Dataset Information

Prioritized concordance index for hierarchical survival outcomes.

ABSTRACT: We propose an extension of Harrell's concordance (C) index to evaluate the prognostic utility of biomarkers for diseases with multiple measurable outcomes that can be prioritized. Our prioritized concordance index measures the probability that, given a random subject pair, the subject with the worst disease status as of a time ? has the higher predicted risk. Our prioritized concordance index uses the same approach as the win ratio, by basing generalized pairwise comparisons on the most severe or clinically important comparable outcome. We use an inverse probability weighting technique to correct for study-specific censoring. Asymptotic properties are derived using U-statistic properties. We apply the prioritized concordance index to two types of disease processes with a rare primary outcome and a more common secondary outcome. Our simulation studies show that when a predictor is predictive of both outcomes, the new concordance index can gain efficiency and power in identifying true prognostic variables compared to using the primary outcome alone. Using the prioritized concordance index, we examine whether novel clinical measures can be useful in predicting risk of type II diabetes in patients with impaired glucose resistance whose disease status can also regress to normal glucose resistance. We also examine the discrimination ability of four published risk models among ever smokers at risk of lung cancer incidence and subsequent death.

SUBMITTER: Cheung LC

PROVIDER: S-EPMC6800570 | biostudies-literature | 2019 Jul

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

Prioritized concordance index for hierarchical survival outcomes.

Cheung Li C LC Pan Qing Q Hyun Noorie N Katki Hormuzd A HA

Statistics in medicine 20190407 15

We propose an extension of Harrell's concordance (C) index to evaluate the prognostic utility of biomarkers for diseases with multiple measurable outcomes that can be prioritized. Our prioritized concordance index measures the probability that, given a random subject pair, the subject with the worst disease status as of a time τ has the higher predicted risk. Our prioritized concordance index uses the same approach as the win ratio, by basing generalized pairwise comparisons on the most severe o ...[more]

PMID: 30957257

Similar Datasets

Project description:Importance:Dry eye is a common ocular surface condition with significant influence on patient quality of life and societal economic burden. There is an urgent need to prioritize new research for dry eye. Objective:To identify and rank research questions and outcomes important to patients with dry eye. Design, Setting, and Participants:This study was conducted using the following 6 steps: (1) identifying research questions from a previous survey of clinicians who treat patients with dry eye; (2) identifying outcomes from existing research (systematic reviews and their cited clinical trials in the Cochrane Eyes and Vision US Satellite database of eyes and vision reviews, and National Eye Institute-funded clinical trials registered on ClinicalTrials.gov) as of June 13, 2017; (3) identifying a sample of patients with dry eye from the email subscribers to the online newsletter KeratoScoop; (4) and (5) conducting a 2-round Delphi survey of those patients online in November and December 2017, respectively; and (6) designating and ranking questions and outcomes as important. Main Outcomes and Measures:Importance assigned to research questions and outcomes for dry eye. A research question or outcome ranked by at least 75% of patients as 6 or higher on a scale of 0 to 10 was considered important. Results:Among the 420 patients from 15 countries who completed both rounds of the Delphi survey, most were 60 years of age or older (233 [56%]), female (348 [83%]), white (393 [94%]), and of non-Hispanic ethnicity (398 [95%]). Among the 12 questions that clinicians had previously prioritized, patients rated 8 as important. The top 3 questions pertained to effectiveness of patient education, environmental modifications, and topical anti-inflammatory eye drops for dry eye. Among the 109 outcomes identified in existing research on dry eye, patients rated 26 as important. Ten of these 26 were unpopular in existing research, with fewer than 10% of 158 studies reporting these outcomes. Of the 10 most important outcomes, 9 were associated with symptoms or quality of life. The 3 outcomes rated most important by patients were ocular burning or stinging, ocular discomfort, and ocular pain. Conclusions and Relevance:This study identified research questions and outcomes important to patients with dry eye. A considerable gap was noted between outcomes in existing research on dry eye and outcomes patients consider important. Future research on dry eye should consider addressing the important research questions and outcomes identified herein, taking into account the patient perspective.

Project description:ObjectivesThere are over 4,000 trials conducted in people with coronavirus disease 2019. However, the variability of outcomes and the omission of patient-centered outcomes may diminish the impact of these trials on decision-making. The aim of this study was to generate a consensus-based, prioritized list of outcomes for coronavirus disease 2019 trials.DesignIn an online survey conducted in English, Chinese, Italian, Portuguese, and Spanish languages, adults with coronavirus disease 2019, their family members, health professionals, and the general public rated the importance of outcomes using a 9-point Likert scale (7-9, critical importance) and completed a Best-Worst Scale to estimate relative importance. Participant comments were analyzed thematically.SettingInternational.SubjectsAdults 18 years old and over with confirmed or suspected coronavirus disease 2019, their family members, members of the general public, and health professionals (including clinicians, policy makers, regulators, funders, and researchers).InterventionsNone.MeasurementsNone.Main resultsIn total, 9,289 participants from 111 countries (776 people with coronavirus disease 2019 or family members, 4,882 health professionals, and 3,631 members of the public) completed the survey. The four outcomes of highest priority for all three groups were: mortality, respiratory failure, pneumonia, and organ failure. Lung function, lung scarring, sepsis, shortness of breath, and oxygen level in the blood were common to the top 10 outcomes across all three groups (mean > 7.5, median ≥ 8, and > 70% of respondents rated the outcome as critically important). Patients/family members rated fatigue, anxiety, chest pain, muscle pain, gastrointestinal problems, and cardiovascular disease higher than health professionals. Four themes underpinned prioritization: fear of life-threatening, debilitating, and permanent consequences; addressing knowledge gaps; enabling preparedness and planning; and tolerable or infrequent outcomes.ConclusionsLife-threatening respiratory and other organ outcomes were consistently highly prioritized by all stakeholder groups. Patients/family members gave higher priority to many patient-reported outcomes compared with health professionals.

Project description:IntroductionColorectal cancer shows high incidence and mortality rates. Immune checkpoint blockade can be used to treat colorectal carcinoma (CRC); however, it shows limited effectiveness in most patients.MethodsTo identify patients who may benefit from immunotherapy using immune checkpoint inhibitors, we constructed an immune-related gene prognostic index (IRGPI) for predicting the efficacy of immunotherapy in patients with CRC. Transcriptome datasets and clinical information of patients with CRC were used to identify differential immune-related genes between tumor and para-carcinoma tissue. Using weighted correlation network analysis and Cox regression analysis, the IRGPI was constructed, and Kaplan-Meier analysis was used to evaluate its predictive ability. We also analyzed the molecular and immune characteristics between IRGPI high-and low-risk subgroups, performed sensitivity analysis of ICI treatment, and constructed overall survival-related receiver operating characteristic curves to validate the IRGPI. Finally, IRGPI genes and tumor immune cell infiltration in CRC model mice with orthotopic metastases were analyzed to verify the results.ResultsThe IRGPI was constructed based on the following 11 hub genes: ADIPOQ, CD36, CCL24, INHBE, UCN, IL1RL2, TRIM58, RBCK1, MC1R, PPARGC1A, and LGALS2. Patients with CRC in the high-risk subgroup showed longer overall survival than those in the low-risk subgroup, which was confirmed by GEO database. Clinicopathological features associated with cancer progression significantly differed between the high- and low-risk subgroups. Furthermore, Kaplan-Meier analysis of immune infiltration showed that the increased infiltration of naïve B cells, macrophages M1, and regulatory T cells and reduced infiltration of resting dendritic cells and mast cells led to a worse overall survival in patients with CRC. The ORC curves revealed that IRGPI predicted patient survival more sensitive than the published tumor immune dysfunction and rejection and tumor inflammatory signature.DiscussionThus, the low-risk subgroup is more likely to benefit from ICIs than the high-risk subgroup. CRC model mice showed higher proportions of Tregs, M1 macrophages, M2 macrophages and lower proportions of B cells, memory B cell immune cell infiltration, which is consistent with the IRGPI results. The IRGPI can predict the prognosis of patients with CRC, reflect the CRC immune microenvironment, and distinguish patients who are likely to benefit from ICI therapy.

Dataset Information

Prioritized concordance index for hierarchical survival outcomes.

Publications

Prioritized concordance index for hierarchical survival outcomes.

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets