Patient-reported Outcomes Research Articles

Improving the Implementation of Patient-Reported Outcome Measure in Clinical Practice: Tackling Current Challenges With Innovative Digital Communication Technologies

Implementation of patient-reported outcome measures (PROMs) in clinical practice is challenging. We believe effective communication is key to realizing the clinical benefits of PROMs. Communication processes for PROMs in clinical practice typically involve (1) health care professionals (HCPs) inviting patients to complete PROMs, (2) patients completing PROMs, (3) HCPs and patients interpreting the resulting patient-reported outcomes (PROs), and (4) HCPs and patients using PROs for health management. Yet, communication around PROMs remains underexplored. Importantly, patients differ in their skills, knowledge, preferences, and motivations for completing PROMs, as well as in their ability and willingness to interpret and apply PROs in managing their health. Despite this, current communication practices often fail to account for these differences. This paper highlights the importance of personalized communication to make PROMs accessible to diverse populations. Personalizing communication manually is highly labor-intensive, but several digital technologies can offer a feasible solution to accommodate various patients. Despite their potential, these technologies have not yet been applied to PROMs. We explore how existing principles and tools, such as automatic data-to-text generation (including multimodal outputs like text combined with data visualizations) and conversational agents, can enable personalized communication of PROMs in practice.

Creation of Text Vignettes Based on Patient-Reported Data to Facilitate a Better Understanding of the Patient Perspective: Design Study.

Patient-reported outcome (PRO) data refer to information systematically reported by patients, or on behalf of patients, without the influence of health care professionals. It is a focal point of the health care system's ambition toward becoming more involving and personalized. It is recognized that PROs provide valuable data. However, despite this recognition, there are challenges related to both patients' and clinicians' accurate interpretations of the quantitative data. To overcome these challenges, this study explores text vignettes as a representation of PROs. This study aimed to develop data-informed text vignettes based on data from the Readiness and Enablement Index for Health Technology (READHY) instrument as another way of representing PRO data and to examine how these are perceived as understandable and relevant for both patients and clinicians. The text vignettes were created from participant responses to the READHY instrument, which encompasses health literacy, health education, and eHealth literacy. The text vignettes were created from 13 individual text strings, each corresponding to a scale in the READHY instrument. This study consisted of 3 sequential parts. In part 1, individuals with chronic obstructive pulmonary disease completed the READHY instrument, providing data to be used to create vignettes based on cluster profiles from the READHY instrument. Part 2 focused on the development of scale-based strings representing all READHY dimensions, which were evaluated through iterative cognitive interviews. In part 3, clinicians and patients assessed the understanding and relevance of the text vignettes. Clinicians and patients both understood and related to the text vignettes. Patients viewed the text vignettes as an accurate reflection of their PRO responses, and clinicians perceived the text vignettes as aligned with their understanding of patients' experiences. Text vignettes can be developed using PRO instruments, with individual scales as input strings. This provides an opportunity to present numeric values in a text format that is understandable and recognizable to most patients and clinicians. Challenges with the vignette's language and layout require customization and clinician training to ensure meaningful interpretation. Findings also support the need to expand the study and enhance clinical relevance with alternative or contextually relevant text vignettes.

Surgical Adverse Events for Primary Tumors of the Spine and Their Impact on Outcomes: An Observational Study From the Primary Tumors Research and Outcomes Network.

Aggressive resection for primary tumors of the spine are associated with a high rate of adverse events (AEs), but the impact of AEs on patient-reported outcomes (PROs) remains unknown and is critical to the shared decision-making. Our primary objective was to assess the impact of surgical AEs on PROs using an international registry. Assessing the impact on clinical outcomes and identifying risk factors for AEs were our secondary objectives. Patients who underwent surgery for a primary spinal tumor were selected through the Primary Tumor Research and Outcomes Network. Our primary outcome was the impact of AEs on PROs at 3 and 12 months after surgery (measured with Spinal Oncology Study Group Outcomes Questionnaire, Short-Form 36, and EuroQol 5 Dimension). We also assessed the impact on clinical outcomes (local control, surgical margins, readmission, reoperation, and mortality). We stratified our results according to severity of AEs, histology, and type of resection. 374 patients met inclusion criteria (219 males/155 females). The mean age of the cohort was 48.7 years. The most frequent histology was chordoma (37.3%) followed by chondrosarcoma (8.8%). Sixty-seven patients (17.9%) experienced at least 1 intraoperative AE and 117 patients (31.3%) had at least 1 postoperative AE within 3 months. Overall, 159 patients (42.5%) experienced AEs. The readmission rate was significantly higher in patients who experienced AEs (Any AE: 10.1% vs no AE: 1.9% within 3 months; P = <0.001). PROs were not significantly affected by AEs in most questionnaires. Local control, risk of reoperation, mortality, and achieving preplanned margins were similar between AE groups. The rate of surgical AEs is considerable in this population. Surgical AEs seem to be associated with a higher number of readmissions, but do not seem to result in significant differences in PROs or in a higher risk of reoperation, mortality, and failure to achieve preplanned margins.

The mere-measurement effect of patient-reported outcomes: a systematic review and meta-analysis.

The mere-measurement effect is the phenomenon in which subjects exposed to measurements have their perceptions and/or behaviors on the inquired topic affected simply through the act of responding. Patient-reported outcomes (PROs) are increasingly used to assess patient perspective and quality of life in clinical trials and different health care settings. This systematic literature review aims to assess what is currently known about the mere-measurement effect of PROs. A systematic literature review and meta-analysis was conducted. We included studies that provided evidence on perceptual or behavioral changes in patients as a result of exposure to questionnaire items assessing PROs. All adult participants were included regardless of demographics. Any study design was considered eligible for inclusion. The databases MEDLINE [PubMed], CINAHL [Ebsco], Web of Science and ScienceDirect were searched. The search resulted in 636 articles which led to a final extraction of nine. Overall, seven of the nine articles reported a significant main effect, i.e. presence of the mere-measurement effect. For the meta-analysis, thirteen different interventions were included. There was a one-directional, positive and significant overall risk ratio of 1.17 [CI95% 1.04;1.30]. This systematic review found significant potential for the mere-measurement effect to shape respondents' behaviors or perceptions for the better, opening the door to the possibility of engineering PROs to serve as a subtle intervention. Future considerations and directions for research are discussed.

Association of alcohol consumption and rotator cuff retear: a case control matched cohort study

Abstract Background Failure of healing or retear after surgical repair of the rotator cuff tendons are still a problem and can cause ongoing shoulder pain and dysfunction. Compromised microcirculation as seen in regular alcohol consumption may lead to poor healing. Purpose To compare the clinical outcomes and tendon integrity of patients after rotator cuff repair with and without regular alcohol intake. Study design Case control matched cohort study; Level of evidence, 3. Methods Patients who underwent arthroscopic rotator cuff repair (ARCR), had regular alcohol intake (according to world health organization (WHO) definition of harmful alcohol consumption) and were at least 2 years postoperative were included, and matched according to age, sex, involved tendon, and tear size with patients who underwent ARCR without regular alcohol intake. Patient-reported outcome (PRO) scores were collected at final follow-up including the Constant Murley Score (CMS), Western Ontario Rotator Cuff Score (WORC), Simple Shoulder Test (SST), and visual analog scale (VAS). Tendon integrity (maintained continuity: yes/no = full thickness) was assessed by ultrasound examination at final follow-up. Complications and revision surgeries are reported. Results Twenty-two patients (versus twenty-two matched—controls) were available for follow-up. There were two female (9%) and twenty male (91%) patients with a mean age of 66.6 years (standard deviation, 36–85 years). The mean follow-up was 4 years (standard deviation, 2–5 years) in the alcohol group and 5 years (standard deviation, 2–10 years) in the non-alcohol group. No differences in mean PRO scores between alcohol and non-alcohol groups were seen except VAS (0.5 (standard deviation, 0–5) vs. 1.6 (standard deviation, 0–8), (P = 0.049*) respectively). Intact tendon insertion was seen in 77% (17/22) for the alcohol group and 100% (22/22) for non-alcohol group, (P = 0.021*). One patient underwent revision surgery (5%) in the alcohol group due to a retear, no further peri- or postoperative complications were noticed. Conclusion Patients with torn rotator cuff tendons benefited similarly from ARCR independently of their alcohol use concerning clinical presentation. However, significantly higher retear rates were recorded in the alcohol group.

Clinical efficacy of the fractional ablative CO2 laser stacking method for depressed scars.

Depressed scars, which are unsightly outcomes of trauma or skin lesions, can lead to significant physical and mental stress. Various treatments have been developed to alleviate these burdens, among which the laser stacking method has proven effective. In this study, we compared the outcomes of laser stacking treatments with those of single-pulse ablative laser treatments for depressed scars. Eighty patients with depressed scars were divided into two therapeutic groups: the ablative laser stacking therapy group (Group 1, n = 40) and the single pulse ablative laser therapy group (Group 2, n = 40). The scars were evaluated using the modified Vancouver Scar Scale (mVSS) before and after treatment. Patient-completed questionnaires were also utilized. Comparative analyses were conducted both before and after treatment, as well as between the two groups. Eighty scars from each patient underwent the complete laser treatment protocols. Group 1 showed significantly greater improvement in the mVSS indices for pigmentation, vascularity, pliability, and height than group 2 (P < 0.05). In terms of patient-reported outcomes, group 1 rated better than group 2 across three indices: appearance, alleviation of concavity, and overall improvement (P < 0.05). Ablative laser stacking therapy effectively alleviated depressed scars, demonstrating significant improvement in both observer- and patient-reported outcomes. An energy level of 20-30mJ was employed, resulting in more favorable outcomes compared to a single pulse laser application of 10-15mJ. Our findings highlight the benefits of the stacking method, which shows notable improvements while minimizing complications. Further research involving various protocols may optimize clinical outcomes and patient satisfaction.

PREVALENCE AND PATTERNS OF OPIOID USE IN CHRONIC PANCREATITIS.

Opioids are used to treat pain in chronic pancreatitis (CP), but little is known about current use patterns. The aims of this study were to characterize the utilization of opioids, and associations with clinical characteristics in adult patients with CP. This cross-sectional analysis utilized baseline data from participants with definite CP enrolled in a cohort study in the US (PROCEED). Data on demographics, pain medication use, health care utilization, disability, and pain patterns were systematically collected in case report forms while quality of life (QOL) was assessed with patient-reported outcome instruments. Opioid use was classified according to strength (weak or strong) and frequency (scheduled or as-needed). A total of 681 participants (n=364, 53% male) were included: 299 (44%) were current opioid users (22% only weak opioids and 22% at least one strong opioid). Increasing frequency and severity of pain was associated with increase of weak, strong, as-needed, or scheduled opioids. Neuromodulators were used by ∼40% of participants; increasing use was associated with increasing frequency and severity of pain. On multivariate analysis, independent predictors associated with strength and frequency of current opioid use were pain patterns (ORs 1.84-8.32 and ORs 1.92-8.52, respectively, p<0.001) and prior celiac plexus block (OR 3.54, 95% CI 1.82-6.87) and (OR 3.42, 95% CI 1.76-6.64), respectively. Participants using opioids had higher prevalence of disability, healthcare utilization and poorer QOL. Opioid use in CP is common and associated with increased pain severity and constancy. These data provide foundational estimates for future trials that can elucidate the complex interactions between patient factors, pain, and interventions.

The Childbirth Experience Survey (CBEX): An Analysis of Qualitative Survey data.

In collaboration with community research partners, a national cross-sectional online Childbirth Experience Survey (CBEX) of pregnant and postpartum birthing people was administered in 2016. The linked antepartum-postpartum survey included items across 18 domains (e.g., labor management, pain management, newborn care and feeding), and identified 23 childbirth-specific postpartum patient-reported outcomes (PROs) that were associated with hospital satisfaction. CBEX was implemented in 16 California hospitals to identify hospital-specific opportunities for improvement in care. We analyzed postpartum qualitative survey responses (1) to evaluate the content validity to test the representativeness of existing CBEX domains, (2) to assess for any potential new domains or topics of interest within existing domains, and (3) to use these data to provide hospitals with actionable information for practice improvement. This was an analysis of qualitative survey data based on the following CBEX item: "Is there anything else you would like to share about your birth experience?" Patients could provide multiple comments. Using Atlas.ti Version 8, we mapped participant responses to the 18 existing CBEX domains. Of 525 surveys completed between Oct 2018 and Sept 2020, 172 patients responded to the qualitative item. A total of 235 comments were analyzed and all were able to be mapped to the 18 domains. Qualitative responses highlighted subtleties within several CBEX domains: (1) labor management: pressure from the care team to have a labor induction; (2) pain management: epidural effectiveness, timing, dosage, and education; (3) empathy and respect: issues related to students and residents; and (4) newborn feeding: rough physical handling of patients by nurses, specifically during lactation consults. CBEX survey data is currently being utilized in hospitals to inform childbirth-specific quality improvement initiatives. By capturing detailed voluntary participant responses, CBEX provides the opportunity to document and explore nuanced aspects of the childbirth experience and subtleties that may be contributing to maternal dissatisfaction.

Physiotherapists’ Experiences with the Hip Disability and Knee Injury Osteoarthritis Outcome Score Following Total Hip and Knee Arthroplasty: A Qualitative Interview Study

Background: Clinical guidelines for physiotherapy following total hip and knee arthroplasty (THA/TKA) recommend using Patient-Reported Outcome Measures (PROMs) like the Hip disability and Knee injury Osteoarthritis Outcome Score (HOOS/KOOS). However, it remains unclear how physiotherapists use these PROMs in daily clinical practice. Objective: To explore primary care physiotherapists’ experiences with the HOOS/KOOS in daily clinical practice following THA and TKA. Methods: Thirteen physiotherapists in the Netherlands were recruited via convenience sampling. Data were collected through semi-structured interviews, which explored HOOS/KOOS use in clinical practice, administrative regulations, and applications beyond patient care, as well as think-aloud interviews to capture perceptions of the content of these PROMs and interpretations of hypothetical patient scores. Interviews were analysed using reflexive thematic analysis. Results: The physiotherapists’ ages ranged from 25 to 54y, with annual THA/TKA volumes from 5 to 50 patients. Three themes emerged, as follows: (1) “Physiotherapists use the HOOS/KOOS for various purposes in daily clinical practice”, including complementing history taking and monitoring patient progress; (2) “Perceptions of the relevance of the HOOS/KOOS for daily clinical practice vary per item, domain, and version,” with items related to daily life activities and psychosocial factors being perceived as more valuable; and (3) “Practical aspects of HOOS/KOOS administration influence their use in daily clinical practice,” with electronic health records (EHRs) that facilitate PROM administration enhancing their use, while administrative regulations limit this. Conclusions: Physiotherapists experience HOOS/KOOS items related to daily life activities and psychosocial factors useful for history-taking and monitoring patient progress, particularly when embedded in EHRs that facilitate PROM administration.

Association among financial toxicity, depression and fear of cancer recurrence in young breast cancer patient-family caregiver dyads: an actor-partner interdependence mediation model.

This study aimed to investigate the depression situation and the mediating role of fear of cancer recurrence (FCR) in the relationship between financial toxicity and depression in young breast cancer (BC) patient-family caregiver dyads. A total of 196 young BC patient-family caregiver dyads at four hospitals in China were investigated. The Comprehensive scores for financial toxicity based on patient-reported outcome measures, the Hospital Anxiety and Depression Scale, and the FCR Inventory Short Form were assessed. The actor-partner interdependence mediation model using structural equation modelling in AMOS software was applied to examine the direct and indirect effects. In this study, there were 196 pairs of patients and family caregivers. The findings indicated a significant correlation between financial toxicity and FCR in both young BC patients and their family caregivers. Two significant partner effects were observed: the family caregiver's financial toxicity significantly influenced the patient's FCR (β=-0.450, P < 0.001), and the patient's FCR influenced the family caregiver's depression (β = 0.570, P < 0.001). Furthermore, financial toxicity in both young BC patients and family caregivers markedly affected both the actor and partner effects on dyadic depression, primarily through the patients' FCR. Depression in young BC patients was affected not only by themselves but also by their family caregivers. Emphasis should be placed on the interplay between financial toxicity and FCR of patients and family caregivers, with the aim of improving depression for young BC patients. The study emphasized the importance of addressing the experiences of both patient and family caregivers in clinical interventions. By demonstrating how financial toxicity and FCR are interlinked with depression in both parties, the study supports the development of we offer empirical support for developing comprehensive intervention strategies to alleviate mental distress and enhance mental health for patients and family caregivers.

Clinical outcome of vertebroplasty alone versus short-segment posterior instrumentation with vertebroplasty in osteoporotic vertebral fracture: a propensity-score-matched analysis.

Retrospective cohort study with propensity-score-matched analysis. To compare the efficacy of vertebroplasty (VP) versus short-segment posterior instrumentation (SS) with VP in patients with osteoporotic vertebral fractures (OVFs). OVFs significantly affect the aging population, causing pain, reduced mobility, and increased dependence. Treatment guidelines vary, and a consensus on the most effective approach remains unclear. To the best of our knowledge, no previous report focused on the efficacy comparison of VP alone versus SS with VP. The study included patients with OVFs undergoing VP with or without SS from 2017 to 2021. Baseline demographic and patient-reported outcome scores, including Oswestry Disability Index (ODI) and European Quality-of-Life-5 Dimensions (EQ-5D), were collected preoperatively and 1 year postoperatively. Radiographic outcomes, including Cobb angle, sagittal angle reduction, and kyphotic progression, were assessed. Perioperative data were gathered. Propensity-score matching was conducted to compare both groups after adjusting for baseline characteristics. This study included 60 patients. The subsequent analyses included 19 patients in both the SS+VP group and the VP groups after matching patient cohorts across various covariates. The SS+VP group demonstrated better ODI (30.38±17.12 vs. 49.68±19.43, p=0.0025) and EQ-5D scores (0.80±0.19 vs. 0.6±0.31, p=0.0018) at 1 year postoperative. Sagittal angle correction was higher in the SS+VP group (10.63°±6.34° vs. 5.74°±5.91°, p=0.0188). The SS+VP group exhibited higher blood loss and longer operative time. Perioperative complications, kyphotic progression, adjacent fractures, and reoperation rates were similar between the two groups. SS with VP generated superior patient-reported outcomes and sagittal angle correction for OVFs when evaluated one year postoperatively compared to VP alone. Perioperative complications, kyphotic progression, adjacent fractures, and reoperation rates were similar despite increased blood loss and extended operative time.

Application of Patient-Generated Health Data Among Older Adults With Cancer: Scoping Review.

The advancement of information and communication technologies has spurred a growing interest in and increased applications of patient-generated health data (PGHD). In particular, PGHD may be promising for older adults with cancer who have increased survival rates and experience a variety of symptoms. This scoping review aimed to identify the characteristics of research on PGHD as applied to older adults with cancer and to assess the current use of PGHD. Guided by Arksey and O'Malley as well as the JBI (Joanna Briggs Institute) methodology for scoping reviews, 6 electronic databases were searched: PubMed, Embase, CINAHL, Cochrane Library, Scopus, and Web of Science. In addition, the reference lists of the selected studies were screened to identify gray literature. The researchers independently screened the literature according to the predefined eligibility criteria. Data from the selected studies were extracted, capturing study, participant, and PGHD characteristics. Of the 1090 identified studies, 88 were selected. The publication trend gradually increased, with a majority of studies published since 2017 (69/88, 78%). Almost half of the studies were conducted in North America (38/88, 43%), followed by Europe (30/88, 34%). The most common setting in which the studies were conducted was the participant's home (69/88, 78%). The treatment status varied; the median sample size was 50 (IQR 33.8-84.0). The devices that were used to measure the PGHD were classified as research-grade wearable devices (57/113, 50.4%), consumer-grade wearable devices (28/113, 24.8%), or smartphones or tablet PCs for mobile apps (23/113, 20.4%). More than half of the studies measured physical activity (69/123, 56.1%), followed by patient-reported outcomes (23/123, 18.7%), vital signs (13/123, 10.6%), and sleep (12/123, 9.8%). The PGHD were mainly collected passively (63/88, 72%), and active collection methods were used from 2015 onward (20/88, 23%). In this review, the stages of PGHD use were classified as follows: (1) identification, monitoring, review, and analysis (88/88, 100%); (2) feedback and reporting (32/88, 39%); (3) motivation (30/88, 34%); and (4) education and coaching (19/88, 22%). This scoping review provides a comprehensive summary of the overall characteristics and use stages of PGHD in older adults with various types and stages of cancer. Future research should emphasize the use of PGHD, which interacts with patients to provide patient-centered care through patient engagement. By enhancing symptom monitoring, enabling timely interventions, and promoting patient involvement, PGHD have the potential to improve the well-being of older adults with cancer, contributing to better health management and quality of life. Therefore, our findings may provide valuable insights into PGHD that health care providers and researchers can use for geriatric cancer care. Open Science Framework Registry OSF.IO/FZRD5; https://doi.org/10.17605/OSF.IO/FZRD5.

Do the Provisional Paediatric Rheumatology International Trials Organisation Enthesitis/Spondylitis-Related Juvenile Idiopathic Arthritis Criteria Capture Youth With Axial Spondyloarthritis?

The Paediatric Rheumatology International Trials Organisation (PRINTO) recently undertook an effort to better harmonize the pediatric and adult arthritis criteria. These provisional criteria are being refined for optimal performance. We aimed to investigate differences between patients who did and did not fulfill these PRINTO criteria among youth diagnosed with juvenile spondyloarthritis (SpA) that met axial juvenile SpA (axJSpA) classification criteria. This was a retrospective cross-sectional sample of youth diagnosed with juvenile SpA who met the axJSpA classification criteria. Demographics, clinical manifestations, and physician and patient-reported outcomes were abstracted from medical records. Magnetic resonance imaging (MRI) scans underwent central imaging review by at least two central raters. Differences between groups were compared using Wilcoxon signed-rank test or chi-square test, as appropriate. Of 158 patients who met axJSpA criteria, 107 patients (68%) met the PRINTO provisional criteria for enthesitis/spondylitis-related arthritis. A total of 41 patients (26%) did not fulfill any of the three major PRINTO criteria due to lack of peripheral disease manifestations. Demographics, prevalence of inflammatory or structural lesions on MRI, family history of SpA, and duration of pain were not statistically different between those who did and did not meet PRINTO criteria. Those who fulfilled the PRINTO criteria had significantly more peripheral arthritis, enthesitis, and HLA-B27 positivity but reported less sacral/buttock pain. Phenotypic differences of children with axJSpA between those who were and were not classified by the PRINTO criteria were primarily due to peripheral disease manifestations and HLA-B27 positivity. Modification of the PRINTO provisional criteria may facilitate capture of youth with primarily axial disease.

Clinical Outcomes of Fully Arthroscopic Versus Arthroscopically Assisted Latissimus Dorsi Transfer for Irreparable Subscapularis Tear.

Untreated chronic subscapularis (SSC) tears pose a challenging problem to treat owing to the resultant tendon retraction, atrophy, fatty infiltration, and changes in humeral head position, which complicate surgical options. Anterior latissimus dorsi (LD) transfer has shown effective results in treating these tears without glenohumeral arthritis. This study introduces and evaluates fully arthroscopic and arthroscopically assisted anterior LD transfer techniques for reconstructing irreparable SSC tears. The authors hypothesize that both methods will yield promising and comparable clinical outcomes. Cohort study; Level of evidence, 3. This retrospective study reviewed patients who underwent anterior LD tendon transfer between February 2014 and April 2022. Indications for surgery included persistent shoulder pain and functional limitations unresponsive to nonoperative treatment, irreparable SSC tears (Lafosse grade ≥4), significant fatty infiltration (Goutallier grade ≥3), and minimal glenohumeral arthritis (Hamada stage <3). Patients were excluded if they had <2 years of follow-up or if data were lost. For comparison, the study divided patients into 2 groups based on the surgical methods: fully arthroscopic LD tendon transfer and arthroscopically assisted LD tendon transfer. Clinical assessments included pain levels (visual analog scale), Constant score, Subjective Shoulder Value score, and range of motion. Radiologic measurement and complications were assessed. The study included 34 patients (mean ± SD age, 62.4 ± 7.5 years; follow-up, 35.4 ± 15.9 months). Significant improvements were observed in all patient-reported outcomes (pre- to posttest visual analog scale, 7.5 ± 1.2 to 1.2 ± 1.6; Constant score, 28.3 ± 6.0 to 68.8 ± 15.8; Subjective Shoulder Value, 23.2 ± 8.7 to 65.3 ± 22.0; all P < .001), range of motion in all directions, and internal rotation strength. Fully arthroscopic (n = 18) and arthroscopically assisted (n = 16) LD tendon transfers produced comparable clinical outcomes. Complications included 3 retears, 3 infections, and 5 cases of arthritis progression. Fully arthroscopic and arthroscopically assisted anterior LD transfers significantly reduce pain, enhance range of motion, and strengthen internal rotation in patients with irreparable SSC tears, with no significant differences in complications. These techniques offer comparable clinical outcomes, providing different options for surgeons.

Associations among positive child health measures in the environmental influences on child health outcomes (ECHO) cohort.

Effective measurement of positive child health is critical in improving public health. A proposed measure of positive health, a positive child health index (PCHI), is based on how many of 11 specific physical, developmental, and mental health conditions a child has (ranging from 0 to 11). Accepted measures of positive health, Patient-Reported Outcome Measurement Information System (PROMIS®) measures of global health, meaning and purpose, and life satisfaction, are based on child and caregiver perceptions. The sample comprised 3713 children aged 5 to 17years from the NIH Environmental influences on Child Health Outcomes (ECHO) Cohort with data to calculate PCHI and at least 1 child- or caregiver-reported PROMIS measure. Linear regressions were performed to test the associations between each PROMIS measure T-score and the PCHI, adjusting for gestational age, child sex, child age, and maternal factors (age, education, income). The PROMIS measure associated most strongly with PCHI was caregiver-reported global health, followed by child-reported global health. Caregiver-reported life satisfaction and child-reported meaning and purpose were higher for children with a PCHI = 0 compared with children with 3 or more health conditions but not when compared with children with only 1 or 2 conditions. Among children with 4 or more conditions, girls reported lower global health than boys. Sex differences were not found for caregiver-reported measures. PROMIS measures and PCHI offer complementary information on positive child health. PROMIS measures are intended as measures of a person's perception of their health, whereas PCHI reflects a cumulative impact of chronic health conditions from the perspective of health care systems. Both viewpoints are informative in public health promotion.

Autologous bone grafting combined with spheroid-based matrix-induced autologous chondrocyte implantation for osteochondral defects of the knee: Good clinical outcomes alongside abnormal postoperative gait patterns.

This study aimed to evaluate the clinical and functional outcomes of autologous bone grafting with spheroid-based matrix-induced autologous chondrocyte implantation (MABCI) for osteochondral defects of the knee by analysing pre- and postoperative patient-reported outcome measures (PROMs). Postoperative gait analysis was conducted and compared with a matched healthy control group to investigate biomechanical deviations. A total of 35 patients (m: 21, f: 14; mean defect size: 4.2 ± 2.4 cm², localisation: femoral condyle: 31, patellofemoral: 5) were analysed. The mean follow-up was 42.6 ± 22.8 months. International Knee Documentation Committee (IKDC), Knee Injury and Osteoarthritis Outcome Score (KOOS), PROMIS 29 profile, and a questionnaire on patient perception of treatment success were assessed to evaluate PROMs. 3D-instrumented gait analysis (GRAIL, Motek) was used to assess lower extremity kinematics, kinetics and vertical ground reaction forces, compared to sex-, age- and body mass index-matched healthy controls. All clinical scores showed significant improvement compared to the preoperative condition (IKDC: 73.1 ± 10.1 vs. 56.6 ± 17.2, p < 0.01; KOOS subcategories: pain 82.0 [±12.7] vs. 70.7 [±16.7] [p < 0.01], symptoms 79.1 [±20.3] vs. 68.9 [±13.9] [p < 0.01], activities of daily living 90.1 [±11.2] vs. 80.5 [±15.6] [p < 0.01], sport and recreational function: 65.3 [±19.3] vs. 51.3 [±26.29] [p < 0.01], quality of life 52.2 [±18.6] vs. 42.6 [±18.6] [p < 0.01]; numeric pain rating scale: 2.7 ± 2.0 vs. 5.0 ± 2.5, p < 0.01). The analysed patients reported a high satisfaction rate (94.3%). Self-selected walking speed was significantly lower than in healthy controls (1.17 ± 0.17 m/s vs. 0.98 ± 0.18 m/s, p < 0.01). Peak knee flexion angle (PKA) during loading response was significantly smaller (9.6° ± 7.0 vs. 17.7° ± 4.6, p < 0.01), and knee extension moment was significantly reduced (0.1 Nm/kg ± 0.2 vs. 0.4 Nm/kg ± 0.2, p < 0.01). MABCI is an effective treatment for osteochondral knee defects, showing significant improvements in all evaluated PROMs. Postoperative gait analysis revealed abnormal gait patterns, including reduced PKA and lower knee extension moment, suggesting a need for further rehabilitation to optimise functional recovery. Level III.

Real-World Effectiveness of Risankizumab in Patients with Moderate-to-Severe Psoriasis: Interim Analysis from the VALUE Global Prospective Post-marketing Observational Study at 25Months.

Risankizumab is approved for treating moderate-to-severe psoriasis. This interim analysis at 25 months evaluated the effectiveness of risankizumab compared with other approved biologics (OtherBios) among patients with moderate-to-severe psoriasis in the 37-month VALUE post-marketing observational study. Patients diagnosed with psoriasis were enrolled in a 2:1 ratio to risankizumab or OtherBios, as prescribed by their physicians. A ≥ 90% improvement in Psoriasis Area Severity Index (PASI) 90 at months 4, 13, and 25 and the time to first treatment change at 25 months were evaluated. Additionally, PASI 100 and 75, static Physician Global Assessment (sPGA 0/1), Dermatology Life Quality Index (DLQI), and Treatment Satisfaction Questionnaire for Medication (TSQM) scores were evaluated. All patients treated with ≥ 1 dose of biological therapy with ≥ 1 post-baseline measurement were included in the analysis. Modified non-responder imputation was used to handle missing data, and propensity score matching accounted for imbalances between comparison groups. Overall, 1765 patients received risankizumab and 874 received OtherBios. At baseline, the mean (SD) age of the overall population was 48.5 (14.7) years and mean (standard deviation [SD]) PASI scores were 15.0 (9.0) and 13.9 (8.8) in the risankizumab and OtherBios groups, respectively. At 25 months, 70.9% of those treated with risankizumab vs. 51.5% of those treated with OtherBios achieved PASI 90. The cumulative treatment change probability was 0.16 (95%, confidence interval [CI] 0.14, 0.18) in the risankizumab group and 0.29 (95% CI 0.26, 0.32) in the OtherBios group. At 25 months, a higher proportion of patients achieved PASI 100 (56.6% vs. 40.2%), PASI 75 (84.3% vs. 67.7%), sPGA 0/1 (82.6% vs. 66.2%), and DLQI 0/1 (70.0% vs. 52.9%) in the risankizumab vs. OtherBios group, respectively, and the change in mean TSQM global score was higher in the risankizumab group (86.0 vs. 79.4). All comparisons were nominally significant (P < 0.0001). No new safety signals were identified. In this prospective study, risankizumab demonstrated higher effectiveness, longer drug survival, and better improvement of patient-reported outcomes at 25 months compared with OtherBios. ClinicalTrials.gov identifier: NCT03982394.

Patient-reported outcome improvements following scalp hair regrowth among patients with Alopecia Areata: analysis of the ALLEGRO-2b/3 trial

Purpose: Alopecia areata (AA), an autoimmune disorder characterized by non-scarring hair loss, is detrimental to the psychological health and quality of life of people living with AA. Clinically meaningful hair regrowth is possible, but the relationship with downstream patient-reported outcomes (PROs) is complex. Materials and methods: This post hoc analysis of ALLEGRO-2b/3 (NCT03732807) longitudinal data from Weeks 24–48 compared improvements in PROs between patients who achieved (responders) or did not achieve (non-responders) clinically meaningful scalp hair regrowth. Responders were defined by a Week 24 Severity of Alopecia Tool (SALT) score ≤20 (SALT20) or ≤10 (SALT10). Across 6 PROs assessing multiple AA-related health domains, response proportions and mean changes from baseline were estimated for Weeks 24–48. Results: Among 650 included participants, 114 (17.5%) were SALT20 responders, of which 76 (11.7%) were also SALT10 responders. Generally, more responders than non-responders reported improvements in AA and related symptoms or limitations and satisfaction with hair regrowth. Responders additionally reported greater improvement from baseline than non-responders for measures of AA-related emotional symptoms, mental health, and work or activity limitations. Conclusions: These results support a positive relationship between scalp hair regrowth and downstream PROs—including satisfaction and psychosocial burden—demonstrating an association between clinically meaningful hair regrowth and patient-reported treatment benefits.

Treatment satisfaction and patient reported outcomes among people with opioid use disorder participating in an open-label, non-randomised trial of long-acting injectable buprenorphine treatment in Australian custodial settings.

A trial of long-acting injectable buprenorphine (LAIB) in Australian prisons allowed examination of treatment satisfaction and patient-reported outcomes. UNLOC-T was a 16-week non-randomised open-label study. Men and women aged ≥18 years with moderate/severe DSM-5 opioid use disorder currently serving a custodial sentence ≥6 months were recruited. Participants not in opioid agonist treatment (OAT) commenced LAIB (n = 67); those already stable on oral methadone treatment were recruited to a comparison arm (n = 62). The Treatment Satisfaction Questionnaire for Medication (TSQM), Patient Satisfaction Visual Analogue Scale (PS-VAS) and Treatment Burden Questionnaire assessed treatment satisfaction; the Kessler Psychological Distress Scale (K10), 12-Item Short Form Health Survey (SF-12) and the Australian Treatment Outcomes Profile (ATOP) measured mental health, physical health and quality of life. Among participants receiving LAIB, TSQM global satisfaction scores significantly increased from 68.2 (SD 16.6) to 77.0 (SD 18.4) by week 16 (p = 0.0041), as did satisfaction measured by the PS-VAS (62.5 [SD 29.2] vs. 79.4 [SD 25.5], p = 0.0005). Statistically significant improvements between baseline and week 16 were also observed for K10, SF-12 (total) and SF-12 (mental health) scores. By the end of the study, 'successful' treatment outcomes were observed in the ATOP domains of psychological health (84%), physical health (80%) and quality of life (86%). Participants inducted and stabilised on LAIB reported high treatment satisfaction and improved health and wellbeing. Results suggest LAIB is acceptable to people with opioid use disorder in custody, supporting scaleup of this medication to increase coverage of OAT in these settings. https://www.anzctr.org.au ACTRN12618000942257.

The Spectrum of Functional, Structural and Patient-Reported Outcomes in Intermediate Age-Related Macular Degeneration – A MACUSTAR Study Report

Introduction: There is an unmet medical need for therapies in intermediate age-related macular degeneration (iAMD). The prospective European multi-center cohort study MACUSTAR validates structural, functional and patient-reported iAMD endpoints for use in future trials. The multiplicity of assessments allows characterizing iAMD in more dimensions than previously available. We describe the heterogeneity of assessments in the iAMD baseline cohort of the MACUSTAR study. Methods: A wide range of assessments were administered across 20 European study sites in accordance with established guidelines. These assessments encompassed multiple structural evaluations, such as color fundus photography, fundus autofluorescence, and optical coherence tomography. Additionally, functional tests were conducted, including assessments of best-corrected and low-luminance visual acuity (VA), Moorfields acuity, contrast sensitivity, reading speed, mesopic and scotopic microperimetry, and dark adaptometry. Moreover, patient-reported outcome assessments, specifically the Vision Impairment in Low Luminance questionnaire, were also incorporated into the evaluation process. Associations between variables were investigated using Phi coefficients, Pearson correlation coefficients and age-corrected regression models. Results: Five-hundred eighty-five individuals with iAMD (66% women; mean (standard deviation) age: 72±7 years) were included in the MACUSTAR study. Forty-nine percent had pigmentary abnormalities, 27% had reticular pseudodrusen; 10% and 9% had incomplete and complete retinal pigment epithelium and outer retinal atrophy at baseline, respectively. Mean best-corrected VA, low-luminance VA and mesopic average threshold on microperimetry at baseline were 0.03±0.11 logMAR, 0.24±0.16 logMAR and 23.3±3.7 dB. Mean VILL subscale scores at baseline were 2±2 to 2±3 logits. Phi coefficients between structural assessments ranged between 0.17 and 0.22 (median 0.21); correlation coefficients between function tests ranged between 0.07 and 0.69 (median 0.34) and between VILL scores ranged between 0.21 and 0.68 (median 0.23). Conclusion: The findings from this broad and comprehensive spectrum of assessments of structure, function, and patient-reported outcomes in iAMD suggest that the disease spectrum is diverse and heterogeneous and that further efforts are necessary to fully understand and characterize iAMD in all its complexities. A further in-depth characterization will enable novel enrichment strategies for clinical trials in iAMD. Trial registration: ClinicalTrials.gov, NCT03349801. Registered on November 22, 2017