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Currently, eculizumab is the main effective treatment for paroxysmal nocturnal hemoglobinuria (PNH). The aim of this randomized multicenter noninferiority study was to evaluate the efficacy and safety of the Biosimilar (Elizaria) in comparison with the Originator (Soliris) in patients with PNH. Biosimilar and Originator were administered at a dose of 600 mg weekly for 4 weeks at the initial stage in naive patients, as well as for maintenance therapy at a dose of 900 mg every 2 weeks in all patients. The primary endpoint was a comparative assessment of hemolytic activity based on the area under the lactate dehydrogenase (LDH) concentration–time curve during the maintenance therapy. Thirty-two (32) patients were randomized for therapy with Biosimilar (n = 16) or Originator (n = 16). The mean values of LDH concentration–time curve were similar in both treatment groups without statistically significant differences (p > 0.05). Evaluation of secondary endpoints has shown no statistically significant differences between the groups. Safety values were comparable in both treatment groups. The data obtained confirm that the Biosimilar is not inferior to the Originator in terms of the main efficacy parameter, and is also comparable with it in terms of safety and additional efficacy parameters. Clinicaltrials.gov identifier: NCT04463056

El siguiente trabajo presenta el caso ficticio de M, una mujer de 71 años ingresada en un centro residencial y diagnosticada con Atrofia Multisistémica (AMS). Este trastorno neurodegenerativo se clasifica dentro de los parkinsonismos atípicos. Se cree que es causado por depósitos anómalos de α-sinucleína en algunas estructuras cerebrales, ocasionando atrofia olivopontocerebelosa y degeneración estriatonígrica. Sus principales síntomas incluyen disautonomía, parkinsonismo y disfunción cerebelosa, y se clasifica en función de si hay un predominio de clínica parkinsoniana (AMS-P) o predominio de clínica cerebelosa (AMS-C). Tradicionalmente, la presencia de alteraciones cognitivas se ha considerado un criterio de exclusión para el diagnóstico de la AMS. No obstante, en las últimas décadas han salido a la luz evidencias de que no solo son compatibles las alteraciones cognitivas en pacientes con AMS, sino que se ha podido establecer un perfil neuropsicológico característico. La evaluación neuropsicológica realizada muestra alteraciones en la orientación temporal, la atención compleja, la memoria verbal a corto plazo y las funciones ejecutivas. A nivel neuropsiquiátrico se observa estado ansioso y alteraciones conductuales. Al tratase de un trastorno neurodegenerativo, el objetivo de la intervención ha sido mantener y preservar las capacidades cognitivas y mejorar el estado emocional y conductual de la paciente. Para ello se ha diseñado una intervención neuropsicológica de seis meses que incluye estimulación cognitiva individual, grupal y terapia emocional y conductual individual. Con esta intervención, se espera ralentizar el avance de las alteraciones cognitivas detectadas y mejorar la calidad de vida reduciendo las alteraciones conductuales y el estado ansioso. The following work presents the fictitious case of M, a 71-year-old woman in an elderly residence who is diagnosed with Multiple Systemic Atrophy (MSA). This neurodegenerative disorder is classified within the atypical parkinsonisms. It is believed to be caused by abnormal deposits of α-synuclein in some brain structures, leading to olivopontocerebellar atrophy and striatonigral degeneration. Its main symptoms include dysautonomia, parkinsonism and cerebellar dysfunction, and it is classified depending on whether there is a predominance of parkinsonian symptoms (MSA-P) or a predominance of cerebellar symptoms (MSA-C). Traditionally, the presence of cognitive impairment has been considered an exclusion criterion for the diagnosis of MSA. However, in recent decades evidence has come to light that not only are cognitive impairment compatible in patients with MSA, but a characteristic neuropsychological profile has been established. The neuropsychological evaluation that was carried out reveals alterations in temporal orientation, complex attention, short-term verbal memory and executive functions. Observed neuropsychiatric symptoms are a state of anxiety and behavioral alterations. Being a neurodegenerative disorder, the objective of the intervention has been to maintain and preserve cognitive function and improve the emotional and behavioral state of the patient. For this, a six-month neuropsychological intervention has been designed including individual and group cognitive stimulation, and individual emotional and behavioral therapy. This intervention is expected to slow down the progress of the cognitive impairment and improve the quality of life by reducing behavioral alterations and anxiety. El següent treball presenta el cas fictici de M, una dona de 71 anys ingressada en un centre residencial i diagnosticada amb Atròfia Multisistèmica (AMS). Aquest trastorn neurodegeneratiu es classifica dins dels parkinsonismes atípics. Es creu que és causat per dipòsits anòmals de α-*sinucleína en algunes estructures cerebrals, ocasionant atròfia olivopontocerebelosa i degeneració estriatonígrica. Els seus principals símptomes inclouen disautonomía, parkinsonisme i disfunció cerebel·losa, i es classifica en funció de si hi ha un predomini de clínica parkinsoniana (AMS-P) o predomini de clínica cerebel·losa (AMS-C). Tradicionalment, la presència d'alteracions cognitives s'ha considerat un criteri d'exclusió per al diagnòstic de la AMS. No obstant això, en les últimes dècades han sortit a la llum evidencies que no sols són compatibles les alteracions cognitives en pacients amb AMS, sinó que s'ha pogut establir un perfil neuropsicològic característic. L'avaluació neuropsicològica realitzada mostra alteracions en l'orientació temporal, l'atenció complexa, la memòria verbal a curt termini i les funcions executives. A nivell neuropsiquiátrico s'observa estat ansiós i alteracions conductuals. Al tractés d'un trastorn neurodegeneratiu, l'objectiu de la intervenció ha estat mantenir i preservar les capacitats cognitives i millorar l'estat emocional i conductual de la pacient. Per a això s'ha dissenyat una intervenció neuropsicològica de sis mesos que inclou estimulació cognitiva individual, grupal i teràpia emocional i conductual individual. Amb aquesta intervenció, s'espera alentir l'avanç de les alteracions cognitives detectades i millorar la qualitat de vida reduint les alteracions conductuals i l'estat ansiós.

Con el incremento de la esperanza de vida, se ha producido un aumento de la incidencia de enfermedades características de la tercera edad, entre ellas las enfermedades neurodegenerativas como la Enfermedad de Alzheimer (EA) y, en general, la demencia. Según la OCDE (2018) la prevalencia de demencia en Europa se situa en un 7%, un 8 % en España. Teniendo presente que, como indica la OMS, entre 2015 y 2050 se duplicará el porcentaje de mayores de 60 (2018) queda patente la urgencia de buscar métodos protectores y preventivos ante el desarrollo de la demencia. Actualmente, se están llevando a cabo diversos proyectos, a nivel europeo y mundial, centrados en desarrollar técnicas de prevención y protección de la demencia. En el siguiente trabajo, se ha realizado una revisión de la reciente literatura, tomando en consideración dos de los factores protectores que describe el proyecto FINGER (Kivipelto et al., 2013): la dieta mediterránea y el ejercicio. Se han consultado las bases de datos PubMed, Dialnet, PsycInfo y Scielo, seleccionando un total de 28 artículos para revisión. Los resultados muestran una clara evidencia de la adherencia a la dieta mediterránea como factor preventivo de la demencia, mientras los resultados no son tan claros sobre el ejercicio, debido a la falta de consenso entre el tipo de ejercicio, la intensidad y la frecuencia. Aun así, se sugiere que las intervenciones multimodales –que combinen dieta sana, actividad física, ocio, entrenamiento cognitivo y/o un buen descanso– puedan tener mayores beneficios en la función cognitiva. Se recomienda futuros estudios que amplíen la evidencia. With the increase in life expectancy, there has been an increase in the incidence of diseases characteristic of the elderly, including neurodegenerative diseases such as Alzheimer's Disease (AD) and, in general, dementia. According to the OECD (2018), the prevalence of dementia in Europe stands at 7%, 8% in Spain. Bearing in mind that, as indicated by WHO, that between 2015 and 2050, the proportion of the world's population over 60 years will nearly double (2018), there is the need to seek protective and preventive methods in the development of dementia. Currently, several projects, at a European and worldwide level, are focused on developing prevention protection techniques for dementia. In the following work, I have reviewed the recent literature, considering two of the protective factors that describe the FINGER project (Kivipelto et al., 2013): the Mediterranean diet and exercise. The PubMed, Dialnet, PsycInfo and Scielo databases have been consulted, selecting a total of 28 articles for review. The results presented have a clear evidence of adherence to the Mediterranean diet as a preventive factor for dementia, while the results are not so clear about exercise, due to the lack of consensus between the type of exercise, intensity and frequency. Even so, the review suggests that multimodal interventions - which combine healthy diet, physical activity, leisure, cognitive training and/or a good rest - can have greater benefits in cognitive function. Future studies that expand the evidence are necessary. Amb l'increment de l'esperança de vida, s'ha produït un augment de la incidència de malalties característiques de la tercera edat, entre elles les malalties neurodegeneratives com la Malaltia d'Alzheimer (EA) i, en general, la demència. Segons l'OCDE (2018) la prevalença de demència a Europa es situa en un 7%, un 8% a Espanya. Tenint present que, com indica l'OMS, entre 2015 i 2050 es duplicarà el percentatge de majors de 60 (2018) queda patent la urgència de buscar mètodes protectors i preventius davant el desenvolupament de la demència. Actualment, s'estan duent a terme diversos projectes, a nivell europeu i mundial, centrats en desenvolupar tècniques de prevenció i protecció de la demència. En el següent treball, s'ha realitzat una revisió de la recent literatura, prenent en consideració dues dels factors protectors que descriu el projecte FINGER (Kivipelto et al., 2013): la dieta mediterrània i l'exercici. S'han consultat les bases de dades PubMed, Dialnet, PsycInfo i Scielo, seleccionant un total de 28 articles per a revisió. Els resultats mostren una clara evidència de l'adherència a la dieta mediterrània com a factor preventiu de la demència, mentre els resultats no són tan clars sobre l'exercici, a causa de la falta de consens entre el tipus d'exercici, la intensitat i la freqüència. Així i tot, se suggereix que les intervencions multimodals –que combinin dieta sana, activitat física, oci, entrenament cognitiu i/o un bon descans– puguin tenir majors beneficis en la funció cognitiva. Es recomana futurs estudis que ampliïn l'evidència.

BACKGROUND: \ud \ud Growth guidance sliding treatment devices such as Shilla (Medtronic, USA) or LSZ-4D (Conmet, Russia) used for the treatment of scoliosis in children who have high growth potential have unlocked fixtures allowing rods to slide during growth of the spine which avoids periodical extensions. However, the probability of clinical complications associated with metallosis after the implantation of such devices is poorly understood. The content of metal ions in the blood and tissues of pediatric patients treated for scoliosis using fusionless growth guidance sliding instrumentation, have not yet been investigated.\ud \ud PURPOSE: \ud \ud The aim of this study was to measure the content of metal ions in blood and tissues surrounding implanted growth guidance sliding LSZ-4D devices made of titanium alloy (Ti6Al4V) and to identify the incidence of metallosis associated clinical complications in some patients with these devices.\ud \ud STUDY DESIGN: \ud \ud One center case-control retrospective study.\ud \ud PATIENTS SAMPLE: \ud \ud Study group included 25 patients with high growth potential (3 males, 22 females, average age at primary surgery for scoliosis treatment is 11.4±1.2 years old) who had sliding growth guidance instrumentation LSZ-4D (Conmet, Moscow, Russia) implanted on 13 (range 10-16) spine levels for 6±2 years. The LSZ-4D device was made from titanium alloy Ti6Al4V and consisted of two rectangular section rods and fixture elements. Locked fixtures were used on one spinal level, while the others were unlocked (sliding). The control group consisted of 13 patients (12 females and 1 male, 11±1.2 years old) without any implanted devices.\ud \ud OUTCOME MEASURES: \ud \ud The content of Ti, Al and V metal ions in the whole blood and tissues around the implanted device was measured. Incidence of metallosis associated complications in the study group were recorded.\ud \ud METHODS: \ud \ud Metal ion content was measured by ICP-MC on quadrupolar Nexion 300D (Perkin Elmer, USA).\ud \ud RESULTS: \ud \ud 5 of 25 patients in the study group developed metallosis associated complications (two sinuses and three seromas in the lumbar part of the spine). Revisions were carried out in two of these patients. 90% of patients in the study group had increased content of Ti and V ions in the blood (2.8 and 4 times respectively). Median content of Ti ions in soft tissues adjacent to implanted sliding device was more than 1,500 fold higher compared with the control group. These levels are much higher than previously reported for spinal instrumentation.\ud \ud CONCLUSION: \ud \ud Increased content of Ti and V ions in the blood and especially in tissues around the titanium growth guidance sliding device LSZ-4D accompanied by clinical manifestations (seromas and sinuses) indicate the importance of improving of wear resistance of such instrumentation with the coatings and the necessity to exchange sliding instrumentation once the child is fully grown.

Type 1 (non neuronopathic) Gaucher disease was the first lysosomal storage disorder for which an effective enzyme replacement therapy was developed and it has become a prototype for treatments for related orphan diseases. There are currently four treatment options available to patients with Gaucher disease, nevertheless, almost 25% of type 1 Gaucher patients do not gain timely access to therapy because of delays in diagnosis after the onset of symptoms. Diagnosis of Gaucher disease by enzyme testing is unequivocal, but the rarity of the disease and non-specific and heterogeneous nature of Gaucher disease symptoms may impede consideration of this disease in the differential diagnosis. To help promote timely diagnosis and optimal management of the protean presentations of Gaucher disease, a consensus meeting was convened to develop algorithms for diagnosis and disease management for Gaucher disease.