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BACKGROUND: Data are limited on prostate cancer (PC) management in patients with haemophilia (PWH). AIM: To describe PC screening and diagnosis, treatment modalities and bleeding complications in a group of unselected PWH followed at French Haemophilia Treatment Centres (HTCs) PATIENTS AND METHODS: PC screening, management and bleeding complications were retrospectively investigated at 14 French HTCs between 2003 and 2018. RESULTS: Among> 1549 > 50-year-old PWHs, 73 (4.7%) underwent PC screening (median age 71.1 years; 67/6â¯HA/HB, 17/56 severe-moderate/mild). At diagnosis, haematuria was infrequent. Prophylaxis was administered during 76/86 (88%) prostate biopsies (PB) (n = 67 clotting factor concentrates, CFC; n = 9 desmopressin; n = 17 associated with tranexamic acid, TA). Bleeding (11/86, 12.8%) occurred mainly post-prophylaxis (median delay: 7 days): haematuria (9/11, 81.8%), and rectal bleeding (2/11, 18.2%) including one major (1.2%). PC was confirmed in 50/86 PB and in two prostatectomy specimens (total n = 50 patients, n = 6 with only active surveillance). Surgery (n = 28/44 patients) was managed with CFC. Fifteen patients had radiotherapy/brachytherapy, 10â¯had hormone therapy; CFC-based prophylaxis was only prescribed for brachytherapy (n = 2). Major bleedings occurred in 3/28 (10.7%) and 2/15 (13.3%) patients who underwent surgery and radio/brachytherapy, respectively. No bleeding risk factor was found. CONCLUSION: Our data indicate that PB requires prophylaxis for atleast 7 days, using CFC, desmopressin or TA in function of haemophilia severity. PC surgery should be considered at high bleeding risk. Long-term post-procedural CFC or oral TA could be discussed. Radiotherapy/brachytherapy also should be managed with prophylaxis (CFC or TA).
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Hemofilia A , Neoplasias da Próstata , Idoso , Biópsia , Desamino Arginina Vasopressina/uso terapêutico , Hematúria/complicações , Hemofilia A/complicações , Hemofilia A/tratamento farmacológico , Hemorragia/complicações , Hemorragia/prevenção & controle , Humanos , Masculino , Pessoa de Meia-Idade , Próstata , Neoplasias da Próstata/complicações , Neoplasias da Próstata/terapia , Estudos RetrospectivosRESUMO
BACKGROUND: Nonacog alfa, a standard half-life recombinant factor IX (FIX), is used as a prophylactic treatment in severe haemophilia B (SHB) patients. Its half-life determined in clinical studies involving a limited sampling (72 h) was shown to be rather short. In our clinical practice, we suspected that its half-life could have been underestimated. OBJECTIVES: We aimed to evaluate nonacog alfa pharmacokinetics in real world clinical practice based on FIX levels in patients receiving prophylaxis. METHODS: We retrospectively collected data on patients with SHB receiving prophylaxis from eight centres across France. The terminal half-life (THL), time to reach 5-2 IU/dl and FIX activity at 48, 72 and 96 h were derived by Bayesian estimations using NONMEM analysis. RESULTS AND CONCLUSIONS: Infusion data (n = 455) were collected from 64 patients with SHB. The median THL measured in 92 pharmacokinetic (PK) studies was 43.4 h. In 26 patients ≤12 years of age, 51 PK studies showed a median time to reach 5 IU/dl of FIX of 70.5 h and a median time to reach 2 IU/dl of 121.5 h. In 38 patients 13-75 years of age, 41 PK studies showed a median time to reach 5 IU/dl of FIX of 92.0 h and a median time to reach 2 IU/dl of 167.5 h. Extending the sampling beyond 72 h makes it possible to observe a plateau, with FIX remaining between 2 and 5 IU/dl for several days and shows that the THL of nonacog alfa might be longer than previously described. ESSENTIALS: Nonacog alfa terminal half-life (THL) in patients receiving regular prophylaxis was evaluated in clinical practice. The median THL was estimated to be 36.9 h for patients aged .8-12 years. The median THL was estimated to be 49.9 h for patients aged 13-75 years. For patients aged ≤12 and >12 years, the median times to reach 5 IU/dl were 70.5 and 92 h, respectively; to reach 3 IU/dl, 95.5 and 131.5 h, respectively; to reach 2 IU/dl, 121.5 and 167.5 h, respectively. We suggest that the half-life of nonacog alfa might be longer than previously described in both younger and older patients.
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Fator IX , Hemofilia B , Adulto , Teorema de Bayes , Fator IX/farmacocinética , Fator IX/uso terapêutico , Meia-Vida , Hemofilia B/tratamento farmacológico , Humanos , Pessoa de Meia-Idade , Proteínas Recombinantes/farmacocinética , Proteínas Recombinantes/uso terapêutico , Estudos RetrospectivosRESUMO
BACKGROUND: A transformation is underway regarding how we deal with our health. Mobile devices make it possible to have continuous access to personal health information. Wearable devices, such as Fitbit and Apple's smartwatch, can collect data continuously and provide insights into our health and fitness. However, lack of interoperability and the presence of data silos prevent users and health professionals from getting an integrated view of health and fitness data. To provide better health outcomes, a complete picture is needed which combines informal health and fitness data collected by the user together with official health records collected by health professionals. Mobile apps are well positioned to play an important role in the aggregation since they can tap into these official and informal health and data silos. OBJECTIVE: The objective of this paper is to demonstrate that a mobile app can be used to aggregate health and fitness data and can enable interoperability. It discusses various technical interoperability challenges encountered while integrating data into one place. METHODS: For 8 years, we have worked with third-party partners, including wearable device manufacturers, electronic health record providers, and app developers, to connect an Android app to their (wearable) devices, back-end servers, and systems. RESULTS: The result of this research is a health and fitness app called myFitnessCompanion, which enables users to aggregate their data in one place. Over 6000 users use the app worldwide to aggregate their health and fitness data. It demonstrates that mobile apps can be used to enable interoperability. Challenges encountered in the research process included the different wireless protocols and standards used to communicate with wireless devices, the diversity of security and authorization protocols used to be able to exchange data with servers, and lack of standards usage, such as Health Level Seven, for medical information exchange. CONCLUSIONS: By limiting the negative effects of health data silos, mobile apps can offer a better holistic view of health and fitness data. Data can then be analyzed to offer better and more personalized advice and care.
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Telefone Celular/instrumentação , Aplicativos Móveis/estatística & dados numéricos , Telemedicina/estatística & dados numéricos , Humanos , Aptidão FísicaRESUMO
BACKGROUND: rVIII-SingleChain is a recombinant factor VIII (FVIII) with increased binding affinity to von Willebrand factor compared with other FVIII products. rVIII-SingleChain is indicated for the treatment and prevention of bleeding episodes in patients with hemophilia A. OBJECTIVES: To collect real-world evidence data from patients treated with rVIII-SingleChain to confirm the efficacy and safety established in the clinical trial program and carry out a population pharmacokinetic (PK) analysis. METHODS: This interim analysis includes data, collected between January 2018 - September 2021, from patients treated with rVIII-SingleChain prophylaxis at French Hemophilia Treatment centers. Data on annualized bleeding rates, dosing frequency, and consumption before and after switching to rVIII-SingleChain were recorded. A population PK analysis was also conducted to estimate PK parameters. RESULTS: Overall, 43 patients switched to prophylaxis with rVIII-SingleChain either from a previous prophylaxis regimen or from on-demand treatment. Following the switch to rVIII-SingleChain, patients maintained excellent bleed control. After switching to rVIII-SingleChain, most patients maintained or reduced their regimen. Interestingly, a majority of patients treated >2 ×/weekly with a standard half-life FVIII reduced both injection frequency and FVIII consumption with rVIII-SingleChain. A PK analysis revealed a lower clearance of rVIII-SingleChain (1.9 vs. 2.1 dL/h) and a longer half-life both in adolescents/adults (n = 28) and pediatric (n = 6) patients (15.5 and 11.9 hours, respectively vs. 14.5 and 10.3 hours) than previously reported. CONCLUSIONS: Patients who switched to rVIII-SingleChain prophylaxis demonstrated excellent bleed control and a reduction in infusion frequency. A population PK analysis revealed improved PK parameters compared with those reported in the clinical trial.
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Hemofilia A , Hemostáticos , Adulto , Adolescente , Humanos , Criança , Hemofilia A/tratamento farmacológico , Fator VIII/farmacocinética , Fator de von Willebrand/efeitos adversos , Hemorragia/induzido quimicamente , Hemostáticos/efeitos adversos , Meia-VidaRESUMO
BACKGROUND: Women with hereditary fibrinogen disorders (HFDs) seem to be at an increased risk of adverse obstetrical outcomes, but epidemiologic data are limited. OBJECTIVES: We aimed to determine the prevalence of pregnancy complications; the modalities and management of delivery; and the postpartum events in women with hypofibrinogenemia, dysfibrinogenemia, and hypodysfibrinogenemia. METHODS: We conducted a retrospective and prospective multicentric international study. RESULTS: A total of 425 pregnancies were investigated from 159 women (49, 95, and 15 cases of hypofibrinogenemia, dysfibrinogenemia, and hypodysfibrinogenemia, respectively). Overall, only 55 (12.9%) pregnancies resulted in an early miscarriage, 3 (0.7%) resulted in a late miscarriage, and 4 (0.9%) resulted in an intrauterine fetal death. The prevalence of live birth was similar among the types of HFDs (P = .31). Obstetrical complications were observed in 54 (17.3%) live birth pregnancies, including vaginal bleeding (14, 4.4%), retroplacental hematoma (13, 4.1%), and thrombosis (4, 1.3%). Most deliveries were spontaneous (218, 74.1%) with a vaginal noninstrumental delivery (195, 63.3%). A neuraxial anesthesia was performed in 116 (40.4%) pregnancies, whereas general or no anesthesia was performed in 71 (16.6%) and 129 (44.9%) pregnancies, respectively. A fibrinogen infusion was administered in 28 (8.9%) deliveries. Postpartum hemorrhages were observed in 62 (19.9%) pregnancies. Postpartum venous thrombotic events occurred in 5 (1.6%) pregnancies. Women with hypofibrinogenemia were at an increased risk of bleeding during the pregnancy (P = .04). CONCLUSION: Compared with European epidemiologic data, we did not observe a greater frequency of miscarriage, while retroplacental hematoma, postpartum hemorrhage, and thrombosis were more frequent. Delivery was often performed without locoregional anesthesia. Our findings highlight the urgent need for guidance on the management of pregnancy in HFDs.
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Afibrinogenemia , Hemostáticos , Hemorragia Pós-Parto , Trombose , Feminino , Humanos , Gravidez , Aborto Espontâneo/etiologia , Afibrinogenemia/complicações , Afibrinogenemia/epidemiologia , Fibrinogênio , Hemorragia Gastrointestinal , Hematoma/complicações , Hemorragia Pós-Parto/epidemiologia , Hemorragia Pós-Parto/etiologia , Estudos Prospectivos , Estudos Retrospectivos , Trombose/complicaçõesRESUMO
Smartphones and tablets are slowly but steadily changing the way we look after our health and fitness. Today, many high quality mobile apps are available for users and health professionals and cover the whole health care chain, i.e. information collection, prevention, diagnosis, treatment and monitoring. Our team has developed a mobile health and fitness app called myFitnessCompanion® which has been available via Android market since February 2011. The objective of this paper is to share our experience with rolling out a mobile health and fitness app. We discuss the acceptance of health apps by end-users and healthcare industry. We discuss how mobile health apps will be distributed in the near future, the use of Personal Health Record (PHR) systems such as Microsoft HealthVault and the impact of regulations (FDA) on the future of mobile health apps. The paper is based on seven years of experience by the authors as mobile health and fitness application developers and we discuss the challenges and opportunities for app developers in the health industry.
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Telefone Celular/estatística & dados numéricos , Computadores de Mão , Monitorização Ambulatorial/métodos , Aptidão Física/fisiologia , Software , Telemedicina/instrumentação , Interface Usuário-Computador , Diagnóstico por Computador/estatística & dados numéricos , HumanosRESUMO
Hypertension is a chronic condition caused by a poor lifestyle that affects patients' lives. Adherence to self-management programs increases hypertension self-monitoring, and allows greater prevention and disease management. Patient compliance with hypertension self-management is low in general; therefore, mobile health applications (mHealth-Apps) are becoming a daily necessity and provide opportunities to improve the prevention and treatment of chronic diseases, including hypertension. This research aims to explore Saudi individuals' perspectives and needs regarding designing a hypertension management mobile app to be used by hypertension patients to better manage their illnesses. Semi-structured interviews were conducted with 21 Saudi participants to explore their perspectives and views about the needs and requirements in designing a hypertension mobile technology solution, as well as usability and culture in the Saudi context. The study used NVivo to analyze data and divided the themes into four main themes: the app's perceived health benefits, features and usability, suggestions for the app's content, and security and privacy. The results showed that there are many suggestions for improvements in mobile health apps that developers should take into consideration when designing apps. The mobile health apps should include physical activity tracking, related diet information, and reminders, which are promising, and could increase adherence to healthy lifestyles and consequently improve the self-management of hypertension patients. Mobile health apps provide opportunities to improve hypertension patients' self-management and self-monitoring. However, this study asserts that mobile health apps should not share users' data, and that adequate privacy disclosures should be implemented.
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Hipertensão , Aplicativos Móveis , Telemedicina , Humanos , Hipertensão/prevenção & controle , Pesquisa Qualitativa , Arábia Saudita , TecnologiaRESUMO
BACKGROUND: National implementation of electronic personal health record (ePHR) systems is of vital importance to governments worldwide because this type of technology promises to promote and enhance healthcare. Although there is widespread agreement as to the advantages of ePHRs, the level of awareness and acceptance of this technology among healthcare consumers has been low. OBJECTIVE: The aim of this study was to identify the factors that can influence the acceptance and use of an integrated ePHR system in Saudi Arabia. METHOD: The unified theory of acceptance and use of technology model was extended in this study to include e-health literacy (e-HL) and tested using structural equation modelling. Data were collected via a questionnaire survey, resulting in 794 valid responses. RESULTS: The proposed model explained 56% of the variance in behavioural intention (BI) to use the integrated ePHR system. Findings also highlighted the significance of performance expectancy, effort expectancy, social influence (SI) and e-HL as determinants of Saudi healthcare consumers' intentions to accept and use the integrated ePHR system. Additionally, assessment of the research model moderators revealed that only gender had a moderating influence on the relationship between SI and BI. Finally, findings showed a low level of awareness among Saudi citizens about the national implementation of an integrated ePHR system, suggesting the need to promote a greater and more widespread awareness of the system and to demonstrate its usefulness. CONCLUSION: Findings from this study can assist governments, policymakers and developers of health information technologies and systems by identifying important factors that may influence the diffusion and use of integrated ePHRs.
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Letramento em Saúde , Registros de Saúde Pessoal , Confidencialidade , Eletrônica , Arábia SauditaRESUMO
Process-of-care studies participate in improving the efficiency of the care pathway for patient with haemophilia (CPPH) and rationalize the multidisciplinary management of patients. Our objective is to establish a current overview of the different actors involved in the management of patients with haemophilia and to provide an accurate description of the patient trajectory. This is a qualitative exploratory research based on interviews of the principal health professionals of four haemophilia services, between November 2019 and February 2020, in France. Mapping of the CPPH processes within the different institutions and/or services, as well as the rupture zones, were identified. Treatment delivery and biological analyses were carried out exclusively in healthcare institutions. The main liberal health professionals solicited were nurses, physiotherapists and general practitioner. Obstacles and barriers within the specialized service, with other hospital services and external hospital or private services, community health care providers et community environment and individual one was complex and multiples. Our research identified potential concerns that need to be addressed to improve future studies to identify influential elements. Similarly, other qualitative studies will have to be conducted on the perceptions and literacy of patients with haemophilia to develop a global interactive mapping of their trajectories.
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Clínicos Gerais , Hemofilia A , Procedimentos Clínicos , França , Humanos , Pesquisa QualitativaRESUMO
BACKGROUND: Several innovative drugs liable to lead to changes in healthcare organization are or soon will be available for the management of hemophilia. Analyzing their implementation can shed further light on healthcare decision-making, to anticipate changes and risk of breakdown in the patient's care pathway. METHODS: Multiple criteria decision analysis (MCDA), based on ISPOR recommendations, was used to assess the organizational impact of innovation in hemophilia care management. The MCDA process designed for this specific context involved ten French experts in hemophilia care management (physicians, nurses, pharmacist, physiotherapist and psychologist) in the hemophilia care center of Chambéry, in the Rhône-Alpes Region of France. This pilot study involved seven steps: (i) defining the decision problem; (ii) selecting and structuring criteria; (iii) assessing the relative weight of each criterion with software-assisted simulation based on pairwise comparisons of different organizational change scenarios; (iv) measuring the performance of the selected innovations; (v) scoring alternatives; (vi) calculating aggregate scores; (vii) discussion. The endpoint was to determine the expected overall organizational impact on a 0-100 scale. RESULTS: Seven organizational criteria were selected. "Acceptability for patient/caregiver/association" was the most heavily weighted. Factor VIII by subcutaneous route obtained the highest aggregate score: i.e., low impact on care organization (88.8 out of 100). The innovation with strongest organizational impact was gene therapy (27.3 out of 100). CONCLUSION: This approach provided a useful support for discussion, integrating organizational aspects in the treatment decision-making process, at healthcare team level. The study needs repeating in a few years' time and in other hemophilia centers.
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Hemofilia A , Procedimentos Clínicos , Técnicas de Apoio para a Decisão , Hemofilia A/terapia , Humanos , Inovação Organizacional , Projetos PilotoRESUMO
PURPOSE: Although 3D food printing is expected to enable the creation of visually appealing pureed food for people with disability and dysphagia, little is known about the user experience in engaging with 3D food printing or the feasibility of use with populations who need texture-modified foods. The aim of this study was to explore the feasibility and usability of using domestic-scale 3D food printer as an assistive technology to print pureed food into attractive food shapes for people with dysphagia. MATERIALS AND METHODS: In total, 16 participants engaged in the unfamiliar, novel process of using a domestic-scale 3D food printer (choosing, printing, tasting), designed for printing pureed food, and discussed their impressions in focus group or individual interviews. RESULTS AND CONCLUSIONS: Overall, results demonstrated that informed experts who were novice users perceived the 3D food printing process to be fun but time consuming, and that 3D food printers might not yet be suitable for people with dysphagia or their supporters. Slow response time, lack of user feedback, scant detail on the appropriate recipes for the pureed food to create a successful print, and small font on the user panel interface were perceived as barriers to accessibility for people with disability and older people. Participants expected more interactive elements and feedback from the device, particularly in relation to resolving printer or user errors. This study will inform future usability trials and food safety research into 3D printed foods for people with disability and dysphagia. IMPLICATIONS FOR REHABILITATION3D food printers potentially have a role as an assistive technology in the preparation of texture-modified foods for people with disability and dysphagia.To increase feasibility, 3D food printers should be co-designed with people with disability and their supporters and health professionals working in the field of dysphagia and rehabilitation.Experts struggled to be able to print 3D pureed shapes owing to relatively low usability of the 3D food printer tested with problems using the interface and resolving problems in the print.3D food printing is a fun and novel activity and may help to engage people with disability and dysphagia in making choices around the shape of the food to be printed.
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Mobile application (app) use is increasingly becoming an essential part of our daily lives. Due to their significant usefulness, people rely on them to perform multiple tasks seamlessly in almost all aspects of everyday life. Similarly, there has been immense progress in artificial intelligence (AI) technology, especially deep learning, computer vision, natural language processing, and robotics. These technologies are now actively being implemented in smartphone apps and healthcare, providing multiple healthcare services. However, several factors affect the usefulness of mobile healthcare apps, and usability is an important one. There are various healthcare apps developed for each specific task, and the success of these apps depends on their performance. This study presents a systematic review of the existing apps and discusses their usability attributes. It highlights the usability models, outlines, and guidelines proposed in previous research for designing apps with improved usability characteristics. Thirty-nine research articles were reviewed and examined to identify the usability attributes, framework, and app design conducted. The results showed that satisfaction, efficiency, and learnability are the most important usability attributes to consider when designing eHealth mobile apps. Surprisingly, other significant attributes for healthcare apps, such as privacy and security, were not among the most indicated attributes in the studies.
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Aplicativos Móveis , Telemedicina , Inteligência Artificial , Atenção à Saúde , Humanos , PrivacidadeRESUMO
Congenital hypodysfibrinogenemia is a rare fibrinogen disorder, defined by decreased levels of a dysfunctional fibrinogen. We present the functional and structural characterization of two new fibrinogen variants. A duplication of 32 bases in FGA exon 5, p.Ser382GlyfsTer50 was identified in a patient (P1) with history of hemoptysis and traumatic cerebral bleeding. A missense mutation in FGG exon 8, p.Ala353Ser was identified in two siblings (P2 and P3) with tendency to bruising and menorrhagia. Fibrin polymerization was studied in plasma and in purified fibrinogen by turbidimetry. Fibrin structure was studied by a permeability assay, laser scanning confocal microscopy (LSCM) and scanning electron microscopy (SEM). In both plasma and purified fibrinogen samples, all patients had an abnormal polymerization characterized by a decreased maximal absorption compared to controls. The permeation constant (Ks) was markedly increased in all patients: 31 ± 9 × 10-9 cm2 in P1, and 20 ± 0.1 × 10-9 cm2 in P2 and P3, compared to 6 ± 2 × 10-9 cm2 in the control (p < 0.05). The presence of very large pores that accounts for the increased Ks was confirmed by LSCM and SEM patients' clots images. By SEM, the patients' fibrin fibers diameters were thicker: 90 ± 25 nm in P1, 162 ± 64 nm in P2 and 132 ± 46 nm in P3 compared to 74 ± 25 nm in control (p < 0.0001). In conclusion, both new causative fibrinogen mutations altered clot structure by forming thick fibers, diminishing fiber branching, and increasing pore filling space. These structural changes to clots explain the patients' bleeding phenotypes.
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Afibrinogenemia , Fibrinogênio , Afibrinogenemia/genética , Feminino , Fibrina , Fibrinogênio/genética , Humanos , Microscopia Eletrônica de Varredura , Mutação de Sentido Incorreto , FenótipoRESUMO
BACKGROUND: New therapies provide a favorable evolution in the care management of persons with hemophilia. However, the impact of these new therapies on patient care organization remains to be determined. A qualitative study will be implemented to analyze patients' perception regarding the impact of innovation on the organization of their care management. Secondary objectives will include refining specific factors related to persons with hemophilia (barriers or facilitators, especially the place of treatment) to consider within an organizational impact analysis. PATIENTS AND METHODS: Semi-structured individual interviews will be conducted via videoconferencing or by phone by two researchers using an interview guide. Participants will be recruited from the Rhône-Alpes region, in France. Physicians from two hemophilia treatment centers will identify eligible patients. Moreover, a call for volunteers will be launched by the Rhône-Alpes committee of the French hemophilia association. Interviews will be conducted with adult patients, adolescent patients or parents of a minor with hemophilia regularly treated prophylactically or on demand. Data analysis will be performed with NVivo® software. Each interview will be analyzed by two researchers using an inductive content analytic method. DISCUSSION: The INNOVHEMO study is an original study analyzing the way patients perceive the impact of an innovation on their care management organization. The resulting patient-specific factors, identified as barriers or facilitators, will need to be integrated into a more comprehensive analysis of the impact of innovation on care management organization.
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Turbidity analysis is widely used as a quantitative technique in hereditary dysfibrinogenemia. We aimed to compare several coagulation triggers in hereditary dysfibrinogenemia and control plasmas. We included 20 patients with hereditary dysfibrinogenemia, 19 with hotspot mutations Aα Arg35His (nâ=â9), Aα Arg35Cys (nâ=â2), γ Arg301His (nâ=â6), γ Arg301Cys (nâ=â2), and one with Aα Phe27Tyr, and a commercial pooled normal plasma. Fibrin polymerization was activated by bovine or human thrombin or tissue factor (TF), in the presence or absence of tissue type plasminogen activator. The lag time (min), slope (mOD/s), maximum absorbance (MaxAbs, mOD), and area under the curve (AUCp, ODâs) were calculated from the fibrin polymerization curves and the time for 50% clot degradation (T50, min), AUCf (ODâs) and the overall fibrinolytic potential from fibrinolysis curves. The lag time was significantly shorter and AUC increased in Aα Arg35His patients with bovine thrombin as compared with human thrombin. The MaxAbs and AUCp were significantly higher in γArg301His patients with bovine thrombin compared with human thrombin. Fibrin polymerization parameters of patients' samples were closer to those of control when assessed with TF compared with both human and bovine thrombin. T50 and overall fibrinolytic potential were similar in all samples regardless of the coagulation trigger used, however, with TF the AUCf of Aα Arg35His and γ Arg301His groups were significantly decreased compared with control. Bovine and human thrombin cannot be used equally for studying fibrin polymerization in hotspot hereditary dysfibrinogenemia or control plasmas.
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Afibrinogenemia/sangue , Coagulação Sanguínea , Adolescente , Adulto , Afibrinogenemia/genética , Animais , Testes de Coagulação Sanguínea/métodos , Bovinos , Feminino , Fibrinogênio/genética , Humanos , Indicadores e Reagentes , Masculino , Pessoa de Meia-Idade , Mutação , Adulto JovemRESUMO
BACKGROUND: Despite a high prevalence of angiodysplasia, no specific guidelines are available for the modalities of endoscopic exploration of gastrointestinal (GI) bleeding in von Willebrand disease (VWD). Whether VWD patients could benefit from video capsule endoscopy (VCE) looking for angiodysplasia eligible to endoscopic treatment or at high risk of bleeding is unknown. OBJECTIVES: To assess the diagnostic efficacy for angiodysplasia and the prognostic value of VCE on top of conventional endoscopy in VWD patients with GI bleeding. PATIENTS/METHODS: A survey was sent to the 30 centers of the French-network on inherited bleeding disorders to identify VWD patients referred for endoscopic exploration of GI bleeding from January 2015 to December 2017. Data obtained included patient characteristics, VWD phenotype/genotype, GI bleeding pattern, results of endoscopic investigations, and medical management applied including endoscopic therapy. We assessed by Kaplan-Meier analysis the recurrence-free survival after the first GI bleeding event according to endoscopic categorization and, in patients with angiodysplasia, to the presence of small-bowel localizations on VCE exploration. RESULTS: GI bleeding source localization was significantly improved when including VCE exploration (P < .01), even in patients without history of angiodysplasia (P < .05). Patients with angiodysplasia had more GI bleeding recurrences (P < .01). A lower recurrence-free survival was observed in patients with angiodysplasia (log-rank test, P = .02), and especially when lesions were located in the small bowel (log-rank test, P < .01), even after endoscopic treatment with argon plasma coagulation (log-rank test, P < .01). CONCLUSION: VCE should be more systematically used in VWD patients with unexplained or recurrent GI bleeding looking for angiodysplasia eligible to endoscopic treatment or at high risk of relapse.
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Angiodisplasia , Doenças de von Willebrand , Angiodisplasia/complicações , Angiodisplasia/diagnóstico , Endoscopia , Hemorragia Gastrointestinal/diagnóstico , Humanos , Prognóstico , Doenças de von Willebrand/complicações , Doenças de von Willebrand/diagnósticoRESUMO
BACKGROUND: Obesity and its related illnesses are a major health problem around the world. Saudi Arabia has one of the highest national obesity rates globally; however, it is not easy to intervene to prevent obesity and becoming overweight owing to Saudi Arabia's cultural and social norms, and linguistic barriers. In recent years, there has been an exponential growth in the usage of smartphones and apps in Saudi Arabia. These could be used as a cost-effective tool to facilitate the delivery of behavior-modification interventions for obese and overweight people. There are a variety of health and fitness apps that claim to offer lifestyle-modification tools. However, these do not identify the motivational features required to overcome obesity, consider the evidence-based practices for weight management, or enhance the usability of apps by considering usability attributes. OBJECTIVE: This study aimed to explore the opportunity and the need to develop an Arabic weight-loss app that provides localized content and addresses the issues with existing apps identified here. This study has explained the steps taken to design an Arabic weight-loss app that was developed to facilitate the adjustment of key nutritional and physical activities and behaviors, which considers the social and cultural norms of Saudi Arabia. METHODS: Qualitative studies were conducted with 26 obese Saudi Arabians, who tested the level of usability of 2 weight-loss apps and then provided feedback and recommendations. The app Akser Waznk is an interactive, user-friendly app designed primarily for iPhones. It has several features intended to assist users to monitor and track their food consumption and physical activities. The app provides personalized diet and weight loss advice. Unique features such as Let's Walk are designed to motivate users to walk more. An augmented reality function is implemented to provide information regarding fitness equipment, fruits, and vegetables. The app uses behavior-change techniques to increase activities and healthy behaviors and evidence-informed practices for weight-loss management. The Akser Waznk app considers user privacy and data security by applying a number of guidelines and procedures. RESULTS: The development of the app took 26 months. In all, 7 experts (5 dietitians, and 2 physical activity professionals) evaluated the app's contents. Moreover, 10 potential users (5 men and 5 women) tested the app's level of usability, its features, and performance during a pilot study. They reported that the app's design is interactive, and the motivational features are user-friendly. CONCLUSIONS: Mobile technology, such as mobile apps, has the potential to be an effective tool that facilitates the changing of unhealthy lifestyle behaviors within the Saudi community. To be successful, the target group, the usability, motivational features, and social and cultural norms must be considered.
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A healthy and active lifestyle can significantly improve the well-being and quality of life; however, some elderly people struggle to stay motivated and engaged with any form of exercise. The project Elaine (Elderly, AI and New Experiences) addresses this problem by seeking to improve the quality of life of the elderly through exergames. Currently, the project explores a novel approach in the field of health informatics called asynchronous exergaming. This approach, a new trend in games in the health domain, allows the elderly to workout at their own pace, and in their own time, with their physical activity linked asynchronously to a game. This paper presents the study protocol for Solitaire Fitness, a new asynchronous exergame developed by the team. The game aims at increasing the motivation of the elderly to engage in physical exercise whilst helping to maintain their cognitive abilities. It also describes the protocol for the trial. The result of this research has the potential to benefit elderly that need nudging to be motivated to exercise, health care providers treating people with sedentary lifestyles and researchers investigating ways to encourage the elderly to exercise.
Assuntos
Exercício Físico , Jogos de Vídeo , Idoso , Envelhecimento , Humanos , Estilo de Vida , Qualidade de Vida , Comportamento SedentárioRESUMO
Essentials A novel fibrinogen concentrate was evaluated in patients with congenital fibrinogen deficiency. An open-label, phase 2-3 trial studied pharmacology, efficacy, and safety in patients >6 years. The product offers safe and effective therapy in the treatment and prophylaxis of bleeding. Data in recovery show the need of adjusted treatment and further investigation in children. SUMMARY: Background Single-factor replacement therapy is considered the most suitable treatment option for hereditary fibrinogen deficiency. A triple-secured plasma-derived human fibrinogen product was developed to increase the safety of the former fibrinogen concentrate. Objectives This non-randomized, open-label, prospective study investigated pharmacokinetics, efficacy, and safety of a novel fibrinogen concentrate (FibCLOT® /CLOTTAFACT® LFB, France) in inherited deficiency. Patients/Methods Fourteen patients ≥40 kg received fibrinogen concentrate for pharmacology and 16 ≥ 23 kg received treatment for bleeding or surgery. Each treatment was followed by a 3-week safety observation period. Key outcomes included number of infusions, dose, bleeding control, daily assessment, hemoglobin, blood loss, transfusions, and physicians' global assessment of response. Results Incremental recovery was 2.35 mg mL-1 per mg kg-1 and maximal concentration 1.41 g L-1 (geometric mean) after 0.060 g kg-1 infusion in 14 afibrinogenemic patients. Terminal half-life was 69.3 h (non-compartmental analysis). The maximum clot firmness was increased by a mean of 10.3 mm from baseline to maximal effect. Sixteen patients participated to the efficacy phase: 32 bleeding episodes were treated in 9 patients, and 15 patients underwent 38 surgical/invasive procedures. All patients achieved appropriate hemostasis: response to treatment was successful in all bleeds (95% CI, 0.89-1.00) and procedures (95% CI, 0.91-1.00). Most (94%) bleeds were controlled with a single infusion (median 0.050 g kg-1 ). Two patients experienced asymptomatic distal venous thromboses identified by systematic ultrasound. Conclusion FibCLOT® /CLOTTAFACT® showed a pharmacokinetic profile comparable to that of other fibrinogen concentrates and provides safe and clinically effective substitution therapy for fibrinogen-deficient patients.
Assuntos
Afibrinogenemia/tratamento farmacológico , Fibrinogênio/administração & dosagem , Hemorragia/tratamento farmacológico , Hemostasia/efeitos dos fármacos , Hemostáticos/administração & dosagem , Adolescente , Adulto , Afibrinogenemia/sangue , Afibrinogenemia/congênito , Afibrinogenemia/diagnóstico , Fatores Etários , Criança , Feminino , Fibrinogênio/efeitos adversos , Fibrinogênio/farmacocinética , Hemorragia/sangue , Hemorragia/congênito , Hemorragia/diagnóstico , Hemostáticos/efeitos adversos , Hemostáticos/farmacocinética , Humanos , Masculino , Estudos Prospectivos , Resultado do Tratamento , Adulto JovemRESUMO
5-Hydroxytryptamine (serotonin) (5-HT) is a neurotransmitter with both central and peripheral functions, including the modulation of mood, appetite, hemodynamics, gastrointestinal (GI) sensation, secretion, and motility. Its synthesis is initiated by the enzyme tryptophan hydroxylase (TPH). Two isoforms of TPH have been discovered: TPH1, primarily expressed in the enterochromaffin cells of the gastrointestinal tract, and TPH2, expressed exclusively in neuronal cells. Mice lacking Tph1 contain little to no 5-HT in the blood and GI tract while maintaining normal levels in the brain. Because GI 5-HT is known to play important roles in normal and pathophysiology, we set out to discover and characterize novel compounds that selectively inhibit biosynthesis of GI 5-HT. Here, we describe two of a series of these inhibitors that are potent for TPH activity both in biochemical and cell-based assays. This class of compounds has unique properties with respect to pharmacokinetic and pharmacodynamic effects on GI serotonin production. Similar to the Tph1 knockout results, these TPH inhibitors have the ability to selectively reduce 5-HT levels in the murine GI tract without affecting brain 5-HT levels. In addition, administration of these compounds in a ferret model of chemotherapy-induced emesis caused modest reductions of intestinal serotonin levels and a decreased emetic response. These findings suggest that GI-specific TPH inhibitors may provide novel treatments for various gastrointestinal disorders associated with dysregulation of the GI serotonergic system, such as chemotherapy-induced emesis and irritable bowel syndrome.