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Patient-reported outcome measures in a pharmacokinetic study with sunitinib, a prospective cohort study.

ABSTRACT: PURPOSE:During treatment with tyrosine kinase inhibitors, such as sunitinib, patients experience treatment and/or disease-related symptoms. Although application of patient-reported outcome measures (PROMs) enhances early recognition of symptoms, early clinical trials are focused on symptom severity objectified by the Common Terminology Criteria for Adverse Events (CTCAE) in order to evaluate drug safety and to determine a personalized and/or safe dosage range. To gain insight into patient-reported symptoms in addition to healthcare professional-reported adverse events (AEs), a substudy was conducted in an ongoing pharmacokinetic-guided sunitinib dosing study. METHODS:In patients for whom sunitinib was considered standard therapy or patients with advanced/metastatic tumors for whom no standard therapy was available, patient-reported symptoms and well-being besides healthcare professional-reported AEs were assessed. RESULTS:Twenty-nine patients were included for analysis. Over 50% of them experienced a decreased well-being, caused by symptoms of mild and moderate intensity. Compared to healthcare professionals, all measured symptoms, with the exception of fatigue and vomiting, were reported statistically significantly more often by patients. CONCLUSIONS:Application of PROMs in early clinical trials on personalized or individualized oral targeted anticancer agents is feasible and enhances early recognition of symptom burden due to multiple CTCAE grade 1-2 AEs, just as pro-active symptom management and effect evaluation of interventions performed. Application of PROMs in these trials might be clinically relevant in obtaining dose-limiting toxicities.

SUBMITTER: Koldenhof JJ

PROVIDER: S-EPMC6018583 | biostudies-literature | 2018 Aug

REPOSITORIES: biostudies-literature

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Patient-reported outcome measures in a pharmacokinetic study with sunitinib, a prospective cohort study.

Koldenhof J J JJ Lankheet N A G NAG Steeghs N N Teunissen S C C M SCCM Teunissen S C C M SCCM Witteveen P O PO

Supportive care in cancer : official journal of the Multinational Association of Supportive Care in Cancer 20180221 8

<h4>Purpose</h4>During treatment with tyrosine kinase inhibitors, such as sunitinib, patients experience treatment and/or disease-related symptoms. Although application of patient-reported outcome measures (PROMs) enhances early recognition of symptoms, early clinical trials are focused on symptom severity objectified by the Common Terminology Criteria for Adverse Events (CTCAE) in order to evaluate drug safety and to determine a personalized and/or safe dosage range. To gain insight into patien ...[more]

PMID: 29464389

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Project description:IntroductionTo evaluate the quality of clinical practice, patient-reported outcome measures (PROMs) are important as certain questions could only be answered by the patient himself. PROMs help to get a better understanding what is meaningful to a patient and directly affects daily functioning. To move beyond traditional measures, we are interested in what matters to patients and developed this project. The aim of this article is to provide the protocol for our study collecting PROMs in daily medical practice from patients who undergo knee arthroplasty.Methods and analysisThis study is a single-site, observational, prospective cohort study. We will recruit patients scheduled for a knee arthroplasty in our medical office, situated in a private clinic. After signed informed consent, patients complete self-reported questionnaires before the surgery, after 4 months, 1 year, 2 years, 3 years, 4 years and 5 years. We will use the following PROMs: Knee injury and Osteoarthritis Outcome Score, Forgotten Joint Score, EuroQol five dimensions and satisfaction. Additionally, the surgeon will complete the objective Knee Society Score. Administration of the questionnaires will be electronically or paper-based. We will assess differences between preoperative and postoperative data with paired t-test for continuous variables and Wilcoxon signed-rank test for categorical variables. To assess subgroup differences, we will use unpaired t-test for continuous variables and Mann-Whitney U test for categorical variables. To assess possible presence of bias, we will conduct sensitivity analyses.Ethics and disseminationThe study has been reviewed and approved by the local ethics committee in Basel, Switzerland. Written informed consent will be obtained from all patients. We will disseminate the results of the study through peer-reviewed journals, national and international conference presentations and presentations to relevant stakeholders through appropriate channels.

Project description:BackgroundThe number of hip, knee and shoulder arthroplasties continues to rise worldwide. The Organization for Economic Cooperation and Development has launched an initiative (called PaRIS Initiative) for the systematic collection of Patient Reported Outcome Measures (PROMs) in patients undergoing elective hip and knee arthroplasty. The Rizzoli Orthopedic Institute (IOR) was selected as a pilot center for the launch of the Initiative in Italy given that IOR hosts the Registry of Orthopedic Prosthetic Implants (RIPO), a region-wide registry which collects joint implant data from all the hospitals in the Emilia-Romagna Region. In this specific geographic area information related to PROMs after joint replacement is unknown. This paper describes the protocol of a study (PaRIS-IOR) that aims to implement the collection of a set of PROMs within an existing implant registry in Italy. The study will also investigate the temporal trend of PROMs in relation to the type of prosthesis and the type of surgical intervention.MethodsThe PaRIS-IOR study is a prospective, single site, cohort study that consists of the administration of PROMs questionnaires to patients on the list for elective arthroplasty. The questionnaires will be administered to the study population within 30 days before surgery, and then at 6 and 12 months following surgery. The study population will consist of consecutive adult patients undergoing either hip, knee or shoulder arthroplasty. The collected data will be linked with those routinely collected by the RIPO in order to assess the temporal trend of PROMs in relation to the type of prosthesis and the type of surgical intervention.DiscussionThe PaRIS-IOR study could have important implications in targeting the factors influencing functional outcomes and quality of life reported by patients after hip, knee and shoulder arthroplasty, and will also represent the first systematic collection of PROMs related to arthroplasty in Italy.Trial registrationProtocol version (1.0) and trial registration data are available on the platform www.clinicaltrial.gov with the identifier NCT03790267 , first posted on December 31, 2018.

Project description:BackgroundThe International Consortium for Health Outcomes Measurement has published a set of patient-centered outcome measures for pregnancy and childbirth (PCB set), including patient-reported outcome measures (PROMs) and patient-reported experience measures (PREMs). To establish value-based pregnancy and childbirth care, the PCB set was implemented in the Netherlands, using the outcomes on the patient level for shared decision-making and on an aggregated level for quality improvement.ObjectiveThis study aims to report first outcomes, experiences, and practice insights of implementing the PCB set in clinical practice.MethodsIn total, 7 obstetric care networks across the Netherlands, each consisting of 1 or 2 hospitals and multiple community midwifery practices (ranging in number from 2 to 18), implemented the PROM and PREM domains of the PCB set as part of clinical routine. This observational study included all women participating in the clinical project. PROMs and PREMs were assessed with questionnaires at 5 time points: 2 during pregnancy and 3 post partum. Clinical threshold values (alerts) supported care professionals interpreting the answers, indicating possibly alarming outcomes per domain. Data collection took place from February 2020 to September 2021. Data analysis included missing (pattern) analysis, sum scores, alert rates, and sensitivity analysis.ResultsIn total, 1923 questionnaires were collected across the 5 time points: 816 (42.43%) at T1 (first trimester), 793 (41.23%) at T2 (early third trimester), 125 (6.5%) at T3 (maternity week), 170 (8.84%) at T4 (6 weeks post partum), and 19 (1%) at T5 (6 months post partum). Of these, 84% (1615/1923) were filled out completely. Missing items per domain ranged from 0% to 13%, with the highest missing rates for depression, pain with intercourse, and experience with pain relief at birth. No notable missing patterns were found. For the PROM domains, relatively high alert rates were found both in pregnancy and post partum for incontinence (469/1798, 26.08%), pain with intercourse (229/1005, 22.79%), breastfeeding self-efficacy (175/765, 22.88%), and mother-child bonding (122/288, 42.36%). Regarding the PREM domains, the highest alert rates were found for birth experience (37/170, 21.76%), shared decision-making (101/982, 10.29%), and discussing pain relief ante partum (310/793, 39.09%). Some domains showed very little clinical variation; for example, role of the mother and satisfaction with care.ConclusionsThe PCB set is a useful tool to assess patient-reported outcomes and experiences that need to be addressed over the whole course of pregnancy and childbirth. Our results provide opportunities to improve and personalize perinatal care. Furthermore, we could propose several recommendations regarding methods and timeline of measurements based on our findings. This study supports the implementation of the PCB set in clinical practice, thereby advancing the transformation toward patient-centered, value-based health care for pregnancy and childbirth.

Project description:BackgroundKnee replacement is a very effective and indispensable treatment option for end-stage knee arthritis, and the number of cases has been increasing worldwide. A replaced knee joint without patient joint awareness is thought to be the ultimate goal of artificial knees. Joint awareness reportedly correlates with patient satisfaction. Although numbness around a replaced knee is a minor but common problem, its effect on postoperative outcome is controversial. Joint awareness also is sensitive to subtle abnormalities of the joint, so it must be negatively affected by numbness. Although numbness is minor, it cannot be ignored to further improve knee replacement outcomes. This study investigated the relationship between patient-reported numbness and other patient-reported outcome measures (PROMs), including joint awareness, and kneeling. We developed a numbness score based on a 5-point Likert scale on frequency of numbness, with an intraclass correlation coefficient of 0.76 and higher scores indicating less numbness.MethodsThe numbness score, New Knee Society Score (KSS), Knee Injury and Osteoarthritis Outcome Score (KOOS), Forgotten Joint Score-12 (FJS-12), and other clinical and radiological data from 311 patients (394 primary knee replacements) were analyzed. Kneeling ability was evaluated by using kneeling-specific items in the KSS (KSS-Kneeling).ResultsNo numbness was found in 170 knees (43.1%), and some degree of numbness was found in the remaining 224 knees (56.9%). The numbness score showed weak-to-moderate correlations with KSS-Symptoms (r = 0.44), KSS-Satisfaction (r = 0.41), KSS-Activities (r = 0.29), and all KOOS subscales (r = 0.23-0.44), and FJS-12 (r = 0.42). Multiple regression analyses suggested that midline incision positively affected the numbness score over the anteromedial incision (p = 0.04) and that a better numbness score (p = 0.001), male sex (p < 0.0001), and better postoperative knee flexion angle (0.04) positively affected kneeling.ConclusionsThe numbness score positively correlated with PROMs and positively affected kneeling. Knee replacements performed via an anteromedial incision may be at higher risk for numbness.

Project description:Background and purposeThe use of patient reported outcomes measures (PROMs) may offer utility that are important for stroke survivors. This study assessed the PROMIS-10, which contains Mental health (MH) and Physical Health (PH) domains, with an additional five stroke specific questions. The aim of this study was to evaluate the association between the MH and PH measures following a stroke and pre-existing health conditions.MethodsA multicentre prospective cohort study at 19 hospital sites across England and Wales during 2019 was conducted. The association between each PROMIS-10 domain and demographic and health conditions were calculated using a multilevel multivariable linear and present the adjusted mean difference (aMD).ResultsThe study enrolled 549 stroke survivors within 14 days of the index event, 232 were women (42.3%) and with a mean age of 72.7 years (SD = 12.9, range 25 to 97). The MH domain was scored as poor in 3.9% of participants, and very good or excellent in almost a half (48.4%). In contrast the PH domain was scored as poor in 39.9%, compared to very good or excellent in 8.5%. The MH domain was associated with pre-existing diabetes (aMD = - 2.01; 95%CI -3.91, - 0.12; p = 0.04), previous stroke (aMD = - 3.62; 95%CI -5.86, - 1.39; p = 0.001), age (aMD = 0.07; 95%CI: 0.01, 0.14; p = 0.037), and female sex (aMD = 1.91; 95%CI 0.28, 3.54; p = 0.022). The PH domain was found to be associated with sex (female) (aMD = 2.09; 95%CI 0.54, 3.65; p = 0.008) and previous stroke (aMD = - 3.05; 95%CI -5.17, - 0.93; p = 0.005).ConclusionsAlmost half of stroke survivors reported poor PH using a PROM with less reporting poor MH. age, and sex were associated with both MH and PH domains, and additionally pre-exising diabetes and stroke were associated with poorer MH. Clinical management offers an opportunity to investigate and intervene to prevent long term poorer health in stroke survivors.

Project description:IntroductionThe impact of stroke-related impairment on activities of daily living may vary between patients, and can only be estimated by applying patient-reported outcome measures. The International Consortium for Health Outcome Measurement has developed a standard set of instruments that combine clinical and longitudinal patient-reported outcome measures for stroke. The present study was designed (1) to implement and evaluate the feasibility of the use of it as a consistent outcome measure in clinical routine at the stroke center of a German university hospital, (2) to characterize impairment in everyday life caused by stroke, and (3) to identify predictive factors associated with patient-relevant outcomes.MethodsWe plan to enroll 1040 consecutive patients with the diagnosis of acute ischemic stroke, transient ischemic attack, or intracerebral hemorrhage in a prospective observational study. Demographics, cardiovascular risk factors, and living situation are assessed at inpatient surveillance. At 90?days and 12?months after inclusion, follow-up assessments take place including the Patient-reported Outcomes Measurement Information System 10-Question Short Form (PROMIS-10), the Patient- Health Questionnaire-4, and the simplified modified Ranking Scale questionnaire. The acceptance and feasibility (1) will be assessed by a process evaluation through qualitative semi-structured interviews with clinical staff and patients and quantitative analyses of the data quality evaluating practicability, acceptance, adoption, and fidelity to protocol. The primary outcome of objective 2 and 3 is health-related quality of life measured with the PROMIS-10. Additional outcomes are depressive and anxiety symptoms and patient participation in their social roles. Patient-reported outcomes will be assessed in their longitudinal course using (generalized) mixed regressions. Exploratory descriptive and inference statistical analyses will be used to find patterns of patient characteristics and predictive factors of the outcome domains.PerspectiveThe results will describe and further establish the evaluation of stroke patients of a stroke center by standardized PROMs in everyday life.Trial registrationThe trial is registered at ClinicalTrials.gov (NCT03795948). Approval of the local ethics committee (Ethik-Kommission der Ärztekammer Hamburg) has been obtained.

Project description:BackgroundPatients with chronic kidney disease (CKD) face risks of not only end-stage kidney disease (ESKD), cardiovascular disease (CVD) and death, but also decline in kidney function, quality of life (QOL) and mental and physical well-being. This study describes the multidimensional trajectories of CKD using clinical events, kidney function and patient-reported outcome measures (PROMs). We hypothesized that more advanced CKD stages would associate with more rapid decline in each outcome.MethodsAmong 3939 participants enrolled in the Chronic Renal Insufficiency Cohort (CRIC) Study, we evaluated multidimensional disease trajectories by G- and A-stages of enrollment estimated glomerular filtration rate (eGFR) and albuminuria, respectively. These trajectories included clinical events (ESKD, CVD, heart failure and death), eGFR decline and PROMs [kidney disease QOL (KDQOL) burden, effects and symptoms questionnaires, as well as the 12-item short form mental and physical component summaries]. We also evaluated a group-based multitrajectory model to group participants on the basis of longitudinal PROMs and compared group assignments by enrollment G- and A-stage.ResultsThe mean participant age was 58 years, 45% were women, mean baseline eGFR was 44 mL/min/1.73 m2 and median urine albumin:creatinine ratio was 52 mg/g. The incidence of all clinical events was greater and eGFR decline was faster with more advanced G- and A-stages. While baseline KDQOL and physical component measures were lower with more advanced G- and A-stage of CKD, changes in PROMs were inconsistently related to the baseline CKD stage. Groups formed on PROM trajectories were fairly distinct from existing CKD staging (observed agreement 60.6%) and were associated with the risk of ESKD, CVD, heart failure and death.ConclusionsMore advanced baseline CKD stage was associated with a higher risk of clinical events and faster eGFR decline, and was only weakly related to changes in patient-reported metrics over time.

Project description:BACKGROUND:Retinoblastoma is a childhood retinal cancer with lifelong consequences such as vision loss and increased risk of second cancer. Patient-reported outcome measures (PROMs) are instruments that measure outcomes related to health directly reported by patients. The purpose of this study was to determine the scope, characteristics and quality of PROMs used in retinoblastoma and related fields of pediatric ophthalmology and pediatric oncology. METHODS:Databases MEDLINE and Embase were searched for studies in the English language that reported on PROMs used in retinoblastoma, pediatric oncology, or pediatric ophthalmology; grey literature and studies reporting on developmental PROM phases were excluded. PROMs were grouped by the construct measured and domains assessed, and classified as condition-specific or generic. A subsequent search was then conducted in MEDLINE and Embase for studies assessing measurement properties of the identified PROMs. PROMs with associated studies were assessed for their methodologic quality using the COnsensus-based standard for the Selection of health Measurement INstruments (COSMIN) strategy. RESULTS:Among 110 eligible studies uncovered by the database searches, 143 PROMs were identified: one retinoblastoma-specific, 56 ophthalmology- and 86 oncology-related. The most common construct measured was 'health-related quality of life' and the most common domain assessed was emotional well-being. Of the 143 PROMs, 100 had associated validation studies; the one retinoblastoma-specific PROM was not validated. Quality assessment revealed 34/100 PROMs received a score of sufficient quality in both subcategories of 'overall content validity'; 3/100 received a score of sufficient quality in both subcategories of 'internal structure'; 0/100 received a score of sufficient quality in all three subcategories of 'remaining measurement properties'. The Patient-Reported Outcome Measure Information System (PROMIS) Pediatric Profile-25 was the highest-scoring PROM identified, meeting COSMIN standards for 2/3 measurement property categories (and 5/7 subcategories). Eleven additional PROMs were identified which had sufficient scores in 1/3 measurement property categories (and 5/7 subcategories). CONCLUSION:The study identified several PROMs from the pediatric ophthalmology and pediatric oncology literature that could be relevant to the retinoblastoma population, but many have limits to their validation. Future development of a retinoblastoma-specific PROM, performed in partnership with retinoblastoma patients to support optimal content validity, could first focus on the selection and definition of the optimal construct to measure, followed potentially by adaptation and further validation of the relevant PROMs with strong methodologic quality identified in this study.

Dataset Information

Patient-reported outcome measures in a pharmacokinetic study with sunitinib, a prospective cohort study.

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Patient-reported outcome measures in a pharmacokinetic study with sunitinib, a prospective cohort study.

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