.9 lb; 27.1% had worsened depressive symptoms. In 2020, residents in active COVID-19 SNFs required assistance with an additional 0.36 ADLs (95% CI, 0.34 to 0.38), lost 3.1 lb (95% CI, -3.2 to -3.0 lb) more weight, and were 4.4% (95% CI, 4.1% to 4.7%) more likely to have worsened depressive symptoms, all statistically significant changes. In 2020, residents in no-known COVID-19 SNFs had no significant change in ADLs (-0.06 [95% CI, -0.12 to 0.01]), but lost 1.8 lb (95% CI, -2.1 to -1.5 lb) more weight and were 3.2% more likely (95% CI, 2.3% to 4.1%) to have worsened depressive symptoms, both statistically significant changes.

CONCLUSIONS AND RELEVANCE: Among skilled nursing facilities in the US during the first year of the COVID-19 pandemic and prior to the availability of COVID-19 vaccination, mortality and functional decline significantly increased at facilities with active COVID-19 cases compared with the prepandemic period, while a modest statistically significant decrease in mortality was observed at facilities that had never had a known COVID-19 case. Weight loss and depressive symptoms significantly increased in skilled nursing facilities in the first year of the pandemic, regardless of COVID-19 status.

PMID:36036916 | DOI:10.1001/jama.2022.15071

Pain. 2022 Aug 26. doi: 10.1097/j.pain.0000000000002768. Online ahead of print.

ABSTRACT

Conventional “1-variable-at-a-time” analyses to identify treatment effect modifiers are often underpowered and prone to false positive results. This study used a “risk-modeling” approach guided by the Predictive Approaches to Treatment effect Heterogeneity (PATH) Statement framework: (1) developing and validating a multivariable model to estimate predicted future back-related functional limitations as measured by the Roland-Morris Disability Questionnaire (RMDQ); and (2) stratifying patients from a randomized controlled trial (RCT) of lumbar epidural steroid injections (LESI) for the treatment of lumbar spinal stenosis into subgroups with different individualized treatment effects on RMDQ scores at 3-week follow-up. Model development and validation was conducted in a cohort (n=3259) randomly split into training and testing sets in a 4:1 ratio. The model was developed in the testing set using linear regression with least absolute shrinkage and selection regularization and 5-fold cross-validation. The model was then applied in the testing set, and subsequently in patients receiving the control treatment in the RCT of LESI. R2 values in the training set, testing set and RCT were 0.38, 0.32, and 0.34, respectively. There was statistically significant modification (p=0.03) of the LESI treatment effect according to predicted risk quartile, with clinically relevant LESI treatment effect point estimates in the two quartiles with greatest predicted risk (-3.7 and -3.3 RMDQ points) and no effect in the lowest two quartiles. A multivariable risk-modeling approach identified subgroups of patients with lumbar spinal stenosis with a clinically relevant treatment effect of LESI on back-related functional limitations.

PMID:36036907 | DOI:10.1097/j.pain.0000000000002768

J Urol. 2022 Aug 29:101097JU0000000000002891. doi: 10.1097/JU.0000000000002891. Online ahead of print.

ABSTRACT

INTRODUCTION/BACKGROUND: With increasing telehealth technology, confirming the validity of non-office administration of questionnaires intended for office use only is important. We studied three validated questionnaires: Urinary Distress Inventory 6-Short Form (UDI-6), International Incontinence 7- Short Form (IIQ-7), and one Quality of Life Survey (QoL) using a deception model.

METHODS/MATERIALS: Following IRB approval and power calculations, these 3 questionnaires were prospectively administered in women with and without incontinence by phone and then again in person about 2 weeks later. To avoid recall bias, participants were not informed of the study over the phone. After completing their office visit questionnaire scores, they were told about the study and invited to consent for comparison of their phone and in-person questionnaire scores. Non-English speakers and those with an active urinary tract infection, impaired mental competency, or on fluid diets were excluded.

RESULTS: From June to September 2021, 86 women, including 40 incontinent (30-85) and 46 control (30-85), with similar demographic parameters, met all study criteria. Of the 14 questions studied, only two, the UDI6: Q1 (p=0.033) and IIQ-7: Q7 (p=0.036), showed rather minimal but statistically significant differences in scores. For incontinent women, only the IIQ-7: Q7 (p=0.012) showed a significant score difference.

CONCLUSIONS: The three questionnaire scores were overall comparable when obtained over the phone or during office visit. Women with incontinence, who may otherwise be lost to follow-up or only reachable by telehealth calls, can benefit from the remote administration of these 3 questionnaires.

PMID:36036901 | DOI:10.1097/JU.0000000000002891