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Validation of a patient-reported outcomes symptom measure for patients with nontransfusion-dependent thalassemia (NTDT-PRO© ).

ABSTRACT: This study demonstrates the quantitative characteristics of the first patient-reported outcome (PRO) tool developed for patients with nontransfusion-dependent β-thalassemia (NTDT), the NTDT-PRO© . A multicenter validation study was performed over 24 weeks, involving 48 patients from Italy, Lebanon, Greece, and Thailand. Most patients were female (68.8%), with a median age of 34.5 years (range, 18-52); 66.7% were diagnosed with β-thalassemia intermedia, and median time since diagnosis was 22 years (range, 0-43). The NTDT-PRO comprises 6 items across 2 domains (Tiredness/Weakness and Shortness of Breath [SoB]), and was valid and reliable, with good consistency. At baseline, most patients reported symptoms as present via the NTDT-PRO, and were highly compliant, ≥90% completing the NTDT-PRO tool. In a pairwise correlation analysis, all items were positively correlated. Correlations between NTDT-PRO and existing tools-36-Item Short Form Health Survey version 2 (SF-36v2) and Functional Assessment of Cancer Therapy-Anemia (FACT-An)-were assessed at weeks 1, 3, and 12; robust correlations were seen between SoB and SF-36v2-Vitality (rs = -0.53), and between SoB and Fact-An-Fatigue Experience (rs = -0.66) at week 1. Internal consistency was high for both Tiredness/Weakness (Cronbach alpha, 0.91) and SoB (Spearman-Brown coefficient, 0.78); intraclass correlation coefficients were high (Tiredness/Weakness, 0.88 and 0.97; SoB, 0.92 and 0.98), demonstrating stability. Further studies are required to fully support the validity of this tool, this study demonstrated the usefulness of the NTDT-PRO in the clinical setting and for longitudinal clinical research, particularly in trials where patient health-related quality of life is expected to change.

SUBMITTER: Taher A

PROVIDER: S-EPMC6587834 | biostudies-other | 2019 Feb

REPOSITORIES: biostudies-other

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Validation of a patient-reported outcomes symptom measure for patients with nontransfusion-dependent thalassemia (NTDT-PRO<sup>©</sup> ).

Taher Ali A Cappellini Maria Domenica MD Viprakasit Vip V Sutcharitchan Pranee P Mahmoud Dalia D Laadem Abderrahmane A Khan Anzalee A Gwaltney Chad C Harding Gale G Attie Kenneth K Zhang Xiaosha X Zou Jun J Pariseau Joseph J Henry Hu X X Kattamis Antonis A

American journal of hematology 20181126 2

This study demonstrates the quantitative characteristics of the first patient-reported outcome (PRO) tool developed for patients with nontransfusion-dependent β-thalassemia (NTDT), the NTDT-PRO<sup>©</sup> . A multicenter validation study was performed over 24 weeks, involving 48 patients from Italy, Lebanon, Greece, and Thailand. Most patients were female (68.8%), with a median age of 34.5 years (range, 18-52); 66.7% were diagnosed with β-thalassemia intermedia, and median time since diagnosis ...[more]

PMID: 30394570

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Project description:BackgroundPatient-reported outcome (PRO) measures developed and validated on patients with the currently defined phenotypes of chronic rhinosinusitis (CRS) are needed to support clinical trials in CRS.ObjectiveThis study developed and examined the initial reliability and validity of the CRS-PRO, a new PRO measure of CRS.MethodsInstrument development was performed through structured interviews and focus groups with clinical experts and 45 patients with CRS meeting current definitions of disease, 21 patients with CRS without nasal polyps (CRSsNP), and 24 patients with CRS with nasal polyps (CRSwNP) to identify items important to patients. Then another 50 patients (32 with CRSsNP and 18 with CRSwNP) with stable CRS symptoms were enrolled to evaluate the reliability of the instrument. Each patient completed the CRS-PRO, Sinonasal Outcome Test-22 (SNOT-22), and 4 Patient-Reported Outcome Measurement Information System short forms at the baseline visit and then at least 7 days later.ResultsAfter the development process, 21 items were identified from the conceptual domains of physical symptoms, sensory impairment, psychosocial effects, and life impact. Using the responses of the 50 patients with CRS, 21 draft items were further refined to 12 items by eliminating conceptually similar or highly correlated items or those with low mean symptom severity. The 12-item questionnaire was shown to have excellent internal consistency (Cronbach α, 0.86) and test-retest reliability with a high intraclass correlation coefficient (0.89) and Pearson's correlation (r = 0.82, P < .0001). The 12-item CRS-PRO correlated highly with the longer SNOT-22 (r = 0.83, P < .0001) demonstrating its concurrent validity. We also demonstrated validity and reliability in a separate analysis for patients with CRSsNP and CRSwNP.ConclusionThe CRS-PRO is a concise, valid, and reliable measure that was developed with extensive input from patients with CRS with current disease definitions.

Project description:OBJECTIVES:To assess the reliability, validity, and responsiveness of InFLUenza Patient-Reported Outcome (FLU-PRO©) scores for quantifying the presence and severity of influenza symptoms. METHODS:An observational prospective cohort study of adults (?18 years) with influenza-like illness in the United States, the United Kingdom, Mexico, and South America was conducted. Participants completed the 37-item draft FLU-PRO daily for up to 14 days. Item-level and factor analyses were used to remove items and determine factor structure. Reliability of the final tool was estimated using Cronbach ? and intraclass correlation coefficients (2-day reliability). Convergent and known-groups validity and responsiveness were assessed using global assessments of influenza severity and return to usual health. RESULTS:Of the 536 patients enrolled, 221 influenza-positive subjects comprised the analytical sample. The mean age of the patients was 40.7 years, 60.2% were women, and 59.7% were white. The final 32-item measure has six factors/domains (nose, throat, eyes, chest/respiratory, gastrointestinal, and body/systemic), with a higher order factor representing symptom severity overall (comparative fit index = 0.92; root mean square error of approximation = 0.06). Cronbach ? was high (total = 0.92; domain range = 0.71-0.87); test-retest reliability (intraclass correlation coefficient, day 1-day 2) was 0.83 for total scores and 0.57 to 0.79 for domains. Day 1 FLU-PRO domain and total scores were moderately to highly correlated (?0.30) with Patient Global Rating of Flu Severity (except nose and throat). Consistent with known-groups validity, scores differentiated severity groups on the basis of global rating (total: F = 57.2, P < 0.001; domains: F = 8.9-67.5, P < 0.001). Subjects reporting return to usual health showed significantly greater (P < 0.05) FLU-PRO score improvement by day 7 than did those who did not, suggesting score responsiveness. CONCLUSIONS:Results suggest that FLU-PRO scores are reliable, valid, and responsive to change in influenza-positive adults.

Project description:Background Validity is the ability of an instrument to measure what it claims to measure. It means the degree to which the empirical evidence supports the trustworthiness of interpretations based on the calculated scores. The hematological malignancy (HM) specific patient reported outcome measure (HM-PRO), is a newly developed instrument for use in daily clinical practice as well as in research. This study, provides the evidence for construct validity of the HM-PRO, specifically focusing on the convergent and divergent validity compared to the other established instruments used in hematology. Methods This validation study adopted a prospective cross-sectional design where a heterogeneous group of patients diagnosed with different HMs and different disease state were recruited. A total of 905 patients were recruited from seven secondary care hospitals in the UK and online through five patient organizations. Patients were asked to complete the HM-PRO and other cancer specific PRO’s, FACT-G and EORTC QLQ C-30. Data analysis was performed using IBM SPSS 23 statistical software. Results A total of 486 males (53.7%) and 419 females (46.3%), with a mean age of 64.3 (± 12.4) years and mean time since diagnosis of 4.6 ( ± 5.2) were recruited. The total score of Part A of the HM-PRO highly correlated with the five functional scales of the EORTC QLQ-C30 (Physical = ?0.71, Role = ?0.72, Emotional = ?0.64, Cognitive = ?0.58, Social = ?0.74—p < 0.001). With respect to correlation with FACT-G, the total score of Part A of the HM-PRO highly correlated with Physical (?0.74), Emotional (?0.57), Functional (?0.66) domains and overall score of FACT-G (?0.74). Similarly, the total score of Part B of the HM-PRO highly correlated with three symptoms scales of EORTC QLQ-C30 (Fatigue scale = ?0.74, Nausea and Vomiting = ?0.52, Pain = ?0.59—p < 0.001) and individual symptom items (Dyspnea = 0.51, Insomnia= 0.43, Appetite loss = 0.54—p < 0.001). Conclusion The construct validity evidence presented in this research is a testimony to the HM-PRO’s ability to measure HRQoL issues which it intends to measure. This is of utmost importance when a PRO is used in routine clinical practice so that the interpretation of the scores or response to an individual item is understood by the clinicians/nurses as intended by the patients.

Project description:Accurate documentation of patient symptoms in the electronic medical record (EMR) is important for high-quality patient care.To explore inconsistencies between patient self-report on an Eye Symptom Questionnaire (ESQ) and documentation in the EMR.This investigation was an observational study in comprehensive ophthalmology and cornea clinics at an academic institution among a convenience sample of 192 consecutive eligible patients, of whom 30 declined participation. Patients were recruited at the Kellogg Eye Center from October 1, 2015, to January 31, 2016. Patients were eligible to be included in the study if they were 18 years or older.Concordance of symptoms reported on an ESQ with data recorded in the EMR. Agreement of symptom report was analyzed using ? statistics and McNemar tests. Disagreement was defined as a negative symptom report or no mention of a symptom in the EMR for patients who reported moderate to severe symptoms on the ESQ. Logistic regression was used to investigate if patient factors, physician characteristics, or diagnoses were associated with the probability of disagreement for symptoms of blurry vision, pain or discomfort, and redness.A total of 162 patients (324 eyes) were included. The mean (SD) age of participants was 56.6 (19.4) years, 62.3% (101 of 162) were female, and 84.9% (135 of 159) were white. At the participant level, 33.8% (54 of 160) had discordant reporting of blurry vision between the ESQ and EMR. Likewise, documentation was discordant for reporting glare (48.1% [78 of 162]), pain or discomfort (26.5% [43 of 162]), and redness (24.7% [40 of 162]), with poor to fair agreement (? range, -0.02 to 0.42). Discordance of symptom reporting was more frequently characterized by positive reporting on the ESQ and lack of documentation in the EMR (Holm-adjusted McNemar P?<?.03 for 7 of 8 symptoms except for blurry vision [P?=?.59]). Return visits at which the patient reported blurry vision on the ESQ had increased odds of not reporting the symptom in the EMR compared with new visits (odds ratio, 5.25; 95% CI, 1.69-16.30; Holm-adjusted P?=?.045).Symptom reporting was inconsistent between patient self-report on an ESQ and documentation in the EMR, with symptoms more frequently recorded on a questionnaire. These results suggest that documentation of symptoms based on EMR data may not provide a comprehensive resource for clinical practice or "big data" research.

Dataset Information

Validation of a patient-reported outcomes symptom measure for patients with nontransfusion-dependent thalassemia (NTDT-PRO© ).

Publications

Validation of a patient-reported outcomes symptom measure for patients with nontransfusion-dependent thalassemia (NTDT-PRO<sup>©</sup> ).

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