FRESC: French REAL World Extensive Stage Sclc Cohorts: A Retrospective Study on Patient Characteristics and Treatment STRATEGY Based on KBP-2010

Lung Cancer ◽  
2021 ◽  
Author(s):  
Didier Debieuvre ◽  
Charles Dayen ◽  
Adrien Dixmier ◽  
David Pau ◽  
Anna Sibley-Revelat ◽  
...  
2021 ◽  
Author(s):  
Kenichi Tanaka ◽  
Yosuke Okada ◽  
Akemi Tokutsu ◽  
Yoshiya Tanaka

Abstract Real-world data comparing the effectiveness of various glucagon-like peptide 1 receptor agonists (GLP-1 RAs) in type 2 diabetes mellitus (T2DM) are limited. We investigated the clinical effectiveness of liraglutide and dulaglutide in Japanese T2DM in a real-world setting. This retrospective study included 179 patients with T2DM who were treated with GLP-1 RA for at least 12 months (liraglutide, n=97; dulaglutide, n=82). Changes in glycated hemoglobin (HbA1c) at the end of 12-month treatment were evaluated. Compared with the liraglutide group, the dulaglutide group included significantly older patients with longer disease duration, lower body mass index, and higher proportion of dementia cases. HbA1c was significantly lower at 12 months in both groups (liraglutide, 8.9 to 7.6%; dulaglutide, 8.8 to 7.5%). Hierarchical regression analysis showed no differences in the extent of changes in HbA1c at 12 months between the two agents, after adjustment for differences in patient characteristics and drug adjustments during the 12-month period. High baseline HbA1c, the addition of GLP-1 RA treatment, and in-hospital initiation of GLP-1 RA treatment were identified as significant contributing factors to HbA1c reduction. Although patient characteristics were different between the two treatment groups, comparable HbA1c-lowering effects were noted in real-world settings.


2020 ◽  
Vol 16 (4) ◽  
pp. 327-333
Author(s):  
Shannon Armstrong-Kempter ◽  
Lucinda Beech ◽  
Sarah J. Melov ◽  
Adrienne Kirby ◽  
Roshini Nayyar

Background: The discovery of the benefits of antenatal corticosteroids (ACS) for preterm infants was one of the most significant developments in obstetric care. However, due to the difficulty in predicting preterm delivery, optimal use of ACS, is challenging. Objective: To describe prescribing practices for antenatal corticosteroids (ACS) at a tertiary hospital over five years to determine whether ACS were received at optimal timing; to determine patient characteristics of women receiving ACS at optimal timing; to determine patient characteristics of those who did not receive ACS as indicated and to examine the trend in ACS prescribing over the study period. Methods: We performed a retrospective study of all deliveries from January 2011 to December 2015. The rates of ACS prescription for each group of women (preterm, late preterm, and term) were recorded and analysed. Results: A total of 65% of women who delivered before 34 weeks’ gestation received ACS. Of these women, 63% delivered within 7 days of receiving ACS. Women most likely to receive ACS with optimal timing were primiparous (relative risk [RR], 1.25 [CI, 1.08-1.45]), or women diagnosed with pre-eclampsia (RR, 1.34 [CI 1.10-1.63]), preterm premature rupture of membranes (RR, 1.33 [CI, 1.15-1.54]) or threatened preterm labour (RR, 1.42 [CI, 1.22-1.65]). Conclusion: A significant number of women and babies are exposed to ACS without commensurate benefit, and a significant number who deliver preterm do not receive ACS. The percentage of preterm and term infants receiving ACS should be determined to optimise service delivery.


2021 ◽  
Vol 10 (7) ◽  
pp. 1527
Author(s):  
Jamie Duckers ◽  
Beth Lesher ◽  
Teja Thorat ◽  
Eleanor Lucas ◽  
Lisa J. McGarry ◽  
...  

Cystic fibrosis (CF) is a rare, progressive, multi-organ genetic disease. Ivacaftor, a small-molecule CF transmembrane conductance regulator modulator, was the first medication to treat the underlying cause of CF. Since its approval, real-world clinical experience on the use of ivacaftor has been documented in large registries and smaller studies. Here, we systematically review data from real-world observational studies of ivacaftor treatment in people with CF (pwCF). Searches of MEDLINE and Embase identified 368 publications reporting real-world studies that enrolled six or more pwCF treated with ivacaftor published between January 2012 and September 2019. Overall, 75 publications providing data from 57 unique studies met inclusion criteria and were reviewed. Studies reporting within-group change for pwCF treated with ivacaftor consistently showed improvements in lung function, nutritional parameters, and patient-reported respiratory and sino-nasal symptoms. Benefits were evident as early as 1 month following ivacaftor initiation and were sustained over long-term follow-up. Decreases in pulmonary exacerbations, Pseudomonas aeruginosa prevalence, and healthcare resource utilization also were reported for up to 66 months following ivacaftor initiation. In studies comparing ivacaftor treatment to modulator untreated comparator groups, clinical benefits similarly were reported as were decreases in mortality, organ-transplantation, and CF-related complications. The safety profile of ivacaftor observed in these real-world studies was consistent with the well-established safety profile based on clinical trial data. Our systematic review of real-world studies shows ivacaftor treatment in pwCF results in highly consistent and sustained clinical benefit in both pulmonary and non-pulmonary outcomes across various geographies, study designs, patient characteristics, and follow-up durations, confirming and expanding upon evidence from clinical trials.


2021 ◽  
pp. jim-2020-001633
Author(s):  
Florentino Carral San Laureano ◽  
Mariana Tomé Fernández-Ladreda ◽  
Ana Isabel Jiménez Millán ◽  
Concepción García Calzado ◽  
María del Carmen Ayala Ortega

There are not many real-world studies evaluating daily insulin doses requirements (DIDR) in patients with type 1 diabetes (T1D) using second-generation basal insulin analogs, and such comparison is necessary. The aim of this study was to compare DIDR in individuals with T1D using glargine 300 UI/mL (IGlar-300) or degludec (IDeg) in real clinical practice. An observational, retrospective study was designed in 412 patients with T1D (males: 52%; median age 37.0±13.4 years, diabetes duration: 18.7±12.3 years) using IDeg and IGla-300 ≥6 months to compare DIDR between groups. Patients using IGla-300 (n=187) were more frequently males (59% vs 45.8%; p=0.004) and had lower glycosylated hemoglobin (HbA1c) (7.6±1.2 vs 8.1%±1.5%; p<0.001) than patients using IDeg (n=225). Total (0.77±0.36 unit/kg/day), basal (0.43±0.20 unit/kg/day) and prandial (0.33±0.23 unit/kg/day) DIDR were similar in IGla-300 and IDeg groups. Patients with HbA1c ≤7% (n=113) used significantly lower basal (p=0.045) and total (p=0.024) DIDR, but not prandial insulin (p=0.241), than patients with HbA1c between 7.1% and 8% and >8%. Patients using IGla-300 and IDeg used similar basal, prandial and total DIDR regardless of metabolic control subgroup. No difference in basal, prandial and total DIDR was observed between patients with T1D using IGla-300 or IDeg during at least 6 months in routine clinical practice.


Blood ◽  
2020 ◽  
Author(s):  
Tina Dutt ◽  
Rebecca J Shaw ◽  
Matthew James Stubbs ◽  
Jun Yong ◽  
Benjamin Bailiff ◽  
...  

The cornerstone of life-saving therapy in immune mediated thrombotic thrombocytopenic purpura (iTTP) has been plasma exchange (PEX) combined with immunomodulatory strategies. Caplacizumab, a novel anti-von Willebrand factor nanobody, trialled in two multicentre, randomised-placebo-controlled trials leading to EU and FDA approval, has been available in the UK through a patient-access scheme. Data was collected retrospectively from 2018-2020 for 85 patients receiving caplacizumab, including 4 children, from 22 UK hospitals. Patient characteristics and outcomes in the real-world clinical setting were compared with caplacizumab trial endpoints and historical outcomes in the pre-caplacizumab era. 84/85 patients received steroid and rituximab alongside PEX; 26% required intubation. Median time to platelet count normalisation (3 days), duration of PEX (7 days) and hospital stay (12 days) was comparable with RCT data. Median duration of PEX and time from PEX initiation to platelet count normalisation was favourable compared with historical outcomes (p&lt;0.05). TTP recurrence occurred in 5/85 patients; all with persistent ADAMTS13 activity &lt;5iu/dL. Of 31 adverse events in 26 patients, 17/31 (55%) were bleeding episodes and 5/31 (16%) were thrombotic events (two unrelated to caplacizumab); mortality was 6% (5/85), with no deaths attributed to caplacizumab. In 4/5 deaths caplacizumab was introduced &gt;48 hours after PEX initiation (3-21 days). This real-world evidence represents the first and largest series of TTP patients receiving caplacizumab outside clinical trials, including paediatric patients. Representative of true clinical practice, the findings provide valuable information for clinicians treating TTP globally.


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