Endoscopic therapy through dilatation of benign esophageal stenosis

Introduction: Endoscopic dilatation is the first therapeutic option to eliminate benign esophageal stenosis and improve the symptoms and the quality of life of those patients who suffer from it. Objective: To describe the results of endoscopic dilatation in patients with benign esophageal stenosis treated in the National Center for Endoscopic Surgery from January 2015 to December 2016.  Material and Methods:A case series longitudinal observational study was conducted in 59 patients with benign esophageal stenosis. Dilatations were done with Savary-Gilliard bougie and balloons. Results: The mean age was 52,5 years, and the condition predominated in 37 male patients (62,7%). Post-surgical, peptic, and caustic were the most frequent etiologies with 25, 14, and 6 cases, respectively.  Short stenosis predominated in 51 cases. Bougies were used in 48 patients for a total of 149 dilatations, corresponding to a mean of 3,1 dilatations/ patients. Correction of the stenosis was made in 1-3 sessions in 47 % of patients; 11 cases were dilated with balloon, corresponding to a mean of 1- 3 dilatations/ patients. Four patients from the group that were dilated with Savary-Gilliard bougies showed refractoriness. A perforation, and two bleedings occurred.  After the dilatations, dysphagia improved or disappeared in 93,2 % of patients. Conclusions: Endoscopic therapy through dilatation of benign esophageal stenosis indicated to be a good alternative method in achieving corrections in a few dilatation sessions, with a low number of complications, and an improvement of the dysphagia.  Keywords: esophageal stenosis, benign esophageal stenosis, endoscopic therapy, balloon dilatation, dilatation with Savary-Gilliard bougies.</p

Large expert-curated database for benchmarking document similarity detection in biomedical literature search

Document recommendation systems for locating relevant literature have mostly relied on methods developed a decade ago. This is largely due to the lack of a large offline gold-standard benchmark of relevant documents that cover a variety of research fields such that newly developed literature search techniques can be compared, improved and translated into practice. To overcome this bottleneck, we have established the RElevant LIterature SearcH consortium consisting of more than 1500 scientists from 84 countries, who have collectively annotated the relevance of over 180 000 PubMed-listed articles with regard to their respective seed (input) article/s. The majority of annotations were contributed by highly experienced, original authors of the seed articles. The collected data cover 76% of all unique PubMed Medical Subject Headings descriptors. No systematic biases were observed across different experience levels, research fields or time spent on annotations. More importantly, annotations of the same document pairs contributed by different scientists were highly concordant. We further show that the three representative baseline methods used to generate recommended articles for evaluation (Okapi Best Matching 25, Term Frequency-Inverse Document Frequency and PubMed Related Articles) had similar overall performances. Additionally, we found that these methods each tend to produce distinct collections of recommended articles, suggesting that a hybrid method may be required to completely capture all relevant articles. The established database server located at https://relishdb.ict.griffith.edu.au is freely available for the downloading of annotation data and the blind testing of new methods. We expect that this benchmark will be useful for stimulating the development of new powerful techniques for title and title/abstract-based search engines for relevant articles in biomedical research.Peer reviewe

The videofluoroscopic swallowing study shows a sustained improvement of dysphagia in children with Niemann-Pick disease type C after therapy with miglustat

Niemann-Pick disease type C (NPC) is a rare autosomal recessive lysosomal storage disorder characterized by defective intracellular lipid trafficking, with secondary accumulation of free cholesterol, sphingosine, and glycosphingolipids. NPC is clinically characterized by a wide spectrum of manifestations with progressive visceral and neurological involvement, including dysphagia. Neurological manifestations represent the most debilitating findings. Swallowing impairment is a frequent cause of morbidity and disability in NPC patients and progressive dysphagia may be considered a marker of neurological progression. Recently substrate reduction therapy with miglustat has been proposed for the treatment of neurological manifestations in NPC patients. This observational study reports on the long-term use of miglustat in four pediatric patients with NPC and shows the efficacy of the treatment to improve or prevent dysphagia, and persistence after 3 years of treatment or more. We used a videofluoroscopic analysis of liquid barium swallowing to provide additional information on patterns of impairment of the swallowing mechanism and to detect aspiration. In three patients showing dysphagia and aspiration we observed the improvement of the swallowing function and the sustained absence of barium aspiration in the airways after miglustat treatment, while the patient with normal swallowing function at baseline did not show any deterioration. We suggest that the videofluoroscopic study of swallowing should be routinely used to monitor the effects of treatment on swallowing ability in NPC patients

Long-term enzyme replacement therapy for Pompe disease with recombinant human alpha-glucosidase derived from chinese hamster ovary cells.

Pompe disease is a rare autosomal recessive myopathy due to the deficiency of lysosomal acid alpha-glucosidase. Clinical phenotypes range from the severe classic infantile form (hypotonia and hypertrophic cardiomyopathy), to milder late onset forms (skeletal myopathy and absence of significant heart involvement). Enzyme replacement therapy with recombinant human alpha-glucosidase derived from either rabbit milk or Chinese hamster ovary cells has been introduced and is undergoing clinical trials. Reported is a long-term follow-up of 3 Pompe patients presenting without cardiomyopathy, treated with recombinant human alpha-glucosidase derived from Chinese hamster ovary cells. This study suggests that enzyme replacement therapy can lead to significant motor and respiratory improvement in the subgroup of patients who start the therapy before extensive muscle damage has occurred. The recombinant enzyme derived from Chinese hamster ovary cells, administered at doses significantly higher than previously reported, appears to have the same safety as the drug derived from rabbit milk

Terapéutica endoscópica mediante dilataciones en estenosis esofágicas benignas

Introduction: Endoscopic dilatation is the first therapeutic option to eliminate benign esophageal stenosis and improve the symptoms and the quality of life of those patients who suffer from it. Objective: To describe the results of endoscopic dilatation in patients with benign esophageal stenosis treated in the National Center for Endoscopic Surgery from January 2015 to December 2016.  Material and Methods:A case series longitudinal observational study was conducted in 59 patients with benign esophageal stenosis. Dilatations were done with Savary-Gilliard bougie and balloons. Results: The mean age was 52,5 years, and the condition predominated in 37 male patients (62,7%). Post-surgical, peptic, and caustic were the most frequent etiologies with 25, 14, and 6 cases, respectively.  Short stenosis predominated in 51 cases. Bougies were used in 48 patients for a total of 149 dilatations, corresponding to a mean of 3,1 dilatations/ patients. Correction of the stenosis was made in 1-3 sessions in 47 % of patients; 11 cases were dilated with balloon, corresponding to a mean of 1- 3 dilatations/ patients. Four patients from the group that were dilated with Savary-Gilliard bougies showed refractoriness. A perforation, and two bleedings occurred.  After the dilatations, dysphagia improved or disappeared in 93,2 % of patients. Conclusions: Endoscopic therapy through dilatation of benign esophageal stenosis indicated to be a good alternative method in achieving corrections in a few dilatation sessions, with a low number of complications, and an improvement of the dysphagia.  Keywords: esophageal stenosis, benign esophageal stenosis, endoscopic therapy, balloon dilatation, dilatation with Savary-Gilliard bougies.Introducción: Las dilataciones endoscópicas constituyen la primera opción terapéutica para eliminar las estenosis esofágicas benignas y mejorar los síntomas y la calidad de vida de los pacientes que las presentan. Objetivo: Describir los resultados de las dilataciones endoscópicas en pacientes con estenosis esofágicas benignas, atendidos en el Centro Nacional de Cirugía de Mínimo Acceso, durante enero de 2015 a diciembre de 2016. Material y Métodos: Se realizó un estudio observacional longitudinal de serie de casos, en 59 pacientes con estenosis esofágicas benignas. Las dilataciones se efectuaron con Bujías de Savary-Gilliard y balones. Resultados: La edad media fue de 52,5 años, predominaron los hombres con 37 (62,7%) pacientes. Las etiologías más frecuentes fueron la postquirúrgicas, pépticas y caústicas con 25, 14 y 6 casos respectivamente. Predominaron las estenosis cortas en 51 casos. En 48 pacientes se utilizaron bujías, con un total de 149dilataciones, con una media de 3,1 dilataciones/pacientes, 47,5%corrigió la estenosis con 1-3 sesiones; 11 casos se dilataron con balón, con una media de 1,3 dilataciones/pacientes. En el grupo de los dilatados con Bujías de Savary-Guilliard, 4 pacientes mostraron refractariedad y ocurrieron una perforación y 2 sangrados. Posterior a las dilataciones, en 93,2% de los pacientes, mejoró o desapareció la disfagia. Conclusiones: La terapéutica endoscópica mediante dilataciones en las estenosis esofágicas benignas demostró ser una buena alternativa, al lograr su corrección con pocas sesiones de dilataciones, bajo número de complicaciones y mejoría de la disfagia.   Palabras claves: Estenosis de esófago, estenosis benigna de esófago, terapéutica endoscópica, dilatación con balón, dilatación con Bujías de Savary Giliard

Annali storici di Principato Citra, A. 14, n. 2.2 (2016)

A.14, n.2.2(2016): G. Guardia, Editoriale, P. 3 ; F. Sofia, “Si levò una gran tempesta à segno di ponente et lebeccio”: imbarcazioni, uomini, attività lungo le coste cilentane in età moderna (secc. XVII-XVIII)”, P. 9 ; A. Capano, Medici del Cilento in epoca moderna e contemporanea dal conseguimento del diploma di laurea all'attività nel campo sanitario, sociale e politico, P. 92 ; E. Catone, «Il fischio della macchina a vapore». Il mulino-trappeto impiantato nell’ex convento agostiniano di Buccino (1880-1929), P. 121 ; P. Natella, Raffaele Guariglia : un ritratto, P. 150 ; V. Aversano, Una insolita “guida” di Salerno, tra plurime prospettive spazio-temporali centrate sul rapporto verde/edificato. Per il recupero e la valorizzazione dell’identità cittadina, P.192 ; P. F. Giuliani Mazzei, Il centro preistorico megalitico sul Monte della Stella. Un’ipotesi di lavoro, P. 192 ; L. Rossi, Archivi e memorie. Pietro Barbato: Paesi che circondano Stio, P. 242 ; U.Landi, Archivi e memorie. La voce di un prete nella seconda guerra mondiale, P.269 ; V. Amendola, Ernesto Samaritani fotografo : biografia e fondi fotografici, P. 282 ; G. De Luca, I de Marsilio: l’ascesa di una famiglia borghese nella Vallo del Settecento, P. 291 ; F. Innella, L’ultimo caffè: un caso di vebeficio nella Battipaglia borbonica, P. 298 ; M.T. Schiavino, Alcuni aspetti della mobilitazione civile a Salerno nel corso della Prima Guerra Mondiale (1914-1916), P. 301 ; A. Andria, A. Conte, Dei molteplici Orizzonti di Piero Lucia ovvero Diverse storie di politica e cultura, P. 307

Annali storici di Principato Citra, A. 15, n. 1.1 (2017)

A.15, n.1.1(2017): G. Guardia, Editoriale, P. 3 ; P. Natella, Sull’origine ed evoluzione di “Picentia”, P. 5 ; S. Lisi, A tavola con i Romani, P. 21 ; C. Schiavo, Agostino Migliani tra senso dello Stato e legami pericolosi, P. 40 ; U. Landi, I sindaci di Baronissi dal 1861 al 2016, P. 57 ; M. Morra, Quattro secoli di storia a Vallo: dal “Conservatorio di S.Caterina” agli “Istituti Riuniti”, P 115 ; A. Andria, Il Centro Universitario Europeo per i Beni Culturali, P. 127 ; M. Iannone, Un “moltiplico” Istituito da monsignor Luigi Pappacoda, P. 132 ; A. Capano, Celle e Poderia nel Catasto Murattiano, P. 143 ; M. A. del Grosso, P. Trotta, La Rotonda di Nocera, P. 169 ; L. Rossi, Cannalonga dal 1799 alla seconda restaurazione borbonica, P. 177 ; M. Scafidi, La scienza odontoiatrica in età romana. I reperti antropologici della necropoli di S. Marco di Castellabate (SA), P. 212 ; F. De Luca, Strutture socio-economiche in un’universtià del Cilento alla metà del Settecento. Camerota in Principato Citeriore, P. 268 ; M. Di Maio, Il territorio della provincia di Salerno nelle perizie preunitarie (1813-1865), P. 294 ; T. Guardia, Fuori tempo massimo, P. 303

Long term follow-up to evaluate the efficacy of miglustat treatment in Italian patients with Niemann-Pick disease type C

BACKGROUND: Twenty-five patients with Niemann Pick disease type C (age range: 7 months to 44 years) were enrolled in an Italian independent multicenter trial and treated with miglustat for periods from 48 to 96 months. METHODS: Based on the age at onset of neurological manifestations patients' phenotypes were classified as: adult (n = 6), juvenile (n = 9), late infantile (n = 6), early infantile (n = 2). Two patients had an exclusively visceral phenotype. We clinically evaluated patients' neurological involvement, giving a score of severity ranging from 0 (best) to 3 (worst) for gait abnormalities, dystonia, dysmetria, dysarthria, and developmental delay/cognitive impairment, and from 0 to 4 for dysphagia. We calculated a mean composite severity score transforming the original scores proportionally to range from 0 to 1 to summarize the clinical picture of patients and monitor their clinical course. RESULTS: We compared the results after 24 months of treatment in 23 patients showing neurological manifestations. Stabilization or improvement of all parameters was observed in the majority of patients. With the exception of developmental delay/cognitive impairment, these results persisted after 48-96 months in 41 - 55% of the patients (dystonia: 55%, dysarthria: 50%, gait abnormalities: 43%, dysmetria: 41%, respectively). After 24 months of therapy the majority of the evaluable patients (n = 20), demonstrated a stabilization or improvement in the ability to swallow four substances of different consistency (water: 65%, purée: 58%, little pasta: 60%, biscuit: 55%). These results persisted after 48-96 months in 40-50% of patients, with the exception of water swallowing. Stabilization or improvement of the composite severity score was detected in the majority (57%) of 7 patients who were treated early (within 3.5 years from onset) and rarely in patients who received treatment later. CONCLUSIONS: The results of this study suggest that miglustat treatment can improve or stabilize neurological manifestations, at least for a period of time; the severity of clinical conditions at the beginning of treatment can influence the rate of disease progression. This conclusion applies particularly to patients with juvenile or adult onset of the disease. TRIAL REGISTRATION: EudraCT number 2006-005842-3