Patient-centric care in primary immune thrombocytopenia (ITP): shared decision-making and assessment of health-related quality of life.

ABSTRACTImmune thrombocytopenia (ITP), an autoimmune disease characterized by low platelet counts and increased bleeding risk, can impair health-related quality of life (HRQoL), impacting patients' daily lives and mental health. A number of patient-reported outcome (PRO) measures (both generic and specific to ITP) can be used to understand the impact of ITP on HRQoL and generate evidence to guide disease management. As well-developed PRO tools could help in HRQoL assessment, their optimization could help to solidify a patient-centric approach to ITP management. Shared decision-making is a collaborative process between a patient and their healthcare professional in making decisions about care. Treatment decisions based on this shared process between physician and patient are recommended by clinical guidelines. The goal of this narrative review is to discuss treatment decisions with regards to patient-centric ITP management, with a focus on the impact of PRO measures and the process of shared decision-making in practice.

4 Domain Sports PROM in Spanish: Cross-cultural adaptation in the Chilean population and reliability analysis.

Background Patient-Reported Outcome Measures (PROMs) are tools of increasing interest in the sports population. The purpose of this study was to perform the cross-cultural adaptation and reliability analysis of the 4 Domain Sports Patient-Reported Outcome Measure (4 DSP) into Spanish. Methods A six-stage cross-cultural adaptation protocol was executed to obtain the Spanish version of the 4 DSP (S-4DSP). Subsequently, the questionnaire was administered to a population of 108 postoperative athletes with ACL (Anterior Cruciate Ligament) injuries. The questionnaire was administered again after 30 days. Acceptability, floor and ceiling effects, internal consistency (Cronbach's alpha), and reproducibility (Intraclass Correlation) were evaluated. Results The S-4DSP was fully completed by 108 participants (mean age 34 ± 10.75, 26% women), achieving 100% acceptability. No floor effect was detected. The statistical analysis yielded a global Cronbach's alpha for the questionnaire of 0.65, and domain-specific alphas of 0.88, 0.72, 0.27, and 0.68 for the first, second, third, and fourth domains, respectively. The Intraclass Correlation test reached a maximum of 0.94 and a minimum of 0.48 for the first and fifth questions, respectively. Conclusions The S-4DSP is a reliable and useful tool for evaluating Spanish-speaking athletes after ACL reconstruction.

Linking Patient-Reported Outcome Measure (PROM) Scores to Adverse Event Data to Gain Insight in Overestimation of Postoperative PROM Improvement after Total Hip Arthroplasty and Total Knee Arthroplasty Due to Selective Non-Response.

BACKGROUND: To gain insight into how clinically relevant improvement in Patient-Reported Outcome Measure (PROM) scores after Total Hip (THA) and Total Knee Arthroplasty (TKA) may be under- or overestimated, we compared PROM respondents and non-respondents on their adverse event rates and assessed whether adverse event occurrence was associated with clinically relevant PROM improvement from those without adverse events. METHODS: All primary THAs and TKAs performed in 19 Dutch hospitals between January 2017 and December 2019 were included. The Hip disability and Osteoarthritis Outcome Score-Physical function Short form (HOOS-PS) and Knee injury and Osteoarthritis Outcome Score-Physical function Short form (KOOS-PS) were used to assess the physical function after THA and TKA, respectively. Adverse events included 1-year revision, 30-day readmission, 30-day complications, and long (i.e., > 75th percentile) length-of-stay (LOS). A clinically relevant improvement was defined as at least a 10-point decrease in HOOS-PS and 9 points in KOOS-PS scores. Associations between adverse events and clinically relevant HOOS-PS and KOOS-PS improvement were assessed using binary logistic regression models adjusted for patient characteristics and clustering of patients within hospitals. RESULTS: There were 20,338 THA and 18,082 TKA procedures included. Adverse events mostly occurred more frequently in HOOS-PS and KOOS-PS non-respondents than in respondents. The THA patients experiencing revision, complications, or long LOS were less likely to experience clinically relevant HOOS-PS improvements (odds ratios of 0.11 [0.06 to 0.20], 0.44 [0.30 to 0.63], and 0.66 [0.50 to 0.88], respectively). The TKA patients experiencing revision or long LOS were less likely to experience clinically relevant KOOS-PS improvements (odds ratios of 0.26 [0.12 to 0.55] and 0.63 [0.50 to 0.80], respectively). CONCLUSION: Clinically-relevant HOOS-PS and KOOS-PS improvements are likely overestimated, as non-respondents had higher adverse event rates which were associated with lower likelihood to achieve clinically-relevant HOOS-PS and KOOS-PS improvements.

Sustained benefits of onabotulinumtoxinA treatment in chronic migraine: An analysis of the pooled Phase 3 REsearch Evaluating Migraine Prophylaxis Therapy (PREEMPT) randomized controlled trials.

OBJECTIVE: To characterize the long-term (56-week) benefits of continuous onabotulinumtoxinA treatment response in individuals with chronic migraine (CM) who achieved reduction to <15 headache days/month with treatment. BACKGROUND: There are limited data exploring reductions in monthly headache days to levels consistent with episodic migraine among those experiencing CM. Understanding the impact of sustained preventive treatment response in CM can provide important information about the impact of successful therapy. METHODS: The two Phase 3 REsearch Evaluating Migraine Prophylaxis Therapy trials of onabotulinumtoxinA in adults included a 24-week, randomized, double-blind, placebo-controlled phase and a 32-week open-label phase. Data were pooled to determine proportions of individuals with <15 headache days/month while on treatment during several time periods in the double-blind phase (Weeks 21-24; any 12 consecutive weeks; Weeks 13-24) and the entire study (Weeks 53-56; any 12 consecutive weeks; any 4-week period). We assessed the long-term impact on mean monthly headache days and changes from baseline on the six-item Headache Impact Test (HIT-6) and Migraine-Specific Quality of Life questionnaire version 2.1 (MSQv2.1). RESULTS: We analyzed 1384 participants with chronic migraine (double-blind: onabotulinumtoxinA, n = 688; placebo, n = 696; open-label: n = 688 [onabotulinumtoxinA]). The discontinuation rates prior to the completion of the full 56-week treatment period for onabotulinumtoxinA and placebo were 25.4% (n = 175) and 29.3% (n = 204), respectively. During Weeks 13-24 of the double-blind phase, significantly more onabotulinumtoxinA-treated (386/688 [56.1%]) than placebo-treated (342/696 [49.1%]) individuals had <15 headache days/month (p = 0.010), with fewer monthly headache days for onabotulinumtoxinA versus placebo responders. The proportions of participants achieving <15 monthly headache days with onabotulinumtoxinA were 60.9% (419/688) at Weeks 25-56, 81.1% (558/688) at Weeks 53-56, and 79.4% (546/688) during any consecutive 12-week period. Mean changes from baseline on the HIT-6 and MSQv2.1 questionnaire surpassed within-group minimal important difference thresholds in all periods. At Week 24, onabotulinumtoxinA-treated participants who achieved <15 monthly headache days during Weeks 21-24 had a greater mean HIT-6 score reduction (-6.5 vs. -1.4) and greater mean MSQv2.1 Role-Function Restrictive score improvements (21.3 vs. 6.4) than those who did not achieve <15 monthly headache days during the same period. CONCLUSIONS: Participants who achieved <15 monthly headache days with onabotulinumtoxinA treatment achieved meaningful benefits in headache-related disability and migraine-specific quality of life compared with those who remained at or above the 15-monthly headache days threshold. Sustained benefits observed over 56 weeks support long-term onabotulinumtoxinA use for the prevention of CM.

Asking questions that are "close to the bone": integrating thematic analysis and natural language processing to explore the experiences of people with traumatic brain injuries engaging with patient-reported outcome measures.

Introduction: Patient-reported outcomes measures (PROMs) are valuable tools for assessing health-related quality of life and treatment effectiveness in individuals with traumatic brain injuries (TBIs). Understanding the experiences of individuals with TBIs in completing PROMs is crucial for improving their utility and relevance in clinical practice. Methods: Sixteen semi-structured interviews were conducted with a sample of individuals with TBIs. The interviews were transcribed verbatim and analysed using Thematic Analysis (TA) and Natural Language Processing (NLP) techniques to identify themes and emotional connotations related to the experiences of completing PROMs. Results: The TA of the data revealed six key themes regarding the experiences of individuals with TBIs in completing PROMs. Participants expressed varying levels of understanding and engagement with PROMs, with factors such as cognitive impairments and communication difficulties influencing their experiences. Additionally, insightful suggestions emerged on the barriers to the completion of PROMs, the factors facilitating it, and the suggestions for improving their contents and delivery methods. The sentiment analyses performed using NLP techniques allowed for the retrieval of the general sentimental and emotional "tones" in the participants' narratives of their experiences with PROMs, which were mainly characterised by low positive sentiment connotations. Although mostly neutral, participants' narratives also revealed the presence of emotions such as fear and, to a lesser extent, anger. The combination of a semantic and sentiment analysis of the experiences of people with TBIs rendered valuable information on the views and emotional responses to different aspects of the PROMs. Discussion: The findings highlighted the complexities involved in administering PROMs to individuals with TBIs and underscored the need for tailored approaches to accommodate their unique challenges. Integrating TA-based and NLP techniques can offer valuable insights into the experiences of individuals with TBIs and enhance the interpretation of qualitative data in this population.

Confirmation of the Unidimensionality of the Satisfaction and Recovery Index Among Those With Various Musculoskeletal Disorders.

Background A semistructured patient-reported outcome measure (PROM) wherein patients rate the importance of structured items and the magnitude of the psychometric properties to be investigated (e.g., disability and satisfaction) facilitates patient engagement in their treatment and patient-centered clinical practice. The Satisfaction and Recovery Index (SRI) is one such semistructured PROM that was originally developed to measure recovery from a whiplash injury. Exploratory factor analysis demonstrated a one-factor structure among ambulatory community-dwelling people with traumatic musculoskeletal injuries. However, a confirmatory factor analysis has not been conducted among patients with various musculoskeletal disorders, and the internal structure of the SRI has not been established yet. Thus, this study aimed to investigate the internal structure of the SRI among patients with diverse musculoskeletal disorders. Methodology An anonymous survey was performed for patients who were referred for physical therapy for musculoskeletal disorders at a local orthopedic clinic. A confirmatory factor analysis was conducted. The goodness-of-fit criteria were as follows: chi-square/degree of freedom < 3, goodness-of-fit index > 0.90, adjusted goodness-of-fit index > 0.95, and root mean square error of approximation < 0.08. Results Data from 217 participants were analyzed. All goodness-of-fit criteria were satisfied. Conclusion This study confirmed the acceptable internal structure of the SRI among patients with diverse musculoskeletal disorders.

Understanding factors impacting patient-reported outcome measures integration in routine clinical practice: an umbrella review.

PURPOSE: Patient-report outcome measures (PROMs) have gained widespread support as a mechanism to improve healthcare quality. We aimed to map out key enablers and barriers influencing PROMs implementation strategies in routine clinical practice. METHODS: An umbrella review was conducted to identify reviews exploring enablers and barriers related to the integration of PROMs in routine clinical practice from January 2000 to June 2023. Information on key enablers and barriers was extracted and summarised thematically according to the Theoretical Domains Framework. RESULTS: 34 reviews met our criteria for inclusion. Identified reviews highlighted barriers such as limited PROMs awareness among clinicians and patients, perceived low value by clinicians and patients, PROMs that were too complex or difficult for patients to complete, poor usability of PROMs systems, delayed feedback of PROMs data, clinician concerns related to use of PROMs as a performance management tool, patient concerns regarding privacy and security, and resource constraints. Enablers encompassed phased implementation, professional training, stakeholder engagement prior to implementation, clear strategies and goals, 'change champions' to support PROMs implementation, systems to respond to issues raised by PROMs, and integration into patient pathways. No consensus favoured paper or electronic PROMs, yet offering both options to mitigate digital literacy bias and integrating PROMs into electronic health records emerged as important facilitators. CONCLUSIONS: The sustainable implementation of PROMs is a complex process that requires multicomponent organisational strategies covering training and guidance, necessary time and resources, roles and responsibilities, and consultation with patients and clinicians.

Examining the "revisability" benefit of hip resurfacing arthroplasty.

BACKGROUND: Hip resurfacing arthroplasty (HRA) is an alternative to total hip arthroplasty (THA) that is typically reserved for young active patients because it preserves bone. However, the benefits of HRA only hold true if conversion THA after failed HRA provides acceptable outcomes. AIM: To compare patient reported outcomes for conversion THA after HRA failure to primary THA. METHODS: A retrospective review of 36 patients (37 hips) that underwent conversion THA for failed HRA between October 2006 and May 2019 by a single surgeon was performed. Patient reported outcomes [modified Harris Hip Score (mHHS), University of California Los Angeles (UCLA) activity score] were obtained via an email-based responder-anonymous survey. Outcomes were compared to normative data of a primary THA cohort with similar demographics. Subgroup analysis was performed comparing outcomes of conversion THA for adverse local tissue reaction (ALTR) vs all other causes for failure. RESULTS: The study group had a lower mHHS than the control group (81.7 ± 13.8 vs 90.2 ± 11.6, P < 0.01); however, both groups had similar UCLA activity levels (7.5 ± 2.3 vs 7.2 ± 1.6, P = 0.51). Patients that underwent conversion for non-ATLR causes had similar mHHS (85.2 ± 11.5 vs 90.2 ± 11.6, P = 0.11) and higher UCLA activity levels (8.5 ± 1.8 vs 7.2 ± 1.6, P < 0.01) compared to the control group. Patients that underwent conversion for ATLR had worse mHHS (77.1 ± 14.5 vs 90.2 ± 11.6, P < 0.01) and UCLA activity levels (6.1 ± 2.3 vs 7.2 ± 1.6, P = 0.05) when compared to the control group. CONCLUSION: Patient outcomes equivalent to primary THA can be achieved following HRA conversion to THA. However, inferior outcomes were demonstrated for ALTR-related HRA failure. Patient selection and perhaps further studies examining alternative HRA bearing surfaces should be considered.

Growth Factor Concentrate (GFC) for the Management of Osteoarthritis of the Knee: A Systematic Review.

Introduction: The knee is the most commonly affected joint in osteoarthritis (OA), affecting millions of people worldwide. Knee OA significantly impacts the activities of daily living (ADL) along with affecting overall quality of life of patients (QoL), thereby leading to substantial socio-economic burden. Conservative therapies are prioritized, resorting to surgery only when needed. However, these traditional approaches have limitations. Regenerative medicine, involving the use of orthobiologics, including autologous peripheral blood-derived orthobiologics such as growth factor concentrate (GFC), has evolved and shown potential for managing knee OA. The primary goal of this review is to summarize the results of in vitro, preclinical and clinical studies involving GFC for the management of knee OA. Methods: Multiple databases (PubMed, Scopus, Google Scholar, Web of Science and Embase) were searched applying terms for the intervention 'GFC' and treatment 'knee OA' for the studies published in the English language to March 10, 2024. Results: Only three clinical studies met our pre-defined criteria and were included in this review. Conclusion: Intra-articular administration of GFC is safe and potentially efficacious to manage OA of the knee. More, adequately powered, multi-center, prospective, RCTs are warranted to demonstrate the long-term effectiveness of GFC in patients suffering from mild-to-moderate knee OA and to justify its routine clinical use. Further studies evaluating the efficacy of GFC compared to other orthobiologics are also required to allow physicians/surgeons to choose the optimal orthobiologic for the treatment of OA of the knee.

Socioeconomic Differences in Patient Reported Outcome Measures 3 Months After Stroke: A Nationwide Swedish Register-Based Study.

BACKGROUND: There is a well-known association between low socioeconomic status (SES), poor survival, and clinician-reported outcomes after stroke. We aimed to assess socioeconomic differences in Patient Reported Outcome Measures 3 months after stroke. METHODS: This nationwide cohort study included patients registered with acute stroke in the Swedish Stroke Register 2015-2017. Patient Reported Outcome Measures included activities of daily living (mobility, toileting, and dressing), and poststroke symptoms (low mood, fatigue, pain, and poor general health). Information on SES prestroke was retrieved from Statistics Sweden and defined by a composite measure based on education and income tertiles. Associations between SES and Patient Reported Outcome Measures were analyzed using logistic regression adjusting for confounders (sex and age) and additionally for potential mediators (stroke type, severity, cardiovascular disease risk factors, and living alone). Subgroup analyses were performed for stroke type, men and women, and younger and older patients. RESULTS: The study included 44 511 patients. Of these, 31.1% required assistance with mobility, 18% with toileting, and 22.2% with dressing 3 months after stroke. For poststroke symptoms, 12.3% reported low mood, 39.1% fatigue, and 22.7% pain often/constantly, while 21.4% rated their general health as poor/very poor. Adjusted for confounders, the odds of needing assistance with activities of daily living were highest for patients with low income and primary school education, for example, for mobility, odds ratio was 2.06 (95% CI, 1.89-2.24) compared with patients with high income and university education. For poststroke symptoms, odds of poor outcome were highest for patients with low income and university education (eg, odds ratio, 1.79 [95% CI, 1.49-2.15] for low mood). Adjustments for potential mediators attenuated but did not remove associations. The associations were similar in ischemic and hemorrhagic strokes and more pronounced in men and patients <65 years old. CONCLUSIONS: There are substantial SES-related differences in Patient Reported Outcome Measures poststroke. The more severe outcome associated with low SES is more pronounced in men and in patients of working age.

Measurement properties of utility-based health-related quality of life measures in cardiac rehabilitation and secondary prevention programs: a systematic review.

PURPOSE: To identify utility-based patient-reported outcome measures (PROMs) for assessing health-related quality of life (HRQoL) in cardiac rehabilitation and secondary prevention programs (CR) and appraise existing evidence on their measurement properties. Secondly, to link their items to the International Classification of Functioning Disability and Health (ICF) and the International Consortium of Health Outcome Measures (ICHOM) domains for cardiovascular disease (CVD). METHODS: Eight databases were searched. The review followed the COSMIN and JBI guidelines for measurement properties systematic reviews and PRISMA 2020 reporting guidelines. Non-experimental and observational empirical studies of patients ≥ 18 years of age with CVD undergoing CR and assessed quality of life (QoL) or HRQoL using utility-based PROMs or one accompanied by health state utilities were included. RESULTS: Nine PROMs were identified with evidence on measurement properties for three measures: the German translations of SF-12, EQ-5D-5L, and MacNew heart disease HRQoL questionnaire. There was moderate quality evidence for responsiveness and hypothesis testing of the SF-12 and EQ-5D-5L, and high-quality evidence for responsiveness and hypothesis testing for the MacNew. All items of SF-12 and EQ-5D were linked to ICF categories, but four items of the MacNew were not classified or defined. All the PROM domains were mapped onto similar constructs from the ICHOM global sets. CONCLUSION: Three utility-based PROMs validated in CR were identified: the German versions of the EQ-5D and SF-12 and the MacNew questionnaire. These PROMs are linked to a breadth of ICF categories and all ICHOM global sets. Additional validation studies of PROMs in CR are required.

One-stage prosthodontically driven jaw reconstruction in patients with benign and malignant pathologies: A 7- to 11-year cohort study.

OBJECTIVES: One stage functional jaw reconstruction is defined as the resection and reconstruction of segmental defects in conjunction with the placement of dental implants in an ideal prosthetic position and loaded with a provisional restoration, during one surgical procedure. The aim of the study is to describe clinical outcomes of patients who underwent one stage functional jaw reconstruction. METHODS: Patients who underwent one-stage functional jaw reconstruction, from January 2013 to March 2016 were recalled in 2022 and 2023. Planning and execution for the reconstruction utilized either analogue or digital techniques. Outcome parameters recorded were treatment-related outcomes at patient level, implant-related outcomes and patient-reported outcome measures. RESULTS: Eighteen patients underwent one-stage jaw reconstruction with a total of 57 implants. Four patients had maxillary and 14 had mandibular reconstructions. Ten patients underwent postoperative radiotherapy. Ten patients were planned using analogue and eight by digital planning. Three patients had partial flap necrosis, three patients had plate fractures, implant loss was seen in one patient and four patients died during the period. A functional prosthesis was provided in 16 out of the 18 patients. CONCLUSION: One-stage functional jaw reconstruction is a predictable method for providing rehabilitation with successful outcomes at 7-11 years. However, caution should be exercised when the treatment modality is carried out in patients with malignant pathologies who have undergone radiotherapy.

Postsurgical Pain Risk Stratification to Enhance Pain Management Workflow in Adult Patients: Design, Implementation, and Pilot Evaluation.

BACKGROUND: Exposure to opioids after surgery is the initial contact for some people who develop chronic opioid use disorder. Hence, effective postoperative pain management, with less reliance on opioids, is critical. The Perioperative Opioid Quality Improvement (POQI) program developed (1) a digital health platform leveraging patient-survey-reported risk factors and (2) a postsurgical pain risk stratification algorithm to personalize perioperative care by integrating several commercially available digital health solutions into a combined platform. Development was reduced in scope by the COVID-19 pandemic. OBJECTIVE: This pilot study aims to assess the screening performance of the risk algorithm, quantify the use of the POQI platform, and evaluate clinicians' and patients' perceptions of its utility and benefit. METHODS: A POQI platform prototype was implemented in a quality improvement initiative at a Canadian tertiary care center and evaluated from January to September 2022. After surgical booking, a preliminary risk stratification algorithm was applied to health history questionnaire responses. The estimated risk guided the patient assignment to a care pathway based on low or high risk for persistent pain and opioid use. Demographic, procedural, and medication administration data were extracted retrospectively from the electronic medical record. Postoperative inpatient opioid use of >90 morphine milligram equivalents per day was the outcome used to assess algorithm performance. Data were summarized and compared between the low- and high-risk groups. POQI use was assessed by completed surveys on postoperative days 7, 14, 30, 60, 90, and 120. Semistructured patient and clinician interviews provided qualitative feedback on the platform. RESULTS: Overall, 276 eligible patients were admitted for colorectal procedures. The risk algorithm stratified 203 (73.6%) as the low-risk group and 73 (26.4%) as the high-risk group. Among the 214 (77.5%) patients with available data, high-risk patients were younger than low-risk patients (age: median 53, IQR 40-65 years, vs median 59, IQR 49-69 years, median difference five years, 95% CI 1-9; P=.02) and were more often female patients (45/73, 62% vs 80/203, 39.4%; odds ratio 2.5, 95% CI 1.4-4.5; P=.002). The risk stratification was reasonably specific (true negative rate=144/200, 72%) but not sensitive (true positive rate=10/31, 32%). Only 39.7% (85/214) patients completed any postoperative quality of recovery questionnaires (only 14, 6.5% patients beyond 60 days after surgery), and 22.9% (49/214) completed a postdischarge medication survey. Interviewed participants welcomed the initiative but noted usability issues and poor platform education. CONCLUSIONS: An initial POQI platform prototype was deployed operationally; the risk algorithm had reasonable specificity but poor sensitivity. There was a significant loss to follow-up in postdischarge survey completion. Clinicians and patients appreciated the potential impact of preemptively addressing opioid exposure but expressed shortcomings in the platform's design and implementation. Iterative platform redesign with additional features and reevaluation are required before broader implementation.

Capturing patient-reported sleep disturbance in atopic dermatitis clinical trials.

BACKGROUND: Patient-focused approaches to capturing day-to-day variability in sleep disturbance are needed to properly evaluate the sleep benefits of new treatments. Such approaches rely on patient-reported outcome (PRO) measures validated in the target patient population. METHODS: Using atopic dermatitis (AD) as an example of a disease in which sleep is commonly disturbed, we developed a strategy for measuring sleep disturbance in AD trials. In developing this strategy, we conducted a targeted literature review and held concept elicitation interviews with adolescents and adults with AD. We subsequently identified potentially suitable PRO measures and cognitively debriefed them. Finally, we evaluated their psychometric properties using data from phase 2b (NCT03100344) and phase 3 (NCT03985943 and NCT03989349) clinical trials. RESULTS: The literature review confirmed that sleep disturbance is a key impact of AD but failed to identify validated PRO measures for assessing fluctuations in sleep disturbance. Subsequent concept elicitation interviews confirmed the multidimensional nature of sleep disturbance in AD and supported use of a single-item measure to assess overall sleep disturbance severity, complemented by a diary to capture individual components of sleep disturbance. The single-item sleep disturbance numerical rating scale (SD NRS) and multi-item Subject Sleep Diary (SSD)-an AD-adapted version of the Consensus Sleep Diary-were identified as potentially suitable PRO measures. Cognitive debriefing of the SD NRS and SSD demonstrated their content validity and their understandability to patients. Psychometric analyses based on AD trial data showed that the SD NRS is a well-defined, reliable, and fit-for-purpose measure of sleep disturbance in adults with AD. Furthermore, the SD NRS correlated with many SSD sleep parameters, suggesting that most concepts from the SSD can be covered using the SD NRS. CONCLUSIONS: Using these findings, we developed an approach for measuring sleep disturbance in AD trials. Subject to further research, the same approach could also be applied to future trials of other skin diseases where itch causes sleep disturbance.

A protocol for the formative evaluation of the implementation of patient-reported outcome measures in child and adolescent mental health services as part of a learning health system.

BACKGROUND: Mental health conditions affect one in seven young people and research suggests that current mental health services are not meeting the needs of most children and youth. Learning health systems are an approach to enhancing services through rapid, routinized cycles of continuous learning and improvement. Patient-reported outcome measures provide a key data source for learning health systems. They have also been shown to improve outcomes for patients when integrated into routine clinical care. However, implementing these measures into health systems is a challenging process. This paper describes a protocol for a formative evaluation of the implementation of patient-reported measures in a newly operational child and adolescent mental health centre in Calgary, Canada. The purpose is to optimize the collection and use of patient-reported outcome measures. Our specific objectives are to assess the implementation progress, identify barriers and facilitators to implementation, and explore patient, caregivers and clinician experiences of using these measures in routine clinical care. METHODS: This study is a mixed-methods, formative evaluation using the Consolidated Framework for Implementation Research. Participants include patients and caregivers who have used the centre's services, as well as leadership, clinical and support staff at the centre. Focus groups and semi-structured interviews will be conducted to assess barriers and facilitators to the implementation and sustainability of the use of patient-reported outcome measures, as well as individuals' experiences with using these measures within clinical care. The data generated by the patient-reported measures over the first five months of the centre's operation will be analyzed to understand implementation progress, as well as validity of the chosen measures for the centres' population. DISCUSSION: The findings of this evaluation will help to identify and address the factors that are affecting the successful implementation of patient-reported measures at the centre. They will inform the co-design of strategies to improve implementation with key stakeholders, which include patients, clinical staff, and leadership at the centre. To our knowledge, this is the first study of the implementation of patient-reported outcome measures in child and adolescent mental health services and our findings can be used to enhance future implementation efforts in similar settings.

Patient-reported outcome measures for systemic lupus erythematosus: an expert Delphi consensus to guide implementation in routine care.

BACKGROUND: Systemic lupus erythematosus (SLE) may result in great impact on patients' quality of life, social relationships, and work productivity. The use of patient-reported outcome measures (PROMs) in routine care could help capture disease burden to guide SLE management and optimize disease control. We aimed to explore the current situation, appropriateness, and feasibility of PROMs to monitor patients with SLE in routine care, from healthcare professionals' and patients' perspectives. METHODS: A scientific committee developed a Delphi questionnaire, based on a focus group with patients and a literature review, including 22 statements concerning: 1) Use of PROMs in routine care (n = 2); 2) PROMs in SLE management (n = 13); 3) Multidisciplinary management of patients with SLE (n = 4), and 4) Aspects on patient empowerment (n = 3). Statements included in Sects. 2-4 were assessed from three perspectives: current use, appropriateness, and feasibility (with currently available resources). For each statement, panellists specified their level of agreement using a 7-point Likert scale. A consensus was reached when ≥ 70% of the panellists agreed (6,7) or disagreed (1,2) on each statement. RESULTS: Fifty-nine healthcare professionals and 16 patients with SLE participated in the Delphi-rounds. A consensus was reached on the value of PROMs to improve SLE management (83%) and the key role of healthcare professionals (77%) and the need for a digital tool connected to the electronic medical record (85%) to promote and facilitate PROMs collection. PROMs most frequently used in clinical practice are pain (56%), patient's global assessment (44%) and fatigue (39%), all on visual analogue scales. Panellists agreed on the need to implement multidisciplinary consultation (79%), unify complementary tests (88%), incorporate pharmacists into the healthcare team (70%), and develop home medication dispensing and informed telepharmacy programmes (72%) to improve quality of care in patients with SLE. According to panellists, patient associations (82%) and nurses (80%) are critical to educate and train patients on PROMs to enhance patient empowerment. CONCLUSIONS: Although pain, fatigue, and global assessment were identified as the most feasible, PROMs are not widely used in routine care in Spain. The present Delphi consensus can provide a road map for their implementation being key for SLE management.

Exploring subjective quality-of-life indicators in long-term care facilities: a mixed-methods research protocol.

INTRODUCTION: Improving quality of life has become a priority in the long-term care (LTC) sector internationally. With development and implementation guidance, standardised quality-of-life monitoring tools based on valid, self-report surveys could be used more effectively to benefit LTC residents, families and organisations. This research will explore the potential for subjective quality-of-life indicators in the interRAI Self-Reported Quality of Life Survey for Long-Term Care Facilities (QoL-LTCF). METHODS AND ANALYSIS: Guided by the Medical Research Council Framework, this research will entail a (1) modified Delphi study, (2) feasibility study and (3) realist synthesis. In study 1, we will evaluate the importance of statements and scales in the QoL-LTCF by administering Delphi surveys and focus groups to purposively recruited resident and family advisors, researchers, and LTC clinicians, staff, and leadership from international quality improvement organisations. In study 2, we will critically examine the feasibility and implications of risk-adjusting subjective quality-of-life indicators. Specifically, we will collect expert stakeholder perspectives with interviews and apply a risk-adjustment methodology to QoL-LTCF data. In study 3, we will iteratively review and synthesise literature, and consult with expert stakeholders to explore the implementation of quality-of-life indicators. ETHICS AND DISSEMINATION: This study has received approval through a University of Waterloo Research Ethics Board and the Social and Societal Ethics Committee of KU Leuven. We will disseminate our findings in conferences, journal article publications and presentations for a variety of stakeholders.

Real-world evaluation of health-related quality of life in patients with diffuse large B-cell lymphoma based on a multinational survey.

Background: Real-world health-related quality of life (HRQoL) data in patients with diffuse large B-cell lymphoma (DLBCL) are scarce. This study is to compare patient-reported outcomes in patients with DLBCL across therapy lines and countries. Methods: Data were derived from the Adelphi DLBCL Disease Specific Programme™ from January 2021 to May 2021, a survey of physicians and their DLBCL patients in France, Germany, Italy, Spain, United Kingdom (UK), and the United States (US). Results: Overall, analysis was conducted on 441 patients with DLBCL across Europe and the US (mean age 64.6 years, 64% male); 68% had an Ann Arbor stage III and 69% had an Eastern Cooperative Oncology Group Performance Status of 0 to 1. The mean overall GHS/QoL was 54.1; patients on their 3L+ therapy had a lower mean GHS/QoL compared with patients on 1L/2L (P = 0.0033). Further to this, mean EQ-5D-5L utility score was reduced from 0.73 for patients on 1L therapy to 0.66 for patients on 3L+ therapies (P = 0.0149). Mean percentages of impairment while working and overall work impairment were lower for patients receiving 3L+ therapy (12.5% and 17.7%; respectively) than those on 1L therapy (35.6% and 33.8%; respectively). When comparing region, patients in the US had significantly better scores for all functioning and symptomatic scales (per EORTC QLQ-C30) and work impairment (per WPAI) vs. patients with DLBCL in Europe. WPAI scores indicate that the overall activity impairment in the US was 36.6% and in Europe ranged from 42.4% in the UK to 54.9% in Germany. Mean EQ-5D-5L utility score for the US was 0.80, compared to 0.60 - 0.80 across the countries in Europe. Regression analysis showed patients who relapsed after more than one year of treatment were associated with better patient reported outcomes than those who relapse after less than one year. Conclusion: Patient-reported outcomes of DLBCL patients remain poor and patients continue to experience considerable morbidity.

Patient Reported Outcome Measures used in Surgical Evaluation of Obstructive Sleep Apnea: A Systematic Review.

OBJECTIVE: To identify patient-reported outcome measures (PROMs) used to measure adult obstructive sleep apnea (OSA)-related quality of life in sleep surgery and analyze key psychometric properties in the original design and development of each PROM. DATA SOURCES: PubMed, Web of Science, Embase, PsychINFO, and CINAHL. REVIEW METHODS: A systematic review was performed using Preferred Reporting Items for Systematic Reviews and Meta-analyses (PRISMA) guidelines. Disease-specific instruments assessing sleep-related quality of life that were used in sleep surgery studies and validated in patients with sleep disorders were included. The Consensus-based Standards for the Selection of Health Status Measurement Instruments (COSMIN) criteria were used to evaluate methodological quality. RESULTS: A total of 2494 abstracts were retrieved, and 216 underwent full-text review. Seven PROMs assessing sleep-related quality of life were identified. Only one (14%) PROM (SAQLI) was developed using both patient and physician input, and none were rated as "adequate" for content validity. Two PROMs utilized patients with sleep-disordered breathing during content development and none specifically queried patients considering surgery. Six (86%) PROMs demonstrated "very good" analysis of internal consistency per COSMIN criteria, and six (86%) included data on test-retest reliability. CONCLUSION: Several PROMs are utilized in sleep-related quality-of-life assessments for patients treated with OSA surgery. The measurement properties of these PROMs are of variable quality, and notably, no PROMs meet adequate quality measurements for content validity. New and updated PROMs for OSA-related quality of life should consider input from sleep surgery patients and providers. Laryngoscope, 2024.

A cross-sectional survey assessing clinicians' perspectives towards redesigning the surveillance model for head and neck cancer: can we do better?

BACKGROUND: Service delivery of post-treatment surveillance in head and neck cancer (HNC) varies across institutions in Australia. To better understand current practices and develop protocols that maximize service capacity or incorporate emerging technologies, especially in under-resourced regional and remote communities, it is important to obtain the perspectives of clinicians that regularly manage patients with HNC. DESIGN: This cross-sectional study utilized an online survey distributed via email to specialists recruited from HNC-associated networks across Australia. The survey captured information on current practices and explored clinician perspectives towards re-designing the current surveillance model to incorporate telehealth or patient-reported outcome measures (PROMs). Quantitative data was analyzed using descriptive statistics while open-ended survey comments were analyzed using a content analysis approach. RESULTS: Forty participants completed the survey (25 surgeons, 9 medical oncologists, 5 radiation oncologists and 1 oral medicine specialist). Most clinicians used either institution-specific guidelines (44%) or National Comprehensive Cancer Network guidelines (39%), with the remaining 17% using surveillance intervals based on patient symptoms. Following treatment, 53% of participants imaged patients only when there was clinical suspicion of recurrence or new symptoms. Planned surveillance imaging was conducted at 6 or 12-monthly intervals based on the HNC subtype. Fifty-seven percent of clinicians were open to redesigning the surveillance model, specifically in low-risk patients who did not require nasoendoscopic examination. Seventy-one percent had concerns regarding the feasibility of telehealth appointments, citing disparities in digital health equity. Additionally, 61% felt PROMs are currently underutilized and were open to incorporating HNC-specific PROMS into surveillance. Open-ended responses indicated that within the current surveillance model, "fragmented service provision" and "administration issues" were significantly impacting on timing of care. CONCLUSION: Surveyed HNC clinicians feel that current post-treatment surveillance can be fragmented and potentially lead to delayed care. They are open to incorporating PROMS to assist in surveillance scheduling, especially in low-risk patients.