Patient-preferred Outcomes Research Articles

Is There Room for Individual Patient-Specified Preferences in the Patient-Reported Outcome Measurement Revolution?

The study aim was to test the feasibility of collecting qualitative patient-preferred outcomes or goals and the degree of their attainment as an addition to a standardized process for collecting quantitative composite patient-reported outcome measures (PROMs) from patients undergoing knee joint replacement. Patients of a large Midwestern medical group scheduled to have total replacement of their knee joint have been asked to complete a PROMs survey preoperatively and at 3 and 12 months after surgery since 2014. In March 2020, an open-ended question about their most important preferred outcome was added to the existing questionnaire. The responses for all 3 time periods from the first 6 months of this addition were summarized quantitatively and analyzed by 2 reviewers. During that 6-month period, 1481 people completed the main survey while 1463 (98.8%) also completed the open-ended question. At baseline, 90.8% of the 590 baseline respondents identified a preferred outcome. If multiple-choice categories had been used, 82.7% of the responses would have lost some or a large amount of their preferred goals' meaning. Of the 144 who completed surveys at both baseline and 3 months, 86.1% reported another outcome in addition to pain relief, while 54.2% reported "Complete or Mostly" achieving their self-identified preferred outcome. Most people who have joint replacement surgery and respond to a quantitative PROMs survey are willing to report on their other preferred outcomes as well. Adding an open-ended question to PROMs surveys may increase clinician focus on addressing outcomes important to each patient.

Patient-preferred outcomes in patients with vestibular schwannoma: a qualitative content analysis of symptoms, side effects and their impact on health-related quality of life

PurposeDuring counseling and management of patients with vestibular schwannoma (VS), the emphasis is shifting from tumour control and nerve preservation towards maintaining or improving health-related quality of life (HRQoL). Understanding the patients’ perspective and impact of VS is, therefore, of utmost importance. The current study aimed to identify treatment outcomes preferred by patients and to explore the patient-reported VS symptoms and management-related side effects and their impact on HRQoL.MethodsPatients with VS were contacted through the Dutch VS association Stichting Hoormij and questioned using a semi-structured, cross-sectional online survey. Patients were asked to report and rank symptoms and side effects, with their impact on HRQoL and frequency of occurrence. Results were structured through qualitative content analysis. Coded symptoms, side effects, impacts, frequencies, and patient-preferred outcomes were analysed and summarized with descriptive statistics.ResultsOf the 231 respondents, 71% were actively treated. Hearing (symptoms vs. side effects: 78.8% vs. 63.6%), balance (62.3%; 48.8%), and energy issues (33.8%; 32.6%) were the most frequently mentioned symptoms and management-related side effects. Fatigue, deafness, headaches, and hearing loss had the highest impact on HRQoL. The majority of patients identified hearing preservation (61%), balance preservation (38.5%), and reduced tinnitus (34.6%) to be the patient-preferred outcomes.ConclusionThis qualitative study demonstrates that in this population many patients with VS encounter participation difficulties in their daily physical and social activities and value hearing and balance preservation, reduced tinnitus, and restored energy as preferred outcomes as they are hampered by symptoms and side effects related to hearing, balance, and energy. Healthcare professionals should consider these key points and use these and the patient-preferred outcomes in consultation, shared decision making, treatment, and follow-up to optimize patient-centred care.

Glucocorticoids in rheumatoid arthritis: Balancing benefits and harm by leveraging the therapeutic window of opportunity.

Glucocorticoids have been available since the early 1950s and have since become an integral part of the management of rheumatoid arthritis (RA). Due to their rapid effect, glucocorticoids have an appealing profile for treating flares or as "bridging" agents in early RA. The efficacy of glucocorticoids to treat RA has been well established, both to control disease activity and to delay the progression of joint damage. However, despite their benefits, glucocorticoids have equally well-known adverse effects. It is generally accepted that long-term use of glucocorticoids, particularly at higher doses, is not advisable, and recent guidelines for the management of RA therefore either recommend against the use of glucocorticoids or suggest using them only as bridging therapy. Perceptions on the harmful effects of glucocorticoids remain, although mainly based on observational studies. Prolonged glucocorticoid therapy at low doses is still highly prevalent in clinical practice, but recent data suggest a rather favourable risk-benefit balance for this strategy, even in senior patients. Balancing the benefits and risks of treating RA with glucocorticoids thus remains a somewhat controversial topic. This narrative review outlines the historical and current position of glucocorticoids in the management of RA, while summarising recent evidence on their beneficial and detrimental effects. Furthermore, practical strategies for the current use and tapering of glucocorticoids in RA are formulated.

Unmet needs in clinical trials in CKD: questions we have not answered and answers we have not questioned.

Many advances have been made in the field of nephrology over the last decade. These include an increasing focus on patient-centred involvement in trials, exploration of innovative trial designs and methodology, the growth of personalized medicine and, most importantly, novel therapeutic agents that are disease-modifying for large groups of patients with and without diabetes and chronic kidney disease. Despite this progress, many questions remain unanswered and we have not critically evaluated some of our assumptions, practices and guidelines despite emerging evidence to challenge current paradigms and discrepant patient-preferred outcomes. How best to implement best practices, diagnose various conditions, examine better diagnostic tools, treat laboratory values versus patients and understand prediction equations in the clinical context remain unanswered. As we enter a new era in nephrology, there are extraordinary opportunities to change the culture and care. Rigorous research paradigms enabling both the generation and the use of new information should be explored. We identify here some key areas of interest and suggest renewed efforts to describe and address these gaps so that we can develop, design and execute trials of importance to all.

Looking Beyond HbA1c: What Outcomes Matter to Patients With Type 1 Diabetes When it Comes to Adopting Virtual Care for Self-Management Support

Virtual health-care interventions have the potential to improve T1D outcomes, including metabolic control. We sought to understand the perspectives of individuals with T1D on self-management intervention outcomes that matter most to them. We conducted six focus groups (N=24), and performed an inductive analysis to understand self-management practices and patient-preferred outcomes towards technology and virtual care. This study will inform outcome measures used in a new virtual health-care intervention study (T1ME). Participants recounted that to adopt a self-management intervention (e.g. mobile app), it would need to decrease the time spent on self-management, ease the burden of decision-making, and safely minimize the impact of diabetes on their life. Additionally, participants felt that T1D self-management support must be individualized and strive to meet people “where they are.” We further identified the over-arching theme of “control” in T1D self-management, which was categorized into three stages: 1) learning how to adapt and apply self-management strategies to meet evolving needs; 2) managing the constant workload of living with T1D while mitigating disruptions to other aspects of daily life; and 3) gaining confidence and competence in one’s capacity to control their diabetes. Moreover, these phases are not unidirectional; instead, participants recounted moving between them depending on their changing life-stage and needs. This study highlights the importance of patient-oriented approach to health technology development, and various stages in T1D self-management where health-care interventions need to target.

The aspects of healthcare quality that are important to health professionals and patients: A qualitative study

ObjectiveExisting quality of care frameworks insufficiently integrate the perspectives of physicians, nurses and patients. We collected narrative accounts from these three groups to explore if their perspectives might add new content to these existing definitions. MethodsNinety-seven descriptions of “good” and “poor” care episodes were collected from a convenience sample of physicians, nurses and outpatients at eight regional hospitals. Two coders classified the narrative contents into themes related to structures, processes and outcomes of care. ResultsThe physicians, nurses and patients raised the following “quality of care” aspects: Successful communication among staff, with patients and care companions; staff motivation; frequency of knowledge errors; prioritization of patient-preferred outcomes; institutional emphasis on building “quality cultures”; and organizational implementation of fluid system procedures. ConclusionRespondents primarily referred to care processes in their descriptions of “quality of care.” “Hippocratic pride” (in response to care successes) and “Rapid reactivity” (in response to (near) failures) emerged as two new outcome indicators of high-quality care. Practice implicationsThis study provides a first qualitative fundament for understanding the components of “quality of care” from a triangulated frontline perspective. Future research needs to validate our findings with quantitative data to explore their usefulness for completing existing quality frameworks.

What do patients prefer? A multinational, longitudinal, qualitative study on patient-preferred treatment outcomes in early rheumatoid arthritis

ObjectivesTo explore treatment outcomes preferred by patients with early rheumatoid arthritis (RA) and how these change throughout the early disease stage across three European countries.MethodsA longitudinal, qualitative, multicentre study was...

Patients' preferences of cutaneous leishmaniasis treatment outcomes: Findings from an international qualitative study.

BackgroundCutaneous leishmaniasis (CL) is a disease that often affects exposed skin areas and may heal leaving lifelong scars. Patients’ expectations from treatment are rarely considered in drug development for CL. An initiative aiming to address shortcomings in clinical trial design and conduct for CL treatments involving the researchers’ community is on-going. This manuscript presents patient-preferred outcomes for CL and an assessment on how to consider these in the conduct of future trials.Methodology/Principal findingsWe report preferred treatment outcomes by 74 patients with confirmed CL in endemic regions of Brazil, Burkina Faso, Colombia, Iran, Morocco, Peru and Tunisia during individual in-depth interviews. Beyond outcomes customarily considered in trials (such as lesion appearance and adverse events), patients talked about a large number of outcomes related to quality of life, such as pain, scar formation, and others affecting their work and daily activities. They also reported fears around getting rid of the parasite, disease recurrence, and possible sequelae.Conclusions/SignificanceThe study results provide a rich insight into important outcomes for CL treatments, as well as related topics, from the perspective of a diverse patient population. Among the outcomes identified, we argue that those related to quality of life as well as recurrence should be included to a greater extent for assessment in clinical trials, and discuss the suitability of measurement instruments such as the Dermatology Quality of Life Index (DLQI). Interviews also point out the potential need to address concerns related to parasitological cure or scar formation, such as social stigmatization and disability. In addition, patients should be given information in order to clarify reported misconceptions. This study therefore suggests a methodology for consulting CL patients on outcomes as elements of clinical trial design, and how to incorporate these outcomes in trials. It also discusses how reported outcomes could be addressed in clinical care.

European Qualitative research project on Patient-preferred outcomes in Early Rheumatoid Arthritis (EQPERA): rationale, design and methods of a multinational, multicentre, multilingual, longitudinal qualitative study

IntroductionIncluding the patient perspective is important to achieve optimal outcomes in the treatment of rheumatoid arthritis (RA). Ample qualitative studies exist on patient outcomes in RA. A Belgian study recently...

Patient-Preferred Outcomes Measurement After Post-Mastectomy Radiation Therapy and Immediate Reconstruction

Women who undergo mastectomy with immediate breast reconstruction (RC) for breast cancer may receive postmastectomy radiation therapy (PMRT) to improve local control and survival. However, PMRT can negatively impact RC outcomes by increasing complications and worsening the cosmetic results. It is unclear how women prioritize these various outcomes. In the course of designing a larger trial of PMRT with a patient-centered approach, we conducted a survey study to assess patient-reported outcomes and prioritization of these outcomes by patients who had undergone immediate RC and PMRT. Sixty-two breast cancer patients who had undergone implant-based RC and completed PMRT 6-24 months prior at a single institution were approached for study participation. Twenty-nine (47%) completed interviews (by phone or in person) to evaluate opinions of the following validated quality-of-life (QOL) instruments: BREAST-Q, FACT-B, and LYMPH-ICF. These 3 instruments were selected by a group of patient advocates with the goal of capturing important outcomes while minimizing redundancy. After completing the instruments, study participants were asked to rank the relative importance of each domain of the instruments. Clinical data were abstracted from the electronic medical record, including patient age, initial tumor stage and adjuvant therapy. Linear regression modeling was performed to assess associations between patient-reported outcomes and clinical and treatment variables. The median age was 47 years (range, 26-66) and the median interval from PMRT to interview was 22 months (range, 8-27). Patients had T1-3, N0-3 disease and underwent unilateral (52%) or bilateral (48%) mastectomy. Twenty-seven (93%) received chemotherapy. The median total radiation dose was 5000 cGy (range, 4256-6400); 14 (48%) received a scar boost. The mean FACT-B total score was 112.2. Univariate predictors of higher FACT-B total scores included older age, lower N-stage and longer interval from end of PMRT to the interview. The physical well-being (PWB) subscale of the FACT-B was selected as the most important measure by 13/29 patients (45%) and was within the top 3 for 20 patients (69%). Multivariable analysis revealed that higher nodal stage (mean difference -8.1 points for N3 vs. N0; 95% CI: -15.8, -0.3) and younger age (-4.3 points for <40 vs. >40; 95% CI: -8.0, -0.7) were independently associated with a worse PWB score on the FACT-B instrument. These results indicate that among patients who agreed to participate in this study, physical well-being was valued as the most important QOL outcome after immediate implant-based reconstruction followed by PMRT. Future PMRT trials should carefully measure PWB and explore why younger patients and those with higher nodal stage may have a diminished QOL. Explicit elicitation by patients of outcomes most important to them would ensure patient-centeredness in trial design.

Unraveling Patient-Preferred Health and Treatment Outcomes in Early Rheumatoid Arthritis: A Longitudinal Qualitative Study.

To unravel the perspective of patients with rheumatoid arthritis (RA) on preferred health and treatment outcomes at 2 time points during the early stage of their disease and treatment. In a longitudinal, qualitative, explorative study, we individually interviewed 26 patients with early RA (ERA) 4-6 months after the start of initial RA treatment. Fourteen of these participants took part in 1 of 3 focus groups at least 1 year after treatment initiation. Interviews were audiotaped, transcribed verbatim, and analyzed using the constant comparison method. Two patient researchers completed the interdisciplinary research team. Patients with ERA ultimately strive to be normal again, literally and figuratively. Outcome preferences inherent to this urge for normality were related to aspects of disease control, physical aspects, aspects of participation, and mental aspects. Initially, patient outcome preferences in ERA were primarily related to pain relief, medication side effects and burden, and emotional well-being. Patient-preferred outcomes evolved over the ERA disease course, with subtle changes in terminology used by participants and with pain relief staying in the foreground. From a patient perspective, normality is the ultimate outcome to target in ERA. Our study produced knowledge for designing more targeted therapeutic interventions aimed at normalizing patients' health and life in all its aspects during a crucial phase of RA.

OP0137-HPR Unravelling Preferred Outcomes of Disease and Treatment in Patients with Early Rheumatoid Arthritis: A Longitudinal Qualitative Interview Study

BackgroundUnravelling patients' preferences with regards to disease and treatment outcomes, is crucial to deliver patient-centred and high-quality care. Previous qualitative studies in Rheumatoid Arthritis (RA) have provided valuable findings in...

AB1147-HPR Investigating Long-Term QOL in Rheumatoid Arthritis Patients with AIMS2 -3-Year Evaluation of Treatment with Biologics-

BackgroundAccording to the EULAR recommendations on the role of the nurse in the management of chronic inflammatory arthritis, nurses should participate in comprehensive disease management to control disease activity, to...

Does Ethnicity Affect Where People with Cancer Die? A Population-Based 10 Year Study

BackgroundAgeing is a growing issue for people from UK black, Asian and minority ethnic (BAME) groups. The health experiences of these groups are recognised as a ‘tracer’ to measure success in end of life patient-preferred outcomes that includes place of death (PoD).AimTo examine patterns in PoD among BAME groups who died of cancer.Material and MethodsMortality data for 93,375 cancer deaths of those aged ≥65 years in London from 2001–2010 were obtained from the UK Office for National Statistics (ONS). Decedent's country of birth was used as a proxy for ethnicity. Linear regression examined trends in place of death across the eight ethnic groups and Poisson regression examined the association between country of birth and place of death.Results76% decedents were born in the UK, followed by Ireland (5.9%), Europe(5.4%) and Caribbean(4.3%). Most deaths(52.5%) occurred in hospital, followed by home(18.7%). During the study period, deaths in hospital declined with an increase in home deaths; trend for time analysis for those born in UK(0.50%/yr[0.36–0.64%]p<0.001), Europe (1.00%/yr[0.64–1.30%]p<0.001), Asia(1.09%/yr[0.94–1.20%]p<0.001) and Caribbean(1.03%/yr[0.72–1.30%]p<0.001). However, time consistent gaps across the geographical groups remained. Following adjustment hospital deaths were more likely for those born in Asia(Proportion ratio(PR)1.12[95%CI1.08–1.15]p<0.001) and Africa(PR 1.11[95%CI1.07–1.16]p<0.001). Hospice deaths were less likely for those born in Asia(PR 0.73 [0.68–0.80] p<0.001), Africa (PR 0.83[95%CI0.74–0.93]p<0.001), and ‘other’ geographical regions (PR0.90[95% 0.82–0.98]p<0.001). Home deaths were less likely for those born in the Caribbean(PR0.91[95%CI 0.85–0.98]p<0.001).ConclusionsLocation of death varies by country of birth. BAME groups are more likely to die in a hospital and less likely to die at home or in a hospice. Further investigation is needed to determine whether these differences result from patient-centred preferences, or other environment or service-related factors. This knowledge will enable strategies to be developed to improve access to relevant palliative care and related services, where necessary.

THU0575 Long-Term QOL Evaluation with AIMS-2 in Patients with Rheumatoid Arthritis - Two-Year Evaluation in Patients Receiving Treatment with Biologicals

BackgroundAccording to the T2T recommendation, the primary target in the treatment of rheumatoid arthritis (RA) is to achieve the state of clinical remission, and the desired treatment target should be...

No evidence favouring one irrigant versus another in root canal treatments

The Cochrane Oral Health Group's Trials Register, CENTRAL, Medline, Embase, and LILACS databases were searched with no restrictions. Randomised controlled trials (RCTs) involving single or multi-rooted permanent teeth requiring root canal treatment (RoCT) which compared irrigants against each other or against inactive irrigant or placebo were included. Combinations of irrigants were allowed and if used in conjunction with EDTA (ethylenediaminetetra-acetic acid) or similar chelating agents. Two review authors independently assessed risk of bias of included trials and extracted data. Eleven trials (851 participants, 879 teeth) were included. Six trials were assessed at high risk of bias, three unclear and two low. Four compared sodium hypochlorite versus chlorhexidine, the other trials compared different interventions and only two of these trials included useable data on the primary outcomes of swelling and pain. Meta-analysis of sodium hypochlorite versus chlorhexidine indicated no strong evidence of a difference in the existence of bacterial growth between the interventions (risk ratio 0.73; 95% confidence interval 0.34 to 1.56; P = 0.41). None of the included trials reported any data on adverse effects nor radiological changes in periapical radiolucency. Although root canal irrigants such as sodium hypochlorite and chlorhexidine appear to be effective at reducing bacterial cultures when compared to saline, most of the studies included in this review failed to adequately report these clinically important and potentially patient-relevant outcomes. There is currently insufficient reliable evidence showing the superiority of any one individual irrigant. The strength and reliability of the supporting evidence was variable, and clinicians should be aware that changes in bacterial counts or pain in the early postoperative period may not be accurate indicators of long-term success. Future trials should report both clinician-relevant and patient-preferred outcomes at clearly defined perioperative, as well as long-term, time points.

Irrigants for non-surgical root canal treatment in mature permanent teeth

Root canal treatment is carried out on teeth in which irreversible pulpitis has led to necrosis of the dental pulp. As a treatment option it is an alternative to dental extraction. Mechanical preparation and irrigation with antiseptic or antibacterial solutions destroys bacteria and cleans the infected root canal. Irrigants should be effective in deactivating bacteria in the entire root canal space without causing any adverse tissue reactions. Sodium hypochlorite (NaOCl) and chlorhexidine are commonly used but there is uncertainty as to which solution, concentration or combination is the most effective. To assess the effects of irrigants used in the non-surgical root canal treatment of mature permanent teeth. We searched the Cochrane Oral Health Group's Trials Register (to 5 July 2012), the Cochrane Central Register of Controlled Trials (CENTRAL) (The Cochrane Library 2012, Issue 4), MEDLINE via Ovid (1950 to 5 July 2012), EMBASE via Ovid (1980 to 5 July 2012), LILACS via BIREME (1980 to 5 July 2012). There were no restrictions regarding language or date of publication. Randomised controlled trials in single or multi-rooted permanent teeth with pulpal or periapical pathology or both, which require root canal treatment. Irrigants either against each other or against inactive irrigant or placebo. Combinations of irrigants were allowed and if used in conjunction with EDTA (ethylenediaminetetra-acetic acid) or similar chelating agents. Two review authors independently assessed risk of bias of included trials and extracted data. We included 11 trials involving 851 participants with 879 teeth which had undergone root canal treatment and involved the use of irrigants. Two trials were assessed as being at low risk of bias, with six unclear and three high.Four trials compared sodium hypochlorite versus chlorhexidine, however, no primary outcomes and only one secondary outcome, bacterial growth cultures, was reported for two of these trials (20% and 50% of teeth in the control group had positive bacterial culture).The meta-analysis indicated no strong evidence of a difference in the existence of bacterial growth between the interventions (risk ratio 0.73; 95% confidence interval 0.34 to 1.56; P = 0.41).The seven remaining trials each compared different interventions and only two of these trials included useable data on the primary outcomes of swelling and pain. One trial compared sodium hypochlorite 5.25% alone versus sodium hypochlorite 5.25% combined with hydrogen peroxide 3%, and versus normal saline and reported pain at 3 to 14 days after the procedure.There was no evidence of a difference in pain between the three groups.The other trial compared sodium hypochlorite 5% versus sodium hypochlorite with 'proteolytic enzyme', and there was no evidence ofa difference in swelling between the groups. Two further trials reported bacterial growth, and three trials failed to report any data which could be used in the review. None of the included trials reported any data on adverse effects nor radiological changes in periapical radiolucency. Although root canal irrigants such as sodium hypochlorite and chlorhexidine appear to be effective at reducing bacterial cultures when compared to saline, most of the studies included in this review failed to adequately report these clinically important and potentially patient-relevant outcomes. There is currently insufficient reliable evidence showing the superiority of any one individual irrigant. The strength and reliability of the supporting evidence was variable and clinicians should be aware that changes in bacterial counts or pain in the early postoperative period may not be accurate indicators of long-term success. Future trials should report both clinician-relevant and patient-preferred outcomes at clearly defined perioperative, as well as long-term, time points.

Evaluating the triptans

The debilitating effect of migraine has fueled the search for more specific agents to treat its characteristic and associated symptoms. Second-generation oral triptans have shown an improved efficacy profile in comparison with the pioneer sumatriptan and with the over-the-counter medications and prescription analgesics that have been staples of migraine treatment. Although all triptans exert effects through the 5-hydroxytryptamine 1B/1D receptors, each triptan has distinctive pharmacokinetic properties that determine its efficacy and tolerability profile. Empirical findings based on clinical trials have led to associations between triptan pharmacology and efficacy. With the expanded treatment choices, the onus is on healthcare providers (especially primary care physicians, who see the majority of patients with migraine) to determine which treatment has an efficacy profile that best suits the individual patient's needs. Patients prefer pharmacotherapy with a rapid onset of action that facilitates complete pain relief and no recurrence. Data from published comparator trials, based on commonly used efficacy end points and pharmacokinetic properties underlying patient-preferred outcomes, are reviewed in this article.