scholarly journals Trends in Real-World Neovascular AMD Treatment Outcomes in the UK

2020 ◽  
Vol Volume 14 ◽  
pp. 3331-3342
Author(s):  
Hemal Mehta ◽  
Leah N Kim ◽  
Thibaud Mathis ◽  
Pardis Zalmay ◽  
Faruque Ghanchi ◽  
...  
Eye ◽  
2016 ◽  
Vol 30 (11) ◽  
pp. 1462-1468 ◽  
Author(s):  
G Liew ◽  
◽  
A Y Lee ◽  
J Zarranz-Ventura ◽  
I Stratton ◽  
...  

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<0.05). TTP recurrence occurred in 5/85 patients; all with persistent ADAMTS13 activity <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 >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.


2020 ◽  
Vol 41 (Supplement_2) ◽  
Author(s):  
P McEwan ◽  
L Hoskin ◽  
K Badora ◽  
D Sugrue ◽  
G James ◽  
...  

Abstract Background Patients with chronic kidney disease (CKD), heart failure (HF), resistant hypertension (RHTN) and diabetes are at an increased risk of hyperkalaemia (HK) which can be potentially life-threatening, as a result of cardiac arrhythmias, cardiac arrest leading to sudden death. In these patients, renin-angiotensin-aldosterone system inhibitors (RAASi), are used to manage several cardiovascular and renal conditions, and are associated with an increased risk of HK. Assessing the burden of HK in real-world clinical practice may concentrate relevant care on those patients most in need, potentially improving patient outcomes and efficiency of the healthcare system. Purpose To assess the burden of HK in a real-world population of UK patients with at least one of: RHTN, Type I or II diabetes, CKD stage 3+, dialysis, HF, or in receipt of a prescription for RAASi. Methods Primary and secondary care data for this retrospective study were obtained from the UK Clinical Practice Research Datalink (CPRD) and linked Hospital Episode Statistics (HES). Eligible patients were identified using READ codes defining the relevant diagnosis, receipt of indication-specific medication, or, in the case of CKD, an estimated glomerular filtration rate (eGFR) ≤60 ml/min/1.73m2 within the study period (01 January 2008 to 30 June 2018) or in the five-year lookback period (2003–2007). The index date was defined as 01 January 2008 or first diagnosis of an eligible condition or RAASi prescription, whichever occurred latest. HK was defined as K+ ≥5.0 mmol/L; thresholds of ≥5.5 mmol/L and ≥6.0 mmol/L were explored as sensitivity analyses. Incidence rates of HK were calculated with 95% confidence intervals (CI). Results The total eligible population across all cohorts was 931,460 patients. RHTN was the most prevalent comorbidity (n=317,135; 34.0%) and dialysis the least prevalent (n=4,415; 0.5%). The majority of the eligible population were prescribed RAASi during follow-up (n=754,523; 81.0%). At a K+ threshold of ≥5.0 mmol/L, the dialysis cohort had the highest rate of HK (501.0 events per 1,000 patient-years), followed by HF (490.9), CKD (410.9), diabetes (355.0), RHTN (261.4) and the RAASi cohort (211.2) (Figure 1). This pattern was still observed at alternative threshold definitions of HK. Conclusion This large real-world study of UK patients demonstrates the burden of hyperkalaemia in high-risk patient populations from the UK. There is a need for effective prevention and treatment of HK, particularly in patients with CKD, dialysis or HF where increased incidence rates are observed which in turn will improve patient outcomes and healthcare resource usage. Figure 1. Rates of HK by condition Funding Acknowledgement Type of funding source: Private company. Main funding source(s): AstraZeneca


Author(s):  
Ioannis N. Anastopoulos ◽  
Chloe K. Herczeg ◽  
Kasey N. Davis ◽  
Atray C. Dixit

While the clinical approval process is able to filter out medications whose utility does not offset their adverse drug reaction profile in humans, it is not well suited to characterizing lower frequency issues and idiosyncratic multi-drug interactions that can happen in real world diverse patient populations. With a growing abundance of real-world evidence databases containing hundreds of thousands of patient records, it is now feasible to build machine learning models that incorporate individual patient information to provide personalized adverse event predictions. In this study, we build models that integrate patient specific demographic, clinical, and genetic features (when available) with drug structure to predict adverse drug reactions. We develop an extensible graph convolutional approach to be able to integrate molecular effects from the variable number of medications a typical patient may be taking. Our model outperforms standard machine learning methods at the tasks of predicting hospitalization and death in the UK Biobank dataset yielding an R2 of 0.37 and an AUC of 0.90, respectively. We believe our model has potential for evaluating new therapeutic compounds for individualized toxicities in real world diverse populations. It can also be used to prioritize medications when there are multiple options being considered for treatment.


2021 ◽  
Vol 21 ◽  
pp. S332-S333
Author(s):  
Fadi Nasr ◽  
Intissar Yehia ◽  
Reem El Khoury ◽  
Saada Diab ◽  
Ahmad Al Ghoche ◽  
...  

Author(s):  
Amanda McDonell ◽  
Urpo Kiiskinen ◽  
Danielle Zammit ◽  
Robert Kotchie ◽  
Per-Olof Thuresson ◽  
...  
Keyword(s):  
The Real ◽  

2021 ◽  
Vol 2021 ◽  
pp. 1-8
Author(s):  
Jae Hui Kim ◽  
Jong Woo Kim ◽  
Chul Gu Kim

Background. To evaluate the proportion of eyes that do not meet the eligibility criteria of clinical trials on neovascular age-related macular degeneration (AMD) and the reasons for exclusion. Methods. This retrospective, observational study included 512 eyes of 463 patients diagnosed with treatment-naïve neovascular AMD. The proportion of eyes that did not meet the eligibility criteria of the Vascular Endothelial Growth Factor Trap-Eye: Investigation of Efficacy and Safety in Wet AMD (VIEW) studies were evaluated. The two most common reasons for exclusion were also evaluated in each subtype of neovascular AMD (typical neovascular AMD, polypoidal choroidal vasculopathy (PCV), and type 3 neovascularization). Results. Among the 512 eyes, 229 (44.7%) did not meet the eligibility criteria. In all the included eyes, the most common reasons for exclusion were good or poor visual acuity (169 eyes, 33.0%), followed by the presence of subretinal hemorrhage (47 eyes, 9.5%). Moreover, good or poor visual acuity was the most common reason for exclusion in all three subtypes of neovascular AMD. The second most common reason was a fovea-involving scar or fibrosis in typical neovascular AMD, subretinal hemorrhage in PCV, and other vascular diseases affecting the retina in type 3 neovascularization. Conclusions. Among the included cases, 44.7% did not meet the eligibility criteria for VIEW study, suggesting that the conclusion derived from clinical trials may not directly reflect the real-world outcomes. Additionally, the reasons for ineligibility differed among the different subtypes of neovascular AMD.


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