scholarly journals The B-team: Equal but different?

2021 ◽  
Vol 8 (1) ◽  
pp. 133-135
Author(s):  
Luke Pembroke
Keyword(s):  
Gene Therapy ◽  
Treatment Options ◽  
Haemophilia A ◽  
Pharmacokinetic Modelling ◽  
Haemophilia B ◽  
Tailored Treatment ◽  
Dosing Frequency ◽  
Perception Bias ◽  
Respective Treatment ◽  
The Impact

Abstract As a person with haemophilia B, I have known there are differences between haemophilia A and haemophilia B and their respective treatment throughout my life – though I was shocked when I learnt about the impact inhibitors can have when it comes to bleeding. Despite being very rare, as well as difficult to manage, in a recent survey reported by Chaplin et al., many nurses had experience in managing haemophilia B inhibitors. Nurses in the survey also thought extended half-life (EHL) factor products would remain the optimal treatment for haemophilia B in 2025. Ongoing clinical trials for novel molecules like concuzimab and fitusiran signal the start of more treatment options for haemophilia B, and the development of gene therapy has focused on haemophilia B in the first instance. But the fact remains that the pharmaceutical industry has focused on developing treatments for the larger haemophilia A market. Could this have distorted perceptions around treatment? In a further ‘perception bias’ that impacts management, some nurses feel there are differences in bleeding phenotype between haemophilia A and B. Garner et al.'s paper discussing rIX-FX, suggests that treatment adherence is better in haemophilia B due to lower dosing frequency, making it an easier treatment option than for haemophilia A. The patient perception may be somewhat different. While dosing schedules in haemophilia B have been more consistent for longer, there has been less pharmacokinetic modelling in haemophilia B and, arguably, less opportunity for truly tailored treatment. Gene therapy has been shown to be more ‘successful’ for haemophilia B than haemophilia A, but emicizumab has raised questions about the need for gene therapy in haemophilia A. Having an ‘emi-equivalent’ for haemophilia B will raise the same questions and may give people haemophilia B and inhibitors an effective treatment that is as transformative as emicizumab has been in the haemophilia A population.

scholarly journals Haemophilia specialist nurses’ perceptions of haemophilia B

2021 ◽  
Vol 8 (1) ◽  
pp. 119-127
Author(s):  
Steve Chaplin ◽  
Maj Friberg Birkedal ◽  
Erica Crilly ◽  
Simon Fletcher ◽  
Sara Garcia ◽  
...  
Keyword(s):  
Clinical Practice ◽  
Online Survey ◽  
Optimal Treatment ◽  
Haemophilia A ◽  
Current Clinical Practice ◽  
Haemophilia B ◽  
Health Related ◽  
Specialist Nurses ◽  
Nurses Perceptions ◽  
The Impact

Abstract Introduction Some clinicians believe that haemophilia B is associated with less bleeding than haemophilia A, yet there appears to be little difference in health-related outcomes. Current clinical practice reduces the risk of bleeds, making differences difficult to measure. We surveyed specialist haemophilia nurses to discern their opinions about the impact of haemophilia B compared to haemophilia A. Methods Between July and September 2020, European and Canadian nurses were invited to complete an online survey (25 questions) about perceptions of management and treatment of haemophilia B. Results Fifty-nine nurses (46 European, 13 Canadian) completed the survey. Bleeding was reported as different in haemophilia B by 37% of respondents, and treatment as different by over half. Opinions and experience around using extended half-life (EHL) products varied. Self-reported confidence in using EHL products was rated at a mean of 7.1 (range 3–10) with 47% believing these would remain the optimal treatment in 2025. Conclusion Some nurses believe haemophilia A and B are managed differently. Variations in experience and levels of confidence in the use of EHL products, combined with a belief that these products will remain an optimal treatment for haemophilia B for the next five years, indicates a need for education to promote confidence and competence.

Surgery for Lung Cancer and the Consequences for the Swallow

2016 ◽  
Vol 1 (13) ◽  
pp. 162-168
Author(s):  
Pippa Hales ◽  
Corinne Mossey-Gaston
Keyword(s):  
Lung Cancer ◽  
Treatment Options ◽  
Vocal Cord Palsy ◽  
Subsequent Treatment ◽  
Physiological Reserve ◽  
Palliative Phase ◽  
And Function ◽  
The Impact ◽  
Swallow Function

Lung cancer is one of the most commonly diagnosed cancers across Northern America and Europe. Treatment options offered are dependent on the type of cancer, the location of the tumor, the staging, and the overall health of the person. When surgery for lung cancer is offered, difficulty swallowing is a potential complication that can have several influencing factors. Surgical interaction with the recurrent laryngeal nerve (RLN) can lead to unilateral vocal cord palsy, altering swallow function and safety. Understanding whether the RLN has been preserved, damaged, or sacrificed is integral to understanding the effect on the swallow and the subsequent treatment options available. There is also the risk of post-surgical reduction of physiological reserve, which can reduce the strength and function of the swallow in addition to any surgery specific complications. As lung cancer has a limited prognosis, the clinician must also factor in the palliative phase, as this can further increase the burden of an already compromised swallow. By understanding the surgery and the implications this may have for the swallow, there is the potential to reduce the impact of post-surgical complications and so improve quality of life (QOL) for people with lung cancer.

Management of Haemophilia in Sweden

1976 ◽  
Vol 35 (03) ◽  
pp. 510-521 ◽  
Author(s):  
Inga Marie Nilsson
Keyword(s):  
Factor Viii ◽  
Total Population ◽  
Age Groups ◽  
Factor Ix ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Haemophilia B ◽  
Human Fraction ◽  
Mild Haemophilia

SummaryThe incidence of living haemophiliacs in Sweden (total population 8.1 millions) is about 1:15,000 males and about 1:30,000 of the entire population. The number of haemophiliacs born in Sweden in 5-year periods between 1931-1975 (June) has remained almost unchanged. The total number of haemophilia families in Sweden is 284 (77% haemophilia A, 23% haemophilia B) with altogether 557 (436 with A and 121 with B) living haemophiliacs. Of the haemophilia A patients 40 % have severe, 18 % moderate, and 42 % mild, haemophilia. The distribution of the haemophilia B patients is about the same. Inhibitors have been demonstrated in 8% of the patients with severe haemophilia A and in 10% of those with severe haemophilia B.There are 2 main Haemophilia Centres (Stockholm, Malmo) to which haemophiliacs from the whole of Sweden are admitted for diagnosis, follow-up and treatment for severe bleedings, joint defects and surgery. Minor bleedings are treated at local hospitals in cooperation with the Haemophilia Centres. The concentrates available for treatment in haemophilia A are human fraction 1-0 (AHF-Kabi), cryoprecipitate, Antihaemophilic Factor (Hyland 4) and Kryobulin (Immuno, Wien). AHF-Kabi is the most commonly used preparation. The concentrates available for treatment in haemophilia B are Preconativ (Kabi) and Prothromplex (Immuno). Sufficient amounts of concentrates are available. In Sweden 3.2 million units of factor VIII and 1.0 million units of factor IX are given per year. Treatment is free of charge.Only 5 patients receive domiciliary treatment, but since 1958 we in Sweden have practised prophylactic treatment of boys (4–18 years old) with severe haemophilia A. At about 5-10 days interval they receive AHF in amounts sufficient to raise the AHF level to 40–50%. This regimen has reduced severe haemophilia to moderate. The joint score is identical with that found in moderate haemophilia in the same age groups. For treatment of patients with haemophilia A and haemophilia B complicated by inhibitors we have used a large dose of antigen (factor VIII or factor IX) combined with cyclophosphamide. In most cases this treatment produced satisfactory haemostasis for 5 to 30 days and prevented the secondary antibody rise.

The impact of a physician’s recommendation and gender on informed decision making: A randomized controlled study in a simulated decision situation

2020 ◽  
Author(s):  
Marie Eggeling ◽  
Anna Meinhardt ◽  
Ulrike Cress ◽  
Joachim Kimmerle ◽  
Martina Bientzle
Keyword(s):  
Decision Making ◽  
Cruciate Ligament ◽  
Treatment Options ◽  
General Information ◽  
Treatment Preference ◽  
Controlled Study ◽  
Treatment Preferences ◽  
Hypothetical Scenario ◽  
And Gender ◽  
The Impact

Objective: This study examined the influence of physicians’ recommendations and gender on the decision-making process in a preference-sensitive situation. Methods: N = 201 participants were put in a hypothetical scenario in which they suffered from a rupture of the anterior cruciate ligament (ACL). They received general information on two equally successful treatment options for this injury (surgery vs. physiotherapy) and answered questions regarding their treatment preference, certainty and satisfaction regarding their decision, and attitude toward the treatment options. Then participants watched a video that differed regarding physician’s recommendation (surgery vs. physiotherapy) and physician’s gender (female vs. male voice and picture). Afterward, they indicated again their treatment preference, certainty, satisfaction, and attitude, as well as the physician’s professional and social competence.Results: Participants changed their treatment preferences in the direction of the physician’s recommendation (P<.001). Decision certainty (P<.001) and satisfaction (P<.001) increased more strongly if the physician’s recommendation was congruent with the participant’s prior attitude than if the recommendation was contrary to the participant’s prior attitude. Finally, participants’ attitudes toward the recommended treatment became more positive (surgery recommendation: P<.001; physiotherapy recommendation: P<.001). We found no influence of the physician’s gender on participants’ decisions, attitudes, or competence assessments.Conclusion: This research indicates that physicians should be careful with recommendations when aiming for shared decisions, as they might influence patients even if the patients have been made aware that they should take their personal preferences into account. This could be particularly problematic if the recommendation is not in line with the patient’s preferences.

Artificial Intelligence as a Business Partner in Cardiovascular Precision Medicine: an Emerging Approach for Disease Detection and Treatment Optimization

2020 ◽  
Vol 28 ◽  
Author(s):  
Valeria Visco ◽  
Germano Junior Ferruzzi ◽  
Federico Nicastro ◽  
Nicola Virtuoso ◽  
Albino Carrizzo ◽  
...  
Keyword(s):  
Artificial Intelligence ◽  
Heart Failure ◽  
Atrial Fibrillation ◽  
Precision Medicine ◽  
Clinical Decision Making ◽  
Disease Detection ◽  
Physician Patient Relationship ◽  
Invasive Approach ◽  
Tailored Treatment ◽  
The Impact

Background: In the real world, medical practice is changing hand in hand with the development of new Artificial Intelligence (AI) systems and problems from different areas have been successfully solved using AI algorithms. Specifically, the use of AI techniques in setting up or building precision medicine is significant in terms of the accuracy of disease discovery and tailored treatment. Moreover, with the use of technology, clinical personnel can deliver a very much efficient healthcare service. Objective: This article reviews AI state-of-the-art in cardiovascular disease management, focusing on diagnostic and therapeutic improvements. Methods: To that end, we conducted a detailed PubMed search on AI application from distinct areas of cardiology: heart failure, arterial hypertension, atrial fibrillation, syncope and cardiovascular rehabilitation. Particularly, to assess the impact of these technologies in clinical decision-making, this research considers technical and medical aspects. Results: On one hand, some devices in heart failure, atrial fibrillation and cardiac rehabilitation represent an inexpensive, not invasive or not very invasive approach to long-term surveillance and management in these areas. On the other hand, the availability of large datasets (big data) is a useful tool to predict the development and outcome of many cardiovascular diseases. In summary, with this new guided therapy, the physician can supply prompt, individualised, and tailored treatment and the patients feel safe as they are continuously monitored, with a significant psychological effect. Conclusion: Soon, tailored patient care via telemonitoring can improve the clinical practice because AI-based systems support cardiologists in daily medical activities, improving disease detection and treatment. However, the physician-patient relationship remains a pivotal step.

scholarly journals Protein Tyrosine Kinases: Their Roles and Their Targeting in Leukemia

Cancers ◽  
2021 ◽  
Vol 13 (2) ◽  
pp. 184
Author(s):  
Kalpana K. Bhanumathy ◽  
Amrutha Balagopal ◽  
Frederick S. Vizeacoumar ◽  
Franco J. Vizeacoumar ◽  
Andrew Freywald ◽  
...  
Keyword(s):  
Growth Factor ◽  
Protein Kinase ◽  
Tyrosine Kinase ◽  
Protein Kinases ◽  
Tyrosine Kinases ◽  
Treatment Options ◽  
Growth Factor Receptor ◽  
Tyrosine Protein Kinase ◽  
Mesenchymal Epithelial Transition Factor ◽  
The Impact

Protein kinases constitute a large group of enzymes catalysing protein phosphorylation and controlling multiple signalling events. The human protein kinase superfamily consists of 518 members and represents a complicated system with intricate internal and external interactions. Protein kinases are classified into two main families based on the ability to phosphorylate either tyrosine or serine and threonine residues. Among the 90 tyrosine kinase genes, 58 are receptor types classified into 20 groups and 32 are of the nonreceptor types distributed into 10 groups. Tyrosine kinases execute their biological functions by controlling a variety of cellular responses, such as cell division, metabolism, migration, cell–cell and cell matrix adhesion, cell survival and apoptosis. Over the last 30 years, a major focus of research has been directed towards cancer-associated tyrosine kinases owing to their critical contributions to the development and aggressiveness of human malignancies through the pathological effects on cell behaviour. Leukaemia represents a heterogeneous group of haematological malignancies, characterised by an uncontrolled proliferation of undifferentiated hematopoietic cells or leukaemia blasts, mostly derived from bone marrow. They are usually classified as chronic or acute, depending on the rates of their progression, as well as myeloid or lymphoblastic, according to the type of blood cells involved. Overall, these malignancies are relatively common amongst both children and adults. In malignant haematopoiesis, multiple tyrosine kinases of both receptor and nonreceptor types, including AXL receptor tyrosine kinase (AXL), Discoidin domain receptor 1 (DDR1), Vascular endothelial growth factor receptor (VEGFR), Fibroblast growth factor receptor (FGFR), Mesenchymal–epithelial transition factor (MET), proto-oncogene c-Src (SRC), Spleen tyrosine kinase (SYK) and pro-oncogenic Abelson tyrosine-protein kinase 1 (ABL1) mutants, are implicated in the pathogenesis and drug resistance of practically all types of leukaemia. The role of ABL1 kinase mutants and their therapeutic inhibitors have been extensively analysed in scientific literature, and therefore, in this review, we provide insights into the impact and mechanism of action of other tyrosine kinases involved in the development and progression of human leukaemia and discuss the currently available and emerging treatment options based on targeting these molecules.

scholarly journals Role of the Pharmacist in Managing Treatment-Resistant Depression: A Focus on Ketamine

Pharmacy ◽  
2021 ◽  
Vol 9 (3) ◽  
pp. 118
Author(s):  
Linda Xing Yu Liu ◽  
Marina Golts ◽  
Virginia Fernandes
Keyword(s):  
Current Practice ◽  
Quality Care ◽  
Treatment Options ◽  
Critical Role ◽  
Transitions Of Care ◽  
Treatment Resistant Depression ◽  
Treatment Resistant ◽  
Resistant Depression ◽  
Multiple Treatments ◽  
The Impact

The impact of depression is well described in the literature, and it is most prominent in patients who have trialed multiple treatments. Treatment-resistant depression (TRD) is particularly debilitating, and it is associated with significant morbidity and mortality. Despite this, there seems to be therapeutic inertia in adopting novel therapies in current practice. Ketamine is an N-methyl-D-aspartate receptor antagonist and anesthetic agent which has recently been shown to be effective in the management of TRD when administered intravenously or intranasally. The treatments, however, are not easily accessible due to restrictions in prescribing and dispensing, high costs, and the slow uptake of evidence-based practice involving ketamine within the Canadian healthcare system. Given the limited treatment options for TRD, novel approaches should be considered and adopted into practice, and facilitated by a multi-disciplinary approach. Pharmacists play a critical role in ensuring access to quality care. This includes dissemination of evidence supporting pharmacological treatments and facilitating translation into current practice. Pharmacists are uniquely positioned to collaborate with prescribers and assess novel treatment options, such as ketamine, address modifiable barriers to treatment, and triage access to medications during transitions of care. Extending the reach of these novel psychiatric treatments in both tertiary and primary care settings creates an emerging role for pharmacists in the collaborative effort to better manage treatment-resistant depression.

scholarly journals Made to Measure: Patient-Tailored Treatment of Multiple Sclerosis Using Cell-Based Therapies

2021 ◽  
Vol 22 (14) ◽  
pp. 7536
Author(s):  
Inez Wens ◽  
Ibo Janssens ◽  
Judith Derdelinckx ◽  
Megha Meena ◽  
Barbara Willekens ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Autoimmune Diseases ◽  
Stromal Cells ◽  
Mononuclear Cells ◽  
Treatment Options ◽  
Cell Types ◽  
Peripheral Blood Mononuclear ◽  
Tailored Treatment ◽  
Key Issues ◽  
The Central Nervous System

Currently, there is still no cure for multiple sclerosis (MS), which is an autoimmune and neurodegenerative disease of the central nervous system. Treatment options predominantly consist of drugs that affect adaptive immunity and lead to a reduction of the inflammatory disease activity. A broad range of possible cell-based therapeutic options are being explored in the treatment of autoimmune diseases, including MS. This review aims to provide an overview of recent and future advances in the development of cell-based treatment options for the induction of tolerance in MS. Here, we will focus on haematopoietic stem cells, mesenchymal stromal cells, regulatory T cells and dendritic cells. We will also focus on less familiar cell types that are used in cell therapy, including B cells, natural killer cells and peripheral blood mononuclear cells. We will address key issues regarding the depicted therapies and highlight the major challenges that lie ahead to successfully reverse autoimmune diseases, such as MS, while minimising the side effects. Although cell-based therapies are well known and used in the treatment of several cancers, cell-based treatment options hold promise for the future treatment of autoimmune diseases in general, and MS in particular.

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