Browsing by Subject "Outcome assessment"

Now showing 1 - 7 of 7
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    Burosumab treatment in adults with X-linked hypophosphataemia: 96-week patient-reported outcomes and ambulatory function from a randomised phase 3 trial and open-label extension
    (BMJ, 2021) Briot, Karine; Portale, Anthony A.; Brandi, Maria Luisa; Carpenter, Thomas O.; Cheong, Hae Ii; Cohen-Solal, Martine; Crowley, Rachel K.; Eastell, Richard; Imanishi, Yasuo; Ing, Steven; Insogna, Karl; Ito, Nobuaki; de Beur, Suzanne Jan; Javaid, Muhammad K.; Kamenicky, Peter; Keen, Richard; Kubota, Takuo; Lachmann, Robin H.; Perwad, Farzana; Pitukcheewanont, Pisit; Ralston, Stuart H.; Takeuchi, Yasuhiro; Tanaka, Hiroyuki; Weber, Thomas J.; Yoo, Han-Wook; Nixon, Annabel; Nixon, Mark; Sun, Wei; Williams, Angela; Imel, Erik A.; Medicine, School of Medicine
    Objectives: To report the impact of burosumab on patient-reported outcomes (PROs) and ambulatory function in adults with X-linked hypophosphataemia (XLH) through 96 weeks. Methods: Adults diagnosed with XLH were randomised 1:1 in a double-blinded trial to receive subcutaneous burosumab 1 mg/kg or placebo every 4 weeks for 24 weeks (NCT02526160). Thereafter, all subjects received burosumab every 4 weeks until week 96. PROs were measured using the Western Ontario and the McMaster Universities Osteoarthritis Index (WOMAC), Brief Pain Inventory-Short Form (BPI-SF) and Brief Fatigue Inventory (BFI), and ambulatory function was measured with the 6 min walk test (6MWT). Results: Subjects (N=134) were randomised to burosumab (n=68) or placebo (n=66) for 24 weeks. At baseline, subjects experienced pain, stiffness, and impaired physical and ambulatory function. At week 24, subjects receiving burosumab achieved statistically significant improvement in some BPI-SF scores, BFI worst fatigue (average and greatest) and WOMAC stiffness. At week 48, all WOMAC and BPI-SF scores achieved statistically significant improvement, with some WOMAC and BFI scores achieving meaningful and significant change from baseline. At week 96, all WOMAC, BPI-SF and BFI achieved statistically significant improvement, with selected scores in all measures also achieving meaningful change. Improvement in 6MWT distance and percent predicted were statistically significant at all time points from 24 weeks. Conclusions: Adults with XLH have substantial burden of disease as assessed by PROs and 6MWT. Burosumab treatment improved phosphate homoeostasis and was associated with a steady and consistent improvement in PROs and ambulatory function.
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    Endoscopic versus surgical management for colonic volvulus hospitalizations in the United States
    (Korean Society of Gastrointestinal Endoscopy, 2023) Dahiya, Dushyant Singh; Perisetti, Abhilash; Goyal, Hemant; Inamdar, Sumant; Singh, Amandeep; Garg, Rajat; Cheng, Chin-I; Al-Haddad, Mohammad; Sanaka, Madhusudhan R.; Sharma, Neil; Medicine, School of Medicine
    Background/aims: Colonic volvulus (CV), a common cause of bowel obstruction, often requires intervention. We aimed to identify hospitalization trends and CV outcomes in the United States. Methods: We used the National Inpatient Sample to identify all adult CV hospitalizations in the United States from 2007 to 2017. Patient demographics, comorbidities, and inpatient outcomes were highlighted. Outcomes of endoscopic and surgical management were compared. Results: From 2007 to 2017, there were 220,666 CV hospitalizations. CV-related hospitalizations increased from 17,888 in 2007 to 21,715 in 2017 (p=0.001). However, inpatient mortality decreased from 7.6% in 2007 to 6.2% in 2017 (p<0.001). Of all CV-related hospitalizations, 13,745 underwent endoscopic intervention, and 77,157 underwent surgery. Although the endoscopic cohort had patients with a higher Charlson comorbidity index, we noted lower inpatient mortality (6.1% vs. 7.0%, p<0.001), mean length of stay (8.3 vs. 11.8 days, p<0.001), and mean total healthcare charge ($68,126 vs. $106,703, p<0.001) compared to the surgical cohort. Male sex, increased Charlson comorbidity index scores, acute kidney injury, and malnutrition were associated with higher odds of inpatient mortality in patients with CV who underwent endoscopic management. Conclusion: Endoscopic intervention has lower inpatient mortality and is an excellent alternative to surgery for appropriately selected CV hospitalizations.
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    Factors associated with optimal patient outcomes after operative repair of isolated hip fractures in the elderly
    (BMJ, 2020-12-22) deMeireles, Alirio J.; Gerhardinger, Laura; Oliphant, Bryant W.; Jenkins, Peter C.; Cain-Nielsen, Anne H.; Scott, John W.; Hemmila, Mark R.; Sangji, Naveen F.; Surgery, School of Medicine
    Background: Increased time to operative intervention is associated with a greater risk of mortality and complications in adults with a hip fracture. This study sought to determine factors associated with timeliness of operation in elderly patients presenting with an isolated hip fracture and the influence of surgical delay on outcomes. Methods: Trauma quality collaborative data (July 2016 to June 2019) were analyzed. Inclusion criteria were patients ≥65 years with an injury mechanism of fall, Abbreviated Injury Scale (AIS) 2005 diagnosis of hip fracture, and AIS extremity ≤3. Exclusion criteria included AIS in other body regions >1 and non-operative management. We examined the association of demographic, hospital, injury presentation, and comorbidity factors on a surgical delay >48 hours and patient outcomes using multivariable regression analysis. Results: 10 182 patients fit our study criteria out of 212 620 patients. Mean age was 82.7±8.6 years and 68.7% were female. Delay in operation >48 hours occurred in 965 (9.5%) of patients. Factors that significantly increased mortality or discharge to hospice were increased age, male gender, emergency department hypotension, functionally dependent health status (FDHS), advanced directive, liver disease, angina, and congestive heart failure (CHF). Delay >48 hours was associated with increased mortality or discharge to hospice (OR 1.52; 95% CI 1.13 to 2.06; p<0.01). Trauma center verification level, admission service, and hip fracture volume were not associated with mortality or discharge to hospice. Factors associated with operative delay >48 hours were male gender, FDHS, CHF, chronic renal failure, and advanced directive. Admission to the orthopedic surgery service was associated with less incidence of delay >48 hours (OR 0.43; 95% CI 0.29 to 0.64; p<0.001). Discussion: Hospital verification level, admission service, and patient volume did not impact the outcome of mortality/discharge to hospice. Delay to operation >48 hours was associated with increased mortality. The only measured modifiable characteristic that reduced delay to operative intervention was admission to the orthopedic surgery service. Level of evidence: III.
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    Goal Attainment Scaling: An Idiographic Measure Sensitive to Parent and Teacher Report of IEP Goal Outcome Assessment for Students with ASD
    (Springer, 2022) Ruble, Lisa; McGrew, John; Dale, Brittany; Yee, Madison; Psychology, School of Science
    Young students with ASD have instructional needs in social, communication, and learning skills that should be reflected in their Individualized Education Program (IEP). Research suggests that many of these goal areas present a challenge for special educators because of problems with measurability. The current study utilized an idiographic approach called Goal Attainment Scaling (GAS) for measuring IEP progress of individualized skills. Cross-sectional analysis of the associations between GAS ratings from an independent observer were correlated against teacher and parent ratings of IEP progress at the end of the school year and with standardized measures of the Behavior Assessment System for Children and Vineland Adaptive Behavior Scales (VABS). GAS scores were associated with parent and teacher ratings, including the VABS.
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    Recommendations for outcome measurement for deprescribing intervention studies
    (Wiley, 2022) Bayliss, Elizabeth A.; Albers, Kathleen; Gleason, Kathy; Pieper, Lisa E.; Boyd, Cynthia M.; Campbell, Noll L.; Ensrud, Kristine E.; Gray, Shelly L.; Linsky, Amy M.; Mangin, Derelie; Min, Lillian; Rich, Michael W.; Steinman, Michael A.; Turner, Justin; Vasilevskis, Eduard E.; Dublin, Sascha; Medicine, School of Medicine
    Interpreting results from deprescribing interventions to generate actionable evidence is challenging owing to inconsistent and heterogeneous outcome definitions between studies. We sought to characterize deprescribing intervention outcomes and recommend approaches to measure outcomes for future studies. A scoping literature review focused on deprescribing interventions for polypharmacy and informed a series of expert panel discussions and recommendations. Twelve experts in deprescribing research, policy, and clinical practice interventions participating in the Measures Workgroup of the US Deprescribing Research Network sought to characterize deprescribing outcomes and recommend approaches to measure outcomes for future studies. The scoping review identified 125 papers reflecting 107 deprescribing studies. Common outcomes included medication discontinuation, medication appropriateness, and a broad range of clinical outcomes potentially resulting from medication reduction. Panel recommendations included clearly defining clinically meaningful medication outcomes (e.g., number of chronic medications, dose reductions), ensuring adequate sample size and follow-up time to capture clinical outcomes resulting from medication discontinuation (e.g., quality of life [QOL]), and selecting appropriate and feasible data sources. A new conceptual model illustrates how downstream clinical outcomes (e.g., reduction in falls) should be interpreted in the context of initial changes in medication measures (e.g., reduction in mean total medications). Areas needing further development include implementation outcomes specific to deprescribing interventions and measures of adverse drug withdrawal events. Generating evidence to guide deprescribing is essential to address patient, caregiver, and clinician concerns about the benefits and harms of medication discontinuation. This article provides recommendations and an initial conceptual framework for selecting and applying appropriate intervention outcomes to support deprescribing research.
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    Review of menopausal palpitations measures
    (BMC, 2021-05-31) Sheng, Ying; Carpenter, Janet S.; Elomba, Charles D.; Alwine, Jennifer S.; Yue, Min; Pike, Caitlin A.; Chen, Chen X.; Tisdale, James E.; School of Nursing
    Palpitations are reported commonly by women around the time of menopause as skipped, missed, irregular, and/or exaggerated heartbeats or heart pounding. However, much less is known about palpitations than other menopausal symptoms such as vasomotor symptoms. The objective of this review was to integrate evidence on menopausal palpitations measures. Keyword searching was done in PubMed, CINAHL, and PsycINFO for English-language, descriptive articles containing data on menopause and palpitations and meeting other pre-specified inclusion criteria. Of 670 articles, 110 met inclusion criteria and were included in the review. Results showed that 11 different measures were used across articles, with variability within and between measures. Inconsistencies in the wording of measurement items, recall periods, and response options were observed even when standardized measures were used. Most measures were limited to assessing symptom presence and severity. Findings suggest that efforts should be undertaken to (1) standardize conceptual and operational definitions of menopausal palpitations and (2) develop a patient-friendly, conceptually clear, psychometrically sound measure of menopausal palpitations.
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    Violent injury prevention does not equal to violent crime prevention: an analysis of violence intervention program efficacy using propensity score methods
    (BMJ, 2022-10-17) Holler, Emma; Ortiz, Damaris; Mohanty, Sanjay; Meagher, Ashley D.; Boustani, Malaz; Zarzaur, Ben L.; Simons, Clark J.; Surgery, School of Medicine
    Objective: The purpose of this study was to determine if exposure to Prescription for Hope (RxH), a hospital-based violence intervention program (HVIP), is associated with reduced violent reinjury and new convictions for violent crime in the 2-year period after index hospitalization for a violent injury. Methods: This was a retrospective cohort study analyzing patients from two level I trauma centers in Indianapolis, Indiana. RxH participants (n=260) enrolled between January 1, 2015 and December 31, 2018 and who had trauma registry data were included. RxH eligibility criteria: admitted for a violent injury (excluding sexual violence), at least 15 years of age, live in Marion County, Indiana, and stay in the hospital for at least 24 hours. RxH exclusion criteria: heavy active substance use, acute psychosis, dementia, severe traumatic brain injury, intentional self-harm, and incarceration. All patients admitted to IU Health Methodist Hospital, a nearby level I trauma center, for an assault, stabbing, or gunshot wound during the same period and met the RxH eligibility criteria were included as a comparison group (n=732). Doubly adjusted logistic regression with inverse probability of treatment weighting was used to estimate the average treatment effect of RxH participation on violent reinjury and new convictions for violent crime. Results: Data from 992 patients were analyzed. RxH was significantly associated with reduced odds of violent reinjury (OR=0.35, 95% CI 0.20 to 0.59) and increased odds of conviction for a violent crime (OR=2.43, 95% CI 1.64 to 3.61). Conclusion: RxH was associated with decreased odds of violent reinjury but increased odds of new conviction for a violent crime. Our results highlight the importance of robust, routine evaluation of HVIP efficacy and recommend inclusion of other outcomes in addition to violent reinjury when evaluating program success. High-quality randomized controlled trials are needed to further investigate the impact of HVIPs on a variety of outcomes.