Assessment Of Clinical Outcomes Research Articles

Assessment of clinical outcomes in patients with inflammatory arthritis: analysis from the UK Medical Cannabis Registry.

The aim of this study was to assess changes in validated patient-reported outcome measures after initiation of cannabis-based medicinal products (CBMPs) and the safety of CBMPs in patients with inflammatory arthritis. A prospective case series from the UK Medical Cannabis Registry was analyzed. The primary outcomes changes were in Brief Pain Inventory, McGill Pain Questionnaire, EuroQol 5-dimension 5-level (EQ-5D-5L), Generalised Anxiety Disorder-7 questionnaire, and Single-Item Sleep Quality Scale at 1, 3, 6, and 12 months of follow-up compared with baseline. Adverse events were analyzed in accordance with Common Terminology Criteria for Adverse Events, v.4.0. Statistical significance was defined as a P-value less than 0.050. Eighty-two patients met the inclusion criteria. Initiation of CBMP treatment was associated with improvements in Brief Pain Inventory, McGill Pain Questionnaire, EQ-5D-5L, Generalised Anxiety Disorder-7 questionnaire, and Single-Item Sleep Quality Scale at 1, 3, 6, and 12 months compared with baseline (P < 0.050). There were 102 (44.35%) mild adverse events, 97 (42.17%) moderate adverse events, and 31 (13.48%) severe adverse events recorded by 21 (25.61%) participants. This study suggests that CBMP treatment is associated with pain improvement and increased health-related quality of life for inflammatory arthritis patients. While causality cannot be inferred in this observational study, the results support the development of randomized control trials for inflammatory arthritis pain management with CBMPs.

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

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

An open-label pilot study of losmapimod to evaluate the safety, tolerability, and changes in biomarker and clinical outcome assessments in participants with facioscapulohumeral muscular dystrophy type 1

IntroductionFacioscapulohumeral muscular dystrophy (FSHD) is a genetic disease caused by aberrant DUX4 expression, leading to progressive muscle weakness. No effective pharmaceutical treatment is available. Losmapimod, a small molecule selective inhibitor of p38 α/β MAPK, showed promising results in a phase 1 trial for the treatment of FSHD, prompting additional studies. We report the findings of an open-label phase 2 trial (NCT04004000) investigating the safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of losmapimod in participants with FSHD1. MethodsThis study was conducted at a single site in the Netherlands from August 2019 to March 2021, with an optional, ongoing open-label extension. Participants aged 18 to 65 years with FSHD1 took 15 mg of losmapimod twice daily for 52 weeks. Primary endpoints were measures of losmapimod safety and tolerability. Secondary endpoints were assessments of losmapimod pharmacokinetics and pharmacodynamics. ResultsFourteen participants were enrolled. No deaths, serious treatment-emergent adverse events (TEAEs), or discontinuations due to TEAEs were reported. Losmapimod achieved blood concentrations and target engagements that were previously associated with decreased DUX4 expression in vitro. Clinical outcome measures showed a trend toward stabilization or improvement. ConclusionsLosmapimod was well tolerated and may be a promising new treatment for FSHD; a larger phase 3 study is ongoing.

PCR24 Exploring the Content and Psychometric Validity of Clinical Outcome Assessments in Pancreatic Ductal Adenocarcinoma Versus the Patient Reported Symptoms and Impacts

PCR24 Exploring the Content and Psychometric Validity of Clinical Outcome Assessments in Pancreatic Ductal Adenocarcinoma Versus the Patient Reported Symptoms and Impacts

CO12 A Review of Goal Attainment Scaling Implementation As a Personalized Clinical Outcome Assessment over the Last Decade

CO12 A Review of Goal Attainment Scaling Implementation As a Personalized Clinical Outcome Assessment over the Last Decade

PCR137 Within-Trial Interviews to Contextualize the Patient Experience and Supplement Clinical Outcomes Assessments in a Pivotal Trial of a Newly Described, Fatal and Ultra-Rare Pediatric Disease

PCR137 Within-Trial Interviews to Contextualize the Patient Experience and Supplement Clinical Outcomes Assessments in a Pivotal Trial of a Newly Described, Fatal and Ultra-Rare Pediatric Disease

PCR76 Exploring the Content and Psychometric Validity of Clinical Outcome Assessments in Renal Cell Carcinoma Versus the Patient Reported Symptoms and Impacts

PCR76 Exploring the Content and Psychometric Validity of Clinical Outcome Assessments in Renal Cell Carcinoma Versus the Patient Reported Symptoms and Impacts

PCR167 Targeted Literature Review to Understand the Patient Experience of Essential Thrombocythemia and to Evaluate Existing Clinical Outcome Assessment Measures

PCR167 Targeted Literature Review to Understand the Patient Experience of Essential Thrombocythemia and to Evaluate Existing Clinical Outcome Assessment Measures

1695-P: The Evaluation of Clinical Outcomes Assessments and Digital Health Technologies in Obesity Clinical Trials

1695-P: The Evaluation of Clinical Outcomes Assessments and Digital Health Technologies in Obesity Clinical Trials

Perampanel as an Add-On Therapy in Patients with Refractory Status Epilepticus and Postanoxic Encephalopathy: A Real-Life Single-Center Retrospective Cohort Study.

Data on the efficacy of perampanel in refractory status epilepticus (RSE) and postanoxic encephalopathy (PAE) are limited; its use in such conditions is currently off-label. We conducted a retrospective cohort study of consecutive adult patients with RSE, including PAE, exhibiting electroencephalographic patterns indicative of status epilepticus who were treated at our center (January 2018 to December 2022) with assessment of clinical and electroencephalographic outcomes. Thirty-six patients were included in the study, of whom 29 had nonanoxic RSE and 7 had PAE. Within the nonanoxic RSE subgroup, 45% (13 of 29; 95% confidence interval [CI] 27-63%) of study participants were responders, 34% (10 of 29; 95% CI 17-52%) were partial responders, and 21% (6 of 29; 95% CI 6-35%) were nonresponders. In the PAE subgroup (n = 7), no patients fully responded to perampanel; 43% (3 of 7; 95% CI 6-80%) were partial responders, and 57% (4 of 7; 95% CI 20-95%) were nonresponders. Responder and nonresponder study participants exhibited overlapping baseline characteristics. No significant differences in duration of hospitalization were observed between responders and nonresponders in both subgroups. Responders in the RSE subgroup had a median discharge modified Rankin Scale score of 3 (interquartile range 3-4), and nonresponders had a median discharge modified Rankin Scale score of 5 (interquartile range 5-6). Despite limitations from the retrospective design and the small population size, this study suggests that perampanel use in nonanoxic RSE appears to yield promising results at moderate doses, including a tendency toward a better functional outcome at discharge, without significant adverse effects. However, in patients with PAE, the drug seems to show suboptimal performance. Perampanel appears to have promising efficacy as an add-on therapy in nonanoxic RSE. However, in patients with PAE, its efficacy seems to be lower. Further studies are warranted to confirm these observations.

Validation of the Manchester Cosmetic Clinician-Reported Outcomes Questionnaire (MCCRO-Q)

SummaryPatient-reported outcome measures (PROM) are collected pre- and post-operatively following cosmetic surgery. Clinicians assess their own outcomes following surgery, but without a validated clinician-reported outcome measure (CROM), there is no way to compare clinical results between clinicians or compare these to PROM. Assessment of clinical outcomes is important to address patients' expectations in the consenting process and to provide recommendations to patients pre-operatively to improve post-operative appearance.To address the lack of validated CROM for cosmetic surgery, the Manchester Cosmetic Clinical Reported Outcome Questionnaire (MCCRO-Q) was developed. This study assessed the inter-rater reliability of assessors using the four-point scale pre-operative and post-operative MCCRO-Q questionnaires. Fifteen assessors reviewed photos from pre- and post-operative assessments in the UK between July 2016 and February 2020. Ninety-five patients were included in this validation study, with each patient assessed by a minimum of three assessors.MCCRO-Q showed consistency between reviewers, with all intra-class coefficient averages >0.5 for pre-operative assessment and post-operative assessment. Twenty-six per cent of preoperative assessments demonstrated perfect agreement, 56% had a maximum one-point difference, and 18% had a two-point difference to the median score. One per cent of post-operative assessments post-operatively demonstrated perfect agreement, 36% had a one-point difference and 63% had a maximum point difference of two to the median score.The inter-rate agreement shows MCCRO-Q is a reliable tool when used with three or more assessors to judge patient appearance clinically, pre-operatively and post-operatively, concerning abdominoplasty.

Appendicular lean mass index and motor function in ambulatory patients with Duchenne muscular dystrophy.

Appendicular lean mass index (ALMI) has been linked to motor function in patients with Duchenne muscular dystrophy (DMD). However, quantification of the relationship between ALMI and disease-specific clinical outcome assessment trajectories is needed. The purpose of this study was to determine associations between dual-energy x-ray absorptiometry (DXA) derived estimates of ALMI and motor function in ambulatory patients with DMD. A retrospective analysis of longitudinal clinical visit data from 137 glucocorticoid-treated patients with DMD collected via structured motor assessment protocol evaluated associations between ALMI and motor function indexed by the North Star Ambulatory Assessment (NSAA) and 10 Meter Walk/run Test (10MWT). Body composition was assessed using DXA. ALMI was calculated by dividing arm and leg lean mass by height in m2; fat mass index (FMI) was calculated by dividing whole body fat mass by height in m2. Linear mixed-effects models were used to estimate associations between ALMI and motor function, controlling for age and FMI. The full prediction model (age, age,2 ALMI, and FMI) explained 57% of the variance in NSAA scores and 63% of the variance in 10MWT speed. A 1 kg/m2 higher ALMI value predicted a 5.4-point higher NSAA score (p < .001) and 0.45 m/s faster 10MWT speed (p < .001). A 1 kg/m2 higher FMI value predicted a 1.5-point lower NSAA score (p < .001) and 0.14 meters/second slower 10MWT speed (p < .001). DXA-derived estimates of ALMI and FMI are associated with motor function in DMD and may explain variation in DMD disease progression.

Decentralized clinical trials for medications to reduce the risk of dementia: Consensus report and guidance.

Recent growth in the functionality and use of technology has prompted an increased interest in the potential for remote or decentralized clinical trials in dementia. There are many potential benefits associated with decentralized medication trials, but we currently lack specific recommendations for their delivery in the dementia field. A modified Delphi method engaged an expert panel to develop recommendations for the conduct of decentralized medication trials in dementia prevention. A working group of researchers and clinicians with expertise in dementia trials further refined the recommendations. Overall, the recommendations support the delivery of decentralized trials in dementia prevention provided adequate safety checks and balances are included. A total of 40 recommendations are presented, spanning aspects of decentralized clinical trials, including safety, dispensing, outcome assessment, and data collection. These recommendations provide an accessible, pragmatic guide for the design and conduct of remote medication trials for dementia prevention. Clinical trials of medication have begun adopting decentralized approaches. Researchers in the field lack guidance on what would be appropriate circumstances and frameworks for what would be appropriate circumstances and frameworks for the use of decentralized trial methods in dementia prevention. The present report provides consensus-based expert recommendations for decentralized clinical trials for dementia prevention.

Trends and landscape of clinical outcome assessment use to support oncology product approvals.

e23186 Background: The regulatory landscape in oncology trials has evolved with growing emphasis on patient-focused drug development. Further to tumor and survival outcomes, measuring how patients feel and function via clinical outcome assessments (COAs) provides critical information on symptoms and treatment burden and may help to inform risk/benefit evaluation for new treatments. This study aimed to examine trends in the use of COAs to support efficacy or safety in oncology product labeling. Methods: Product labeling information was obtained from PROLABELS (Mapi Research Trust), a database containing COA-related data sourced from the efficacy or safety sections of FDA Product labels and EMA SmPC where at least one COA is mentioned. Oncology product labels were screened based on therapeutic indications and study information. The clinical study details published in the label/SPC between 1990 and 2022 were used for further analysis. Results: A total of 180 EMA and FDA approved drugs and devices for oncology indications where COAs were mentioned in the product label were identified. 288 studies with COAs supported these drug approvals. These studies were in 52 different indications, with most in multiple myeloma (31), followed by non-small-cell lung cancer (24), prostate cancer (22), and breast cancer (22). Of the COAs that were used, 47% were patient-reported outcomes, 18% were clinician-reported outcomes, 30% were composites, and 5% were unspecified. The number of studies conducted with COAs supporting a product label increased over time, with 3x more studies in the past 10 years (189 from 2013-2022) compared to the previous 10 years (60 from 2003-2012). For studies listing specific COA names, 63 unique COAs were used. Excluding response criteria assessments, the most common COAs were the EORTC-QLQ-C30 (57 studies), EORTC-QLQ-LC13 (18 studies), and EQ-5D-3L (17 studies). The two indications with the largest variety of COAs used were prostate cancer (10 unique COAs) and non-small-cell lung cancer (9 unique COAs). Conclusions: In line with regulators highlighting the utility of COAs in evaluating clinical benefit, results show an increase in studies using COAs to support efficacy and safety in oncology product approvals. With COAs providing critical information on symptoms, side effect impact, and quality of life, including this information on labels may help patients and providers make more informed healthcare decisions, and for market differentiation.

Screening oncology articles in a qualitative literature review using large language models: A comparison of GPT4 versus fine-tuned open source models using expert-annotated data.

e23196 Background: Clinical Outcome Assessment (COA) conceptual gap analyses for oncology are complex and time consuming. Artificial Intelligence may efficiently reduce time to completion of such analyses. We aimed to assess two AI models’ performance for literature screening to identify relevant qualitative oncology research. We also compared accuracy and run-time for both AI models. Methods: We manually curated a dataset of title/abstract screening (n = 1,700 study references) across 17 landscape reviews. Among these, 11 landscape reviews (n = 951 study references) spanning over were in oncology, including 8 solid cancers (breast, lung, urothelial, colorectal, esophageal, head and neck, pancreatic, and stomach) and 3 non-solid cancers (lymphoma, acute myeloid leukemia, and multiple myeloma). Each citation was annotated for eligibility (Y/N) by population, study design (qualitative), and reporting of concepts (how patients feel or function). We then compared the accuracy of two AI models at predicting the screening decisions of expert researchers. The two AI models were Generative Pre-trained Transformers 4 (GPT4, OpenAI) prompts and a fine-tuned SciFive biomedical large language model (LLM). We used 70% of the data for training and 30% for test. Accuracy estimates were obtained only for the models’ ability to label eligibility within the 11 oncology datasets. Results: Both LLMs performed well for assessing relevance by oncology population, with F1-scores for the GPT4 and SciFive models being 0.92 and 0.83 respectively (precision was 0.92 and 0.93 respectively). For concept reporting the fine-tuned SciFive model outperformed GPT4 with an F1-score and precision 0.88 and 0.92 versus 0.81 and 0.79. The same was true but less pronounced for eligibility by study design, with an F1-score and precision 0.81 and 0.90 versus 0.86 and 0.76. For overall eligibility, the customized SciFive model outperformed the GPT4 model with an F1-score and precision of 0.84 and 0.92 versus 0.85 and 0.82. Lastly, it took the GPT4 prompts between 10-30 minutes to screen 100 abstracts. By contrast, the customized SciFive model took 1-2 minutes on a computer with a Quadro RTX 8000 GPU. Conclusions: In conclusion, both AI models are promising. The fine-tuned SciFive model appears slightly more accurate and performs substantially faster than the GPT4 model.

Real-world treatment patterns and clinical outcomes among patients with HR+/HER2- advanced/metastatic breast cancer receiving palbociclib in Brazil: IRIS study.

e13088 Background: Palbociclib was approved for hormone receptor-positive (HR+) and human epidermal growth factor receptor 2-negative (HER2-) advanced/metastatic breast cancer (aBC/mBC) in Brazil in 2018. Worldwide studies explore palbociclib's real-world use and outcomes; however, this data is not available for Brazilian patients. IRIS Brazil study aimed to describe demographic/clinical characteristics, treatment and clinical outcomes of adult female patients who have received palbociclib as first-line treatment for HR+/HER2- aBC/mBC in private healthcare institutions in Brazil. Methods: This retrospective descriptive study included females aged 18 years or older diagnosed with HR+/HER2- aBC/mBC and those who had received palbociclib as their initial line of therapy. The study analyzed variables such as demographics, clinical characteristics, treatment history, palbociclib combinations, and clinical outcomes. Progression-free survival (PFS) and overall survival (OS) were calculated using Kaplan-Meier estimates at 6- and 12-month time points. Results: A total of 121 patients were included in the study. The mean age was 54.4 years, and 82 (67.7%) were menopausal at diagnosis. A total of 51 patients (42.1%) were treated with palbociclib + fulvestrant, while 67 (55.8%) were treated with palbociclib + aromatase inhibitors. The majority of patients (n = 79; 65.3%) did not require any dose adjustments. Among the 40 patients (33.1%) who required dose adjustments, the main reason was adverse events/toxicity (n = 36; 90%). The PFS rates at 6 and 12 months were 78% and 60%, respectively. The OS rates at 6 and 12 months were 86% and 70%, respectively. Conclusions: Results from this initial real-world assessment of clinical outcomes in Brazil suggest that Palbociclib combinations exhibit favorable effectiveness in treating HR+/HER2- aBC/mBC disease, as measured by PFS and OS rates.

Multi-dimensional measurements of long-term quality of life and recovery patterns of patients with cervical cancer after treatment.

e17533 Background: Cervical cancer is a prevalent malignancy affecting the female reproductive system globally, and its incidence among younger individuals is on the rise. This study employed the CC-PRO137 scale to evaluate the long-term quality of life and recovery patterns of cervical cancer patients post- treatment. Clinicians are guided to provide individualized treatment and psychological support. Methods: This study recruited patients with primary cervical cancer who underwent primary surgical treatment at the Obstetrics and Gynecology Hospital of Fudan University from October 2018 to June 2021. The CC-PRO137 (Cervical Cancer Patient Reported Clinical Outcome Assessment Scale) was used for follow-up to assess changes in the patients' physical, psychological, and social life states. The data was analyzed to examine variations in the recovery status of the patients across 13 modules: urinary function (URI), bowel function (BOW), pelvic organ prolapse (POP), ovarian function (OVA), lymphatic function (LYM), nervous function (NEU), sexual function (SEX), anxiety (ANX), depression (DEP), shame (SHA), belief (BEL), social support (SUP), and daily life (LIF). Subgroup analysis was conducted based on different stages, surgical methods, and adjuvant treatment plans. Results: A total of 304 participants were tested both 6 months and 12 months after the completion of treatment. The scores for URI, NEU, and SEX were significantly higher after one year compared to within 6 months (URI: t=-3.519, p=0.001; NEU: t=-2.138, p=0.033; SEX: t=-2.338, p=0.021). This suggests a notable improvement in urinary function, nervous function, and sexual function after 12 months. In terms of the psychological domain, DEP scores were significantly higher after 12 months compared to within 6 months ( t=-2.517, p=0.012). The LIF index showed a significant improvement after 12 months ( t=-5.224, p&lt;0.001). Subgroup analysis revealed that Stage II had worse outcomes compared to Stages I and III, particularly in terms of URI ( F(2,297) = 10.619, p &lt; 0.001) and POP ( F(2,299) = 8.559, p&lt; 0.001) indexes. The laparoscopy had a greater impact on the urinary system ( t = 2.157, p = 0.046). The adverse effects of concurrent chemoradiotherapy on URI ( F(2,297) = 11.497, p &lt; 0.001) and DEP ( F(2,299) = 4.149, p = 0.017) functions were more significant than those of chemotherapy. Conclusions: The recovery of patients following cervical cancer treatment is primarily observed in improvements in urinary, bowel, and nervous functions in the physiological domain. In the psychological domain, there is a noticeable improvement in depressive state, while the social life domain also shows signs of improvement over time. By focusing on the disease impact within a limited time frame, the scale helps clinicians provide targeted guidance and advice based on the main issues.

Listener effort quantifies clinically meaningful progression of dysarthria in people living with amyotrophic lateral sclerosis.

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative motor neuron disease that causes progressive muscle weakness. Progressive bulbar dysfunction causes dysarthria and thus social isolation, reducing quality of life. The Everything ALS Speech Study obtained longitudinal clinical information and speech recordings from 292 participants. In a subset of 120 participants, we measured speaking rate (SR) and listener effort (LE), a measure of dysarthria severity rated by speech pathologists from recordings. LE intra- and inter-rater reliability was very high (ICC 0.88 to 0.92). LE correlated with other measures of dysarthria at baseline. LE changed over time in participants with ALS (slope 0.77 pts/month; p<0.001) but not controls (slope 0.005 pts/month; p=0.807). The slope of LE progression was similar in all participants with ALS who had bulbar dysfunction at baseline, regardless of ALS site of onset. LE could be a remotely collected clinically meaningful clinical outcome assessment for ALS clinical trials.

Factors Associated With Early Motor Function Trajectories in DMD After Glucocorticoid Initiation: Post Hoc Analysis of the FOR-DMD Trial.

Clinical trials in Duchenne muscular dystrophy (DMD) require 3-6 months of stable glucocorticoids, and the primary outcome is explored at 48-52 weeks. The factors that influence the clinical outcome assessment (COA) trajectories soon after glucocorticoid initiation are relevant for the design and analysis of clinical trials of novel drugs. We describe early COA trajectories, associated factors, and the time from glucocorticoid initiation to COA peak. This was a prospective 18-month analysis of the Finding the Optimum Corticosteroid Regimen for Duchenne Muscular Dystrophy study. Four COAs were investigated: rise from supine velocity (RFV), 10-meter walk/run velocity (10MWRV), North Star Ambulatory Assessment (NSAA) total score, and 6-minute walk test distance (6MWT). The relationships of baseline age (4-5 vs 6-7 years), COA baseline performance, genotype, and glucocorticoid regimen (daily vs intermittent) with the COA trajectories were evaluated using linear mixed-effects models. One hundred ninety-six glucocorticoid-naïve boys with DMD aged 4-7 years were enrolled. The mean age at baseline was 5.9 ± 1.0 years, 66% (n = 130) were on daily regimens, 55% (n = 107) showed a 6MWT distance >330 metres; 41% (n = 78) showed RFV >0.2 rise/s; 76% (n = 149) showed 10MWRV >0.142 10m/s, and 41.0% (n = 79) showed NSAA total score >22 points. Mean COA trajectories differed by age at glucocorticoid initiation (p < 0.01 for RFV, 10MWRV, and NSAA; p < 0.05 for 6MWT) and regimen (p < 0.01 for RFV, 10MWRV, and NSAA). Boys younger than 6 years reached their peak performance 12-18 months after glucocorticoid initiation. Boys aged 6 years or older on a daily regimen peaked between months 9 and 12 and those on an intermittent regimen by 9 months. The baseline COA performance was associated with the NSAA (p < 0.01) and the 6MWT trajectory in boys younger than 6 years on a daily regimen (p < 0.01). Differences in the mean trajectories by genotype were not significant. Glucocorticoid regimen, age, duration of glucocorticoid exposure, and baseline COA performance need to be considered in the design and analysis of clinical trials in young boys with DMD.

Bayesian Spatio-Temporal Multilevel Modelling of Patient-Reported Quality of Life following Prostate Cancer Surgery.

Globally, prostate cancer is the second leading cause of cancer deaths among males. It is the most commonly diagnosed cancer in Australia. The quality of life of prostate cancer patients is poorer when compared to the general population due to the disease itself and its related complications. However, there is limited research on the geographic pattern of quality of life and its risk factors in Victoria. Therefore, an examination of the spatio-temporal pattern and risk factors of poor quality of life, along with the impact of spatial weight matrices on estimates and model performance, was conducted. A retrospective study was undertaken based on the Prostate Cancer Outcome Registry-Victoria data. Patient data (n = 5238) were extracted from the Prostate Cancer Outcome Registry, a population-based clinical quality outcome assessment from 2015 to 2021. A Bayesian spatio-temporal multilevel model was fitted to identify risk factors for poor quality of life. This study also evaluated the impact of distance- and adjacency-based spatial weight matrices. Model convergence was assessed using Gelman-Rubin statistical plots, and model comparison was based on the Watanabe-Akaike Information Criterion. A total of 1906 (36.38%) prostate cancer patients who had undergone surgery experienced poor quality of life in our study. Belonging to the age group between 76 and 85 years (adjusted odds ratio (AOR) = 2.90, 95% credible interval (CrI): 1.39, 2.08), having a prostate-specific antigen level between 10.1 and 20.0 (AOR = 1.33, 95% CrI: 1.12, 1.58), and being treated in a public hospital (AOR = 1.35, 95% CrI: 1.17, 1.53) were significantly associated with higher odds of poor quality of life. Conversely, residing in highly accessible areas (AOR = 0.60, 95% CrI: 0.38, 0.94) was significantly associated with lower odds of poor prostate-specific antigen levels. Variations in estimates and model performance were observed depending on the choice of spatial weight matrices. Belonging to an older age group, having a high prostate-specific antigen level, receiving treatment in public hospitals, and remoteness were statistically significant factors linked to poor quality of life. Substantial spatio-temporal variations in poor quality of life were observed in Victoria across local government areas. The distance-based weight matrix performed better than the adjacency-based matrix. This research finding highlights the need to reduce geographical disparities in quality of life. The statistical methods developed in this study may also be useful to apply to other population-based clinical registry settings.