Browsing by Subject "Outcomes research"
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Item Advocating for the inclusion of kidney health outcomes in neonatal research: best practice recommendations by the Neonatal Kidney Collaborative(Springer Nature, 2024) Reidy, Kimberly J.; Guillet, Ronnie; Selewski, David T.; Defreitas, Marissa; Stone, Sadie; Starr, Michelle C.; Harer, Matthew W.; Todurkar, Namrata; Vuong, Kim T.; Gogcu, Semsa; Askenazi, David; Tipple, Trent E.; Charlton, Jennifer R.; Pediatrics, School of MedicineAcute kidney injury (AKI) occurs in nearly 30% of sick neonates. Chronic kidney disease (CKD) can be detected in certain populations of sick neonates as early as 2 years. AKI is often part of a multisystem syndrome that negatively impacts developing organs resulting in short- and long-term pulmonary, neurodevelopmental, and cardiovascular morbidities. It is critical to incorporate kidney-related data into neonatal clinical trials in a uniform manner to better understand how neonatal AKI or CKD could affect an outcome of interest. Here, we provide expert opinion recommendations and rationales to support the inclusion of short- and long-term neonatal kidney outcomes using a tiered approach based on study design: (1) observational studies (prospective or retrospective) limited to data available within a center's standard practice, (2) observational studies involving prospective data collection where prespecified kidney outcomes are included in the design, (3) interventional studies with non-nephrotoxic agents, and (4) interventional studies with known nephrotoxic agents. We also provide recommendations for biospecimen collection to facilitate ancillary kidney specific research initiatives. This approach balances the costs of AKI and CKD ascertainment with knowledge gained. We advocate that kidney outcomes be included routinely in neonatal clinical study design. Consistent incorporation of kidney outcomes across studies will increase our knowledge of neonatal morbidity.Item Artificial intelligence reveals features associated with breast cancer neoadjuvant chemotherapy responses from multi-stain histopathologic images(Springer Nature, 2023-01-27) Huang, Zhi; Shao, Wei; Han, Zhi; Alkashash, Ahmad Mahmoud; De la Sancha, Carlo; Parwani, Anil V.; Nitta, Hiroaki; Hou, Yanjun; Wang, Tongxin; Salama, Paul; Rizkalla, Maher; Zhang, Jie; Huang, Kun; Li, Zaibo; Electrical and Computer Engineering, School of Engineering and TechnologyAdvances in computational algorithms and tools have made the prediction of cancer patient outcomes using computational pathology feasible. However, predicting clinical outcomes from pre-treatment histopathologic images remains a challenging task, limited by the poor understanding of tumor immune micro-environments. In this study, an automatic, accurate, comprehensive, interpretable, and reproducible whole slide image (WSI) feature extraction pipeline known as, IMage-based Pathological REgistration and Segmentation Statistics (IMPRESS), is described. We used both H&E and multiplex IHC (PD-L1, CD8+, and CD163+) images, investigated whether artificial intelligence (AI)-based algorithms using automatic feature extraction methods can predict neoadjuvant chemotherapy (NAC) outcomes in HER2-positive (HER2+) and triple-negative breast cancer (TNBC) patients. Features are derived from tumor immune micro-environment and clinical data and used to train machine learning models to accurately predict the response to NAC in breast cancer patients (HER2+ AUC = 0.8975; TNBC AUC = 0.7674). The results demonstrate that this method outperforms the results trained from features that were manually generated by pathologists. The developed image features and algorithms were further externally validated by independent cohorts, yielding encouraging results, especially for the HER2+ subtype.Item Bone health effects of androgen-deprivation therapy and androgen receptor inhibitors in patients with nonmetastatic castration-resistant prostate cancer(Springer Nature, 2021) Hussain, Arif; Tripathi, Abhishek; Pieczonka, Christopher; Cope, Diane; McNatty, Andrea; Logothetis, Christopher; Guise, Theresa; Medicine, School of MedicineBackground: Osteoporosis is a skeletal disorder characterized by compromised bone strength, resulting in increased fracture risk. Patients with prostate cancer may have multiple risk factors contributing to bone fragility: advanced age, hypogonadism, and long-term use of androgen-deprivation therapy. Despite absence of metastatic disease, patients with nonmetastatic castrate-resistant prostate cancer receiving newer androgen receptor inhibitors can experience decreased bone mineral density. A systematic approach to bone health care has been hampered by a simplistic view that does not account for heterogeneity among prostate cancer patients or treatments they receive. This review aims to raise awareness in oncology and urology communities regarding the complexity of bone health, and to provide a framework for management strategies for patients with nonmetastatic castrate-resistant prostate cancer receiving androgen receptor inhibitor treatment. Methods: We searched peer-reviewed literature on the PubMed database using key words "androgen-deprivation therapy," "androgen receptor inhibitors," "bone," "bone complications," and "nonmetastatic prostate cancer" from 2000 to present. Results: We discuss how androgen inhibition affects bone health in patients with nonmetastatic castrate-resistant prostate cancer. We present data from phase 3 trials on the three approved androgen receptor inhibitors with regard to effects on bone. Finally, we present management strategies for maintenance of bone health. Conclusions: In patients with nonmetastatic castrate-resistant prostate cancer, aging, and antiandrogen therapy contribute to bone fragility. Newer androgen receptor inhibitors were associated with falls or fractures in a small subset of patients. Management guidelines include regular assessment of bone density, nutritional guidance, and use of antiresorptive bone health agents when warranted.Item Cinacalcet, dialysate calcium concentration, and cardiovascular events in the EVOLVE trial(Wiley, 2016-07) Pun, Patrick H.; Abdalla, Safa; Block, Geoffrey A.; Chertow, Glenn M.; Correa-Rotter, Ricardo; Dehmel, Bastian; Drüeke, Tilman B.; Floege, Jürgen; Goodman, William G.; Herzog, Charles A.; London, Gerard M.; Mahaffey, Kenneth W.; Moe, Sharon M.; Parfrey, Patrick S.; Wheeler, David C.; Middleton, John P.; Medicine, School of MedicineAmong patients receiving hemodialysis, abnormalities in calcium regulation have been linked to an increased risk of cardiovascular events. Cinacalcet lowers serum calcium concentrations through its effect on parathyroid hormone secretion and has been hypothesized to reduce the risk of cardiovascular events. In observational cohort studies, prescriptions of low dialysate calcium concentration and larger observed serum-dialysate calcium gradients have been associated with higher risks of in-dialysis facility or peri-dialytic sudden cardiac arrest. We performed this study to examine the risks associated with dialysate calcium and serum-dialysate gradients among participants in the Evaluation of Cinacalcet Hydrochloride Therapy to Lower Cardiovascular Events (EVOLVE) trial. In EVOLVE, 3883 hemodialysis patients were randomized 1:1 to cinacalcet or placebo. Dialysate calcium was administered at the discretion of treating physicians. We examined whether baseline dialysate calcium concentration or the serum-dialysate calcium gradient modified the effect of cinacalcet on the following adjudicated endpoints: (1) primary composite endpoint (death or first non-fatal myocardial infarction, hospitalization for unstable angina, heart failure, or peripheral vascular event); (2) cardiovascular death; and (3) sudden death. In EVOLVE, use of higher dialysate calcium concentrations was more prevalent in Europe and Latin America compared with North America. There was a significant fall in serum calcium concentration in the cinacalcet group; dialysate calcium concentrations were changed infrequently in both groups. There was no association between baseline dialysate calcium concentration or serum-dialysate calcium gradient and the endpoints examined. Neither the baseline dialysate calcium nor the serum-dialysate calcium gradient significantly modified the effects of cinacalcet on the outcomes examined. The effects of cinacalcet on cardiovascular death and major cardiovascular events are not altered by the dialysate calcium prescription and serum-dialysate calcium gradient.Item Delirium severity does not differ between medical and surgical intensive care units after adjusting for medication use(Springer Nature, 2022-08-24) Ortiz, Damaris; Lindroth, Heidi L.; Braly, Tyler; Perkins, Anthony J.; Mohanty, Sanjay; Meagher, Ashley D.; Khan, Sikandar H.; Boustani, Malaz A.; Khan, Babar A.; Surgery, School of MedicineSevere delirium is associated with an increased risk of mortality, institutionalization, and length of stay. Few studies have examined differences in delirium severity between different populations of critically ill patients. The objective of the study was to compare delirium severity and the presence of the four core features between adults in the surgical intensive care unit (SICU) and medical intensive care unit (MICU) while controlling for variables known to be associated with delirium. This is a secondary analysis of two parallel randomized multi-center trials conducted from March 2009 to January 2015 at 3 Indianapolis hospitals. A total of 474 adults with delirium were included in the analysis. Subjects were randomized in a 1:1 ratio in random blocks of 4 by a computer program. Patients were randomized to either haloperidol prescribing or de-prescribing regimen vs usual care. Delirium severity was assessed daily or twice-daily using the CAM-ICU-7 beginning after 24 h of ICU admission and until discharge from the hospital, death, or 30 days after enrollment. Secondary outcomes included hospital length of stay, hospital and 30-day mortality, and delirium-related adverse events. These outcomes were compared between SICU and MICU settings for this secondary analysis. Out of 474 patients, 237 were randomized to intervention. At study enrollment, the overall cohort had a mean age of 59 (SD 16) years old, was 54% female, 44% African-American, and 81% were mechanically ventilated upon enrollment. MICU participants were significantly older and severely ill with a higher premorbid cognitive and physical dysfunction burden. In univariate analysis, SICU participants had significantly higher mean total CAM-ICU-7 scores, corresponding to delirium severity, (4.15 (2.20) vs 3.60 (2.32), p = 0.02), and a lower mean RASS score (- 1.79 (1.28) vs - 1.53 (1.27), p < 0.001) compared to MICU participants. Following adjustment for benzodiazepines and opioids, delirium severity did not significantly differ between groups. The presence of Feature 3, altered level of consciousness, was significantly associated with the SICU participants, identifying as Black, premorbid functional impairment, benzodiazepines, opioids, and dexmedetomidine. In this secondary analysis examining differences in delirium severity between MICU and SICU participants, we did not identify a difference between participant populations following adjustment for administered benzodiazepines and opioids. We did identify that an altered level of consciousness, core feature 3 of delirium, was associated with SICU setting, identifying as Black, activities of daily living, benzodiazepines and opioid medications. These results suggest that sedation practice patterns play a bigger role in delirium severity than the underlying physiologic insult, and expression of core features of delirium may vary based on individual factors.Item Existing Data Analysis in Pediatric Critical Care Research(Frontiers Media, 2014-07-29) Bennett, Tellen D.; Spaeder, Michael C.; Matos, Renée I.; Watson, R. Scott; Typpo, Katri V.; Khemani, Robinder G.; Crow, Sheri; Benneyworth, Brian D.; Thiagarajan, Ravi R.; Dean, J. Michael; Markovitz, Barry P.; Pediatric Acute Lung Injury and Sepsis Investigators (PALISI); Pediatrics, School of MedicineOur objectives were to review and categorize the existing data sources that are important to pediatric critical care medicine (PCCM) investigators and the types of questions that have been or could be studied with each data source. We conducted a narrative review of the medical literature, categorized the data sources available to PCCM investigators, and created an online data source registry. We found that many data sources are available for research in PCCM. To date, PCCM investigators have most often relied on pediatric critical care registries and treatment- or disease-specific registries. The available data sources vary widely in the level of clinical detail and the types of questions they can reliably answer. Linkage of data sources can expand the types of questions that a data source can be used to study. Careful matching of the scientific question to the best available data source or linked data sources is necessary. In addition, rigorous application of the best available analysis techniques and reporting consistent with observational research standards will maximize the quality of research using existing data in PCCM.Item Functional outcome following inpatient rehabilitation among individuals with complete spinal cord injury in Nepal(Springer Nature, 2021-10-07) Khatri, Prakriti; Jalayondeja, Chutima; Dhakal, Raju; Groves, Christine C.; Physical Medicine and Rehabilitation, School of MedicineObjectives: To describe functional outcomes using Spinal Cord Independence Measure III (SCIM III) following inpatient rehabilitation among individuals with complete spinal cord injury (SCI) in the low-income setting of Nepal; to evaluate functional changes from rehabilitation admission to discharge and to compare functional outcomes between neurological levels of injury (NLI) at discharge. Setting: Spinal Injury Rehabilitation Centre (SIRC), Kavrepalanchowk, Nepal. Methods: We present data of all individuals with complete SCI who completed rehabilitation at SIRC in 2017. Data collected included: demographics, aetiology, neurological assessment, admission/discharge SCIM III scores, and length of stay. Data were analyzed using descriptive statistics. Pre/post-SCIM III scores were analyzed using Related-Samples Wilcoxon signed-rank test. Comparative analysis between NLIs was done using the Kruskal Wallis ANOVA test followed by pairwise Mann-Whitney U tests. Results: Ninety-six individuals were included. Mean (SD) age was 33.5 (14.2) years, with a male/female ratio of 3.4:1. Median admission and discharge total SCIM III scores for cervical, thoracic and lumbosacral levels were 10 and 21, 16 and 61, and 41 and 79.5, respectively. Median total SCIM III score change between admission and discharge were 11 (p = 0.003), 43 (p < 0.001) and 40 (p = 0.068) for cervical, thoracic and lumbar groups, respectively. Conclusions: This study is the first of its kind to describe functional outcomes among individuals with complete SCI in the low-income setting of Nepal. All SCI groups showed a positive trend in SCIM III from admission to discharge, with improvements reaching statistical significance among groups with cervical and thoracic NLIs.Item Medical and surgical interventions and outcomes for infants with trisomy 18 (T18) or trisomy 13 (T13) at children's hospitals neonatal intensive care units (NICUs)(Springer Nature, 2021) Acharya, Krishna; Leuthner, Steven R.; Zaniletti, Isabella; Niehaus, Jason Z.; Bishop, Christine E.; Coghill, Carl H.; Datta, Ankur; Dereddy, Narendra; DiGeronimo, Robert; Jackson, Laura; Ling, Con Yee; Matoba, Nana; Natarajan, Girija; Pritha Nayak, Sujir; Brown Schlegel, Amy; Seale, Jamie; Shah, Anita; Weiner, Julie; Williams, Helen O.; Wojcik, Monica H.; Fry, Jessica T.; Sullivan, Kevin; Palliative Care and Ethics Focus Group of the Children’s Hospital Neonatal Consortium (CHNC); Pediatrics, School of MedicineObjectives: To examine characteristics and outcomes of T18 and T13 infants receiving intensive surgical and medical treatment compared to those receiving non-intensive treatment in NICUs. Study design: Retrospective cohort of infants in the Children's Hospitals National Consortium (CHNC) from 2010 to 2016 categorized into three groups by treatment received: surgical, intensive medical, or non-intensive. Results: Among 467 infants admitted, 62% received intensive medical treatment; 27% received surgical treatment. The most common surgery was a gastrostomy tube. Survival in infants who received surgeries was 51%; intensive medical treatment was 30%, and non-intensive treatment was 72%. Infants receiving surgeries spent more time in the NICU and were more likely to receive oxygen and feeding support at discharge. Conclusions: Infants with T13 or T18 at CHNC NICUs represent a select group for whom parents may have desired more intensive treatment. Survival to NICU discharge was possible, and surviving infants had a longer hospital stay and needed more discharge supports.Item Monotherapy Anticoagulation to Expedite Home Treatment of Patients Diagnosed With Venous Thromboembolism in the Emergency Department: A Pragmatic Effectiveness Trial(American Heart Association, 2021) Kline, Jeffrey A.; Adler, David H.; Alanis, Naomi; Bledsoe, Joseph R.; Courtney, Daniel M.; d’Etienne, James P.; Diercks, Deborah B.; Garrett, John S.; Jones, Alan E.; Mackenzie, David C.; Madsen, Troy; Matuskowitz, Andrew J.; Mumma, Bryn E.; Nordenholz, Kristen E.; Pagenhardt, Justine; Runyon, Michael S.; Stubblefield, William B.; Willoughby, Christopher B.; Emergency Medicine, School of MedicineBackground: The objective was to test if low-risk emergency department patients with vitamin K antagonist (venous thromboembolism [VTE]; including venous thrombosis and pulmonary embolism [PE]) can be safely and effectively treated at home with direct acting oral (monotherapy) anticoagulation in a large-scale, real-world pragmatic effectiveness trial. Methods: This was a single-arm trial, conducted from 2016 to 2019 in accordance with the Standards for Reporting Implementation Studies guideline in 33 emergency departments in the United States. Participants had newly diagnosed VTE with low risk of death based upon either the modified Hestia criteria, or physician judgment plus the simplified PE severity index score of zero, together with nonhigh bleeding risk were eligible. Patients had to be discharged within 24 hours of triage and treated with either apixaban or rivaroxaban. Effectiveness was defined by the primary efficacy and safety outcomes, image-proven recurrent VTE and bleeding requiring hospitalization >24 hours, respectively, with an upper limit of the 95% CI for the 30-day frequency of VTE recurrence below 2.0% for both outcomes. Results: We enrolled 1421 patients with complete outcomes data, including 903 with venous thrombosis and 518 with PE. The recurrent VTE requiring hospitalization occurred in 14/1421 (1.0% [95% CI, 0.5%-1.7%]), and bleeding requiring hospitalization occurred in 12/1421 (0.8% [0.4%-1.5%). The rate of severe bleeding using International Society for Thrombosis and Haemostasis criteria was 2/1421 (0.1% [0%-0.5%]). No patient died, and serious adverse events occurred in 2.5% of venous thrombosis patients and 2.3% of patients with PE. Medication nonadherence was reported by patients in 8.0% (6.6%-9.5%) and was associated with a risk ratio of 6.0 (2.3-15.2) for VTE recurrence. Among all patients diagnosed with VTE in the emergency department during the period of study, 18% of venous thrombosis patients and 10% of patients with PE were enrolled. Conclusions: Monotherapy treatment of low-risk patients with venous thrombosis or PE in the emergency department setting produced a low rate of bleeding and VTE recurrence, but may be underused. Patients with venous thrombosis and PE should undergo risk-stratification before home treatment. Improved patient adherence may reduce rate of recurrent VTE.Item Participant-reported personal utility of genetic testing for Parkinson's disease and interest in clinical trial participation(Springer Nature, 2024-10-25) Oas, Hannah; Cook, Lola; Schwantes-An, Tae-Hwi; Walsh, Laurence E.; Wills, Anne-Marie; Mata, Ignacio F.; Nance, Martha A.; Beck, James C.; Naito, Anna; Marder, Karen; Alcalay, Roy N.; Verbrugge, Jennifer; Medical and Molecular Genetics, School of MedicineGenetic testing for Parkinson's disease (PD) is infrequently performed due to perceptions of low utility. We investigated the personal utility in PD GENEration and how results lead to enrollment in additional research studies. Participants (n = 972) underwent genetic testing, results disclosure, genetic counseling, and completed a survey examining the perceived personal utility of their results and interest in participating in additional studies. Most participants found their genetic test results useful, including satisfying curiosity (81%), feeling good about helping the medical community (80%), and having information to share with family (77%). There were no significant differences in responses based on result type. Forty-five percent of participants expressed interest in participating in research studies; whereas 16% of participants confirmed enrollment. Our results suggest that participants find personal utility in genetic testing regardless of results. Although participants may be interested in enrolling in additional research, they may need support and resources.