Benefits of rIX-FP prophylaxis in patients with Haemophilia B: real-world evidence from a Spanish reference centre

Haemophilia B; pharmacokinetic; Real world evidenceHemofilia B; Farmacocinética; Evidencia del mundo realHemofília B; Farmacocinètica; Evidència del món realStandard FIX prophylaxis for PWHB require frequent injections, which has led to the development of extended half-life products like rIX-FP (albutrepenonacog alfa) that has shown good efficacy in clinical studies. This ambispective study aims to report a real-world experience with rIX-FP in a Spanish centre with PWHB who switched from SHL-FIX or began prophylaxis with rIX-FP. Five PWHB were included in this study, Four PTP switched to rIX-FP with prophylaxis every 7 days whilst one PUP started with an every-14-days regimen. 3 PTPs extended their dosing intervals to every 14 days or every 21 days. In all PTPs, median annualized spontaneous and joint bleeding rates were maintained at 0.00 and median (range) of ABR was 0.92 (0.00–2.77) after switch to rIX-FP. Mean trough level with previous product was 3.68% (SD = 2.06), while it was 7.08% (SD = 3) with all rIX-FP dosing intervals. After switching to rIX-FP, all PTP reduced their annual infusion rate between 50 and 84% and their annual FIX consumption by 61% (59–67%). This is the first reported real-world experience with albutrepenonacog alfa in a small cohort in Spain and demonstrates good bleeding control together with a reduction of the infusion rate, factor consumption and higher through factor level than previous treatment

Manejo de la obesidad y diabetes mellitus tipo 2 con cirugía bariátrica. Una revisión sistemática

Introduction: Bariatric surgery is the surgical modification of the stomach, intestine or both to lose weight, which leads to remission of DM2, in patients with severe obesity. Objective: To describe the techniques used in bariatric surgery for the management of type 2 diabetes mellitus and obesity. Specific objectives : 1) Compare results between bariatric surgery versus lifestyle change without surgery in patients with diabetes and obesity. 2 ) Knowing the long-term benefits of bariatric surgery. Materials and methods: For the present systematic review scientific medical articles published in the last 5 years were used, following the inclusion and exclusion criteria established, The databases Pubmed, IntechOpen, ScienceDirect, Mendeley, Wiley Online Library, Google Scholar, with the title search: bariatric surgery in the remission of type 2 diabetes mellitus were used as reference for the preparation of this manuscript. Results: Bariatric surgery is effective in patients with type 2 diabetes mellitus with body mass index (BMI) of 30 to 35 kg/m 2, in the remission of diabetes mellitus showed better results than changes in lifestyle and diet. According to the results obtained in the randomization of patients for bariatric surgery, compared with patients who had only lifestyle intervention, bariatric surgery showed a 70% resolution of nonalcoholic steatohepatitis. Conclusions: Bariatric and metabolic surgery in the management of type 2 diabetes with severe obesity, has great utility and benefit in the group of patients suffering from this pathology, obtaining good results according to the surgical technique used, whose remission is higher, compared to the change in lifestyle, also reduces long-term mortality, proving very useful in patients with class III or morbid obesity, and in patients with class II and class I obesity suffering from decompensated diabetesIntroducción: La cirugía bariátrica es la modificación quirúrgica del estómago, el intestino o ambos para perder peso, la cual conduce a remisión de la DM2, en los pacientes con obesidad severa.  Objetivo General: Describir las técnicas utilizadas en cirugía bariátrica para el manejo de la diabetes mellitus tipo 2 y obesidad. Objetivos específicos : 1) Comparar los resultados entre cirugía bariátrica versus cambio del estilo de vida sin cirugía en pacientes con diabetes y obesidad. 2 )  Conocer los  beneficios a largo plazo de la cirugía bariátrica. Materiales y métodos: Para la presente revisión sistemática se utilizaron artículos médicos científicos publicados en los últimos 5 años, siguiendo los criterios de inclusión y exclusión establecidos, para la elaboración del presente manuscrito se utilizó como referencia las bases de datos Pubmed, IntechOpen, ScienceDirect, Mendeley, Wiley Online Library, Google Académico, con el título de búsqueda: cirugía bariátrica en la remisión de la diabetes mellitus tipo 2. Resultados:  La cirugía bariátrica es efectiva en pacientes con diabetes mellitus tipo 2 con índice de masa corporal (IMC) de 30 a 35 kg/m 2, en la remisión de la diabetes mellitus mostro mejores resultados que los cambios de los hábitos de la vida y la dieta. De acuerdo a los resultados obtenidos en la aleatorización de los pacientes para cirugía bariátrica, comparada con los pacientes que tuvieron solo intervención en el estilo de vida, la cirugía bariátrica demostró un 70% de resolución de la esteatohepatitis no alcohólica.Conclusiones: La cirugía bariátrica y metabólica en el manejo de la diabetes tipo 2 con obesidad severa, tiene gran utilidad y beneficio en el grupo de pacientes que padecen esta patología, obteniéndose buenos resultados de acuerdo a la técnica quirúrgica empleada, cuya remisión es superior, comparado con  el cambio en el  estilo de vida, además reduce la mortalidad a largo plazo, demostrando  gran utilidad en los pacientes con obesidad clase III o mórbida, y en los pacientes con obesidad clase II y clase I que padezcan de diabetes descompensad

Clinical Efficacy and Safety of Fanhdi^®, a Plasma-Derived VWF/Factor VIII Concentrate, in von Willebrand Disease in Spain: A Retrospective Study

Objective: To evaluate the efficacy and safety of a plasma-derived factor VIII concentrate containing von Willebrand Factor (pdVWF/FVIII) in standard clinical practice in von Willebrand Disease (VWD) patients. Methods: A retrospective, multicentric, observational study of VWD patients treated with Fanhdi®, a pdVWF/FVIII concentrate, from January 2011 to December 2017 was conducted at 14 centers in Spain. Efficacy and safety were evaluated for acute bleeding episodes, for prevention of bleeding in surgeries, and for secondary long-term prophylaxis. Results: Seventy-two eligible patients, type 1, 2, 3 VWD (25%/38.9%/36.1%) were treated for spontaneous and traumatic bleeding (140 episodes, n = 41 patients), to prevent surgical bleeding (69 episodes, n = 43 patients); and for secondary long-term prophylaxis (18 programs, n = 13 patients). Replacement therapy with pdVWF/FVIII showed an excellent to good clinical efficacy in 96.7% of the bleeding episodes, 100% during surgical procedures and 100% during prophylaxis. No adverse events (AEs), nor serious AEs related to the product were observed. Conclusions: Fanhdi® was effective, safe and well tolerated in the management of bleeding episodes, the prevention of bleeding during surgeries, and for secondary long-term prophylaxis in VWD patientsThe author(s) disclosed receipt of the followingfinancial support forthe research, authorship, and/or publication of this article: This workwas supported by Grifols, manufacturer of the pdVWF/FVIII,Fanhdi

Clinical Efficacy and Safety of Fanhdi ®, a Plasma-Derived VWF/Factor VIII Concentrate, in von Willebrand Disease in Spain : A Retrospective Study

UDHEBRONTo evaluate the efficacy and safety of a plasma-derived factor VIII concentrate containing von Willebrand Factor (pdVWF/FVIII) in standard clinical practice in von Willebrand Disease (VWD) patients. A retrospective, multicentric, observational study of VWD patients treated with Fanhdi ®, a pdVWF/FVIII concentrate, from January 2011 to December 2017 was conducted at 14 centers in Spain. Efficacy and safety were evaluated for acute bleeding episodes, for prevention of bleeding in surgeries, and for secondary long-term prophylaxis. Seventy-two eligible patients, type 1, 2, 3 VWD (25%/38.9%/36.1%) were treated for spontaneous and traumatic bleeding (140 episodes, n = 41 patients), to prevent surgical bleeding (69 episodes, n = 43 patients); and for secondary long-term prophylaxis (18 programs, n = 13 patients). Replacement therapy with pdVWF/FVIII showed an excellent to good clinical efficacy in 96.7% of the bleeding episodes, 100% during surgical procedures and 100% during prophylaxis. No adverse events (AEs), nor serious AEs related to the product were observed. Fanhdi ® was effective, safe and well tolerated in the management of bleeding episodes, the prevention of bleeding during surgeries, and for secondary long-term prophylaxis in VWD patients

Traumatismo craneoencefálico en adultos jóvenes: una serie de casos y análisis de su pronóstico

Introduction: traumatic brain injury (TBI) and associated traumatic injuries continue to stand out as one of the main causes of morbidity and mortality. Pre-hospital management, as well as the time of arrival and admission to the emergency area, the period to enter the operating room, postoperative management and in the intensive care unit are vital. Objectives: the general objective is to present a series of 3 clinical cases of young patients with head trauma who suffered motorcycle accidents on the coast of Ecuador and who were treated in Guayaquil, to emphasize the prevention of complications, in addition to describing the diagnosis. and neurocritical treatment performed. Materials and methods: clinical cases are presented; authorization is obtained from the teaching and research department for access and review of medical records and images. Clinical cases: all the cases corresponded to young, male patients who presented a motorcycle traffic accident without a helmet, the first case presented diffuse axonal injury, and required several months in the intensive care unit, the second case was a patient with a fracture. sunken right parietal artery, which was diagnosed and treated opportunely receiving surgical treatment with good results, the third case presented epidural hematoma, which underwent a hemispheric decompressive craniectomy on the right side and drainage of the epidural hematoma, obtaining satisfactory results. Discussion: in this population, a lack of safety measures was observed, such as the use of a helmet, which resulted in patients presenting: diffuse axonal injury, fractures with collapse, and bruises. Nuclear magnetic resonance and computerized axial tomography of the brain were important in the evaluation of the lesions, since they allowed making a timely decision and choosing the type of treatment, as well as knowing the prognosis in each case. Conclusions: Head trauma is an emergency that requires immediate resolution, it is a growing problem and the main cause of mortality and morbidity in young adults, with a high burden on health care, causing disability in previously healthy patients, the hospital transfer system and time influence its prognosis, interrelated with diagnosis and timely treatment. The cases presented were promptly resolved with a good response. The patient who presented diffuse axonal injury had severe neurological sequelae. It is important to highlight that the three cases presented were due to a motorbike traffic accident, without the use of a helmet, a situation that must be prevented.Introducción: el traumatismo craneoencefálico (TCE) y las lesiones traumáticas asociadas, continúan destacándose como una de las principales causas de morbilidad y mortalidad. El manejo pre hospitalario, así como el tiempo de llegada e ingreso al área de emergencia, el lapso para ingresar a quirófano, el manejo posoperatorio y en la unidad de cuidados intensivos son vitales. Objetivos: el objetivo general es presentar una serie de 3 casos clínicos de pacientes jóvenes, con traumatismo craneoencefálico que sufrieron accidentes en moto en la costa de Ecuador y que fueron atendidos en Guayaquil, para enfatizar en la  prevención de  complicaciones, además de describir el diagnóstico y tratamiento neurocrítico realizado. Materiales y métodos: se realiza presentación de casos clínicos, se obtiene autorización del departamento de docencia e investigación para  acceso  y revisión de las historias clínicas e imágenes.  Casos clínicos:  todos los casos correspondían a pacientes jóvenes, masculinos que presentaron accidente de tránsito en moto sin casco,  el primer caso presentó lesión axonal difusa, y requirió varios meses en unidad de cuidados intensivos, el segundo caso se trato de un paciente con fractura parietal derecha hundida, que fue diagnosticado y atendido oportunamente recibiendo tratamiento quirúrgico con buenos resultados, el tercer caso presentó hematoma epidural, al cual se le  realizó una craniectomía descompresiva hemisférica del lado derecho y drenaje del hematoma epidural, obteniéndose resultados satisfactorios. Discusión: en esta población se observó falta de medidas de seguridad, como es el uso del casco, lo cual trajo como consecuencia que los pacientes presenten: lesión axonal difusa, fracturas con hundimiento y hematomas. La resonancia nuclear magnética y la tomografía axial computarizada de cerebro fueron importantes en la valoración de las lesiones, ya que, permitieron tomar una decisión oportuna y elección del tipo de tratamiento, así como también conocer el pronóstico en cada caso. Conclusiones: El trauma craneoencefálico es una emergencia que amerita resolución inmediata, es un problema creciente y principal causa de mortalidad y morbilidad en adultos jóvenes, con una carga elevada sobre la atención en salud, ocasionando discapacidad en pacientes previamente sanos, el sistema de traslado hospitalario y el tiempo influyen en su pronóstico, interrelacionado con el diagnóstico y tratamiento oportuno. Los casos presentados fueron resueltos oportunamente con buena respuesta, el paciente que presentó lesión axonal difusa, tuvo secuelas neurológicas graves. Es importante destacar que los tres casos presentados fueron por accidente de tránsito en moto, sin utilización del casco, situación que se debe prevenir

Activated prothrombin complex concentrate to treat bleeding events in acquired hemophilia A: BAHAS study

[Objective] Activated prothrombin complex concentrate (aPCC) is a bypassing agent indicated to treat bleeds in patients with acquired hemophilia A (AHA). Nevertheless, its efficacy and safety in the real-world setting have not often been addressed.[Methods] We report the experience of Spanish reference centers for coagulation disorders and from acquired hemophilia Spanish Registry (AHASR) from August 2012 to February 2021. Follow-up period of 30 days after aPCC withdrawal.[Results] Thirty patients with a median age of 70 years old, suffering from 51 bleeds treated with aPCC were finally evaluated. As first-line treatment, aPCC stopped bleeding in 13 of 14 (92.9%) cases. aPCC as the second line after recombinant factor VIIa failure, stopped bleeding in all cases. In 17 patients, aPCC was used far from initial bleed control as prophylaxis of rebleeding with 94% effectiveness. No thromboembolic episodes were communicated. One patient developed hypofibrinogenemia, which did not prevent aPCC from halting bleeding. No other serious adverse events possibly or probably associated with aPCC were reported.[Conclusions] This data support aPCC as hemostatic treatment in AHA with high effectiveness and excellent safety profile in acute bleeds and as extended use to prevent rebleedings, even in aging people with high cardiovascular risk.Shire IIR-ES-002899.Peer reviewe

Adelante / Endavant

Séptimo desafío por la erradicación de la violencia contra las mujeres del Institut Universitari d’Estudis Feministes i de Gènere "Purificación Escribano" de la Universitat Jaume

Alterations produced in child pronator foot after endurance activities and its involvement with the physical fatigue

[Resumen] Objetivos: Este estudio se plantea con el objetivo de determinar si existen cambios en la postura del pie y en la huella plantar de los niños tras una actividad deportiva de resistencia y si estos cambios son mayores en los niños con pies pronadores respecto a los que presentan una tipología de pies neutros. A su vez relacionar todo esto con el cansancio físico desencadenado y los dolores musculares que aparecen tras la actividad física. Nos planteamos llevar a cabo este estudio con la idea de conocer, entre tanta controversia, si el pie pronador presenta algún tipo de desventaja respecto al pie neutro, y si precisaría por tanto de tratamiento ortopodológico como medida de prevención y mejora de las alteraciones biomecánicas en etapas infantiles.Material y Método: Se valoró la postura del pie mediante el Índice de Postura del Pie (en adelante FPI-6), y la huella plantar mediante una pedigrafía, antes y después de ejercicios de resistencia de tipo aeróbicos, para posteriormente calcular el Arch Index mediante el programa informático AutoCad® a cada huella.Resultados y conclusiones: Según los resultados, se puede concluir que la postura del pie y la huella plantar de los niños se modificó tras los ejercicios de resistencia, los pies pronadores se cansaron más y refirieron dolor en pies y piernas durante y/o post actividad, siendo estos resultados estadísticamente significativos (P0.05) de que estos cambios fuesen mayores en los niños con pies pronadores ni que éstos rindieran menos que los niños con pies neutros durante la actividad.[Abstract] Objectives: This study was made with the objective of determine if there are alterations in child’s foot position and his footprint after a endurance activity and to establish wether these alterations are increased in pronator child compared to child who present neutral feet. At the same time, we relate all the above with the extreme physical fatigue and muscle aches that appear after physical activity. The aim of making this essay was to get to know, despite the controversy this topic arouses, if the pronator foot has some kind of disadvantage compared to the neutral foot and wheter it would need orthopedic podiatric treatment as a way to prevent and improve the child biomechanic alterations.Material and Method: Foot position was consider through the Foot Posture Index (FPI-6), and the footprint through a pedigraphy, before and after aerobic endurance activities in order to calculate the Arch Index by applying the computer program AutoCad® to every print.Results and Conclusions: According to the results, it can be concluded that the foot position and the children footprint was altered after endurance activities, pronators were more fatigated and complained about feet and legs aches during and/or after the activities. These results are statistically significant (P0.05) that these changes were higher in children with pronator feet or that their efficiency was lower during the exercise compared to neutral feet childre

VHrare study: Prevalence, clinical features and management of severe rare bleeding disorders in a large cohort

Abstract Introduction: Rare bleeding disorders (RBD) constitute 5% of total hereditary bleeding disorders, although the number could be higher, due to the presence of undiagnosed asymptomatic patients. The objective of this study was to analyze the prevalence and characteristics of patients with severe RBDs in our area. Material and methods: We analyzed the patients with RBD followed at a tertiary‐level hospital between January 2014 and December 2021. Results: A total of 101 patients were analyzed, with a median age at diagnosis of 27.67 years (range 0–89), of which 52.47% were male. The most frequent RBD in our population was FVII deficiency. Regarding the diagnostic reason, the most frequent cause was a preoperative test and only 14.8% reported bleeding symptoms at the time of diagnosis. A genetic study was carried out in 63.36% of patients and the most frequent mutation type found was finding a missense mutation. Conclusions: The distribution of RBDs in our centre is similar to the one reported in the literature. The majority of RBDs were diagnosed from a preoperative test and this allowed preventive treatment prior to invasive procedures to avoid bleeding complications. 83% of patients did not have a pathological bleeding phenotype according to ISTH‐BA

Expert opinion paper on the treatment of hemophilia B with albutrepenonacog alfa

Altres ajuts: CSL Behring.Introduction: Current guidelines recommend prophylactic treatment of hemophilia B with the missing coagulation factor IX, either with standard half-life or extended half-life products. Extended half-life products have half-lives three to six times longer than the former, allowing a reduction in the number of weekly injections and therefore, potentially impacting on treatment adherence and quality of life. Albutrepenonacog alfa is an extended half-life fusion protein of coagulation factor IX with recombinant human albumin, indicated for both on-demand and prophylactic treatment for bleeding in patients with hemophilia B of all ages. Areas covered: The authors review the clinical and pharmacokinetic characteristics of albutrepenonacog alfa, as well as the available information regarding trough levels and real-world evidence. Given the availability of other factor IX products in the market, indirect comparisons of clinical and pharmacokinetic characteristics are presented. Expert opinion: The authors exhibit their expert opinion on which patient profiles are candidates for prophylactic treatment with albutrepenonacog alfa, and on the management of patients in terms of dosing, regimens of administration and protocols for switching the treatment