Patient-reported Outcomes Research Articles

Early osteointegration in "one-step" resection and reconstruction using porous hydroxyapatite custom implants for skull-infiltrating tumors: a monocentric prospective series.

Early reconstruction of the skull represents the gold standard after resection of bone infiltrating cranial tumors. Customized hydroxyapatite porous ceramics are an excellent option for covering skull bone defects. The authors illustrate the surgical technique and investigate the effectiveness of the"one-step" procedure in terms of aesthetic results and early degree of osteointegration. A prospective study was conducted, including all patients operated on for skull bone infiltrating lesions at our center between January 2020 and June 2022. Stereolithography was the technique used for shaping the epoxy-resin model, suitable for both designing the craniotomy and manufacturing the custom-made hydroxyapatite prosthesis. Clinical outcome, results of early (6-week) and late (3-month) osteointegration evaluated on CT and MRI, and level of patient satisfaction measured by the FACE-Q questionnaire were reported. Fourteen patients (13 adults and a 7-year-old boy) and a total of 15 implants were included. The average percentage of early osteointegration calculated at the edge of the prosthesis, was 72.2%, that increased to 82.6% after 3months. Patient-reported outcomes indicated a high level of satisfaction across all patients. "One-step" resection and reconstruction using customized hydroxyapatite porous implants for treatment of skull infiltrating tumors is a safe, simple and effective technique, in particular when the bone defect is large. Bone regeneration around and inside the prosthesis seems to start early after surgery.

When is staging complex adult spinal deformity advantageous? Identifying subsets of patients who benefit from staged interventions.

The objective of this study was to identify baseline patient and surgical factors predictive of optimal outcomes in staged versus same-day combined-approach surgery. Adult spinal deformity (ASD) patients with baseline and perioperative (by 6 weeks) data were stratified based on single-stage (same-day) or multistage (staged) surgery, excluding planned multiple hospitalizations. Means comparison analyses were used to assess baseline demographic, radiographic, and surgical differences between cohorts. Backstep logistic regression and conditional inference tree analysis were used to identify variable thresholds associated with study-specific definitions of an optimal outcome in each cohort, defined as no intraoperative or surgery-related in-hospital adverse event. There were 439 patients with complex ASD in the dataset (mean age 64.0 ± 9.3 years, 68% female, mean BMI 28.7 ± 5.5 kg/m2). Overall, 58.8% of patients were in the same-day group, while 41.2% were in the staged group. Demographically, cohorts were not significantly different (p > 0.05), but staged patients were more frail per total Edmonton Frail Scale score (p = 0.043). Staged patients also reported greater numeric rating scale scores for back pain than same-day patients (p = 0.002). Cohorts were comparable in magnitude of planned correction of C7-S1 sagittal vertical axis, pelvic incidence-lumbar lordosis (PI-LL) mismatch, and T4-12 kyphosis (all p > 0.05). Controlling for baseline age, frailty, and number of levels fused, staged patients reported significantly higher PROMIS Discretionary Social Activities scores by 6 weeks (p = 0.029). Radiographic outcomes by 6 weeks were comparable between cohorts, in terms of both magnitude of change from baseline and overall result (all p > 0.05). Same-day patients were significantly more likely to experience in-hospital complications (p = 0.013). When considering frailty thresholds for staging, only a Charlson Comorbidity Index ≤ 1.0 was associated with optimal outcome in same-day patients, while Edmonton Frail Scale score ≥ 7 (p = 0.036), ≥ 9 levels fused (p = 0.016), and baseline PI-LL mismatch ≥ 15.3° (p = 0.028) were associated with optimal outcome for staged patients. Yet, staging alone was not significantly associated with an optimal outcome perioperatively (p = 0.056). While staged and same-day combined-approach surgeries yield comparable radiographic and patient-reported outcomes, certain subsets of complex ASD patients may benefit from staged surgery despite the invariably increased hospital length of stay. Individuals with increased frailty, moderate to severe PI-LL mismatch, and increased anticipated number of levels fused may experience a lower risk of perioperative adverse events if they undergo a staged procedure. Clinical trial registration no.: NCT04194138 (ClinicalTrials.gov).

Integrating Clinical Data and Patient-Reported Outcomes for Analyzing Gender Differences and Progression in Multiple Sclerosis Using Machine Learning.

Multiple sclerosis (MS) is a complex neurodegenerative disease with a variable prognosis that complicates effective management and treatment. This study leverages machine learning (ML) to enhance the understanding of disease progression and uncover gender-based differences in MS by analyzing clinical data integrated with patient-reported outcomes (PROMs). We conducted a prospective cohort study involving 250 MS patients at a secondary care hospital in Spain over an 18-month period. Using REDCap for data management, we collected comprehensive demographic, clinical, and PROMs data. Our analysis utilized Decision Trees, Random Forest, and Support Vector Machine algorithms to classify patients based on disease evolution and infer Expanded Disability Status Scale (EDSS) levels. Additionally, we employed propensity score matching to analyze gender differences, focusing on clinical outcomes and quality of life measures. The results could indicate that integrating diverse data sets through ML would significantly improve the diagnostic accuracy and serve as a support for clinician's decision making. Our models achieved high accuracy in classifying MS types and predicting disability levels, demonstrating the potential of ML in personalized treatment planning. Furthermore, our findings suggest notable gender differences in disease progression and response to treatment. These insights advocate for a gender-specific approach in MS management and highlight the importance of personalized medicine. This study underscores the transformative potential of ML in enhancing the understanding and management of MS through integrated data analysis.

Dupilumab Treatment for Aspirin-Exacerbated Respiratory Disease in a Real-World Setting: Impact on Quality of Life and Healthcare Utilization.

Patients with aspirin-exacerbated respiratory disease (AERD) have difficult-to-treat asthma and chronic rhinosinusitis with nasal polyps (CRSwNP) and often require treatment with biologic therapy for asthma or CRSwNP. Healthcare utilization in patients with AERD has not been well described since the advent of respiratory biologics. To determine real-world healthcare utilization and quality of life among patients with AERD and to understand the impact of dupilumab, a monoclonal antibody targeting the interleukin 4 receptor, on patient-reported health outcomes and healthcare utilization. We conducted a longitudinal survey study of 98 patients with AERD recruited from the Brigham and Women's Hospital AERD registry. Patients completed online questionnaires describing their medication history, healthcare utilization, and quality of life every 3 months for 2 years. At the end of 24 months, participants who were on dupilumab at the start of the study and those who started dupilumab had a significant reduction in the number of reported poor health days in the preceding month compared to patients not on dupilumab (P < .001 and P < .01, respectively). Participants on dupilumab and those who started dupilumab also had significantly lower overall sinonasal outcome test-22 and asthma control test scores compared to those not on dupilumab over 24 months (P < .05 for both groups). Dupilumab therapy significantly improves health-related quality of life in patients with AERD, specifically as it pertains to patient assessment of days of overall poor health and quality of life related to sinonasal and asthma symptoms.

Ablation Depth-Dependent Survival Analysis of Phototherapeutic Keratectomy for Recurrent Corneal Erosion Syndrome.

Phototherapeutic keratectomy (PTK) is a treatment for recurrent corneal erosion syndrome (RCES). The aim of this study was to investigate whether deeper ablations yielded greater success rates. Retrospective case notes review with prospective patient-reported outcome measures for all patients who had undergone PTK for RCES at a single tertiary referral unit. Patients received treatment with the Schwind Amaris® 750s excimer laser. The primary outcome measure was recurrence-free survival of patients with ablation depth ≥ 15µm compared to < 15µm. Seventy eyes of 63 patients were included for analysis, of whom 39 (56%) had preceding trauma and 20 (29%) had epithelial basement membrane dystrophy (EBMD), with the remaining 11 (15%) of unknown aetiology. Twenty eyes (29%) received an ablation ≥ 15µm (mean ablation depth 16.85 ± 3.4µm) and 50 (71%) received < 15µm (mean ablation depth 9.26 ± 1.5µm). Overall, 65 eyes (93%) reported a subjective improvement in symptoms, with 46 (66%) remaining completely symptom free at the last follow-up [mean follow-up 24 (range, 9-48)months]. Eighty-five per cent of eyes in the ≥ 15µm group remained symptom free compared to 58% of those with < 15µm (p = 0.036). PTK is an effective treatment for RCES, with deeper ablations yielding longer symptom-free survival.

Biological augmentation of anterior cruciate ligament reconstruction with bone marrow aspirate concentrate: a systematic review and meta-analysis of randomised controlled trials.

Biological augmentation of anterior cruciate ligament (ACL) reconstruction with bone marrow aspirate concentrate (BMAC) is gaining attention for its theoretical potential to enhance postoperative healing and recovery. However, its clinical benefits remain uncertain, and its high cost raises questions about efficacy. Hence, we systematically reviewed randomised controlled trials (RCTs) to evaluate the effectiveness of BMAC in ACL reconstruction. Our search included Cochrane, EMBASE, OVID, PubMed, and Scopus databases for RCTs evaluating the use of BMAC in ACL reconstruction. Primary outcomes focused on International Knee Documentation Committee (IKDC) scores and Lysholm scores. Secondary outcomes included MRI-related outcomes and postoperative complications. Statistical analysis was conducted using Review Manager 5.4 (Cochrane Collaboration), with heterogeneity assessed using Cochrane's Q test and I2 statistics. 221 patients from five RCTs were included, with 109 (49.3%) receiving BMAC augmentation. Follow-up ranged from 11.05 to 24 months. No significant differences were found in postoperative IKDC scores between the BMAC and control groups at, three, six and 12 months. The BMAC group had significantly higher IKDC scores at 24 months; however, this difference was unlikely to be clinically significant. No significant differences were observed in postoperative Lysholm scores at 12 or 24 months. MRI-related outcomes suggested potential graft recovery improvement with BMAC, and complication rates were comparable between groups. In summary, biological augmentation with BMAC in ACL reconstruction does not significantly improve early patient-reported outcomes but offers potential benefits in graft recovery without increasing complication rates.

Smoking Does Not Negatively Impact Outcomes Following Cervical Laminoplasty: A Quality Outcomes Database Study.

Retrospective cohort study. To assess the impact of smoking on outcomes following elective cervical laminoplasty for degenerative cervical myelopathy (DCM). The detrimental effect of cigarette smoking on cervical spinal fusion surgery outcomes is well documented. However, the impact of smoking on outcomes following nonfusion cervical spine procedures is unknown. One commonly utilized nonfusion technique for cervical decompression is laminoplasty. Adult smokers or nonsmokers who underwent primary elective laminoplasty for DCM were included. The propensity score (PS) was constructed for being a current smoker based on covariates. Only patients contained in the PS overlapped region were included in the analysis set. Patient-reported outcomes (PROs) at baseline and 12 months postoperation included Visual Analog Scale (VAS) neck and arm pain, neck disability index (NDI)%, EuroQol-5 Dimension (EQ-5D), and modified Japanese Orthopedic Association (mJOA) scores. Other outcomes included perioperative complications, 3-month readmissions/reoperations, and patient satisfaction. Wilcoxon and Pearson tests were used to compare outcomes between smokers and nonsmokers. The study included 132 patients: 30 were smokers (22.7%) and 102 were nonsmokers (77.3%). No significant differences were found at baseline between groups in demographics or medical history. Smokers had significantly worse baseline VAS neck pain (5.7±3.2 vs. 4.4±3.04, P=0.028) and arm pain (5.7±3.5 vs. 4.3±3.2, P=0.045) scores, but all other baseline PROs were not statistically different between groups. No differences were found between smokers and nonsmokers in any 12-month PRO, or in rates of perioperative complications, 3-month readmissions, or 3-month reoperations. On multivariable logistic regression analysis, smoking had no significant impact on any outcome of interest. Clinical and PROs following elective laminoplasty for DCM are not significantly different between smokers and nonsmokers. Laminoplasty should be considered a good surgical option in smokers presenting with DCM.

Efficacy and safety of mirikizumab in patients with moderately-to-severely active Crohn's disease: a phase 3, multicentre, randomised, double-blind, placebo-controlled and active-controlled, treat-through study.

Mirikizumab, a humanised monoclonal antibody that inhibits IL-23p19, is effective in moderate-to-severe ulcerative colitis. We aimed to evaluate the efficacy and safety of mirikizumab in patients with moderately-to-severely active Crohn's disease. VIVID-1 was a global phase 3, randomised, double-blind, double-dummy, placebo-controlled and active-controlled, treat-through study. The study enrolled adult patients at 324 sites (hospitals or medical centres, clinical practices, and clinical research sites) in 33 countries across Europe, Asia, North America, Central America, South America, and Australia. Adult patients with moderately-to-severely active Crohn's disease and previous inadequate response, loss of response, or intolerance to one or more approved biological therapies or conventional therapies were randomly assigned 6:3:2 to receive mirikizumab 900 mg intravenously at weeks 0, 4, and 8, then 300 mg subcutaneously every 4 weeks from weeks 12 to 52; ustekinumab about 6 mg/kg intravenously at week 0, then 90 mg subcutaneously every 8 weeks from weeks 8 to 52; or placebo. The coprimary endpoints assessing superiority of mirikizumab over placebo were composite endpoints: patient-reported outcome (PRO) clinical response at week 12 and endoscopic response at week 52 (endoscopic response-composite), and PRO clinical response at week 12 and Crohn's Disease Activity Index (CDAI) clinical remission at week 52 (CDAI clinical remission-composite). The adjusted risk differences were calculated, and the comparison was performed by the Cochran-Mantel-Haenszel test. Non-responder imputation was used. VIVID-1 was registered on ClinicalTrials.gov, NCT03926130, and is now complete. Between July 23, 2019, and Aug 23, 2023, 1150 patients were randomly assigned and received study treatment (safety population); 1065 patients were included in the efficacy population and received mirikizumab (n=579), ustekinumab (n=287), or placebo (n=199). Both coprimary endpoints were met: endoscopic response-composite was reached in 220 (38·0%) of 579 patients on mirikizumab versus 18 (9·0%) of 199 on placebo (99·5% CI 20·6-36·8; p<0·0001); CDAI clinical remission-composite was reached in 263 (45·4%) of 579 patients on mirikizumab versus 39 (19·6%) of 199 patients on placebo (99·5% CI 15·9-35·6; p<0·0001). The incidence rates of overall adverse events and discontinuations in patients treated with mirikizumab were lower compared with placebo. The most common adverse event across the three groups was COVID-19. Serious adverse events were reported in 65 (10·3%) of 630 patients on mirikizumab, 33 (10·7%) of 309 patients on ustekinumab, and 36 (17·1%) of 211 patients on placebo. There were three deaths during VIVID-1, one in the ustekinumab group, and two in the placebo group, including one in a placebo non-responder who switched to mirikizumab after week 12. None of the deaths were considered related to the study drug. The safety of mirikizumab in Crohn's disease was consistent with its known favourable profile. Mirikizumab was safe and effective as induction and maintenance treatment for patients with moderately-to-severely active Crohn's disease who had intolerance, inadequate response, or loss of response to standard therapy. Eli Lilly and Company.

Long-Term Management of Right Ventricular Outflow Tract Dysfunction in Repaired Tetralogy of Fallot: A Scientific Statement From the American Heart Association.

Right ventricular outflow dysfunction, manifesting as stenosis, regurgitation, or both, is nearly universal in patients with repaired tetralogy of Fallot, precipitating a complex pathophysiological cascade that leads to increasing rates of morbidity and mortality with advancing age. As the number of adolescent and adult patients with repaired tetralogy of Fallot continues to grow as a result of excellent survival during infancy, the need to improve late outcomes has become an urgent priority. This American Heart Association scientific statement provides an update on the current state of knowledge of the pathophysiology, methods of surveillance, risk stratification, and latest available therapies, including transcatheter and surgical pulmonary valve replacement strategies, as well as management of life-threatening arrhythmias. It reviews emerging evidence on the roles of comorbidities and patient-reported outcomes and their impact on quality of life. In addition, this scientific statement explores contemporary evidence for clinical choices such as transcatheter or surgical pulmonary valve replacement, discusses criteria and options for intervention for failing implanted bioprosthetic pulmonary valves, and considers a new approach to determining optimal timing and indications for pulmonary valve replacement.

Outcomes of medial collateral ligament reconstruction with suture-augmented semitendinosus autograft.

This study evaluates patient-reported outcomes among patients who underwent medial collateral ligament (MCL) reconstruction with suture-augmented semitendinosus autograft (SASA). Patients who underwent SASA MCL reconstruction between 2017 and 2022 participated in preoperative and postoperative surveys for patient-reported outcomes: Visual Analog Pain Scale (VAS), Knee Injury and Osteoarthritis Outcomes Score (KOOS), Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC), Single Assessment Numeric Evaluation (SANE), Marx Activity Rating Scale (MARS), and Veterans Rand 12 (VR-12). Paired t-tests were performed to compare preoperative and postoperative scores. Postoperative complications were analyzed for all patients. A total of 19 operations were identified during the study period, and 16 patients were included in the study. Patients reported significant decreases in VAS (mean [95% CI] of -3.86 [-6.09, -1.63], p = 0.0022) and WOMAC (-24.87 [-40.30, -9.4], p = 0.0037) scores postoperatively. Patients also reported significant increases in KOOS (22.60 [9.79, 35.40], p = 0.0019), SANE (38.06 [18.83, 57.27], p = 0.0007), and VR-12 Physical (14.32 [6.38, 22.27], p = 0.0017) scores. Patients did not report significant changes in MARS (0.87 [-1.88, 3.63], p = 0.5081) or VR-12 Mental (-2.90 [-9.37, 3.56], p = 0.3516) scores after surgery. Four patients required reoperation for either arthrofibrosis (n = 3) or ACL reinjury following a multiligament procedure that did not require revision to the MCL reconstruction (n = 1). In this cohort of patients undergoing MCL reconstruction with SASA, patients reported significant improvement in functional outcomes and reduction in pain postoperatively. SASA is a safe and effective technique for MCL reconstruction.

Health-related quality of life in children with bronchopulmonary dysplasia: examining agreement between child self-report and parent proxy.

In school-aged children with BPD, agreement between parents and children on HRQL scales is poor to moderate. Caregivers are biased towards lower HRQL. When possible, HRQL should be assessed in the patient directly. • Children with chronic respiratory diseases often assess their own health related quality of life different than their caregiver would report for the child. • Recent data suggests that parent reported health related quality of life for children with BPD is similar to the normal population. • There is a consistent bias for parents to report worse health related quality of life on the Patient Reported Outcomes Measurement Information System (PROMIS) standardized assessment than their school-aged children with BPD.

Self-assessed performance-based function test versus patient-reported outcome measures for knee and hip osteoarthritis

BackgroundPhysical function constitutes a key component of outcome assessment for almost all osteoarthritis interventions. The aim was to compare physical function measured using a self-assessed performance-based test versus self-reported function using questionnaires among individuals with knee or hip osteoarthritis (OA) participating in a digital exercise and education therapy.MethodsWe analysed data from individuals aged 40 + years participating in the digital program. We extracted data on the self-assessed 30-second chair stand test (30s CST) and the function subscales of Knee injury/Hip disability and Osteoarthritis Outcome Score 12 (KOOS-12/HOOS-12) at enrolment and 3- (n = 10884) and 12-month (n = 3554) follow-ups. Participants completed Numeric Rating Scale (NRS) pain, EQ-5D-5L, and an external anchor: global rating of change scale. Correlations were assessed using the Spearman correlation coefficient, responsiveness using standardized response mean (SRM) and receiver operating characteristic (ROC) curves, and agreement using weighted percent of agreement and weighted Gwet’s agreement coefficient.ResultsCorrelations were weak between the 30s CST and KOOS-12/HOOS-12 function (r < 0.35 for raw and r < 0.20 for change scores). Correlations with NRS pain and EQ-5D-5L were stronger for the KOOS-12/HOOS-12 function subscale than for 30s CST. Greater internal (SRM > 1 vs. SRM < 0.5) and lower external responsiveness were observed for the 30s CST versus the KOOS-12/HOOS-12 function, even though external responsiveness was generally inadequate for both (the area under the ROC curves < 0.7). The direction of change was similar for the two function measures for about 70% of subjects with moderate agreement between them (weighted Gwet’s agreement coefficient range 0.45 to 0.50).ConclusionWeak correlations and moderate agreements between function measured using performance-based test and self-reported using KOOS-12/HOOS-12 in people with knee or hip OA suggest that they may capture different aspects of functional abilities in this population.

Anifrolumab Study for Treatment Effectiveness in the Real World (ASTER) among patients with systemic lupus erythematosus: protocol for an international observational effectiveness study

IntroductionSystemic lupus erythematosus (SLE) is a chronic autoimmune disease with a diverse clinical presentation that involves multiple organ systems and may lead to organ damage and increased risk of mortality....

The Atopic Dermatitis Control Tool: Adaptation and Content Validation for Children and Caregivers of Children with Atopic Dermatitis.

The Atopic Dermatitis Control Tool (ADCT) assesses six concepts regarding patient-perceived control of atopic dermatitis (AD) in adults and adolescents with AD. This study aimed to develop two modified ADCT versions, one for children with AD aged 8-11years and another for caregivers of children with AD aged 6months to 11years. Following the US Food and Drug Administration patient-reported outcomes guidance, the ADCT was modified to produce draft Child and Caregiver ADCT versions, maintaining the original six concepts. The instruments were refined and finalized through an iterative process using input from children with AD and caregivers of children with AD via qualitative interviews. Inclusion criteria were clinician diagnosis of AD, prescription treatment use in the past 3months, and itching/scratching or rash in the past month. Interviews consisted of concept elicitation to identify perceptions of AD control and cognitive debriefing to test and refine the ADCT items. In total, 19 children (mean age 9.2years, 74% male) and 17 caregivers (mean age 36.3years, 100% female) were interviewed. During concept elicitation, children and caregivers reported similar symptoms and described the cycling and unpredictability of AD. Most participants reported that daily activities were impacted negatively by AD symptoms. The concept of AD control resonated with children and caregivers, and respondents were able to describe their experiences related to AD symptom severity. Children were unfamiliar with the term AD, so the Child ADCT version was named the Child Eczema Control Tool (ECT). Children and caregivers both reported that the instruments assessed relevant concepts, comprehensively measured AD control, and demonstrated content and face validity. The Child ECT and Caregiver ADCT were developed and qualitatively validated for assessing AD control in patients aged 6months to 11years and may offer simple ways to assess disease control and optimize treatment decisions. Video Abstract.

The FORWARD Psoriasis Registry: Patient-Reported Outcomes in a Novel Psoriasis Registry and Comparison of Traditional, Dermatologist-Led Enrollment With Web-Based Patient Enrollment.

Real-world evidence describing the natural history of all manifestations and severities of psoriasis is needed, as existing studies often recruit from a restricted patient population, and treatment failure and dissatisfaction is common. The FORWARD Psoriasis Registry collects patient-reported data directly online from participants independent of clinician involvement. To test the feasibility of this new registry design and compare baseline characteristics, patient-reported disease outcomes, and treatment utilization between participants enrolling through their clinician (primary enrollment group) and participants self-enrolling online (secondary). We summarized cross-sectional enrollment data from adults with clinician-diagnosed psoriasis who enrolled in the registry from September 2023 through June 2024 and compared baseline characteristics between enrollment groups. The registry enrolled 1560 adults with clinician-diagnosed psoriasis from 42 states and territories in the United States. In the primary enrollment group, 68% were female, mean age was 51years, 34% of participants had moderate or severe psoriasis based on PREPI, and 27% reported clinician-diagnosed psoriatic arthritis. Forty six percent actively used systemic therapies while 65% used topical therapies for psoriasis, and 20% were dissatisfied or very dissatisfied with their treatment. Comparatively, the secondary enrollment group reported statistically significantly worse psoriasis burden for nearly all disease outcomes. The FORWARD Psoriasis Registry rapidly enrolled a large, national cohort of participants with psoriasis, demonstrating feasibility of participant-driven data collection. We found important differences between participants enrolling through their clinician and self-enrolled participants, highlighting the need to collect real-world evidence to understand psoriasis regardless of access to clinical care.

Return to Sport Following Anterior Cruciate Ligament Reconstruction: A Scoping Review of Criteria Determining Return to Sport Readiness.

Provide a concise review of currently utilized functional metrics and patient reported outcomes measures (PROMs) determining appropriate return to sport following anterior cruciate ligament reconstruction (ACLR). When determining return to sport following ACLR, a limb symmetry index (LSI) ≥ 90% when compared to the contralateral extremity is the most commonly reported functional metric. LSI is most commonly assessed using isokinetic quadriceps strength, followed by single-leg vertical hop and cross-over hop test. A minimum ACL-Return to Sport Index (ACL-RSI) score of 68.2 was reported, as well as a mean International Knee Documentation Committee (IKDC) score of 88.3%. A minimum Knee Injury and Osteoarthritis Outcome Score-Quality of Life (KOOS-QoL) of 62.5% was reported in a single investigation. There remains limited data on reported metrics guiding return to sport following ACLR. Evaluation of LSI when compared to the contralateral extremity is the most commonly reported functional measure, with ACL-RSI, IKDC and KOOS-QoL also being reported. Further investigations examining return to sport rate and the incidence of recurrent injury, factoring in differences in sex, competition level, and the presence or absence of concomitant meniscal injuries, based on functional metrics and PROMs is warranted to better understand which outcome measures are predictive of successful return to sport following ACLR.

A comprehensive review of patient-reported outcomes in metabolic dysfunction-associated steatotic liver disease

The global prevalence of obesity and type 2 diabetes has increased, contributing to an increased worldwide prevalence of metabolic dysfunction-associated steatotic liver disease (MASLD). Currently, one in three adults is affected by MASLD and/or its progressive form, metabolic dysfunction-associated steatohepatitis (MASH), making this liver disease a significant public health challenge. Along with MASH-related cirrhosis, these conditions are poised to become the leading causes of chronic liver disease and liver transplants in the near future. Given the growing burden of MASLD and MASH, it is crucial to understand their impact from the patients’ perspective. One way to do this is by assessing patient-reported outcomes (PROs), including health-related quality of life (HRQL). HRQL can be assessed using generic instruments like the short form 36 version (SF-36) and the European quality of life-5 dimensions questionnaire (EQ-5D), or disease-specific tools such as the chronic liver disease questionnaire for nonalcoholic steatohepatitis (CLDQ-NASH). Given the limitations of each instrument, the best approach generally involves using both generic and disease-specific instruments. Evidence indicates that HRQL scores are significantly lower in individuals with MASLD, especially in areas assessing physical activity and the ability to perform daily living tasks. Fatigue and impaired work productivity are also important PROs for those with MASLD/MASH. These decrements in PROs worsen with disease progression but appear to improve with disease regression, including improvements linked to treatment. In this context, measuring PROs enhances the assessment of other patient-centric outcomes and provides insights for the healthcare community to develop interventions that could improve both clinical and humanistic outcomes for individuals living with MASLD/MASH.

Clinical study of a micro-implantable pulse generator for the treatment of peripheral neuropathic pain: 12-month results from the COMFORT-randomized controlled trial

BackgroundThere is paucity of data from randomized controlled trials supporting the use of peripheral nerve stimulation, a well-established therapy for the treatment of chronic pain. This study was undertaken, in...

Patient Reported Outcomes Due to Bladder Neck Obstruction in Women Treated With Botulinum Toxin A Injection.

Primary bladder neck obstruction (BNO) occurs when the bladder neck fails to open during voiding, causing urinary symptoms despite no anatomic obstruction. The cause of BNO is unclear but may involve neurogenic dysregulation related to the sympathic nervous system such as upper motor neuron lesion or peripheral autonomic neuropathy (small fiber neuropathy (SFN)). Another etiology can incuded increased sympathetic tone secondary to anxiety or stress conditons. Botulinum toxin A (BoNT-A) to the bladder neck has been used in our practice to treat women with BNO who failed conventional therapies (alpha blockers, relaxation strategies). This is the first report of patient-reported outcomes after BoNT-A treatment in women with pelvic pain and BNO. We included female patients with pelvic pain and BNO who received BoNT-A to the bladder neck between January 2022 and March 2023, and mailed self-reported outcome questionnaires. The primary outcome was the Global Response Assessment (GRA); secondary outcomes included pain scores on the Visual Analogue Scale (VAS) and symptom checklists. BNO was diagnosed using Nitti Criteria (high bladder pressure, low flow in bladder neck on video urodynamics), supplemented by additional criteria (e.g., high voiding pressure, prolonged attempts, Valsalva effort, and cystoscopic evidence or symptoms related to BNO). Urodynamic studies followed International Continence Society standards. Additionally, due to BNO's association with SFN, patient history of biopsy-confirmed SFN was recorded. Our inclusion criteria was satisfied among 18 patietns, with 17 completing the questionnaire. Out of those, 14 patients (77%) reported improvement on the GRA, with an average VAS of 8.3 ("Very Helpful"). The most commonly improved symptoms were feeling of incomplete emptying, difficulty starting stream, urethral burning, pain with urination, and pelvic pain. Postoperative symptom flares were reported in six patients for an average of 16 days. All six patients with biopsy-confirmed SFN showed significant improvement (VAS 8.7). Total of 14 patients (77%) indicated they would repeat the procedure. BoNT-A to the bladder neck significantly improved pelvic pain and refractory hesitancy in women with bladder neck obstruction, especially in those with small fiber neuropathy. Symptom flares are common, but did not reduce interest in repeated treatments, and scheduling injections before symptom recurrence may mitigate these flares.

Use of the Obstetric Quality-of-Recovery Score (ObsQoR-11) to Measure the Impact of an Enhanced Recovery Programme for Elective Cesarean Section

(Int J Obstet Anesth. 2024;57:103955. https://doi.org/10.1016/j.ijoa.2023.103955) Cesarean deliveries (CD) are increasingly common worldwide, yet recovery after CD is often slower than after vaginal birth, impacting individuals, families, and workplaces. Enhanced recovery after cesarean (ERAC) programs aim to improve this recovery process. However, there is variability in how ERAC outcomes are reported, hindering comparisons and recommendations. Many current measures, like length of stay (LOS) and pain scores, need to be improved in capturing overall patient recovery. Patient-reported outcome measures (PROMs), such as the Obstetric Quality-of-Recovery (ObsQoR) score, offer a more comprehensive view from the patient’s perspective. Despite their importance, ERAC studies often overlook PROMs. The CRADLE study recently identified vital outcomes, including ObsQoR, for evaluating ERAC programs. We proposed using ObsQoR-11 to assess ERAC’s impact on patient recovery post-CS, expecting improved scores with ERAC implementation.