Real-world Outcomes Research Articles

Emapalumab Use in Patients With Rheumatologic Disease-Associated Hemophagocytic Lymphohistiocytosis in the United States: the REAL-HLH Study.

Rheumatologic disease-associated hemophagocytic lymphohistiocytosis (HLH), a rare, life-threatening, systemic hyperinflammatory syndrome, occurs as a complication of underlying rheumatologic disease. Real-world evidence is lacking on emapalumab, a fully human monoclonal antibody that neutralizes the proinflammatory cytokine interferon-gamma, approved for treating patients with primary HLH. REAL-HLH, a retrospective medical chart review study conducted across 33 US hospitals, assessed real-world treatment patterns and outcomes in patients with HLH treated with ≥1 dose of emapalumab between November 20, 2018, and October 31, 2021. Data are presented for the subset of patients with rheumatologic disease-associated HLH. Fifteen of 105 patients (14.3%) had rheumatologic disease-associated HLH. Of these, 9 (60.0%) had systemic juvenile idiopathic arthritis, and 1 (6.7%) had adult-onset Still's disease. Median (range) age at HLH diagnosis was 5 (0.9-39) years. Most (9/15; 60.0%) patients initiated emapalumab in an intensive care unit. Emapalumab was most frequently initiated for treating refractory or recurrent (10/15; 66.7%) disease. Most patients received HLH-related therapies prior to (10/15; 66.7%) and concurrently (15/15; 100.0%) with emapalumab. Emapalumab-containing regimens stabilized or achieved physician-determined normalization of most laboratory parameters including fibrinogen (11/13; 84.6%), chemokine ligand 9 (7/8; 87.5%), and absolute neutrophil count (6/10; 60%), and reduced glucocorticoid dose by 80%. Overall survival and 12-month survival probability from emapalumab initiation were 86.7%. Emapalumab-containing regimens stabilized or normalized most key laboratory parameters, reduced glucocorticoid dose, and were associated with low disease-related mortality, thereby demonstrating potential benefits in patients with rheumatologic disease-associated HLH.

Switching hemophilia A patients to rVIII-SingleChain: The Iberian experience.

The real-world outcomes of lonoctocog alfa (rVIII-SingleChain), a long-acting factor VIII (FVIII) with a favorable safety and efficacy profile in trials, were assessed in patients with hemophilia A in Iberian (Spain and Portugal). This was a retrospective study involving patients switching to rVIII-SingleChain from other FVIIIs in 7 Spanish and Portuguese hospitals. The efficacy and safety of replacement therapies were compared between 12 months before switching and the period from switching to the end of the study. Twenty-nine patients (median age 25 years; severe hemophilia A, 37.9%) were recruited. Before switching, 12 were on prophylaxis and were followed-up for a median of 12 months. After switching, 17 received prophylaxis with rVIII-SingleChain and were followed-up for a median of 41 months. Those with ≤2 weekly infusions increased from 37.5% before switching to 60.7% after switching to rVIII-SingleChain. The median monthly consumption was 312 IU/kg with prior FVIIIs and 273 IU/kg with rVII-SingleChain. Six spontaneous bleeds were reported in each period in the prophylaxis patients. In the entire cohort, 50 bleeds were reported with prior FVIIIs and 33 were reported after switching to rVIII-SingleChain. Patients requiring ≤1 dose for hemostasis increased from 44.0% with prior FVIIIs to 60.6% with rVIII-SingleChain. Responses were rated good/excellent in 95.4% of cases. No safety concerns were reported. Patients who switched to rVIII-SingleChain prophylaxis had excellent bleeding control and reduced infusion frequency in regular clinical practice, with the subsequent increase in quality-of-life.

Outcomes of first-line treatment and their association with pretreatment neutrophil-to-lymphocyte ratio in patients with advanced renal cell carcinoma: Insights from a tertiary care institute in Pakistan.

Renal cell carcinomas (RCCs) are renal parenchymal neoplasms that contribute to <5% of cancer cases worldwide. Within the diverse group of renal tumours, clear cell carcinoma is the most common subtype. The recommended first-line treatment for metastatic disease is a tyrosine kinase inhibitor given either as monotherapy or in combination with an immune checkpoint inhibitor, based on improved survival outcomes. These endpoints are not only influenced by the initial risk stratification but also by certain variables such as the neutrophil-to-lymphocyte (NLR) ratio. A retrospective review was conducted to evaluate the progression-free survival (PFS) with first-line treatment in patients with metastatic RCC treated at our institute from the year 2017-2021. We also investigated the association of PFS with both Memorial Sloan Kettering Cancer Center risk groups and the pretreatment NLR ratio. Overall, 35 patients were enrolled after fulfilling the eligibility criteria. Of these, 25 patients received Pazopanib, 5 patients were treated with Sunitinib and the remaining patients were administered Pembrolizumab with Axitinib. Two-thirds of the study population belonged to the intermediate-risk group. The median PFS for all participants was 16 months. Among the overall population, patients in the favourable-risk group demonstrated superior PFS. Patients with elevated pretreatment NLR experienced shorter PFS compared to the patients with low to normal NLR. This review highlights the prognostic significance of initial risk stratification and pretreatment NLR in predicting the response to first-line treatment in metastatic RCC patients. As this is a comprehensive study emphasizing the outcomes of metastatic RCC in Pakistan, it fills a void in the literature by providing invaluable perspectives on the real-world outcomes of patients. This not only enhances our understanding of disease management in this region but also lays the foundation for future investigations.

A Closer Look at Race, Ethnicity, and Gender Neutral Models

Abstract Litigation cases involving the expected earnings of minor children provide an ideal environment to examine the implications of public policies that attempt to mitigate racial, ethnic, and gender discrimination in the calculation of economic damages. This paper extends the existing literature on the implications of race, ethnicity, and gender neutral models by evaluating both educational attainment probabilities and estimated lifetime earnings within educational attainment categories. Recent related studies have focused on the educational attainment portion of the calculation only. We perform thousands of simulations of a child’s lifetime earnings using neutral estimations—a proxy for removing discrimination—and compare those estimates to ones in which race, ethnicity, and gender are taken into account. We find wide variation within racial and ethnic groups when earnings are incorporated into the analysis, consistent with prior research. Importantly, however, our findings pertain primarily to females, suggesting that interactions between gender and race and ethnicity are an added complexity. The practical implication of our findings is that neutral models can adversely impact individual members of protected groups. The results provide further guidance to policymakers who might be interested in the real-world outcomes associated with legislation that restricts how economists calculate economic damages.

Real-World Clinical Characteristics and Outcomes with Daptomycin Use in Pediatric Patients: A Retrospective Case Series.

Daptomycin (DAP) is a cyclic lipopeptide that exhibits potent in vitro activity against many drug-resistant gram-positive organisms, including methicillin-resistant Staphylococcus aureus (MRSA) and vancomycin-resistant enterococci (VRE). Despite substantial reports evaluating the clinical outcomes of DAP within the adult population, real-world data are lacking in children. The primary goal of this evaluation was to describe the clinical characteristics and outcomes of DAP use in pediatric patients across a wide range of infections. This retrospective evaluation included patients < 18 years of age who were treated with DAP from January 2014 to May 2023. The primary objective was to evaluate the composite clinical success, which was defined as a 30-day survival, the lack of a 30-day microbiological recurrence, and the resolution of signs and symptoms of an acute infection without therapy modifications based on clinical failures. Secondary objectives included adverse effects potentially attributable to DAP and reasons for DAP utilization. Forty patients were included, which were predominately male (62.5%) and white (52.5%), with a median age of 8.7 [IQR, 4.4-16.0] years. DAP was used for a wide range of infections, including central line-associated bloodstream infections (CLABSIs; 32.5%), infective endocarditis (15.0%), surgical-site infections (12.5%), and osteomyelitis (12.5%). The most common pathogen isolated was MRSA (37.5%), and most patients were bacteremic (60.0%). The median DAP dose was 8 [IQR, 6-10] mg/kg, and the median duration of the DAP therapy was 11.5 [IQR, 4.8-18.8] days. Most patients achieved composite clinical success (75.0%). An adverse effect occurred in 5.0% of the patients. DAP was prescribed the most for its ease of use/ability to facilitate discharge (40.0%) and/or for issues with alternative therapies (37.5%). Most pediatric patients that received DAP demonstrated clinical success with a low incidence of adverse effects. Larger, real-world studies of DAP use are necessary to further assess clinical outcomes.

Real World Outcome of High-Risk Multiple Myeloma: An Indian Tertiary Care Centre Experience

High risk myeloma is a heterogeneous disease cohort with significant variation in risk stratification across literatures. Real world outcomes differ from controlled clinical trials and affected by socioeconomical determinants of health. This study from an Indian tertiary care set up highlights’ treatment patterns and outcome of real-world high risk myeloma patients. Out of 384,76(19.7%) high risk myeloma patients were analyzed (median age 58 years). Most common HRCA was 1 q gain 36(47.4%) followed by del17p 32(42.1%). 61/76(80.2%) received Bortezomib based triplets and 15(19.74%) daratumumab based quadruplets induction, 31(40.79%) received ASCT. Median duration of follow up was 19.5 months. The 2 year OS and PFS was 73.8%, 52.6% respectively. Estimated 3 year OS was 74.7% in ASCT cohort versus 52.9% (P = .0067) without. Estimated 3-year PFS in the ASCT cohort was 72.1% versus 30.3% (P = .0026) without. Estimated 3-year OS for single hit and multi hit ultra HRMM was 67.7% and 61.9% (P = .642) whereas PFS was 58.2% and 35.2% (P = .486) respectively. In multivariate analysis ASCT correlated with better OS (HR 0.3, P = .041) and PFS (HR 0.35,P = .012). Absence of baseline renal impairment correlated with better OS (HR 4.12, P = .004) only. Early aggressive therapy with prompt ASCT translates to a better survival in high risk myeloma. Emphasis on real world clinical outcome is the need of the hour for addressing practical issues and improving global myeloma outcome.

Real-World Evaluation of Treatment Patterns and Clinical Outcomes among Patients With Chronic Myeloid Leukemia in Chronic Phase Treated With Asciminib in Clinical Practice in the United States: Real-world asciminib treatment outcomes in CML-CP

BackgroundTyrosine kinase inhibitors (TKIs) are the mainstay treatment for chronic myeloid leukemia in chronic phase (CML-CP). Asciminib, an ABL/BCR::ABL1 inhibitor which binds to the myristoyl pocket, was recently approved in the US for patients with CML-CP previously treated with ≥2 TKIs or with the T315I mutation. This study described treatment patterns and real-world clinical outcomes among patients with CML-CP treated with asciminib in US clinical practice. MethodsElectronic health record data from adult patients with CML-CP who initiated asciminib after ≥2 prior TKIs, without the T315I mutation, were obtained from the Flatiron Health database. Time-to-treatment discontinuation and molecular response (MR; time-to-BCR::ABL ≤0.1% and time-to-BCR::ABL1 ≤1%, separately) were evaluated from asciminib initiation (index date) using Kaplan–Meier analyses. ResultsOverall, 97 patients initiated asciminib (median age: 63 years, 50.5% female, 64.9% White) after either 2 (47.4%) or 3 (24.7%), or ≥4 (27.8%) prior TKIs. In total, 85.7% and 78.1% of patients remained on asciminib by 12- and 24-weeks postindex, respectively. Among patients with ≥1 MR assessment postindex, 31.3% (95% confidence interval [CI]: 21.6%, 43.9%) and 49.7% (95% CI: 38.1%, 62.6%) achieved or maintained BCR::ABL1 ≤0.1%, while 51.3% (95% CI: 40.1%, 63.6%) and 64.2% (95% CI: 52.6%, 75.6%) achieved or maintained BCR::ABL1 ≤1%, by 12- and 24-weeks, respectively. ConclusionsResults of this real-world study describing clinical outcomes among patients with CML-CP treated with asciminib after ≥2 prior TKIs in the US demonstrated that asciminib was well-tolerated and effective. These findings were consistent with results from the ASCEMBL trial.

Credit card fraud detection with advanced graph based machine learning techniques

&lt;p&gt;In the realm of credit card fraud detection, the landscape is continually evolving, demanding innovative approaches to stay ahead of increasingly sophisticated fraudulent activities. Our research pioneers a groundbreaking methodology that amalgamates the power of bipartite graph visualization with advanced machine learning techniques. This fusion yields a comprehensive framework capable of effectively evaluating the efficacy of a random forest classifier in uncovering fraudulent credit card transactions. Our study showcases the compelling application of this methodology, offering a paradigm shift in how we analyze and understand credit card fraud detection systems. By seamlessly integrating machine learning algorithms with network analysis, we provide a holistic view of the data, unveiling intricate patterns hidden within. At the heart of our approach lies the innovative use of bipartite graphs, which serve as a dynamic visual bridge between model predictions and real-world outcomes. This visual representation not only enhances interpretability but also facilitates a deeper understanding of the classifier’s performance. By visually mapping the relationships between transactions and their respective classifications, our methodology offers actionable insights into both successful detection and potential areas for improvement. Empowering analysts and stakeholders, our approach facilitates informed decision-making by enabling them to fine-tune model parameters and enhance the overall effectiveness of fraud detection systems. Through this synergy between cutting-edge machine learning and network analysis techniques, we provide a powerful tool to combat the critical challenge of credit card fraud prevention. Step into the future of fraud detection with our innovative methodology, where every transaction is scrutinized with precision, and where security is not just a possibility, but a promise fulfilled.&lt;/p&gt;

1992P Real-world (RW) characteristics and outcomes in patients (pts) with muscle-invasive urothelial carcinoma (MIUC) treated with adjuvant nivolumab (NIVO) with or without neoadjuvant chemotherapy (NAC)

1992P Real-world (RW) characteristics and outcomes in patients (pts) with muscle-invasive urothelial carcinoma (MIUC) treated with adjuvant nivolumab (NIVO) with or without neoadjuvant chemotherapy (NAC)

CT-305 Real-World Clinical Outcomes in Patients With Paroxysmal Nocturnal Hemoglobinuria Treated With Eculizumab or Ravulizumab in the US: A Retrospective Claims Database Analysis

CT-305 Real-World Clinical Outcomes in Patients With Paroxysmal Nocturnal Hemoglobinuria Treated With Eculizumab or Ravulizumab in the US: A Retrospective Claims Database Analysis

374P Real-world (Rw) outcomes in patients (pts) with hormone receptor-positive and human epidermal growth factor receptor-2 negative (HR+/HER2-) metastatic breast cancer (mBC) treated with chemotherapy (CT) in France and Germany

374P Real-world (Rw) outcomes in patients (pts) with hormone receptor-positive and human epidermal growth factor receptor-2 negative (HR+/HER2-) metastatic breast cancer (mBC) treated with chemotherapy (CT) in France and Germany

1352P Real-world second-line outcomes of NSCLC patients receiving first-line chemotherapy plus immunotherapy

1352P Real-world second-line outcomes of NSCLC patients receiving first-line chemotherapy plus immunotherapy

Abstract P412: Pulmonary Hypertension in Patients with Preeclampsia: Prevalence, Predictors and Associated In-Hospital Adverse Events

Pulmonary hypertension (PH) is prevalent pregnant women, but there is a lack of research into its co-occurence with preeclampsia. Our study aimed to characterize and evaluate the real-world prevalence, predictors and outcomes of PH in preeclamptic patients Data were obtained from the National Inpatient Sample database from 2016 to 2019.Primary outcomes assessed were baseline and hospital admission characteristics and comorbidities for patients with preeclampsia with or without PH, outcomes for preeclamptic patients with PH, and predictors of mortality. Of 255,513 preeclamptic patients, 497 (.194%) developed PH. Patients with PH had a significantly elevated likelihood of having IDA (9.7% vs 2.6%, p&lt;.001), CHF (25.5% vs 0.4%, p&lt;.001), pulmonary disease (17.7% vs 7.6%, p&lt;.001), coagulopathy (2.0% vs 0.8%, p=.002), T2DM (7.2% vs 2.8%, p&lt;.001), hypertension (0.8% vs 0.3%, p=.014), alcohol abuse (1.4% vs 0.1%, p&lt;.001), liver disease (2.0% vs 0.4%, p&lt;.001), atrial fibrillation (0.6% vs 0.1%, p&lt;.001), hypothyroidism (7.0% vs 4.6%, p=.01), CAD (1.8% vs 0.1%, p&lt;.001), OSA (8.7% vs 0.7%, p&lt;.001), opioid use (1.8% vs 0.9%, p=.026), obesity (33.2% vs 19.4%, p&lt;.001), and ESRD (0.8% vs 0.0%, p&lt;.001). Univariate analysis showed that PH in preeclamptic patients was associated with higher rates of cardiac arrest (0.8% vs 0.0% p&lt;.001), permanent pacemaker (1.4% vs 0.1% p&lt;.001), ventricular fibrillation (0.2% vs 0.0% p&lt;.001), angioplasty (0.8% vs 0.0% p&lt;.001), PE (1.0% vs 0.0% p&lt;.001), shock after delivery (1.2% vs 0.1% p&lt;.001), PPH (10.1% vs 7.3% p=.017), right heart catheterization (2.0% vs 0.0% p&lt;.001), left heart catheterization (0.6% vs 0.0% p&lt;.001), cardiogenic shock (1.0% vs 0.0% p&lt;.001), medical ventilation (3.6% vs 0.1% p&lt;.001), vasopressor use (1.2% vs 0.1%, p&lt;.001), STEMI (0.2% vs 0.0%, p&lt;.001), and NSTEMI (1.2% vs 0.0% p&lt;.001). Predictors of mortality included age at admission (OR 1.06, 95% CI 1.0–1.1, p&lt;.05), Asian and Pacific Islander race (OR 4.89, 95% CI 1.9–12.7 p&lt;0.05), CHF (OR 19.41 , 95% CI –, p&lt;.05), coagulopathy (OR 19.4, 95% CI 6.4–58.8 p&lt;.05), liver disease (OR 6.5, 95% CI 2.0–21.3 p&lt;.05), eclampsia (OR 17.3, 95% CI 5.3–55.9 p&lt;.05), and HELLP syndrome (OR 6.2, 95% CI 2.8–13.5 p&lt;.05). PH in preeclampsia is associated with an increased risk of maternal mortality, heart failure, arrhythmia, pulmonary embolism. The present study found age at admission, AAPI race, CHF, coagulopathy, liver disease, eclampsia, and HELLP syndrome to be predictors of mortality.

1926MO Radioiodine-refractory differentiated thyroid cancer (RAI-R DTC) patients treated with lenvatinib monotherapy: Real-world treatment patterns and clinical outcomes in Europe and Canada

1926MO Radioiodine-refractory differentiated thyroid cancer (RAI-R DTC) patients treated with lenvatinib monotherapy: Real-world treatment patterns and clinical outcomes in Europe and Canada

1387P Real-world data, tolerability and clinical outcomes in patients with advanced NSCLC treated with sotorasib in the UK

1387P Real-world data, tolerability and clinical outcomes in patients with advanced NSCLC treated with sotorasib in the UK

1923P Real-world management and outcome of thymic carcinomas in a tertiary German center

1923P Real-world management and outcome of thymic carcinomas in a tertiary German center

Real-World Insights into CAR T-Cell Therapy: Efficacy and Safety of Kymriah in B-ALL and NHL

Background: Chimeric Antigen Receptor (CAR) T-cell therapy has emerged as a revolutionary treatment for certain cancers, particularly B-cell malignancies. This study focused on the real-world outcomes of Kymriah (tisagenlecleucel), a CAR T-cell therapy targeting CD19, by analyzing data from the Cellular Immunotherapy Data Resource (CIDR). A total of 410 patients with relapsed or refractory B-cell Acute Lymphoblastic Leukemia (B-ALL) and Non-Hodgkin Lymphoma (NHL) were included. Methods: Data were collected from 73 treatment centers across the U.S. and Canada. Key outcomes measured included Cytokine Release Syndrome (CRS), Immune-effector Cell-Associated Neurotoxicity Syndrome (ICANS), overall response rate (ORR), duration of response (DOR), and survival rates. Statistical analyses were performed using descriptive statistics, Kaplan-Meier methods, and logistic regression. Results: CRS was observed in 54.3% of B-ALL patients, with severe cases in 16.1%. The complete remission rate in B-ALL patients was 85.5%, with a 12-month overall survival rate of 76.4%. In NHL patients, the overall response rate was 62.4%, with 38.2% achieving complete remission. Severe CRS and ICANS were less frequent in NHL patients. Conclusion: Kymriah demonstrated high efficacy in both B-ALL and NHL, with manageable side effects. However, ongoing monitoring is essential to optimize outcomes and reduce adverse events. This study provides valuable real-world data, contributing to the growing understanding of CAR T-cell therapy’s potential in clinical practice.

1798P Relapsed and refractory systemic therapy real-world outcomes in the Canadian small cell lung cancer database (CASCADE)

1798P Relapsed and refractory systemic therapy real-world outcomes in the Canadian small cell lung cancer database (CASCADE)

Real-World Outcomes of First-Line Treatment With Anti–PD(L)1-Based Combination Therapy for Nonsquamous Metastatic Non–Small Cell Lung Cancer: A Multiregional Chart Review in Europe, Japan, and the United States

Real-World Outcomes of First-Line Treatment With Anti–PD(L)1-Based Combination Therapy for Nonsquamous Metastatic Non–Small Cell Lung Cancer: A Multiregional Chart Review in Europe, Japan, and the United States

463P Real-world outcomes of patients with non-small cell lung cancer with and without intracranial metastatic disease: A retrospective cohort analysis

463P Real-world outcomes of patients with non-small cell lung cancer with and without intracranial metastatic disease: A retrospective cohort analysis