Inclusion Of Patient-reported Outcomes Research Articles

Integration of Patient Reported Quality-of-life Data into Risk Assessment in Heart Failure

Optimal management of outpatients with heart failure (HF) requires serially updating the estimates of their risk for adverse clinical outcomes to guide treatment. Patient-reported outcomes (PROs) are becoming increasingly used in clinical care. The purpose of this study was to determine whether the inclusion of PROs can improve the risk prediction for HF hospitalization and death in ambulatory patients with HF. We included consecutive patients with HF with reduced ejection fraction (HFrEF) and HF with preserved EF (HFpEF) seen in a HF clinic between 2015 and 2019 who completed PROs as part of routine care. Cox regression with a least absolute shrinkage and selection operator regularization and gradient boosting machine analyses were used to estimate risk for a combined outcome of HF hospitalization, heart transplant, left ventricular assist device implantation, or death. The performance of the prediction models was evaluated with the time-dependent concordance index (Cτ). Among 1165 patients with HFrEF (mean age 59.1 ± 16.1, 68% male), the median follow-up was 487 days. Among 456 patients with HFpEF (mean age 64.2 ± 16.0 years, 55% male) the median follow-up was 494 days. Gradient boosting regression that included PROs had the best prediction performance - Cτ 0.73 for patients with HFrEF and 0.74 in patients with HFpEF, and showed very good stratification of risk by time to event analysis by quintile of risk. The Kansas City Cardiomyopathy Questionnaire overall summary score, visual analogue scale and Patient Reported Outcomes Measurement Information System dimensions of satisfaction with social roles and physical function had high variable importance measure in the models. PROs improve risk prediction in both HFrEF and HFpEF, independent of traditional clinical factors. Routine assessment of PROs and leveraging the comprehensive data in the electronic health record in routine clinical care could help more accurately assess risk and support the intensification of treatment in patients with HF.

Key factors in the choice of appropriate outcomes for clinical trials.

Health research is the foundation of medical knowledge and healthcare system recommendations. Therefore, choosing appropriate outcomes in studies of therapeutic interventions is a fundamental step in producing evidence and, subsequently, for decision-making. In this article, we propose three key factors for the choice of outcomes: the inclusion of patient-reported outcomes, since they focus on the patient's perception of their health status and quality of life; the consideration of clinically relevant outcomes, which are direct measurements of the patient's health status and, therefore, will be decisive in decision-making; and the use of core outcome sets, a tool that standardizes the measurement and interpretation of outcomes, facilitating the production and synthesis of appropriate evidence for the evidence ecosystem. The correct choice of outcomes will help health decision-makers and clinicians deliver appropriate patient-centered care and optimize the use of resources in healthcare and clinical research.

Defining Priorities in Value-Based Cancer Care: Insights From the Alliance for Clinical Trials in Oncology National Cooperative Group Survey.

We determine how stakeholders prioritize the importance of oncologic outcomes, patient-reported outcomes (PROs), and cancer-related health care costs. A survey was distributed to the National Clinical Trials Network Alliance for Clinical Trials in Oncology cooperative group membership from May 14 to June 30, 2022. Respondents were asked to rate (5-point Likert scale) and rank (1-9) evidence-based value domains: overall survival, treatment toxicities/complications, quality of life (QOL), financial toxicity, access to care, compliance with evidence-based care, health system performance, scientific discovery and innovation, and cost to the health care system. A total of 514 members responded, including researchers (24.7%), nurses (19.5%), medical oncologists (17.9%), administrators (9.3%), surgical and radiation oncologists (9.1%), patient advocates (3.1%), and nonphysician providers (16.4%). Participants represented various practice settings including National Cancer Institute-designated cancer centers (29.8%), university-affiliated academic cancer centers (21%), hospital-owned oncology practices (21.8%), and others (27.4%). There was agreement in how respondents prioritized value domains (W = 0.39, P < .001). Respondents ranked patient QOL (mean rank: 2.6 ± 1.9) as most important above all other metrics including survival (mean rank: 3.5 ± 0.3) and access to care (mean rank: 3.5 ± 2.1; P < .001). Members engaged in direct patient care also ranked access to care of higher importance than nonclinicians (P = .026). Cost to the health care system (mean rank: 7.5 ± 2.1) and health system performance (mean rank: 7 ± 2) were ranked as least important (P < .001). Inclusion of PROs into therapeutic assessment (59.3%) was the most frequently selected priority of future cooperative group initiatives. Oncology community stakeholders deemed patient-centered value domains as most important and considered patient QOL the highest priority. Inclusion of PROs into clinical trials was endorsed as an important component of therapeutic assessment. These findings can be taken into consideration when creating a value framework for inclusion in cancer clinical trials.

Opioid Prescribing Variation After Laparoscopic Cholecystectomy in the US Military Health System

IntroductionAfter laparoscopic cholecystectomy (LC), there is a wide variation in opioid prescription miligram morphine equivalent dose (MED) and refills across US medical institutions. Given wide variation and opioid prescription guidelines, it is essential to conduct thorough health services research across medical, surgical, and patient-level factors that can be implemented to improve system-wide prescribing practices. Therefore, this study describes discharge MED variation and opioid refill probability after emergent and nonemergent LC. Materials and methodsThis retrospective cohort study included medical record data of adult patients (N = 20,025) undergoing LC from January 2016 to June 2021 in the US Military Health System. Data visualizations and bivariate analyses examined prescription patterns across hospitals and evaluated the relationship between patient-level, care-level, and system-level factors and each outcome: discharge MED and opioid refill probability. Two generalized additive mixed models evaluated the relationship between predictors and each outcome. ResultsThere was a significant variation in opioid and nonopioid pain medication prescribing practices across hospitals. While several factors were associated with discharge MED and opioid refill probability, the strongest effects were related to time period (before versus after a June 2018 Defense Health Agency policy release) and receipt of an opioid/nonopioid combination medication. Despite decreases in MED, the MED remained almost twice the recommended dose per prior research. ConclusionsVariation by hospital suggests the need for system-level changes that target genuine practice change and opioid stewardship. Inclusion of patient-reported outcomes, electronic health record decision support tools, and academic detailing programs may support system-level improvements.

The inclusion of health-related quality of life and other patient-reported outcomes in landmark trials for Food and Drug Administration oncology drug approvals: An analysis of cancer drug approvals between 2006 and 2022.

1569 Background: How patients feel and function are essential elements of the clinical benefit of interventions which is often not captured by traditional measures of clinical benefit such as progression-free survival and overall survival. Health-related quality of life, functional status, symptom burden, and other patient-reported outcomes (PROs) assess the impact of interventions from the patient’s point of view. The aim of this study is to assess the inclusion of PROs in landmark trials leading to FDA oncology drug approvals. Methods: This is a retrospective cross-sectional study of pivotal trials leading to FDA oncology approvals between January 2006 and December 2022 based on the FDA oncology approval notifications. The trial protocols of all pivotal trials were evaluated for inclusion of health-related quality of life or other PROs as an outcome measure. The approved therapies were classified into first-line, adjuvant/maintenance, and second or later line of treatment. Chi-square tests were used to assess the relationship between the line of treatment and the inclusion of PROs in the trial protocol. Results: There were 420 oncology approvals during the study period with the highest number of approvals in a single year in 2020 (56) and the least in 2007 (7). Lung cancer has the highest number of approvals (61, 14.5%) followed by lymphoma (50, 12%), leukemia (49, 11.7%), genitourinary malignancies (48, 11.4%), breast cancer (36, 8.6%), gastrointestinal malignancies (32, 7.6%), plasma cell disorders (30, 7.1%) melanoma (20, 4.8%), gynecological malignancies (20, 4.8%), hepatobiliary malignancies (14, 3.3%) and head/neck cancers (14, 3.3%). The approved therapies were first line (146, 34.8%), second or higher line (226, 53.8%), and adjuvant/maintenance therapies (48, 11.4%). PROs were included in 40.7% of the pivotal trials. There is a rising trend in the inclusion of PROs from 5.5% of pivotal trials in 2006 to 31% in 2022. Trials on first-line therapies are more likely to include PROs compared to second or higher-line therapies (44.5% vs 36.7%; p = 0.13). Conclusions: Although there is a rising trend in the inclusion of health-related quality of life and other PROs in pivotal trials for FDA oncology drug approvals, most pivotal trials did not include PROs as outcome measures.[Table: see text]

Making decisions about clinical trial enrollment: What’s important to the patient?

e18658 Background: Understanding patient (pt) perspectives on clinical trials is paramount for trial accrual. We qualitatively explored factors pts felt were important when deciding to enroll in clinical trials. Methods: Pts with recurrent ovarian cancer took part in semi-structured interviews which were audio-recorded, transcribed, and consensus coded. Results: 40 pts were interviewed. Median age = 62.4 yrs (41.2, 79.0), 82.5% (33/40) were white, 60% (24/40) were college educated. Median time from diagnosis = 5.5 yrs (0.80, 17.8); 42.5% (17/40) had prior enrollment in a clinical trial. Most pts cited hope for clinical benefit and desire to help others as key motivators for trial participation. Quality of life (QoL) was also an integral factor. Many expressed a willingness to sacrifice QoL for clinical benefit: “I don’t want to, you know, spend the last months suffering…but, if they say ‘Well, we can try this….Maybe you’ll get 6 more months out of it’, which we know that’s hope for 6 more months. So then the suffering might be worth it.” Others would refuse enrollment due to fear of compromised QoL, or poor outcome: “ If I was healthy at the time, I would have difficulty going into a clinical trial. Because my thought would be…“I’m healthy. I feel good. You wanna place me on a clinical trial, and what if it goes bad? What if it doesn’t work?” Another asserted: “I would not participate in a clinical trial if it was going to make me feel worse before it supposedly made me feel better.” A motivator and potential barrier was trust in providers. “I would like to know… this is their…business or work, they're serious about doin' it…and they kind of professional people, someone…that believe in doin' a good job on somethin', not just get up and do it because maybe they gonna get this amount of money for it, but somethin'…at heart that they really believe in…I'll have to know somethin' about the person or the persons that's in charge of that clinical trial.” Another noted: “I would have gone with whatever [my doctor] recommended…at the end of the day, it’s trust. If I didn’t trust my doctor, I’m not so sure I would just jump into this…” Pts cited trial logistics as potential barriers to participation: “Was it something that I would be able to incorporate into my life, both travel-wise and…taking the drug? Was it going to really be a demanding schedule in terms of meals, in terms of sleep…? Those are all things I looked at. ’ Access and affordability were other barriers cited by pts. Conclusions: Pts weigh many factors when considering clinical trials. QoL information is highly desired. Inclusion of patient-reported outcomes in developmental therapeutic trials may inform QoL for future pts. While motivators for participation were apparent, efforts to overcome potential barriers to enrollment by building trust with providers, and increasing education, navigation and decentralized trials may help diversify and improve representation in trial accrual.

P39 Patient-Reported Outcomes As Endpoint in Oncology Trials: An Analysis of FDA and EMA Claims

Patient-Reported Outcomes (PROs) capture patient’s subjective perception of the effects of a disease or treatments. Previous reports have indicated that regulators have different approaches in inclusion of PROs as endpoint measures in the drug approval process. We aim at highlighting such differences by investigating the current use of PROs, as endpoints in oncology trials and label claims granted by the FDA in comparison with the EMA.

Trends in Patient-Reported Outcomes Reporting in Breast Reconstruction: A Scoping Literature Review.

Patient-reported outcomes (PROs) provide essential information in reconstructive surgery, where interventions center on patients' functional and aesthetic goals. Although multiple patient-reported outcome measures (PROMs) have been validated for breast reconstruction since 2009, no studies have assessed recent frequency and consistency in use. This study aims to characterize recent trends in inclusion of PROs in recent breast reconstruction literature. Articles published between 2015 and 2021 pertaining to autologous and/or prosthetic breast reconstruction in Annals of Plastic Surgery and Journal of Plastic and Reconstructive Surgery were considered in a scoping review. Original breast reconstruction articles were reviewed for use of PROMs and characteristics of administration in accordance with PRISMA-Scr guidelines. Previously defined scoping review criteria were considered, including PROM used, timeline of collection, and topics addressed, with trends in frequency and consistency of usage assessed over the designated period. Of the 877 articles reviewed and 232 articles included, 24.6% reported using any PROM. The majority used BREAST-Q (n = 42, 73.7%), with the remainder being institutional surveys or previously validated questionnaires. Patient-reported outcomes were most often collected retrospectively (n = 20, 64.9%) and postoperatively (n = 33, 57.9%). The average time point of postoperative survey administration was 16.03 months (SD, 19.185 months). χ2 Analysis revealed no significant association between the numbers of articles, including PROMs and the year (P = 0.1047). This study demonstrates that only one-fourth of breast reconstruction articles report the use of PROMs with no interval increase over recent years. Patient-reported outcome measures were predominantly used retrospectively and postoperatively with notable variation in timing of administration. The findings highlight the need for improved frequency and consistency of PROM collection and reporting, as well as for further exploration into barriers and facilitators of PROM use.

Real-world data for precision cancer medicine-A European perspective.

Leveraging real-world data (RWD) for drug access is necessary to overcome a key challenge of modern precision oncology: tackling numerous low-prevalence oncogenic mutations across cancers. Withholding a potentially active medication in patients with rare mutations for the sake of control chemotherapy or "best" supportive care is neither practicable nor ethically justifiable anymore, particularly as RWD could meanwhile be used instead, according to scientific principles outlined by the US Food and Drug Administration, European Medicines Agency and other stakeholders. However, practical implementation varies, with occasionally opposite recommendations based on the same evidence in different countries. In the face of growing need for precision drugs, more transparency of evaluation, a priori availability of guidance for the academia and industry, as well as a harmonized framework for health technology assessment across the European Union (EU) are imperative. These could in turn trigger infrastructural changes in national and pan-European registries, cancer management guidelines (e.g., frequency of routine radiologic restaging, inclusion of patient-reported outcomes), and the health data space, to ensure conformity with declared standards and facilitate extraction of RWD sets (including patient-level data) suitable for approval and pricing with minimal effort. For an EU-wide unification of precision cancer medicine, collective negotiation of drug supply contracts and funding solidarity would additionally be required to handle the financial burden. According to experience from pivotal European programs, off-label use could potentially also be harmonized across EU-states to accelerate availability of novel drugs, streamline collection of valuable RWD, and mitigate related costs through wider partnerships with pharmaceutical companies.

Using the Pediatric Patient-reported Outcomes–common Terminology Criteria for Adverse Events to Longitudinally Measure Symptom Adverse Events of Children With Advanced Cancer

Background: Children with advanced cancer are frequently enrolled on clinical trials and experience multiple symptoms. Inclusion of patient-reported outcomes in pediatric cancer trials to monitor for symptom adverse events (AEs) of treatment is limited. Objective: To (1) determine the feasibility and acceptability of administering the Ped-PRO-CTCAE biweekly over a 6-month period to children with advanced cancer, and (2) describe symptom AEs. Interventions/Methods: A multisite prospective longitudinal design was used to electronically collect information about symptom AEs using the Ped-PRO-CTCAE and acceptability of a biweekly assessment interval in children 2–18 of age with a diagnosis of advanced cancer who spoke English or Spanish. Descriptive statistics were used to analyze sample characteristics and symptom AE data. Results: Forty-nine children completed the study with a mean age of 11.1 years with diverse cancer diagnoses. Participants completed 85% of 515 administered symptom measures over an average of 16 weeks. Fatigue severity and frequency attributes for anxiety, pain, nausea, and anorexia were the most reported symptom AEs across all participants and time points, while severity attributes of pain, nausea, headache, and abdominal pain had the highest mean scores. Conclusions: Biweekly electronic symptom assessment using the Pediatric PRO-CTCAE is feasible and acceptable. Implications for Practice: Using the Ped-PRO-CTCAE to longitudinally measure symptom AEs in children with advanced cancer may support clinicians and researchers alike to understand patient-reported symptom experiences and toxicities from treatment across the continuum of care. What is Foundational: Inclusion of patient-reported symptom AE monitoring in therapeutic and nontherapeutic clinical trials is necessary to illuminate symptom experiences.

Leveraging Value-Based Payment Models to Reduce Health Inequities in Radiology

Leveraging Value-Based Payment Models to Reduce Health Inequities in Radiology

Temporal trends in the inclusion of patient-reported outcomes in heart failure randomized trials published in high-impact medical journals: a systematic bibliometric review

Abstract Background Patient reported outcomes (PROs) are important measures of health, particularly in symptomatic conditions such as heart failure (HF), and regulatory agencies have encouraged their collection in randomized controlled trials (RCTs). Purpose To assess temporal trends in the inclusion of patient-reported outcomes (PROs) in heart failure (HF) randomized controlled trials (RCTs) published in high-impact medical journals, explore trial characteristics associated with inclusion of PROs, and describe the quality of PRO reporting in HF RCTs. Methods We searched MEDLINE, EMBASE and CINAHL for studies published between January 1, 2000 and July 17, 2020. We included RCTs published in journals with impact factor ≥10. We assessed temporal trends using the Jonckheere-Terpstra test and conducted multivariable logistic regression analysis to explore trial characteristics independently associated with inclusion of PROs. We assessed the quality of PRO reporting using the Consolidated Standards for Trial Reporting PRO extension statement. Results We identified 12,342 articles, of which 417 RCTs met inclusion criteria. PROs were included in 224 RCTs (53.7%, 95% confidence interval [CI]: 48.8%-58.6%), of which 44 (19.6%) reported PRO as primary or co-primary endpoint. The proportion of RCTs with PROs increased significantly between 2000–2003 (37.4%) and 2016–2020 (65.1%) (p&amp;lt;0.001). PROs had higher odds of inclusion in RCTs that were multicenter (odds ratio [OR]: 1.95; 95% CI: 1.05–3.64; p=0.036); medium-sized (n=51–250) (OR: 2.29; 95% CI: 1.24–4.23; p=0.008); coordinated in in Central and South America (OR: 6.79; 95% CI: 1.34–34.36; p=0.021); and assessed health services (OR: 4.21; 1.97–8.98; p&amp;lt;0.001), device / surgical (OR: 6.24; 95% CI 3.05–12.80; p&amp;lt;0.001), or exercise and rehabilitation interventions (OR: 3.98; 95% CI 1.59–9.97; p=0.003). A majority (54.9%) of the 224 RCTs reported four or less of the eleven CONSORT-PRO items. The median number of CONSORT-PRO items reported was 4 (interquartile range [IQR] 3–6 items per trial), with improved reporting in trials with PRO as primary or co-primary endpoint. Conclusions PROs are included in in just over half of HF RCTs, with an increased reporting of PROs between 2000 and 2020. Large, pharmacotherapy trials conducted in North America and Europe were less likely to include PROs. The quality of PRO reporting in HF RCTs was modest, with trials most often reporting four of eleven CONSORT-PRO items. Funding Acknowledgement Type of funding sources: Public grant(s) – National budget only. Main funding source(s): Canadian Institutes of Health Research

Inclusion of Patient-Reported Outcomes in Adolescent and Young Adult Phase III Therapeutic Trials: An Analysis of Cancer Clinical Trials Registered on ClinicalTrials.gov

ObjectivesThere is a paucity of research on the impact of cancer treatment on the health-related quality of life (HRQOL) of adolescent and young adult (AYA) patients with cancer. Patient-reported outcomes (PROs) are self-report measures used to assess HRQOL and symptom burden. The extent to which PROs have been included in trials that include common AYA cancer types has not been previously assessed. MethodsTherapeutic phase 3 trials among common AYA cancer types (Hodgkin lymphoma, non-Hodgkin lymphoma, acute lymphoblastic leukemia, sarcomas, and germ cell tumors) initiated between 2007 and 2020 were identified on ClinicalTrials.gov. The proportions and characteristics of trials including a PRO endpoint were assessed. For comparison with an older population, the proportion of breast and colorectal therapeutic phase 3 trials including PRO endpoints were assessed. ResultsEighty-seven studies met the inclusion criteria. Overall, 20.7% of therapeutic phase 3 AYA trials included a PRO endpoint, and only one trial published PRO data. Germ cell tumors (42.9%) and non-Hodgkin lymphoma (40%) trials had the highest proportions of PRO inclusion. The European Organization for Research and Treatment of Cancer generic, cancer-specific quality of life questionnaire was the most commonly used PRO measure; nevertheless, the measures used varied within and between cancer types. The proportion of trials including a PRO endpoint did not change significantly between 2007 to 2013 and 2014 to 2020 (18.6% vs 22.7%, P=.79). ConclusionsFew therapeutic phase 3 AYA cancer trials include PRO endpoints, fewer publish PRO data, and there is no homogeneity in the measures administered. Therapeutic trials represent an underused opportunity to capture PRO data in the AYA population with the goal of improving HRQOL outcomes.

What Is the Comparison in Robot Time per Screw, Radiation Exposure, Robot Abandonment, Screw Accuracy, and Clinical Outcomes Between Percutaneous and Open Robot-Assisted Short Lumbar Fusion?: A Multicenter, Propensity-Matched Analysis of 310 Patients.

Multicenter cohort. To compare the robot time/screw, radiation exposure, robot abandonment, screw accuracy, and 90-day outcomes between robot-assisted percutaneous and robot-assisted open approach for short lumbar fusion (1- and 2-level). There is conflicting literature on the superiority of robot-assisted minimally invasive spine surgery to open techniques. A large, multicenter study is needed to further elucidate the outcomes and complications between these two approaches. We included adult patients (≥18 yrs old) who underwent robot-assisted short lumbar fusion surgery from 2015 to 2019 at four independent institutions. A propensity score matching algorithm was employed to control for the potential selection bias between percutaneous and open surgery. The minimum follow-up was 90 days after the index surgery. After propensity score matching, 310 patients remained. The mean (standard deviation) Charlson comorbidity index was 1.6 (1.5) and 53% of patients were female. The most common diagnoses included high-grade spondylolisthesis (grade >2) (48%), degenerative disc disease (22%), and spinal stenosis (25%), and the mean number of instrumented levels was 1.5(0.5). The operative time was longer in the open (198 min) versus the percutaneous group (167 min, P value = 0.007). However, the robot time/screw was similar between cohorts (P value > 0.05). The fluoroscopy time/ screw for percutaneous (14.4 s) was longer than the open group (10.1 s, P value = 0.021). The rates for screw exchange and robot abandonment were similar between groups (P value > 0.05). The estimated blood loss (open: 146 mL vs. percutaneous: 61.3 mL, P value < 0.001) and transfusion rate (open: 3.9% vs. percutaneous: 0%, P value = 0.013) were greater for the open group. The 90-day complication rate and mean length of stay were not different between cohorts (P value > 0.05). Percutaneous robot-assisted spine surgery may increase radiation exposure, but can achieve a shorter operative time and lower risk for intraoperative blood loss for short-lumbar fusion. Percutaneous approaches do not appear to have an advantage for other short-term postoperative outcomes. Future multicenter studies on longer fusion surgeries and the inclusion of patient-reported outcomes are needed.Level of Evidence: 3.

Cautious optimism for machine learning techniques for prediction of heart failure outcomes

Cautious optimism for machine learning techniques for prediction of heart failure outcomes

Under Reporting of Patient Reported Outcomes (PROs) in Myeloproliferative Neoplasm (MPN) Clinical Trials

Introduction Improvements in overall survival (OS) and quality of life (QoL) are clinically relevant outcomes that should guide clinical decision-making. Patients with MPNs can live with their disease for a long period of time, which increases the importance of understanding the impact of treatment in regards to a patients' Health-Related QoL. Therefore, including PROs in clinical trials has become paramount in facilitating informed treatment decisions made by health care providers and patients. Furthermore, the FDA encourages the implementation of patient-centric measures in clinical trials. Objective We aimed to evaluate the frequency at which PRO measures are utilized as study endpoints and are made publicly available when trial results are published. Methods A review was conducted to characterize studies that have evaluated PRO in patients undergoing treatment for myeloproliferative neoplasms. We searched Citeline® Trialtrove database, a registry of clinical trials, for randomized clinical trials including patients with all myeloproliferative neoplasms, initiated between the years 2006-2016, utilizing at least 1 PRO. We excluded trials that evaluated supportive care and studies with no publications. For included trials, we recorded the following data: indication, treatment and comparator, clinical development phase, endpoints, trial sponsorship, and type of scale or questionnaire used for PRO endpoint. We then identified all available publications associated with the trial, and recorded type of publication (abstract or full text), year, number of randomized patients, and reported outcomes. Results Thirty-five MPN trials were obtained through our search. Trials included patients with chronic myelogenous leukemia (CML), polycythemia vera (PV), essential thrombocythemia (ET), primary myelofibrosis, and mastocytosis. Of these 35 trials, 16 were excluded; two studies were classified as supportive care, three studies did not have a publication or were terminated without results, and 11 studies did not have at least one PRO. 19 trials (19/30; 63%) included at least one PRO assessment as an endpoint and were included in the analysis. Among these 19 trials, the commonly used PRO assessments were EQ-5D, EORTC-QLQ C30, Total Symptom Score (TSS), FACT-Leu, FACT-G, MPN-SAF, and Patient Global Impression of Change (PGIC). Three of the 19 trials (15.8%) utilized at least one PRO as a primary endpoint. Among the 19 trials, thirteen (68.4%) were sponsored by industry, three (15.8%) were sponsored by industry-academic collaborations, and three (15.8%) were sponsored solely by academic institutions. 12 studies (63.2%) reported PRO results within their publications. Six studies (31.6%) reported PRO data within their initial publication. Nine of the19 trials (47.4%) included a comprehensive report of PRO data within their publications, while three (15.8%) only partially reported PRO data. Of the 12 trials with published PRO data, four of 12 (33.3%) reported the PRO more than six months after the initial full publication. Most of the included trials focused on disease control outcome and overall survival benefit was not shown. Conclusions: Despite a growing emphasis on QoL and the inclusion of PROs in oncology clinical trials, a significant number of the observed MPN randomized trials did not include a PRO measure. Of those trials that included a PRO measurement, many lacked the comprehensive reporting needed to inform the assessment of clinical benefit and respective decisions. Early and timely reporting of PRO data should be valued, especially in this indication; however, the publication of PRO data along with other critical trial endpoints was not seen in many of the initial trial reports. The collection of PRO data should result in routine, timely, and appropriate reporting as part of the trial outcome publication, to allow for a thorough assessment of investigational clinically relevant treatment effects. If the primary trial publication does not include the PRO results, there should be a reference or acknowledgment to the impending PRO publication. Future research in this field may evaluate the reasons why PROs are not implemented more often in clinical trials and what causal factors have influenced the under-reporting of PRO data in trial publications. Table Disclosures No relevant conflicts of interest to declare.

Inclusion of patient-reported outcomes (PROs) in randomized controlled trials (RCTs) with elderly cancer patients: A systematic review.

e18217 Background: Inclusion of PROs in RCTs involving elderly cancer patients may be particularly important, as the elderly are often frail and vulnerable, and treatment decisions need to carefully balance potential burden against benefit. We aimed to determine how many RCTs involving elderly patients have included a PRO endpoint, and identified the most relevant PRO information available in this area. Methods: A systematic review in PubMed/Medline and Cochrane Library identified RCTs with PRO endpoint that enrolled a cancer sample (breast, colorectal, lung, prostate, gynaecological and bladder cancer) with a mean/ median age ≥70 years, published from January 2004 to June 2018. The quality of PRO reporting was evaluated using the ISOQOL-PRO recommended criteria. Two reviewers independently performed data extraction. RCTs meeting at least two-thirds of the recommended criteria were considered as “probably-robust” and therefore most likely to be able to inform patient care. Results: Out of the 610 RCTs with PRO endpoint identified, only 67 RCTs (11%) enrolled a sample that met the above criteria. In 19 RCTs (28.4%) PROs were the primary endpoint and 35 RCTs (52.2%) were conducted in a metastatic population. Less than one-third of these trials (n = 21) were considered as probably-robust. In 10 (47.6%) out of the 21 probably-robust RCTs, PROs favored the experimental arm and in 8 (38.1%) the arms did not differ. Overall survival (OS) was an endpoint in 13 of the probably-robust RCTs. In only 3 of these RCTs (23.1%) did OS improve in the experimental arm and in 10 (76.9%) there was no difference in OS between arms. In about half of the probably-robust trials evaluating OS (n = 7, 53.8%), PROs provided information that contrasted with survival findings. In two RCTs, OS improved in the experimental arm, while PROs either did not change between arms (n = 1) or favoured the control arm (n = 1). Conversely, in 5 RCTs (38.5%), OS did not differ between arms whereas PROs favoured the experimental arm. Conclusions: Among cancer RCTs including PROs, the proportion of those conducted in the elderly is low. However, PRO data may provide useful information for these type of patients and their clinicians.

Patient perspectives in gout: a review.

Recent studies have produced evidence regarding the patient perspectives in gout including from disease experience to disease outcomes. Therefore, an overview on the topic can help improve our understanding of the patient experience. This article explores several aspects of the patient perspective including the impact of gout on a patient's life, patient knowledge and beliefs regarding gout and its treatments, patient-perceived barriers to optimal medication adherence in gout and patient's perception of their gout. This article also summarizes any evidence of the association of patient perceptions to patient outcomes in gout. A recognition of patient perspectives in gout has the potential to positively impact clinical care for gout. Discussion of disease impact, misperceptions about benefits/harms of urate-lowering therapy (ULT), and patient values/preferences regarding pharmacological and nonpharmacological treatments can lead to a better shared decision-making and improved outcomes in gout. These findings emphasize the importance of inclusion of patient perspective not only in clinical care and quality improvement and research initiatives but also in the design and implementation of the research agenda in gout. Inclusion of patient-reported outcomes in clinical research is likely to improve its relevance to patients with gout.

The effectiveness of alternating rapid maxillary expansion and constriction combined with maxillary protraction in the treatment of patients with a class III malocclusion: a systematic review and meta-analysis.

To establish the effectiveness of Alternate Rapid Maxillary Expansion and Constriction combined with Protraction Facial Mask (Alt-RAMEC/PFM) approach in treating Class III growing patients compared with PFM combined with conventional Rapid Maxillary Expansion (RME/PFM). Search sources: Unrestricted search in five electronic databases and manual searching were undertaken up to February 2018. Randomised clinical trials (RCTs) evaluating the effectiveness of Alt-RAMEC/PFM were selected. Screening of references, data extraction and assessment of bias risk were evaluated independently by two reviewers. Five RCTs comparing the Alt-RAMEC/PFM with RME/PFM met the inclusion criteria. Small but statistically significant mean differences favouring Alt-RAMEC/PFM protocol as measured by SNA angle (1.16°; 95% CI 0.65 to 1.66), SNB angle (0.67°; 95% CI 0.32 to 1.02) and ANB angle (0.66°; 95% CI 0.08 to 1.25) were noted. Alt-RAMEC/PFM exhibited a more favourable overjet correction when compared to RME/PFM, however, differences in other dental changes were insignificant. There is limited evidence with high risk of bias that Alt-RAMEC/PFM can result in a statistically significant increase in maxillary protraction compared with RME/PFM in Chinese subjects over the short-term. High-quality long-term RCTs with inclusion of patient-reported outcomes are required.

Patient-reported outcome measures for use in clinical trials of SLE: a review

Inclusion of patient-reported outcomes is important in SLE clinical trials as they allow capture of the benefits of a proposed intervention in areas deemed pertinent by patients. We aimed to...