scholarly journals Myeloproliferative Neoplasms: Emerging Treatment Options for Myelofibrosis

2020 ◽  
Vol 18 (12.5) ◽  
pp. 1788-1791
Author(s):  
Aaron T. Gerds

Myelofibrosis (MF) is a symptom-forward disease, and its treatment focuses on alleviating those symptoms, as well as improving survival. An initial disease risk assessment is critical for deciding on a course of therapy (and a number of models can be used depending on the available patient information), and anemia can be considered a special case within the treatment algorithms for MF. JAK-STAT inhibition is currently the cornerstone of treatment for MF, but these inhibitors are not perfect. Future research will focus on the microenvironment in reversing fibrosis, immunotherapies, proliferative signaling pathways, epigenetic regulators, and stem cells.

2020 ◽  
Vol 14 ◽  
Author(s):  
Hui-Zhong Li ◽  
Xiang-Dong Liu ◽  
Xin Shi

Background: Modern medical researches show that population health is closely related to population behavior, environmental factors, social and economic conditions, aging and genetic susceptibility. Therefore, it is very important to study the relationship between these factors and population health. Objective: It reviews the latest research progress in population health management and life/disease risk assessment, analyzes the existing problems in relevant researches, and proposes some improvement plans to solve the problems. Methods: It introduces the general methods of human health management and life/disease risk assessment, analyzes the defects of these methods in practical application, and then draws lessons from the methods of industrial system equipment health management and residual life prediction, and puts forward some new ideas to solve the problems. Results: In the study of health assessment, although self-assessment of health is a common method of health variables, its subjectivity and inaccuracy is a problem that cannot be ignored. In life/disease risk assessment studies, medical effect and efficacy are mainly evaluation subjects, and little consideration is given to other factors, such as behaviour, environment, socio-economic status. In the construction of statistical graphs, great individual differences, internal symbiotic variables and external impact variables are seldom considered. Conclusion: This study helps to understand the basic ideas and methods in health management and life/disease risk assessment, and proposes some future research programs based on the author's knowledge background.


2019 ◽  
Vol 14 (2) ◽  
pp. 146-151 ◽  
Author(s):  
Junaid Khan ◽  
Amit Alexander ◽  
Mukta Agrawal ◽  
Ajazuddin ◽  
Sunil Kumar Dubey ◽  
...  

Diabetes and its complications are a significant health concern throughout the globe. There are physiological differences in the mechanism of type-I and type-II diabetes and the conventional drug therapy as well as insulin administration seem to be insufficient to address the problem at large successfully. Hypoglycemic swings, frequent dose adjustments and resistance to the drug are major problems associated with drug therapy. Cellular approaches through stem cell based therapeutic interventions offer a promising solution to the problem. The need for pancreatic transplants in case of Type- I diabetes can also be by-passed/reduced due to the formation of insulin producing β cells via stem cells. Embryonic Stem Cells (ESCs) and induced Pluripotent Stem Cells (iPSCs), successfully used for generating insulin producing β cells. Although many experiments have shown promising results with stem cells in vitro, their clinical testing still needs more exploration. The review attempts to bring into light the clinical studies favoring the transplantation of stem cells in diabetic patients with an objective of improving insulin secretion and improving degeneration of different tissues in response to diabetes. It also focuses on the problems associated with successful implementation of the technique and possible directions for future research.


2021 ◽  
pp. 238008442110144
Author(s):  
N.R. Paul ◽  
S.R. Baker ◽  
B.J. Gibson

Introduction: Patients’ decisions to undergo major surgery such as orthognathic treatment are not just about how the decision is made but what influences the decision. Objectives: The primary objective of the study was to identify the key processes involved in patients’ experience of decision making for orthognathic treatment. Methods: This study reports some of the findings of a larger grounded theory study. Data were collected through face-to-face interviews of patients who were seen for orthognathic treatment at a teaching hospital in the United Kingdom. Twenty-two participants were recruited (age range 18–66 y), of whom 12 (male = 2, female = 10) were 6 to 8 wk postsurgery, 6 (male = 2, female = 4) were in the decision-making stage, and 4 (male = 0, female = 4) were 1 to 2 y postsurgery. Additional data were also collected from online blogs and forums on jaw surgery. The data analysis stages of grounded theory methodology were undertaken, including open and selective coding. Results: The study identified the central role of dental care professionals (DCPs) in several underlying processes associated with decision making, including legitimating, mediating, scheduling, projecting, and supporting patients’ decisions. Six categories were related to key aspects of decision making. These were awareness about their underlying dentofacial problems and treatment options available, the information available about the treatment, the temporality of when surgery would be undertaken, the motivations and expectation of patients, social support, and fear of the surgery, hospitalization, and potentially disliking their new face. Conclusion: The decision-making process for orthognathic treatment is complex, multifactorial, and heavily influenced by the role of DCPs in patient care. Understanding the magnitude of this role will enable DCPs to more clearly participate in improving patients’ decision-making process. The findings of this study can inform future quantitative studies. Knowledge Transfer Statement: The results of this study can be used both for informing clinical practice around enabling decision making for orthognathic treatment and also for designing future research. The findings can better inform clinicians about the importance of their role in the patients’ decision-making process for orthognathic treatment and the means to improve the patient experience. It is suggested that further research could be conducted to measure some of the key constructs identified within our grounded theory and assess how these change during the treatment process.


1990 ◽  
Vol 16 (3) ◽  
pp. 399-427
Author(s):  
Pamela D. Harvey

Environmental pollution threatens public health. The search for solutions has advanced the frontiers of science and law. Efforts to protect the environment and public health begin with describing potential adverse consequences of human activities and characterizing the predicted risk. The National Environmental Policy Act requires the preparation of environmental impact statements to describe the effects of proposed federal projects and provide information for agency decisionmakers and the public.Risks to public health are particularly difficult to quantify because of uncertainty about the relation between exposure to environmental contamination and disease. Risk assessment is the current scientific tool to present estimates of risk. The methodology has created controversy, however, when underlying assumptions and uncertainties are not clearly presented. Critics caution that the methodology is vulnerable to bias. This Note evaluates the use of risk assessment in the environmental impact statement process and offers recommendations to ensure informed decisions.


2020 ◽  
Vol 11 ◽  
Author(s):  
Courtney B. Johnson ◽  
Jizhou Zhang ◽  
Daniel Lucas

Hematopoiesis in the bone marrow (BM) is the primary source of immune cells. Hematopoiesis is regulated by a diverse cellular microenvironment that supports stepwise differentiation of multipotent stem cells and progenitors into mature blood cells. Blood cell production is not static and the bone marrow has evolved to sense and respond to infection by rapidly generating immune cells that are quickly released into the circulation to replenish those that are consumed in the periphery. Unfortunately, infection also has deleterious effects injuring hematopoietic stem cells (HSC), inefficient hematopoiesis, and remodeling and destruction of the microenvironment. Despite its central role in immunity, the role of the microenvironment in the response to infection has not been systematically investigated. Here we summarize the key experimental evidence demonstrating a critical role of the bone marrow microenvironment in orchestrating the bone marrow response to infection and discuss areas of future research.


Parasitology ◽  
1999 ◽  
Vol 117 (7) ◽  
pp. 205-212 ◽  
Author(s):  
C. J. GIBSON ◽  
C. N. HAAS ◽  
J. B. ROSE

Throughout the past decade much research has been directed towards identifying the occurrence, epidemiology, and risks associated with waterborne protozoa. While outbreaks are continually documented, sporadic cases of disease associated with exposure to low levels of waterborne protozoa are of increasing concern. Current methodologies may not be sensitive enough to define these low levels of disease. However, risk assessment methods may be utilised to address these low level contamination events. The purpose of this article is to provide an introduction to microbial risk assessment for waterborne protozoa. Risk assessment is a useful tool for evaluating relative risks and can be used for development of policies to decrease risks. Numerous studies have been published on risk assessment methods for pathogenic protozoa including Cryptosporidium and Giardia. One common notion prevails: microbial risk assessment presents interesting complications to the traditional chemical risk assessment paradigm. Single microbial exposures (non-threshold) are capable of causing symptomatic illness unlike traditional chemical exposures, which require a threshold to be reached. Due to the lack of efficient recovery and detection methods for protozoa, we may be underestimating the occurrence, concentration and distribution of these pathogenic micro-organisms. To better utilize the tool of microbial risk assessment for risk management practices, future research should focus in the area of exposure assessment.


2012 ◽  
Vol 30 (24) ◽  
pp. 2995-3001 ◽  
Author(s):  
Malin Hultcrantz ◽  
Sigurdur Yngvi Kristinsson ◽  
Therese M.-L. Andersson ◽  
Ola Landgren ◽  
Sandra Eloranta ◽  
...  

PurposeReported survival in patients with myeloproliferative neoplasms (MPNs) shows great variation. Patients with primary myelofibrosis (PMF) have substantially reduced life expectancy, whereas patients with polycythemia vera (PV) and essential thrombocythemia (ET) have moderately reduced survival in most, but not all, studies. We conducted a large population-based study to establish patterns of survival in more than 9,000 patients with MPNs.Patients and MethodsWe identified 9,384 patients with MPNs (from the Swedish Cancer Register) diagnosed from 1973 to 2008 (divided into four calendar periods) with follow-up to 2009. Relative survival ratios (RSRs) and excess mortality rate ratios were computed as measures of survival.ResultsPatient survival was considerably lower in all MPN subtypes compared with expected survival in the general population, reflected in 10-year RSRs of 0.64 (95% CI, 0.62 to 0.67) in patients with PV, 0.68 (95% CI, 0.64 to 0.71) in those with ET, and 0.21 (95% CI, 0.18 to 0.25) in those with PMF. Excess mortality was observed in patients with any MPN subtype during all four calendar periods (P < .001). Survival improved significantly over time (P < .001); however, the improvement was less pronounced after the year 2000 and was confined to patients with PV and ET.ConclusionWe found patients with any MPN subtype to have significantly reduced life expectancy compared with the general population. The improvement over time is most likely explained by better overall clinical management of patients with MPN. The decreased life expectancy even in the most recent calendar period emphasizes the need for new treatment options for these patients.


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