Evolocumab cost effective for atherosclerotic CVD, familial hypercholesterolaemia in KSA

2021 ◽  
Vol 888 (1) ◽  
pp. 10-10
Keyword(s):  
Familial Hypercholesterolaemia ◽  
Cost Effective

scholarly journals Familial Hypercholesterolaemia: An Updated Overall Management

2020 ◽  
Vol 5 (2) ◽  
pp. 19
Author(s):  
Noor Alicezah Mohd Kasim ◽  
Yung An Chua ◽  
Hapizah Mohd Nawawi
Keyword(s):  
High Risk ◽  
Familial Hypercholesterolaemia ◽  
Density Lipoprotein ◽  
Cost Effective ◽  
Response To Treatment ◽  
Lipid Lowering ◽  
Risk Category ◽  
Premature Coronary Artery Disease ◽  
Statin Intolerance ◽  
Cascade Screening

Familial hypercholesterolaemia (FH), the commonest and serious but potentially treatable form of inherited dyslipidaemias, is characterised by severely elevated plasma low-density lipoprotein-cholesterol (LDL-C) level, which subsequently leads to premature coronary artery disease (pCAD). Effectiveness of FH early detection and treatment is supported by the outcome of several international cohort studies. Optimal FH management relies on prescription of statins either alone or together with other lipid-lowering therapies (LLT). Intensive lifestyle intervention is required in parallel with LLT, which should be commenced at diagnosis in adults and childhood. Treatment with high intensity statin should be started as soon as possible. Combination with ezetimibe and/or bile acid sequestrants is indicated if target LDL-C is not achieved. For FH patients in the very-high risk category, if their LDL-C targets are not achieved, despite being on maximally tolerated statin dose and ezetimibe, proprotein convertase subtilisin/kexin type1 inhibitor (PCSK9i) is recommended. In statin intolerance, ezetimibe alone, or in combination with PCSK9i may be considered. Clinical evaluation of response to treatment and safety are recommended to be done about 4-6 weeks following initiation of treatment. Homozygous FH (HoFH) patients should be treated with maximally tolerated intensive LLT and, when available, with lipoprotein apheresis. This review highlights the overall management, and optimal treatment combinations in FH in adults and children, newer LLT including PCSK9i, microsomal transfer protein inhibitor, allele-specific oligonucleotide to ApoB100 and PCSK9 mRNA. Family cascade screening and/or screening of high-risk individuals, is the most cost-effective way of identifying FH cases and initiating early and adequate LLT.

scholarly journals P41 Pharmacokinetics-based estimation of evolocumab dosing for homozygous familial hypercholesterolemia (HoFH) in patients aged 6 to 12 years old

2020 ◽  
Vol 105 (9) ◽  
pp. e27.3-e28
Author(s):  
Chris Paget ◽  
Adam Sutherland
Keyword(s):  
Liver Transplantation ◽  
Therapeutic Drug Monitoring ◽  
Adverse Effects ◽  
Drug Monitoring ◽  
Familial Hypercholesterolaemia ◽  
Cost Effective ◽  
Therapeutic Drug ◽  
List Type ◽  
Lipid Apheresis ◽  
Homozygous Familial Hypercholesterolaemia

BackgroundHomozygous familial hypercholesterolaemia (HoFH) is a rare genetic disorder characterised by high plasma cholesterol levels and premature development of atherosclerotic cardiovascular disease.1Evolocumab is a high-cost monoclonal antibody to PCSK-9, an enzyme critical in cholesterol homeostasis. It is a subcutaneous injection commissioned for HoFH in children ≥12 years with persistently raised LDL-cholesterol (LDL-C) despite maximal tolerated lipid-lowering therapy.2 3 There is an unmet clinical need to allow patients ≥6 years meeting the same treatment threshold to access evolocumab and attenuate progression to invasive lipid apheresis or liver transplantation. However, as there are no published studies with PCSK-9 inhibitors in children <12 years, no established dosing regimen exists.Aims and objectivesPropose a safe, efficacious and cost-effective dose of evolocumab to be administered to eligible patients aged 6 to 12 years old with HoFH.Review all patients at 12 weeks to determine if 30% target LDL-C reduction is achieved thereby warranting treatment continuation in line with commissioning criteria.Review all patients at 12 months to establish if LDL-C reduction sustained.Assess all patients for incidence and nature of treatment-related adverse effects.MethodProposed evolocumab dosing determined using four criteria: potential dosing adjustments required based on population physiological and pharmacokinetic data, drug safety profile, practicalities of administration and cost implications.Clinic letters for all patients were reviewed 12 weeks and 12 months after treatment commenced.ResultsIn children <12 years dosing was proposed to start at 140 mg every 2 weeks, as this is the lowest administrable dose but is clinically equivalent to the 420 mg monthly dose (if information is extrapolated from heterozygous familial hypercholesterolemia studies4) and more cost-effective in terms of number of injections required. Furthermore, dose reduction in younger patients unlikely to be required as blood volume-dependent clearance of monoclonal antibodies and synthetic rates of PCSK-9 production do not significantly vary with age. Wide therapeutic index is implied as doses can be increased to 420 mg every 2 weeks and PCSK-9 has a limited physiological role with negligible ‘off target’ toxicity due to its inhibition.2 3 Therapeutic drug monitoring is not currently an option. Evolocumab was initiated using the proposed dose regimen in two eligible patients. Patient 1 had a 65% reduction in LDL-C (5.9 mmol/L to 1.9 mmol/L) at week 12, marginally subsiding at 12 months (2.7 mmol/L). No adverse effects reported and patient not yet progressed to lipid apheresis or liver transplantation. Patient 2 had only a 7% reduction in LDL-C (6.97 mmol/L to 6.48 mmol/L) at week 12 therefore evolocumab was stopped. No adverse effects were experienced. Lipid apheresis was continued throughout treatment.Conclusions and DiscussionExtrapolated dose of 140 mg every 2 weeks was safe and well-tolerated. Larger patient numbers are needed to further determine efficacy and safety, particularly due to promising significant and sustained LDL-C reduction in one patient. Licensed dose increases due to poor response in patients ≥12 years warrant investigation in younger population to allow potential treatment escalation for refractory patients. Therapeutic drug monitoring and antibody level testing are possible future research opportunities.ReferencesCuchel M, Bruckert E., Ginsberg HN, Homozygous familial hypercholesterolaemia: new insights and guidance for clinicians to improve detection and clinical management. A position paper from the Consensus Panel on Familial Hypercholesterolaemia of the European Atherosclerosis Society. Eur Heart J, 2014;35:2146–57.Amgen Ltd., Summary of Product Characteristics for Repatha Sureclick. 2016. Available from www.medicines.org.uk [accessed: 24/05/2018]. Last updated 08/03/2018.NHS England, NHSE statement on evolocumab for the treatment of homozygous familial hypercholesterolaemia (circular). September 2016.National Institute for Health and Care Excellence, Evolocumab for treating primary hypercholesterolaemia and mixed dyslipidaemia [TA394]. 2016. Available from www.nice.org.uk [accessed:22/06/2018].

The application of electron microscopy to diagnosis in gynecologic neoplasms and tumor-like conditions

1984 ◽  
Vol 42 ◽  
pp. 102-105
Author(s):  
Lawrence M. Roth
Keyword(s):  
Electron Microscopy ◽  
Reproductive Tract ◽  
Malignant Tumors ◽  
Frozen Section ◽  
Cost Effective ◽  
Female Reproductive Tract ◽  
Ultrastructural Examination ◽  
Specific Diagnosis ◽  
Potential Value ◽  
Gynecologic Neoplasms

The female reproductive tract may be the site of a wide variety of benign and malignant tumors, as well as non-neoplastic tumor-like conditions, most of which can be diagnosed by light microscopic examination including special stains and more recently immunoperoxidase techniques. Nevertheless there are situations where ultrastructural examination can contribute substantially to an accurate and specific diagnosis. It is my opinion that electron microscopy can be of greatest benefit and is most cost effective when applied in conjunction with other methodologies. Thus, I have developed an approach which has proved useful for me and may have benefit for others. In cases where it is deemed of potential value, glutaraldehyde-fixed material is obtained at the time of frozen section or otherwise at operation. Coordination with the gynecologic oncologist is required in the latter situation. This material is processed and blocked and is available if a future need arises.

The IBM-PC in electron microscopy

1996 ◽  
Vol 54 ◽  
pp. 612-613
Author(s):  
James F. Mancuso
Keyword(s):  
Head Start ◽  
Image Acquisition ◽  
Cost Effective ◽  
Financial Applications ◽  
Number Of Factors ◽  
Instrument Control ◽  
The Cost ◽  
Training And Support ◽  
Control Image ◽  
Ibm Pc

IBM PC compatible computers are widely used in microscopy for applications ranging from control to image acquisition and analysis. The choice of IBM-PC based systems over competing computer platforms can be based on technical merit alone or on a number of factors relating to economics, availability of peripherals, management dictum, or simple personal preference.IBM-PC got a strong “head start” by first dominating clerical, document processing and financial applications. The use of these computers spilled into the laboratory where the DOS based IBM-PC replaced mini-computers. Compared to minicomputer, the PC provided a more for cost-effective platform for applications in numerical analysis, engineering and design, instrument control, image acquisition and image processing. In addition, the sitewide use of a common PC platform could reduce the cost of training and support services relative to cases where many different computer platforms were used. This could be especially true for the microscopists who must use computers in both the laboratory and the office.

A Review of 21 iPad Applications for Augmentative and Alternative Communication Purposes

2012 ◽  
Vol 21 (2) ◽  
pp. 60-71 ◽  
Author(s):  
Ashley Alliano ◽  
Kimberly Herriger ◽  
Anthony D. Koutsoftas ◽  
Theresa E. Bartolotta
Keyword(s):  
Augmentative And Alternative Communication ◽  
Cost Effective ◽  
Alternative Form ◽  
Alternative Communication ◽  
Text To Speech ◽  
Reference Guide ◽  
Expressive Communication ◽  
Communication Needs ◽  
User Friendly ◽  
Ipad Applications

Abstract Using the iPad tablet for Augmentative and Alternative Communication (AAC) purposes can facilitate many communicative needs, is cost-effective, and is socially acceptable. Many individuals with communication difficulties can use iPad applications (apps) to augment communication, provide an alternative form of communication, or target receptive and expressive language goals. In this paper, we will review a collection of iPad apps that can be used to address a variety of receptive and expressive communication needs. Based on recommendations from Gosnell, Costello, and Shane (2011), we describe the features of 21 apps that can serve as a reference guide for speech-language pathologists. We systematically identified 21 apps that use symbols only, symbols and text-to-speech, and text-to-speech only. We provide descriptions of the purpose of each app, along with the following feature descriptions: speech settings, representation, display, feedback features, rate enhancement, access, motor competencies, and cost. In this review, we describe these apps and how individuals with complex communication needs can use them for a variety of communication purposes and to target a variety of treatment goals. We present information in a user-friendly table format that clinicians can use as a reference guide.

Teletinnitus Program at the VA Gulf Coast Veterans Health Care System

2014 ◽  
Vol 4 (1) ◽  
pp. 23-29
Author(s):  
Constance Hilory Tomberlin
Keyword(s):  
Health Care ◽  
Patient Satisfaction ◽  
Patient Care ◽  
Health Care System ◽  
Gulf Coast ◽  
New Technology ◽  
Cost Effective ◽  
Veterans Health ◽  
Potential Benefits ◽  
Care System

There are a multitude of reasons that a teletinnitus program can be beneficial, not only to the patients, but also within the hospital and audiology department. The ability to use technology for the purpose of tinnitus management allows for improved appointment access for all patients, especially those who live at a distance, has been shown to be more cost effective when the patients travel is otherwise monetarily compensated, and allows for multiple patient's to be seen in the same time slots, allowing for greater access to the clinic for the patients wishing to be seen in-house. There is also the patient's excitement in being part of a new technology-based program. The Gulf Coast Veterans Health Care System (GCVHCS) saw the potential benefits of incorporating a teletinnitus program and began implementation in 2013. There were a few hurdles to work through during the beginning organizational process and the initial execution of the program. Since the establishment of the Teletinnitus program, the GCVHCS has seen an enhancement in patient care, reduction in travel compensation, improvement in clinic utilization, clinic availability, the genuine excitement of the use of a new healthcare media amongst staff and patients, and overall patient satisfaction.

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