Observational Registry Study Research Articles

A US payer budget impact analysis of Flurpiridaz-PET-MPI compared to SPECT-MPI in the diagnosis of coronary artery disease

Aims This economic model was developed to assess the budget impact of a novel radiotracer, Flurpiridaz (F18-PET-MPI), compared to SPECT-MPI from a US payer perspective. Materials and Methods The model was developed comparing F18-PET-MPI and SPECT-MPI, with F18-PET-MPI modality share increasing from 0.5% to 2.5% of the total MPI modality share, over a 5-year time horizon. The model estimates the impact of diagnostic performance on downstream healthcare resource utilization (HCRU) including invasive coronary angiography (ICA), revascularization, pharmacological treatment, and cardiac outcomes (CO) such as cardiac mortality (CM) and myocardial infarction (MI). Four suspected CAD populations, including general and difficult-to-image subgroups, were analyzed. Clinical inputs used to support the parameterization of the model were sourced from a systematic literature search and included claims-based real-world evidence, observational, and multicenter registry studies to inform the rates of HCRU and CO, and head-to-head comparative clinical trial data advised diagnostic performance inputs. Reimbursement codes informed MPI modality costs. Results are reported as per-member per-month (PMPM) based on a hypothetical health plan. Results In all suspected CAD populations analyzed, there was a nominal cost increase in the world with F18-PET-MPI. The 5-year average PMPM incremental budget impact ranged from $0.02 to $0.05 across all suspected CAD subgroups. Cost-savings were associated with decreased downstream CO such as CM, MI, and ICA. Limitations and Conclusion The available literature to source all parameters in the model was limited; therefore, assumptions and additional calculations were made based on published evidence to inform the model. A one-way sensitivity analysis was performed to confirm and address uncertainty in key parameters. This comprehensive analysis illustrates that the superior diagnostic performance of F18-PET-MPI may result in reduced adverse CO events and associated costs, increased appropriate identification and treatment of CAD, and a minimal increase in overall costs among general and difficult-to-image patient subgroups.

CTNI-75. TRIALS IN PROGRESS: REGISTRY OF SAFETY & EFFICACY IN ADULTS WITH PRIMARY & METASTATIC INTRACRANIAL TUMORS TREATED WITH START (GAMMATILES (CS-131 SOURCE BRACHY-THERAPY)): NCT04427384

Abstract INTRO This is the first observational registry study of R+STaRT, delivered by Cs-131 sources in permanently implanted resorbable collagen tile carriers, for patients with intracranial tumors. METHODS Since October 2020, 37 sites to-date have enrolled 359 patients with primary & metastatic intracranial tumors into the R+STaRT registry to assess the safety & efficacy of the addition of Gamma Tile to medically needed resection. Local control, overall survival, QOL, neurocognition, functional decline, and surgical and radiation associated AE’s are collected at 1, 3, 6, 9,12, 18 and 24 months, then every 6 months through 5 years. RESULTS Demographics include 359 patients, avg. age 57 y.o.. 162 malignant gliomas, 125 metastatic tumors, 38 meningiomas, and 34 rare other tumors. 197 Males (163 White, 23 Black, 5 Asian, and other races) and 157 Females (White 121, 28 Black, 0 Asian, and 8 other races). No unexpected outcomes reported in the hands of experienced providers have occurred, although close clinical-radiographic follow-up, to ensure early detection of possible treatment-effects, is paramount. Steroid-use, and above endpoints, continue to be collected. CONCLUSIONS In this observational registry of Gamma Tiles (Cs-131 source brachy-therapy) added to medically needed resections for patients with primary and metastatic intracranial tumors continues. Data will be used to benchmark clinical outcomes of R+STaRT therapy and allow for comparisons to existing standard-of-care treatments. The outcome measures captured will allow for evaluation of the potential risks and benefits of this treatment approach for patients in a real-world setting.

Disparities in casemix, acute interventions, discharge destinations and mortality of patients with traumatic brain injury between Europe and India.

Traumatic brain injury (TBI) is a major global health problem that disproportionally affects low- and middle-income countries. The needs for patients with TBI therefore may differ between levels of national development. We aimed to describe differences in epidemiology and acute care provision of TBI between India and Europe. We used data from two prospective observational registry studies - the Collaborative Indian NeuroTrauma Effectiveness Research in TBI (CINTER-TBI) and the Collaborative European NeuroTrauma Effectiveness Research in TBI (CENTER-TBI), which included TBI patients with an indication for brain CT-scan presenting to 65 centres across Europe and Israel and two trauma centres in India. We performed descriptive analyses of demographic, injury, and treatment characteristics and used random-effects logistic regression with covariate adjustment to examine the likelihood of acute neurosurgical interventions and in-hospital mortality. We included 22 849 patients from CENTER-TBI and 3904 from CINTER-TBI. The median age in Europe was 55 years (IQR = 32-76) compared to 27 years (IQR = 18-40) in India. The most common cause of TBI in Europe were falls (n = 12150 (53%), while traffic incidents predominated in India (n = 2130 (55%)). The proportion of patients with severe TBI was higher in India (n = 867 (22%)) than in Europe (n = 1661 (7%). Professional pre-hospital care involving ambulance service was utilised by three-fourths (n = 17203 (75%)) of European and less than a one-tenth (n = 224 (6%)) of Indian patients in our sample. Patients with severe TBI were more likely to undergo surgical contusion/haematoma evacuation in India compared to Europe (OR = 2.0; 95% CI = 1.7-2.5) and Indian patients had higher odds of undergoing intracranial pressure monitor placement (OR = 2.3; 95% CI = 2.0-2.7). A primary decompressive craniectomy was likewise more often performed in the Indian cohort (OR = 5.1; 95% CI = 3.5-7.5). Discharge destinations in Europe included rehabilitation centres (n = 1261 (6%)) or nursing homes (n = 1208 (5%)), which was rarely the case in India (n = 13 (0%) and n = 9 (0%), respectively). Substantial disparities between India and Europe exist along the neurotrauma care chain, with both systems being likely to face unique features and challenges in the future.

Effect of patient factors and procedure on proximal tibia fracture recovery - A retrospective analysis.

Tibia plateau fractures account for 1-2% of all fractures and might adversely affect the knee joint. The current research aimed to evaluate the effect of surgical procedures, age, and gender on proximal tibia fracture functional outcomes. The present data for the retrospective analysis was obtained from an observational longitudinal cohort trauma registry study, initiated at a tertiary care hospital in June 2015. A total of 51 isolated tibia plateau fracture patients were routinely treated with open reduction internal fixation or external fixator and assessed for functional outcomes till twelve months by the Rasmussen scoring scale. Patients were divided into five age groups. The functional outcome association to gender, age groups, and surgical procedures was assessed by the Fisher Exact test and logistic regression analysis. The mean age of the total 51 patients was 40±11 years. Patients operated with Open Reduction and Internal Fixation (74.5%) or with an external fixator (25.5%). Road traffic accidents were the common mechanism of injury. There were non-significantly different functional outcomes observed among age groups or between surgical procedures at all follow-ups but significantly different between genders at three-month follow-ups. Males recovered better than females (p=0.02). Tibia Plateau fracture patients respond almost equally to both external fixation and Open Reduction Internal Fixation procedures. Age does not influence the outcome. However, males had better functional outcomes at three months post-surgery indicating that gender could affect the outcomes. Further exploration might assist in planning gender-based proximal tibia fracture treatment strategies for optimum outcomes.

Longitudinal outcomes of iStent inject with cataract surgery compared to cataract surgery alone: real-world data from the Fight Glaucoma Blindness registry.

To compare combined phacoemulsification and iStent inject to phacoemulsification alone. Fight Glaucoma Blindness (FGB) registry - Australia. Prospective observational registry study. Eyes that had combined phacoemulsification with iStent inject (iStent group) were compared to eyes that had phacoemulsification only (cataract alone). Primary outcome was percentage of eyes achieving >20% IOP reduction from baseline 12 months after surgery. Secondary outcomes included mean IOP and medication reduction, adverse events and secondary procedures. At 12 months the percentage of eyes achieving >20% reduction of IOP at 12 months was significantly higher in the iStent group compared to the cataract alone group (40.9% vs 30.1% p<0.001). Adverse events occurred at a low rate in both groups, and secondary IOP lowering procedures were significantly less frequent in the iStent group compared to cataract alone. Real-world outcomes from the FGB registry at 12 months show phacoemulsification combined with iStent inject has higher efficacy and a reduced need for secondary IOP lowering surgery compared to phacoemulsification alone.

Predictors of Care Home Admission and Survival Rate in Patients With Syndromes Associated With Frontotemporal Lobar Degeneration in Europe.

Data on care home admission and survival rates of patients with syndromes associated with frontotemporal lobar degeneration (FTLD) are limited. However, their estimation is essential to plan trials and assess the efficacy of intervention. Population-based registers provide unique samples for this estimate. The aim of this study was to assess care home admission rate, survival rate, and their predictors in incident patients with FTLD-associated syndromes from the European FRONTIERS register-based study. We conducted a prospective longitudinal multinational observational registry study, considering incident patients with FTLD-associated syndromes diagnosed between June 1, 2018, and May 31, 2019, and followed for up to 5 years till May 31, 2023. We enrolled patients fulfilling diagnosis of the behavioral variant frontotemporal dementia (bvFTD), primary progressive aphasia (PPA), progressive supranuclear palsy (PSP) or corticobasal syndrome (CBS), and FTD with motor neuron disease (FTD-MND). Kaplan-Meier analysis and Cox multivariable regression models were used to assess care home admission and survival rates. The survival probability score (SPS) was computed based on independent predictors of survivorship. A total of 266 incident patients with FTLD were included (mean age ± SD = 66.7 ± 9.0; female = 41.4%). The median care home admission rate was 97 months (95% CIs 86-98) from disease onset and 57 months (95% CIs 56-58) from diagnosis. The median survival was 90 months (95% CIs 77-97) from disease onset and 49 months (95% CIs 44-58) from diagnosis. Survival from diagnosis was shorter in FTD-MND (hazard ratio [HR] 4.59, 95% CIs 2.49-8.76, p < 0.001) and PSP/CBS (HR 1.56, 95% CIs 1.01-2.42, p = 0.044) compared with bvFTD; no differences between PPA and bvFTD were found. The SPS proved high accuracy in predicting 1-year survival probability (area under the receiver operating characteristic curve = 0.789, 95% CIs 0.69-0.87), when defined by age, European area of residency, extrapyramidal symptoms, and MND at diagnosis. In FTLD-associated syndromes, survival rates differ according to clinical features and geography. The SPS was able to predict prognosis at individual patient level with an accuracy of ∼80% and may help to improve patient stratification in clinical trials. Future confirmatory studies considering different populations are needed.

Clinical Benefits and Safety of Medical Cannabis Products: A Narrative Review on Natural Extracts.

Interest in medical cannabis and cannabis-based medicinal products (CBMPs) has increased greatly in recent years. Two cannabinoids are of principal importance; delta-9-tetrahydrocannabinol (∆9-THC), the primary psychoactive component, and also cannabidiol (CBD), considered non-intoxicating. Each has distinct mechanisms of action and different therapeutic potentials. CBMPs differ in their ∆9-THC and CBD components; predominantly ∆9-THC, balanced formulations with equivalent ∆9-THC and CBD elements, and CBD-predominant products. In this narrative review, we evaluate the published evidence for the clinical benefits of CBMPs and overall benefits in well-being. We also review the overall safety profile and discuss the potential for dependence with CBMPs. Evidence can be drawn from a wide range of randomized and other controlled studies and from observational real-world studies. Most data from observational registry studies are supportive of ∆9-THC-based products (∆9-THC-predominant or balanced CBMPs) in the management of chronic neuropathic pain. Balanced products are also effective in reducing spasticity in multiple sclerosis. Most CBMPs show benefit in providing symptomatic benefits in reducing anxiety, nausea, and in improving sleep, but the place of specific products is more subtle, and choice guided by specific circumstances. Symptomatic improvements are accompanied by improved quality of life and well-being. Safety data indicate that CBMPs are generally well tolerated in most patients without specific contraindications. The majority of adverse effects are non-serious, and transient; most are principally associated with ∆9-THC and are dose-dependent. In contrast to recreational cannabis use, there is little evidence from clinical studies that CBMPs have any potential for dependence.

Stemless anatomic and reverse shoulder arthroplasty in patients under 55 years of age with primary glenohumeral osteoarthritis: an analysis of the Australian Orthopedic Association National Joint Replacement Registry at 5 years

BackgroundPrimary glenohumeral osteoarthritis in young patients poses challenging treatment decisions. Arthroplasty options have different failure profiles and implant survivorship patterns. This registry study aims to analyze the cumulative per cent revision rate (CPR) of different types of arthroplasties conducted for primary osteoarthritis in patients under 55 years of age. MethodsThis comparative observational national registry study included all shoulder arthroplasty for osteoarthritis in patients under 55 years of age undertaken between January 1st, 2005, and December 31st, 2022. Partial hemi resurfacing and hemi stemless procedures were excluded. The cumulative percentage of revision (CPR) was determined using Kaplan-Meier estimates of survivorship and hazard ratios (HR) from Cox proportional hazard models adjusted for gender. Reasons for revision of each type of arthroplasty and cumulative incidence of revision diagnoses were analyzed. Results2111 primary shoulder arthroplasties were compared. Glenoid erosion is the predominant cause of revision for humeral resurfacing (29.8%) and hemiarthroplasty (35.5%). Instability is the predominant cause of revision for stemmed anatomic total shoulder arthroplasty (ATSA) and reverse total shoulder arthroplasty (RTSA), while loosening is the predominant cause of revision for stemless ATSA. The 6-year CPR is 12.8% for humeral resurfacing (HRA), 14.1% for hemiarthroplasty (HA), 12.4% for stemmed (ATSA), 7.0% for stemless ATSA, and 6.5% for (RTSA). Stemmed ATSA had a higher revision rate than RTSA (entire period HR=2.04 (95% confidence interval (CI) 1.16, 3.57), p=0.012). In contrast, the revision rate of stemless ATSA was not different from RTSA (HR =1.05 (95% CI 0.51, 2.19), p=0.889). Males outnumber females for all shoulder arthroplasty categories. DiscussionRTSA and stemless ATSA are viable options in young patients with primary osteoarthritis. Their short—to medium-term revision rates are comparable to those of older patients and lower than those associated with HRA, HA, and stemmed ATSA. ConclusionIn the predominantly male patient population under the age of 55, reverse shoulder arthroplasty and stemless ATSA have a lower short-term revision risk than stemmed ATSA.

Elevation of Donor-derived Cell-free DNA Before Biopsy-proven Rejection in Kidney Transplant.

Standard-of-care biomarkers for renal allograft rejection are lagging indicators, signaling existing organ injury. This precludes early intervention, when immunological cascades leading to rejection are most susceptible. Donor-derived cell-free DNA (dd-cfDNA) shows promise as an early indicator of rejection, allowing earlier and possibly more effective treatment. This analysis was designed to assess this promise using real-world dd-cfDNA testing evidence. This retrospective analysis of the prospective, observational ProActive registry study (NCT04091984) assessed dd-cfDNA and serum creatinine levels before biopsy in 424 patients with ≥1 dd-cfDNA test (n = 1013) in the 6 mo before biopsy. Of 4667 enrolled patients, 1631 patients had ≥18 mo of follow-up data, of which 424 had a biopsy and were included in this analysis. Twenty-six biopsies showed antibody-mediated rejection (ABMR), 62 showed T cell-mediated rejection, and 336 showed nonrejection; each from a unique patient. dd-cfDNA fractions were significantly elevated 5 mo before ABMR biopsies, and 2 mo before T cell-mediated rejection biopsies, compared with nonrejection biopsies. In contrast, serum creatinine did not discriminate between rejection and nonrejection in advance, or concurrent with biopsy. Among patients with nonrejection biopsies, estimated glomerular filtration rate was significantly lower in cases with ≥2 increased dd-cfDNA results (≥1%), compared with those with 0 or 1 increased dd-cfDNA result. These data indicate that dd-cfDNA is an early indicator of biopsy-proven rejection, especially ABMR, suggesting a greater role for dd-cfDNA in surveillance to identify patients at high risk of ongoing or future rejection, thus requiring closer monitoring, biopsy, or other management changes.

Registry-based cohort study comparing percentages of patients reaching PASS for knee function outcomes after revision ACLR compared to primary ACLR

ObjectivesReaching the Patient-Acceptable Symptom State (PASS) threshold for the Knee injury and Osteoarthritis Outcome Score (KOOS) has previously been reported to successfully identify individuals experiencing clinical success after anterior cruciate...

The Symptomatic Expression in Chinese Patients with Suspected COVID-19 Omicron Variant Infection: A Clinical Case Registry Study

Little systematic research has been conducted into the symptomatic expression of COVID-19 infections in patients. It is known that symptomatic expression varies between patients, but the nature and extent of this variability is poorly understood. This paper elaborates on the symptoms reported by Chinese patients infected with COVID-19 during the Omicron wave, and compares this with available data from other countries. This was an observational clinical case registry study of Chinese patients with suspected Omicron variant COVID-19 infection. Symptoms were prospectively collected via a 171-item questionnaire and entered into the Clificol COVID-19 Clinical Case Registry. Two types of symptoms were distinguished: A) common clinical symptoms as identified by a search of available/published data, and B) homeopathic symptoms, used for the selection of the most suitable homeopathic medicine. Data were mainly analysed descriptively. Additionally, we compared the prevalence of the reported symptoms with available symptom data from the UK and France. Twenty one Chinese practitioners collected questionnaires on 388 cases that received a first homeopathic prescription between 5 December 2021 and 8 April 2022. With respect to A), the most frequently reported clinical symptoms were respectively cough (71%), fever (65%), extreme tiredness (58%), headache (51%), sore throat (46%), runny nose (34%), unusual muscle pains (31%), hoarseness (21%), eye soreness (8%) and brain fog (6%). With respect to B), homeopathic symptoms related to cough and fever were particularly prevalent. This study identified the reported symptoms of Chinese COVID-19 patients with suspected Omicron variant infection. A more detailed understanding of the clinical presentation of COVID-19 variants in different countries is important for the selection of test-triggering symptoms.

Medical costs of Swedish nursing home residents at the end of life: a retrospective observational registry study

BackgroundThere are many studies of medical costs in late life in general, but nursing home residents’ needs and the costs of external medical services and interventions outside of nursing home services are less well described.MethodsWe examined the direct medical costs of nursing home residents in their last year of life, as well as limited to the period of stay in the nursing home, adjusted for age, sex, Hospital Frailty Risk Score (HFRS), and diagnosis of dementia or advanced cancer. This was an observational retrospective study of registry data from all diseased nursing home residents during the years 2015–2021 using healthcare consumption data from the Stockholm Regional Council, Sweden. T tests, Wilcoxon rank sum tests and chi-square tests were used for comparisons of groups, and generalized linear models (GLMs) were constructed for univariable and multivariable linear regressions of health cost expenditures to calculate risk ratios (RRs) with 95% confidence intervals (95% CIs).ResultsAccording to the adjusted (multivariable) models for the 38,805 studied nursing home decedents, when studying the actual period of stay in nursing homes, we found significantly greater medical costs associated with male sex (RR 1.29 (1.25–1.33), p < 0.0001) and younger age (65–79 years vs. ≥90 years: RR 1.92 (1.85–2.01), p < 0.0001). Costs were also greater for those at risk of frailty according to the Hospital Frailty Risk Score (HFRS) (intermediate risk: RR 3.63 (3.52–3.75), p < 0.0001; high risk: RR 7.84 (7.53–8.16), p < 0.0001); or with advanced cancer (RR 2.41 (2.26–2.57), p < 0.0001), while dementia was associated with lower medical costs (RR 0.54 (0.52–0.55), p < 0.0001). The figures were similar when calculating the costs for the entire last year of life (regardless of whether they were nursing home residents throughout the year).ConclusionsDespite any obvious explanatory factors, male and younger residents had higher medical costs at the end of life than women. Having a risk of frailty or a diagnosis of advanced cancer was strongly associated with higher costs, whereas a dementia diagnosis was associated with lower external, medical costs. These findings could lead us to consider reimbursement models that could be differentiated based on the observed differences.

Three-year follow-up of the grip concept: an open, prospective, observational registry study on biomechanically calculated abdominal wall repair for complex incisional hernias

PurposeWe studied the effectiveness of biomechanically calculated abdominal wall reconstructions for incisional hernias of varying complexity in an open, prospective observational registry trial.MethodsFrom July 1st, 2017 to December 31st, 2020, four hospitals affiliated with the University of Heidelberg recruited 198 patients with complex incisional hernias. Hernias were repaired using biomechanically calculated reconstructions and materials classified on their gripping force towards cyclic load. This approach determines the required strength preoperatively based on the hernia size, using the Critical Resistance to Impacts related to Pressure. The surgeon is supported in reliably determining the Gained Resistance, which is based on the mesh-defect-area-ratio, as well as other mesh and suture factors, and the tissue stability. Tissue stability is defined as a maximum distension of 1.5 cm upon a Valsalva maneuver. In complex cases, a CT scan of the abdomen can be used to assess unstable tissue areas both at rest and during Valsalva’s maneuver.ResultsLarger and stronger gripping meshes were required for more complex cases to achieve a durable repair, especially for larger hernia sizes. To achieve durable repairs, the number of fixation points increased while the mesh-defect area ratio decreased. Performing these repairs required more operating room time. The complication rate remained low. Less than 1% of recurrences and low pain levels were observed after 3 years.ConclusionsBiomechanical stability, defined as the resistance to cyclic load, is crucial in preventing postoperative complications, including recurrences and chronic pain.

Surgery for carpal tunnel syndrome in patients with and without diabetes-Is there a difference in the frequency of surgical procedures?

Carpal tunnel syndrome (CTS) occurs more often among individuals with diabetes. The aim of this retrospective observational registry study was to examine whether individuals with diabetes and CTS are treated surgically to the same extent as individuals with CTS but without diabetes. Data on CTS diagnosis and surgery were collected from the Skåne Healthcare Register (SHR). A total of 35,105 individuals (age ≥ 18 years) diagnosed with CTS from 2004-2019 were included. Data were matched to the Swedish National Diabetes Register (NDR. Cox regression models were used to calculate the risk of the use of surgical treatment. Of the 35,105 included individuals with a CTS diagnosis, 17,662 (50%) were treated surgically, and 4,966 (14%) had diabetes. A higher number of individuals with diabetes were treated surgically (2,935/4,966, 59%) than individuals without diabetes (14,727/30,139, 49%). In the Cox regression model, diabetes remained a significant risk factor for surgical treatment (PR 1.14 (95% CI 1.11-1.17)). Individuals with type 1 diabetes were more frequently treated surgically (490/757, 65%) than individuals with type 2 diabetes (2,445/4,209, 58%). There was no difference between the sexes and their treatment. The duration of diabetes was also a risk factor for surgical treatment in diabetes type 2, but high HbA1c levels were not. Individuals with diabetes are more likely to be treated surgically for CTS than individuals without diabetes. Individuals with type 1 diabetes are more likely to be treated surgically for CTS than individuals with type 2 diabetes.

Abstract PO1-02-11: Characterization of MammaPrint UltraLow Risk tumors in more than 1400 patients from the real-world evidence FLEX study

Abstract BACKGROUND: MammaPrint is a gene expression signature used to determine the risk of distant recurrence for patients with early-stage breast cancer. Previous studies from MINDACT, FOCUS, IKA, and STO-3 trials have demonstrated that postmenopausal node-negative patients with MammaPrint UltraLow Risk tumors are considered indolent and patients have excellent long-term survival outcomes up to 20 years with little to no endocrine treatment. These studies suggest that patients with UltraLow Risk tumors are ideal candidates for treatment optimization which has resulted in the inclusion of the UltraLow Risk result in the NCCN guidelines. Here, we characterized patients with UltraLow Risk tumors from the real-world evidence FLEX study. METHODS: FLEX (NCT03053193) is a prospective, observational registry study with 99 sites open in the United States, Canada, Greece, and Israel. Patients enrolled in FLEX have early-stage breast cancer and receive MammaPrint testing as standard of care (with or without BluePrint molecular subtyping), and consent to full transcriptome analysis and clinical data collection. MammaPrint is a gene expression signature of 70 genes that classifies tumors as having a Low Risk or High Risk of distant recurrence. Within Low Risk tumors, a subcategory of UltraLow Risk (MammaPrint Index &amp;gt; 0.355) has been defined in previous studies. Clinical data for this analysis was locked February 2023. Clinical risk was defined as low risk or high risk based on Adjuvant Online criteria. RESULTS: Among the 12,328 patients enrolled in FLEX, 1465 (11.9%) have UltraLow Risk tumors. All tumors with available BluePrint molecular subtyping were classified as Luminal-Type. Of the patients with detailed clinical data, the majority had clinically low risk tumors (78.8%; Table 1). Notably, 34.6% of patients with UltraLow Risk tumors had Ki67 tumor staining of greater than 10%, 16.7% were pre- or peri-menopausal, 43.6% had grade 2-3 tumors, and 20.1% had tumors &amp;gt; 2 cm (Table 1). Among patients with treatment information, 6.5% received a combination of chemotherapy with or without endocrine and HER2-targeted therapy, 82.4% received endocrine therapy, 4.8% received other treatment (endocrine with targeted therapy (CDK4/6), radiation alone, novel treatment regimen, or supporting agents), and 6.3% did not receive treatment (Table 1). CONCLUSIONS: Previous trials have demonstrated the utility of MammaPrint UltraLow Risk in patients with clinically low risk features. In this large real-world evidence study of patients with early-stage breast cancer, UltraLow Risk tumors can be identified across all clinical categories. Overall, we report a substantial number of premenopausal patients with UltraLow Risk tumors, and these young women would be good candidates for treatment optimization. Future studies will assess long-term outcomes in this cohort further stratified by treatment. Table 1. Clinical and genomic characteristics of UltraLow Risk tumors Data are presented as n (%). Patients with unknown or missing data for each category have not been included. Citation Format: Cathy Graham, Kent Hoskins, Vijayakrishna K. Gadi, Suzanne Hoekstra, Gordon Srkalovic, Douglas Marks, Beth Sieling, Patricia Dauer, Andrea Menicucci, William Audeh, Joyce O'Shaughnessy, FLEX Investigators' Group. Characterization of MammaPrint UltraLow Risk tumors in more than 1400 patients from the real-world evidence FLEX study [abstract]. In: Proceedings of the 2023 San Antonio Breast Cancer Symposium; 2023 Dec 5-9; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2024;84(9 Suppl):Abstract nr PO1-02-11.

Predictors and long-term patterns of medication adherence to glaucoma treatment in Denmark—an observational registry study of 30 100 Danish patients with glaucoma

BackgroundSelf-treatment with glaucoma medication (eye drops) has been associated with adherence challenges. Poor adherence results in worse outcomes in terms of visual field loss.ObjectiveTo investigate patterns in medication adherence among...

Characteristics associated with rerevision of primary inlay and onlay reverse shoulder arthroplasty. Analysis from the Australian Orthopaedic Association National Joint Replacement Registry

BackgroundTwo designs of primary reverse total shoulder arthroplasty (rTSA), inlay reverse total shoulder arthroplasty (in-rTSA) and onlay reverse total shoulder arthroplasty (on-rTSA) that had undergone an aseptic revision were compared to determine differences in the rate of rerevision. MethodsIn this comparative observational national registry study between January 1, 2012, and December 31, 2021, all rTSA utilizing either a modular inlay or onlay metaphyseal humeral component that had been revised for aseptic reasons formed 2 cohort groups. The cumulative percentage rerevision (2nd CPR) was determined using Kaplan-Meier estimates of survivorship and hazard ratios (HRs) from Cox proportional hazard models adjusted for age and sex. A minor category revision involved exchange of parts not fixed to bone whilst major revisions did. The primary and revision diagnoses, surgeon primary volume experience, and revision category were compared. Shoulder Modular Replacement (SMR)/SMR L1 or L2 combination (Lima Corporate, San Daniele del Friuli, Italy) was excluded at subanalysis. ResultsThe 2nd CPR at 3 years was 20.4% (95% confidence interval 17.1, 24.1) for in-rTSA (n = 571) and 16.1%(11.6, 22.2) for on-rTSA (n = 249). The risk of rerevision was not different between the 2 cohort groups. Primary diagnosis fracture was associated with an increased risk of rerevision for on-rTSA (entire period on-rTSA HR = 3.16(1.50, 6.68), P = .002), and in-rTSA at subanalysis (entire period on-rTSA HR = 2.91(1.33, 6.33), P = .007). 59.9% of in-rTSA and 24.1% of on-rTSA aseptic revisions were minor. The revision diagnosis, the surgical experience of rTSA and if the revision was major or minor did not change the rate of rerevision. The most common reason for both in-rTSA (50%) and (43.2%) on-rTSA rerevision was instability/dislocation. DiscussionRerevision rates of in-rTSA and on-rTSA after aseptic revision are high. The primary rather the revision diagnosis changed rerevision rates in contemporary rTSA surgery. Minor revisions did not reduce rerevision rates for in-rTSA or on-rTSA compared to humeral/glenoid revision. Increased surgical experience of primary rTSA did not change the rate of rerevision of in-rTSA or on-rTSA.

ELLIPSOID ZONE RECOVERY IN MACULAR TELANGIECTASIA TYPE 2.

To describe imaging features of macular telangiectasia type 2 eyes experiencing ellipsoid zone (EZ) recovery. Macular telangiectasia type 2 patients with EZ recovery were identified from the Natural History and Observational Registry study and underwent retinal imaging including optical coherence tomography and fundus photography. Eyes were graded according to the classification system by Gass and Blodi, the EZ-loss area was measured, and optical coherence tomography parameters were assessed by two independent readers. Parameters were analyzed for their presence before EZ recovery. Twenty-four eyes of 21 patients (12 female, 57.12%; mean age 68 ± 8.54 years) were included in this study and followed for 21.25 months ± 12.79 months. At baseline, mean EZ-loss area was 0.036 mm 2 ± 0.028 mm 2 and 0.01 mm 2 ± 0.013 mm 2 at follow-up ( P < 0.001). A persisting external limiting membrane overlaying the EZ-loss was detected in 16 cases (66%), and hyperreflective changes in the outer retina were present in 18 cases (75%). Best-corrected visual acuity was 0.23 (20/32) ± 0.33 logMAR at baseline and 0.34 (20/40) ± 0.34 logMAR at follow-up ( P = 0.3). Distinct optical coherence tomography features precede ellipsoid zone recovery in macular telangiectasia type 2 and warrant further studies investigating implications for patient care and clinical trial interpretation.

Machine Learning Did Not Outperform Conventional Competing Risk Modeling to Predict Revision Arthroplasty.

Estimating the risk of revision after arthroplasty could inform patient and surgeon decision-making. However, there is a lack of well-performing prediction models assisting in this task, which may be due to current conventional modeling approaches such as traditional survivorship estimators (such as Kaplan-Meier) or competing risk estimators. Recent advances in machine learning survival analysis might improve decision support tools in this setting. Therefore, this study aimed to assess the performance of machine learning compared with that of conventional modeling to predict revision after arthroplasty. Does machine learning perform better than traditional regression models for estimating the risk of revision for patients undergoing hip or knee arthroplasty? Eleven datasets from published studies from the Dutch Arthroplasty Register reporting on factors associated with revision or survival after partial or total knee and hip arthroplasty between 2018 and 2022 were included in our study. The 11 datasets were observational registry studies, with a sample size ranging from 3038 to 218,214 procedures. We developed a set of time-to-event models for each dataset, leading to 11 comparisons. A set of predictors (factors associated with revision surgery) was identified based on the variables that were selected in the included studies. We assessed the predictive performance of two state-of-the-art statistical time-to-event models for 1-, 2-, and 3-year follow-up: a Fine and Gray model (which models the cumulative incidence of revision) and a cause-specific Cox model (which models the hazard of revision). These were compared with a machine-learning approach (a random survival forest model, which is a decision tree-based machine-learning algorithm for time-to-event analysis). Performance was assessed according to discriminative ability (time-dependent area under the receiver operating curve), calibration (slope and intercept), and overall prediction error (scaled Brier score). Discrimination, known as the area under the receiver operating characteristic curve, measures the model's ability to distinguish patients who achieved the outcomes from those who did not and ranges from 0.5 to 1.0, with 1.0 indicating the highest discrimination score and 0.50 the lowest. Calibration plots the predicted versus the observed probabilities; a perfect plot has an intercept of 0 and a slope of 1. The Brier score calculates a composite of discrimination and calibration, with 0 indicating perfect prediction and 1 the poorest. A scaled version of the Brier score, 1 - (model Brier score/null model Brier score), can be interpreted as the amount of overall prediction error. Using machine learning survivorship analysis, we found no differences between the competing risks estimator and traditional regression models for patients undergoing arthroplasty in terms of discriminative ability (patients who received a revision compared with those who did not). We found no consistent differences between the validated performance (time-dependent area under the receiver operating characteristic curve) of different modeling approaches because these values ranged between -0.04 and 0.03 across the 11 datasets (the time-dependent area under the receiver operating characteristic curve of the models across 11 datasets ranged between 0.52 to 0.68). In addition, the calibration metrics and scaled Brier scores produced comparable estimates, showing no advantage of machine learning over traditional regression models. Machine learning did not outperform traditional regression models. Neither machine learning modeling nor traditional regression methods were sufficiently accurate in order to offer prognostic information when predicting revision arthroplasty. The benefit of these modeling approaches may be limited in this context.

Treatment Patterns with Mirabegron and Antimuscarinics for Overactive Bladder: A Prospective, Registry Study in Taiwan and South Korea (FAITH).

This study aimed to assess overactive bladder (OAB) treatment patterns and factors associated with effectiveness and persistence. A prospective, longitudinal, observational registry study of adults starting OAB therapy with mirabegron or antimuscarinics was undertaken. Primary endpoints were time from treatment initiation to discontinuation/switching; proportion who discontinued/switched; and reasons for discontinuation/switching. Secondary endpoints included OAB Symptom Score (OABSS), OAB Questionnaire: Short Form, and OAB Bladder Assessment Tool scores; factors associated with effectiveness and persistence; and safety. In total, 556 patients initiating mirabegron and 250 initiating antimuscarinics were enrolled. There was no treatment switch, change, or discontinuation in 68.5% of the mirabegron initiator group and median time to treatment change was not reached. Mean initial treatment duration was 130.8days. In multivariable models, baseline OABSS was the only variable significantly associated with change from baseline in OABSS, and patients with mild and moderate OAB had significantly better persistence with mirabegron than those with severe OAB. Urinary tract infection was the most common adverse event with mirabegron. There was no treatment switch, change, or discontinuation in 60.4% of the antimuscarinics initiator group and median time to treatment change was not reached. Solifenacin was the most frequent initial treatment (66.0%). Mean treatment duration was 122.2days. In multivariable models, baseline OABSS was the only variable significantly associated with change from baseline in OABSS, while patients with OAB medication in the 12months before enrollment had significantly better persistence with antimuscarinics than those with no previous OAB medication. Dry mouth was the most common adverse event with antimuscarinics. Mirabegron and solifenacin were commonly prescribed as first-line OAB medications. There was no treatment switch, change, or discontinuation in more than 60% of the mirabegron initiator and antimuscarinics initiator groups. Mean initial treatment duration was 130.8days and 122.2days for mirabegron and antimuscarinics, respectively. Graphical Abstract available for this article. ClinicalTrials.gov NCT03572231.