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Nevin Manimala Statistics

Estimation of planned and unplanned missing individual scores in longitudinal designs using continuous-time state-space models

Psychol Methods. 2024 May 16. doi: 10.1037/met0000664. Online ahead of print.

ABSTRACT

Latent change score (LCS) models within a continuous-time state-space modeling framework provide a convenient statistical approach for analyzing developmental data. In this study, we evaluate the robustness of such an approach in the context of accelerated longitudinal designs (ALDs). ALDs are especially interesting because they imply a very high rate of planned data missingness. Additionally, most longitudinal studies present unexpected participant attrition leading to unplanned missing data. Therefore, in ALDs, both sources of data missingness are combined. Previous research has shown that ALDs for developmental research allow recovering the population generating process. However, it is unknown how participant attrition impacts the model estimates. We have three goals: (a) to evaluate the robustness of the group-level parameter estimates in scenarios with empirically plausible unplanned data missingness; (b) to evaluate the performance of Kalman scores (KS) imputations for individual data points that were expected but unobserved; and (c) to evaluate the performance of KS imputations for individual data points that were outside the age ranged observed for each case (i.e., to estimate the individual trajectories for the complete age range under study). In general, results showed lack of bias in the simulated conditions. The variability of the estimates increased with lower sample sizes and higher missingness severity. Similarly, we found very accurate estimates of individual scores for both planned and unplanned missing data points. These results are very important for applied practitioners in terms of forecasting and making individual-level decisions. R code is provided to facilitate its implementation by applied researchers. (PsycInfo Database Record (c) 2024 APA, all rights reserved).

PMID:38753382 | DOI:10.1037/met0000664

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Nevin Manimala Statistics

Value of Real-World Evidence for Treatment Selection: A Case Study in Colon Cancer

JCO Clin Cancer Inform. 2024 May;8:e2300186. doi: 10.1200/CCI.23.00186.

ABSTRACT

PURPOSE: Real-world evidence (RWE)-derived from analysis of real-world data (RWD)-has the potential to guide personalized treatment decisions. However, because of potential confounding, generating valid RWE is challenging. This study demonstrates how to responsibly generate RWE for treatment decisions. We validate our approach by demonstrating that we can uncover an existing adjuvant chemotherapy (ACT) guideline for stage II and III colon cancer (CC)-which came about using both data from randomized controlled trials and expert consensus-solely using RWD.

METHODS: Data from the population-based Netherlands Cancer Registry from a total of 27,056 patients with stage II and III CC who underwent curative surgery were analyzed to estimate the overall survival (OS) benefit of ACT. Focusing on 5-year OS, the benefit of ACT was estimated for each patient using G-computation methods by adjusting for patient and tumor characteristics and estimated propensity score. Subsequently, on the basis of these estimates, an ACT decision tree was constructed.

RESULTS: The constructed decision tree corresponds to the current Dutch guideline: patients with stage III or stage II with T stage 4 should receive surgery and ACT, whereas patients with stage II with T stage 3 should only receive surgery. Interestingly, we do not find sufficient RWE to conclude against ACT for stage II with T stage 4 and microsatellite instability-high (MSI-H), a recent addition to the current guideline.

CONCLUSION: RWE, if used carefully, can provide a valuable addition to our construction of evidence on clinical decision making and therefore ultimately affect treatment guidelines. Next to validating the ACT decisions advised in the current Dutch guideline, this paper suggests additional attention should be paid to MSI-H in future iterations of the guideline.

PMID:38753347 | DOI:10.1200/CCI.23.00186

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Nevin Manimala Statistics

Systemic Anticancer Therapy and Overall Survival in Patients With Very Advanced Solid Tumors

JAMA Oncol. 2024 May 16. doi: 10.1001/jamaoncol.2024.1129. Online ahead of print.

ABSTRACT

IMPORTANCE: Two prominent organizations, the American Society of Clinical Oncology and the National Quality Forum (NQF), have developed a cancer quality metric aimed at reducing systemic anticancer therapy administration at the end of life. This metric, NQF 0210 (patients receiving chemotherapy in the last 14 days of life), has been critiqued for focusing only on care for decedents and not including the broader population of patients who may benefit from treatment.

OBJECTIVE: To evaluate whether the overall population of patients with metastatic cancer receiving care at practices with higher rates of oncologic therapy for very advanced disease experience longer survival.

DESIGN, SETTING, AND PARTICIPANTS: This nationwide population-based cohort study used Flatiron Health, a deidentified electronic health record database of patients diagnosed with metastatic or advanced disease, to identify adult patients (aged ≥18 years) with 1 of 6 common cancers (breast cancer, colorectal cancer, non-small cell lung cancer [NSCLC], pancreatic cancer, renal cell carcinoma, and urothelial cancer) treated at health care practices from 2015 to 2019. Practices were stratified into quintiles based on retrospectively measured rates of NQF 0210, and overall survival was compared by disease type among all patients treated in each practice quintile from time of metastatic diagnosis using multivariable Cox proportional hazard models with a Bonferroni correction for multiple comparisons. Data were analyzed from July 2021 to July 2023.

EXPOSURE: Practice-level NQF 0210 quintiles.

MAIN OUTCOME AND MEASURE: Overall survival.

RESULTS: Of 78 446 patients (mean [SD] age, 67.3 [11.1] years; 52.2% female) across 144 practices, the most common cancer types were NSCLC (34 201 patients [43.6%]) and colorectal cancer (15 804 patients [20.1%]). Practice-level NQF 0210 rates varied from 10.9% (quintile 1) to 32.3% (quintile 5) for NSCLC and 6.8% (quintile 1) to 28.4% (quintile 5) for colorectal cancer. No statistically significant differences in survival were observed between patients treated at the highest and the lowest NQF 0210 quintiles. Compared with patients seen at practices in the lowest NQF 0210 quintiles, the hazard ratio for death among patients seen at the highest quintiles varied from 0.74 (95% CI, 0.55-0.99) for those with renal cell carcinoma to 1.41 (95% CI, 0.98-2.02) for those with urothelial cancer. These differences were not statistically significant after applying the Bonferroni-adjusted critical P = .008.

CONCLUSIONS AND RELEVANCE: In this cohort study, patients with metastatic or advanced cancer treated at practices with higher NQF 0210 rates did not have improved survival. Future efforts should focus on helping oncologists identify when additional therapy is futile, developing goals of care communication skills, and aligning payment incentives with improved end-of-life care.

PMID:38753341 | DOI:10.1001/jamaoncol.2024.1129

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Nevin Manimala Statistics

Clotrimazole-Betamethasone Dipropionate Prescribing for Nonfungal Skin Conditions

JAMA Netw Open. 2024 May 1;7(5):e2411721. doi: 10.1001/jamanetworkopen.2024.11721.

NO ABSTRACT

PMID:38753333 | DOI:10.1001/jamanetworkopen.2024.11721

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Nevin Manimala Statistics

Effectiveness of a Web-Based Cognitive Behavioral Self-Help Intervention for Binge Eating Disorder: A Randomized Clinical Trial

JAMA Netw Open. 2024 May 1;7(5):e2411127. doi: 10.1001/jamanetworkopen.2024.11127.

ABSTRACT

IMPORTANCE: Binge eating disorder (BED) is one of the most frequent eating pathologies and imposes substantial emotional and physical distress, yet insufficient health care resources limit access to specialized treatment. Web-based self-help interventions emerge as a promising solution, offering more accessible care.

OBJECTIVE: To examine the effectiveness of a web-based cognitive behavioral self-help intervention for individuals with BED.

DESIGN, SETTING, AND PARTICIPANTS: This 2-arm, parallel-group randomized clinical trial conducted from January 15, 2021, to August 3, 2022, in Germany and other German-speaking countries enrolled patients aged 18 to 65 years who met the diagnostic criteria for BED (according to the Diagnostic and Statistical Manual of Mental Disorders [Fifth Edition]). Data analysis occurred between January 27 and September 4, 2023, following our statistical analysis plan.

INTERVENTIONS: Participants were randomized to a web-based self-help intervention or a waiting-list control condition.

MAIN OUTCOMES AND MEASURES: The primary outcome was a change in objective binge eating episodes from baseline to after treatment. Secondary outcomes included global eating pathology, clinical impairment, work capacity, well-being, comorbid psychopathology, self-esteem, and emotion regulation.

RESULTS: A total of 1602 patients were screened, of whom 154 (mean [SD] age, 35.93 [10.59] years; 148 female [96.10%]) fulfilled the criteria for BED and were randomized (77 each to the intervention and control groups). The web-based intervention led to significant improvements in binge eating episodes (Cohen d, -0.79 [95% CI, -1.17 to -0.42]; P < .001), global eating psychopathology (Cohen d, -0.71 [95% CI, -1.07 to -0.35]; P < .001), weekly binge eating (Cohen d, -0.49 [95% CI, -0.74 to -0.24]; P < .001), clinical impairment (Cohen d, -0.75 [95% CI, -1.13 to -0.37]; P < .001), well-being (Cohen d, 0.38 [95% CI, 0.01 to 0.75]; P = .047), depression (Cohen d, -0.49 [95% CI, -0.86 to -0.12]; P = .01), anxiety (Cohen d, -0.37 [95% CI, -0.67 to -0.07]; P = .02), self-esteem (Cohen d, 0.36 [95% CI, 0.13 to 0.59]; P = .003), and emotion regulation (difficulties: Cohen d, -0.36 [95% CI, -0.65 to -0.07]; P = .01 and repertoire: Cohen d, 0.52 [95% CI, 0.19 to 0.84]; P = .003).

CONCLUSION AND RELEVANCE: In this randomized clinical trial of a web-based self-help intervention for patients with BED, the findings confirmed its effectiveness in reducing binge eating episodes and improving various mental health outcomes, highlighting a scalable solution to bridge the treatment gap for this condition.

TRIAL REGISTRATION: ClinicalTrials.gov Identifier: NCT04876183.

PMID:38753330 | DOI:10.1001/jamanetworkopen.2024.11127

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Nevin Manimala Statistics

Hospice Readmission, Hospitalization, and Hospital Death Among Patients Discharged Alive from Hospice

JAMA Netw Open. 2024 May 1;7(5):e2411520. doi: 10.1001/jamanetworkopen.2024.11520.

ABSTRACT

IMPORTANCE: Transitions in care settings following live discharge from hospice care are burdensome for patients and families. Factors contributing to risk of burdensome transitions following hospice discharge are understudied.

OBJECTIVE: To identify factors associated with 2 burdensome transitions following hospice live discharge, as defined by the Centers for Medicare & Medicaid Services.

DESIGN, SETTING, AND PARTICIPANTS: This population-based retrospective cohort study included a 20% random sample of Medicare fee-for-service beneficiaries using 2014 to 2019 Medicare claims data. Data were analyzed from April 22, 2023, to March 4, 2024.

EXPOSURE: Live hospice discharge.

MAIN OUTCOMES AND MEASURES: Multivariable logistic regression examined associations among patient, health care provision, and organizational characteristics with 2 burdensome transitions after live hospice discharge (outcomes): type 1, hospice discharge, hospitalization within 2 days, and hospice readmission within 2 days; and type 2, hospice discharge, hospitalization within 2 days, and hospital death.

RESULTS: This study included 115 072 Medicare beneficiaries discharged alive from hospice (mean [SD] age, 84.4 [6.6] years; 71892 [62.5%] female; 5462 [4.8%] Hispanic, 9822 [8.5%] non-Hispanic Black, and 96 115 [83.5%] non-Hispanic White). Overall, 10 381 individuals (9.0%) experienced a type 1 burdensome transition and 3144 individuals (2.7%) experienced a type 2 burdensome transition. In adjusted models, factors associated with higher odds of burdensome transitions included identifying as non-Hispanic Black (type 1: adjusted odds ratio [aOR], 1.47; 95% CI, 1.36-1.58; type 2: aOR, 1.70; 95% CI, 1.51-1.90), hospice stays of 7 days or fewer (type 1: aOR, 1.13; 95% CI, 1.06-1.21; type 2: aOR, 1.71; 95% CI, 1.53-1.90), and care from a for-profit hospice (type 1: aOR, 1.78; 95% CI, 1.62-1.96; type 2: aOR, 1.32; 95% CI, 1.15-1.52). Nursing home residence (type 1: aOR, 0.66; 95% CI, 0.61-0.72; type 2: aOR, 0.47; 95% CI, 0.40-0.54) and hospice stays of 180 days or longer (type 1: aOR, 0.63; 95% CI, 0.59-0.68; type 2: aOR, 0.60; 95% CI, 0.52-0.69) were associated with lower odds of burdensome transitions.

CONCLUSION AND RELEVANCE: This retrospective cohort study of burdensome transitions following live hospice discharge found that non-Hispanic Black race, short hospice stays, and care from for-profit hospices were associated with higher odds of experiencing a burdensome transition. These findings suggest that changes to clinical practice and policy may reduce the risk of burdensome transitions, such as hospice discharge planning that is incentivized, systematically applied, and tailored to needs of patients at greater risk for burdensome transitions.

PMID:38753329 | DOI:10.1001/jamanetworkopen.2024.11520

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Nevin Manimala Statistics

Pharmacogenomics, Race, and Treatment Outcome in Pediatric Acute Myeloid Leukemia

JAMA Netw Open. 2024 May 1;7(5):e2411726. doi: 10.1001/jamanetworkopen.2024.11726.

ABSTRACT

IMPORTANCE: Disparities in outcomes exist between Black and White patients with acute myeloid leukemia (AML), with Black patients experiencing poorer prognosis compared with their White counterparts.

OBJECTIVE: To assess whether varying intensity of induction therapy to treat pediatric AML is associated with reduced disparities in treatment outcome by race.

DESIGN, SETTING, AND PARTICIPANTS: A comparative effectiveness analysis was conducted of 86 Black and 359 White patients with newly diagnosed AML who were enrolled in the AML02 trial from 2002 to 2008 or the AML08 trial from 2008 to 2017. Statistical analysis was conducted from July 2023 through January 2024.

INTERVENTIONS: Patients in AML02 were randomly assigned to receive standard low-dose cytarabine-based induction therapy or augmented high-dose cytarabine-based induction therapy, whereas patients in AML08 received high-dose cytarabine-based therapy.

MAIN OUTCOMES AND MEASURES: Cytarabine pharmacogenomic 10-single-nucleotide variant (ACS10) scores were evaluated for association with outcome according to race and treatment arm.

RESULTS: This analysis included 86 Black patients (mean [SD] age, 8.8 [6.5] years; 54 boys [62.8%]; mean [SD] leukocyte count, 52 600 [74 000] cells/µL) and 359 White patients (mean [SD] age, 9.1 [6.2] years; 189 boys [52.6%]; mean [SD] leukocyte count, 54 500 [91 800] cells/µL); 70 individuals with other or unknown racial and ethnic backgrounds were not included. Among all patients without core binding factor AML who received standard induction therapy, Black patients had significantly worse outcomes compared with White patients (5-year event-free survival rate, 25% [95% CI, 9%-67%] compared with 56% [95% CI, 46%-70%]; P = .03). By contrast, among all patients who received augmented induction therapy, there were no differences in outcome according to race (5-year event-free survival rate, Black patients, 50% [95% CI, 38%-67%]; White patients, 48% [95% CI, 42%-55%]; P = .78). Among patients who received standard induction therapy, those with low ACS10 scores had a significantly worse 5-year event-free survival rate compared with those with high scores (42.4% [95% CI, 25.6%-59.3%] and 70.0% [95% CI, 56.6%-83.1%]; P = .004); however, among patients who received augmented induction therapy, there were no differences in 5-year event-free survival rates according to ACS10 score (low score, 60.6% [95% CI, 50.9%-70.2%] and high score, 54.8% [95% CI, 47.1%-62.5%]; P = .43).

CONCLUSIONS AND RELEVANCE: In this comparative effectiveness study of pediatric patients with AML treated in 2 consecutive clinical trials, Black patients had worse outcomes compared with White patients after treatment with standard induction therapy, but this disparity was eliminated by treatment with augmented induction therapy. When accounting for ACS10 scores, no outcome disparities were seen between Black and White patients. Our results suggest that using pharmacogenomics parameters to tailor induction regimens for both Black and White patients may narrow the racial disparity gap in patients with AML.

PMID:38753328 | DOI:10.1001/jamanetworkopen.2024.11726

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Nevin Manimala Statistics

Calculation of Overall Hospital Quality Star Ratings With and Without Inclusion of the Peer Grouping Step

JAMA Netw Open. 2024 May 1;7(5):e2411933. doi: 10.1001/jamanetworkopen.2024.11933.

ABSTRACT

IMPORTANCE: The Centers for Medicare & Medicaid Services (CMS) Overall Star Rating is widely used by patients and consumers, and there is continued stakeholder curiosity surrounding the inclusion of a peer grouping step, implemented to the 2021 Overall Star Rating methods.

OBJECTIVE: To calculate hospital star rating scores with and without the peer grouping step, with the former approach stratifying hospitals into 3-, 4-, and 5-measure group peer groups based on the number of measure groups with at least 3 reported measures.

DESIGN, SETTING, AND PARTICIPANTS: This cross-sectional study used Care Compare website data from January 2023 for 3076 hospitals that received a star rating in 2023. Data were analyzed from April 2023 to December 2023.

EXPOSURE: Peer grouping vs no peer grouping.

MAIN OUTCOMES AND MEASURES: The primary outcome was the distribution of star ratings, with 1 star being the lowest-performing hospitals and 5 stars, the highest. Analyses additionally identified the number of hospitals with a higher, lower, or identical star rating with the use of the peer grouping step compared with its nonuse, stratified by certain hospital characteristics.

RESULTS: Among 3076 hospitals that received a star rating in 2023, most were nonspecialty (1994 hospitals [64.8%]), nonteaching (1807 hospitals [58.7%]), non-safety net (2326 hospitals [75.6%]), non-critical access (2826 hospitals [91.9%]) hospitals with fewer than 200 beds (1822 hospitals [59.2%]) and located in an urban geographic designations (1935 hospitals [62.9%]). The presence of the peer grouping step resulted in 585 hospitals (19.0%) being assigned a different star rating than if the peer grouping step was absent, including considerably more hospitals receiving a higher star rating (517 hospitals) rather than a lower (68 hospitals) star rating. Hospital characteristics associated with a higher star rating included urbanicity (351 hospitals [67.9%]), non-safety net status (414 hospitals [80.1%]), and fewer than 200 beds (287 hospitals [55.6%]). Collectively, the presence of the peer grouping step supports a like-to-like comparison among hospitals and supports the ability of patients to assess overall hospital quality.

CONCLUSIONS AND RELEVANCE: In this cross-sectional study, inclusion of the peer grouping in the CMS star rating method resulted in modest changes in hospital star ratings compared with application of the method without peer grouping. Given improvement in face validity and the close association between the current peer grouping approach and stakeholder needs for peer-comparison, the current CMS Overall Star Rating method allows for durable comparisons in hospital performance.

PMID:38753326 | DOI:10.1001/jamanetworkopen.2024.11933

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Nevin Manimala Statistics

Intraoperative intravenous versus periarticular injection of glucocorticoids in improving clinical outcomes after total knee arthroplasty: A prospective, randomized and controlled study

J Orthop Surg (Hong Kong). 2024 May-Aug;32(2):10225536241256554. doi: 10.1177/10225536241256554.

ABSTRACT

BACKGROUND: Glucocorticoids have been widely used in perioperative period for postoperative pain relief after total knee arthroplasty (TKA). However, the optimal administration protocols of glucocorticoids remain controversial. This study aims to compare the efficacy of glucocorticoids between intravenous and periarticular injection on clinical outcomes.

METHODS: A total of 114 patients were randomly assigned to intravenous (IV) group (n = 57) and periarticular injection (PI) group (n = 57). The IV group received 10 mg dexamethasone intravenously and the PI group received periarticular injection of 10 mg dexamethasone during the procedure. The clinical outcomes were assessed using visual analogue scale (VAS), knee society score (KSS), range of motion (ROM), knee swelling, inflammation markers and complications after TKA.

RESULTS: The VAS score during walking at 2nd day postoperatively was lower in the PI group compared with the IV group (2.08 ± 1.45 vs 2.73 ± 1.69, p = .039), and there was no significant difference at the other time points of VAS score in two groups. The inflammation markers, knee swelling, knee ROM and KSS score were not statistically different. Vomiting and other complications occurrence were not significantly different between the two groups.

CONCLUSIONS: Intraoperative periarticular injection of glucocorticoids has similar analgesic effect compared to intravenous in the postoperative period following TKA and may be even more effective on the second postoperative day. In addition, periarticular injection of glucocorticoids does not impose an excess risk or complication on patients.

PMID:38753310 | DOI:10.1177/10225536241256554

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Nevin Manimala Statistics

Modernizing public health communication competencies in Canada: A survey of the Canadian public health workforce

Can J Public Health. 2024 May 16. doi: 10.17269/s41997-024-00890-w. Online ahead of print.

ABSTRACT

OBJECTIVES: Since the publication of the Core Competencies for Public Health in Canada in 2008, the public health and communication landscape has changed dramatically. Digital media and infodemics have shifted how practitioners must communicate and respond to health information. The age of the current competency framework, which is relied on for workforce development, alongside emerging public health challenges, have prompted calls for modernized competency statements. This study aims to (i) measure self-reported communication competence in the public health workforce, (ii) measure agreement with new communication competency statements, (iii) identify variation in agreement between sub-groups of professionals, and (iv) explore current and needed communication training.

METHODS: Using a mixed-methods online survey, a sample of 378 participants in various Canadian public health roles and regions were asked to rate their current communication competence and agreement with a modernized, evidence-based draft communication competency framework. The survey was distributed in both official languages through partner organizations and social media. Descriptive statistics were performed to assess agreement and variation was analyzed in relation to public health roles and experience.

RESULTS: While most participants self-reported communication competence, specific areas were rated lower. All 21 proposed competency statements received high agreement with some variation observed between expertise and experience levels. Demand for communication training is high.

CONCLUSION: Strong agreement with statements indicates support for a modernized communication competency framework among sampled professionals. Research to gather more evidence surrounding the communication demands of the public health workforce and observed variation in strong agreement for the proposed statements is underway.

PMID:38753267 | DOI:10.17269/s41997-024-00890-w