Patient-Reported Outcome Measures in Symptomatic, Non–Limb-Threatening Peripheral Artery Disease: A State-of-the-Art Review

Patient-reported outcome measures (PROMs) are health outcomes directly reported by the patient that can be used to measure the effect of disease and treatments on patient perceived well-being. This review summarizes current evidence regarding the validation of PROMs in people with symptomatic, nonlimb-threatening peripheral artery disease. A literature search was conducted to identify studies of symptomatic peripheral artery disease without limb-threatening ischemia that included PROMs and had sample sizes ≥25. PROMs were summarized along a continuum of validation using classical test theory framework and according to whether they fulfilled defined criteria for (1) content validity; (2) psychometric validation; and (3) further validation evidence base expansion. Of 2198 articles identified, 157 (7.1%) met inclusion criteria. Twenty-four PROMs in patients with symptomatic peripheral artery disease were reviewed. Among disease-specific PROMs, 8 of 15 had excellent reliability as measured by a Cronbach alpha ≥0.80. Based on established criteria for PROM responsiveness, 6 of 15 disease-specific PROMs demonstrated excellent sensitivity to change. Of these, the disease-specific peripheral artery questionnaire, vascular quality of life questionnaire, and walking impairment questionnaire met criteria for validation at each stage of the continuum. For generic (nondisease specific) PROMs, the European Quality of Life 5-Dimension and SF-36 had the most extensive evidence of validation. Evidence from this review can inform selection of PROMs aligned with scientific and clinical goals, given the variable degree of validation and potential complementary nature of the measures.

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Patient-Reported Outcome Measures (PROMs) in Pediatric Non-Malignant Hematology: A Systematic Review

Blood ◽

10.1182/blood-2019-127248 ◽

2019 ◽

Vol 134 (Supplement_1) ◽

pp. 2180-2180

Author(s):

Robert J Klaassen ◽

Julia Y. Kinahan ◽

Johann M. I. Graham ◽

Yamilée V. Hébert ◽

Katie O'Hearn

Keyword(s):

Quality Of Life ◽

Systematic Review ◽

Outcome Measures ◽

Well Being ◽

Patient Reported Outcome Measures ◽

Patient Reported Outcome ◽

Clinical Environment ◽

Patient Reported ◽

Disease Specific

Introduction: Patient reported outcome measures (PROMs) are questionnaires completed by patients or caregivers without interpretation by healthcare professionals. As such, they allow patient concerns about a variety of healthcare issues to be identified and addressed in an efficient and actionable manner. PROMs can be generic, with questions relevant to multiple disease groups or disease-specific, with questions targeting the symptoms, limitations, and feelings common to the disease group. This systematic review identified generic and disease-specific PROMs for monitoring symptoms and health-related quality of life (HRQoL) in 4 pediatric non-malignant hematologic disease groups: thalassemia, hemophilia, immune thrombocytopenia (ITP), and sickle cell disease (SCD). Methods: Databases (MEDLINE, Embase, HaPI, CINAHL, and PsycTESTS) were searched to identify publications that either validated or used PROMs as an outcome measure in the four disease groups. Articles were excluded when <30% of the population was pediatric (<18 years), when the study setting was inpatient, when the tool had not been validated, or when the article did not report the use of a PROM for monitoring symptoms or HRQoL. Notably, hemophilia records published prior to 2016 were not screened as a systematic review by Limperg et al. (2017) identified validated PROMs in the pediatric hemophilia population and was used to include relevant articles. Results: The search identified 1176 unique records, with 902 records remaining for title and abstract screening after removal of 274 hemophilia articles published prior to the systematic review. Including hemophilia records identified from the 2017 review, 217 articles met inclusion criteria incorporating 107 generic and 20 disease-specific PROMs. Of the generic tools, the most frequent categories identified include psychological well-being (26 tools), general quality of life (19 tools), and family impact (19 tools). The most frequently used tool was the PedsQL 4.0 Generic Core Scales (66 studies), appearing 33 times in SCD, 25 times in thalassemia, 5 times in ITP, and 3 times in hemophilia. Other commonly used generic tools include the Short Form Health Survey, Child Health Questionnaire, PROMIS Health Measures, and Child Behaviour Checklist (Table). Disease-specific tools identified in the review include the PedsQL SCD Module, Kids ITP Tool, Haemo-QoL, CHO-KLAT, and TranQol (Table). In addition, 10 studies reported on pain diaries and 9 of these studies were SCD focused, the other being hemophilia focused. Conclusion: This systematic review identified several generic and disease-specific PROMs that have been used in pediatric non-malignant hematology. Although generic tools have been used more frequently, many disease-specific tools have been validated and are available for use in the clinical environment. We are currently conducting focus groups with patients, parents, and clinicians to determine the optimal choice of tools for monitoring symptoms and HRQoL in the pediatric non-malignant clinical environment. Disclosures No relevant conflicts of interest to declare.

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Systematic Review of the Use of Patient Reported Outcome Measures in Studies of Electively-Managed Hand Conditions

The Journal of Hand Surgery (Asian-Pacific Volume) ◽

10.1142/s2424835519500425 ◽

2019 ◽

Vol 24 (03) ◽

pp. 329-341

Author(s):

Hawys Lloyd-Hughes ◽

Luke Geoghegan ◽

Jeremy Rodrigues ◽

Michele Peters ◽

David Beard ◽

...

Keyword(s):

Quality Of Life ◽

Outcome Measures ◽

Patient Reported Outcome Measures ◽

Patient Reported Outcome ◽

Inclusion Criteria ◽

Site Specific ◽

Patient Reported ◽

Generic Quality ◽

Disease Specific

Background: Electively-managed conditions account for over 100 000 inpatient surgeries a year in the English National Health Service alone, with further procedures in other regions of the UK, or performed on an outpatient basis. To quality assure this care and to conduct research, effective outcome measurement is critical. Traditional surgeon-centric outcome measures correlate poorly with hand function and are seldom important to patients. There has been an advent in the use of patient reported outcome measures (PROMs) in hand surgery although consensus of PROM choice appears to be lacking. This systematic review aimed to describe the use of relevant PROMs in clinical research of electively-managed hand conditions. Methods: A PRISMA-compliant methodology was used. A bespoke search strategy was developed in conjunction with a search strategist, and applied to Medline, EMBASE, CINAHL and PSYCHINFO from 1992 to June 2017. Pre-specified stepwise inclusion criteria were used to identify studies describing adult patients undergoing treatment for electively-managed hand conditions distal to the distal radius, with clinical outcomes measured using one or more PROMs. Results: Of 4554 results, 834 met inclusion criteria. PROMs identified included 9 disease-specific, 8 site-specific and 4 generic quality of life measures. Across all, the Disabilities of the Arm, Shoulder and Hand (DASH) was the most commonly used (overall frequency 41.0%). The most commonly reported disease-specific measure was the Boston Carpal Tunnel Questionnaire (overall frequency 23.0%). The most commonly reported generic quality of life of measure was the SF-36 (overall frequency 4%). Time-analysis demonstrated predominance of site-specific PROMs since the year 2000. Conclusions: Various PROMs have been used to study electively-managed hand conditions, with site-specific PROMs most popular. However, there appears to be limited consensus on choices. A future systematic evaluation of the published psychometric properties of identified PROMs may inform standardisation of measurement.

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