|
1
|
Ferrara L, Ardito V, Tozzi VD, Tarricone R. Economic Evaluations of Health Service Interventions Targeting Patients with Multimorbidities: A Scoping Literature Review. Int J Integr Care 2025; 25:3. [PMID: 39866291 PMCID: PMC11760752 DOI: 10.5334/ijic.8623] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Grants] [Track Full Text] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 03/06/2024] [Accepted: 01/14/2025] [Indexed: 01/28/2025] Open
Abstract
Introduction Multimorbid patients have been growing, leading to an exponential increase in healthcare costs and patterns of resource utilization. Despite the heightened interest toward integrated care programs as a response to the complex need of multimorbid patients, economic evaluations of these programs remain scarce. This work investigated the economic evaluations of service interventions targeting multimorbid patients, to identify the characteristics of these programs and the methods applied to their evaluation. Methods We conducted a scoping review of papers published between 2010 and 2021 on PubMed, Science Direct, EconLit and Web Of Science. The search strategy was built around three keyword blocks: service interventions, multimorbidity, economic evaluations. We selected economic evaluations of service interventions delivered through multiple care settings and targeting patients with 2+ chronic conditions. Results Twenty-five articles were included. Interventions were categorized as organizational-type versus patient-oriented. The selected studies often targeted patients with one chronic disease, associated with a mental disorder, like depression or anxiety. Included studies were mostly cost-utility analyses conducted with the healthcare perspective. Discussions and conclusions This work confirmed that economic evaluations of service interventions for multimorbid patients are limited in number. This could suggest that decision-making regarding the delivery of healthcare services for multimorbid patients may not always be based on a solid evidence base. More economic analyses are needed to inform evidence-based coverage decision-making.
Collapse
Affiliation(s)
- Lucia Ferrara
- Center for Research on Health and Social Care Management (CERGAS), SDA Bocconi School of Management, Milan, Italy
| | - Vittoria Ardito
- Center for Research on Health and Social Care Management (CERGAS), SDA Bocconi School of Management, Milan, Italy
| | - Valeria D. Tozzi
- Center for Research on Health and Social Care Management (CERGAS), SDA Bocconi School of Management, Milan, Italy
| | - Rosanna Tarricone
- Center for Research on Health and Social Care Management (CERGAS), SDA Bocconi School of Management, Milan, Italy
- Department of Social and Political Sciences, Bocconi University, Milan, Italy
| |
Collapse
|
|
2
|
Polok K, Fronczek J, Putowski Z, Czok M, Guidet B, Jung C, de Lange D, Leaver S, Moreno R, Flatten H, Szczeklik W. Validity of the total SOFA score in patients ≥ 80 years old acutely admitted to intensive care units: a post-hoc analysis of the VIP2 prospective, international cohort study. Ann Intensive Care 2023; 13:98. [PMID: 37798561 PMCID: PMC10555975 DOI: 10.1186/s13613-023-01191-0] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 07/15/2023] [Accepted: 09/17/2023] [Indexed: 10/07/2023] Open
Abstract
BACKGROUND Little is known about the performance of the Sequential Organ Failure Assessment (SOFA) score in older critically ill adults. We aimed to evaluate the prognostic impact of physiological disturbances in the six organ systems included in the SOFA score. METHODS We analysed previously collected data from a prospective cohort study conducted between 2018 and 2019 in 22 countries. Consecutive patients ≥ 80 years old acutely admitted to intensive care units (ICUs) were eligible for inclusion. Patients were followed up for 30 days after admission to the ICU. We used logistic regression to study the association between increasing severity of organ dysfunction and mortality. RESULTS The median SOFA score among 3882 analysed patients was equal to 6 (IQR: 4-9). Mortality was equal to 26.1% (95% CI 24.7-27.5%) in the ICU and 38.7% (95% CI 37.1-40.2%) at day 30. Organ failure defined as a SOFA score ≥ 3 was associated with variable adjusted odds ratios (aORs) for ICU mortality dependant on the organ system affected: respiratory, 1.53 (95% CI 1.29-1.81); cardiovascular 1.69 (95% CI 1.43-2.01); hepatic, 1.74 (95% CI 0.97-3.15); renal, 1.87 (95% CI 1.48-2.35); central nervous system, 2.79 (95% CI 2.34-3.33); coagulation, 2.72 (95% CI 1.66-4.48). Modelling consecutive levels of organ dysfunction resulted in aORs equal to 0.57 (95% CI 0.33-1.00) when patients scored 2 points in the cardiovascular system and 1.01 (0.79-1.30) when the cardiovascular SOFA equalled 3. CONCLUSIONS Different components of the SOFA score have different prognostic implications for older critically ill adults. The cardiovascular component of the SOFA score requires revision.
Collapse
Affiliation(s)
- Kamil Polok
- Center for Intensive Care and Perioperative Medicine, Jagiellonian University Medical College, ul. Wrocławska 1-3, 30 - 901, Kraków, Poland
- Department of Pulmonology, Jagiellonian University Medical College, Kraków, Poland
| | - Jakub Fronczek
- Center for Intensive Care and Perioperative Medicine, Jagiellonian University Medical College, ul. Wrocławska 1-3, 30 - 901, Kraków, Poland
| | - Zbigniew Putowski
- Center for Intensive Care and Perioperative Medicine, Jagiellonian University Medical College, ul. Wrocławska 1-3, 30 - 901, Kraków, Poland
| | - Marcelina Czok
- Center for Intensive Care and Perioperative Medicine, Jagiellonian University Medical College, ul. Wrocławska 1-3, 30 - 901, Kraków, Poland
| | - Bertrand Guidet
- Sorbonne Universités, UPMC Univ Paris 06, INSERM, UMR_S 1136, Institut Pierre Louis d'Epidémiologie Et de Santé Publique, Equipe: Epidémiologie Hospitalière Qualité Et Organisation Des Soins, 75012, Paris, France
- Assistance Publique-Hôpitaux de Paris, Paris, France
| | - Christian Jung
- Department of Cardiology, Pulmonology and Vascular Medicine, Medical Faculty, Heinrich-Heine-University Duesseldorf, Moorenstraße 5, 40225, Duesseldorf, Germany
| | - Dylan de Lange
- Department of Intensive Care Medicine, University Medical Center, University Utrecht, Utrecht, The Netherlands
| | - Susannah Leaver
- Department of Critical Care, St George's Hospital, London, UK
| | - Rui Moreno
- Hospital de São José, Centro Hospitalar Universitário de Lisboa Central, Faculdade de Ciências Médicas de Lisboa (Nova Médical School), Lisbon, Portugal
- Faculdade de Ciências da Saúde, Universidade da Beira Interior, Covilhã, Portugal
| | - Hans Flatten
- Department of Anaesthesia and Intensive Care, Haukeland University Hospital, Bergen, Norway
- Department of Clinical Medicine, University of Bergen, Bergen, Norway
| | - Wojciech Szczeklik
- Center for Intensive Care and Perioperative Medicine, Jagiellonian University Medical College, ul. Wrocławska 1-3, 30 - 901, Kraków, Poland.
| |
Collapse
|
|
3
|
Lau VI, Johnson JA, Bagshaw SM, Rewa OG, Basmaji J, Lewis KA, Wilcox ME, Barrett K, Lamontagne F, Lauzier F, Ferguson ND, Oczkowski SJW, Fiest KM, Niven DJ, Stelfox HT, Alhazzani W, Herridge M, Fowler R, Cook DJ, Rochwerg B, Xie F. Health-related quality-of-life and health-utility reporting in critical care. World J Crit Care Med 2022; 11:236-245. [PMID: 36051941 PMCID: PMC9305682 DOI: 10.5492/wjccm.v11.i4.236] [Citation(s) in RCA: 3] [Impact Index Per Article: 1.0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 02/03/2022] [Revised: 04/16/2022] [Accepted: 05/28/2022] [Indexed: 02/06/2023] Open
Abstract
Mortality is a well-established patient-important outcome in critical care studies. In contrast, morbidity is less uniformly reported (given the myriad of critical care illnesses and complications of each) but may have a common end-impact on a patient’s functional capacity and health-related quality-of-life (HRQoL). Survival with a poor quality-of-life may not be acceptable depending on individual patient values and preferences. Hence, as mortality decreases within critical care, it becomes increasingly important to measure intensive care unit (ICU) survivor HRQoL. HRQoL measurements with a preference-based scoring algorithm can be converted into health utilities on a scale anchored at 0 (representing death) and 1 (representing full health). They can be combined with survival to calculate quality-adjusted life-years (QALY), which are one of the most widely used methods of combining morbidity and mortality into a composite outcome. Although QALYs have been use for health-technology assessment decision-making, an emerging and novel role would be to inform clinical decision-making for patients, families and healthcare providers about what expected HRQoL may be during and after ICU care. Critical care randomized control trials (RCTs) have not routinely measured or reported HRQoL (until more recently), likely due to incapacity of some patients to participate in patient-reported outcome measures. Further differences in HRQoL measurement tools can lead to non-comparable values. To this end, we propose the validation of a gold-standard HRQoL tool in critical care, specifically the EQ-5D-5L. Both combined health-utility and mortality (disaggregated) and QALYs (aggregated) can be reported, with disaggregation allowing for determination of which components are the main drivers of the QALY outcome. Increased use of HRQoL, health-utility, and QALYs in critical care RCTs has the potential to: (1) Increase the likelihood of finding important effects if they exist; (2) improve research efficiency; and (3) help inform optimal management of critically ill patients allowing for decision-making about their HRQoL, in additional to traditional health-technology assessments.
Collapse
Affiliation(s)
- Vincent Issac Lau
- Department of Critical Care Medicine, University of Alberta, Edmonton T6G 2B7, AB, Canada
| | - Jeffrey A Johnson
- School of Public Health, Inst Hlth Econ, University of Alberta, Edmonton T6G 2B7, AB, Canada
| | - Sean M Bagshaw
- Department of Critical Care Medicine, University of Alberta, Edmonton T6G 2B7, AB, Canada
| | - Oleksa G Rewa
- Department of Critical Care Medicine, University of Alberta, Edmonton T6G 2B7, AB, Canada
| | - John Basmaji
- Department of Medicine, Division of Critical Care, Western University, London N6A 5W9, Canada
| | - Kimberley A Lewis
- Division of Critical Care, McMaster University, Hamilton L8N 4A6, Canada
| | - M Elizabeth Wilcox
- Interdepartmental Division of Critical Care, University of Toronto, Toronto M5T 2S8, Canada
| | - Kali Barrett
- Interdepartmental Division of Critical Care, University of Toronto, Toronto M5T 2S8, Canada
| | | | - Francois Lauzier
- Departments of Medicine and Anesthesiology, University Laval, Laval G1V 4G2, Canada
| | - Niall D Ferguson
- Department Critical Care Medicine, University of Toronto, Toronto M5G 2C4, Canada
| | - Simon J W Oczkowski
- Department of Medicine, McMaster Clin, Hamilton Gen Hosp, McMaster University, Hamilton L8N 4A6, Canada
| | - Kirsten M Fiest
- Department of Community Health Sciences & Institute for Public Health, University of Calgary, Calgary T2N 2T9, Canada
| | - Daniel J Niven
- Department of Critical Care Medicine, University Calgary, Calgary T2N 2T9, Canada
| | - Henry T Stelfox
- Department of Community Health Sciences, University of Calgary, Calgary T2N 2T9, Canada
| | - Waleed Alhazzani
- Department of Medicine, McMaster University, Hamilton L8N 4A6, Canada
| | - Margaret Herridge
- Indepartmental Division of Critical Care, University Health Network, Toronto M5G 2C4, Canada
| | - Robert Fowler
- Departments of Medicine and Critical Care Medicine, Sunnybrook Health Sciences Center, Institute of Health Policy Management and Evaluation, University of Toronto, Toronto M4N 3M5, Canada
| | - Deborah J Cook
- Department of Medicine, McMaster University, Hamilton L8N 4A6, Canada
| | - Bram Rochwerg
- Department of Medicine, McMaster University, Hamilton L8N 4A6, Canada
| | - Feng Xie
- Health Research Methods, Evidence and Impact, McMaster University, Hamilton L8N 3Z5, Canada
| |
Collapse
|
|
4
|
Luo Y, Huang Z, Liu H, Xu H, Su H, Chen Y, Hu Y, Xu B. Development and Validation of a Multimorbidity Index Predicting Mortality Among Older Chinese Adults. Front Aging Neurosci 2022; 14:767240. [PMID: 35370612 PMCID: PMC8965437 DOI: 10.3389/fnagi.2022.767240] [Citation(s) in RCA: 3] [Impact Index Per Article: 1.0] [Reference Citation Analysis] [Abstract] [Grants] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 08/30/2021] [Accepted: 02/16/2022] [Indexed: 12/13/2022] Open
Abstract
Objective This study aimed to develop and validate a multimorbidity index using self-reported chronic conditions for predicting 5-year mortality risk. Methods We analyzed data from the Chinese Longitudinal Healthy Longevity Survey (CLHLS) and included 11,853 community-dwelling older adults aged 65–84 years. Restrictive association rule mining (ARM) was used to identify disease combinations associated with mortality based on 13 chronic conditions. Data were randomly split into the training (N = 8,298) and validation (N = 3,555) sets. Two multimorbidity indices with individual diseases only (MI) and disease combinations (MIDC) were developed using hazard ratios (HRs) for 5-year morality in the training set. We compared the predictive performance in the validation set between the models using condition count, MI, and MIDC by the concordance (C) statistic, the Integrated Discrimination Improvement (IDI), and the Net Reclassification Index (NRI). Results A total of 13 disease combinations were identified. Compared with condition count (C-statistic: 0.710), MIDC (C-statistic: 0.713) showed significantly better discriminative ability (C-statistic: p = 0.016; IDI: 0.005, p < 0.001; NRI: 0.038, p = 0.478). Compared with MI (C-statistic: 0.711), the C-statistic of the model using MIDC was significantly higher (p = 0.031), while the IDI was more than 0 but not statistically significant (IDI: 0.003, p = 0.090). Conclusion Although current multimorbidity status is commonly defined by individual chronic conditions, this study found that the multimorbidity index incorporating disease combinations showed supreme performance in predicting mortality among community-dwelling older adults. These findings suggest a need to consider significant disease combinations when measuring multimorbidity in medical research and clinical practice.
Collapse
Affiliation(s)
- Yan Luo
- Department of Epidemiology and Biostatistics, School of Public Health, Peking University, Beijing, China
- Medical Informatics Center, Peking University, Beijing, China
| | - Ziting Huang
- Department of Epidemiology and Biostatistics, School of Public Health, Peking University, Beijing, China
- Medical Informatics Center, Peking University, Beijing, China
| | - Hui Liu
- Medical Informatics Center, Peking University, Beijing, China
| | - Huiwen Xu
- Department of Epidemiology and Biostatistics, School of Public Health, Peking University, Beijing, China
- Medical Informatics Center, Peking University, Beijing, China
| | - Hexuan Su
- Department of Epidemiology and Biostatistics, School of Public Health, Peking University, Beijing, China
- Medical Informatics Center, Peking University, Beijing, China
| | - Yuming Chen
- Department of Epidemiology and Biostatistics, School of Public Health, Peking University, Beijing, China
- Medical Informatics Center, Peking University, Beijing, China
| | - Yonghua Hu
- Department of Epidemiology and Biostatistics, School of Public Health, Peking University, Beijing, China
- Medical Informatics Center, Peking University, Beijing, China
| | - Beibei Xu
- Medical Informatics Center, Peking University, Beijing, China
- *Correspondence: Beibei Xu,
| |
Collapse
|
|
5
|
de Vos J, Visser LA, de Beer AA, Fornasa M, Thoral PJ, Elbers PWG, Cinà G. The Potential Cost-Effectiveness of a Machine Learning Tool That Can Prevent Untimely Intensive Care Unit Discharge. VALUE IN HEALTH : THE JOURNAL OF THE INTERNATIONAL SOCIETY FOR PHARMACOECONOMICS AND OUTCOMES RESEARCH 2022; 25:359-367. [PMID: 35227446 DOI: 10.1016/j.jval.2021.06.018] [Citation(s) in RCA: 11] [Impact Index Per Article: 3.7] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Subscribe] [Scholar Register] [Received: 01/11/2021] [Revised: 05/06/2021] [Accepted: 06/29/2021] [Indexed: 06/14/2023]
Abstract
OBJECTIVES The machine learning prediction model Pacmed Critical (PC), currently under development, may guide intensivists in their decision-making process on the most appropriate time to discharge a patient from the intensive care unit (ICU). Given the financial pressure on healthcare budgets, this study assessed whether PC has the potential to be cost-effective compared with standard care, without the use of PC, for Dutch patients in the ICU from a societal perspective. METHODS A 1-year, 7-state Markov model reflecting the ICU care pathway and incorporating the PC decision tool was developed. A hypothetical cohort of 1000 adult Dutch patients admitted in the ICU was entered in the model. We used the literature, expert opinion, and data from Amsterdam University Medical Center for model parameters. The uncertainty surrounding the incremental cost-effectiveness ratio was assessed using deterministic and probabilistic sensitivity analyses and scenario analyses. RESULTS PC was a cost-effective strategy with an incremental cost-effectiveness ratio of €18 507 per quality-adjusted life-year. PC remained cost-effective over standard care in multiple scenarios and sensitivity analyses. The likelihood that PC will be cost-effective was 71% at a willingness-to-pay threshold of €30 000 per quality-adjusted life-year. The key driver of the results was the parameter "reduction in ICU length of stay." CONCLUSIONS We showed that PC has the potential to be cost-effective for Dutch ICUs in a time horizon of 1 year. This study is one of the first cost-effectiveness analyses of a machine learning device. Further research is needed to validate the effectiveness of PC, thereby focusing on the key parameter "reduction in ICU length of stay" and potential spill-over effects.
Collapse
Affiliation(s)
- Juliette de Vos
- Pacmed B.V., Amsterdam, The Netherlands; Erasmus School of Health Policy & Management, Erasmus University Rotterdam, Rotterdam, The Netherlands.
| | - Laurenske A Visser
- Erasmus School of Health Policy & Management, Erasmus University Rotterdam, Rotterdam, The Netherlands
| | | | | | - Patrick J Thoral
- Department of Intensive Care Medicine, Laboratory for Critical Care Computational Intelligence, Amsterdam Medical Data Science, Amsterdam UMC, Vrije Universiteit, Amsterdam, The Netherlands
| | - Paul W G Elbers
- Department of Intensive Care Medicine, Laboratory for Critical Care Computational Intelligence, Amsterdam Medical Data Science, Amsterdam UMC, Vrije Universiteit, Amsterdam, The Netherlands
| | | |
Collapse
|
|
6
|
Lau VI, Xie F, Basmaji J, Cook DJ, Fowler R, Kiflen M, Sirotich E, Iansavichene A, Bagshaw SM, Wilcox ME, Lamontagne F, Ferguson N, Rochwerg B. Health-Related Quality-of-Life and Cost Utility Analyses in Critical Care: A Systematic Review. Crit Care Med 2021; 49:575-588. [PMID: 33591013 DOI: 10.1097/ccm.0000000000004851] [Citation(s) in RCA: 7] [Impact Index Per Article: 1.8] [Reference Citation Analysis] [Abstract] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 12/16/2022]
Abstract
OBJECTIVES Cost utility analyses compare the costs and health outcome of interventions, with a denominator of quality-adjusted life year, a generic health utility measure combining both quality and quantity of life. Cost utility analyses are difficult to compare when methods are not standardized. It is unclear how cost utility analyses are measured/reported in critical care and what methodologic challenges cost utility analyses pose in this setting. This may lead to differences precluding cost utility analyses comparisons. Therefore, we performed a systematic review of cost utility analyses conducted in critical care. Our objectives were to understand: 1) methodologic characteristics, 2) how health-related quality-of-life was measured/reported, and 3) what costs were reported/measured. DESIGN Systematic review. DATA SOURCES We systematically searched for cost utility analyses in critical care in MEDLINE, Embase, American College of Physicians Journal Club, CENTRAL, Evidence-Based Medicine Reviews' selected subset of archived versions of UK National Health Service Economic Evaluation Database, Database of Abstracts of Reviews of Effects, and American Economic Association electronic databases from inception to April 30, 2020. SETTING Adult ICUs. PATIENTS Adult critically ill patients. INTERVENTIONS None. MEASUREMENTS AND MAIN RESULTS Of 8,926 citations, 80 cost utility analyse studies were eligible. The time horizon most commonly reported was lifetime (59%). For health utility reporting, health-related quality-of-life was infrequently measured (29% reported), with only 5% of studies reporting baseline health-related quality-of-life. Indirect utility measures (generic, preference-based health utility measurement tools) were reported in 85% of studies (majority Euro-quality-of-life-5 Domains, 52%). Methods of estimating health-related quality-of-life were seldom used when the patient was incapacitated: imputation (19%), assigning fixed utilities for incapacitation (19%), and surrogates reporting on behalf of incapacitated patients (5%). For cost utility reporting transparency, separate incremental costs and quality-adjusted life years were both reported in only 76% of studies. Disaggregated quality-adjusted life years (reporting separate health utility and life years) were described in only 34% of studies. CONCLUSIONS We identified deficiencies which warrant recommendations (standardized measurement/reporting of resource use/unit costs/health-related quality-of-life/methodological preferences) for improved design, conduct, and reporting of future cost utility analyses in critical care.
Collapse
Affiliation(s)
- Vincent I Lau
- Department of Critical Care Medicine, Faculty of Medicine and Dentistry, University of Alberta, and Alberta Health Services, Edmonton, AB, Canada
- Department of Health Research Methods, Evidence & Impact, McMaster University, Hamilton, ON, Canada
| | - Feng Xie
- Department of Health Research Methods, Evidence & Impact, McMaster University, Hamilton, ON, Canada
| | - John Basmaji
- Department of Medicine, Division of Critical Care Medicine, Western University, London, ON, Canada
| | - Deborah J Cook
- Department of Health Research Methods, Evidence & Impact, McMaster University, Hamilton, ON, Canada
- Department of Medicine, Division of Critical Care Medicine, McMaster University, Hamilton, ON, Canada
| | - Robert Fowler
- Interdepartmental Division of Critical Care Medicine, University of Toronto, Toronto, ON, Canada
- Department of Medicine, University Health Network, Ontario, ON, Canada
| | - Michel Kiflen
- Department of Health Research Methods, Evidence & Impact, McMaster University, Hamilton, ON, Canada
- Population Health Research Institute, McMaster University, Hamilton, ON, Canada
| | - Emily Sirotich
- Department of Health Research Methods, Evidence & Impact, McMaster University, Hamilton, ON, Canada
| | | | - Sean M Bagshaw
- Department of Critical Care Medicine, Faculty of Medicine and Dentistry, University of Alberta, and Alberta Health Services, Edmonton, AB, Canada
| | - M Elizabeth Wilcox
- Interdepartmental Division of Critical Care Medicine, University of Toronto, Toronto, ON, Canada
- Department of Medicine, University Health Network, Ontario, ON, Canada
| | - François Lamontagne
- Centre de Recherche du CHU de Sherbrooke, Université de Sherbrooke, Sherbrooke, QC, Canada
| | - Niall Ferguson
- Interdepartmental Division of Critical Care Medicine, University of Toronto, Toronto, ON, Canada
- Department of Medicine, University Health Network, Ontario, ON, Canada
| | - Bram Rochwerg
- Department of Health Research Methods, Evidence & Impact, McMaster University, Hamilton, ON, Canada
- Department of Medicine, Division of Critical Care Medicine, McMaster University, Hamilton, ON, Canada
| |
Collapse
|
|
7
|
Fresh Red Cells for Transfusion in Critically Ill Adults: An Economic Evaluation of the Standard Issue Transfusion Versus Fresher Red-Cell Use in Intensive Care (TRANSFUSE) Clinical Trial. Crit Care Med 2020; 47:e572-e579. [PMID: 31008734 DOI: 10.1097/ccm.0000000000003781] [Citation(s) in RCA: 4] [Impact Index Per Article: 0.8] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 11/26/2022]
Abstract
OBJECTIVES Trials comparing the effects of transfusing RBC units of different storage durations have considered mortality or morbidity as outcomes. We perform the first economic evaluation alongside a full age of blood clinical trial with a large population assessing the impact of RBC storage duration on quality-of-life and costs in critically ill adults. DESIGN Quality-of-life was measured at 6 months post randomization using the EuroQol 5-dimension 3-level instrument. The economic evaluation considers quality-adjusted life year and cost implications from randomization to 6 months. A generalized linear model was used to estimate incremental costs (2016 U.S. dollars) and quality-adjusted life years, respectively while adjusting for baseline characteristics. SETTING Fifty-nine ICUs in five countries. PATIENTS Adults with an anticipated ICU stay of at least 24 hours when the decision had been made to transfuse at least one RBC unit. INTERVENTIONS Patients were randomized to receive either the freshest or oldest available compatible RBC units (standard practice) in the hospital transfusion service. MEASUREMENTS AND MAIN RESULTS EuroQol 5-dimension 3-level utility scores were similar at 6 months-0.65 in the short-term and 0.63 in the long-term storage group (difference, 0.02; 95% CI, -0.00 to 0.04; p = 0.10). There were no significant differences in resource use between the two groups apart from 3.0 fewer hospital readmission days (95% CI, -5.3 to -0.8; p = 0.01) during follow-up in the short-term storage group. There were no significant differences in adjusted total costs or quality-adjusted life years between the short- and long-term storage groups (incremental costs, -$2,358; 95% CI, -$5,586 to $711) and incremental quality-adjusted life years: 0.003 quality-adjusted life years (95% CI, -0.003 to 0.008). CONCLUSIONS Without considering the additional supply cost of implementing a freshest available RBC strategy for critical care patients, there is no evidence to suggest that the policy improves quality-of-life or reduces other costs compared with standard transfusion practice.
Collapse
|
|
8
|
The Cost-Effectiveness of Interventions to Increase Utilization of Prone Positioning for Severe Acute Respiratory Distress Syndrome. Crit Care Med 2020; 47:e198-e205. [PMID: 30779719 DOI: 10.1097/ccm.0000000000003617] [Citation(s) in RCA: 9] [Impact Index Per Article: 1.8] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 12/13/2022]
Abstract
OBJECTIVES Despite strong evidence supporting proning in acute respiratory distress syndrome, few eligible patients receive it. This study determines the cost-effectiveness of interventions to increase utilization of proning for severe acute respiratory distress syndrome. DESIGN We created decision trees to model severe acute respiratory distress syndrome from ICU admission through death (societal perspective) and hospital discharge (hospital perspective). We assumed patients received low tidal volume ventilation. We used short-term outcome estimates from the PROSEVA trial and longitudinal cost and benefit data from cohort studies. In probabilistic sensitivity analyses, we used distributions for each input that included the fifth to 95th percentile of its CI. SETTING ICUs that care for patients with acute respiratory distress syndrome. SUBJECTS Patients with moderate to severe acute respiratory distress syndrome. INTERVENTIONS The implementation of a hypothetical intervention to increase the appropriate utilization of prone positioning. MEASUREMENTS AND MAIN RESULTS In the societal perspective model, an intervention that increased proning utilization from 16% to 65% yielded an additional 0.779 (95% CI, 0.088-1.714) quality-adjusted life years at an additional long-term cost of $31,156 (95% CI, -$158 to $92,179) (incremental cost-effectiveness ratio = $38,648 per quality-adjusted life year [95% CI, $1,695-$98,522]). If society was willing to pay $100,000 per quality-adjusted life year, any intervention costing less than $51,328 per patient with moderate to severe acute respiratory distress syndrome would represent good value. From a hospital perspective, the intervention yielded 0.072 (95% CI, 0.008-0.147) more survivals-to-discharge at a cost of $5,242 (95% CI, -$19,035 to $41,019) (incremental cost-effectiveness ratio = $44,615 per extra survival [95% CI, -$250,912 to $558,222]). If hospitals were willing to pay $100,000 per survival-to-discharge, any intervention costing less than $5,140 per patient would represent good value. CONCLUSIONS Interventions that increase utilization of proning would be cost-effective from both societal and hospital perspectives under many plausible cost and benefit assumptions.
Collapse
|
|
9
|
Economic Evaluation of Venovenous Extracorporeal Membrane Oxygenation for Severe Acute Respiratory Distress Syndrome. Crit Care Med 2019; 47:186-193. [PMID: 30312186 DOI: 10.1097/ccm.0000000000003465] [Citation(s) in RCA: 20] [Impact Index Per Article: 3.3] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 12/11/2022]
Abstract
OBJECTIVES Venovenous extracorporeal membrane oxygenation is increasingly being used to support patients with severe acute respiratory distress syndrome, but its cost-effectiveness is unknown. We assessed the cost-utility of venovenous extracorporeal membrane oxygenation for severe acute respiratory distress syndrome in adults compared with standard lung protective ventilation from the perspective of the healthcare system. DESIGN We conducted a cost-utility analysis with a cohort state transition decision model using a lifetime time horizon, 1.5% discount rate, and outcomes reported as cost per quality-adjusted life year. Literature reviews were conducted to inform the model variables. Deterministic and probabilistic sensitivity analyses were conducted to assess uncertainty in the model. SETTING Canadian publicly funded healthcare system. PATIENTS Hypothetical cohort of adults with severe acute respiratory distress syndrome. INTERVENTIONS Venovenous extracorporeal membrane oxygenation or standard lung protective ventilation. MEASUREMENTS AND MAIN RESULTS In our model, the use of venovenous extracorporeal membrane oxygenation compared with lung protective ventilation resulted in a gain of 5.2 life years and 4.05 quality-adjusted life years, at an additional lifetime cost of $145,697 Canadian dollars. The incremental cost-effectiveness ratio was $36,001/quality-adjusted life year. Sensitivity analyses show that the incremental cost-effectiveness ratio is sensitive to the efficacy of extracorporeal membrane oxygenation therapy and costs. CONCLUSIONS Based on current data, venovenous extracorporeal membrane oxygenation is cost-effective for patients with severe acute respiratory distress syndrome. Additional evidence on the efficacy of venovenous extracorporeal membrane oxygenation for acute respiratory distress syndrome and in different subgroups of patients will allow for greater certainty in its cost-effectiveness.
Collapse
|
|
10
|
Santos HGD, Zampieri FG, Normilio-Silva K, Silva GTD, Lima ACPD, Cavalcanti AB, Chiavegatto Filho ADP. Machine learning to predict 30-day quality-adjusted survival in critically ill patients with cancer. J Crit Care 2019; 55:73-78. [PMID: 31715534 DOI: 10.1016/j.jcrc.2019.10.015] [Citation(s) in RCA: 11] [Impact Index Per Article: 1.8] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 05/22/2019] [Revised: 08/13/2019] [Accepted: 10/31/2019] [Indexed: 02/04/2023]
Abstract
PURPOSE To develop and compare the predictive performance of machine-learning algorithms to estimate the risk of quality-adjusted life year (QALY) lower than or equal to 30 days (30-day QALY). MATERIAL AND METHODS Six machine-learning algorithms were applied to predict 30-day QALY for 777 patients admitted in a prospective cohort study conducted in Intensive Care Units (ICUs) of two public Brazilian hospitals specialized in cancer care. The predictors were 37 characteristics collected at ICU admission. Discrimination was evaluated using the area under the receiver operating characteristic (AUROC) curve. Sensitivity, 1-specificity, true/false positive and negative cases were measured for different estimated probability cutoff points (30%, 20% and 10%). Calibration was evaluated with GiViTI calibration belt and test. RESULTS Except for basic decision trees, the adjusted predictive models were nearly equivalent, presenting good results for discrimination (AUROC curves over 0.80). Artificial neural networks and gradient boosted trees achieved the overall best calibration, implying an accurately predicted probability for 30-day QALY. CONCLUSIONS Except for basic decision trees, predictive models derived from different machine-learning algorithms discriminated the QALY risk at 30 days well. Regarding calibration, artificial neural network model presented the best ability to estimate 30-day QALY in critically ill oncologic patients admitted to ICUs.
Collapse
Affiliation(s)
| | | | - Karina Normilio-Silva
- Research Institute, Heart Hospital (Hospital do Coração - Hcor), São Paulo, São Paulo, Brazil; Cancer Institute of the State of São Paulo (Instituto do Câncer do Estado de São Paulo - ICESP), São Paulo, São Paulo, Brazil
| | - Gisela Tunes da Silva
- Department of Statistics, Institute of Mathematics and Statistics, University of São Paulo, São Paulo, Brazil
| | | | - Alexandre Biasi Cavalcanti
- Research Institute, Heart Hospital (Hospital do Coração - Hcor), São Paulo, São Paulo, Brazil; Cancer Institute of the State of São Paulo (Instituto do Câncer do Estado de São Paulo - ICESP), São Paulo, São Paulo, Brazil
| | | |
Collapse
|
|
11
|
Turbil E, Galerneau LM, Terzi N, Schwebel C, Argaud L, Guérin C. Positive-end expiratory pressure titration and transpulmonary pressure: the EPVENT 2 trial. J Thorac Dis 2019; 11:S2012-S2017. [PMID: 31632813 DOI: 10.21037/jtd.2019.06.34] [Citation(s) in RCA: 4] [Impact Index Per Article: 0.7] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 01/19/2023]
Affiliation(s)
- Emanuele Turbil
- Anesthesiology and Intensive Care, Università degli Studi di Sassari, Sassari, Italy
| | - Louis Marie Galerneau
- Medical ICU, University Hospital, Grenoble, France.,University of Grenoble-Alpes, Grenoble, France
| | - Nicolas Terzi
- Medical ICU, University Hospital, Grenoble, France.,University of Grenoble-Alpes, Grenoble, France
| | - Carole Schwebel
- Medical ICU, University Hospital, Grenoble, France.,University of Grenoble-Alpes, Grenoble, France
| | - Laurent Argaud
- Medical ICU, University Hospital Lyon Center, Lyon, France.,University of Lyon, Lyon, France
| | - Claude Guérin
- University of Lyon, Lyon, France.,INSERM 955, Créteil, France
| |
Collapse
|
|
12
|
Caruana E, Foucher Y, Tessier P, Frenel JS, Classe JM, Dantan E. Patient-centered simulations to assess the usefulness of the 70-gene signature for adjuvant chemotherapy administration in early-stage breast cancer. Breast Cancer Res Treat 2019; 174:537-542. [PMID: 30603997 DOI: 10.1007/s10549-018-05107-6] [Citation(s) in RCA: 1] [Impact Index Per Article: 0.2] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 09/04/2018] [Accepted: 12/16/2018] [Indexed: 12/12/2022]
Abstract
PURPOSE From the MINDACT trial, Cardoso et al. did not demonstrate a significant efficacy for adjuvant chemotherapy (CT) for women with early-stage breast cancer presenting high clinical and low genomic risks. Our objective was to assess the usefulness of the 70-gene signature in this population by using an alternative endpoint: the number of Quality-Adjusted Life-Years (QALYs), i.e., a synthetic measure of quantity and quality of life. METHODS Based on the results of the MINDACT trial, we simulated a randomized clinical trial consisting of 1497 women with early-stage breast cancer presenting high clinical and low genomic risks. The individual preferences for the different health states and corresponding decrements were obtained from the literature. RESULTS The gain in terms of 5-year disease-free survival was 2.8% (95% CI from - 0.1 to 5.7%, from 90.4% for women without CT to 93.3% for women with CT). In contrast, due to the associated side effects, CT significantly reduced the number of QALYs by 62 days (95% CI from 55 to 70 days, from 4.13 years for women without CT to 3.96 years for women with CT). CONCLUSION Our results support the conclusions published by Cardoso et al. by providing additional evidence that the 70-gene signature can be used to avoid overtreatment by CT for women with high clinical risk but low genomic risk.
Collapse
Affiliation(s)
- Emmanuel Caruana
- INSERM UMR 1246 -SPHERE, Nantes University, Tours University, Nantes, France
| | - Yohann Foucher
- INSERM UMR 1246 -SPHERE, Nantes University, Tours University, Nantes, France.,Nantes University Hospital, Nantes, France
| | - Philippe Tessier
- INSERM UMR 1246 -SPHERE, Nantes University, Tours University, Nantes, France
| | - Jean-Sébastien Frenel
- Institut de Cancérologie de l'Ouest, Centre René Gauducheau, Bd Jacques Monod, 44800, Saint-Herblain, France
| | - Jean-Marc Classe
- Institut de Cancérologie de l'Ouest, Centre René Gauducheau, Bd Jacques Monod, 44800, Saint-Herblain, France
| | - Etienne Dantan
- INSERM UMR 1246 -SPHERE, Nantes University, Tours University, Nantes, France.
| |
Collapse
|
|
13
|
Self WH, Liu D, Strayer N, Russ S, Ward MJ, Shapiro NI, Rice TW, Semler MW. Charge Reductions Associated With Shorter Time to Recovery in Septic Shock. Chest 2018; 155:315-321. [PMID: 30419234 DOI: 10.1016/j.chest.2018.10.034] [Citation(s) in RCA: 1] [Impact Index Per Article: 0.1] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 04/10/2018] [Revised: 10/18/2018] [Accepted: 10/29/2018] [Indexed: 01/12/2023] Open
Abstract
BACKGROUND Septic shock therapies that shorten the time to physiologic and clinical recovery may result in financial savings. However, the financial implications of improving these nonmortal outcomes are not well characterized. Therefore, we quantified hospital charges associated with four outcomes: ICU length of stay, duration of invasive mechanical ventilation, duration of vasopressor use, and new renal replacement therapy. METHODS This was an observational study using administrative data from a large academic hospital in the United States. The analysis included adults treated with vasopressors for septic shock in a medical ICU. Linear regression modeling with ordinary least square was used to estimate the incremental hospital charges associated with 1 day of ICU length of stay, 1 day of mechanical ventilation, 1 day of vasopressor use, and new renal replacement therapy. RESULTS The study population included 587 adults with septic shock, including 180 (30.7%) who died in the hospital. The median charge for a septic shock hospitalization was $98,583 (interquartile range [IQR], $61,177-$136,672). Decreases in ICU length of stay, mechanical ventilation duration, and vasopressor duration of 1 day were associated with charge reductions of $15,670 (IQR, $15,023-$16,317), $15,284 (IQR, $13,566-$17,002), and $17,947 (IQR, $16,344-$19,549), respectively. Avoidance of new renal replacement therapy was associated with a charge reduction of $36,051 (IQR, $22,353-$49,750). CONCLUSIONS Septic shock therapies that reduce the duration of organ support and ICU care have the potential to lead to substantial financial savings.
Collapse
Affiliation(s)
- Wesley H Self
- Department of Emergency Medicine, Vanderbilt University Medical Center, Nashville, TN.
| | - Dandan Liu
- Department of Biostatistics, Vanderbilt University Medical Center, Nashville, TN
| | - Nicholas Strayer
- Department of Biostatistics, Vanderbilt University Medical Center, Nashville, TN
| | - Stephan Russ
- Department of Emergency Medicine, Vanderbilt University Medical Center, Nashville, TN
| | - Michael J Ward
- Department of Emergency Medicine, Vanderbilt University Medical Center, Nashville, TN
| | - Nathan I Shapiro
- Department of Emergency Medicine, Beth Israel Deaconess Medical Center, Boston, MA
| | - Todd W Rice
- Department of Medicine, Division of Allergy, Pulmonary and Critical Care Medicine, Vanderbilt University Medical Center, Nashville, TN
| | - Matthew W Semler
- Department of Medicine, Division of Allergy, Pulmonary and Critical Care Medicine, Vanderbilt University Medical Center, Nashville, TN
| |
Collapse
|
|
14
|
One more lesson from a great man! The intensive care ethical dilemma exposed. TRENDS IN ANAESTHESIA AND CRITICAL CARE 2018. [DOI: 10.1016/j.tacc.2018.05.008] [Citation(s) in RCA: 5] [Impact Index Per Article: 0.7] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 11/22/2022]
|
|
15
|
Gates S, Lall R, Quinn T, Deakin CD, Cooke MW, Horton J, Lamb SE, Slowther AM, Woollard M, Carson A, Smyth M, Wilson K, Parcell G, Rosser A, Whitfield R, Williams A, Jones R, Pocock H, Brock N, Black JJ, Wright J, Han K, Shaw G, Blair L, Marti J, Hulme C, McCabe C, Nikolova S, Ferreira Z, Perkins GD. Prehospital randomised assessment of a mechanical compression device in out-of-hospital cardiac arrest (PARAMEDIC): a pragmatic, cluster randomised trial and economic evaluation. Health Technol Assess 2018; 21:1-176. [PMID: 28393757 DOI: 10.3310/hta21110] [Citation(s) in RCA: 26] [Impact Index Per Article: 3.7] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 12/15/2022] Open
Abstract
BACKGROUND Mechanical chest compression devices may help to maintain high-quality cardiopulmonary resuscitation (CPR), but little evidence exists for their effectiveness. We evaluated whether or not the introduction of Lund University Cardiopulmonary Assistance System-2 (LUCAS-2; Jolife AB, Lund, Sweden) mechanical CPR into front-line emergency response vehicles would improve survival from out-of-hospital cardiac arrest (OHCA). OBJECTIVE Evaluation of the LUCAS-2 device as a routine ambulance service treatment for OHCA. DESIGN Pragmatic, cluster randomised trial including adults with non-traumatic OHCA. Ambulance dispatch staff and those collecting the primary outcome were blind to treatment allocation. Blinding of the ambulance staff who delivered the interventions and reported initial response to treatment was not possible. We also conducted a health economic evaluation and a systematic review of all trials of out-of-hospital mechanical chest compression. SETTING Four UK ambulance services (West Midlands, North East England, Wales and South Central), comprising 91 urban and semiurban ambulance stations. Clusters were ambulance service vehicles, which were randomly assigned (approximately 1 : 2) to the LUCAS-2 device or manual CPR. PARTICIPANTS Patients were included if they were in cardiac arrest in the out-of-hospital environment. Exclusions were patients with cardiac arrest as a result of trauma, with known or clinically apparent pregnancy, or aged < 18 years. INTERVENTIONS Patients received LUCAS-2 mechanical chest compression or manual chest compressions according to the first trial vehicle to arrive on scene. MAIN OUTCOME MEASURES Survival at 30 days following cardiac arrest; survival without significant neurological impairment [Cerebral Performance Category (CPC) score of 1 or 2]. RESULTS We enrolled 4471 eligible patients (1652 assigned to the LUCAS-2 device and 2819 assigned to control) between 15 April 2010 and 10 June 2013. A total of 985 (60%) patients in the LUCAS-2 group received mechanical chest compression and 11 (< 1%) patients in the control group received LUCAS-2. In the intention-to-treat analysis, 30-day survival was similar in the LUCAS-2 (104/1652, 6.3%) and manual CPR groups [193/2819, 6.8%; adjusted odds ratio (OR) 0.86, 95% confidence interval (CI) 0.64 to 1.15]. Survival with a CPC score of 1 or 2 may have been worse in the LUCAS-2 group (adjusted OR 0.72, 95% CI 0.52 to 0.99). No serious adverse events were noted. The systematic review found no evidence of a survival advantage if mechanical chest compression was used. The health economic analysis showed that LUCAS-2 was dominated by manual chest compression. LIMITATIONS There was substantial non-compliance in the LUCAS-2 arm. For 272 out of 1652 patients (16.5%), mechanical chest compression was not used for reasons that would not occur in clinical practice. We addressed this issue by using complier average causal effect analyses. We attempted to measure CPR quality during the resuscitation attempts of trial participants, but were unable to do so. CONCLUSIONS There was no evidence of improvement in 30-day survival with LUCAS-2 compared with manual compressions. Our systematic review of recent randomised trials did not suggest that survival or survival without significant disability may be improved by the use of mechanical chest compression. FUTURE WORK The use of mechanical chest compression for in-hospital cardiac arrest, and in specific circumstances (e.g. transport), has not yet been evaluated. TRIAI REGISTRATION Current Controlled Trials ISRCTN08233942. FUNDING This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 21, No. 11. See the NIHR Journals Library website for further project information.
Collapse
Affiliation(s)
- Simon Gates
- Warwick Clinical Trials Unit, University of Warwick, Coventry, UK
| | - Ranjit Lall
- Warwick Clinical Trials Unit, University of Warwick, Coventry, UK
| | - Tom Quinn
- Surrey Peri-operative Anaesthesia Critical Care Collaborative Research Group, Faculty of Health and Medical Sciences, University of Surrey, Guildford, UK (current address: Faculty of Health, Social Care and Education, Kingston University London and St George's, University of London, London, UK)
| | - Charles D Deakin
- South Central Ambulance Service NHS Foundation Trust, Otterbourne, UK
| | - Matthew W Cooke
- Warwick Clinical Trials Unit, University of Warwick, Coventry, UK.,Heart of England NHS Foundation Trust, Birmingham, UK
| | - Jessica Horton
- Warwick Clinical Trials Unit, University of Warwick, Coventry, UK
| | - Sarah E Lamb
- Warwick Clinical Trials Unit, University of Warwick, Coventry, UK.,Oxford Clinical Trials Research Unit, University of Oxford, Oxford, UK
| | | | - Malcolm Woollard
- Surrey Peri-operative Anaesthesia Critical Care Collaborative Research Group, Faculty of Health and Medical Sciences, University of Surrey, Guildford, UK (current address: Faculty of Health, Social Care and Education, Kingston University London and St George's, University of London, London, UK)
| | - Andy Carson
- West Midlands Ambulance Service NHS Foundation Trust, Brierley Hill, UK
| | - Mike Smyth
- Warwick Clinical Trials Unit, University of Warwick, Coventry, UK.,West Midlands Ambulance Service NHS Foundation Trust, Brierley Hill, UK
| | - Kate Wilson
- West Midlands Ambulance Service NHS Foundation Trust, Brierley Hill, UK
| | - Garry Parcell
- West Midlands Ambulance Service NHS Foundation Trust, Brierley Hill, UK
| | - Andrew Rosser
- West Midlands Ambulance Service NHS Foundation Trust, Brierley Hill, UK
| | | | | | | | - Helen Pocock
- South Central Ambulance Service NHS Foundation Trust, Otterbourne, UK
| | - Nicola Brock
- South Central Ambulance Service NHS Foundation Trust, Otterbourne, UK
| | - John Jm Black
- South Central Ambulance Service NHS Foundation Trust, Otterbourne, UK
| | - John Wright
- North East Ambulance Service NHS Foundation Trust, Newcastle upon Tyne, UK.,Royal Victoria Infirmary, Newcastle upon Tyne, UK
| | - Kyee Han
- North East Ambulance Service NHS Foundation Trust, Newcastle upon Tyne, UK
| | - Gary Shaw
- North East Ambulance Service NHS Foundation Trust, Newcastle upon Tyne, UK
| | - Laura Blair
- North East Ambulance Service NHS Foundation Trust, Newcastle upon Tyne, UK
| | - Joachim Marti
- Academic Unit of Health Economics, Leeds Institute of Health Sciences, University of Leeds, Leeds, UK
| | - Claire Hulme
- Academic Unit of Health Economics, Leeds Institute of Health Sciences, University of Leeds, Leeds, UK
| | - Christopher McCabe
- Department of Emergency Medicine Research, University of Alberta, Edmonton, AB, Canada
| | - Silviya Nikolova
- Academic Unit of Health Economics, Leeds Institute of Health Sciences, University of Leeds, Leeds, UK
| | - Zenia Ferreira
- Academic Unit of Health Economics, Leeds Institute of Health Sciences, University of Leeds, Leeds, UK
| | - Gavin D Perkins
- Warwick Clinical Trials Unit, University of Warwick, Coventry, UK.,Heart of England NHS Foundation Trust, Birmingham, UK
| |
Collapse
|
|
16
|
Colantuoni E, Scharfstein DO, Wang C, Hashem MD, Leroux A, Needham DM, Girard TD. Statistical methods to compare functional outcomes in randomized controlled trials with high mortality. BMJ 2018; 360:j5748. [PMID: 29298779 PMCID: PMC5751848 DOI: 10.1136/bmj.j5748] [Citation(s) in RCA: 59] [Impact Index Per Article: 8.4] [Reference Citation Analysis] [Abstract] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Indexed: 12/11/2022]
Abstract
Mortality is a common primary endpoint in randomized controlled trials of patients with a high severity of illness, such as critically ill patients. However, researchers are increasingly evaluating functional outcomes, such as quality of life. Importantly, in such trials some patients may die before the assessment of a functional outcome, resulting in the functional outcome being “truncated due to death.” As described in this paper, defining and testing treatment effects on functional outcomes in this setting requires careful consideration. Data from a completed trial of critically ill patients are used to highlight key differences among three statistical approaches used when analyzing such trials.
Collapse
Affiliation(s)
- Elizabeth Colantuoni
- Outcomes After Critical Illness and Surgery (OACIS) Group, Johns Hopkins University, Baltimore, MD, USA
- Department of Biostatistics, Bloomberg School of Public Health, Johns Hopkins University, Baltimore, MD, USA
| | - Daniel O Scharfstein
- Outcomes After Critical Illness and Surgery (OACIS) Group, Johns Hopkins University, Baltimore, MD, USA
- Department of Biostatistics, Bloomberg School of Public Health, Johns Hopkins University, Baltimore, MD, USA
| | - Chenguang Wang
- Division of Biostatistics and Bioinformatics, Johns Hopkins University School of Medicine, Baltimore, MD, USA
| | - Mohamed D Hashem
- Outcomes After Critical Illness and Surgery (OACIS) Group, Johns Hopkins University, Baltimore, MD, USA
- Division of Pulmonary and Critical Care Medicine, Johns Hopkins University School of Medicine, Baltimore, MD, USA
| | - Andrew Leroux
- Department of Biostatistics, Bloomberg School of Public Health, Johns Hopkins University, Baltimore, MD, USA
| | - Dale M Needham
- Outcomes After Critical Illness and Surgery (OACIS) Group, Johns Hopkins University, Baltimore, MD, USA
- Division of Pulmonary and Critical Care Medicine, Johns Hopkins University School of Medicine, Baltimore, MD, USA
- Department of Physical Medicine and Rehabilitation, Johns Hopkins University School of Medicine, Baltimore, MD, USA
| | - Timothy D Girard
- Clinical Research, Investigation, and Systems Modeling of Acute illness (CRISMA) Center in the Department of Critical Care Medicine, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA
| |
Collapse
|
|
17
|
Abu Al-Saad N, Skedgel C, Nortje J. Principles of resource allocation in critical care. BJA Educ 2017. [DOI: 10.1093/bjaed/mkx029] [Citation(s) in RCA: 7] [Impact Index Per Article: 0.9] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 01/07/2023] Open
|
|
18
|
Jolley SE, Hough CL, Clermont G, Hayden D, Hou S, Schoenfeld D, Smith NL, Thompson BT, Bernard GR, for the ARDS Network Investigators. Relationship between Race and the Effect of Fluids on Long-term Mortality after Acute Respiratory Distress Syndrome. Secondary Analysis of the National Heart, Lung, and Blood Institute Fluid and Catheter Treatment Trial. Ann Am Thorac Soc 2017; 14:1443-1449. [PMID: 28708421 PMCID: PMC5711400 DOI: 10.1513/annalsats.201611-906oc] [Citation(s) in RCA: 14] [Impact Index Per Article: 1.8] [Reference Citation Analysis] [Abstract] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 11/18/2016] [Accepted: 06/09/2017] [Indexed: 11/20/2022] Open
Abstract
RATIONALE Short-term follow-up in the Fluid and Catheter Treatment Trial (FACTT) suggested differential mortality by race with conservative fluid management, but no significant interaction. OBJECTIVE In a post hoc analysis of FACTT including 1-year follow-up, we sought to estimate long-term mortality by race and test for an interaction between fluids and race. METHODS We performed a post hoc analysis of FACTT and the Economic Analysis of Pulmonary Artery Catheters (EAPAC) study (which included 655 of the 1,000 FACTT patients with near-complete 1-year follow up). We fit a multistate Markov model to estimate 1-year mortality for all non-Hispanic black and white randomized FACTT subjects. The model estimated the distribution of time from randomization to hospital discharge or hospital death (available on all patients) and estimated the distribution of time from hospital discharge to death using data on patients after hospital discharge for patients in EAPAC. The 1-year mortality was found by combining these estimates. RESULTS Non-Hispanic black (n = 217, 25%) or white identified subjects (n = 641, 75%) were included. There was a significant interaction between race and fluid treatment (P = 0.012). One-year mortality was lower for black subjects assigned to conservative fluids (38 vs. 54%; mean mortality difference, 16%; 95% confidence interval, 2-30%; P = 0.027 between conservative and liberal). Conversely, 1-year mortality for white subjects was 35% versus 30% for conservative versus liberal arms (mean mortality difference, -4.8%; 95% confidence interval, -13% to 3%; P = 0.23). CONCLUSIONS In our cohort, conservative fluid management may have improved 1-year mortality for non-Hispanic black patients with ARDS. However, we found no long-term benefit of conservative fluid management in white subjects.
Collapse
Affiliation(s)
- Sarah E. Jolley
- Louisiana State University, Section of Pulmonary/Critical Care Medicine, New Orleans, Louisiana
| | - Catherine L. Hough
- University of Washington, Division of Pulmonary/Critical Care Medicine, Seattle, Washington
| | - Gilles Clermont
- University of Pittsburgh, Division of Pulmonary/Critical Care Medicine, Pittsburgh, Pennsylvania
| | - Douglas Hayden
- Massachusetts General Hospital, Biostatistics Center, Department of Medicine, Boston, Massachusetts
| | - Suqin Hou
- Massachusetts General Hospital, Biostatistics Center, Department of Medicine, Boston, Massachusetts
| | - David Schoenfeld
- Massachusetts General Hospital, Biostatistics Center, Department of Medicine, Boston, Massachusetts
| | - Nicholas L. Smith
- University of Washington, Department of Epidemiology, Seattle, Washington
| | - Boyd Taylor Thompson
- Massachusetts General Hospital, Division of Pulmonary/Critical Care Medicine, Boston, Massachusetts; and
| | - Gordon R. Bernard
- Vanderbilt University, Division of Pulmonary/Critical Care, Nashville, Tennessee
| | - for the ARDS Network Investigators
- Louisiana State University, Section of Pulmonary/Critical Care Medicine, New Orleans, Louisiana
- University of Washington, Division of Pulmonary/Critical Care Medicine, Seattle, Washington
- University of Pittsburgh, Division of Pulmonary/Critical Care Medicine, Pittsburgh, Pennsylvania
- Massachusetts General Hospital, Biostatistics Center, Department of Medicine, Boston, Massachusetts
- University of Washington, Department of Epidemiology, Seattle, Washington
- Massachusetts General Hospital, Division of Pulmonary/Critical Care Medicine, Boston, Massachusetts; and
- Vanderbilt University, Division of Pulmonary/Critical Care, Nashville, Tennessee
| |
Collapse
|
|
19
|
Marti J, Hulme C, Ferreira Z, Nikolova S, Lall R, Kaye C, Smyth M, Kelly C, Quinn T, Gates S, Deakin CD, Perkins GD. The cost-effectiveness of a mechanical compression device in out-of-hospital cardiac arrest. Resuscitation 2017; 117:1-7. [DOI: 10.1016/j.resuscitation.2017.04.036] [Citation(s) in RCA: 14] [Impact Index Per Article: 1.8] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 09/29/2016] [Revised: 03/21/2017] [Accepted: 04/30/2017] [Indexed: 10/19/2022]
|
|
20
|
Long-Term Survival, Quality of Life, and Quality-Adjusted Survival in Critically Ill Patients With Cancer. Crit Care Med 2017; 44:1327-37. [PMID: 26998653 DOI: 10.1097/ccm.0000000000001648] [Citation(s) in RCA: 37] [Impact Index Per Article: 4.6] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 12/12/2022]
Abstract
OBJECTIVES To assess the long-term survival, health-related quality of life, and quality-adjusted life years of cancer patients admitted to ICUs. DESIGN Prospective cohort. SETTING Two cancer specialized ICUs in Brazil. PATIENTS A total of 792 participants. INTERVENTIONS None. MEASUREMENTS AND MAIN RESULTS The health-related quality of life before ICU admission; at 15 days; and at 3, 6, 12, and 18 months was assessed with the EQ-5D-3L. In addition, the vital status was assessed at 24 months. The mean age of the subjects was 61.6 ± 14.3 years, 42.5% were female subjects and half were admitted after elective surgery. The mean Simplified Acute Physiology Score 3 was 47.4 ± 15.6. Survival at 12 and 18 months was 42.4% and 38.1%, respectively. The mean EQ-5D-3L utility measure before admission to the ICU was 0.47 ± 0.43, at 15 days it was 0.41 ± 0.44, at 90 days 0.56 ± 0.42, at 6 months 0.60 ± 0.41, at 12 months 0.67 ± 0.35, and at 18 months 0.67 ± 0.35. The probabilities for attaining 12 and 18 months of quality-adjusted survival were 30.1% and 19.1%, respectively. There were statistically significant differences in survival time and quality-adjusted life years according to all assessed baseline characteristics (ICU admission after elective surgery, emergency surgery, or medical admission; Simplified Acute Physiology Score 3; cancer extension; cancer status; previous surgery; previous chemotherapy; previous radiotherapy; performance status; and previous health-related quality of life). Only the previous health-related quality of life and performance status were associated with the health-related quality of life during the 18-month follow-up. CONCLUSIONS Long-term survival, health-related quality of life, and quality-adjusted life year expectancy of cancer patients admitted to the ICU are limited. Nevertheless, these clinical outcomes exhibit wide variability among patients and are associated with simple characteristics present at the time of ICU admission, which may help healthcare professionals estimate patients' prognoses.
Collapse
|
|
21
|
Agus A, Hulme C, Verghis RM, McDowell C, Jackson C, O'Kane CM, Laffey JG, McAuley DF. Simvastatin for patients with acute respiratory distress syndrome: long-term outcomes and cost-effectiveness from a randomised controlled trial. Crit Care 2017; 21:108. [PMID: 28511660 PMCID: PMC5434552 DOI: 10.1186/s13054-017-1695-0] [Citation(s) in RCA: 16] [Impact Index Per Article: 2.0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 01/19/2017] [Accepted: 05/02/2017] [Indexed: 11/17/2022] Open
Abstract
Background Simvastatin therapy for patients with acute respiratory distress syndrome (ARDS) has been shown to be safe and associated with minimal adverse effects, but it does not improve clinical outcomes. The aim of this research was to report on mortality and cost-effectiveness of simvastatin in patients with ARDS at 12 months. Methods This was a cost-utility analysis alongside a multicentre, double-blind, randomised controlled trial carried out in the UK and Ireland. Five hundred and forty intubated and mechanically ventilated patients with ARDS were randomly assigned (1:1) to receive once-daily simvastatin (at a dose of 80 mg) or identical placebo tablets enterally for up to 28 days. Results Mortality was lower in the simvastatin group (31.8%, 95% confidence interval (CI) 26.1–37.5) compared to the placebo group (37.3%, 95% CI 31.6–43.0) at 12 months, although this was not significant. Simvastatin was associated with statistically significant quality-adjusted life year (QALY) gain (incremental QALYs 0.064, 95% CI 0.002–0.127) compared to placebo. Simvastatin was also less costly (incremental total costs –£3601, 95% CI –8061 to 859). At a willingness-to-pay threshold of £20,000 per QALY, the probability of simvastatin being cost-effective was 99%. Sensitivity analyses indicated that the results were robust to changes in methodological assumptions with the probability of cost-effectiveness never dropping below 90%. Conclusion Simvastatin was found to be cost-effective for the treatment of ARDS, being associated with both a significant QALY gain and a cost saving. There was no significant reduction in mortality at 12 months, Trial registration ISRCTN, 88244364. Registered 26 November 2010. Electronic supplementary material The online version of this article (doi:10.1186/s13054-017-1695-0) contains supplementary material, which is available to authorized users.
Collapse
Affiliation(s)
- A Agus
- Northern Ireland Clinical Trials Unit, Elliot Dynes Building, The Royal Hospitals, Grosvenor Road, Belfast, BT12 6BA, UK.
| | - C Hulme
- Academic Unit of Health Economics, University of Leeds, Charles Thackrah Building, Clarendon Road, Leeds, LS2 9LJ, UK
| | - R M Verghis
- Northern Ireland Clinical Trials Unit, Elliot Dynes Building, The Royal Hospitals, Grosvenor Road, Belfast, BT12 6BA, UK.,Centre for Infection and Immunity, Queen's University of Belfast, Belfast, BT9 7AE, UK
| | - C McDowell
- Northern Ireland Clinical Trials Unit, Elliot Dynes Building, The Royal Hospitals, Grosvenor Road, Belfast, BT12 6BA, UK
| | - C Jackson
- Northern Ireland Clinical Trials Unit, Elliot Dynes Building, The Royal Hospitals, Grosvenor Road, Belfast, BT12 6BA, UK
| | - C M O'Kane
- Centre for Infection and Immunity, Queen's University of Belfast, Belfast, BT9 7AE, UK
| | - J G Laffey
- Department of Anaesthesia, School of Medicine, HRB Galway Clinical Research Facility, Clinical Sciences Institute, National University of Ireland, Galway, Ireland.,Department of Anesthesia, Centre for Critical Care Research, Keenan Research Centre for Biomedical Science, St. Michael's Hospital, University of Toronto, Toronto, Canada
| | - D F McAuley
- Northern Ireland Clinical Trials Unit, Elliot Dynes Building, The Royal Hospitals, Grosvenor Road, Belfast, BT12 6BA, UK.,Centre for Infection and Immunity, Queen's University of Belfast, Belfast, BT9 7AE, UK.,Regional Intensive Care Unit, The Royal Hospitals, Grosvenor Road, Belfast, BT12 6BA, UK
| |
Collapse
|
|
22
|
Hough CL. Beyond mortality or being mortal? Challenges in understanding and improving life after ARDS. Thorax 2017; 72:302-303. [DOI: 10.1136/thoraxjnl-2016-209082] [Citation(s) in RCA: 1] [Impact Index Per Article: 0.1] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 11/04/2022]
|
|
23
|
Abstract
OBJECTIVE To evaluate all published pediatric randomized controlled trials of patients with septic shock from any cause to examine the outcome measures used, the strengths and limitations of these measurements and whether the trial outcomes met feasibility criteria. DATA SOURCES We used a previously published database of pediatric critical care randomized controlled trials (PICUtrials.net) derived from searches of MEDLINE, EMBASE, LILACS, and CENTRAL. STUDY SELECTION We included randomized controlled trials of interventions to children admitted to a PICU with septic or dengue hemorrhagic shock which were published in English. DATA EXTRACTION Study characteristics and outcomes were retrieved by two independent reviewers with disagreement being resolved by a third reviewer. We defined feasibility as 1) recruitment of at least 90% of the targeted sample size and agreement of the observed outcome rate in the control group with the rate used for the sample size calculation to within 10% or 2) finding of a statistically significant difference in an interim or final analysis. DATA SYNTHESIS Nineteen of 321 identified articles were selected for review. Fourteen of 19 studies (74%) provided an a priori definition of their primary outcome measure in their "Methods section." Mortality rate was the most commonly reported primary outcome (8/14; 57%), followed by duration of shock (4/14; 29%) followed by organ failure (1/14; 7%). Only three of 19 included trials met feasibility criteria. CONCLUSIONS Our review found that use of mortality alone as a primary outcome in pediatric septic shock trials was associated with significant limitations and that long-term patient-centered outcomes were not used in this setting. Composite outcomes incorporating mortality and long-term outcomes should be explored for use in future pediatric septic shock trials.
Collapse
|
|
24
|
Sepsis: frontiers in supportive care, organisation and research. Intensive Care Med 2017; 43:496-508. [DOI: 10.1007/s00134-017-4677-4] [Citation(s) in RCA: 43] [Impact Index Per Article: 5.4] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 10/18/2016] [Accepted: 01/03/2017] [Indexed: 01/05/2023]
|
|
25
|
Dantan E, Foucher Y, Lorent M, Giral M, Tessier P. Optimal threshold estimator of a prognostic marker by maximizing a time-dependent expected utility function for a patient-centered stratified medicine. Stat Methods Med Res 2016; 27:1847-1859. [PMID: 28937334 DOI: 10.1177/0962280216671161] [Citation(s) in RCA: 2] [Impact Index Per Article: 0.2] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 11/16/2022]
Abstract
Defining thresholds of prognostic markers is essential for stratified medicine. Such thresholds are mostly estimated from purely statistical measures regardless of patient preferences potentially leading to unacceptable medical decisions. Quality-Adjusted Life-Years are a widely used preferences-based measure of health outcomes. We develop a time-dependent Quality-Adjusted Life-Years-based expected utility function for censored data that should be maximized to estimate an optimal threshold. We performed a simulation study to compare estimated thresholds when using the proposed expected utility approach and purely statistical estimators. Two applications illustrate the usefulness of the proposed methodology which was implemented in the R package ROCt ( www.divat.fr ). First, by reanalysing data of a randomized clinical trial comparing the efficacy of prednisone vs. placebo in patients with chronic liver cirrhosis, we demonstrate the utility of treating patients with a prothrombin level higher than 89%. Second, we reanalyze the data of an observational cohort of kidney transplant recipients: we conclude to the uselessness of the Kidney Transplant Failure Score to adapt the frequency of clinical visits. Applying such a patient-centered methodology may improve future transfer of novel prognostic scoring systems or markers in clinical practice.
Collapse
Affiliation(s)
- Etienne Dantan
- 1 EA 4275 SPHERE - methodS in Patient-centered outcomes & HEalth ResEarch, Nantes University, France
| | - Yohann Foucher
- 1 EA 4275 SPHERE - methodS in Patient-centered outcomes & HEalth ResEarch, Nantes University, France.,2 Nantes University Hospital, Nantes, France
| | - Marine Lorent
- 1 EA 4275 SPHERE - methodS in Patient-centered outcomes & HEalth ResEarch, Nantes University, France
| | - Magali Giral
- 3 Transplantation, Urology and Nephrology Institute (ITUN), CHU Nantes, RTRS Centaure, Nantes and Inserm U1064, Labex Transplantex, Nantes University, France.,4 Centre d'Investigation Clinique biothérapie, Nantes, France
| | - Philippe Tessier
- 1 EA 4275 SPHERE - methodS in Patient-centered outcomes & HEalth ResEarch, Nantes University, France
| |
Collapse
|
|
26
|
Kyeremanteng K, Wan C, D'Egidio G, Neilipovitz D. Approach to economic analysis in critical care. J Crit Care 2016; 36:92-96. [PMID: 27546754 DOI: 10.1016/j.jcrc.2016.07.004] [Citation(s) in RCA: 20] [Impact Index Per Article: 2.2] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 01/28/2016] [Revised: 06/06/2016] [Accepted: 07/01/2016] [Indexed: 12/30/2022]
Abstract
There are 4 general economic analyses used in health care: cost minimization, cost-benefit, cost-effectiveness, and cost utility. In this review, we provide an overview of each of these analyses and examine their appropriateness and effectiveness in assessing critical care costs. In the intensive care unit setting, it is particularly important to consider the patients' quality of life following the treatment of critical illness and to adopt a societal perspective when conducting economic analyses. Therefore, of the 4 economic analyses we cover, we recommend the use of cost-effectiveness and cost utility analyses.
Collapse
Affiliation(s)
| | - Cynthia Wan
- University of Ottawa, Ottawa, Ontario, Canada.
| | | | | |
Collapse
|
|
27
|
Aberegg S. Challenging orthodoxy in critical care trial design: physiological responsiveness. ANNALS OF TRANSLATIONAL MEDICINE 2016; 4:147. [PMID: 27162797 DOI: 10.21037/atm.2016.03.45] [Citation(s) in RCA: 3] [Impact Index Per Article: 0.3] [Reference Citation Analysis] [Track Full Text] [Subscribe] [Scholar Register] [Indexed: 01/04/2023]
|
|
28
|
Ost DE, Jimenez CA, Lei X, Cantor SB, Grosu HB, Lazarus DR, Faiz SA, Bashoura L, Shannon VR, Balachandran D, Noor L, Hashmi YB, Casal RF, Morice RC, Eapen GA. Quality-adjusted survival following treatment of malignant pleural effusions with indwelling pleural catheters. Chest 2014; 145:1347-1356. [PMID: 24480929 DOI: 10.1378/chest.13-1908] [Citation(s) in RCA: 43] [Impact Index Per Article: 3.9] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 12/30/2022] Open
Abstract
BACKGROUND Malignant pleural effusions (MPEs) are a frequent cause of dyspnea in patients with cancer. Although indwelling pleural catheters (IPCs) have been used since 1997, there are no studies of quality-adjusted survival following IPC placement. METHODS With a standardized algorithm, this prospective observational cohort study of patients with MPE treated with IPCs assessed global health-related quality of life using the SF-6D to calculate utilities. Quality-adjusted life days (QALDs) were calculated by integrating utilities over time. RESULTS A total of 266 patients were enrolled. Median quality-adjusted survival was 95.1 QALDs. Dyspnea improved significantly following IPC placement (P < .001), but utility increased only modestly. Patients who had chemotherapy or radiation after IPC placement (P < .001) and those who were more short of breath at baseline (P = .005) had greater improvements in utility. In a competing risk model, the 1-year cumulative incidence of events was death with IPC in place, 35.7%; IPC removal due to decreased drainage, 51.9%; and IPC removal due to complications, 7.3%. Recurrent MPE requiring repeat intervention occurred in 14% of patients whose IPC was removed. Recurrence was more common when IPC removal was due to complications (P = .04) or malfunction (P < .001) rather than to decreased drainage. CONCLUSIONS IPC placement has significant beneficial effects in selected patient populations. The determinants of quality-adjusted survival in patients with MPE are complex. Although dyspnea is one of them, receiving treatment after IPC placement is also important. Future research should use patient-centered outcomes in addition to time-to-event analysis. TRIAL REGISTRY ClinicalTrials.gov; No.: NCT01117740; URL: www.clinicaltrials.gov.
Collapse
Affiliation(s)
- David E Ost
- Department of Pulmonary Medicine, Department of Biostatistics, Section of Health Services Research, The University of Texas MD Anderson Cancer Center.
| | - Carlos A Jimenez
- Department of Pulmonary Medicine, Department of Biostatistics, Section of Health Services Research, The University of Texas MD Anderson Cancer Center
| | - Xiudong Lei
- Section of Pulmonary, Critical Care, and Sleep Medicine, Baylor College of Medicine
| | | | - Horiana B Grosu
- Department of Pulmonary Medicine, Department of Biostatistics, Section of Health Services Research, The University of Texas MD Anderson Cancer Center
| | | | - Saadia A Faiz
- Department of Pulmonary Medicine, Department of Biostatistics, Section of Health Services Research, The University of Texas MD Anderson Cancer Center
| | - Lara Bashoura
- Department of Pulmonary Medicine, Department of Biostatistics, Section of Health Services Research, The University of Texas MD Anderson Cancer Center
| | - Vickie R Shannon
- Department of Pulmonary Medicine, Department of Biostatistics, Section of Health Services Research, The University of Texas MD Anderson Cancer Center
| | - Dave Balachandran
- Department of Pulmonary Medicine, Department of Biostatistics, Section of Health Services Research, The University of Texas MD Anderson Cancer Center
| | - Lailla Noor
- Department of Pulmonary Medicine, Department of Biostatistics, Section of Health Services Research, The University of Texas MD Anderson Cancer Center
| | - Yousra B Hashmi
- Department of Pulmonary Medicine, Department of Biostatistics, Section of Health Services Research, The University of Texas MD Anderson Cancer Center
| | | | - Rodolfo C Morice
- Department of Pulmonary Medicine, Department of Biostatistics, Section of Health Services Research, The University of Texas MD Anderson Cancer Center
| | - George A Eapen
- Department of Pulmonary Medicine, Department of Biostatistics, Section of Health Services Research, The University of Texas MD Anderson Cancer Center
| |
Collapse
|
|
29
|
Yan L, Qing Y, Xingyi J, Hongbo Q. Etiologic Diagnosis and Clinical Treatment of Multiple Drug-Resistant Bacteria Infection in Elderly Patients with Stroke-Associated Pneumonia After Neurosurgery. Cell Biochem Biophys 2014; 71:731-4. [DOI: 10.1007/s12013-014-0256-2] [Citation(s) in RCA: 5] [Impact Index Per Article: 0.5] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 11/28/2022]
|
|
30
|
|
|
31
|
Harhay MO, Wagner J, Ratcliffe SJ, Bronheim RS, Gopal A, Green S, Cooney E, Mikkelsen ME, Kerlin MP, Small DS, Halpern SD. Outcomes and statistical power in adult critical care randomized trials. Am J Respir Crit Care Med 2014; 189:1469-78. [PMID: 24786714 DOI: 10.1164/rccm.201401-0056cp] [Citation(s) in RCA: 139] [Impact Index Per Article: 12.6] [Reference Citation Analysis] [Abstract] [Key Words] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 01/04/2023] Open
Abstract
RATIONALE Intensive care unit (ICU)-based randomized clinical trials (RCTs) among adult critically ill patients commonly fail to detect treatment benefits. OBJECTIVES Appraise the rates of success, outcomes used, statistical power, and design characteristics of published trials. METHODS One hundred forty-six ICU-based RCTs of diagnostic, therapeutic, or process/systems interventions published from January 2007 to May 2013 in 16 high-impact general or critical care journals were studied. MEASUREMENT AND MAIN RESULTS Of 146 RCTs, 54 (37%) were positive (i.e., the a priori hypothesis was found to be statistically significant). The most common primary outcomes were mortality (n = 40 trials), infection-related outcomes (n = 33), and ventilation-related outcomes (n = 30), with positive results found in 10, 58, and 43%, respectively. Statistical power was discussed in 135 RCTs (92%); 92 cited a rationale for their power parameters. Twenty trials failed to achieve at least 95% of their reported target sample size, including 11 that were stopped early due to insufficient accrual/logistical issues. Of 34 superiority RCTs comparing mortality between treatment arms, 13 (38%) accrued a sample size large enough to find an absolute mortality reduction of 10% or less. In 22 of these trials the observed control-arm mortality rate differed from the predicted rate by at least 7.5%. CONCLUSIONS ICU-based RCTs are commonly negative and powered to identify what appear to be unrealistic treatment effects, particularly when using mortality as the primary outcome. Additional concerns include a lack of standardized methods for assessing common outcomes, unclear justifications for statistical power calculations, insufficient patient accrual, and incorrect predictions of baseline event rates.
Collapse
Affiliation(s)
- Michael O Harhay
- 1 Department of Biostatistics and Epidemiology, Center for Clinical Epidemiology and Biostatistics
| | | | | | | | | | | | | | | | | | | | | |
Collapse
|
|
32
|
Andrei AC, Grady KL. Visualization and dynamics of multidimensional health-related quality-of-life-adjusted overall survival: a new analytic approach. Qual Life Res 2014; 23:1411-9. [DOI: 10.1007/s11136-013-0608-1] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Accepted: 12/12/2013] [Indexed: 11/28/2022]
|
|
33
|
|
|
34
|
|
|
35
|
Scales DC, Ferguson ND. Reply: Quality-adjusted Life Years or Composite Outcomes? Am J Respir Crit Care Med 2013; 188:622-3. [DOI: 10.1164/rccm.201303-0451le] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 11/16/2022] Open
Affiliation(s)
- Damon C. Scales
- The University of TorontoToronto, Ontario, CanadaandSunnybrook Health Science CentreToronto, Ontario, Canada
| | - Niall D. Ferguson
- The University of TorontoToronto, Ontario, CanadaandUniversity Health Network & Mount Sinai HospitalToronto, Ontario, Canada
| |
Collapse
|
|
36
|
Les outils de mesure de la qualité de vie sont-ils pertinents ? MEDECINE INTENSIVE REANIMATION 2013. [DOI: 10.1007/s13546-013-0714-7] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 10/26/2022]
|