Stroke is the 5 th -leading cause of death in the US and the major cause of disability. Many patients who have suffered from strokes are left with permanent neurological deficits, new occupational and social difficulties, and reductions in quality of life that may be idiosyncratic or vary considerably from patient to patient. Solicitation of patient reported outcomes (PROs) in the treatment and recovery from strokes is therefore imperative. We examined a list compiled in 2013 for Stroke by Magin et al. of representative stroke-related, randomized clinical trials published in 10 high-impact journals between 2002 and 2012 to assess whether PROs were collected and which constructs were reported. We categorized PRO measures as stroke-specific (e.g. Stroke Impact Scale), health profile and utility scales (e.g. Beck Depression Scale), or general (e.g. pain visual analog scale). Two reviewers independently abstracted PRO measures from each article and disagreements were resolved by consensus. Fisher’s exact test was used for statistical analyses. Of the 99 articles that met study inclusion criteria, 20% concerned prevention, 22% acute treatment, and 58% rehabilitation. A plurality of trials were European (43%) followed by studies based in the US (25%), Asian countries (10%), and Australia (10%). Altogether, 37% of studies used a PRO of any kind. Stroke-specific PROs were collected in 17% of studies, health profile and utility scales were used in 17%, and general PROs were used in 23%. There were no significant differences in PRO use with regards to year of publication or study location. Health profile and utility scales (p=0.01) and unclassified PROs (p<0.001) were most-commonly reported in rehabilitation trials. Stroke-specific PROs were most commonly published in the journals Brain and Journal of Stroke and Cerebrovascular Diseases (p=0.001). Overall, our review and analysis detected a low prevalence and a large degree of heterogeneity of PRO measures reported in stroke-related clinical trials. Future stroke research must routinely incorporate PROs into the study design to help patients, caregivers, and providers make informed decisions about stroke prevention, treatment, and rehabilitation options that yield outcomes of greatest importance to them.
13Development of National Research and Clinical Agendas for Patient-Reported Outcomes in IR: Proceedings from a Multidisciplinary Consensus Panel
Development of National Research and Clinical Agendas for Patient-Reported Outcomes in IR: Proceedings from a Multidisciplinary Consensus Panel
Read moreAbstract 153: Use Of Patient-reported Outcome Measures (PROMS) In The Primary Publications Of Acute Stroke Clinical Trials (2010-2020)
Background: Patient-reported outcome measures (PROMs) describe health status from the perspective of the patient. There is growing interest in the use of PROMs in clinical trials and there have been major efforts to develop PROMs for neurological conditions (e.g., Neuro-QOL, PROMIS). However, the extent to which such measures are used in stroke trials is uncertain. We sought to determine how often contemporary acute stroke trials included PROMs as primary or secondary outcome measures. Methods: We searched MEDLINE for completed RCTs published in one of nine major journals between 2010 and 2020. Eligible studies were phase 2 or 3 trials undertaken to test therapeutic interventions within one month of stroke onset. For each trial we accessed the primary publication as well as the published protocol and supplement when available. We abstracted key trial characteristics including all primary and secondary efficacy outcome measures. We defined PROMs using online databases (qolid.org) with the focus on self-reported measures of quality of life, symptoms, and function collected without interpretation of an external party. Results: 115 trials met eligibility criteria. Only one trial used a PROM for its primary outcome measure (visual analogue pain scale), but 40 trials (35%) listed at least one PROM as a secondary outcome measure in its primary publication. An additional 15 trials (13%) listed at least one PROM in their protocol. Among the 55 trials that defined at least one PROM as a secondary outcome measure, the most commonly used measure was Euro-QOL (n=42, 76%) followed by SIS-16 (n=9, 16%), SF-36 (n=7, 13%), and the stroke-specific QOL scale (n=5, 9%). Other PROMs included various measures of anxiety and depression (n=9, 16%). Only one study used a Neuro-QOL based measure. Of the 41 trials that listed a PROM as a primary or secondary outcome in the primary publication, 39 (95%) provided PROM results, but of these only 7 (18%) found statistically significant treatment differences. Conclusions: While about half of acute stroke trials listed at least one PROM as a secondary outcome, they played a minor role in the presentation of trial results. Inclusion of PROMs in acute stroke trials requires greater attention during both the design and reporting phases of the trial.
Read more92Added Value of Patient‐Reported Outcome Measures in Stroke Clinical Practice
BackgroundThere is uncertainty regarding the clinical utility of the data obtained from patient‐reported outcome measures (PROMs) for patient care. We evaluated the incremental information obtained by PROMs compared to the clinician‐reported modified Rankin Scale (mRS).Methods and ResultsThis was an observational study of 3283 ischemic stroke patients seen in a cerebrovascular clinic from September 14, 2012 to June 16, 2015 who completed the routinely collected PROMs: Stroke Impact Scale‐16 (SIS‐16), EQ‐5D, Patient Health Questionnaire‐9, PROMIS Physical Function, and PROMIS fatigue. The amount of variation in the PROMs explained by mRS was determined using r2 after adjustment for age and level of stroke impairment. The proportion with meaningful change was calculated for patients with ≥2 visits. Concordance with change in the other scales and the ability to discriminate changes in health state as measured by c‐statistic was evaluated for mRS versus SIS‐16. Correlation between PROMs and mRS was highest for SIS‐16 (r=−0.64, P<0.01). The r2 ranged from 0.11 (PROMIS fatigue) to 0.56 (SIS‐16). Change in scores occurred in 51% with mRS and 35% with SIS‐16. There was lower agreement and less ability to discriminate change in mRS than in SIS‐16 with change in the other measures.ConclusionsPROMs provide additional valuable information compared to the mRS alone in stroke patients seen in the ambulatory setting. SIS‐16 may have a better ability to identify change than mRS in health status of relevance to the patient. PROMs may be a useful addition to mRS in the assessment of health status in clinical practice.
Read more1Patient-Reported Outcomes of Bicruciate Multiligament Versus Single Cruciate Multiligament Knee Injuries
Background: Multiligament knee injuries (MLKIs) are heterogeneous, and bicruciate knee ligament injuries are considered a serious form of this injury. The current literature tends not to distinguish between single and bicruciate MLKI when reporting outcomes. Purpose: To investigate patient-reported outcomes after surgical treatment of MLKI comparing single cruciate MLKI with bicruciate MLKI. The secondary aim was to investigate the influence of different factors on patient-reported outcomes after surgery. Study Design: Cohort study; Level of evidence, 3. Methods: This study was designed as a cross-sectional cohort study. Patients who underwent surgical treatment for MLKI at a single level 1 trauma center between January 2013 and December 2020 were included in this study. Patient-reported outcomes included the Tegner score, Lysholm score, International Knee Documentation Committee (IKDC) subjective knee form, Knee injury and Osteoarthritis Outcome Survey (KOOS), and a visual analog scale for pain. Results: Of the 191 patients meeting the inclusion criteria, 124 (65%) agreed to participate and had a complete data set with a follow-up time at a mean 74 ± 27 months. Patients with single cruciate MLKI (type I) had significantly higher scores for IKDC (P = .007), Lysholm (P = .012), KOOS Pain (P = .04), KOOS Activities of Daily Living (P = .01), KOOS Sport and Recreation (P = .005), KOOS Quality of Life (P = .04), KOOS4 (which considers the subscales of Pain, Symptoms, Sport and Recreation, and Quality of Life) (P = .01), Tegner (P = .04), and visual analog scale for pain during activity (P = .004) when compared with patients with bicruciate MLKI (type II-type IV). Furthermore, age was significantly associated with a lower IKDC (P = .001), and an increased severity of injury was significantly associated with IKDC (P = .015), KOOS4 (P = .022), and Lysholm (P = .029) scores. Conclusion: MLKIs involving a single cruciate ligament had significantly higher patient-reported postoperative outcome measures compared with bicruciate MLKIs. Age and type of injury were important predictors for outcomes. Patients presenting with dislocated knees had lower patient-reported outcomes; however, there was no significant difference in outcomes between bicruciate MLKIs and patients presenting with dislocated knees.
Read more4Under Reporting of Patient Reported Outcomes (PROs) in Myeloproliferative Neoplasm (MPN) Clinical Trials
Under Reporting of Patient Reported Outcomes (PROs) in Myeloproliferative Neoplasm (MPN) Clinical Trials
9Implementation of patient-reported outcome measures in appropriateness criteria of surgery for degenerative lumbar scoliosis
Implementation of patient-reported outcome measures in appropriateness criteria of surgery for degenerative lumbar scoliosis
Read more48Patient-Reported Outcomes Assessment in Cancer Trials: Evaluating and Enhancing the Payoff to Decision Making
In recent years, there has been a growing interest in incorporating patients’ perspectives when measuring and interpreting the clinical benefit of cancer therapies. Patient-reported outcomes (PROs) are used in a variety of cancer clinical trials. These studies range from using PROs to better understand the burden of cancer and the adverse effects of cancer therapy (such as pain), to studies that use PROs as the primary end point of the clinical trial, such as reducing symptoms like nausea or fatigue that can be caused by certain cancer therapies. The evolution of PROs in cancer trials has been recently documented by the National Cancer Institute (NCI)–supported Cancer Outcomes Measurement Working Group and by the US Food and Drug Administration’s recent guidance for the use of PROs in product labeling. PRO research also has been recognized as an important element in the NCI strategic planning process. A major NCI goal is to support research that enhances our understanding of the burden of disease among patients with cancer and cancer survivors, and reduces the sequellae of cancer throughout the continuum of care. NCI has taken an important role in developing new ways to measure patient-reported symptoms such as pain and fatigue, and aspects of health-related quality of life across a wide range of cancers and other diseases, through the Patient Reported Outcomes Measurement Information System (PROMIS). As a National Institutes of Health Roadmap initiative, PROMIS will provide clinical research communities with a reliable, valid, and standardized approach to capture these clinically relevant, but subjective and difficult-to-measure outcomes in clinical trials. Standardization of these data will provide additional benefits to the cancer clinical trial enterprise because it will eventually facilitate their incorporation into the cancer bioinformatics grid (ca-BIG), thereby making PROs available to all researchers who participate in and use the grid to support correlative research studies in connection with clinical trials. Through the NCI Clinical Trials Working Group (CTWG), NCI also has undertaken a major reassessment of the design, review, funding, and implementation of cancer clinical treatment trials and symptom management trials. Among the many recommendations contained in the CTWG report are ones to establish a new funding mechanism and prioritization process for supporting correlative science and quality-of-life studies using PROs, that can be initiated in association with clinical trials. As part of the CTWG process, NCI recently created a Symptom Management and Health-Related Quality of Life (SxQOL) Steering Committee to prioritize and review studies that include PROs as primary end points in symptom management clinical trials, and PROs as secondary end points in phase III Cooperative Group cancer treatment trials sponsored by the NCI. In addition, the SxQOL committee will provide guidance on how to improve the utility of PROs in cancer treatment trials and in symptom management trials. Although PRO data have potential utility for a variety of analyses in cancer clinical trials, the issues surrounding their appropriate and effective application are complex. In September 2006, the NCI convened a 2-day conference entitled “PatientReported Outcomes Assessment in Cancer Trials (PROACT): Evaluating and Enhancing the Payoff to Decision-Making.” This conference was cosponsored by the American Cancer Society and represented a collaborative effort among the leadership from all major NCI extramural divisions involved with clinical trials—the Division of Cancer Treatment and Diagnosis, the Division of Cancer Prevention, and the Division of Cancer Control and Population Sciences. The goal of the PROACT conference was to bring together scientists working in this area to inform NCI and the broader cancer research and policy community on how the NCI clinical trial infrastructure is currently organized to support PRO research in clinical trials, and to identify the issues and lessons learned from the experiences of NCI-sponsored cooperative groups and Community Cancer Oncology Programs; other national and international researchers; and US pharmaceutical industry experts in using PROs in phase II and III cancer treatment trials, as well as in symptom management trials. Before the conference, NCI charged a multidisciplinary Scientific Program Committee of internationally recognized experts to identify key determinants of the successful application of PROs in JOURNAL OF CLINICAL ONCOLOGY O V E R V I E W VOLUME 25 NUMBER 32 NOVEMBER 1
Read more20Quality of life assessment using patient-reported outcome (PRO) measures: still a Cinderella outcome?
Quality of life assessment using patient-reported outcome (PRO) measures: still a Cinderella outcome?
204Patient reported outcome measures in rare diseases: a narrative review
BackgroundRare diseases can lead to a significant reduction in quality of life for patients and their families. Ensuring the patients voice is central to clinical decision making is key to delivering, evaluating and understanding the efficacy of therapeutic interventions. Patient reported outcome measures (PROMs) are used to capture the patient’s views about their health status and facilitate our understanding of the impact of these diseases and their treatments on patient’s quality of life and symptoms.Main textThis review explores some of the current issues around the utilisation of PROMs in rare diseases, including small patient populations and dearth of valid PROMs. Difficulties in validating new or current PROMs for use in clinical trials and research are discussed. The review highlights potential solutions for some of the issues outlined in the review and the implementation of PROMs in research and clinical practice are discussed.ConclusionPatient input throughout the development of PROMs including qualitative research is essential to ensure that outcomes that matter to people living with rare disease are appropriately captured. Given the large number of rare diseases, small numbers of patients living with each condition and the cost of instrument development, creative and pragmatic solutions to PROM development and use may be necessary. Solutions include qualitative interviews, modern psychometrics and resources such as item banking and computer adaptive testing. Use of PROMs in rare disease research and clinical practice offers the potential to improve patient care and clinical outcomes.
Read moreAbstract 11518: Patient Reported Outcomes Measures Are Infrequently Used in Clinical Studies of Heart Transplant Recipients
Introduction / Background: The goals of heart transplantation (HT) are to improve symptoms, restore quality of life, and extend survival. Clinical trials in HT recipients have largely focused on objective outcomes such as measures of cardiac function and survival; however, a paucity of data exists regarding the use of patient reported outcome measures (PROMs) as outcomes in these studies. Aims: We aimed to characterize the use of PROMs in registered clinical trials of HT recipients. Methods: All clinical studies of adult HT recipients were queried from ClinicalTrials.gov, and studies were stratified by inclusion of PROMs. Studies reporting PROMs were identified via specific search terms using the “outcomes measures” field. Summary statistics compared characteristics of studies that did versus did not report PROMs. Results/Data: Between November 1999 and August 2022, 227 studies of HT recipients were registered on ClinicalTrials.gov. At least one PROM was reported in 11% (n=24/227) of registered studies. Studies reporting PROMs were more likely to be conducted outside of the United States (91.7% vs 54.2%, p<0.001) and report a greater number of total primary/secondary outcomes (PROMs: median 7 [IQR: 4, 9] vs. No PROMs: median 3 [IQR: 2, 6]; p<0.001). The majority of studies reporting PROMs (58.3%) were initiated after 2020. Twenty-one distinct PROM tools/domains were reported as outcome measures, with the Short Form Health Survey 36 being most frequently used (n=10/24). Thirteen of the 21 PROMs included questions assessing mental health, whereas only three PROM tools were cardiac-specific (Figure). Conclusions: Only about one in ten registered clinical trials of HT recipients include PROMs, and mental health was the most commonly assessed domain when a PROM was included. Development and validation of PROM tools is needed to assess the multifaceted nature of health-related quality of life in HT recipients.
Read moreDeveloping a core outcome set for sciatica: a scoping review of outcome measures.
Outcome measures used in sciatica research lack standardisation, making it difficult to combine data for analysis. This scoping review identified and categorised Patient Reported Outcome Measures (PROMs) employed in randomised controlled trials investigating sciatica interventions, providing a foundation for developing a consensus-based core outcome set. Scoping review. A systematic search was conducted across MEDLINE, Embase and Cochrane Central for research published between 1999 and 2024. We included randomised controlled trials that involved patients with sciatica and used at least one PROM. Screening and data extraction were performed independently by at least two reviewers. PROMs were categorised using the OMERACT Filter 2.0 framework, inductively sub-categorised into domains, and then the frequency was counted to identify patterns of use. Collection time points and intervention type were also assessed. 187 studies met the inclusion criteria. These studies employed 69 different PROMs, collected 548 times across all papers. The Visual Analogue Scale for pain (n=115), Oswestry Disability Index (n=109) and Numeric Pain Rating Scale (n=74) were most frequently used. PROMs predominantly addressed the pathophysiological (n=274) and life impact (n=262) domains, with minimal attention to resource use/economic impact (n=12). Injection-based interventions were the most studied treatment approach. Follow-up periods using the same PROMs varied considerably between studies, with trends by intervention type. This review identified and categorised PROMs from numerous research studies, revealing substantial heterogeneity in outcome measurement for sciatica trials. This demonstrates the need for a standardised core outcome set. The predominance of use of non-sciatica-specific pain and disability measures suggests potential gaps in capturing sciatica-specific outcomes. Inconsistent follow-up durations and administration methods further highlight the requirement for standardisation.
Read more1Variations in patient-reported outcome (PRO) collection and reporting in novel FDA approved anticancer therapies.
e18202 Background: Patient-reported outcomes (PROs) are increasingly valued as a key tool in patient-focused treatment decisions. However, a lack of standardization leads to significant variability in PRO collection and reporting in ground-breaking clinical trials of novel agents. We sought to characterize the mechanisms of assessment and variability by which PROs are reported for newly approved anti-cancer therapies. Methods: We reviewed the U.S. Food and Drug Administration (FDA) approvals between 2011 and 2017 for anti-cancer new molecular entities (NMEs) and new biologic approvals (BLAs). For each therapy, the pivotal clinical trial leading to FDA approval was identified using the national clinical trial (NCT) number and assessed for inclusion of PROs. A separate PubMed search was conducted to evaluate for PRO publication distinct from the original trial based on national clinical trial registry number. Results: From 2011 to 2017, the FDA approved 66 NMEs/BLAs based on 74 clinical trials for cancer treatment. Of the 74 clinical trial publications, 21 (28%) of the trials published PRO data in their original clinical publication, 18 (24%) published a separate PRO analysis, and 35 (47%) did not publish PRO data in either format. Among the 32 clinical trials (43%) that listed PROs as pre-specified outcomes, 72% published PROs (23/32). The separate PRO analyses (N = 18) were published considerably later following FDA approval (mean 605 days) than the original clinical trials (mean 20 days, N = 74, P < 0.001). Conclusions: As cancer treatment options expand, therapy decisions become increasingly nuanced. PROs assist decision-making by providing detailed information on important aspects of quality of life and tolerability. Our research has identified a significant lag in the publication of companion studies of PRO data associated with pivotal clinical trials, representing a meaningful gap in information critical to patients and oncologists in the process of making informed decisions.
Read more14Moving from clinician-defined to patient-reported outcome measures for survivors of high-grade glioma
BackgroundPersons with high-grade glioma face both neurological and cancer-related symptoms from the tumor itself and its treatment affecting their daily lives. Survival alone is not an adequate outcome, the quality of the survivorship experience needs to be regarded with equal importance. Patient-reported outcome (PRO) measures can be used to evaluate treatment effects and symptom management interventions.PurposeThe aim of this review was to identify the use, challenges, and potential of PRO measures in survivors of high-grade glioma.MethodsA narrative expert opinion review was performed on the subject. In addition to our own experiences we searched PubMed, Cumulative Index to Nursing and Allied Health Literature, the Cochrane Library, and PsycINFO for brain tumor-specific PRO measures used in the population of adult patients with high-grade glioma, both original articles and reviews were included.ResultsThere are several PRO measures that have been validated for patients with primary brain tumors including high-grade glioma. PRO measures are used both in clinical trials to evaluate the effect of treatment on health-related quality of life, and in daily clinical practice for holistic needs assessment and symptom management. Common PRO measures used for patients with high-grade glioma are European Organization for Research and Treatment of Cancer general instrument for patients with cancer together with brain tumor module, Functional Assessment of Cancer Therapy-Brain, and MD Anderson Symptom Inventory for Brain Tumor. Neurologic and cognitive disorders often occur in patients with high-grade glioma, which affects patients’ ability to self-report over time, making it more challenging in this population. PRO as a primary outcome seems underutilized.ConclusionFor clinical research, PRO measures need to be used together with other clinical outcome measures rather than replacing traditional outcome measures. Moving to more use of PRO measures in survivorship care has potential to improve patient-caregiver-healthcare team communication, symptom management, and quality of care. Implementing PROs in survivorship care should also involve caregivers and a response based on the results.
Read more1What's New in Sports Medicine.
What's New in Sports Medicine.
P-129: Quality of life reported outcomes in published research on AL Amyloidosis: a systematic literature review
P-129: Quality of life reported outcomes in published research on AL Amyloidosis: a systematic literature review
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