Patient Related Outcome Measures最新文献

We briefly review the history of measuring perceptions of health and quality of life, followed by an examination of conceptual issues related to terminology that have led to potentially conflicting ontologies. Then, we discuss challenges posed by the lack of consensus on common meaning and the proliferation of measures. Next, we suggest a solution grounded in an ontology adopted by the National Institutes of Health (NIH) funded Patient-Reported Outcomes Measurement Information System (PROMIS) project. We conclude by discussing issues associated with mapping the PROMIS domain framework onto other familiar ontologies and recommend a way forward for PROMIS to provide a sustainable ontological structure to enable coherent common measurement.

Purpose: The hemophilia-specific health-related quality of life (HRQOL) questionnaire (Haemo-QOL-A) is validated for detecting QOL changes following standard therapy for hemophilia A, but has not been rigorously evaluated after gene therapy. This post hoc analysis evaluated the psychometric properties of Haemo-QOL-A in adult people with severe hemophilia A (PWSHA) receiving valoctocogene roxaparvovec (AAV5-hFVIII-SQ) in 2 clinical trials (phase 1/2, NCT02576795; phase 3, NCT03370913).

Patients and methods: Adult PWSHA (factor VIII levels ≤1 IU/dL) received 1 AAV5-hFVIII-SQ infusion (6×10¹³ vg/kg). Participants were assessed using the Haemo-QOL-A and the EuroQOL (EQ)-5D-5L and visual analog scale (VAS) questionnaires pre- and post-infusion. Psychometric analyses included convergent and discriminant validity, internal consistency, and reliability. Clinically important difference (CID) was estimated using 3-point change in EQ-5D-5L VAS as anchor.

Results: Haemo-QOL-A data were analyzed from 7 (phase 1/2, 3-year follow-up) and 16 participants (phase 3, 26-week analysis). Change in Haemo-QOL-A Total Scores correlated with EQ-5D-5L VAS score change at 26 weeks (Pearson's correlation 0.77). At 26 weeks, increased Haemo-QOL-A Physical Functioning was associated with decreased EQ-5D-5L Pain and Discomfort and decreased Anxiety and Depression (Spearman's Rank correlations -0.73 and -0.62, respectively, P <0.01). Internal consistency analysis showed good reliability for all domains (Cronbach's alpha >0.7) except Treatment Concern (Cronbach's alpha = 0.31). Anchor-based CID estimates were met for Haemo-QOL-A Total Score (≥5.5) and domain scores (≥6) for Consequences of Bleeding, Physical Functioning, Role Functioning, and Worry.

Conclusion: Our preliminary results suggest that the Haemo-QOL-A is a valid, reliable instrument for HRQOL assessment in PWSHA undergoing gene therapy. Future research should be undertaken to confirm these findings in a larger number of participants.

Introduction: Benign paroxysmal positional vertigo (BPPV) is the most frequently occurring peripheral vestibular disorder. Clinical practice guidelines (CPG) for BPPV exist; however, little is known about how affected patients perceive their condition is being managed. We aimed to leverage registry data to evaluate how adults who report BPPV are managed.

Material and methods: We retrospectively analyzed of data from 1,262 adults (58.4 ± 12.6 years old, 81.1% female, 91.1% White) who were enrolled in the Vestibular Disorders Association Registry from 2014 to 2020. The following patient-reported outcomes were analyzed by proportions for those who did and did not report BPPV: symptoms experienced, falls reported, diagnostics undertaken, interventions received (eg, canalith repositioning maneuvers [CRMs], medications), and responses to interventions.

Results: Of the 1,262 adults included, 26% reported being diagnosed with BPPV. Many adults who reported BPPV (83%) also endorsed receiving additional vestibular diagnoses or may have had atypical BPPV. Those with BPPV underwent magnetic resonance imaging and were prescribed medications more frequently than those without BPPV (76% vs 57% [χ²=36.51, p<0.001] and 85% vs 78% [χ²=5.60, p=0.018], respectively). Falls were experienced by similar proportions of adults with and without BPPV (55% vs 56% [χ²⁼=11.26, p=0.59]). Adults with BPPV received CRMs more often than those without BPPV (86% vs 48%, χ²=127.23, p<0.001). More registrants with BPPV also endorsed benefit from CRMs compared to those without BPPV (51% vs 12% [χ²=105.30, p<0.001]).

Discussion: In this registry, BPPV was often reported with other vestibular disorders. Healthcare utilization was higher than would be expected with care based on the CPG. The rates of falls in those with and without BPPV are higher than previously reported. Adults with BPPV reported significant differences in how their care is managed and their overall outcomes compared to those without BPPV.

Conclusion: Patient-reported outcomes provide useful information regarding the lived experience of adults with BPPV.

Purpose: The use of the Disability of the Arm, Shoulder and Hand (DASH) questionnaire and its shortened version, the QuickDASH, have been used to assess upper extremity function following a transradial percutaneous coronary intervention (TR-PCI). However, the use of these scores has not yet been validated for TR-PCI induced complications in the upper extremity. The aim of this study was to establish the validity of the DASH and the QuickDASH, for the assessment of upper extremity dysfunction following a transradial percutaneous coronary intervention (TR-PCI).

Patients and methods: This study was a diagnostic retrospective analysis of the ARCUS study, of whom 440 underwent TR-PCI and 62 control patients were treated via the transfemoral approach. All participants completed the DASH and QuickDASH questionnaire prior to the procedure and at each follow-up visit up to six months of follow-up. Receiver operating characteristics (ROC) were constructed to determine the validity of the questionnaires, using physical examinations to determine the occurrence of upper extremity dysfunction, according to the ARCUS study.

Results: At each follow-up moment, the area under the curve (AUC) showed a poor ability of the DASH and QuickDASH to discriminate between patients with and without upper extremity dysfunction (AUC: 0.565-0.586). There was no significant difference between the questionnaires (p > 0.05).

Conclusion: The DASH and QuickDASH questionnaires are both equally incapable of discriminating between patients with and without upper extremity dysfunction following a TR-PCI. Study results suggest that the DASH and QuickDASH questionnaires are incapable of discerning changes in upper extremity function as a result of procedural complications following a TR-PCI vs cardiac induced activity cessation.

Oral health problems are associated with poor quality of life, with the potential to cause functional, aesthetic, nutritional, and psychological difficulties, in addition to pain and suffering. Traditionally, dental treatment outcomes are measured using purely clinical parameters; however, this may be ineffective as these parameters cannot adequately capture the full impact of poor oral health on the patient, or their respective coping strategies. From this perspective, there are significant benefits when the patient's perception of their care is considered, and included in treatment planning and delivery. The impacts perceived by the patient on their treatment outcomes can be measured using patient-reported outcomes (PROS), or more specifically with dPROS, focused on dental patient-reported outcomes. Although there are some instruments available for measuring these outcomes in clinical trials, very little information is available for explaining the context in which these outcomes are considered, and also how to capture this information using appropriate instruments, specially in evidence-based dental practice. This article aims to review the literature, seeking to describe what has been considered about assessing patient's outcomes, as well as how to measure them, and explore the potential benefits of using dPROS in evidence-based prosthodontics and clinical care of partially and fully edentulous patients.

Introduction: Patient centred care is needed now more than ever in the treatment of opioid use disorder. Trials, policy makers, and service providers have most often used treatment retention and opioid urine screens as measures of treatment effectiveness. However, patients receiving medication for opioid use disorder treatment (MOUD) may prioritise the use of different ways to assess treatment success.

Objective: The aim of this review is to synthesize literature examining the self-reported goals patients would like to achieve in MOUD for opioid use disorder.

Methods: We searched MEDLINE, EMBASE, PsycINFO, Cumulative Index to Nursing and Allied Health Literature, Web of Science, Cochrane Library, Cochrane Clinical Trials Registry, the National Institutes for Health Clinical Trials Registry, and the WHO International Clinical Trials Registry Platform from inception until April 30th, 2021. No restrictions were placed on language, age, or type of MOUD. A qualitative synthesis is presented given that a meta-analysis was not possible.

Results: The search yielded a total of 21,082 records from which 8 met criteria for inclusion in the qualitative synthesis. We identified a total of 43 patient-reported treatment goals from the 8 studies. Twelve domains were created from the 43 goals reported. These domains cover a range of important areas for patients' goals related to living a normal life, physical health, mental health, treatment, and substance use specific areas.

Conclusion: This review highlights several patient goals that they would like to achieve during treatment for opioid use disorder that are not commonly considered as markers of treatment effectiveness. Goals related to health, living a normal life, and overall substance use concerns by patients should be taken into consideration by clinical trialists, researchers, policy makers, service providers, patients, and communities engaged in developing and tailoring treatment plans for opioid use disorder.

Systematic review registration: PROSPERO CRD42018095553.

Purpose: This review aims to provide an overview of the impact of TNFis biosimilars, with marketing authorization, in patient-reported outcome measures (PROMs) scores and explore how PROMs endpoints might add value in biosimilars uptake in RA patients.

Patients and methods: A comprehensive search of Medline, Scopus, Lilacs, and CINAHL databases was performed for papers published between January 2012 and December 2021. For inclusion, studies had to be prospective, published in a peer-reviewed journal, published in English or Spanish language; studies using PROMs as an outcome measure. After screening title and abstracts and assessing the remaining full texts fulfilling the inclusion criteria, 31 papers were used in this narrative review.

Results: PROMs were used as secondary outcomes in included studies. The most frequently employed domains to assess biosimilar efficacy include physical function, patient global assessment (PtGA), health-related quality of life (HRQoL), and fatigue. The results of randomized clinical trials uniformly showed that mean change in PROMs scores is comparable between biosimilar and reference biologic treatment groups. However, open-label and real-world studies revealed high rates of discontinuation of therapy, mainly for subjective worsening of disease activity or non-specific adverse events. Even without objective clinical evidence of inflammation, patients who are considered to have active disease (higher scores on PtGA) have higher discontinuation rates of biosimilars. The available information suggests that the nocebo effect is the most likely cause for the discontinuation of biosimilars.

Conclusion: There is scarce literature surrounding the impact of biosimilars in PROMs, especially in open-label studies. In real-life studies, biosimilars have a higher discontinuation rate than reference products. TNFis biosimilars treatment efficacy in RA depends on disease activity and other factors such as PtGA and fatigue. The nocebo effect is the best explanation for biosimilar's discontinuation.