12 research outputs found

    Actividad anti-inflamatoria de compuestos sesquiterpénicos aislados de Psacalium decompositum (Gray) Rob. & Brett. en modelo de TPA y en la línea celular de macrófagos RAW 264.7

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    Inflammation is an adaptative response, triggered by various harmful stimuli, its aim is to eliminate injurious agents, as well as the restoration and normal functioning of the affected tissue or organ. However, this response can be altered and produce permanent damage, which often have pathological consequences, like cardiovascular, respiratory, digestive or autoimune diseases. For this reason, there is a large number of anti-inflammatory drugs nowadays, such as esteroideal and non-steroideal drugs, they have pharmacological action mainly by blocking initiation and amplification mediators of the inflammatory response. It has been reported, nevertheless, that the consumption of anti-inflammatory drugs for long periods may cause adverse effects, which brings us to the pursuit of new anti-inflammatory compounds. Mexico is a country with a very large variety of plant species with diverse medicinal uses, an example of this is Psacalium decompositum, a plant with diverse uses in traditional medicine, among which its antidiabetical and anti-inflammatory properties stand out. Among the main compounds that have been identified in P. decompositum, are cacalol and a rich in fructooligosaccharides (FOS) fraction. It remains unknown if this compounds are responsible for anti-inflammatory action.Objective. Determine the anti-inflammatory activity of the cacalol, cacalol acetate and the fraction rich in fructooligosaccharides of Psacalium decompositum in RAW 264.7 macrophages. Materials and methods. For the in vivo model, male mice of the CD-1 strain were used to induce topical inflammation by the administration of TPA (2.5 μg) in the ear and subsequently the compounds cacalol, cacalol acetate and the fraction FOS as well as the hexanic extract and the aqueous extract (1 μg/ear). For the in vitro study, RAW 264.7 macrophages were cultured and stimulated with LPS (5 μg/mL) to induce an inflammatory response an inflammatory response and subsequently treated at different times (15, 30, 60, 90 and 240 minutes) with the compounds cacalol (100 µM), cacalol acetate (100 µM), the FOS fraction (1µg/mL) and the aqueous extract (10 μg/mL), using dexamethasone as positive control (50 µM). After the treatments, the expression of the TNF- α, IL-6, IL-1β, IL-10 and COX.2 mRNAs was assessed by qPCR, cytokine concentrations (TNF-α, IL-6, IL-1β, IL-10) were measured in the culture medium by the ELISA method and phosphorylation of p65 subunit of NF-κB by the ELISA method. Results. The results showed that both cacalol and cacalol acetate significantly inhibited the development of ear edema by up to 40% compared to the control (only treated with vehicle), unlike the hexanic extract that inhibited 39.2% and the FOS fraction which only inhibited it by 28%. Furthermore, in RAW 264.7 macrophages the compounds significantly decreased the expression of TNF-α, IL-6 and IL-1β, as well as their concentrations in the culture medium. No changes were detected in the expression or concentration of IL-10. The concentrations of the phosphorylated p65 subunit decreased with the extracts and with the isolated compounds.Conclusion. Cacalol, cacalol acetate, as well as the FOS fraction and hexane and aqueous extract, reduce inflammation through the modulation of inflammatory cytokines, suppressing the pro-inflammatory ones; however, they do not modify anti-inflammatory citokines. This effects could be involved in the inhibition of the NF-κB pathway as it decreases the level of the phosphorylated p65 subunit. Even so, it is necessary to continue the study with the transcription factors involved in the regulation of these cytokines.La inflamación es una repuesta adaptativa que se desencadena por diversos estímulos nocivos y cuyo objetivo es la eliminación de un agente dañino, así como el restablecimiento y funcionamiento normal del tejido u órgano afectado. Esta respuesta puede alterarse produciendo daño permanente y consecuencias patológicas asociadas con enfermedades cardiovasculares, respiratorias, digestivas o autoinmunes. Hoy en día existe un gran número de fármacos antiinflamatorios, esteroideos y no esteroideos, los cuales ejercen su acción farmacológica mediante el bloqueo de moléculas mediadoras de la iniciación y amplificación de la respuesta inflamatoria. Sin embargo, se ha reportado que, independientemente de la naturaleza esteroidea o no esteroidea de los fármacos antiinflamatorios, cuando se consumen por periodos largos pueden generar efectos adversos, lo cual justifica la búsqueda de nuevos compuestos antiinflamatorios. México cuenta con gran variedad de especies vegetales con diferentes usos medicinales. Psacalium decompositum se usa en la medicina tradicional como antidiabética y antiinflamatoria. Entre los principales compuestos identificados en P. decompositum se encuentran el cacalol y una fracción rica en fructooligosacáridos (FOS), los cuales se han reportado con actividad antinflamatoria en modelos de inflamación aguda y en modelo de síndrome metabólico respectivamente. Sin embargo, se desconoce el mecanismo por el cual estos compuestos ejercen su acción antiinflamatoria.Objetivo. Determinar la actividad antiinflamatoria del cacalol, acetato de cacalol y la fracción rica en fructooligosacáridos de Psacalium decompositum en macrófagos RAW 264.7. Materiales y métodos. Para el modelo in vivo se utilizaron ratones machos de la cepa CD-1, a los que se les indujo inflamación tópica mediante la administración de TPA (2.5 µg) en la oreja; posteriormente se administraron los compuestos cacalol, acetato de cacalol y la fracción FOS, así como el extracto hexánico y el extracto acuoso (1 µg/oreja). Para el estudio in vitro se cultivaron macrófagos de la línea RAW 264.7, los cuales fueron estimulados con LPS (5 µg/mL) para inducir una respuesta inflamatoria; posteriormente se trataron por diferentes tiempos (15, 30, 60, 90 y 240 minutos), con los compuestos cacalol (100 µM), acetato de cacalol (100 µM) y la fracción FOS (1 µg/mL), así como el extracto hexánico (10 µg/mL) y el extracto acuoso (10 µg/mL), utilizando como control positivo dexametasona (50 µM). Al final de los tratamientos la expresión del ARNm de TNF-α, IL-6, IL-1β, IL-10 y COX-2 se midió por qPCR; las concentraciones de las citocinas TNF- α, IL-6, IL-1β e IL-10 en el medio de cultivo y la fosforilación de la subunidad p65 de NF-κB se determinaron por el método de ELISA. Resultados. Los resultados mostraron que tanto el cacalol como su acetato inhibieron significativamente el desarrollo del edema auricular en un 40% con respecto al control (vehículo), el extracto hexánico lo inhibió en un 39.2% y la fracción FOS en un 28%. En macrófagos RAW 264.7 los compuestos disminuyeron significativamente la expresión de TNF-α, IL-6 e IL-1β, así como sus concentraciones en el cultivo. No se detectaron cambios en la expresión ni en la concentración de IL-10. La concentración de la subunidad p65 fosforilada disminuyó con los extractos y con los compuestos aislados. Conclusión. El cacalol, el acetato de cacalol, la fracción FOS y los extracto hexánico y acuoso redujeron la inflamación y suprimieron las concentraciones de citocinas proinflamatorias, sin afectar las antiinflamatorias, lo cual podría estar relacionado con inhibición de la vía NF-κB, ya que los nivel de la subunidad de p65 fosforilada fueron reducidos. Es necesario continuar el estudio de losfactores de transcripción implicados en la regulación de dichas citocinas

    Acción antiinflamatoria del acetato de cacalol sobre la vía de señalización de NF-KB activada por LPS

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    La inflamación es una respuesta biológica natural en donde interaccionan factores solubles celulares, diferentes poblaciones celulares del sistema inmune innato y adaptativo y otras poblaciones celulares como las células endoteliales. Este proceso puede surgir en cualquier tejido vascularizado en respuesta a estímulos externos (lesión traumática o infecciones) o internos (reacciones autoinmunes) que han lesionado su integridad. El proceso normalmente al control y resolución de la infección, a la recuperación tisular y la curación. La inflamación parece estar diseñada para funcionar en microambientes tisulares de forma rápida y aislada. Las vías de señalización intracelular que activan a las células que participan en la reacción inflamatoria requieren de la activación del sistema del control transcripcional NF-ΚB. En ocasiones esta respuesta puede alterarse produciendo daño permanente y consecuencias patológicas asociadas con enfermedades cardiovasculares, respiratorias, digestivas o autoinmunes. Un caso extremo se presenta durante el choque séptico en donde deja de haber procesos locales y la inflamación se convierte en un proceso sistémico. La presentación de formas descontroladas de la inflamación han llevado a la búsqueda y desarrollo de compuestos capaces de interferir con el proceso inflamatorio diferentes niveles. Existe un gran número de fármacos antiinflamatorios, esteroideos y no esteroideos, los cuales ejercen su acción farmacológica mediante el bloqueo de moléculas mediadoras de la iniciación y amplificación de la respuesta inflamatoria. Sin embargo, se ha reportado que, independientemente de la naturaleza esteroidea o no esteroidea de los fármacos antiinflamatorios, cuando se consumen por periodos largos pueden generar efectos adversos, lo cual justifica la búsqueda de nuevos compuestos antiinflamatorios. México cuenta con gran variedad de especies vegetales con diferentes usos medicinales. Psacalium decompositum se usa en la medicina tradicional como antidiabética y antiinflamatoria. Entre los principales compuestos identificados en P. decompositum se encuentra el cacalol el cual se ha reportado que presenta actividad antinflamatoria en modelos de inflamación aguda. Así mismo se ha reportado que el compuesto es muy fácil de oxidar, pero, al acetilarlo en su grupo OH su estructura se vuelve es más estable, produciendo acetato de cacalol (CA). Sin embargo, poco se ha avanzado en el conocimiento de las actividades de este compuesto y del mecanismo de acción antiinflamatoria que podría presentar.Inflammation is a natural biological response in which soluble cellular factors, different populations of the innate and adaptive immune response in others cells type, such the endothelial cells interact. This process can arise in any vascularized tissue in response to external stimuli (traumatic injury or infections) or internal stimuli (autoimmune reactions) that have damaged its integrity. Inflammation is designed to function within the tissue-microenvironment to provide a local and insolated response. The signaling pathway activated in this cells populations is NF-ΚB system. The process normally leads to recovery from infection and healing. Sometimes this response can be altered, producing permanent damage and pathological consequences associated with cardiovascular, respiratory, digestive or autoimmune diseases. A extreme case occurs during septic shock were the local effect becomes systemic. There is a large number of steroidal and non-steroidal anti-inflammatory drugs, which exert their pharmacological action by blocking molecules that mediate the initiation and amplification of the inflammatory response. However, it has been reported that, regardless of the steroidal or non-steroidal nature of anti-inflammatory drugs, when consumed for long periods of time they can generate adverse effects, which justifies the search for new anti-inflammatory compounds. Mexico has a great variety of plant species with different medicinal uses. Psacalium decompositum is used in traditional medicine as both an antidiabetic and anti-inflammatory. Among the identified compounds in P. decompositum the main is cacalol, which has been reported to have anti-inflammatory activity in acute inflammation models. Likewise, it has been reported that the compound is very easy to oxidize, but, when acetylated in its OH group, its structure becomes more stable, producing cacalol acetate (CA). However, little progress has been made in understanding the activities of this compound and the mechanism of anti-inflammatory action that it could present

    Detecção de Trypanosoma cruzi em tecido e sangue murinos por PCR convencional e em tempo real

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    El objetivo del presente trabajo fue comparar la detección de ADN de Trypanosoma cruzi mediante PCR en tiempo real (qPCR) y PCR convencional en sangre periférica (n=25) y músculo esquelético (n=20) de ratones tratados con drogas tripanomicidas luego de 6 meses post-tratamiento. En las muestras de sangre se detectaron un total de 7 positivas por qPCR, mientras que por PCR convencional sólo se detectaron 2. En músculo esquelético, 15 muestras fueron positivas por qPCR y 3 por PCR convencional. Los resultados obtenidos demuestran que la fuerza de concordancia es débil entre las técnicas de PCR utilizadas para la detección de ADN de T. cruzi (k=0,37; 49% positivas por qPCR vs. 11% por PCR convencional, p=0,0001). En las muestras de sangre, los valores diagnósticos de qPCR con respecto a la PCR convencional fueron: 100% sensibilidad; 78% especificidad; 30% VPP; 100% VPN; 4,6 RVP; 0 RVN. Para las muestras de músculo esquelético se obtuvieron los siguientes valores diagnósticos de qPCR: 100% sensibilidad; 29% especificidad; 20% VPP; 100% VPN; 1,4 RVP; 0 RVN. Ambas técnicas fueron igualmente sensibles en el rango de mediana-alta concentración, pero qPCR fue más efectiva para detectar bajas cargas parasitarias, en particular en las muestras de tejido.The aim of this work was to compare detection of Trypanosoma cruzi DNA by real time (qPCR) and conventional PCR in peripheral blood (n=25), and skeletal muscle (n=20) of mice treated with trypanocidal compounds after 6 months post-treatment. A total of 7 blood samples were positive by qPCR; whereas, by conventional PCR only 2 were detected. In skeletal muscle, 15 samples were regarded positive by qPCR and 3 by conventional PCR. These results showed a weak concordance strength among PCR techniques employed to detect T. cruzi DNA in the studied samples (k=0.37; 49% positives by qPCR vs. 11% by conventional PCR, p=0.0001). In blood samples, qPCR diagnostic values in comparison with conventional PCR were: 100% sensibility; 78% specificity; 30% PPV; 100% NPV; 4.6 PVR; 0 NVR. For skeletal muscle samples, qPCR diagnostic values were: 100% sensibility; 29% specificity; 20% PPV; 100% NPV; 1.4 PVR; 0 NVR. Both techniques were equally sensitive in the medium-high concentration range, but qPCR was more effective to detect low parasitic burden, particularly in skeletal muscle samples.O objetivo deste estudo foi comparar a detecção de DNA de Trypanosoma cruzi por PCR em tempo real (qPCR) e PCR convencional no sangue periférico (N=25) e músculo esquelético (N= 20) de camundongos tratados com medicamentos tripanomicidas depois de 6 meses de pós-tratamento. Nas amostras de sangue foi detectado um total de sete positivas por qPCR; enquanto que apenas foram encontradas 2 por PCR convencional. No músculo esquelético, 15 amostras foram positivas por qPCR e 3 por PCR convencional. Os resultados mostram que a força de concordância é fraca entre as técnicas de PCR utilizadas para a detecção de DNA de T. cruzi (k=0,37, 49% positivas por qPCR vs. 11% para a PCR convencional, p=0,0001). Nas amostras de sangue, os valores diagnósticos de qPCR em relação a PCR convencional foram de 100% sensibilidade; 78% de especificidade; 30% de VPP; 100% VPN; 4,6 RVP; 0 RVN. Para as amostras de músculo esquelético, os seguintes valores diagnósticos de qPCR foram obtidos: 100% sensibilidade; 29% de especificidade; 20% de VPP; 100% VPN; 1,4 RVP; 0 RVN. Ambas as técnicas são igualmente sensíveis na faixa de concentração média-alta, mas qPCR foi mais eficaz na detecção de baixas cargas parasitárias, especialmente em amostras de tecido.Fil: Davies, Carolina. Consejo Nacional de Investigaciones Científicas y Técnicas. Centro Científico Tecnológico Salta. Instituto de Patología Experimental; ArgentinaFil: Poma, Hugo Ramiro. Consejo Nacional de Investigaciones Científicas y Técnicas. Centro Científico Tecnológico Salta. Instituto de Investigación Para la Industria Química (i); ArgentinaFil: Cardozo, Rubén Marino. Provincia de Salta. Ministerio de Salud Publica; ArgentinaFil: Mora, Maria Celia. Consejo Nacional de Investigaciones Científicas y Técnicas. Centro Científico Tecnológico Salta. Instituto de Patología Experimental; ArgentinaFil: Ramos, Federico. Consejo Nacional de Investigaciones Científicas y Técnicas. Centro Científico Tecnológico Salta. Instituto de Patología Experimental; ArgentinaFil: Rajal, Verónica Beatriz. Consejo Nacional de Investigaciones Científicas y Técnicas. Centro Científico Tecnológico Salta. Instituto de Investigación Para la Industria Química (i); ArgentinaFil: Basombrio, Miguel Angel Manuel. Consejo Nacional de Investigaciones Científicas y Técnicas. Centro Científico Tecnológico Salta. Instituto de Patología Experimental; Argentin

    Machine Learning Improves Risk Stratification in Myelofibrosis: An Analysis of the Spanish Registry of Myelofibrosis

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    Myelofibrosis (MF) is a myeloproliferative neoplasm (MPN) with heterogeneous clinical course. Allogeneic hematopoietic cell transplantation remains the only curative therapy, but its morbidity and mortality require careful candidate selection. Therefore, accurate disease risk prognostication is critical for treatment decision-making. We obtained registry data from patients diagnosed with MF in 60 Spanish institutions (N = 1386). These were randomly divided into a training set (80%) and a test set (20%). A machine learning (ML) technique (random forest) was used to model overall survival (OS) and leukemia-free survival (LFS) in the training set, and the results were validated in the test set. We derived the AIPSS-MF (Artificial Intelligence Prognostic Scoring System for Myelofibrosis) model, which was based on 8 clinical variables at diagnosis and achieved high accuracy in predicting OS (training set c-index, 0.750; test set c-index, 0.744) and LFS (training set c-index, 0.697; test set c-index, 0.703). No improvement was obtained with the inclusion of MPN driver mutations in the model. We were unable to adequately assess the potential benefit of including adverse cytogenetics or high-risk mutations due to the lack of these data in many patients. AIPSS-MF was superior to the IPSS regardless of MF subtype and age range and outperformed the MYSEC-PM in patients with secondary MF. In conclusion, we have developed a prediction model based exclusively on clinical variables that provides individualized prognostic estimates in patients with primary and secondary MF. The use of AIPSS-MF in combination with predictive models that incorporate genetic information may improve disease risk stratification

    Effectiveness of an intervention for improving drug prescription in primary care patients with multimorbidity and polypharmacy:Study protocol of a cluster randomized clinical trial (Multi-PAP project)

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    This study was funded by the Fondo de Investigaciones Sanitarias ISCIII (Grant Numbers PI15/00276, PI15/00572, PI15/00996), REDISSEC (Project Numbers RD12/0001/0012, RD16/0001/0005), and the European Regional Development Fund ("A way to build Europe").Background: Multimorbidity is associated with negative effects both on people's health and on healthcare systems. A key problem linked to multimorbidity is polypharmacy, which in turn is associated with increased risk of partly preventable adverse effects, including mortality. The Ariadne principles describe a model of care based on a thorough assessment of diseases, treatments (and potential interactions), clinical status, context and preferences of patients with multimorbidity, with the aim of prioritizing and sharing realistic treatment goals that guide an individualized management. The aim of this study is to evaluate the effectiveness of a complex intervention that implements the Ariadne principles in a population of young-old patients with multimorbidity and polypharmacy. The intervention seeks to improve the appropriateness of prescribing in primary care (PC), as measured by the medication appropriateness index (MAI) score at 6 and 12months, as compared with usual care. Methods/Design: Design:pragmatic cluster randomized clinical trial. Unit of randomization: family physician (FP). Unit of analysis: patient. Scope: PC health centres in three autonomous communities: Aragon, Madrid, and Andalusia (Spain). Population: patients aged 65-74years with multimorbidity (≥3 chronic diseases) and polypharmacy (≥5 drugs prescribed in ≥3months). Sample size: n=400 (200 per study arm). Intervention: complex intervention based on the implementation of the Ariadne principles with two components: (1) FP training and (2) FP-patient interview. Outcomes: MAI score, health services use, quality of life (Euroqol 5D-5L), pharmacotherapy and adherence to treatment (Morisky-Green, Haynes-Sackett), and clinical and socio-demographic variables. Statistical analysis: primary outcome is the difference in MAI score between T0 and T1 and corresponding 95% confidence interval. Adjustment for confounding factors will be performed by multilevel analysis. All analyses will be carried out in accordance with the intention-to-treat principle. Discussion: It is essential to provide evidence concerning interventions on PC patients with polypharmacy and multimorbidity, conducted in the context of routine clinical practice, and involving young-old patients with significant potential for preventing negative health outcomes. Trial registration: Clinicaltrials.gov, NCT02866799Publisher PDFPeer reviewe

    Burnout among surgeons before and during the SARS-CoV-2 pandemic: an international survey

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    Background: SARS-CoV-2 pandemic has had many significant impacts within the surgical realm, and surgeons have been obligated to reconsider almost every aspect of daily clinical practice. Methods: This is a cross-sectional study reported in compliance with the CHERRIES guidelines and conducted through an online platform from June 14th to July 15th, 2020. The primary outcome was the burden of burnout during the pandemic indicated by the validated Shirom-Melamed Burnout Measure. Results: Nine hundred fifty-four surgeons completed the survey. The median length of practice was 10 years; 78.2% included were male with a median age of 37 years old, 39.5% were consultants, 68.9% were general surgeons, and 55.7% were affiliated with an academic institution. Overall, there was a significant increase in the mean burnout score during the pandemic; longer years of practice and older age were significantly associated with less burnout. There were significant reductions in the median number of outpatient visits, operated cases, on-call hours, emergency visits, and research work, so, 48.2% of respondents felt that the training resources were insufficient. The majority (81.3%) of respondents reported that their hospitals were included in the management of COVID-19, 66.5% felt their roles had been minimized; 41% were asked to assist in non-surgical medical practices, and 37.6% of respondents were included in COVID-19 management. Conclusions: There was a significant burnout among trainees. Almost all aspects of clinical and research activities were affected with a significant reduction in the volume of research, outpatient clinic visits, surgical procedures, on-call hours, and emergency cases hindering the training. Trial registration: The study was registered on clicaltrials.gov "NCT04433286" on 16/06/2020

    Actas de las V Jornadas ScienCity 2022. Fomento de la Cultura Científica, Tecnológica y de Innovación en Ciudades Inteligentes

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    ScienCity es una actividad que viene siendo continuada desde 2018 con el objetivo de dar a conocer los conocimientos y tecnologías emergentes siendo investigados en las universidades, informar de experiencias, servicios e iniciativas puestas ya en marcha por instituciones y empresas, llegar hasta decisores políticos que podrían crear sinergias, incentivar la creación de ideas y posibilidades de desarrollo conjuntas, implicar y provocar la participación ciudadana, así como gestar una red internacional multidisciplinar de investigadores que garantice la continuación de futuras ediciones. En 2022 se recibieron un total de 48 trabajos repartidos en 25 ponencias y 24 pósteres pertenecientes a 98 autores de 14 instituciones distintas de España, Portugal, Polonia y Países Bajos.Fundación Española para la Ciencia y la Tecnología-Ministerio de Ciencia, Innovación y Universidades; Consejería de la Presidencia, Administración Pública e Interior de la Junta de Andalucía; Estrategia de Política de Investigación y Transferencia de la Universidad de Huelva; Cátedra de Innovación Social de Aguas de Huelva; Cátedra de la Provincia; Grupo de investigación TEP-192 de Control y Robótica; Centro de Investigación en Tecnología, Energía y Sostenibilidad (CITES

    LC-MS Fingerprinting Development for Standardized Precipitate from <i>Agastache mexicana</i>, Which Induces Antihypertensive Effect through NO Production and Calcium Channel Blockade

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    The aim of this work was to evaluate the vasorelaxant and antihypertensive effects of a standardized precipitate of the hydroalcoholic extract from Agastache mexicana (PPAm), comprising ursolic acid, oleanolic acid, acacetin, luteolin and tilianin, among others. In the ex vivo experiments, preincubation with L-NAME (nonspecific inhibitor of nitric oxide synthases) reduced the relaxation induced by PPAm; nevertheless, preincubation with indomethacin (nonspecific inhibitor of cyclooxygenases) did not generate any change in the vasorelaxation, and an opposed effect was observed to the contraction generated by CaCl2 addition. Oral administration of 100 mg/kg of PPAm induced a significant acute decrease in diastolic (DBP) and systolic (SBP) blood pressure in spontaneously hypertensive rats, without changes in heart rate. Additionally, PPAm showed a sustained antihypertensive subacute effect on both DBP and SBP for 10 days compared to the control group. On the other hand, human umbilical vein cells treated with 10 µg/mL of PPAm showed a significant reduction (p Am induces a significant antihypertensive effect in acute- and subacute-period treatments, due to its direct vasorelaxant action on rat aortic rings through NO production and Ca2+ channel blockade

    Presence of acute and chronic renal failure in patients with paroxysmal nocturnal hemoglobinuria: results of a retrospective analysis from the Spanish PNH Registry

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    Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening blood disease. With the advent of eculizumab treatment, renal function has substantially improved, although no data from real-world clinical practice are available. An observational, retrospective, multicenter study was conducted in Spain on clinical data obtained from outpatient visits of patients with PNH (Spanish PNH Registry) who had experienced acute (ARF) or chronic (CRF) renal failure. Of the 128 patients registered (April 2014), 60 were diagnosed with classic PNH. Twenty-seven (45.0%) patients with a mean age of 48.5 (±16.2) years had renal failure, ARF or CRF, and were included in this study. Near half of the patients (n = 13; 48.1%) presented with ARF alone, 33.3% (n = 9) had CRF with episodes of ARF, while 18.5% (n = 5) were diagnosed with CRF alone. For patients with diagnosis of PNH and renal failure (n = 27), the median time to the first ARF episode was 6.5 (CI 95%; 2.2, 14.9) years, whereas the median to the diagnosis of CRF was 14.5 (CI 95%; 3.8, 19.2) years after the diagnosis of PNH. Patients with ARF (n = 22) were treated with eculizumab and did not experience new episodes of ARF, except for one patient with sepsis. Of the patients with CRF, two received treatment without experiencing further episodes of ARF. Sixteen patients who completed treatment (11 with ARF and 5 with ARF + CRF) recovered from the episode of ARF or from CRF. Of the remaining patients treated with eculizumab, one patient improved from stages III to II, three patients stabilized without showing disease progression, and one patient progressed from stages III to IV. Treatment with eculizumab in PNH patients has beneficial effects on renal function, preventing ARF and progression to CRF.This study was funded by Alexion Pharma Spain, SL (Barcelona, Spain).Peer reviewe
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