Cost-utility analysis of insulin analogues compared with multiple daily injections of human insulin for the treatment of 15 years old or older patients with type 1 diabetes mellitus in Colombia

A609-A609Insulin analogues offer certain advantages over regular human insulin. Our objective was to establish the incremental cost-effectiveness ratio (ICER) of insulin analogues compared with human insulin, with multiple daily injections (MID) in adult patients with type 1 diabetes mellitus (DM1) in Colombia. We designed a Markov model of annual cycles, time horizon of up to 55 years, from a third-party payer perspective (only direct medical costs for the Colombian healthcare system) and applying discount rate of 3.5% both for costs and outcomes. Quality-adjusted life years (QALYs), taken from published literature, were used as a measure of effectiveness with a threshold of three times the per capita gross domestic product (GDP), approximately € 17,550. The comparison between analog and human insulin was performed separately for short and long term. The costs were in Colombian pesos (1 € = COP 2.660), and were built through base cases designed by interdisciplinary expert panels, with drug prices and costs of interventions or adverse events were estimated using official tariff manuals and databases. Univariate and probabilistic sensitivity analysis were performed. Despite certain clinical advantages, insulin analogues gain, on average, very few QALYs (from 0.02 to 0.1 depending on scenarios). With this result, the average ICER for short-term insulins would be € 78,900; while long duration insulin analogues have an ICER of € 94,550. Results were highly sensitive to price of medication, as well as to disutility or costs of hypoglycemic events. Given the assumptions and limitations of our model, insulin analogues would not be considered cost-effective within the Colombian healthcare system compared with multiple daily injections for adult patients with DM1

Cost utility analysis of continuous infusion pump with integrated monitoring compared with multiple daily injection treatment for patients 15 years or older with type 1 diabetes mellitus in Colombia

Q1Q1360-361Objectives To estimate the incremental cost-utility ratio (ICUR) for the use of a continuous infusion pump with integrated monitoring (SAP) compared with the application of multiple daily injections (MDI) in adult (>15 year-old) patients with type 1 diabetes mellitus (DM1) in Colombia. Methods We designed an annual cycle Markov model, with transition probabilities obtained from a systematic review of the literature. Outcomes included in the search were ketoacidosis, severe hypoglycemia, microvascular complications (retinopathy, nephropathy, neuropathy), quality of life (expressed in quality-adjusted life years, QALYs, and obtained from Tufts database), as well as mortality. Other features of the model were: discount rate 3.5% for costs and QALYs, third party payer perspective (only direct medical costs), and a lifespan time horizon (up to 55 years). Threshold used was three times per capita gross domestic product (GDP), equivalent to € 17,547. Costs were estimated in 2014 Colombian pesos (1 euro = 2,660 COP), from base case scenarios (built by expert panels) and official tariff manuals. Univariate and probabilistic sensitivity analysis were performed. Results For the average patient, continuous infusion pump has a total expected cost of € 199,296, and generates 16.40 QALYs, while multiple daily injections would cost € 104737, and generate 14.48 QALYs (ICER € 49,302 per additional QALY gained). Sensitivity analysis shows that the only critical variable is the annual cost of continuous infusion pump treatment, which would have to be reduced by 40% to reach the cost-utility threshold. Conclusions Under the assumptions of the model, treatment with continuous infusion pumps would not be cost-effective for the average adult DM1 patient in Colombia. Further analysis would be required, addressed to certain selected subgroups of patients

Use of Machine-Learning Algorithms in Intensified Preoperative Therapy of Pancreatic Cancer to Predict Individual Risk of Relapse

Background: Although surgical resection is the only potentially curative treatment for pancreatic cancer (PC), long-term outcomes of this treatment remain poor. The aim of this study is to describe the feasibility of a neoadjuvant treatment with induction polychemotherapy (IPCT) followed by chemoradiation (CRT) in resectable PC, and to develop a machine-learning algorithm to predict risk of relapse. Methods: Forty patients with resectable PC treated in our institution with IPCT (based on mFOLFOXIRI, GEMOX or GEMOXEL) followed by CRT (50 Gy and concurrent Capecitabine) were retrospectively analyzed. Additionally, clinical, pathological and analytical data were collected in order to perform a 2-year relapse-risk predictive population model using machine-learning techniques. Results: A R0 resection was achieved in 90% of the patients. After a median follow-up of 33.5 months, median progression-free survival (PFS) was 18 months and median overall survival (OS) was 39 months. The 3 and 5-year actuarial PFS were 43.8% and 32.3%, respectively. The 3 and 5-year actuarial OS were 51.5% and 34.8%, respectively. Forty-percent of grade 3-4 IPCT toxicity, and 29.7% of grade 3 CRT toxicity were reported. Considering the use of granulocyte colony-stimulating factors, the number of resected lymph nodes, the presence of perineural invasion and the surgical margin status, a logistic regression algorithm predicted the individual 2-year relapse-risk with an accuracy of 0.71 (95% confidence interval [CI] 0.56-0.84, p = 0.005). The model-predicted outcome matched 64% of the observed outcomes in an external dataset. Conclusion: An intensified multimodal neoadjuvant approach (IPCT + CRT) in resectable PC is feasible, with an encouraging long-term outcome. Machine-learning algorithms might be a useful tool to predict individual risk of relapse. A small sample size and therapy heterogeneity remain as potential limitations

Clinical Characteristics and Treatment Patterns of Children and Adults With IgA Nephropathy or IgA Vasculitis: Findings From the CureGN Study

Introduction: The Cure Glomerulonephropathy Network (CureGN) is a 66-center longitudinal observational study of patients with biopsy-confirmed minimal change disease, focal segmental glomerulosclerosis, membranous nephropathy, or IgA nephropathy (IgAN), including IgA vasculitis (IgAV). This study describes the clinical characteristics and treatment patterns in the IgA cohort, including comparisons between IgAN versus IgAV and adult versus pediatric patients. Methods: Patients with a diagnostic kidney biopsy within 5 years of screening were eligible to join CureGN. This is a descriptive analysis of clinical and treatment data collected at the time of enrollment. Results: A total of 667 patients (506 IgAN, 161 IgAV) constitute the IgAN/IgAV cohort (382 adults, 285 children). At biopsy, those with IgAV were younger (13.0 years vs. 29.6 years, P < 0.001), more frequently white (89.7% vs. 78.9%, P = 0.003), had a higher estimated glomerular filtration rate (103.5 vs. 70.6 ml/min per 1.73 m2, P < 0.001), and lower serum albumin (3.4 vs. 3.8 g/dl, P < 0.001) than those with IgAN. Adult and pediatric individuals with IgAV were more likely than those with IgAN to have been treated with immunosuppressive therapy at or prior to enrollment (79.5% vs. 54.0%, P < 0.001). Conclusion: This report highlights clinical differences between IgAV and IgAN and between children and adults with these diagnoses. We identified differences in treatment with immunosuppressive therapies by disease type. This description of baseline characteristics will serve as a foundation for future CureGN studies

Impact of the COVID-19 pandemic on faecal immunochemical test-based colorectal cancer screening programmes in Australia, Canada, and the Netherlands: a comparative modelling study

Background: Colorectal cancer screening programmes worldwide have been disrupted during the COVID-19 pandemic. We aimed to estimate the impact of hypothetical disruptions to organised faecal immunochemical test-based colorectal cancer screening programmes on short-term and long-term colorectal cancer incidence and morta

Sickle Cell Disease: A Paradigm for Venous Thrombosis Pathophysiology

Venous thromboembolism (VTE) is an important cause of vascular morbidity and mortality. Many risk factors have been identified for venous thrombosis that lead to alterations in blood flow, activate the vascular endothelium, and increase the propensity for blood coagulation. However, the precise molecular and cellular mechanisms that cause blood clots in the venous vasculature have not been fully elucidated. Patients with sickle cell disease (SCD) demonstrate all the risk factors for venous stasis, activated endothelium, and blood hypercoagulability, making them particularly vulnerable to VTE. In this review, we will discuss how mouse models have elucidated the complex vascular pathobiology of SCD. We review the dysregulated pathways of inflammation and coagulation in SCD and how the resultant hypercoagulable state can potentiate thrombosis through down-regulation of vascular anticoagulants. Studies of VTE pathogenesis using SCD mouse models may provide insight into the intersection between the cellular and molecular processes involving inflammation and coagulation and help to identify novel mechanistic pathways