Achieving Diagnostic Excellence: Roadmaps to Develop and Use Patient-Reported Measures With an Equity Lens.

BACKGROUND: Diagnostic excellence refers to the optimal process to attain an accurate and precise explanation about a patient's condition and incorporates the perspectives of patients and their care partners. Patient-reported measures (PRMs), designed to capture patient-reported information, have potential to contribute to achieving diagnostic excellence. We aimed to craft a set of roadmaps illustrating goals and guiding the development of PRMs for diagnostic excellence ("Roadmaps"). METHODS: We used iterative inputs from environmental literature scans, expert consultations, and patient voice and employed human-centred design (HCD) and equity-focused road-mapping. The culminating activity of these approaches was an Expert Convening. RESULTS: Use of PRMs can achieve multiple goals for diagnostic excellence, including but not limited to: (1) PRMs for diagnostic continuity, (2) diagnostic PRM alerts, (3) PRM-based quality improvement, (4) PRMs for research, (5) PRMs for routine screening, (6) PRM-based diagnostic excellence population-level patterns, and (7) PRMs supporting patient storytelling. Equity is considered as a cross-cutting goal. Altogether these and future goals support operationalising a vision of patient-reported diagnostic excellence. Roadmaps were developed as a dynamic tool to illustrate PRMs in relation to specific steps with feedback loops to accomplish goals, anticipated timeframes (8-15 years), synergies to foster, and challenges to overcome. Roadmaps are practical in their following PRMs through the stages of development, endorsement, implementation and scaling, and acting upon those measures. Timeframe estimates assume immediate transitions between these stages and no acceleration through incentives and active coordination. CONCLUSION: PRMs for diagnostic excellence have potential to connect patient perspectives, equity, and achievable goals. Roadmaps offer a design approach to enable coordinating measurement activities among diverse stakeholders. Roadmaps also highlight versatility in ways patient-reported information can be collected and used, from clinical settings to public health contexts. Patient-reported diagnostic excellence cannot be established as a solely top-down endeavour, but inherently benefits from bottom-up approaches.

Longitudinal Assessment of Clinical Outcomes After Arthroscopic Treatment for Hip Synovial Chondromatosis and the Effect of Residual Loose Bodies: Minimum 4-Year and 8-Year Follow-up.

BACKGROUND: Arthroscopic treatment is recommended for hip synovial chondromatosis. However, evidence regarding long-term clinical outcomes is limited. PURPOSE: To evaluate long-term patient-reported outcomes (PROs) and survival, and to determine the potential effect of residual loose bodies, as evaluated by immediate postoperative computed tomography (CT), on clinical outcomes. STUDY DESIGN: Case series; Level of evidence, 4. METHODS: A consecutive cohort of patients undergoing arthroscopic treatment and diagnosed with synovial chondromatosis between March 2010 and May 2015 were included in the study. Preoperative radiography, CT, and magnetic resonance imaging were performed. Preoperative, midterm (minimum of 4 years), and long-term (minimum of 8 years) PROs were collected for visual analog scale for pain, modified Harris Hip Score (mHHS), Non-Arthritic Hip Score (NAHS), and 12-item international Hip Outcome Tool (iHOT-12). The percentages achieving minimal clinically important difference (MCID) were calculated. PROs and survival were compared between patients with and without residual loose bodies evident on immediate postoperative CT scan. RESULTS: A total of 28 patients (20% of patients were lost to follow-up) were included in the study with a mean follow-up period of 104.9 months (range, 96-139 months). PROs including visual analog scale for pain (preoperative, 3.8 ± 1.2; midterm, 0.9 ± 1.7; long-term, 0.8 ± 1.4), mHHS (preoperative, 66.4 ± 14.4; midterm, 92.8 ± 12.3; long-term, 93.5 ± 10.5), NAHS (preoperative, 45.2 ± 16.2; midterm, 81.8 ± 15.3; long-term, 83.1 ± 12.9), and iHOT-12 (preoperative, 48.4 ± 15.6; midterm, 69.3 ± 11.7; long-term, 72.7 ± 11.4) were improved at both midterm and long-term follow-up (all with P < .001). In total, 27 (96.4%), 28 (100%), and 26 (92.9%) patients achieved MCID for mHHS, NAHS and iHOT-12, respectively, at the long-term follow-up. No significant difference was found in any of the PROs and the rate of achieving MCID between midterm and long-term follow-up (all with P > .05). One patient (3.6%) underwent revision surgery. Among the 23 patients who had loose bodies on preoperative CT or radiographs, 14 patients (60.9%) with residual loose bodies evident on immediate postoperative CT demonstrated lower NAHS (P = .045) and iHOT-12 (P = .037) scores but a comparable survival (P > .05) at long-term follow-up compared with those who did not have loose bodies. CONCLUSION: Arthroscopic treatment for hip synovial chondromatosis achieved satisfactory long-term clinical outcomes with strong survival. Most patients maintained or improved their overall functional status between midterm and long-term follow-up. Furthermore, patients with residual loose bodies had less favorable clinical outcomes, although the survival rate was comparable.

Isolated Medial Patellofemoral Ligament Reconstruction for Recurrent Patellar Instability Regardless of Tibial Tubercle-Trochlear Groove Distance and Patellar Height: Minimum 5-Year Outcomes.

BACKGROUND: It remains unclear which subset of patients with recurrent patellofemoral instability would benefit from a concomitant bony realignment procedure in addition to a medial patellofemoral ligament (MPFL) reconstruction. PURPOSE: To provide midterm results for patients who underwent an isolated MPFL reconstruction as part of an ongoing prospective trial. STUDY DESIGN: Case series; Level of evidence, 4. METHODS: Patients with recurrent patellar instability were prospectively enrolled in an institutional registry beginning in March 2014. Exclusion criteria included history of a previous surgery for patellar instability, an off-loadable (inferior/lateral) chondral defect, anterior knee pain ≥50% of their chief complaint, and a "jumping J" sign. All patients underwent primary, unilateral, isolated MPFL reconstruction regardless of their bony anatomic characteristics. Patient-reported outcome measures (PROMs), episodes of recurrent instability, and ability to return to sport were obtained annually. Radiographic measurements of baseline radiographs and MRI were obtained at baseline. RESULTS: A total of 138 patients underwent isolated MPFL reconstruction between March 2014 and December 2019. The mean radiographic measurements were tibial tubercle-trochlear groove, 15.1 ± 4.9 mm; Caton-Deschamps index, 1.14 ± 0.16; patellar trochlear index, 46.9% ± 15.1%; trochlear depth index, 2.5 ± 1.2 mm; tibial tubercle to lateral trochlear ridge, -8.4 ± 5.7 mm; and patellar tendon to lateral trochlear ridge, 5.7 ± 6.2 mm. Trochlear dysplasia, defined as a trochlear depth index <3 mm, was present in 79/125 (63%) patients. A total of 50 patients reached ≥5 years, of whom 40 (80%) completed follow-up PROMs. A total of 119 patients reached ≥2 years, of whom 89 (75%) completed follow-up PROMs. Six patients (5%) reported recurrent instability with a mean time of 2.97 years after surgery. All PROMs improved over time except for the Pediatric Functional Activity Brief Scale (Pedi-FABS), which had no change. At 2 years, the mean changes from baseline for Knee injury and Osteoarthritis Outcome Score (KOOS) Quality of Life subscale (QOL), Pedi-FABS, International Knee Documentation Committee (IKDC) score, KOOS Physical Function Short Form (PS), and Kujala score were 42.1, 0.6, 35.1, -23.5, and 32.3, respectively. All changes had P values <.001 except for Pedi-FABS, which showed no change and had P > .999. At 5 years, the mean changes from baseline for KOOS-QOL, Pedi-FABS, IKDC, KOOS-PS, and Kujala score were 42.6, -2.8, 32.6, -21.5, and 31.6, respectively. All changes had P values <.001 except for Pedi-FABS, which showed no change and had P > .453. In total, 89% of patients returned to sport with a mean of 9.1 months. CONCLUSION: Midterm outcomes for patients who underwent isolated MPFL reconstruction were favorable and were maintained at 5 years. Outcomes for the expanded cohort of patients with a minimum 2-year follow-up support previously published results.

Pubic bone osteomyelitis and fistulas after radiation therapy of the pelvic region: patient-reported outcomes and urological management of a rare but serious complication.

PURPOSE: This study investigated late urinary adverse events (UAEs) in patients who underwent pelvic radiation therapy, with a focus on occurrence, diagnostic characteristics and the impact of subsequent extirpative surgery with the need of urinary diversion on quality of life. METHODS: A retrospective analysis of 20 patients after pelvic radiotherapy (2016-2022) was conducted. Data included demographics, perioperative details, oncological parameters, and patient-reported outcomes. Imaging (CT, MRI) was examined for early manifestations of late UAEs. RESULTS: In the study cohort, prostate cancer was the primary malignancy in 85% with a mean radiation dose of 84 Gray over 35 days. Time to diagnosis of late UAEs was 4.0 years post-radiation. Radiological assessment demonstrated a progressive increase in typical CT and MRI features of pubic bone osteomyelitis over time. Surgical interventions, mainly cystectomy, were required with variable outcomes in patient-reported post-surgery quality of life. CONCLUSION: Diagnosing and managing late UAEs after pelvic radiation necessitate an understanding of their occurrence, diagnostic features and appropriate management strategies. Early imaging, particularly MRI, is crucial for timely diagnosis and treatment planning. Variable post-surgery quality of life underscores the importance of a multidisciplinary approach in managing late UAEs. The study contributes to understanding these complications and emphasizes their consideration in post-radiation follow-up care.

Real-world accuracy of robotic-assisted total knee arthroplasty and its impact on expedited recovery.

Despite total knee arthroplasty (TKA) being the gold standard for end-stage knee osteoarthritis, 20% of patients remain dissatisfied. Robotic-assisted arthroplasty promises unparalleled control of the accuracy of bone cuts, implant positioning, control of gap balance, and resultant hip-knee-ankle (HKA) axis. Patients underwent clinical and radiological assessments, including knee CT scans and patient-reported outcome measures (PROMs), preoperatively. Follow-up assessments were conducted at 2 weeks, 6 weeks, and 3 months post-operatively, with imaging repeated at 6 weeks. A total of 155 patients underwent robotic-assisted TKA and have completed 3 months of follow-up. Mean pre-operative HKA axis was 7.39 ± 5.52 degrees varus, improving to 1.34 ± 2.22 degrees varus post-operatively. Restoration of HKA axis was 0.76 ± 1.9 degrees from intra-operative planning (p < 0.0005). Implant placement accuracy in the coronal plane was 0.08 ± 1.36 degrees (p = 0.458) for the femoral component and 0.71 ± 1.3 degrees (p < 0.0005) for the tibial component. Rotational alignment mean deviation was 0.39 ± 1.49 degrees (p = 0.001). Most patients (98.1%) had ≤ 2 mm difference in extension-flexion gaps. PROM scores showed improvement and exceeded pre-operative scores by 6 weeks post-surgery. Robotic-assisted knee arthroplasty provides precise control over traditionally subjective factors, demonstrating excellent early post-operative outcomes.Level of evidence Prospective observational study-II.

Feasibility of collecting longitudinal patient-reported outcomes in individuals with relapsed or refractory large B-cell lymphoma who received chimeric antigen receptor T-cell (CART) therapy.

PURPOSE: Chimeric antigen receptor T-cell (CART) therapy has shown clinical efficacy in refractory and relapsed large B-cell lymphomas, but is associated with serious acute and long-term toxicities. To understand the patient perspective, we measured a patient-reported outcome (PRO), specifically, health-related quality of life (HRQoL), at multiple time points over one year. METHODS: This was a prospective feasibility study of a cohort of patients who were eligible for standard of care CART therapy, tisagenlecleucel. Demographic data and disease characteristics were collected. HRQoL was measured using FACT-Lym at baseline, and months 1, 3, 6 and 12. FACT-Lym includes FACT-G (physical, social, emotional and functional well-being domains), plus a lymphoma subscale. RESULTS: Thirty-four of 35 patients approached, consented to participate. Two of them did not receive their infusion due to progressive disease. 50% were female and median age was 62 (23-77). Twenty-nine patients (91%) completed baseline FACT-Lym and 20 of 21 (95%) eligible patients completed 12-month FACT-Lym. 52% completed all 4 post-baseline FACT-Lym measures. Exploratory analyses for changes in FACT-Lym scores are reported. CONCLUSION: It is feasible to measure longitudinal PROs in patients who receive CART therapy. This study will inform future studies in evaluating the patient perspective on CART therapy.

Patient-reported outcomes and daily activity assessed with a digital wearable device in patients with paroxysmal nocturnal hemoglobinuria treated with ravulizumab: REVEAL, a prospective, observational study.

BACKGROUND: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic blood disorder. Symptoms such as fatigue can have a substantial impact on patients' physical activity levels, sleep, quality of life, and work productivity. Ravulizumab treatment can reduce thrombosis risk, improve survival and quality of life, and reduce fatigue in PNH, but information is limited on how it impacts sleep and physical activity. Here, data on resting heart rate, daily physical activity, and sleep in ravulizumab-treated patients with PNH were passively collected via a digital wearable activity-tracking device and patient-reported outcome (PRO) data were collected via weekly surveys in the same cohort. METHODS: REVEAL was a 32-week prospective observational cohort study in individuals with PNH receiving ravulizumab in the USA. A wrist-worn Fitbit™ collected data on resting heart rate, daily step count, and sleep duration from eligible patients. Patients also completed the following electronic weekly surveys: Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue, Patient-Reported Outcomes Measurement Information System (PROMIS) Global Physical Health, PROMIS Global Mental Health, PROMIS Sleep-Related Impairment and Sleep Disturbance, and Work Productivity and Activity Impairment Questionnaire - Specific Health Problem (WPAI-SHP). Data collected from the activity trackers and surveys were compared against US general population values reported in the literature. RESULTS: Twenty-eight ravulizumab-treated patients were included (median age: 34 years; 54% female). PRO scores were within US general population normative values, including FACIT-Fatigue (40.0), PROMIS Global Physical Health (51.0), Global Mental Health (51.0), Sleep-Related Impairment (50.0), and Sleep Disturbance (49.0). Similarly, mean resting heart rate (67 bpm), daily step count (7476), and sleep duration (7.7 h) were within the range of US general population values. Daily step count was positively correlated with PROMIS Global Physical and Mental Health scores. CONCLUSIONS: This was the first study to use digital monitoring technology to collect data on physical activity and sleep in patients with PNH. The findings indicate that ravulizumab treatment enables patients with PNH to achieve activity levels (heart rate, sleep duration, step count) and quality of life that are comparable to those of the US general population. A weak positive correlation was identified between patient-reported physical and mental health and daily physical activity levels.

Patient experience in bariatric surgery: protocol of a French narrative inquiry and qualitative analysis.

INTRODUCTION: The quality of hospital care, especially surgery, is traditionally assessed using indicators derived from healthcare databases or safety indicators. Given the growing importance of placing the patient at the heart of care evaluation, the use of questionnaires such as the Patient-Reported Experience Measures and Patient-Reported Outcome Measures has become widespread in recent years. However, these tools-addressing factors such as satisfaction, pain management or wait times-only imperfectly reflect the patient's experience, and all such attempts at patient-centred care quality assessment rely on questions or indicators defined in advance by healthcare providers and health authorities. A biopsychosocial model may allow to better understand the patient experience and to improve care pathways. This study seeks to construct a narrative of the bariatric surgical care journey with instruments from narrative inquiry, propose a metanarrative that can serve as a basis for more sophisticated and reliable patient-focused care quality models and define indicators linked to patients' feelings and stories. METHODS AND ANALYSIS: To achieve these aims, 16 bariatric surgical patients at the hospital of Créteil, France (Centre Hospitalier Intercommunal de Créteil), will be included and interviewed once before and twice after surgery, at months 3 and 6. Narratives collected will be used to construct a metanarrative intended to encompass all possible narratives. This metanarrative may ultimately inform new patient care quality indicators, furthering care focused on patients and tailored to their needs and predispositions. ETHICS AND DISSEMINATION: The study is funded by the Group of Clinical Research and Innovation in Île-de-France and was approved by CPP SUD-EST VI Clermont-Ferrand (France) Research Ethics Committee. The results will be submitted for publication in peer-reviewed journals. The patient associations will be approached for the dissemination of the study results. TRIAL REGISTRATION NUMBER: NCT05092659.

Clinico-mycological validation of dermatophytosis severity score and its correlation with patient-reported outcome measures.

BACKGROUND: Dermatophytosis impacts a significant portion of the global population. Recent shifts in the disease's presentation, severity and response to treatment, primarily due to emerging drug resistance, underscore the need for reliable assessment tools. The Dermatophytosis Severity Score (DSS) aims to standardise the evaluation of the disease's severity and monitor therapeutic responses. METHODS: In a cross-sectional pilot study, 25 adults with clinically diagnosed dermatophytosis were evaluated using the DSS. The study also aimed to establish the correlation of DSS with different stages of treatment, dermatophyte species and patient-reported outcomes. Participants were recruited from a dermatology outpatient clinic, and the DSS was applied at baseline, Weeks 4 and 8. The validity and reliability of the DSS were assessed using statistical measures, including Cronbach's alpha and intraclass correlation coefficient. RESULTS: The study comprised of a near-equal distribution of male (52%) and female (48%) patients, primarily within the age group of 20-39 years. A high recurrence rate of dermatophytosis (60%) was noted, and more than half of the patients (56%) had used topical steroids before presentation. The mean DSS significantly decreased from baseline to the final visit, mirroring the substantial reduction in the 5D itch scale and Dermatology Life Quality Index, with strong positive correlations observed between these measures. CONCLUSION: The DSS demonstrated high inter-rater reliability and internal consistency, indicating its utility as a reliable clinical tool for assessing dermatophytosis severity. The strong correlation of DSS with itch intensity and quality of life validates its role in patient-centered care. Continued use and further validation of the DSS are recommended to enhance dermatophytosis management and treatment outcomes.

A single-centre RetrospeCtive sTudy Investigating patient-reported outcomeS of extended dressing wear time for incisional healing following orthopaedic surgery: the ARCTIS study.

OBJECTIVE: Orthopaedic surgery is an effective intervention for treating the symptoms of degenerative joint disease or osteoarthritis (OA). Frequent wound dressing changes, unless clinically indicated, can disrupt the healing process and increase the occurrence of incision site contamination. Protection from contamination is critical for surgical incisions and, therefore, undisturbed wound healing (UWH) in surgical wound management is vital. This article describes a retrospective study reporting the clinical performance of a self-adherent, absorbent postoperative dressing, with a focus on dressing wear time. METHOD: A single-centre, retrospective electronic medical record review of a convenience sample of adult patients treated with a dressing (Mepilex Border Post Op; Mölnlycke, Sweden) following elective hip or knee replacement was undertaken. Data relating to dressing wear time, rationale for dressing changes and patient-reported outcomes were extracted from a mobile health application moveUP Therapy (moveUP NV, Belgium). Health-related quality of life assessment was conducted using the EQ-5D-5L questionnaire and orthopaedic-specific quality of life (QoL) indicator tools. RESULTS: Of the 558 records reviewed, 151 respondents (27.1%) reported outcomes relating to dressing wear time and frequency of dressing change. The average wear time of the first dressing was 13.6 days (second dressing: 5.3 days). The proportion of patients who wore the first dressing for 1-7 days, 8-13 days and for ≥14 days was 17.2%, 13.2% and 69.5%, respectively. Data from the completed questionnaires revealed improvement in QoL over time. CONCLUSION: The results of this study are a good indicator of the suitability of the postoperative dressing for a 14-day wear time, in line with the principles of UWH.

Efficacy, safety, and patient-reported outcome of immune checkpoint inhibitor in gynecologic cancers: A systematic review and meta-analysis of randomized controlled trials.

Over the past decades, immune checkpoint inhibitors (ICIs) have shown dramatic efficacy in improving survival rates in multiple malignancies. Recently, gynecological cancer patients also showed to respond favorably to ICI treatment. This study aimed to evaluate the efficacy, safety, and patient-reported outcomes of ICI therapy in gynecological cancers. We conducted a systematic review and meta-analysis by retrieving literature from multiple electronic databases, such as MEDLINE, ScienceDirect, EBSCO, ProQuest, and Google Scholar. The protocol used in this study has been registered in PROSPERO (CRD42022369529). We included a total of 12 trials involving 8 therapies and 8,034 patients. ICI group demonstrated a longer OS (HR: 0.807; 95% CI: 0.719, 0.907; p = 0.000) and greater PFS improvement (HR: 0.809; 95% CI: 0.673, 0.973; p = 0.024) compared to the control group. There was no significant difference in the incidence of treatment-related adverse events [RR: 0.968; 95%CI: 0.936, 1.001; p = 0.061], but a higher incidence of immune-related adverse events (IRAEs) was observed in the ICI group (RR: 3.093; 95%CI: 1.933, 4.798; p = 0.000). Although the mean changes of QOL score from baseline was not significantly different between both groups (SMD: 0.048; 95% CI: -0.106, 0.202; p = 0.542), the time to definitive QOL deterioration was longer in the ICI group (HR: 0.508; 95% CI: 0.461, 0.560; p = 0.000). Despite having a higher incidence of IRAE, ICI was shown to improve survival rates and QOL of patients. Thus, it should be considered as a new standard of care for gynecologic cancers, especially in advanced stages.

Patient-reported symptoms and burden of eosinophilic esophagitis: evidence from real-world clinical practice.

BACKGROUND: Eosinophilic esophagitis is a chronic inflammatory disorder of the esophagus. This real-world study used patient and physician surveys to describe the clinical characteristics and disease burden of eosinophilic esophagitis-overall and in a subgroup of patients with dysphagia despite treatment. METHODS: Data analyzed in this study were collected in 2020 from US and EU patients with eosinophilic esophagitis. Eligible patients were aged ≥ 12 years with a diagnosis of eosinophilic esophagitis, had an esophageal count of ≥ 15 eosinophils/high-power field at diagnosis, and were currently prescribed treatment for eosinophilic esophagitis. RESULTS: Overall, 1001 patients were included, of whom 356 (36%) had dysphagia despite treatment. Demographics and clinical characteristics were similar in both populations. The severity of eosinophilic esophagitis was mild in more patients overall (69%) versus those with dysphagia despite treatment (48%). Patient disease history was similar in both populations, with some exceptions: common patient-reported symptoms were dysphagia (70% and 86%) and heartburn/acid reflux (55% and 49%), and common physician-reported symptoms were dysphagia (75% and 91%) and food impaction (46% and 52%). Treatment history was similar in both populations; overall, the most common treatments were proton pump inhibitors (83%) and topical corticosteroids (51%). Patients reported slightly more days with symptoms, higher impacts on activities of daily living, and slightly higher anxiety or depression in the dysphagia-despite-treatment population versus the overall population. CONCLUSIONS: Eosinophilic esophagitis presents severe symptoms and comorbidities that substantially impact patients' well-being and quality of life. Greater awareness of and novel treatments for eosinophilic esophagitis are needed.

Measuring patient-reported distress from breast magnetic resonance imaging: Development and validation of the MRI-related distress scale (MRI-DS).

OBJECTIVE: Although breast magnetic resonance imaging (MRI) is a valuable screening tool, breast MRI testing burden was associated with cancer worry and quality of life. We aimed to develop and validate the MRI-related distress scale (MRI-DS) to assess comprehensive distress specifically related to breast MRI. METHODS: We enrolled women aged above 18 years, diagnosed breast cancer, had MRI examination at least one time, and who could speak and read Korean in phase I and enrolled women aged above 18 years, visited outpatient clinic of breast general surgery, had undergone MRI examination at least once, and could speak and read Korean in phase II. We excluded patients who had any physical or psychiatric conditions in both phases. We recruited from a tertiary university-based hospital in South Korea between April and August 2023. RESULTS: All 18 items had acceptable levels of item correlation (≥0.30) in the explanatory factor analysis with a four-factor solution. The fit indices for the four-factor solution model were good. The discriminant validity of the MRI-DS had a moderate correlation with general anxiety or quality of life. In the known-group analysis, those who reported MRI as the most burden breast examination had higher total scores. CONCLUSION: The validity of the MRI-DS has been confirmed as a scale for measuring the specific distress caused by breast MRI. The MRI-DS is recommended to health professional to communicate with patients with MRI. CLINICAL IMPLICATIONS: It can be used to assess the distress associated with MRI screening in breast cancer patients. Physician could use MRI-DS to discuss the reasons for distress caused by breast MRI screening and to address specific sources of discomfort associated with it.

Prefabricated contoured foot orthoses to reduce pain and increase physical activity in people with hip osteoarthritis: A randomised feasibility trial.

BACKGROUND: Hip osteoarthritis (OA) is a prevalent and burdensome condition that leads to impaired quality of life and a substantial economic burden. Encouraging physical activity, particularly walking, is crucial for OA management, but many individuals with hip OA fail to meet recommended activity levels. Prefabricated contoured foot orthoses have shown promise in improving hip muscle efficiency during walking in laboratory settings, but their real-world feasibility and efficacy remain uncertain. OBJECTIVE: The aim of this study was to assess the feasibility of conducting a fully powered randomised controlled trial (RCT) to evaluate the effectiveness of prefabricated contoured foot orthoses, prescribed via telehealth, in people with hip OA. METHODS: This feasibility trial randomised 27 participants with hip OA into two groups: prefabricated contoured foot orthoses or flat shoe inserts. Feasibility outcomes were assessed, including recruitment rate, adherence, logbook completion, and dropout rate. Patient-reported outcomes and accelerometer-measured physical activity were collected as secondary outcomes. RESULTS: While the recruitment rate was low (0.88 people/week), adherence to the intervention (59%), logbook completion (93%), and dropout rates (7%) met or exceeded our predefined feasibility parameters. Participants found the intervention acceptable, and practicality was demonstrated with minor adverse events. Preliminary efficacy testing indicated that prefabricated contoured foot orthoses positively affected physical activity (adjusted mean difference = 2590 [260 to 4920] steps/day), with comparable outcomes for hip-related quality of life and pain. CONCLUSION: This trial supports proceeding to a fully powered RCT to assess the effect of teleheath prescribed prefabricated contoured foot orthoses on physical activity in people with hip OA. STUDY REGISTRATION NUMBER: National Institutes of Health Trial Registry (NCT05138380).

Knee Arthroscopic Surgery in Middle-Aged Patients With Meniscal Symptoms: A 10-Year Follow-up of a Prospective, Randomized Controlled Trial.

BACKGROUND: Short- and midterm evaluations of arthroscopic meniscal surgery have shown little or no effect in favor of surgery, although long-term effects, including radiographic changes, are unknown. PURPOSE: To compare the 10-year outcomes in middle-aged patients with meniscal symptoms between a group that received an exercise program alone and a group that received knee arthroscopy in addition to the exercise program with respect to the prevalence of radiographic and symptomatic osteoarthritis (OA), patient-reported outcomes, and clinical status. STUDY DESIGN: Randomized controlled trial; Level of evidence, 1. METHODS: Of 179 eligible patients aged 45 to 64 years, 150 were randomized to undergo either 3 months of exercise therapy (nonsurgery group) or knee arthroscopy in addition to the exercise therapy (surgery group). Surgery usually consisted of partial meniscectomy (n = 56) or diagnostic arthroscopy (n = 8). Radiographs were assessed according to the Kellgren-Lawrence score at the baseline and 5- and 10-year follow-ups. Patient-reported outcome measures were reported at the baseline and 1-, 3-, 5-, and 10-year follow-ups. Clinical status was assessed at a 10-year follow-up. The primary outcomes were radiographic OA and changes in the Knee injury and Osteoarthritis Outcome Score Pain subscale (KOOSPAIN) from the baseline to the 10-year follow-up. The primary analysis was performed using the intention-to-treat approach. RESULTS: At the time of the 10-year follow-up, eight patients had died, leaving 142 eligible patients. Radiographic OA was assessed for 95 patients (67%), questionnaires were answered by 110 (77%), and the clinical status was evaluated for 95 (67%). Radiographic OA was present in 67% of the patients in each group (P≥ .999); symptomatic OA was present in 47% of the nonsurgery group and 57% of the surgery group (P = .301). There were no differences between groups regarding changes from baseline to 10 years in any of the KOOS subscales. CONCLUSION: Knee arthroscopic surgery, in most cases consisting of partial meniscectomy or diagnostic arthroscopy, in addition to exercise therapy in middle-aged patients with meniscal symptoms, did not increase the rates of radiographic or symptomatic OA and resulted in similar patient-reported outcomes at the 10-year follow-up compared with exercise therapy alone. Considering the short-term benefit and no long-term harm from knee arthroscopic surgery, the treatment may be recommended when first-line treatment-including exercise therapy for ≥3 months-does not relieve patient's symptoms. REGISTRATION: Clinical Trials NCT01288768 (ClinicalTrials.gov identifier).

Psychometric properties and moderated mediation analysis of the ICIQ-NQOL in Chinese primary care patients with nocturia.

BACKGROUND: Many individuals consider nocturia a significant nuisance, leading to a reduced health-related quality of life (HRQOL). However, there has been a lack of psychometrically sound patient-reported outcome measures to assess the impact of nocturia on patients in Chinese contexts. This study aimed to translate, culturally adapt, and validate the International Consultation on Incontinence Questionnaire Nocturia Quality of Life Module (ICIQ-NQOL) for use among primary care patients in Hong Kong, China. Additionally, it sought to investigate the mechanisms that link nocturia and sleep quality with HRQOL by employing moderated mediation analysis. METHODS: The traditional Chinese version of the ICIQ-NQOL was developed through iterative translations, cognitive debriefing interviews, and panel reviews. The psychometric evaluation included assessments of factor structure, convergent validity, concurrent validity, known-group validity, internal consistency, test-retest reliability and responsiveness. Study instruments included the ICIQ-NQOL, International Prostate Symptom Score (IPSS), Pittsburgh Sleep Quality Index (PSQI), and a modified Incontinence Impact Questionnaire-Short Form (IIQ-7). RESULTS: A total of 419 primary care patients were recruited from general outpatient clinics, among whom 228 experiencing an average of two or more nocturia episodes per night over the past four weeks. Confirmatory factor analysis supported the two-factor structure of the ICIQ-NQOL. Concurrent validity was confirmed by moderate correlations between the IIQ-7 total score and the total score as well as two domain scores of the ICIQ-NQOL (r ranging from 0.43 to 0.49, all p < 0.001). The ICIQ-NQOL also had moderate correlations with the IPSS total symptom score (r ranging from 0.40 to 0.48, all p < 0.001). Convergent validity was supported by moderate correlations between the global PSQI score and the total score as well as two domain scores of the ICIQ-NQOL (r ranging from 0.42 to 0.52, all p < 0.001). Known-group comparisons showed that the ICIQ-NQOL could differentiate between patients with and without nocturia in terms of sleep/energy domain score (p < 0.001), bother/concern domain score (p < 0.001), and total score (p < 0.001), each demonstrating a moderate Cohen's d effect size. Item-total correlations corrected for overlap exceeded 0.4, and Cronbach's alpha coefficients were greater than 0.7. Test-retest reliability was confirmed with intraclass correlation coefficients exceeding 0.7 among patients reporting no change in their nocturia symptoms at a 2-week follow-up. Regarding responsiveness, the Cohen's d effect sizes for differences in domain and total scores between the baseline and 2-week follow-up assessments were greater than 0.3 among patients showing improvement in nocturia. Our moderated mediation analysis indicated that sleep quality significantly moderated the impact of nocturia on HRQOL, with a notably stronger indirect effect among females compared to males. CONCLUSIONS: The ICIQ-NQOL is a reliable and valid instrument for assessing the HRQOL in primary care patients suffering from nocturia. The findings advocate for gender-specific approaches in the management and treatment of nocturia to optimize HRQOL.

Agreement between child self-report and parent-proxy report for functioning in pediatric chronic pain.

PURPOSE: Accurate assessment of chronic pain and functional disability in children and adolescents is imperative for guiding pain management interventions. Parents have multifaceted roles in their child's pain experience and frequently provide parent-proxy reports of pain-related functioning. However, cross-informant variance is often observed with limited understanding of contributing factors. This study aims to examine the degree of alignment between child and parent-proxy reports for Patient-Reported Outcomes Measurement Information System (PROMIS) pain interference domain among children with chronic pain and to identify factors associated with improved child-parent agreement. METHODS: This study includes a sample of 127 youth (66.1% female) with mixed etiology chronic pain, ranging in age from 8 to 17 (M = 12.24; SD = 1.598), and their parent. Data was collected at an interdisciplinary pediatric pain clinic and online peer support groups. Measures of demographic, pain intensity, and functioning were collected. RESULTS: Means of parent-proxy reports were significantly lower than child self-reports on the PROMIS (p < 0.05). A statistically significant association between child's pain intensity (ß = 0.953, P < 0.05) and the difference between child self-reported and parent-proxy reported PROMIS functional interference scores was found. CONCLUSION: Parents underestimated pain-related functional disability relative to children's self-reports. The difference between the paired child self-report and parent-proxy report of functional disability was significantly associated with greater child self-reported pain intensity. Although parent-proxy reports in pediatric chronic pain is often used in research and practice, findings underscore the importance of incorporating child and adolescent self-report, when possible, to comprehensively capture the child's pain experience and best inform clinical interventions.

Validity of Patient-Reported Outcome Measures in Evaluating Nerve Damage Following Chemotherapy.

Importance: Chemotherapy-induced peripheral neuropathy (CIPN) is a substantial adverse effect of anticancer treatments. As such, the assessment of CIPN remains critically important in both research and clinic settings. Objective: To compare the validity of various patient-reported outcome measures (PROMs) with neurophysiological and sensory functional measures as the optimal method of CIPN assessment. Design, Setting, and Participants: This cohort study evaluated participants treated with neurotoxic chemotherapy across 2 cohorts using a dual-study design. Participants commencing treatment were assessed prospectively at beginning of neurotoxic treatment, midtreatment, and at the end of treatment. Participants who completed treatment up to 5 years prior were assessed cross-sectionally and completed a single assessment time point. Participants were recruited from oncology centers in Australia from August 2015 to November 2022. Data analysis occurred from February to November 2023. Exposures: Neurotoxic cancer treatment including taxanes, platinums, vinca-alkaloids, proteasome inhibitors, and thalidomide. Main Outcomes and Measures: CIPN was assessed via PROMs (European Organization for Research and Treatment of Cancer Quality of Life Questionnaire [EORTC-CIPN20], Functional Assessment of Cancer Therapy/Gynecological Cancer Group Neurotoxicity Questionnaire (FACT/GOG-Ntx), and the patient-reported outcomes version of the Common Terminology Criteria for Adverse Events [PRO-CTCAE]), neurological and neurophysiological assessment (Total Neuropathy Score and sural and tibial compound nerve amplitudes), and sensory measures (Grating orientation, Von Frey monofilament, and 2-point discrimination tasks). Core measurement properties of CIPN outcome measures were evaluated. Convergent and known-groups validity was assessed cross-sectionally following treatment completion, and responsiveness was evaluated prospectively during treatment. Neurological, neurophysiological, and sensory outcome measure scores were compared between those who reported high and low levels of CIPN symptoms using linear regressions. Results: A total of 1033 participants (median [IQR] age, 61 [50-59] years; 676 female [65.4%]) were recruited to this study, incorporating 1623 assessments. PROMs demonstrated best ability to accurately assess CIPN (convergent validity), especially the PRO-CTCAE composite score (r = 0.85; P < .001) and EORTC-CIPN20 (r = 0.79; P < .001). PROMS also demonstrated the best ability to discriminate between CIPN severity (known-groups validity) and to detect changes at onset of CIPN development (responsiveness), especially for EORTC-CIPN20 (d = 0.67; 95% CI, 0.52-0.83), FACT/GOG-Ntx (d = 0.65; 95% CI, 0.49-0.81) and the PRO-CTCAE (d = 0.83; 95% CI, 0.64-1.02). Other measures did not achieve threshold for convergent validity (α < 0.7). Neurophysiological and sensory measures did not demonstrate acceptable responsiveness. In regression models, neurological, neurophysiological, and sensory outcome measures were significantly impaired in participants who reported high levels of CIPN symptoms compared with those who reported low levels of CIPN symptoms. Conclusions and Relevance: In this cohort study of 1033 cancer patients, PROMs were the only measures to satisfy all 3 core measurement property criteria (convergent validity, known-groups validity, and responsiveness). These findings suggest that adoption of PROMs in clinical practice can equip clinicians with valuable information in assessing CIPN morbidity.

Validity, reliability, responsiveness, and clinically meaningful change threshold estimates of the National Comprehensive Cancer Network-Functional Assessment of Cancer Therapy-Breast Cancer Symptom Index (NFBSI-16).

BACKGROUND: Breast cancer is one of the most common cancers in women. Patient-reported outcome measures are used to evaluate patients' health-related quality of life in clinical breast cancer studies. This study evaluated the structure, validity, reliability, and responsiveness of the National Comprehensive Cancer Network-Functional Assessment of Cancer Therapy-Breast Cancer Symptom Index (NFBSI-16) subscales in a clinical trial featuring patients with advanced/metastatic breast cancer (aBC), and estimated NFBSI-16 meaningful change thresholds. METHODS: Data from 101 patients with aBC enrolled in a phase II trial (Xenera-1) were included for psychometric evaluation of the NFBSI-16. Subscale structure was evaluated by assessing inter-item correlations, item-total correlations, and internal consistency (cycles 2 and 5). Validity was assessed using scale-level convergent validity (cycles 2 and 5) and known-groups (Baseline). Reliability was analysed via test-retest at cycles 3-4, and responsiveness to improvement and worsening was evaluated at cycles 5, 7, and 9. Meaningful change thresholds were estimated using anchor-based methods (supported by distribution-based methods) at cycles 5, 7, and 9. RESULTS: NFBSI-16 internal consistency was acceptable, but item-total correlations suggested that its subscales and the GP5 item (side-effect of treatment) scores may be preferred over a total score. Convergent and known-groups evidence supported NFBSI-16 validity. Test-retest reliability was good to excellent for Total and DRS-P (disease-related symptoms: physical) scales, and moderate for the GP5 item. Responsiveness to worsening was generally demonstrated, but responsiveness to improvement could not be demonstrated due to limited observed improvement. Anchor-based meaningful change thresholds were estimated for DRS-P and Total scores. CONCLUSION: This study provides evidence that the NFBSI-16 has desirable psychometric properties for use in clinical studies in aBC. It also provides estimates of group- and individual-level meaningful change thresholds to facilitate score interpretation in future aBC research.