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1.
Health Policy Plan ; 39(5): 486-498, 2024 May 15.
Article in English | MEDLINE | ID: mdl-38544412

ABSTRACT

This article traces the influence of network power on mental health policy in Liberia, a low-income, post-conflict West African country. Based on key informant interviews, focus group discussions and document analysis, the work uses an inductive approach to uncover how a network of civil society groups, government officials, diasporans and international NGOs shaped the passage, implementation and revision of the country's 2009 and 2016 mental health policies. With relations rooted in ties of information, expertise, resources, commitment and personal connections, the network coalesced around a key agent, the Carter Center, which connected members and guided initiatives. Network power was evident when these actors channelled expertise, shared narratives of post-war trauma and mental health as a human right, and financial resources to influence policy. Feedback loops appeared as policy implementation created new associations of mental health clinicians and service users, research entities and training institutes. These beneficiaries offered the network information from lived experiences, while also pressing their own interests in subsequent policy revisions. As the network expanded over time, some network members gained greater autonomy from the key agent. Network power outcomes included the creation of government mental health institutions, workforce development, increased public awareness, civil society mobilization and a line for mental health in the government budget, though concerns about network overstretch and key agent commitment emerged over time. The Liberian case illustrates how networks need not be inimical to development, and how network power may facilitate action on stigmatized, unpopular issues in contexts with low state capacity. A focus on network power in health shows how power can operate not only through discrete resources such as funding but also through the totality of assets that network linkages make possible.


Subject(s)
Health Policy , Mental Health Services , Liberia , Humans , Mental Health Services/organization & administration , Mental Health , Focus Groups , Organizations
2.
Clin J Oncol Nurs ; 27(2): 155-163, 2023 03 16.
Article in English | MEDLINE | ID: mdl-37677836

ABSTRACT

BACKGROUND: Chemotherapy-induced nausea and vomiting (CINV) places a burden on patients receiving blood and marrow transplantation. The effects of complementary aromatherapy on CINV are documented, but more information is needed to implement practice changes. OBJECTIVES: The purpose of this pilot study was to evaluate whether pure peppermint or ginger essential oil reduced the severity of CINV. METHODS: A controlled randomized pilot study was conducted using peppermint oil, ginger oil, and control (canola oil) groups. Nurses applied the assigned oil every four hours. Outcome and patient feedback data were collected. FINDINGS: Twenty patients experienced a level 2 or greater nausea event. Patients in the peppermint oil group were the least likely to experience a nausea event, followed by the ginger oil and control groups. Five patients experienced level 2 vomiting; these events did not differ between groups.


Subject(s)
Antineoplastic Agents , Oils, Volatile , Humans , Pilot Projects , Oils, Volatile/therapeutic use , Bone Marrow , Nausea/chemically induced , Nausea/prevention & control , Vomiting/chemically induced , Vomiting/prevention & control
3.
J Public Health Dent ; 83(3): 325-328, 2023 07.
Article in English | MEDLINE | ID: mdl-37584232

ABSTRACT

BACKGROUND: Efforts to progress oral healthcare reform can be challenging with competing interests of governments and service providers to achieve the intended outcomes. The value-based health care approach has been adopted in many areas of healthcare but has had limited applications to oral healthcare systems. Dental Health Services Victoria, an Australian state government funded entity, commenced its journey to value-based health care in 2016, to shift away from traditional dental service models that reward activity and volume towards a stronger emphasis on value and outcomes. AIMS: To maintain the value-based health care agenda focus, Dental Health Services Victoria developed three key principles, which can be adopted by other organisations engaged in reforming oral healthcare, to improve the oral health for the population it serves. MATERIALS & METHODS: In 2018, Dental Health Services Victoria developed a value-based health care framework, which has informed strategic organisation priorities for action. In 2023, the following three key principles are identified as being essential to support the operationalisation and development of effective models of oral healthcare: Principle 1 - Care is co-designed with the person or population Principle 2 - Prevention and early intervention are prioritised. Principle 3 - Consistent measurement of health outcomes and costs are embedded. DISCUSSION: The exploration of the three key principles is an important communication tool to translate value-based health care into practice with key stakeholders. Further work is required to socialise them to within dental teams. CONCLUSION: Organisations looking to commence the value-based health care agenda can apply Dental Health Services Victoria's three key principles as a first step.


Subject(s)
Dental Health Services , Oral Health , Humans , Value-Based Health Care , Australia , Delivery of Health Care
4.
Health Promot Int ; 38(4)2023 Aug 01.
Article in English | MEDLINE | ID: mdl-37555701

ABSTRACT

Dental caries, a non-communicable disease, is one of the most prevalent diseases globally and share common modifiable risk factors with obesity such as excess sugar intake. However, prioritization by governments to improve population oral health has been limited and is typically excluded from the discourse of public health policy development. Therefore, interventions that target dental caries can have other co-benefits including obesity prevention. In Victoria, Australia, local government authorities have a regulatory requirement to develop their Municipal Health and Wellbeing Plans. The aim of this paper is to identify whether prioritization for oral health by local government authorities in Victoria has changed through the subsequent renewal of the Victorian Public Health and Wellbeing Plans 2011-2015 and 2019-2023. Three desktop audits for all publicly available Municipal Health and Wellbeing Plans by local government authorities in Victoria were conducted between 2014 and 2022. Key terms related to oral health was searched within these policy documents and categorized into six indicators: (i) included oral health as a priority, (ii) linked healthy eating and oral health, (iii) supported the Achievement Program, (iv) included the Smiles 4 Miles program, (v) advocated for fluoridated drinking water, and (vi) included other strategies related to oral health. Overall, there was statistically significant reduction in five of the six indicators, with the exception for prioritization of other strategies related to oral health such as targeting excess sugar intake and smoking. A multi-sectoral approach, that includes oral health would be advantageous to address the growing burden of non-communicable diseases.


Subject(s)
Dental Caries , Oral Health , Humans , Dental Caries/epidemiology , Dental Caries/prevention & control , Victoria , Health Policy , Public Policy , Obesity/prevention & control , Local Government , Sugars
5.
J Clin Apher ; 38(6): 654-663, 2023 Dec.
Article in English | MEDLINE | ID: mdl-37522733

ABSTRACT

Symptoms of hypocalcemia are reported in up to 50% of patients undergoing leukapheresis procedures. There is no set standard of practice for administering calcium supplementation in the prevention or treatment of hypocalcemia symptoms. The goal of this descriptive, retrospective study was to determine the prevalence of baseline hypocalcemia and symptomatic hypocalcemia during leukapheresis with acid citrate dextrose solution A and to identify patient characteristics associated with symptomatic hypocalcemia. Three percent of patients were found to have hypocalcemia before leukapheresis with 35% experiencing hypocalcemia symptoms during leukapheresis. Older age, higher albumin levels, and longer procedure time were associated with increased risk of hypocalcemia symptoms.


Subject(s)
Hypocalcemia , Leukapheresis , Humans , Leukapheresis/methods , Hypocalcemia/etiology , Hypocalcemia/prevention & control , Retrospective Studies , Prevalence , Calcium
6.
Ther Adv Rare Dis ; 4: 26330040231164425, 2023.
Article in English | MEDLINE | ID: mdl-37197559

ABSTRACT

Background: Patient advocacy groups (PAGs) serve a vital role for rare disease patients and families by providing educational resources, support, and a sense of community. Motivated by patient need, PAGs are increasingly at the forefront of policy, research, and drug development for their disease of interest. Objectives: The study explored the current landscape of PAGs in order to guide new and existing PAGs on available resources and challenges to research engagement. We aim to inform industry, advocates, and healthcare personnel about PAG achievements and ways they are increasingly involved in research. Design: We chose PAGs from the Rare Diseases Clinical Research Network (RDCRN) Coalition for Patient Advocacy Groups (CPAG) listserv and the National Organization for Rare Disorders (NORD) 'Find a patient organization'. Methods: We surveyed eligible PAG leaders about the demographics, goals, and research activities of their organization. For analysis, PAGs were bucketed by size, age, prevalence of disease, and budget. Data were de-identified for cross-tabulation and multinomial logistic regression analysis with R. Results: Research engagement was an extremely important goal for most PAGs (81%), though ultra-rare disease and high-budget PAGs were most likely to cite it as the top priority. In total, 79% reported research engagement in some capacity, including registries, translational research, and clinical trials. 'Ultra-rare' PAGs were less likely than 'rare' PAGs to have an ongoing clinical trial. Conclusion: While PAGs of varying sizes, budgets, and maturity levels reported an interest in research, limited funding and lack of disease awareness continue to create barriers to achieving their goals. While support tools exist to make research more accessible, often their utility depends on the funding, sustainability, maturity of the PAG itself, and the level of investment of collaborators. Despite the availability of current support systems, there are challenges related to both the start-up and sustainability of patient-centric research efforts.

8.
Infect Immun ; 89(10): e0030121, 2021 09 16.
Article in English | MEDLINE | ID: mdl-34097506

ABSTRACT

Recent studies suggest an anti-inflammatory protective role for class B scavenger receptor BI (SR-BI) in endotoxin-induced inflammation and sepsis. Other data, including ours, provide evidence for an alternative role of SR-BI, facilitating bacterial and endotoxin uptake and contributing to inflammation and bacterial infection. Enhanced endotoxin susceptibility of SR-BI-deficient mice due to their anti-inflammatory glucocorticoid deficiency complicates the understanding of SR-BI's role in endotoxemia/sepsis, calling for the use of alternative models. In this study, using human SR-BI (hSR-BI) and hSR-BII transgenic mice, we found that SR-BI and, to a lesser extent, its splicing variant SR-BII protect against LPS-induced lung damage. At 20 h after intratracheal LPS instillation, the extent of pulmonary inflammation and vascular leakage was significantly lower in hSR-BI and hSR-BII transgenic mice than in wild-type mice. Higher bronchoalveolar lavage fluid (BALF) inflammatory cell count and protein content and lung tissue neutrophil infiltration found in wild-type mice were associated with markedly (2 to 3 times) increased proinflammatory cytokine production compared to these parameters in transgenic mice following LPS administration. The markedly lower endotoxin levels detected in BALF of transgenic versus wild-type mice and the significantly increased BODIPY-LPS uptake observed in lungs of hSR-BI and hSR-BII mice 20 h after the i.t. LPS injection suggest that hSR-BI- and hSR-BII-mediated enhanced LPS clearance in the airways could represent the mechanism of their protective role against LPS-induced acute lung injury.


Subject(s)
Acute Lung Injury/metabolism , Lysosomal Membrane Proteins/metabolism , Receptors, Scavenger/metabolism , Scavenger Receptors, Class B/metabolism , A549 Cells , Acute Lung Injury/chemically induced , Animals , Bronchoalveolar Lavage Fluid , Cell Line, Tumor , Cytokines/metabolism , Disease Models, Animal , Endotoxemia/metabolism , Humans , Inflammation/immunology , Lipopolysaccharides/pharmacology , Male , Mice , Mice, Inbred C57BL , Mice, Transgenic , Neutrophils/metabolism , Sepsis/metabolism
9.
J Biol Chem ; 297(2): 100889, 2021 08.
Article in English | MEDLINE | ID: mdl-34181944

ABSTRACT

APOBEC3s are innate single-stranded DNA cytidine-to-uridine deaminases that catalyze mutations in both pathogen and human genomes with significant roles in human disease. However, how APOBEC3s mutate a single-stranded DNA that is available momentarily during DNA transcription or replication in vivo remains relatively unknown. In this study, utilizing hepatitis B virus (HBV) viral mutations, we evaluated the mutational characteristics of individual APOBEC3s with reference to the HBV replication process through HBV whole single-strand (-)-DNA genome mutation analyses. We found that APOBEC3s induced C-to-T mutations from the HBV reverse transcription start site continuing through the whole (-)-DNA transcript to the termination site with variable efficiency, in an order of A3B >> A3G > A3H-II or A3C. A3B had a 3-fold higher mutation efficiency than A3H-II or A3C with up to 65% of all HBV genomic cytidines being converted into uridines in a single mutation event, consistent with the A3B localized hypermutation signature in cancer, namely, kataegis. On the other hand, A3C expression led to a 3-fold higher number of mutation-positive HBV genome clones, although each individual clone had a lower number of C-to-T mutations. Like A3B, A3C preferred both 5'-TC and 5'-CC sequences, but to a lesser degree. The APOBEC3-induced HBV mutations were predominantly detected in the HBV rcDNA but were not detectable in other intermediates including HBV cccDNA and pgRNA by primer extension of their PCR amplification products. These data demonstrate that APOBEC3-induced HBV genome mutations occur predominantly when the HBV RNA genome was reversely transcribed into (-)-DNA in the viral capsid.


Subject(s)
APOBEC Deaminases/metabolism , DNA, Viral/genetics , Hepatitis B virus/genetics , Hepatitis B/virology , Mutation , RNA, Viral/genetics , APOBEC Deaminases/genetics , Cell Line, Tumor , Genome, Viral , Hepatitis B/pathology , Hepatitis B virus/isolation & purification , Hepatitis B virus/pathogenicity , Humans , RNA, Viral/metabolism , Reverse Transcription
10.
Am Nat ; 198(1): E12-E26, 2021 07.
Article in English | MEDLINE | ID: mdl-34143719

ABSTRACT

AbstractEarly warning signals (EWSs) have the potential to predict tipping points where catastrophic changes occur in ecological systems. However, EWSs are plagued by false negatives, leading to undetected catastrophes. One reason may be because EWSs do not occur equally for all species in a system, so whether and how strongly EWSs are detected depends on which species is being observed. Here, we illustrate how the strength of EWSs is determined by each species' relationship to properties of the noise, the system's response to that noise, and the occurrence of critical slowing down (the dynamical phenomenon that gives rise to EWSs). Using these relationships, we present general rules for maximizing EWS detection in ecological communities. We find that for two-species competitive and mutualistic systems, one should generally monitor the species experiencing smaller intraspecific effects to maximize EWS performance, while in consumer-resource systems, one should monitor the species imposing the smaller interspecific effects. These guidelines appear to hold for at least some larger communities as well. We close by extending the theoretical basis for our rules to systems with any number of species and more complex forms of noise. Our findings provide important guidance on how to monitor systems for EWSs to maximize detection of tipping points.


Subject(s)
Biota , Ecosystem , Symbiosis
11.
Int J Health Policy Manag ; 10(9): 585-587, 2021 09 01.
Article in English | MEDLINE | ID: mdl-32801222

ABSTRACT

This commentary focuses on Latin America, a region known for its rich variety of populist politicians and some of the most extensive welfare states in the Global South. Contemporary Latin America offers examples of left-wing and right-wing populist leaders, none of whom demonstrate the same focus on excluding immigrants from welfare state benefits as that noted by Chiari Rinaldi and Marleen Bekker in the European context. We see this contrast not because immigrants' access to health services is less important in Latin America, but because Latin American populists are more focused on internal "enemies." The commentary concludes with observations regarding Latin American populist leaders' handling of the Coronavirus disease 2019 (COVID-19) pandemic.


Subject(s)
COVID-19 , Population Health , COVID-19/epidemiology , COVID-19/prevention & control , Europe , Health Policy , Humans , Latin America , Politics , SARS-CoV-2
12.
PLoS One ; 15(10): e0240659, 2020.
Article in English | MEDLINE | ID: mdl-33057430

ABSTRACT

SR-BI binds various lipoproteins, including HDL, LDL as well as VLDL, and mediates selective cholesteryl ester (CE) uptake. HDL derived CE accumulates in cellular lipid droplets (LDs), which also store triacylglycerol (TAG). We hypothesized that SR-BI could significantly facilitate LD formation, in part, by directly transporting LDL derived neutral lipids (NL) such as CE and TAG into LDs without lipolysis and de novo lipid synthesis. SR-BI overexpression greatly increased LDL uptake and LD formation in stably transfected HeLa cells (SR-BI-HeLa). LDs isolated from SR-BI-HeLa contained 4- and 7-times more CE and TAG, respectively, than mock-transfected HeLa (Mock-HeLa). In contrast, LDL receptor overexpression in HeLa (LDLr-HeLa) greatly increased LDL uptake, degradation with moderate 1.5- and 2-fold increases of CE and TAG, respectively. Utilizing CE and TAG analogs, BODIPY-TAG (BP-TAG) and BODIPY-CE (BP-CE), for tracking LDL NL, we found that after initial binding of LDL to SR-BI-HeLa, apoB remained at the cell surface, while BP-CE and BP-TAG were sorted and simultaneously transported together to LDs. Both lipids demonstrated limited internalization to lysosomes or endoplasmic reticulum in SR-BI-HeLa. In LDLr-HeLa, NLs demonstrated clear lysosomal sequestration without their sorting to LDs. An inhibition of TAG and CE de novo synthesis by 90-95% only reduced TAG and CE LD content by 45-50%, and had little effect on BP-CE and BP-TAG transport to LDs in SR-BI HeLa. Furthermore, intravenous infusion of 1-2 mg of LDL increased liver LDs in normal (WT) but not in SR-BI KO mice. Mice transgenic for human SR-BI demonstrated higher liver LD accumulation than WT mice. Finally, Electro Spray Infusion Mass Spectrometry (ESI-MS) using deuterated d-CE found that LDs accumulated up to 40% of unmodified d-CE LDL. We conclude that SR-BI mediates LDL-induced LD formation in vitro and in vivo. In addition to cytosolic NL hydrolysis and de novo lipid synthesis, this process includes selective sorting and transport of LDL NL to LDs with limited lysosomal NL sequestration and the transport of LDL CE, and TAG directly to LDs independently of de novo synthesis.


Subject(s)
Lipid Droplets/metabolism , Lipids/chemistry , Lipoproteins, LDL/metabolism , Scavenger Receptors, Class B/metabolism , Animals , Biological Transport/drug effects , Boron Compounds/metabolism , Cholesterol Esters/metabolism , Coenzyme A Ligases/antagonists & inhibitors , Coenzyme A Ligases/metabolism , Enzyme Inhibitors/pharmacology , HeLa Cells , Humans , Lipid Droplets/drug effects , Liver/drug effects , Liver/metabolism , Lysosomes/drug effects , Lysosomes/metabolism , Mice, Inbred C57BL , Mice, Knockout , Receptors, LDL/metabolism , Triazenes/pharmacology , Triglycerides/metabolism
13.
Clin J Oncol Nurs ; 24(5): 489-494, 2020 10 01.
Article in English | MEDLINE | ID: mdl-32945795

ABSTRACT

BACKGROUND: Falls experienced by patients undergoing blood and marrow transplantation or treatment with cellular immunotherapy (BMT-CI) may result in injury or death. An algorithm was developed using the patient fall circumstances identified in a chart analysis from 2016. OBJECTIVES: This study aimed to determine if the Moffitt BMT-CI Orthostatic Vital Signs Algorithm could decrease inpatient falls. METHODS: A pre-/post-test program evaluation was conducted for one year pre- and postimplementation of the algorithm on newly admitted inpatients. Adherence rate of nurses using the algorithm was monitored. FINDINGS: Overall falls decreased from 5.38% to 3.44%, with zero falls or injuries related to orthostasis for newly admitted patients. Adherence of nurses using the algorithm increased from 60% to 93%. The fall rate has been sustained less than baseline with 100% adherence, and the algorithm has been adopted as standard of practice.


Subject(s)
Bone Marrow , Vital Signs , Algorithms , Humans , Immunotherapy/adverse effects
14.
Int J Health Policy Manag ; 9(10): 429-431, 2020 10 01.
Article in English | MEDLINE | ID: mdl-32610732

ABSTRACT

Political scientists bring important tools to the analysis of the coronavirus disease 2019 (COVID-19) pandemic, particularly a focus on the crucial role of power in global health politics. We delineate different kinds of power at play during the COVID-19 crisis, showing how a dearth of compulsory, institutional, and epistemic power undermined global cooperation and fueled the pandemic, with its significant loss to human life and huge economic toll. Through the pandemic response, productive and structural power became apparent, as issue frames stressing security and then preserving livelihoods overwhelmed public health and human rights considerations. Structural power rooted in economic inequalities between and within countries conditioned responses and shaped vulnerabilities, as the crisis threatened to deepen power imbalances along multiple lines. Calls for global health security will surely take on a new urgency in the aftermath of the pandemic and the forms of power delineated here will shape their outcome.


Subject(s)
COVID-19/prevention & control , COVID-19/therapy , Global Health/legislation & jurisprudence , Health Policy/legislation & jurisprudence , Power, Psychological , Public Health/legislation & jurisprudence , Humans , Politics , SARS-CoV-2
15.
Glob Public Health ; 15(6): 805-817, 2020 06.
Article in English | MEDLINE | ID: mdl-32013785

ABSTRACT

This article outlines an agenda for political science engagement with global mental health. Other social sciences have tackled the topic, investigating such questions as the link between poverty and mental health disorders. Political science is noticeably absent from these explorations. This is striking because mental health disorders affect one billion people globally, governments spend only about 2% of their health budgets on these disorders, and most people lack access to treatment. With its focus on power, political science could deepen knowledge on vulnerabilities to mental illness and explain weak policy responses. By illustrating how various forms of power pertaining to governance, knowledge, and moral authority work through the concepts of issue framing, collective action, and institutions, the article shows that political science can deepen knowledge on this global health issue. Political science can analyse how incomplete knowledge leads to contentious framing, thus hobbling advocacy. It can explain why states shirk their obligations in mental health, and it can question how incentives drive mental health mobilisation. The discipline can uncover how power undergirds institutional responses to global mental health at the international, national, and community levels. Political science should collaborate with other social sciences in research networks to improve policy outcomes.


Subject(s)
Global Health , Mental Health , Health Policy , Humans , Politics , Poverty
16.
Child Neurol Open ; 6: 2329048X19876199, 2019.
Article in English | MEDLINE | ID: mdl-31763346

ABSTRACT

Calcium-sensing receptor gain-of-function mutations are known to cause autosomal dominant hypocalcemia and independently an epilepsy syndrome. We report the unique case of a child with both intractable generalized epilepsy and a chronic abnormality in calcium homeostasis due to a calcium-sensing receptor gene mutation. She is a 16-year-old female who began having staring events around 3 years of age. After her first generalized convulsion at age 5 years, investigations revealed hypocalcemia, hypercalciuria, and central nervous system calcifications. Her electroencephalogram demonstrated generalized epileptiform discharges, a hyperventilation-induced electroclinical seizure, and a photoconvulsive response. She has since been diagnosed with intellectual impairment, behavior disorder, and intractable childhood-onset seizures, the latter of which include eyelid myoclonia with absences. We conclude that calcium-sensing receptor gain-of-function mutations may precipitate an intractable generalized epilepsy syndrome with a comorbid endocrinopathy and that further investigations should be pursued in children with seizures presumed to be provoked by hypocalcemia.

17.
Nat Commun ; 10(1): 734, 2019 02 08.
Article in English | MEDLINE | ID: mdl-30737404

ABSTRACT

The original version of this Article contained errors in Fig. 5. In panels i and j the three rightmost x-axis labels inadvertently read 'Tmc1' instead of 'Tmc2'. These errors have been corrected in both the PDF and HTML versions of the Article.

18.
PLoS One ; 14(1): e0209918, 2019.
Article in English | MEDLINE | ID: mdl-30699139

ABSTRACT

Tropical deforestation is one of the most pressing threats to biodiversity, and substantially reduces ecosystem services at the global scale. Little is known however about the global spatial distribution of the actors behind tropical deforestation. Newly available maps of global cropland field size offer an opportunity to gain understanding towards the spatial distribution of tropical deforestation actors. Here we use a map of global cropland field size and combine it with maps of forest loss to study the spatial association between field size and deforestation while accounting for other anthropogenic and geographical drivers of deforestation. We then use linear mixed-effects models and bootstrapping to determine what factors affect field sizes within deforested areas across all countries in the global tropics and subtropics. We find that field size within deforested areas is largely determined by country-level effects indicating the importance of socio-economic, cultural and institutional factors on the distribution of field sizes. Typically, small field sizes appear more commonly in deforested areas in Africa and Asia while the association was with larger field sizes in Australia and the Americas. In general, we find that smaller field sizes are associated with deforestation in protected areas and large field sizes with areas with lower agricultural value, although these results have low explanatory power. Our results suggest that the spatial patterns of actors behind deforestation are aggregated geographically which could help target conservation and sustainable land-use strategies.


Subject(s)
Biodiversity , Conservation of Natural Resources , Crop Production , Forests , Tropical Climate , Socioeconomic Factors
19.
Nat Commun ; 10(1): 236, 2019 01 22.
Article in English | MEDLINE | ID: mdl-30670701

ABSTRACT

Fifty percent of inner ear disorders are caused by genetic mutations. To develop treatments for genetic inner ear disorders, we designed gene replacement therapies using synthetic adeno-associated viral vectors to deliver the coding sequence for Transmembrane Channel-Like (Tmc) 1 or 2 into sensory hair cells of mice with hearing and balance deficits due to mutations in Tmc1 and closely related Tmc2. Here we report restoration of function in inner and outer hair cells, enhanced hair cell survival, restoration of cochlear and vestibular function, restoration of neural responses in auditory cortex and recovery of behavioral responses to auditory and vestibular stimulation. Secondarily, we find that inner ear Tmc gene therapy restores breeding efficiency, litter survival and normal growth rates in mouse models of genetic inner ear dysfunction. Although challenges remain, the data suggest that Tmc gene therapy may be well suited for further development and perhaps translation to clinical application.


Subject(s)
Deafness/genetics , Genetic Predisposition to Disease , Genetic Therapy/methods , Hearing Loss/genetics , Labyrinth Diseases/genetics , Membrane Proteins/genetics , Animals , Deafness/therapy , Hair Cells, Auditory/physiology , Hair Cells, Vestibular/physiology , Hearing Loss/therapy , Labyrinth Diseases/therapy , Mice , Mice, Mutant Strains
20.
J Adv Pract Oncol ; 10(Suppl 3): 29-40, 2019.
Article in English | MEDLINE | ID: mdl-33520344

ABSTRACT

Two anti-CD19 chimeric antigen receptor (CAR) T-cell therapies are approved for adults with relapsed or refractory large B-cell lymphoma after more than two lines of therapy. Although CAR T-cell therapy has demonstrated significant efficacy for some patients, the treatment process can be long and complex and each phase is associated with unique challenges. Care for patients receiving CAR T-cell therapy also involves many health-care players, including physicians, advanced practitioners, nurse coordinators, nurse educators, apheresis nurses, inpatient and outpatient nurses, cellular therapy technologists, case managers, social workers, and many more. Dedicated educational efforts for patients, caregivers, and clinical providers that make up the multidisciplinary care team are warranted for optimal treatment. Here we present an overview of key elements for education along the treatment journey with CAR T-cell therapy, including considerations for each stage of therapy and specific recommendations for care teams, patients, and caregivers. We also present examples of educational programs that have been implemented at our institution.

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