Importance Of Patient-reported Outcomes Research Articles

Predictors of health-related quality of life after non-catastrophic injury sustained in a road traffic crash.

Health-related quality of life (HRQoL) is an important patient-reported outcome that warrants greater attention in individuals who sustained a non-catastrophic injury in a road traffic crash. Additional robust data on HRQoL outcomes after a non-catastrophic injury are needed to effectively identify potential targets for studies of tertiary prevention of poor recovery after a crash. We assessed the association between non-catastrophic road traffic crash injuries and HRQoL and factors that independently affect long-term HRQoL. In this inception cohort study, injured participants (≥17years old) were identified from various sources including hospital emergency departments by a medical examination by a registered healthcare professional in New South Wales (Australia) and followed up after 12months. HRQoL was measured by the European Quality of Life-5 Dimensions (EQ-5D-3L) and Medical Outcomes Survey Short Form 12 items (SF-12). A range of socio-demographic, pre-injury health, psychological, and injury-related factors were considered potential predictors of HRQoL in regression analyses. Among 2019individuals identified, 1201 (59.5%) were followed up after 12months. HRQoL significantly improved between baseline and 12-month follow-up: EQ-5D-3L summary score (0.41-unit difference); SF-12 physical component summary score (PCS; 13.6-unit difference) and mental component summary score (MCS; 3.5-unit difference). Over 12months, HRQoL score was lower for people claiming compensation than others (P<0.0001). Key predictors of better 12-month EQ-5D-3L summary score and visual analogue scale score for pain were age, not claiming compensation, reduced body mass index, less pain severity, less pain-related disability, and less general psychological distress. Significant predictors of SF-12 PCS score were injury to the neck (P=0.02) or head or face (P=0.01), being a driver or passenger at the time of the crash (P<0.0001), hospital admission (P<0.0001) and pain severity (P<0.0001). Baseline variables associated with 12-month SF-12 MCS scores were head or face injury (P=0.02), pre-injury health (P=0.04), pre-injury psychological conditions (P=0.04), trauma-related distress (P=0.0002) and general psychological distress (P<0.0001). A wide spectrum of biopsychosocial factors contribute to HRQoL after a road traffic crash injury. These epidemiological data are potentially important because they could identify potential targets for studies of tertiary prevention of persistently poor HRQoL after such an injury.

A Patient-Reported Outcomes Analysis Of Lanreotide In The Treatment Of NETs Patients With Carcinoid Syndrome: Evidence From The ELECT Trial.

PurposeThe purpose of this analysis of patient-reported outcomes from the ELECT (Evaluation of Lanreotide Depot/Autogel Efficacy and Safety as a Carcinoid Syndrome Treatment) trial (NCT00774930) was to explore the effect of lanreotide on symptoms of carcinoid syndrome. Specifically, this post hoc analysis was designed to identify the most important patient-reported outcomes for patients in ELECT.MethodsThe post hoc analysis of ELECT, a placebo-controlled study of lanreotide in patients with neuroendocrine tumors, evaluated patient-reported outcomes during the double-blind phase of the trial, specifically daily diarrhea and flushing symptoms, octreotide rescue use, and the EORTC QLQ-C30 and QLQ-GINET21 questionnaires at baseline and week 12. Principal component (PC) analysis was applied on baseline data to identify independent variable clusters and clinically meaningful summary measures that highly correlated to these PCs. From those, the minimum clinical important differences were derived so to perform a responder analysis.ResultsThe three largest PCs captured 42.9% of the variation among baseline variables. The C30 summary score (C30-SS), diarrhea burden, and flushing burden were highly correlated with PC1, PC2, and PC3, respectively. Lanreotide patients were more likely to experience an improvement on the C30-SS (risk ratio [RR] 2.42; P=0.023), diarrhea burden (RR 2.85; P=0.005), and flushing burden (RR 1.39; P=0.31) compared to placebo patients. Lanreotide-treated patients have a higher probability of being a responder on at least one of the three domains of C30-SS, diarrhea burden, or flushing burden compared to placebo patients (RR 1.48; P=0.06).ConclusionThe higher response rates in the diarrhea burden are consistent with the previously reported effects of lanreotide on octreotide rescue medication use, while the findings of a greater efficacy of lanreotide vs placebo in the quality-of-life domains represent a novel aspect in the benefits of lanreotide.Trial registrationClinicalTrials.gov identifier: NCT00774930.

Determinants of self-rated health in older adults before and 3 months after an emergency department visit: a prospective study.

Self-rated health (SRH) is an important patient-reported outcome, but little is known about SRH after a visit to the emergency department (ED). We investigated the determinants of decline in SRH during 3 months after an ED visit in older patients. This was a multicenter prospective cohort study including acutely presenting older ( ≥ 70 years) patients in the ED (the Netherlands). Patients were asked to self-rate their health between 0 and 10. The main outcome was a decline in SRH defined as a transition of a SRH of at least 6 to a SRH of less than 6, 3 months after the patient's visit to the ED. Three months after the ED visit, 870 (71.4%) patients had a stable SRH and 209 (11.5%) patients declined in SRH. Independent predictors with a decline in SRH were: male gender (OR 1.83) living alone (OR 1.56), living in residential care or nursing home (OR 2.75), number of different medications (OR 1.08), using a walking device (OR 1.70), and the Katz-ADL score (OR 1.22). Patients with functional decline 3 months after an ED visit show a steeper decline in the mean SRH (0.68 points) than patients with no functional decline (0.12 points, P < 0.001). Decline in SRH after an ED visit in older patients is at least partly dependent on factors of functional capacity and functional decline. Preventive interventions to maintain functional status may be the solution to maintain SRH, but more research is needed to further improve and firmly establish the clinical usability of these findings.

Development of a Patient-reported Experience and Outcome Measures in Pediatric Patients Undergoing Bowel Management for Constipation and Fecal Incontinence.

The aim of this review is develop a reliable and valid questionnaire that assesses patient-reported experience measures (PREMs) and patient-reported outcome measures (PROMs) of caregivers, families, and patients with severe constipation and fecal incontinence who failed conservative treatments and require a formal bowel management program (BMP). A 5-step iterative process was utilized to ensure the reliability and validity of the final instrument. Parents or guardians of patients undergoing our week-long BMP were first asked 20 open-ended questions aimed at understanding the challenges in managing their child's condition. Responses were transformed into an initial 41-item survey constituted of 2 constructs relating to PREMs (24 items) and PROMs (17 items). Survey items were then administered to 359 parents and guardians undergoing BMP. Cognitive interviews were performed with 20 families to assess face validity and to further refine the survey. Instrument readability and reliability was assessed by Flesch-Kincaid and Crohnbach α analyses. Items that weakly correlated were deleted to yield a final instrument that was both valid and reliable. A 33-statement survey was developed that encompasses important physical and emotional health concerns, quality of life, treatment success, financial considerations, schooling, parental employment, and social concerns. The final instrument, the Patient-Reported Experience and Outcome Measure in a Bowel Management Program (PREOM-BMP), is divided into 2 separate constructs that assess important patient-reported experience (22 items) and outcome (11 items) measures. Reliability analyses on the final instrument yielded a Crohnbach α of 0.925. The PREOM-BMP offers physicians and nursing professionals a brief and valid tool to measure the impact of a bowel management program on both the child and the family unit.

Determination of Multiple Sclerosis Indicators for Value-Based Contracting Using the Delphi Method.

Value-based contracts link medication payments to performance measures with the ultimate goal of lowering costs while improving patient outcomes. Previous multiple sclerosis (MS) value-based contracts have focused on indicators easily collected from claims or electronic health record data as their value-based outcomes, even though numerous other MS clinical indicators of interest exist. Uncertainty remains regarding which MS indicators are most meaningful to all stakeholders affected by a value-based contract. To identify meaningful MS indicators among key stakeholders for the purpose of informing a value-based contract for MS medications. Using a modified Delphi method, we surveyed 26 diverse stakeholders, including 8 patients and caregivers; 9 providers (neurologists, nurses, physician assistants, and specialty pharmacists); 2 pharmaceutical company representatives; 5 payers; and 2 pharmacy benefits managers. A list of 12 MS indicators was created from subject matter expert consultation and a literature review. All stakeholders reported on the meaningfulness and value of these 12 indicators through a 5-point Likert scale and forced selection of the 3 most meaningful indicators. All nonpatient stakeholders were additionally surveyed on collection feasibility of the same 12 indicators using a 5-point Likert scale. We defined consensus as ≥ 75% agreement on the meaningfulness and feasibility of an indicator (Likert scores 4 or 5). We performed a Fisher's exact test to assess differences between nonpatient and patient stakeholder rankings of indicators. Consensus was reached for at least 1 indicator for all questions after 2 rounds. "Worsening physical disability" and "functional impairment" achieved 92% agreement on a Likert-scale question assessing indicator value, and 100% of participants selected "worsening physical disability" when asked to choose the 3 most meaningful indicators. "MS flares requiring an emergency department visit" and "MS flares requiring inpatient admission" were rated as the 2 most feasibly collected indicators (both received 89% agreement). Using the Delphi method, we identified that disability and functional impairment are meaningful MS indicators to diverse stakeholders. These findings support the incorporation of important patient-reported outcomes into value-based contracts for MS medications. This study was supported by a grant from Express Scripts Holding Company, which provided research funding to the UPMC Center for Value-Based Pharmacy Initiatives for work on this study. Swart, Neilson, Good, and Parekh are employed by the UPMC Center for Value-Based Pharmacy Initiatives. Manolis is the Chief Pharmacy Officer of UPMC Health Plan, and Shrank was the Chief Medical Officer of UPMC Insurance Services Division at the time of this study. Henderson is employed by Express Scripts Holding Company.

Change, predictors and correlates of weight- and health-related quality of life in adolescents 2-years following bariatric surgery

Background/Objectives:Weight-related quality of life (WRQOL) and generic health-related quality of life (HRQOL) have been identified as important patient reported outcomes for obesity treatment and outcome research. This study evaluated patterns of WRQOL and HRQOL outcomes for adolescents at 24-months post-bariatric surgery relative to a non-surgical comparator sample of youth with severe obesity, and examined potential weight-based (e.g., BMI, weight dissatisfaction) and psychosocial predictors and correlates of these outcomes.Subjects/Methods:Multi-site data from 139 adolescents undergoing bariatric surgery (Mage=16.9; 79.9% female, 66.2% White; MBody Mass Index [BMI]= 51.5kg/m2) and 83 comparators (Mage=16.1; 81.9 % female, 54.2% White; MBMI= 46.9kg/m2) were collected at pre-surgery/baseline, 6-, 12-, and 24-months post-surgery/baseline with high participation rates across time points (>85%). Self-reports with standardized measures of WRQOL/HRQOL as well as predictors/covariates (e.g., weight dissatisfaction, social support, peer victimization, family dysfunction, loss of control eating, self-worth, and internalizing symptoms) were obtained. Growth curve models using structural equation modeling examined WRQOL/HRQOL over time and linear regressions examined predictors and correlates of WRQOL/HRQOL outcomes.Results:Significant improvement in WRQOL and Physical HRQOL, particularly in the first post-operative year with a leveling off subsequently, was found for the surgical group relative to comparators, but with no significant Mental HRQOL change. At 24 months, the surgical group had signficantly greater WRQOL/HRQOL across most subscales. Within the surgical group at 24 months, weight-based variables were signficantly associated with WRQOL and Physical HRQOL, but not Mental HRQOL. Mental HRQOL was associated with greater internalizing symptoms and loss of control eating.Conclusions:For adolescents undergoing bariatric surgery, most clinically meaningful changes in WRQOL and Physical HRQOL occurred early post-operatively, with weight-based variables as the primary drivers of 24-month levels. In contrast, expectations for Mental HRQOL improvement following surgery should be tempered, with 24-month levels significantly associated with psychosocial rather than weight-based correlates.

Outcomes of treatment of foot and ankle fractures: which are important to the patient?

Background: Fractures of the ankle and foot are common and may be associated with long-term disabilities. Previous studies have examined functional status as an important patient-reported outcome, but little is known about patient expectations and satisfaction after their treatment. The aim of this study was to assess which outcomes are important to patients with an ankle or calcaneus fracture and to assess which background and fracture characteristics might be associated with these outcomes. Methods: A cross-sectional survey among 335 patients (response rate: 58%, n = 194) treated for a Weber B/C or calcaneus fracture, was performed. Patients were asked to identify and rank 5 treatment outcomes (out of 22 outcomes) that were the most important to them. The weighted importance of each outcome was calculated and averaged on group level. Stepwise multivariable regression analyses were performed to assess patient characteristics that are associated with patients’ preferences for the most important outcomes. Results: “Self-sufficiency” was reported most frequently (in 50/194; 25.8%) as the most important outcome, followed by “walking” and “complete recovery”. The top 5 of most important outcomes differed somewhat between patient subgroups. The weighted importance of the outcomes was associated with various patient characteristics. Conclusions: Self-sufficiency, walking and complete recovery, are generally valued most by patients with an ankle or calcaneal fracture. The surgeon should be aware of this in order to manage any pre-operative discrepancies between surgeons’ and patients’ expectations. Furthermore, evaluation of care should become more focussed on the outcome parameters that matter to the patient, as part of enhanced approaches to providing person-centered healthcare.

Patient-Reported Outcomes in AtrialFibrillation Research: Results of a Clinicaltrials.gov Analysis.

This study sought to determine how frequently patient-reported outcomes (PROs) are collected in registered clinical studies of atrial fibrillation (AF). Improving symptom burden and quality of life are important goals in the treatment of AF and are best measured with PROs. Data from Clincaltrials.gov were studied to identify PROs in AF studies. All studies reporting AF as the disease condition were included, and PROs were identified by search terms within the outcome measures field. Generic and AF-specific PROs were identified and assessed by study type and year. Clinicaltrials.gov reporting was compared with published reports of linked studies in PubMed. From 1999 to 2018, 1,709 studies including AF patients were posted; 238 studies (14%) included PROs. Collection of PROs was reported in 22% of trials (n= 83 of 386) studying procedural interventions and 11% of all Phase 3studies (n= 18 of 168). Among the 238 studies with PROs, most described "quality of life" (n= 194 [82%]), and most(n= 198 [83%]) included only generic (not AF-specific) PROs. Only 17% of studies (n= 40) reporting PROs specified a previously published AF-specific tool, most commonly the AFEQT (Atrial Fibrillation Effect on QualiTy-of-life) form (n= 20 [8.4%]). Among the available PubMed citations of 391 studies, 74 studies (19%) described collecting a specific PRO tool (n= 29 [7.4%]) for an AF-specific PRO. Despite increased emphasis on the importance of PROs in AF, a minority of registered clinical trialsreported collecting PROs, with very few using validated, AF-specific PROs. Improving outcomes that are mostimportant to patients will necessitate increased emphasis on these PROs in pivotal clinical studies.

Responsiveness of the PROMIS and its Concurrent Validity with Other Region- and Condition-specific PROMs in Patients Undergoing Carpal Tunnel Release.

The Patient-reported Outcome Measurement Information System (PROMIS) continues to be an important universal patient-reported outcomes measure (PROM) in orthopaedic surgery. However, there is concern about the performance of the PROMIS as a general health questionnaire in hand surgery compared with the performance of region- and condition-specific PROMs such as the Michigan Hand Questionnaire (MHQ) and the Boston Carpal Tunnel Questionnaire (BCTQ), respectively. To ensure that PROMIS domains capture patient-reported outcomes to the same degree as region- and condition-specific PROMs do, comparing PROM performance is necessary. (1) Which PROMs demonstrate high responsiveness among patients undergoing carpal tunnel release (CTR)? (2) Which of the PROMIS domains (Physical Function [PF], Upper Extremity [UE], and Pain Interference [PI]) demonstrate concurrent validity with the HHQ and BCTQ domains? In this prospective study, between November 2014 and October 2016, patients with carpal tunnel syndrome visiting a single surgeon who elected to undergo CTR completed the BCTQ, MHQ, and PROMIS UE, PF, and PI domains at each visit. A total of 101 patients agreed to participate. Of these, 31 patients (31%) did not return for a followup visit at least 6 weeks after CTR and were excluded, leaving a final sample of 70 patients (69%). We compared the PROMIS against region- and condition-specific PROMs in terms of responsiveness and concurrent validity. Responsiveness was determined using Cohen's d or the effect-size index (ESI). The larger the absolute value of the ESI, the greater the effect size. Using the ESI allows surgeons to better quantify the impact of CTR, with a medium ESI (that is, 0.5) representing a visible clinical change to a careful observer. Concurrent validity was determined using Spearman's correlation coefficient with correlation strengths categorized as excellent (> 0.7), excellent-good (0.61-0.70), good (0.4-0.6), and poor (< 0.4). Significance was set a priori at p < 0.05. Among PROMIS domains, the PI demonstrated the best responsiveness (ESI = 0.74; 95% CI, 0.39-1.08), followed by the UE (ESI = -0.66; 95% CI, -1.00 to -0.31). For the MHQ, the Satisfaction domain had the largest effect size (ESI = -1.48; 95% CI, -1.85 to -1.09), while for the BCTQ, the Symptom Severity domain had the best responsiveness (ESI = 1.54; 95% CI, 1.14-1.91). The PROMIS UE and PI domains demonstrated excellent-good to excellent correlations to the total MHQ and BCTQ-Functional Status scores (preoperative UE to MHQ: ρ = 0.68; PI to MHQ: ρ = 0.74; UE to BCTQ-Functional Status: ρ = 0.74; PI to BCTQ-Functional Status: ρ = 0.67; all p < 0.001), while the PROMIS PF demonstrated poor correlations with the same domains (preoperative PF to MHQ; ρ = 0.33; UE to BCTQ-Functional Status: ρ = 0.39; both p < 0.01). The PROMIS UE and PI domains demonstrated slightly worse responsiveness than the MHQ and BCTQ domains that was nonetheless acceptable. The PROMIS PF domain was unresponsive. All three PROMIS domains correlated with the MHQ and BCTQ, but the PROMIS UE and PI domains had notably stronger correlations to the MHQ and BCTQ domains than the PF domain did. We feel that the PROMIS UE and PI can be used to evaluate the clinical outcomes of patients undergoing CTR, while also providing more robust insight into overall health status because they are general PROMs. However, we do not recommend the PROMIS PF for evaluating patients undergoing CTR. Level II, diagnostic study.

AMCP Partnership Forum: Building the Foundation for Patient-Reported Outcomes-Infrastructure and Methodologies.

Section 3002 of the 21st Century Cures Act, which was signed into law in December 2016, requires the FDA to develop one or more sets of new guidances regarding the collection of patient experience data. To explore how patient-reported outcomes (PROs) can support value-based care and discuss challenges to using PROs more widely, the Academy of Managed Care Pharmacy (AMCP) convened a stakeholder forum in 2017 on PROs and the role they can play in defining the value of health care interventions. This forum achieved consensus on the importance of PROs in improving patient care through implementing value-based payment models and the need for strong organizational systems to fully adopt and use PROs in health care decision making. To explore strategies to build on these findings and implement them in practice, AMCP convened a second forum on October 25, 2018, in Orlando, FL, to focus on identifying specific methods and infrastructure to support the ideal use of PROs in practice. In light of the FDA's plans to propose new guidance for incorporating PROs in the regulatory process, this forum focused on the following: (a) describing the current state of using PROs for FDA regulatory and value-based coverage decisions; (b) defining a process for collecting and sharing PROs with the FDA and managed care organizations for value-based decisions; (c) identifying the necessary infrastructure needed to support the ideal use of PROs; and (d) identifying the health information technology strategies needed to support PRO use across managed care stakeholders. Strategies for the development and implementation of PROs were discussed throughout the forum, including a focus on appropriate activities to address the needs of patients, health care providers, managed care organizations, and life science companies and researchers. Health care leaders representing health plans, integrated delivery systems, pharmacy benefit managers, employers, national associations of health care professionals, patient advocacy organizations, health infrastructure technology companies and biopharmaceutical companies participated in the forum. The AMCP forum was supported in collaboration with Allergan, Amgen, Boehringer Ingelheim, Bristol-Myers Squibb, Merck, Novo Nordisk, PhRMA, Sanofi, Takeda, and Xcenda. DISCLOSURES: The AMCP Partnership Forum and the development of the proceedings document were supported by Allergan, Amgen, Boehringer Ingelheim, Bristol-Myers Squibb, Merck, Novo Nordisk, PhRMA, Sanofi, Takeda, and Xcenda.

HSR19-109: The Healthy Days Measure in a Metastatic Castration-Resistant Prostate Cancer Population Treated With Novel Oral Therapies

Background: Health-related quality of life (HRQoL) is an important patient-reported outcome to assess in patients with prostate cancer (PC). A variety of general and disease-specific measures have been developed, but there is a lack of evidence on performance of the Centers for Disease Control and Prevention (CDC) Healthy Days (HDs) measure in a population with metastatic disease relative to other validated scales. Objective: To report and understand general HRQoL using the HDs instrument in patients with metastatic PC taking novel oral therapies and compare results to validated scales. Methods: Patients currently enrolled in a Medicare Advantage or Prescription Drug plan treated with enzalutamide or abiraterone acetate were eligible for inclusion. Patients undertook a telephone-based survey including 2 HRQoL questionnaires (Short-Form 36, version 2 [SF-36] survey and the CDC HDs measures), the Service Satisfaction with Cancer Care (SCA) instrument, and the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue). Higher scores indicated higher HRQoL on all surveys, except the HDs scale, where higher numbers indicated lower HRQoL (ie, unhealthy days). Pearson correlations with SF-36 and HDs measures and SCA and FACIT-Fatigue were assessed. Results: 1,745 patients were eligible to participate; 269 completed the survey and were included in the analysis. The mean (standard deviation) age was 74.4 (±7.3) years and 82% were Caucasian. Patients had lower physical HRQoL and a higher number of physically unhealthy days, compared to mental health impacts on both scales. The mean Physical Component Summary (PCS) and Mental Component Summary (MCS) norm-based scores for the SF-36 were 39.9 (±10.6) and 53.4 (±10.4). The mean number of physically and mentally unhealthy days reported in a 30-day period were 9.2 (±11.3) and 3.9 (±7.7), respectively. The unadjusted correlations between scores were all statistically significant (P&lt;.05). Conclusions: The SF-36 and HDs measures both indicated physical health was impacted more than mental health in these patients. The correlations with both HRQoL measures and the other scales were similar in terms of magnitude and significance. This indicates that the HDs instrument may be a useful measure for assessment of general HRQoL in patients with metastatic PC and provides an understanding of general HRQoL in this population that can be utilized to identify opportunities for enhancing patient-centered care.

Quality of Life and not Health Status Improves After Major Amputation in the Elderly Critical Limb Ischaemia Patient

A patient-oriented appraisal of treatment has become extremely important, particularly in elderly patients with critical limb ischaemia (CLI). Quality of life (QoL) is an important patient-reported outcome in vascular surgery. Frequently, the physical domain of QoL questionnaires represents an 'objective' evaluation of performing activities, which is expected to be impaired after major limb amputation. However, an objective appraisal of physical function is an assessment of health status (HS) and not of QoL. Little is known about the subjective appraisal of physical health (QoL). The goal of this study was to evaluate, prospectively, QoL in relation to HS in elderly CLI patients undergoing major limb amputation. Patients suffering from CLI aged 70 years or older were included in a prospective observational cohort study with a follow-up period of 1 year. Patients were divided according to having had an amputation or not. The World Health Organization Quality Of Life-BREF (WHOQOL-BREF) was used to asses QoL. The 12-Item Short Form Health Survey (SF-12) was used to measure HS. These self-reported questionnaires were completed five times during follow-up. Two-hundred patients were included of whom 46 underwent a major limb amputation within one year. Amputees had a statistically significant improvement of their physical QoL after six months (14.0 vs. 9.0 (95% CI -7.84;-1.45),p=0.005) and after a one-year follow-up (14.0 vs. 9.0 (95% CI -9.58;-1.46),p=0.008). They did not however show any statistically significant difference in HS. For non-amputees, both physical QoL and HS improved. An instant statistically significant improvement of the physical QoL appeared 1 week after inclusion (12.0 vs. 10.9 (95% CI -1.57;-0.63),p<0.001). Similarly, statistically significant improvement in the physical HS first occurred at 1 week follow-up (29.0 vs. 28.9 (95% CI -5.78;-2.23),p=0.003). There is a clear difference between patients' functioning (HS) and the patients' appraisal of functioning (QoL). In elderly CLI patients, this study clearly suggests a discrepancy between the physical QoL (WHOQOL-BREF) and HS (SF-12) measurements in vascular amputees. This raises the question, which outcome measurement is the most relevant for elderly CLI patients. Individual treatment goals should be kept in mind when assessing the HS or QoL outcome of patients undergoing hospital care. With respect to shared decision making, distinctive and subjective QoL questionnaires, like the WHOQOL-BREF, provide a very important outcome measurement and should be used in future research.

Health-related quality of life, psychosocial functioning, and unmet health needs in patients with sarcoma: A systematic review.

Health-related quality of life (HRQoL), psychosocial distress, and unmet health needs are important patient-reported outcomes (PROs) for patients with sarcoma treated with curative intent. Syntheses of data on these PROs in patients with sarcoma are limited. A systematic review of peer-reviewed literature published between 2007 and 2017 was conducted using five databases, guided by the PRISMA and Cochrane reporting guidelines. Of 7240 articles identified, 31 were included in this review. Compared with healthy individuals, patients with sarcoma frequently scored lower in physical and psychological HRQoL domains and experienced higher rates of self-image issues, depression, and suicide. However, outcomes for patients with sarcoma were relatively comparable to those with other malignancies. Anxiety symptoms were more common in the diagnosis phase, while depressive symptoms were more common in the treatment phase. Patients who are older, female, and socially isolated often reported lower HRQoL. As a sarcoma-specific HRQoL instrument is not available, and all studies have used generic instruments, key issues may have been omitted. There are few studies reporting data for HRQoL, psychosocial issues, or unmet health needs in this population. A sarcoma-specific HRQoL instrument is needed to accurately describe outcomes in this population. There is a need for further qualitative research to conceptualise the HRQoL issues relevant to this population, against which to map existing HRQoL instruments and guide adaptation or creation of a new instrument.

Issue Highlights

Issue Highlights

Patient-reported outcome measures inthe recovery of adults hospitalised withcommunity-acquired pneumonia: asystematic review.

Symptomatic and functional recovery are important patient-reported outcome measures (PROMs) in community-acquired pneumonia (CAP) that are increasingly used as trial end-points. This systematic review summarises the literature on PROMs in CAP.Comprehensive searches in accordance with the PRISMA statement were conducted to March 2017. Eligible studies included adults discharged from hospital following confirmed CAP and reporting PROMs.15 studies (n=5644 patients) were included; most were of moderate quality. Studies used a wide range of PROMs and assessment tools. At 4-6 weeks post-discharge, the commonest symptom reported was fatigue (45.0-72.6% of patients, three studies), followed by cough (35.3-69.7%) and dyspnoea (34.2-67.1%); corresponding values from studies restricted by age <65 years (two studies) were lower: fatigue 12.1-25.7%, cough 19.9-31.9% and dyspnoea 16.8-27.5%. Functional impairment 4 weeks post-discharge was reported in 18-51% of patients (two studies), while median time to return to normal activities was between 15 and 28 days (three studies).Substantial morbidity is reported by patients up to 6 weeks post-discharge. There is weak methodological consistency across existing studies. A core set of PROMs for use in future studies is suggested.

Satisfaction with social role participation in adults living with chronic conditions: Comparison to a US general population sample

AbstractPurpose: Health-related quality of life (HRQOL) of adults living with a chronic condition or disease is an important patient-reported outcome. There is a need to better understand the socia...

Translation and validation of the MD Anderson Dysphagia Inventory (MDADI) for Spanish-speaking patients.

The main objective of this study was to perform the adaptation and cultural translation and validation of the MD Anderson Dysphagia Inventory (MDADI) questionnaire for the Spanish language. A total of 69 patients were diagnosed with head and neck cancer treated with surgery; radiotherapy and chemoradiotherapy were included. MDADI was translated and a feasibility, internal consistency, test-retest reliability, and construct validity were assessed. The mean overall score of the MDADI was 51.9 (18-85). Internal consistency for total score was 0.908. The overall score of intraclass correlation coefficient was 0.98 and kappa coefficient scores were almost perfect (test-retest reliability). All domains of MDADI were significantly correlated with physical and mental domains of the SF-12. Construct validity was also evaluated with food texture measures, and with TNM classification. The translation and validation of the Spanish version of the MDADI was performed and can be considered an important patient-reported outcomes tool for dysphagia-related quality of life.

Sacral neuromodulation and sexual function: a systematic review and meta-analysis of the literature.

Sexual function is being increasingly recognized as an important patient-reported outcome. Sacral neuromodulation (SNM) is a treatment with an expanding list of indications. The effect of sacral neuromodulation on sexual function has been examined in a number of studies with variable results. In this review, we aim to systematically review the literature and pool the data if appropriate. The literature search was conducted primarily on the Healthcare Databases Advanced Search (HDAS) platform using the Medline, EMBASE and CINHAL search engines. Of 196 initial citations, 17 articles met our predefined inclusion criteria. Thirteen studies reported enough information to be included in our meta-analysis. RevMan5 software was used for analysis. Eight of 17 studies reported a positive effect of SNM on sexual function. Pooled analysis of data from 11 studies involving 573 patients before SNM and 438 patients after SNM showed significant improvement in sexual function (SMD = -0.39; 95% CI: -0.58 to -0.19; p = 0.0001). The results remained significant in most subgroup analyses except in patients suffering from fecal incontinence. SNM in women with pelvic floor disorders, especially bladder dysfunction, seems to have a positive effect on sexual function. This needs to be verified in adequately powered primary research using sexual function as the primary outcome.

The importance of patient-reported outcomes in clinical trials and strategies for future optimization.

Patient-reported outcomes (PROs) can be included in clinical trials as primary or secondary endpoints and are increasingly recognized by regulators, clinicians, and patients as valuable tools to collect patient-centered data. PROs provide unique information on the impact of a medical condition and its treatment from the patient’s perspective; therefore, PROs can be included in clinical trials to ensure the impact of a trial intervention is comprehensively assessed. This review first discusses examples of how PRO endpoints have added value to clinical trial interpretation. Second, it describes the problems with current practices in designing, implementing, and reporting PRO studies, and how these problems may be addressed by complying with guidance for protocol development, selecting appropriate PRO measures to match clinically motivated PRO hypotheses, minimizing the rates of avoidable missing PRO data, analyzing and interpreting PRO data, and transparently reporting PRO findings.

Pilot study of iPad-based patient-reported outcomes at the MGH Cancer Center.

190 Background: Patient reported outcomes (PROs) have been shown to improve outcomes in cancer patients in clinical trials and have also been shown to improve the patient experience, provide real-time access to patient reported symptoms and are increasingly used across disciplines. Given the importance of PROs, at the MGH Cancer Center, we implemented a pilot using iPADs to capture patient reported outcomes for patients receiving IV chemotherapy in the breast and lymphoma clinics as well all IV chemotherapy patients at one of the MGH satellite clinics where patients across all disease groups are seen. Methods: 12 measures from the PRO-CTCAE were selected to be applicable to all cancer patients. Working with the technology team and EPIC team at MGH the selected PROs were loaded into iPADs and are automatically assigned to patients receiving IV chemotherapy. Staff and clinicians were trained in the breast, lymphoma and satellite clinic. Patient who were receiving IV chemotherapy were assigned iPADs at check in. Patients completed the questionnaire and in real-time, the data was available in the EPIC chart. The objectives of the pilot were to assess the feasibility of collecting iPAD based PROs at the MGH Cancer Center. Results: In the 4-month pilot, there were 2,304 visit types that were eligible to receive an iPAD for PRO collection, 79% (N = 1,1816) patients were assigned an iPAD. Of the 1,816 patients assigned an iPAD, 65% (N = 1,173) patients completed the PROs, 4% (N = 36) partially completed the PROs. In terms of completion at the main disease specialty clinic vs the satellite, completion rates were higher at the satellite (77% vs 44%). During one week, we asked the front desk to record why patients did not complete the PROs-93 patients were assessed, 45% of patient refused the iPAD, in 28% of these patients the staff did not capture the reason and 22% were not given an iPAD. Conclusions: This pilot demonstrates the feasibility of collecting PROs in an oncology clinic for patients receiving IV chemotherapy. We plan to continue to improve the assignment of PROs to eligible patients, educate staff, providers and patients on the important of PROs and plan to expand to all the clinics at MGH Cancer Center and satellite centers.