Real-world Evidence Studies Research Articles

Clinical characteristics and outcomes of patients with takotsubo syndrome and dynamic left ventricular outflow tract obstruction

Abstract Background Left ventricular outflow tract obstruction (LVOTO) is recognized as a serious complication of Takotsubo syndrome (TTS). However, its impact on patient prognosis remains insufficiently investigated. Purpose This study aimed to assess clinical characteristics and short- and long-term outcomes of patients with TTS complicated by LVOTO. Methods Patients with TTS were enrolled from the Takotsubo Italian Network (TIN). The primary outcome was the composite of acute heart failure, cardiogenic shock, and mortality during hospitalization. Secondary outcomes included the single components of the primary outcome and the occurrence of death or TTS recurrence during follow-up. Results Overall, 881 patients were included in the study [median age 72.0 (63.0–78.0) years; 91.3% female], with LVOTO detected in 59 patients (6.7%). Low diastolic blood pressure, ST-segment elevation, and moderate-to-severe mitral regurgitation were independently associated with occurrence of LVOTO. Propensity score weighting regression analysis revealed a significant higher risk of the primary outcome (OR, 2.99; 95% CI, 2.22–4.02), acute heart failure (OR, 2.19; 95% CI, 1.59–3.02), cardiogenic shock (OR, 4.56; 95% CI, 3.07–6.78), and in-hospital mortality (OR, 2.42; 95% CI, 1.13–5.21) in patients with TTS complicated by LVOTO. No significant association between LVOTO and long-term mortality or TTS recurrence was observed during follow-up. Conclusions In this real-world evidence study, LVOTO was detected in about 7% of TTS patients and was associated with a significantly higher risk of adverse events during hospitalization, including mortality. Early detection of LVOTO can aid in prognostic assessment and guide therapeutic management in patients with TTS.

Collaborative Real-World Evidence Among Regulators: Lessons and Perspectives.

The International Coalition of Medicines Regulatory Authorities (ICMRA), comprising 38 global medicines regulatory authorities, collaborates on shared challenges, notably during the COVID-19 pandemic. This article focuses on the ICMRA COVID-19 Real-World Evidence (RWE) and Observational Studies Working Group. The Working Group aimed to address challenges related to RWE and observational studies during the pandemic, resulting in impactful studies and ICMRA statements on international collaboration for RWE and COVID-19 vaccine safety. Reflecting on 3 years of collaboration, the Working Group surveyed members for insights, and recommendations were formulated to enhance research preparedness, collaboration, and response to future public health emergencies. The lessons learned highlight the importance of global collaborations, governance structures for rapid decision-making, and effective utilization of existing networks. Recommendations include the establishment of an international governance structure, a "coalition of the willing" for swift research collaboration, dedicated sub-groups, periodic workshops, common protocols, joint timelines, and data model templates, leveraging existing infrastructure, and strengthening outreach for transparency and engagement. The Working Group envisions repurposing into an RWE strategic and operational entity, contributing to global public health emergency response mechanisms. In conclusion, the Working Group's success lies in effective communication, collaborative research, and leveraging existing infrastructure, with ongoing contributions to global emergency response mechanisms.

Prognostic factors and overall survival among patients with ovarian cancer in the pre-PARP inhibitor era: the OCRWE-Finland study.

Despite recent treatment advances in ovarian cancer (OC), more real-world evidence studies investigating patient outcomes are needed. OCRWE-Finland was an observational cohort study investigating OC outcomes in Finland during the pre-PARP inhibitor era. Patients were diagnosed with OC between 2014 and 2019 in Finland. This analysis reports baseline characteristics of all patients, patients with high-grade serous OC (HGSOC), and overall survival (OS) for patients with HGSOC. Among 1,711 patients diagnosed with OC, 867 (51%) had HGSOC. The absence versus presence of visible residual disease post-debulking surgery was associated with improved OS for patients at stage III (n = 303; median: NR vs. 43 months; p = 0.005), but not stage IV (n = 118; median: 37 months vs. 40 months; p = 0.96). Bevacizumab treatment at any line at stages III/IV improved OS in the short-term only. Receiving versus not receiving bevacizumab at first-line for patients with visible residual disease post-debulking surgery was associated with improved OS at stage III (median: 48 months vs. 36 months; p = 0.003), but not stage IV (median: 42 months vs. 37 months; p = 0.26). Multivariate Cox regression analyses showed that stage IV at initial diagnosis and the presence of R2 classification post-debulking surgery resulted in poorer OS. In the pre-PARP inhibitor era, the absence versus presence of visible residual disease post-debulking surgery was associated with improved OS in stage III, but not stage IV HGSOC. First-line bevacizumab seemed to be beneficial in patients with stage III HGSOC and visible residual disease.

A Mixed Methods Systematic Review of the Effectiveness, Acceptability, and Feasibility of Advanced Care Plans for Older Adults Without Life-Limiting Conditions

Context: Advance care planning (ACP) is a comprehensive procedure aimed at helping individuals understand and articulate their values, life goals, and preferences concerning future medical treatment. Despite extensive research on ACP in populations with terminal illnesses, dementia, and those in long-term care, there is a lack of focus on its impact among older adults without life-limiting conditions. Objectives: To investigate the effectiveness, acceptability, and feasibility of ACP for older adults without life-limiting conditions. Methods: We searched 11 electronic databases, spanning from inception to November 2023, complemented by extensive grey literature and reference searches. Rigorous quality assessments were conducted using the Mixed Methods Appraisal Tool (MMAT). Our approach adopted a dynamic convergent mixed-methods design, incorporating thematic and narrative synthesis. Findings: From over 12,600 citations, a total of 16 studies, comprising four randomised controlled trials (RCTs), nine qualitative studies, and three mixed methods studies met the inclusion criteria. Limited findings indicate that ACP interventions are generally effective in improving document completion rates and reducing hospital readmissions. Acceptability and feasibility of these interventions were high, although significantly influenced by cultural and religious factors. Limitations: Evidence supporting the effectiveness, acceptability, and feasibility of ACP in reducing unplanned hospital admissions in older adults without life-limiting conditions was limited. We excluded observations based on unwell older people, though some material with unclear distinctions may have been included. Quality assessments revealed that one of four RCTs had a high risk of bias, three had unclear risks; all nine qualitative studies had low risk; and one of three mixed methods studies had low risk while two had unclear risks. Implications: Further targeted research is necessary to assess the intervention’s value for this population. Rigorous real-world evidence studies involving older adults without life-limiting conditions and their proxies is crucial to enabling providers to tailor ACPs to diverse populations, whilst respecting individuals’ religious and cultural beliefs.

Real-world evidence study on the impact of SPECT MPI, PET MPI, cCTA and stress echocardiography on downstream healthcare utilisation in patients with coronary artery disease in the US.

Coronary artery disease (CAD) is associated with a large clinical and economic burden. However, consensus on the optimal approach to CAD diagnosis is lacking. This study sought to compare downstream healthcare resource utilisation following different cardiac imaging modalities, to inform test selection for CAD diagnosis. Claims and electronic health records data from the Decision Resources Group Real-World Evidence US Data Repository were analysed for 2.5 million US patients who underwent single-photon emission computed tomography myocardial perfusion imaging (SPECT MPI), positron emission tomography myocardial perfusion imaging (PET MPI), coronary computed tomography angiography (cCTA), or stress echocardiography between January 2016 and March 2018. Patients were stratified into nine cohorts based on suspected or existing CAD diagnosis, pre-test risk, and prior events or interventions. Downstream healthcare utilisation, including additional diagnostic imaging, coronary angiography, and cardiac-related health system encounters, was compared by cohort and index imaging modality. Among patients with suspected CAD diagnosed within 3 months of the index test, PET MPI was associated with lower downstream utilisation; 25-37% of patients who underwent PET MPI required additional downstream healthcare resources compared with 40-49% of patients who received SPECT MPI, 35-41% of patients who underwent cCTA, and 44-47% of patients who received stress echocardiography. Patients who underwent PET MPI experienced fewer acute cardiac events (5.3-9.4%) and generally had lower rates of healthcare encounters (0.8-4.1%) and invasive coronary angiography (ICA, 15.4-24.2%) than those who underwent other modalities. SPECT MPI was associated with more downstream ICA (31.3-38.2%) and a higher rate of cardiac events (9.5-13.2%) compared with PET MPI (5.3-9.4%) and cCTA (6.9-9.9%). Across all cohorts, additional diagnostic imaging was 1.6 to 4.7 times more frequent with cCTA compared with PET MPI. Choice of imaging modality for CAD diagnosis impacts downstream healthcare utilisation. PET MPI was associated with lower utilisation across multiple metrics compared with other imaging modalities studied.

Bedaquiline versus injectable containing regimens for rifampicin-resistant and multidrug-resistant tuberculosis in a reference center in Brazil – a real-world evidence study using a retrospective design

BackgroundDrug resistance (DR) is one of the several challenges to global tuberculosis (TB) control. The implementation of bedaquiline (BED) for DR-TB after more than 40 years was expected to improve treatment outcomes as well as microbiologic conversion and adverse events (AE) occurrence.MethodsRetrospective cohort study based on secondary data of patients with rifampicin-resistant (RR) or multidrug-resistant (MDR) TB reported to the Outpatient Clinic of Mycobacterial Diseases of the Thorax Diseases Institute – Federal University of Rio de Janeiro - Brazil, between 2016 and 2023. We aimed to evaluate microbiologic conversion, AE and TB treatment outcomes and compare them according to the treatment regimen used for RR/MDR-TB patients under routine conditions [Injectable Containing Regimens (ICR) versus BED Containing Regimens (BCR)]. Logistic regression and survival analysis using Cox regression and Kaplan Meier curve were used for statistical analysis.ResultsOf the 463 DR-TB patients notified during the study period, 297 (64.1%) were included for analysis (ICR = 197 and BCR = 100). Overall AEs were more frequent (83.7 vs. 16.3%, p < 0.001) and occurred earlier in the ICR group (15 days vs. 65 days, p = 0.003). There were no cases of cardiotoxicity requiring interruption of BED treatment. None of the regimens of treatment tested were associated with smear or culture conversion on Cox regression analysis (p = 0.60 and 0.88, respectively). BED-containing regimens were also associated with favorable outcomes in multivariable logistic regression [adjusted odds ratio (aOR) = 2.63, 95% confidence interval (CI)1.36–5.07, p = 0.004], as higher years of schooling, primary drug resistance, and no previous TB treatment. In the survival analysis, BCR was inversely associated with the occurrence of AE during treatment follow-up (aHR 0.24, 95% CI 0.14–0.41, p < 0.001). In addition, TB treatment regimens with BED were also associated with favorable outcomes (aHR 2.41, 95% CI 1.62–3.57, p < 0.001), along with no illicit drug use and primary drug resistance.ConclusionsThe implementation of a fully oral treatment for RR/MDR-TB in a reference center in Brazil was safe and associated with favorable outcomes under routine conditions, despite social, demographic, and behavioral factors that may influence TB treatment completion.

Comparative effectiveness of endoscopic plantar fasciotomy, needle knife therapy, and conventional painkillers in the treatment of plantar fasciitis: a real-world evidence study

Plantar fascia (PF) is the commonest causes of foot pain in the adult population. Several surgical treatments are available to treat PF. This study was aimed to investigate the clinical efficacy of three different treatments for plantar fasciitis. It was conducted among 60 patients, divided equally into three treatment groups named Needle Knife Therapy, Endoscopic Plantar Fasciotomy, and Conventional Painkillers. Descriptive and analytical analysis were done by using SPSS 25 software. VAS and AOFAS scores were analyzed. The maximum (n = 31) participants were in 41–60 year age range group with normal BMI. These differences of mean VAS and AOFAS pain scores between different treatment groups were statistically significant (One-way ANOVA, p < 0.01). Both scores found lower in the Endoscopic Plantar Fasciotomy group. This study opens a new window of knowledge to achieve sustained pain relief and functional improvement. Moreover, the superiority of Endoscopic Plantar Fasciotomy in treating plantar fasciitis compared to Needle Knife Therapy and Conventional Painkillers was explored.

A real world evidence (RWE) study to evaluate the adherence to a nutritional support program and its relationship with the nutritional status evaluated by morphofunctional assessment in malnourished cancer patients. The OnaVIDA study.

A real world evidence (RWE) study to evaluate the adherence to a nutritional support program and its relationship with the nutritional status evaluated by morphofunctional assessment in malnourished cancer patients. The OnaVIDA study.

Long-Term Outcomes of Patients with FSGS: A Multicenter Real-World Evidence (RWE) Study

Long-Term Outcomes of Patients with FSGS: A Multicenter Real-World Evidence (RWE) Study

Real world evidence study to assess incidence, treatment patterns, clinical outcomes, and health care resource utilization in early-stage, high-risk HER2-negative breast cancer in Alberta, Canada

BackgroundData are needed to improve the current understanding of the epidemiology of patients with high-risk, HER2-negative, early breast cancer (eBC) (hormone receptor positive [HR+]/HER2-negative BC and triple-negative BC [TNBC]). Patients and MethodsThis retrospective longitudinal cohort study used real-world, population-level data that included all individuals newly diagnosed with high-risk, HER2-negative eBC in Alberta, Canada, between 2010-2019. Data on treatment, laboratory results and pathology findings were collected through electronic health records and administrative databases. ResultsThe annual cumulative incidence of high-risk, HER2-negative eBC ranged from 6-9% of all incident BC cases. Individuals with TNBC were more likely to be younger, had stage II disease, grade 3 histology and received systemic therapy at a community centre (p < 0.05) compared to individuals with HR+/HER2-negative eBC. Only 14% of individuals diagnosed in 2010-2017 underwent germline BRCA testing post-diagnosis. Neoadjuvant systemic therapy was given to 37% of individuals. Adjuvant systemic therapy use increased from 77% (2012-2015) to 84% (2019). The 5-year overall survival (OS) from initiation of adjuvant systemic therapy or date of surgery (for individuals who did not receive adjuvant systemic therapy) was 77% (95% CI: 75 to 79). OS was significantly worse among individuals who were older, had grade 3 histology, had stage III disease, or had nodal involvement (p < 0.05). OS among individuals with TNBC between 2016-2019 who initiated adjuvant capecitabine was markedly worse compared to the overall cohort (2-year OS: 70% vs. 89%). ConclusionOutcomes analyses in this high-risk, HER2-negative eBC population suggest a continued unmet clinical need. Micro abstractWe characterized treatment patterns and clinical outcomes in early-stage, high-risk HER2-negative breast cancer (BC) (HR+/HER2-negative BC and triple-negative BC). Adjuvant systemic therapy use increased from 77% (2012-2015) to 84% (2019). The 5-year overall survival from initiation of adjuvant systemic therapy or date of surgery (for individuals who did not receive adjuvant systemic therapy) was 77% (95% CI: 75 to 79).

A-133 Real-world evidence study of brentuximab vedotin retreatment in patients with cutaneous T-cell lymphoma

A-133 Real-world evidence study of brentuximab vedotin retreatment in patients with cutaneous T-cell lymphoma

Patient Profile-Based Management with Nintedanib in Patients with Idiopathic Pulmonary Fibrosis.

A severe and progressive interstitial lung disease (ILD) known as idiopathic pulmonary fibrosis (IPF) has an unknown etiology with poorly defined mechanisms of development. Among the currently prescribed pharmacological interventions for IPF, nintedanib demonstrates the ability to decelerate the deterioration of lung function and yield positive clinical outcomes. Multiple randomized placebo-controlled trials have confirmed the efficacy and acceptable safety profile of nintedanib. Real-world evidence studies also support the use of nintedanib in IPF, being an efficient and well-tolerated treatment option. It has the potential to stabilize the disease progression in patients with ILD. Patients with IPF frequently have comorbidities like diabetes and hypertension, which can exacerbate the course of disease, reduce quality of life, and decrease treatment adherence. For well-informed decision-making, it is important for healthcare professionals to recognize the position of nintedanib therapy in IPF with comorbidities. The gastrointestinal adverse effects, notably diarrhea, dominate the nintedanib safety profile. These can be effectively controlled by closely monitoring side effects, administering anti-diarrheal and anti-emetic drugs, reducing the nintedanib dose, and discontinuing it in case of severe symptoms with an option to reintroduce the treatment after side effects subside. Symptomatic interventions and monitoring of liver enzymes may reduce the occurrence of permanent treatment discontinuations.

Treatment patterns in hormone-sensitive metastatic prostate cancer: data from a referral hospital in Bogota, real-world evidence study

Treatment patterns in hormone-sensitive metastatic prostate cancer: data from a referral hospital in Bogota, real-world evidence study

Outcomes of a patient support programme for subcutaneous immunoglobulin therapy in patients with primary or secondary immunodeficiencies or chronic inflammatory demyelinating polyneuropathy.

Subcutaneous immunoglobulin (SCIg) therapy is important in the treatment of primary (PID) and secondary immunodeficiencies (SID) and chronic inflammatory demyelinating polyneuropathy (CIDP). Patient support programmes (PSPs) help patients self-administer medication regimens and play a more active role in the self-management of their medical conditions. To describe the effectiveness of the CSL Behring CARES PSP in optimising the quality use of SCIg in a hospital-free environment. This retrospective, observational study analysed records of patients enroled in the CSL Behring CARES PSP. Key outcomes were accessibility and effectiveness. Data were extracted from the patient database and analysed using descriptive methods. Seven hundred eighty-nine patients with PID (30.8%), SID (53.4%) and CIDP (15.8%) were enroled in the CARES PSP, 92.8% of whom were referred from public hospitals and the remaining from private hospitals. Of the total patient population, 697 (88.3%) received the nurse-led SCIg self-administration training and education (COACH), out of which 656 (94.1%) completed training and achieved competency after an average of 2.3 training sessions. The proportions of patients who achieved competency were similar across age groups and prior SCIg hospital education status. This is the largest real-world evidence study that describes the effectiveness of SCIg PSPs across three therapeutic disease states. These PSPs can optimise hospital resources such as infusion nurse time and allocation of infusion chairs that were once used for intravenous immunoglobulin infusions, improve patient access to SCIg therapy and enable patients self-administer SCIg outside a hospital environment.

The impact of different censoring methods for analyzing survival using real-world data with linked mortality information: a simulation study

BackgroundEvaluating outcome reliability is critical in real-world evidence studies. Overall survival is a common outcome in these studies; however, its capture in real-world data (RWD) sources is often incomplete and supplemented with linked mortality information from external sources. Conflicting recommendations exist for censoring overall survival in real-world evidence studies. This simulation study aimed to understand the impact of different censoring methods on estimating median survival and log hazard ratios when external mortality information is partially captured.MethodsWe used Monte Carlo simulation to emulate a non-randomized comparative effectiveness study of two treatments with RWD from electronic health records and linked external mortality data. We simulated the time to death, the time to last database activity, and the time to data cutoff. Death events after the last database activity were attributed to linked external mortality data and randomly set to missing to reflect the sensitivity of contemporary real-world data sources. Two censoring schemes were evaluated: (1) censoring at the last activity date and (2) censoring at the end of data availability (data cutoff) without an observed death. We assessed the performance of each method in estimating median survival and log hazard ratios using bias, coverage, variance, and rejection rate under varying amounts of incomplete mortality information and varying treatment effects, length of follow-up, and sample size.ResultsWhen mortality information was fully captured, median survival estimates were unbiased when censoring at data cutoff and underestimated when censoring at the last activity. When linked mortality information was missing, censoring at the last activity date underestimated the median survival, while censoring at the data cutoff overestimated it. As missing linked mortality information increased, bias decreased when censoring at the last activity date and increased when censoring at data cutoff.ConclusionsResearchers should consider the completeness of linked external mortality information when choosing how to censor the analysis of overall survival using RWD. Substantial bias in median survival estimates can occur if an inappropriate censoring scheme is selected. We advocate for RWD providers to perform validation studies of their mortality data and publish their findings to inform methodological decisions better.

A systematic review of health-related quality of life outcomes in patients with advanced breast cancer treated with palbociclib.

Aim: To evaluate the impact of palbociclib treatment on health-related quality of life (HRQoL) in patients with hormone receptor-positive, human epidermal growth factor 2-negative advanced breast cancer (HR+/HER2- aBC) or metastatic breast cancer (mBC) in both the clinical and real-world setting. Materials & methods: A systematic literature review was conducted to identify clinical trials and real-world evidence studies up to June 2023 that reported HRQoL outcomes in patients with HR+/HER2- aBC or mBC treated with Palbociclib. Results: 15 unique studies reported across 35 records were identified. Of these, seven were randomized controlled trials (RCTs), three were single-arm clinical trials and five were real-world evidence (RWE) studies. HRQoL was generally found to be maintained in patients with HR+/HER2- aBC or mBC across RCTs, single-arm clinical trials and RWE studies. HRQoL measures across instruments, study types and line of therapy, were largely reported to be at least maintained if not improved from baseline among patients treated with palbociclib and were observed to be comparable or better in the palbociclib group versus monotherapy control arm in RCTs. Similar results were seen for treatment-related outcomes (e.g., sexual functioning, upset by hair loss, systemic therapy side effects etc.), and important individual patient outcomes, including pain, fatigue and physical functioning. Findings were also consistent across key clinical characteristics (visceral metastases, neutropenia), as well as patient populations often underrepresented in clinical trials (Asian patients, older adults). Conclusion: Overall, current evidence suggests that HRQoL is largely preserved with the addition of palbociclib to endocrine therapy in patients with HR+/HER2- aBC or mBC across study types and populations.

High-dimensional Iterative Causal Forest (hdiCF) for Subgroup Identification Using Health Care Claims Data.

We recently developed a machine-learning subgrouping algorithm, iterative causal forest (iCF), to identify subgroups with heterogeneous treatment effects (HTEs) using predefined covariates. However, such predefined covariates may miss or poorly define important features leading to inaccurate subgrouping. To address such limitations, we developed a new semi-automatic subgrouping algorithm, hdiCF, which adapts methodology from high-dimensional propensity score for feature recognition in claims data. The hdiCF algorithm has 3 steps: 1) high-dimensional feature identification by International Classification of Diseases, Current Procedural Terminology, and Anatomical Therapeutic Chemical codes (in/outpatient diagnoses, procedures, prescriptions) and creation of ordinal variables by frequency of occurrence; 2) propensity score trimming and high-dimensional feature preparation; 3) iCF implementation to identify subgroups. We applied hdiCF in a 20% random sample of fee-for-service Medicare beneficiaries who initiated sodium-glucose cotransporter-2 inhibitors (SGLT2i) or glucagon-like peptide-1 receptor agonists to identify subgroups with HTEs for incidence of hospitalized heart failure. HdiCF findings were consistent with studies suggesting SGLT2i to be more beneficial for patients with pre-existing heart failure or chronic kidney disease. HdiCF is not dependent on prior hypotheses about HTEs and identifies subgroups with markers for potential HTEs in real-world evidence studies where active-comparator, new-user study designs limit the potential for unmeasured confounding.

Improved Clinical Outcomes with the Combination Therapy of a Glucagon-like Peptide-1 Receptor Agonists and a Sodium-glucose Cotransporter-2 Inhibitor in Overweight/Obese People with Type 2 Diabetes: Real-world Evidence from the Indian Subcontinent.

Type 2 diabetes (T2D) mellitus is increasing exponentially in India, with overweight/obesity being prime contributors. This study aimed at assessing the clinical impact of the combination therapy of a glucagon-like peptide-1 receptor agonist (GLP-1RA) injection (inj.) and a sodium-glucose cotransporter-2 inhibitor (SGLT-2i) in overweight/obese patients from the Indian subcontinent. In two real-world evidence (RWE) studies (RWE1 and 2), we retrospectively observed the effect of the combination therapy of a GLP-1RA, injection dulaglutide (DU) 1.5 mg/week, and an SGLT-2i, canagliflozin (CAN) 100 mg/day at weeks 16, 32, and 52 on HbA1c, body weight (weight), systolic blood pressure (SBP), lipids, change in doses of antidiabetic agents (ADA) and antihypertensive agents (AHA) in overweight/obese Asian Indian subjects with T2D, who had suboptimal glycemic control. A total of 95 T2D patients [51 males (M), 44 females (F)] completed the two RWE studies. In RWE 1, 40 patients (20 M/20 F), mean [standard deviation (SD)] age 49.4 (10.7) years (Y), weight 92.6 (6.6) kg, body mass index (BMI) 30.6 (2.3) kg/m2, duration of T2D 8.1 (3.2) Y, completed the study. At week 32, the mean (SD) reduction in A1c (%) was -1.3% [8.4 (0.7) to 7.1 (0.3); p < 0.01] and mean (SD) weight (kg) loss was -5.5 [92.6 (6.6) to 87.9 (7.02); p < 0.00001]. This group was then followed up until week 52. In RWE 2, 55 patients (31 M/24 F), age 51 (5.8) Y, weight 92.6 ± 3.4 kg, BMI 31.1 ± 2.1 kg/m2, SBP 142.4 ± 3.9 mm Hg, estimated glomerular filtration rate (eGFR) 62 ± 5 mL/minute/1.73 m2, with 8.4 ± 3.3 Y duration of diabetes met the inclusion criteria. In 71% of subjects, A1c decreased by -1.4% [8.5 ± 0.4 to 7.1 ± 0.2; p < 0.0001] at week 16 and was 6.8 ± 0.3 (p = 0.0002) in 18% at week 32. A statistically significant (SS) improvement in glycemic control, weight, and improvement in cardiovascular (CV) risk factors was observed with the GLP-1RA/SGLT-2i combination, which was well tolerated.

262.5: Effectiveness of the treatment of antibody mediated rejection- a real-world evidence study.

262.5: Effectiveness of the treatment of antibody mediated rejection- a real-world evidence study.

Standardization of coding definitions for sickle cell disease complications: A systematic literature review.

Sickle cell disease (SCD) affects all organ systems and is characterized by numerous acute and chronic complications and comorbidities. Standardized codes are needed for complications/comorbidities used in real-world evidence (RWE) studies that rely on administrative and medical coding. This systematic literature review was conducted to produce a comprehensive list of complications/comorbidities associated with SCD, along with their diagnosis codes used in RWE studies. A search in MEDLINE and Embase identified studies published from 2016 to 2023. Studies were included if they were conducted in US SCD populations and reported complications/comorbidities and respective International Classification of Diseases, Clinical Modification (ICD-CM) codes. All identified complications/comorbidities and codes were reviewed by a certified medical coding expert and hematologist. Of 1851 identified studies, 39 studies were included. The most reported complications/comorbidities were stroke, acute chest syndrome, pulmonary embolism, venous thromboembolism, and vaso-occlusive crisis. Most of the studies used ICD-9-CM codes (n = 21), while some studies used ICD-10-CM codes (n = 3) or both (n = 15), depending on the study period. Most codes reported in literature were heterogeneous across complications/comorbidities. The medical coding expert and hematologist recommended modifications for several conditions. While many studies we identified did not report their codes and were excluded from this review, the studies with codes exhibited diverse coding definitions. By providing a standardized set of diagnosis codes that were reported by studies and reviewed by a coding expert and hematologist, our review can serve as a foundation for accurately identifying complications/comorbidities in future research, and may reduce heterogeneity, enhance transparency, and improve reproducibility. Future efforts focused on validating these code lists are needed.