Social and Psychological Factors Associated with Health Care Transition for Young Adults Living with Sickle Cell Disease

Introduction: Due to advances in disease management, mortality rates in children with sickle cell disease (SCD) have decreased. However, mortality rates for young adults (YA) increased, and understanding of social and psychological factors is critical. The aim of this study was to explore factors associated with health care transition experiences for YA with SCD. Method: This was a qualitative descriptive study. A 45-minute semistructured interview was conducted with 13 YA (M = 21.5 years, SD = 1.73). Results: Results suggest that social and psychological factors and self-management experiences influence health care transition. Eight themes emerged: “need for accessible support”; “early assistance with goal setting”; “incongruence among expectations, experiences, and preparation”; “spiritual distress”; “stigma”; “need for collaboration”; “appreciation for caring providers”; and “feeling isolated.” Discussion: Consideration of cultural contexts will guide nurses in supporting health care transition. Designing culturally relevant interventions that address unique needs for YA living with SCD is warranted

Blood Cell Membrane Omega-3 (n-3) Fatty Acid Abnormality and Supplementation in Patients with Sickle Cell Anemia

Sickle cell disease (SCD) is a group of autosomal recessive genetic blood disorders caused by a mutation in the sixth codon of the β goblin gene that results in abnormal hemoglobin (Hemoglobin S, HbS) (Knight-Perry et al., 2009; Rees et al., 2010; Serjeant and Serjeant, 2001). The principal phenotypes are homozygous sickle cell (HbSS) disease, sickle cell-hemoglobin C , sickle cell-β0 thalassemia, sickle cell-β1 thalassemia, HbSOArab and HbSDPunjab and HbSLepore Boston SCD (Nagel et al., 2003; Serjeant and Serjeant, 2001). Deoxygenated HbS forms insoluble rigid polymers (sickle) under hypoxic conditions and reverts back to normal on re-oxygenation. However, with repeated cycles of sickling and unsickling, erythrocytes become irreversibly sickled and lose their biconcave shape and fluidity. The primary pathological process in SCD, namely vasoocclusive crisis is a recurrent occlusion of blood vessels which causes ischemia, severe pain episodes (painful crisis), and damage to the brain, eyes, lungs, spleen, liver, and other vital organs (Ballas et al., 2010; Serjeant and Serjeant, 2001). Despite the apparent genetic simplicity, patients with SCD display a remarkable diversity in clinical manifestations and disease severity (Chui and Dover, 2001; Fertrin and Costa, 2010). The Cooperative Study of Sickle Cell Disease (Platt et al., 1991) found that 39% of 3578 patients with SCD did not have painful episodes, whereas 1% had more than six per year. It appears that type and severity of the complication of the disease are modulated by genetic, environmental, and other factors (Sebastiani et al., 2005; Steinberg, 2005)

Selecting and implementing overview methods: implications from five exemplar overviews

This is the final version of the article. Available from BioMed Central via the DOI in this record.Background Overviews of systematic reviews are an increasingly popular method of evidence synthesis; there is a lack of clear guidance for completing overviews and a number of methodological challenges. At the UK Cochrane Symposium 2016, methodological challenges of five overviews were explored. Using data from these five overviews, practical implications to support methodological decision making of authors writing protocols for future overviews are proposed. Methods Methods, and their justification, from the five exemplar overviews were tabulated and compared with areas of debate identified within current literature. Key methodological challenges and implications for development of overview protocols were generated and synthesised into a list, discussed and refined until there was consensus. Results Methodological features of three Cochrane overviews, one overview of diagnostic test accuracy and one mixed methods overview have been summarised. Methods of selection of reviews and data extraction were similar. Either the AMSTAR or ROBIS tool was used to assess quality of included reviews. The GRADE approach was most commonly used to assess quality of evidence within the reviews. Eight key methodological challenges were identified from the exemplar overviews. There was good agreement between our findings and emerging areas of debate within a recent published synthesis. Implications for development of protocols for future overviews were identified. Conclusions Overviews are a relatively new methodological innovation, and there are currently substantial variations in the methodological approaches used within different overviews. There are considerable methodological challenges for which optimal solutions are not necessarily yet known. Lessons learnt from five exemplar overviews highlight a number of methodological decisions which may be beneficial to consider during the development of an overview protocol.The overview conducted by Pollock [19] was supported by a project grant from the Chief Scientist Office of the Scottish Government. The overview conducted by McClurg [21] was supported by a project grant by the Physiotherapy Research Foundation. The overview by Hunt [22] was supported as part of doctoral programme funding by the National Institute for Health Research (NIHR) Collaboration for Leadership in Applied Health Research and Care South West Peninsula (PenCLAHRC). The overview conducted by Estcourt [20] was supported by an NIHR Cochrane Programme Grant for the Safe and Appropriate Use of Blood Components. The overview conducted by Brunton [23] was commissioned by the Department of Health as part of an ongoing programme of work on health policy research synthesis. Alex Pollock is employed by the Nursing, Midwifery and Allied Health Professions (NMAHP) Research Unit, which is supported by the Chief Scientist Office of the Scottish Government. Pauline Campbell is supported by the Chief Nurses Office of the Scottish Government

Pulmonary hypertension in adolescents with sickle cell disease

Sickle cell disease consists of a group of disorders that have a similar mutation in at least one of the beta-globin chains of hemoglobin. This results in a change of the hemoglobin to sickle shaped cells when in the deoxygenated state. It is these sickled cells that lead to the symptoms and complications characteristic of sickle cell disease, including vaso-occlusion. The recurrence of sickling and polymerization in the blood vessels throughout the body ultimately results in tissue and organ damage that increases the mortality of patients. Pulmonary hypertension is the increase in blood pressure in the pulmonary vasculature causing less blood to reach the lungs. The right side of the heart has to pump harder to compensate for this, which can ultimately lead to right heart failure. With vaso-occlusion being the major complication in sickle cell disease and affecting many tissues and organs like the lungs, pulmonary hypertension has become a major risk factor in these patients, especially in children. Most research has been with adults so the seriousness of this condition is still being discovered in children. This review will cover the important aspects of both sickle cell disease and pulmonary hypertension then discuss how pulmonary hypertension has become a significant risk factor for death in those with sickle cell disease. From there the importance of this finding in children will be discussed and a plan for future endeavors will be proposed

The Evolving Pharmacotherapeutic Landscape for the Treatment of Sickle Cell Disease.

Sickle cell disease (SCD) is an extremely heterogeneous disease that has been associated with global morbidity and early mortality. More effective and inexpensive therapies are needed. During the last five years, the landscape of the pharmacotherapy of SCD has changed dramatically. Currently, 54 drugs have been used or under consideration to use for the treatment of SCD. These fall into 3 categories: the first category includes the four drugs (Hydroxyurea, L-Glutamine, Crizanlizumab tmca and Voxelotor) that have been approved by the United States Food and Drug Administration (FDA) based on successful clinical trials. The second category includes 22 drugs that failed, discontinued or terminated for now and the third category includes 28 drugs that are actively being considered for the treatment of SCD. Crizanlizumab and Voxelotor are included in the first and third categories because they have been used in more than one trial. New therapies targeting multiple pathways in the complex pathophysiology of SCD have been achieved or are under continued investigation. The emerging trend seems to be the use of multimodal drugs (i.e. drugs that have different mechanisms of action) to treat SCD similar to the use of multiple chemotherapeutic agents to treat cancer

Evidence for over-dispersion in the distribution of clinical malaria episodes in children.

BACKGROUND: It may be assumed that patterns of clinical malaria in children of similar age under the same level of exposure would follow a Poisson distribution with no over-dispersion. Longitudinal studies that have been conducted over many years suggest that some children may experience more episodes of clinical malaria than would be expected. The aim of this study was to identify this group of children and investigate possible causes for this increased susceptibility. METHODOLOGY AND PRINCIPAL FINDINGS: Using Poisson regression, we chose a group of children whom we designated as 'more susceptible' to malaria from 373 children under 10 years of age who were followed up for between 3 to 5 years from 1998-2003. About 21% of the children were categorized as 'more susceptible' and although they contributed only 23% of the person-time of follow-up, they experienced 55% of total clinical malaria episodes. Children that were parasite negative at all cross-sectional survey were less likely to belong to this group [AOR = 0.09, (95% CI: 0.14-0.61), p = 0.001]. CONCLUSIONS AND SIGNIFICANCE: The pattern of clinical malaria episodes follows a negative binomial distribution. Use of lack of a clinical malaria episode in a certain time period as endpoints for intervention or immunological studies may not adequately distinguish groups who are more or less immune. It may be useful in such studies, in addition to the usual endpoint of the time to first episode, to include end points which take into account the total number of clinical episodes experienced per child

Public Health Concern and Initiatives on the Priority Action Towards Non-Communicable Diseases in Tanzania

Tanzania is already facing challenges caused by existing burden of communicable diseases, and the growing trend of non-communicable diseases (NCDs), which raises a lot of concerns and challenges. The objective of this review is to provide broad insight of the “silent epidemic” of NCDs, existing policies, strategies and interventions, and recommendations on prioritized actions. A review of existing literature including published articles, technical reports, and proceedings from national and international NCDs meetings was carried out. The burden, existing interventions, socio-economic impact, lessons learnt, and potential for expanding cost effective interventions in Tanzania were explored. Challenges to catch up with global\ud momentum on NCD agenda were identified and discussed. The review has indicated that the burden of NCDs and its underlying risk factors in Tanzania is alarming, and affects people of all socio-economic status. The\ud costs of health care for managing NCDs are high, and thus impoverishing the already poor people. The country\ud leadership has a high political commitment; there are policies and strategies, which need to be implemented to\ud address the growing NCD burden. In conclusion, NCDs in Tanzania are a silent rising health burden and has\ud enormous impact on an individual and country’s social-economical status. From the experience of other countries, interventions for NCDs are affordable, feasible and some are income generating. Multi-sectoral approach, involving national and international partners has a unique role in intensifying action on NCDs.\ud Tanzania should strategize on implementation research on how to adapt the interventions and apply multisectoral\ud approach to control and prevent NCDs in the country.\u

Drawing Students into Learning

In addition to teaching the science of medicine, Professor Joanna Rowe shares the importance of positive energy and creates cartoons to help nursing students understand complex subjects

Towards an interoperable healthcare information infrastructure - working from the bottom up

Historically, the healthcare system has not made effective use of information technology. On the face of things, it would seem to provide a natural and richly varied domain in which to target benefit from IT solutions. But history shows that it is one of the most difficult domains in which to bring them to fruition. This paper provides an overview of the changing context and information requirements of healthcare that help to explain these characteristics.First and foremost, the disciplines and professions that healthcare encompasses have immense complexity and diversity to deal with, in structuring knowledge about what medicine and healthcare are, how they function, and what differentiates good practice and good performance. The need to maintain macro-economic stability of the health service, faced with this and many other uncertainties, means that management bottom lines predominate over choices and decisions that have to be made within everyday individual patient services. Individual practice and care, the bedrock of healthcare, is, for this and other reasons, more and more subject to professional and managerial control and regulation.One characteristic of organisations shown to be good at making effective use of IT is their capacity to devolve decisions within the organisation to where they can be best made, for the purpose of meeting their customers' needs. IT should, in this context, contribute as an enabler and not as an enforcer of good information services. The information infrastructure must work effectively, both top down and bottom up, to accommodate these countervailing pressures. This issue is explored in the context of infrastructure to support electronic health records.Because of the diverse and changing requirements of the huge healthcare sector, and the need to sustain health records over many decades, standardised systems must concentrate on doing the easier things well and as simply as possible, while accommodating immense diversity of requirements and practice. The manner in which the healthcare information infrastructure can be formulated and implemented to meet useful practical goals is explored, in the context of two case studies of research in CHIME at UCL and their user communities.Healthcare has severe problems both as a provider of information and as a purchaser of information systems. This has an impact on both its customer and its supplier relationships. Healthcare needs to become a better purchaser, more aware and realistic about what technology can and cannot do and where research is needed. Industry needs a greater awareness of the complexity of the healthcare domain, and the subtle ways in which information is part of the basic contract between healthcare professionals and patients, and the trust and understanding that must exist between them. It is an ideal domain for deeper collaboration between academic institutions and industry

The road to commercialization in Africa: lessons from developing the sickle-cell drug Niprisan

<p>Abstract</p> <p>Background</p> <p>Developing novel drugs from traditional medicinal knowledge can serve as a means to improve public health. Yet countries in sub-Saharan Africa face barriers in translating traditional medicinal knowledge into commercially viable health products. Barriers in moving along the road towards making a new drug available include insufficient manufacturing capacity; knowledge sharing between scientists and medical healers; regulatory hurdles; quality control issues; pricing and distribution; and lack of financing. The case study method was used to illustrate efforts to overcome these barriers during the development in Nigeria of Niprisan – a novel drug for the treatment of sickle cell anemia, a chronic blood disorder with few effective therapies.</p> <p>Discussion</p> <p>Building on the knowledge of a traditional medicine practitioner, Nigeria’s National Institute for Pharmaceutical Research and Development (NIPRD) developed the traditional herbal medicine Niprisan. The commercialization of Niprisan reached a number of commercial milestones, including regulatory approval in Nigeria; securing US-based commercial partner XeChem; demonstrating clinical efficacy and safety; being awarded orphan drug status by the US Food and Drug Administration; and striking important relationships with domestic and international groups. Despite these successes, however, XeChem did not achieve mainstream success for Niprisan in Nigeria or in the United States. A number of reasons, including inconsistent funding and manufacturing and management challenges, have been put forth to explain Niprisan’s commercial demise. As of this writing, NIPRD is considering options for another commercial partner to take the drug forward.</p> <p>Summary</p> <p>Evidence from the Niprisan experience suggests that establishing benefit-sharing agreements, fostering partnerships with established research institutions, improving standardization and quality control, ensuring financial and managerial due diligence, and recruiting entrepreneurial leaders capable of holding dual scientific and business responsibilities should be incorporated into future drug development initiatives based on traditional medicines. Country-level supporting policies and conditions are also important. With more experience and support, and an improved environment for innovation, developing new drugs from traditional medicines may be an attractive approach to addressing diseases in sub-Saharan Africa and other regions.</p