Changes In Patient-reported Outcomes Research Articles

Association between weight reduction achieved with tirzepatide and quality of life in adults with obesity: Results from the SURMOUNT-1 study.

The SURMOUNT-1 trial investigated effects of tirzepatide, a glucose-dependent insulinotropic polypeptide and glucagon-like peptide-1 receptor agonist, on body weight in participants with obesity or overweight. This analysis evaluated changes in patient-reported outcomes (PROs) assessing physical function, psychosocial well-being, and overall health aspects of participants' health-related quality of life (HRQoL) in SURMOUNT-1. PRO instruments included the Impact of Weight on Quality of Life-Lite Clinical Trials version (IWQOL-Lite-CT), Short Form Survey-36 version 2 (SF-36v2) and EQ-5D-5L. Scores were analysed by treatment group and by categorical degree of weight reduction group: >0 to <5%, ≥5 to <10%, ≥10 to <20% and ≥20%. Relevant PROs were evaluated for participants with or without physical or psychosocial limitations at baseline, as measured by Patient Global Impression of Status for physical activity (PGIS) and Patient Health Questionnaire-2 (PHQ-2), respectively. All tirzepatide groups demonstrated significant improvements in PRO scores versus placebo. There was a consistent trend of incremental PRO improvement with greater degrees of weight reduction, starting from ≥5% weight reduction. Participants achieving ≥20% weight reduction demonstrated the greatest changes from baseline to week 72 (SF-36v2 Physical Component Summary, 4.60; SF-36v2 Mental Component Summary, 0.80; IWQOL-Lite CT Total score, 24.7). Those with baseline physical and psychosocial limitations experienced greater improvements than those without. Tirzepatide treatment was associated with improved HRQoL compared to placebo in people with overweight or obesity. Higher percentages of weight reduction were associated with greater improvements. Clinical trial registration number for SURMOUNT-1: NCT04184622.

Favorable change in patient-reported outcomes following peroneus longus to brevis tendon transfer and lateral ankle ligament reconstruction

BackgroundA peroneus longus to brevis tendon transfer is recommended for a severely torn peroneus tendon, but there is little research on the outcome. We conducted a prospective cohort study to examine patient-reported outcomes after this procedure. MethodsThirty-two patients underwent a peroneus longus to brevis tendon transfer and lateral ankle ligament reconstruction, 11 had an additional calcaneal osteotomy. The Foot and Ankle Outcome Score (FAOS) and Short Form-36 (SF-36) were assessed preoperatively, six and 12 months after surgery. ResultsPreoperative mean FAOS was 51.7 (SD 17.8) compared with 72.7 (SD 21.2) at 12 months, an improvement of 21 (95 % CI 12.7–28.0) (p < 0.0001). SF-36 improved significantly in the three domains involving physical function and bodily pain (p < 0.007). ConclusionPatient-reported outcomes improved significantly through peroneus longus to brevis tendon transfer. This procedure is worth considering for patients with a severely damaged peroneus tendon. Level of evidenceLevel II: Prospective cohort study

Longitudinal changes in patient-reported outcomes (PROs) among early phase clinical trial (EP-CT) participants.

243 Background: Little research has explored associations of longitudinal changes in PROs and clinical outcomes among EP-CT participants. We sought to describe changes in PROs over the first month of EP-CT participation and examine associations of changes with clinical outcomes. Methods: We prospectively enrolled adults participating in EP-CTs at Massachusetts General Hospital from 04/2021-01/2023. We assessed quality of life (QOL) (Functional Assessment of Cancer Therapy-General, with subdomains: physical wellbeing [PWB], emotional wellbeing [EWB], social wellbeing [SWB], functional wellbeing [FWB]); hope (Herth Hope Index); symptom burden (Edmonton Symptom Assessment System); and financial wellbeing (Comprehensive Score for Financial Toxicity) at time of EP-CT enrollment and one month later (1-month). We used descriptive statistics and regression models to describe 1-month changes in PROs and explore associations of patient-reported QOL, hope, symptom burden, and financial wellbeing with patient factors (age, diagnosis, performance status) and clinical outcomes (time on trial [TOT], treatment response, ED visits, hospitalizations, and survival). Results: Of 205 enrolled patients, 159 (77.9%) had baseline and 1-month data evaluable for analysis (median age: 63.0 [range 33.0 – 88.6], 59.8% female, 93.6% metastatic cancer, ECOG 0= 46.2% and ECOG 1=53.8%). Most common cancer types were gastrointestinal (31.5%) and breast (22.0%). There was no significant change from baseline to 1-month in mean QOL (75.2 to 76.2), hope (27.1 to 26.7), ESAS total symptom burden (20.4 to 20.9), ESAS physical symptoms (13.3 to 13.7), and financial wellbeing (28.5 to 28.0). There was no significant change in overall QOL and in each subdomain with the exception of EWB (16.2 to 17.0, p&lt;.001). Older age was associated with increased hope (B=1.29, p=.008) and increased QOL (B=3.02, p=0.024). Increasing EWB at 1-month was associated with longer TOT (HR=0.93, p=.011). Increased symptom burden at 1-month was associated with greater risk of ED visits (HR=1.04, p=.005) and hospitalizations (HR=1.03, p=.012). Improving financial wellbeing at 1-month was associated with lower risk of hospitalization (HR=0.96, p=.046). We found no other significant associations of 1-month changes in PROs with treatment response or survival. Conclusions: We described changes in PROs from baseline to 1-month and identified factors associated with these changes. Despite the rigors and intensity associated with EP-CT initiation, most PROs did not worsen, with significantly improved EWB after 1-month of participation. Results demonstrate changes in physical symptoms predicted for greater risk of ED visits and hospitalizations. Future studies should build upon this work and measure PROs throughout trial participation to identify factors associated with changes and guide intervention development.

Incidence of revision surgery and patient-reported outcomes within 5 years of the index procedure for grade 1 spondylolisthesis: an analysis from the Quality Outcomes Database spondylolisthesis data.

Some patients treated surgically for grade 1 spondylolisthesis require revision surgery. Outcomes after revision surgery are not well studied. The objective of this study was to determine how revision surgery impacts patient-reported outcomes (PROs) in patients undergoing decompression only or decompression and fusion (D+F) for grade 1 spondylolisthesis within 5 years of the index surgery. Patients in the 12 highest Quality Outcomes Database (QOD) enrolling sites with a diagnosis of grade 1 spondylolisthesis were identified and the incidence of revision surgery between the decompression-only and D+F patients were compared. PROs were compared between cohorts requiring revision surgery versus a single index procedure. Of 608 patients enrolled, 409 had complete 5-year data available for this study. Eleven (13.3%) of 83 patients underwent revision in the decompression-only group as well as 32 (9.8%) of 326 in the D+F group. For the entire cohort, patients requiring revision had significantly worse PROs at 5 years: Oswestry Disability Index (ODI) 27.4 versus 19.4, p = 0.008; numeric rating scale for back pain (NRS-BP) 4.1 versus 3.0, p = 0.013; and NRS for leg pain (NRS-LP) 3.4 versus 2.1, p = 0.029. In the decompression-only group, the change in 5-year PROs was not impacted by revision status: ODI 31.9 versus 24.2, p = 0.287; NRS-BP 1.9 versus 2.9, p = 0.325; and NRS-LP 6.2 versus 3.7, p = 0.011. In the D+F group, the change in 5-year PROs was diminished if patients required revision: ODI 19.1 versus 29.1, p = 0.001; NRS-BP 3.0 versus 4.0, p = 0.170; and NRS-LP 2.3 versus 4.6, p = 0.001. The most common reasons for reoperation within 5 years in the decompression-only group were repeat decompression and instability, whereas in the D+F group the most common reason was adjacent-segment disease. The need for revision resulted in modestly diminished benefit compared with patients with no revisions. These differences were greater in the fusion cohort compared with the decompression-only cohort. The mean PRO improvement still far exceeded minimal clinically important difference thresholds for all measures for patients who underwent a revision surgery.

Changes in patient-reported outcomes during admission to a South African psychiatric facility.

Patient-reported outcome measures (PROMs) are used as part of clinical practice to determine the impact of the condition and treatment interventions on a patient's health and quality of life. The Patient Health Questionnaire-9 (PHQ-9) is a self-administered diagnostic tool that has been widely adopted for the detection and monitoring of depression. This analysis reports the change in PHQ-9 scores from admission to discharge in patients admitted for depression to a South African acute psychiatric facility and aims to quantify the treatment effect of the admission using the PHQ-9 as the measurement tool. South African acute psychiatric facility. This was a retrospective observational study of all patients admitted to Netcare Akeso acute psychiatric facilities from 01 January 2018 to 31 October 2022. Patients were included if they were ≥ 18 years of age, admitted with a primary International Classification of Disease (ICD)-10 code for depression (i.e. F32-F33) and fully completed both an admission and discharge PHQ-9 questionnaire. We excluded facilities focusing only on the treatment of patients with specialised conditions such as addiction or eating disorders. This analysis included 13 308 patients admitted for depression at 10 different facilities. The median PHQ-9 score on admission was 19 (interquartile range [IQR] 14-23) and 5 (IQR 2-11) on discharge, with a median change of -12 (IQR -5 to -18). A minimal clinically important difference was seen in 87.6% patients (n = 10 091/11 515); a treatment effect was seen in 74.5% of patients and a clinically significant improvement was seen in 72.1% of patients. With the average patient reporting a four-fold reduction in the severity of their depression scores, PROMs provide a critical patient-centred window into the benefit that an inpatient admission has on those suffering with depression. These changes are consistent with those seen internationally and provide a baseline for understanding the treatment efficacy of an inpatient admission for the treatment of depression.

Effectiveness of Treating Obstructive Sleep Apnea by Surgeries and Continuous Positive Airway Pressure: Evaluation Using Objective Sleep Parameters and Patient-Reported Outcomes.

Background/Objectives: Uvulopalatopharyngoplasty (UPPP), palatal plus nasal surgery (PNS), and continuous positive airway pressure (CPAP) are widely implemented treatments for obstructive sleep apnea (OSA). This study aims to explore the long-term effects on objective sleep parameters and patient-reported outcomes (PROs) following different therapeutic interventions for OSA. Methods: Data from patients with moderate-to-severe OSA were retrospectively collected from a medical center and a regional hospital, spanning from December 2011 to August 2018. Objective evaluations included the Apnea-Hypopnea Index (AHI), minimum O2 saturation, and sleep efficiency. The PROs consisted of the Snore Outcomes Survey and Epworth Sleepiness Scale. Using mixed-effects models, we evaluated longitudinal changes in sleep parameters and PROs, accounting for repeated measures and variations within individuals over time. Results: Among 448 patients with moderate-to-severe OSA, follow-up data were collected for 42 patients undergoing UPPP surgery, 171 undergoing PNS, 127 using CPAP, and 108 in the non-treated group. The mean follow-up was 16.7 months (SD = 11.9, range: 1.6-77.3). Significant improvements were observed in AHI, minimum O2 saturation, and hypersomnia immediately following interventions with UPPP, PNS, and CPAP therapy (p < 0.05). Moreover, the analysis revealed no significant rate of change in these parameters over time, suggesting that the benefits of these treatments were sustained in the long term. Furthermore, all interventions exhibited a significant short-term effect on self-reported snoring when compared to the control group, with a p-value of less than 0.001. However, the magnitude of this improvement gradually decreased over time. The snore scores seemed to return to pre-treatment levels among the UPPP, PNS, and CPAP groups after averages of 46.4, 63.5, and 74.4 months, respectively (all p < 0.05). Conclusions: Surgical interventions and CPAP therapy showed potential long-term effectiveness in managing OSA. Snoring symptoms reappeared about 3.9-5.3 years after surgical treatments, which seemed earlier than the average of 6.2 years in patients receiving CPAP and should be considered in patient-participatory decision-making processes.

Impact of Integrative Health and Medicine on Costs Associated with Adult Health System Beneficiaries with Musculoskeletal Conditions: A Retrospective Cohort Study.

Objective: Owing to perceived additional costs, patients may avoid integrative health and medicine (IHM) treatments, while insurers may not cover IHM. We hypothesized that adult beneficiaries of a health system's employee insurance plan with musculoskeletal (MSK) conditions receiving covered outpatient IHM would have reduced total allowed costs over the 1-year follow-up compared with matched controls, secondarily exploring medical and pharmaceutical cost subsets. Methods: We queried medical records and claims spanning 2018-2023 for beneficiaries aged 18-89 years with a new MSK episode. Patients were divided into cohorts: (1) IHM within 3 months after MSK diagnosis and (2) no IHM after initial primary care. After inflation adjustment and trimming, propensity score matching was used to balance cohorts on demographics, comorbidity, health care utilization, and prior 12-month spend. Least-squares mean total, medical, and pharmaceutical allowed costs (United States Dollar) over the 1-year follow-up were analyzed using a linear mixed model. Findings were compared with a generalized linear model without trimming. Results: There were 251 patients per matched cohort, with adequate covariate balance. There was no meaningful between-cohort difference (IHM minus No IHM) in least-squares mean total cost (+703 [95% CI: -314, 1720]). Secondary outcomes included medical cost (+878 [95% CI: 61, 1695]) and pharmaceutical cost (+6 [95% CI: -71, 83]). A generalized linear model revealed no meaningful difference in estimated mean total medical costs (-2561 [95% CI: -7346, +2224]). Conclusions: IHM use among adult health system beneficiaries with MSK conditions was not associated with meaningful differences in 1-year follow-up total health care costs compared with matched controls. Our study was underpowered for secondary outcomes, which should be interpreted with caution. Future research should include a larger sample of patients and examine longitudinal changes in patient-reported outcomes.

Do class III obese patients achieve similar outcomes and satisfaction to nonobese patients following surgery for cervical myelopathy? A QOD study.

The aim of this study was to compare the rate of achievement of the minimal clinically important difference (MCID) in patient-reported outcomes (PROs) and satisfaction between cervical spondylotic myelopathy (CSM) patients with and without class III obesity who underwent surgery. The authors analyzed patients from the 14 highest-enrolling sites in the prospective Quality Outcomes Database CSM cohort. Patients were dichotomized based on whether or not they were obese (class III, BMI ≥ 35 kg/m2). PROs including visual analog scale (VAS) neck and arm pain, Neck Disability Index (NDI), modified Japanese Orthopaedic Association (mJOA), EQ-5D, and North American Spine Society patient satisfaction scores were collected at baseline and 24 months after cervical spine surgery. Of the 1141 patients with CSM who underwent surgery, 230 (20.2%) were obese and 911 (79.8%) were not. The 24-month follow-up rate was 87.4% for PROs. Patients who were obese were younger (58.1 ± 12.1 years vs 61.2 ± 11.6 years, p = 0.001), more frequently female (57.4% vs 44.9%, p = 0.001), and African American (22.6% vs 13.4%, p = 0.002) and had a lower education level (high school or less: 49.1% vs 40.8%, p = 0.002) and a higher American Society of Anesthesiologists grade (2.7 ± 0.5 vs 2.5 ± 0.6, p < 0.001). Clinically at baseline, the obese group had worse neck pain (VAS score: 5.7 ± 3.2 vs 5.1 ± 3.3), arm pain (VAS score: 5.4 ± 3.5 vs 4.8 ± 3.5), disability (NDI score: 42.7 ± 20.4 vs 37.4 ± 20.7), quality of life (EQ-5D score: 0.54 ± 0.22 vs 0.56 ± 0.22), and function (mJOA score: 11.6 ± 2.8 vs 12.2 ± 2.8) (all p < 0.05). At the 24-month follow-up, however, there was no difference in the change in PROs between the two groups. Even after accounting for relevant covariates, no significant difference in achievement of MCID and satisfaction was observed between the two groups at 24 months. Despite the class III obese group having worse baseline clinical presentations, the two cohorts achieved similar rates of satisfaction and MCID in PROs. Class III obesity should not preclude and/or delay surgical management for patients who would otherwise benefit from surgery for CSM.

Impact of preoperative back pain severity on PROMIS outcomes following minimally invasive lumbar decompression.

To evaluate the effect of baseline back pain severity on PROMIS mental health outcomes following minimally invasive lumbar decompression (LD). Patients undergoing elective, primary, single-level LD were retrospectively reviewed from a prospective single spine surgeon registry. Perioperative characteristics, demographics, and the following patient-reported outcomes (PROs) were extracted: Oswestry Disability Index (ODI)/Patient-Health Questionnaire-9 /PROMIS-Physical Function/Anxiety/Pain Interference/Sleep Disturbance (PROMIS-PF/A/PI/SD). Two cohorts were created: preoperative VAS-B < 7 and VAS-B ≥ 7. Change in PROs (ΔPROs) from baseline to six weeks/final follow-up were determined. Average patient follow-up was 13.4 ± 8.8months. Minimal clinically important difference (MCID) achievement rates were calculated and compared through multivariable logistic regression. Postoperative scores and ΔPROs, were compared with multivariable linear regression while all other data was compared between groups with inferential statistics. Altogether, 347 patients were included, with 190 in the VAS-B < 7 group. VAS-B ≥ 7 reported worse outcomes preoperatively (p ≤ 0.013, all). At six weeks, VAS-B ≥ 7 reported worse VAS-B (p = 0.017), with no other significant differences. At final follow-up, patients with worse VAS-B reported worse ODI (p = 0.040) and VAS-B while all other PROs were similar (p ≥ 0.078, all). VAS-B ≥ 7 experienced greater 6-week improvements in VAS-B/ODI/PROMIS-PI/PROMIS-SD (p ≤ 0.009, all), greater VAS-B/ODI/PROMIS-SD improvement by final follow-up (p ≤ 0.009, all) and greater MCID achievement in ODI/VAS-B (p ≤ 0.027). Patients with worse baseline back pain report inferior baseline scores that converge with those with milder preoperative back pain by 6weeks after LD and reported greater 6-week improvements in disability, pain interference, and sleep disturbance by 6weeks, and greater improvements in disability and sleep disturbance by final follow-up.

Comparison of Patient-Reported Outcomes for Immediate Unrestricted Weightbearing Versus Restricted Rehabilitation Protocols After Osteochondral Allograft Transplantation to the Distal Femur.

There is no standardized rehabilitation protocol after osteochondral allograft (OCA) transplantation surgery to the distal femur. The spectrum of recommendations includes restrictions to toe-touch weightbearing (TTWB) for 6 weeks and immediate weightbearing as tolerated (WBAT). The purpose of this study was to compare outcomes for immediate unrestricted WBAT to restricted TTWB after OCA transplantation to the distal femur. It was hypothesized that the immediate WBAT protocol would be noninferior to delayed, restricted TTWB. Retrospective cohort study. A total of 74 patients who underwent press-fit, dowel technique OCA transplantation to the femoral condyle(s) for contained (International Cartilage Repair Society grade 3-4) lesions were identified in the Metrics of Osteochondral Allograft multicenter database: 36 patients (18 women/18 men) who were prescribed TTWB were allocated to the control cohort and 38 patients (21 women/17 men) who were prescribed WBAT were allocated to the test cohort. Baseline characteristics were similar except for larger grafts in test patients (3.4 vs 2.7 cm2; P = .004) and higher body mass index (BMI) in control patients (27.8 vs 24.9 kg/m2; P = .01). Failure rates, final patient-reported outcome (PRO) scores, and PRO score changes from baseline were compared between the cohorts. Multiple regression was used to control for potential confounders and investigate noninferiority using minimal clinically important differences (MCIDs). The mean follow-up was 2 years (range, 1-5 years) in both cohorts. Both cohorts showed significant improvement in all PRO scores, with no significant between-group differences in failure rates, final PRO scores, or PRO changes from baseline. There were 3 cases of failure in each cohort (control cohort: allograft revision [n = 2], debridement [n = 1]; test cohort: chondroplasty [n = 2], conversion to total knee arthroplasty [n = 1]). Regression analysis showed that adjusted differences in final PRO scores based on weightbearing protocol were minor and less than MCIDs when controlling for age, sex, graft size, BMI, and allograft location. Analysis of the MCIDs with respect to the lower bounds of the confidence intervals indicated that WBAT was noninferior to TTWB with a reasonable degree of confidence (range, 84.1%-99.9% confidence). Results indicated that immediate unrestricted WBAT after OCA transplantation to the distal femur was equally safe and effective compared to restricted TTWB.

Post-Partum Clinical and Patient-Reported Outcome Changes in Mothers with Multiple Sclerosis: Findings from the NAPPREMS Study.

Background and Objective: Pregnancy in mothers with multiple sclerosis (MS) commonly results in significant changes in disease activity and changes in clinical care, including the discontinuation of disease modifying therapy (DMT). This study aimed at understanding the clinical and patient-reported outcomes (PROs) before, during and 1-year after delivery. Materials and Methods: A total of 30 pregnant mothers with MS were recruited as part of the study. Clinical (relapse activity and disability changes), PRO information and MRI outcomes were collected on four separate visits: one baseline visit-0-30 days post-delivery; and 3 follow-up visits at week 24, week 36 and week 52 from the baseline. PRO was assessed using a validated questionnaire called the Fatigue Scale for Motor and Cognitive Function (FSMC). The MRI scans were analyzed, and the count of new T2 lesions and/or contrast-enhancing lesions was determined. Results: The average time between delivery and the start of DMT was 142.5 days. Relapse activity before the pregnancy was numerically linked with the activity during the pregnancy, where up to 57.1% of the activity during pregnancy occurred in pwMS with previously active disease before conception (statistically trending with p = 0.073). The relapse activity after the pregnancy occurred twice as often in pwMS whose MS was clinically active before conception. All five pwMS who experienced a relapse prior to the pregnancy experienced worsening in their physical PRO domain. Conclusions: Pre-pregnancy activity is crucial in the screening of mothers with MS at risk for post-partum relapses, worsening of clinical disability and/or PRO measures. A post-partum MS period may benefit from the routine PRO utilization and screening for its worsening. The inflammatory activity during pregnancy was not associated with short-term disease progression.

O-021 Magnetic Resonance Imaging (MRI) evaluation of Quinagolide Vaginal Ring (QVR) treatment on endometrioma, Deep Infiltrating Endometriosis (DIE), and adenomyosis lesion characteristics: QLARITY trial results

Abstract Study question Can MRI provide a useful assessment of changes to endometrioma, DIE, and adenomyosis lesion burden resulting from QVR treatment? Summary answer MRI successfully measured changes in the number, size, diffusion, and perfusion biomarkers of endometriotic and adenomyotic lesions enabling evaluation of QVR treatment efficacy. What is known already Endometriosis and adenomyosis are associated with chronic pelvic pain, painful menses, and infertility. Determining treatment efficacy is reliant on patient reported outcomes (PRO) which are sensitive to response style effects and may lag changes in underlying pathology. Availability of biomarkers measuring lesion burden could accelerate the development of therapies treating disease rather than managing symptoms. MRI is routinely used in oncology to provide non-invasive biomarkers of treatment effect, a paradigm adapted in this trial. Quinagolide is a dopamine D2 receptor agonist formulated in an extended-release vaginal ring in development for treatment of endometriosis based on presumed inhibition of lesion angiogenesis. Study design, size, duration QLARITY was a randomized, placebo-controlled phase 2 trial investigating the mechanism of action of QVR (1080 µg) administered for four menstrual cycles. Randomization was 1:1 QVR (n = 35) to placebo vaginal ring (n = 32). Primary endpoint was change in the sum of lesion sizes by type from baseline to end of cycle 4 as measured by MRI. Secondary endpoints included changes in lesion volume, PROs and adverse events. MRI-derived imaging biomarkers were analyzed as exploratory endpoints. Participants/materials, setting, methods Women, 18-45 years old with at least one type of lesion (endometrioma, adenomyosis, and/or DIE) visualized by MRI, were enrolled. The analyses were performed by treatment for each of the three lesion types individually and for all types combined. Change in lesion size was analyzed using ANCOVA adjusted for baseline. Number of subjects with lesion regression was analyzed using a Chi-Square test. MRI-derived diffusion and perfusion imaging biomarkers were analyzed as exploratory endpoints. Main results and the role of chance Demographics and baseline characteristics were comparable between treatment groups. Patients had a mean age of 36.1 years and had been diagnosed for 5.25 years on average. Most patients (n = 42, 62.7%) had 2-4 lesions. No statistically significant changes in the sum of lesion sizes or PROs (Numerical Rating Scale, Biberoglu and Behrman Scale, and Endometriosis Health Profile-30) after treatment were noted, when compared to placebo. However, across all lesion types, a statistically significantly higher proportion of QVR patients than placebo patients demonstrated lesion regression of ≥ 65.0% volume (61.3% vs 35.5%, p = 0.042). In the DIE group, lesion regression occurred in more patients treated with QVR than those on placebo (36.8% vs 7.7%, p = 0.016). For diffusion imaging biomarkers, a significant difference in the diffusion coefficient standard deviation of DIE lesions (p = 0.039) was found in the QVR group compared to placebo. For perfusion imaging biomarkers, a statistically significant relationship with the treatment was identified in the endometrioma group for the 25th percentile of difference in fractional volume of extravascular extracellular space (mean difference −1.69, p = 0.009) from baseline. Incidence and severity of adverse events were similar between QVR and placebo. Limitations, reasons for caution MRI cannot currently detect superficial, highly vascularized endometriosis, which may be more responsive to QVR treatment than the more advanced stage of endometriosis (indicated by relatively large endometrioma and DIE) imaged in QLARITY. Moreover, 4 cycles may not be of sufficient duration for optimal treatment of these lesions. Wider implications of the findings QLARITY provides support for the use of MRI to diagnose and characterize changes in endometriotic/adenomyotic lesions. It is also the first MRI characterization of the placebo response in this population. Imaging biomarkers show promise in generating insights. QVR is safe and well tolerated; further studies of its efficacy are warranted. Trial registration number NCT03749109

Comparing the Clinical Outcomes of Percutaneous Cheilectomy to Open Cheilectomy With Moberg Osteotomy for the Treatment of Hallux Rigidus.

Both an open cheilectomy with a Moberg osteotomy and percutaneous cheilectomy have been successfully used to treat hallux rigidus and preserve motion.However, there have been no studies that have compared these 2 procedures using validated patient-reported outcomes such as the Patient Reported Outcome Measurement Information System. A retrospective review of hallux rigidus patients between January 2016 and July 2021 collected 48 percutaneous cheilectomy (PC) patients and 71 open cheilectomy with Moberg (OCM) patients. Preoperative and minimum 1-year postoperative PROMIS scores were collected. The OCM and PC cohorts did not have significant differences in their postoperative PROMIS scores. Both cohorts had modest but significant improvements postoperatively in the physical function, pain interference, and pain intensity domains. The OCM group had a larger degree of improvement in physical function, pain interference, and pain intensity (P = .015, .011, .001, respectively). No significant difference was identified in the reoperation rate. Patients undergoing an OCM had worse preoperative PROMIS scores and a modestly greater change in patient-reported outcomes than patients undergoing a PC. Level III, retrospective review.

Anterior Cruciate Ligament Repair With Suture Tape Augmentation of Proximal Tears and Early Anterior Cruciate Ligament Reconstruction With Suture Tape Augmentation Result in Comparable Clinical Outcomes With Anterior Cruciate Ligament Reconstruction at 2-Year Follow-Up

To compare the postoperative side-to-side laxity and short-term clinical outcomes of patients who received primary anterior cruciate ligament (ACL) repair with suture tape augmentation, acute anterior cruciate ligament reconstruction (ACLR) with suture tape augmentation performed within 8 weeks of injury (ACLRacute), or ACLR beyond 8 weeks of injury. After institutional review board approval was obtained, 100 patients were enrolled in this prospective trial: 34 primary ACL repair with suture tape augmentation, 33 ACLRs performed within 8 weeks of injury (ACLRacute), and 33 ACLRs. Patients were allocated to ACL repair if a proximal avulsion was present with good tissue quality (Sherman type 1), confirmed by intraoperative diagnostic arthroscopy. Preoperative side-to-side anteroposterior knee laxity was assessed with KT-1000 arthrometer, and patient-reported outcomes (PROs) including the visual analog scale, Marx activity scale, Veterans RAND 12-item health survey (VR-12 physical & mental), Single Assessment Numeric Evaluation, Knee Injury and Osteoarthritis Outcome Score survey subscales, and range of motion were collected. These objective and subjective measures were repeated at regular intervals postoperatively through 2 years. Minimal clinically important difference calculations were performed assessing postoperative PRO changes at 2 years compared with preoperative. The average time from injury to surgery was 5.03 ± 1.2 weeks for the ACL repair group, 5.09 ± 0.74 weeks for the ACLRacute, and 43.22 ± 33.5 weeks for the ACLR group. Postoperatively, the KT-1000 side-to-side laxity difference for 30 lbs was determined to be 0.1 ± 0.37 (95% confidence interval [CI] -0.7 to 0.8) for ACL repair versus ACLR (P < .0001), -0.8 ± 0.35 (95% CI -1.5 to -0.1) for ACLRacute versus ACLR (P < .0001), and 0.8 ± 0.40 (95% CI 0.0-1.6) for ACL repair versus ACLRacute (P < .0001). The data reveal ACL repair and ACLRacute are noninferior to ACLR at 2-year follow-up. The postoperative difference from baseline for all PROs demonstrated improvement for all PROs. Magnetic resonance imaging at 1 year revealed tissue healing for the 3 ACL injury treatment groups. Patients who underwent ACL repair of proximal tears with suture tape augmentation or ACL reconstruction within 8 weeks from injury resulted in noninferior side-to-side knee laxity, comparable PROs, and similar range of motion at 2-year follow-up compared with ACLR. Level II, prospective comparative study.

Firearm Injury, It's Not Just Physical: The Adverse Impact on Patient-Reported Socioeconomic, Mental Health, and Quality-of-Life Outcomes.

Background: The burden of firearm injury (FI) extends beyond hospitalization; however, literature focuses mostly on short-term physical outcomes. This study aimed to assess changes in patient-reported outcomes following firearm-related trauma. We hypothesized long-term patient-reported socioeconomic, mental health, and quality-of-life (QoL) outcomes are worse post-FI compared to pre-FI.Methods: This was a retrospective study where a phone survey was conducted with FI survivors admitted between January 2017 and August 2022 at a level 1 trauma center. Survey questions assessed demographics, socioeconomics, and mental and physical health pre-FI vs ≥ 6months post-FI; the McNemar test was used for comparisons. The PROMIS-29 + 2v2.1 NIH validated instrument was used to assess long-term QoL. Standardized NIH PROMIS T-scores were calculated using the HealthMeasures Scoring Service.Results: Of 204 eligible FI survivors, 71 were successfully contacted and 38 surveyed. Respondents were male (86.8%), Black (76%), and aged 18-29 (55.3%), and 68.4% had high school level education. Post-FI, patients were more likely to be unemployed (55.2% vs 13.2%, P < .001) and report increased mental health needs (84.2% vs 21%, P < .001) compared to pre-FI. Most (73.7%) also reported lasting physical disability. Similarly, the PROMIS instrument demonstrated largely worse health-related QoL scores post-FI, particularly high anxiety/fear (T-score 60.2, SE 3.1, CI 54.6-66.3, Table 2), pain resulting in life interference (T-score 60.0, SE 2.3, CI 55.7-63.9), and worse physical function (T-score 42.5, SE 3.0, CI 38.2-46.9).Conclusions: Firearm injury survivors had more unemployment and worse mental health post-FI compared to pre-FI. Firearm injury survivors also reported significantly worse health-related QoL metrics including pain, anxiety, and physical function 6months following their trauma. These long-term patient-reported outcomes are a framework to build future outpatient resources.Level of Evidence: IV.

10-Year Heterogeneity of Minimal Important Change and Patient Acceptable Symptom State After Lumbar Fusions.

Cohort study. To evaluate heterogeneity (fluctuation) in minimal important change (MIC) and patient acceptable symptom state (PASS) for patient-reported outcomes (PROMs) through 10 years after lumbar fusion. PROMs have become key determinants in spine surgery outcomes studies. MIC and PASS were established to aid PROM interpretations. However, their long-term stability has not yet been reported. A consecutive series of elective lumbar fusions were followed-up using the Oswestry Disability Index (ODI) and Visual Analogue Scale (VAS) for pain. Improvement was rated by a 4-point Likert scale into "improved" or "non-improved". Satisfaction-to-treatment was rated by the patients' willingness to undergo surgery again. Receiver operating characteristics (ROC) curve analysis estimated MIC (95% confidence interval, CI) as the PROM change that best predicted improvement at distinct time-points. PASS (CI) was estimated as the lowest PROM score at which the patients were still satisfied. Heterogeneity across thresholds was evaluated using the DeLong algorithm. MIC for ODI represented heterogeneity across 10-years, ranging from -21 (-24 to -16) at 2-years to -8 (-7 to -4) at 5-years, P <0.001. The areas under the ROC curves (AUCs) (0.79-0.85) indicated acceptable to excellent discrimination. Heterogeneity was not significant in the MICs for the pain scores. At 1-year, MIC for back pain was -24 (-38 to -15), AUC 0.77, and for leg pain it was -26 (-44 to -8), AUC 0.78. No significant heterogeneity was observed in 10-year PASS scores. At 1-year, PASS for ODI was 22 (15 to 29), AUC 0.85. Similarly, 1-year PASS for back pain was 38 (20 to 56), AUC 0.81, and for leg pain it was 49 (26 to 72), AUC 0.81. MIC for ODI fluctuated over 10-years after lumbar fusions. PASS values for all PROMs seemed most stable over time. Caution is needed when generic MIC values are used in long-term studies. Therapeutic Level III.

De-escalation of Disease-Modifying Therapy for People with Multiple Sclerosis Due to Safety Considerations: Characterizing 1-Year Outcomes in 25 People Who Switched from Ocrelizumab to Diroximel Fumarate.

Switching disease-modifying therapy (DMT) may be considered for relapsing-remitting multiple sclerosis (RRMS) if a patient's current therapy is no longer optimal. This was particularly important during the recent COVID-19 pandemic because of considerations around immune deficiency and impaired vaccine response associated with Bcell-depleting DMTs. This real-world, single-center study aimed to evaluate change or decline in functional ability and overall disease stability in people with RRMS who were switched from Bcell-depleting ocrelizumab (OCRE) to diroximel fumarate (DRF) because of safety concern related to the COVID-19 pandemic. Adults with RRMS were included if they had been clinically stable for ≥ 1year on OCRE. Data collected at baseline and 1year post switch included relapse rate, magnetic resonance imaging (MRI), blood work for assessment of peripheral immune parameters, the Cognitive Assessment Battery (CAB), optical coherence tomography (OCT), and patient-reported outcomes (PROs). Participants (N = 25) had a mean (SD) age of 52 (9) years, and a mean (SD) duration of 26 (8) months' treatment with OCRE before the switch to DRF. Median washout duration since the last OCRE infusion was 7months (range 4-18months). No participants relapsed on DRF during follow-up, and all remained persistent on DRF after 1year. There were no significant changes in peripheral immune parameters, other than an increase in the percentage of CD19+ cells 1year after switching (p < 0.05). Similarly, there were no significant changes in CAB, OCT, and PROs. These preliminary findings suggest that transition to DRF from OCRE may be an effective treatment option for people with RRMS who are clinically stable but may need to switch for reasons unrelated to effectiveness. Longer follow-up times on larger samples are needed to confirm these observations.

Comprehensive analysis of same day discharge after atrial fibrillation ablation: Clinical, cost, and patient reported outcomes.

Same day discharge (SDD) following atrial fibrillation (AF) ablation procedure has emerged as routine practice, and primarily driven by operator discretion. However, the impacts of SDD on clinical outcomes, healthcare system costs, and patient reported outcomes (PROs) have not been systematically studied. We retrospectively analyzed patients undergoing routine AF ablation procedures with SDD versus overnight observation (NSDD). After propensity adjustment we compared postprocedure adverse events (AEs), healthcare system costs, and changes in PROs. We identified 310 cases, with 159 undergoing SDD and 151 staying at least one midnight in the hospital (NSDD). Compared with NSDD, SDD patients were similar age (mean 64 vs. 66, p = 0.3), sex (26% female vs. 27%, p = 0.8), and with lower mean CHADS2-VA2Sc scores (2.0 vs. 2.7; p < 0.011). The primary outcome of AEs was noninferior in SDD versus NSDD patients (odds ratio 0.45, 95% confidence interval 0.21-0.99; noninferiority margin of 10%). There were also no differences in overall cost to the healthcare system between SDD and NSDD (p = 0.11). PROs numerically favored SDD (p = NS for all scores). Physician selection for SDD appears at least as safe as NSDD with respect to clinical outcomes and SDD is not significantly less costly to the health system. There is a trend towards more favorable, general PROs among SDD patients. Routine SDD should be strongly considered for patients undergoing routine AF ablation procedures.

Longitudinal changes in patient-reported outcomes (PROs) among early phase cancer clinical trial (EP-CT) participants.

e23188 Background: Little research has explored associations of longitudinal changes in PROs and clinical outcomes among EP-CT participants. We sought to describe changes in PROs over the first month of EP-CT participation and examine associations of changes with clinical outcomes. Methods: We prospectively enrolled adults participating in EP-CTs at Massachusetts General Hospital from 04/2021-01/2023. We assessed quality of life (QOL) (Functional Assessment of Cancer Therapy-General, with subdomains: physical wellbeing [PWB], emotional wellbeing [EWB], social wellbeing [SWB], functional wellbeing [FWB]); hope (Herth Hope Index); symptom burden (Edmonton Symptom Assessment System); and financial wellbeing (Comprehensive Score for Financial Toxicity) at time of EP-CT enrollment and one month later (1-month). We used descriptive statistics and regression models to describe 1-month changes in PROs and explore associations of patient-reported QOL, hope, symptom burden, and financial wellbeing with patient factors (age, diagnosis, performance status) and clinical outcomes (time on trial [TOT], treatment response, ED visits, hospitalizations, and survival). Results: Of 205 enrolled patients, 159 (77.9%) had baseline and 1-month data evaluable for analysis (median age: 63.0 [range 33.0 – 88.6], 59.8% female, 93.6% metastatic cancer, ECOG 0 = 46.2% and ECOG 1 = 53.8%). Most common cancer types were gastrointestinal (31.5%) and breast (22.0%). There was no significant change from baseline to 1-month in mean QOL (75.2 to 76.2), hope (27.1 to 26.7), ESAS total symptom burden (20.4 to 20.9), ESAS physical symptoms (13.3 to 13.7), and financial wellbeing (28.5 to 28.0). There was no significant change in overall QOL and in each subdomain with the exception of EWB (16.2 to 17.0, p &lt; .001). Older age was associated with increased hope (B = 1.29, p = .008) and increased QOL (B = 3.02, p = 0.024). Increasing EWB at 1-month was associated with longer TOT (HR = 0.93, p = .011). Increased symptom burden at 1-month was associated with greater risk of ED visits (HR = 1.04, p = .005) and hospitalizations (HR = 1.03, p = .012). Improving financial wellbeing at 1-month was associated with lower risk of hospitalization (HR = 0.96, p = .046). We found no other significant associations of 1-month changes in PROs with treatment response or survival. Conclusions: We described changes in PROs from baseline to 1-month and identified factors associated with these changes. Despite the rigors and intensity associated with EP-CT initiation, most PROs did not worsen, with significantly improved EWB after 1-month of participation. Results demonstrate changes in physical symptoms predicted for greater risk of ED visits and hospitalizations. Future studies should build upon this work and measure PROs throughout trial participation to identify factors associated with changes and guide intervention development.

EPCORE FL-2: Phase 3 trial of epcoritamab with rituximab and lenalidomide (R2) vs chemoimmunotherapy or R2 in previously untreated follicular lymphoma.

TPS7084 Background: Follicular lymphoma (FL) is the most common indolent non-Hodgkin lymphoma. Most patients with advanced-stage FL in need of systemic therapy are treated with an anti-CD20 monoclonal antibody (eg, rituximab [R] or obinutuzumab [G]) with cyclophosphamide, doxorubicin, vincristine, and prednisone [CHOP] or bendamustine [benda]). Recent studies have shown R plus lenalidomide (R2) is a potent FL treatment in the 1L and relapsed or refractory (R/R) settings and is a promising backbone for combination therapies. Epcoritamab, a subcutaneous CD3xCD20 bispecific antibody, received breakthrough therapy designation by the FDA for R/R FL after ≥2 lines of systemic therapy. In the phase 1/2 trial (NCT04663347), epcoritamab plus R2 demonstrated high complete response (CR) rates in the 1L and R/R settings. The objective of this trial is to evaluate the safety and efficacy of 1L epcoritamab plus R2 vs CIT in patients with FL. Methods: EPCORE FL-2 (NCT06191744) is a global, randomized, open-label phase 3 trial. Eligible adult patients must have CD20+ histologically confirmed classic FL (previously grade 1–3A), stage III or IV disease (or bulky stage II), and in need of systemic treatment by meeting GELF criteria. Approximately 1080 patients will be randomized to 3 treatment arms (Table). Patients achieving CR or PR upon 6 cycles will move to epcoritamab on day 1 of cycles 7–12 (28-day cycles) and 13–21 (56-day cycles). After 120 weeks of treatment, patients will be followed for progression and survival. Dual primary endpoints will be CR rate at 30 months and progression-free survival in arm A vs arm B. Key secondary efficacy endpoints include overall survival, minimal residual disease negativity, and changes in patient-reported outcomes on the physical functioning scale of the European Organization for Research and Treatment of Cancer Quality of Life-C30 Questionnaire from baseline to week 25. Safety endpoints include incidence and severity of adverse events (AEs), including AEs of special interest (cytokine release syndrome, immune effector cell–associated neurotoxicity syndrome, and clinical tumor lysis syndrome). The study is open for enrollment. Clinical trial information: NCT06191744 . [Table: see text]