Oncological outcomes of visibly complete transurethral resection prior to neoadjuvant chemotherapy for bladder cancer

ABSTRACT Purpose: To evaluate the potential oncologic benefit of a visibly complete transurethral resection of a bladder tumor (TURBT) prior to neoadjuvant chemotherapy (NAC) and radical cystectomy (RC). Materials and Methods: We identified patients who received NAC and RC between 2011-2021. Records were reviewed to assess TURBT completeness. The primary outcome was pathologic downstaging (<ypT2N0), with complete pathologic response (ypT0N0) and survival as secondary endpoints. Logistic regression and Cox proportional hazards models were utilized. Results: We identified 153 patients, including 116 (76%) with a complete TURBT. Sixty-four (42%) achieved <ypT2N0 and 43 (28%) achieved ypT0N0. When comparing those with and without a complete TURBT, there was no significant difference in the proportion with <ypT2N0 (43% vs 38%, P=0.57) or ypT0N0 (28% vs 27%, P=0.87). After median follow-up of 3.6 years (IQR 1.5-5.1), 86 patients died, 37 died from bladder cancer, and 61 had recurrence. We did not observe a statistically significant association of complete TURBT with cancer-specific or recurrence-free survival (p≥0.20), although the hazard of death from any cause was significantly higher among those with incomplete TURBT even after adjusting for ECOG and pathologic T stage, HR 1.77 (95% CI 1.04-3.00, P=.034). Conclusions: A visibly complete TURBT was not associated with pathologic downstaging, cancer-specific or recurrence-free survival following NAC and RC. These data do not support the need for repeat TURBT to achieve a visibly complete resection if NAC and RC are planned.

A comparative study of knowledge, attitude, and practice of self-medication among medical and nursing students in a government medical college of Eastern India

Background: Self-medication is defined as use of medicines without a doctor’s prescription and is frequently practiced among students in medical and nursing colleges. The purpose of this study was to compare the knowledge, attitude and practice of self-medication between 2nd year medical and nursing students in a government medical college of West Bengal, India. Aims and Objectives: The objectives of the study were to assess the knowledge, attitude and practice of self-medication among 2nd year medical students and nursing students and to compare their differences if any. Materials and Methods: A cross-sectional, observational, and questionnaire based study conducted between two groups of students, medical, and nursing, who were interviewed with a prevalidated questionnaire. Demographic data and questions regarding knowledge, attitude, and practice of self-medication were included. Details and purpose of the study were explained to the students. The collected data were analyzed statistically and the comparison between the two groups was done. Results: A total of 260 students were interviewed, of whom 231 participants were selected involving 172 medical students and 59 nursing students. Although 89% of the students have practiced self-medication within the past 1 year, only 61% students agreed recommending self-treatment to others. Accordingly, there were significant differences among the two groups in terms of source of information, preference, frequency of practice, disadvantages, and drugs/drug groups taken for self-medication. Conclusion: Self-medication is a common form of health care with potential benefits and hazards. Our study suggests self-medication being employed more commonly by medical students than their nursing counterparts. Hence, it is very important to increase the awareness about the pros and cons of self-medication for both medical and nursing students to improve their knowledge, attitude, and practice and eventually to increase the awareness in the society.

Helicobacter pylori infection and UBT-13C values are associated with changes in body mass index in children and adults / La infección por Helicobacter pylori y los valores de UBT-13C se asocian con el índice de masa corporal en niños y adultos

BACKGROUND: The urea breath test (UBT-13C) is a non-invasive technique that allows the diagnosis and confirmation of eradication of Helicobacter pylori infection. Aim: To evaluate H. pylori positivity and values of UBT-13C among infected Chilean children and adults, and to analyze its variation in relation to sex, nutritional status, and age of the patients. Material and Methods: Retrospective study of 1141 patients aged 6 to 94 years, with an indication for a UBT-13C either for diagnosis or for confirmation of eradication of H. pylori infection. 13C enrichment was measured using an infrared spectrometer calculating the delta 13C values before and after the ingestion of 13C marked urea. The clinical data of the patients were obtained at the time of the examination. Results: We included 241 children and 900 adults. Infected children obtained lower UBT-13C delta values than infected adults (16.1 ± 8.7 and 37 ± 52.9, respectively). The rates of infection were higher in males who were recruited for diagnosis. Significant differences were obtained between positivity for H. pylori in overweight and obese children but not adults. UBT-13C titers were significantly associated with the body mass index (BMI) only in adults. Conclusions: H. pylori infection rates are similar between sexes and are higher in children probably because of selection bias. In children, H. pylori positivity is associated with higher BMI and excess malnutrition although with similar UBT-13C values. In adults, H. pylori infection is not related with BMI, but a higher BMI impacts UBT-13C titers.

ANTECEDENTES: La prueba de aliento con urea (UBT-13C) es una técnica no invasiva que permite el diagnóstico y confirmación de erradicación de la infección por Helicobacter pylori. Objetivo: Evaluar los valores de UBT- 13C en niños y adultos chilenos infectados y analizar su variación en relación al sexo, diagnóstico nutricional y edad de los pacientes. Material y Métodos: Estudio retrospectivo de 1.141 pacientes de 6 a 94 años. El enriquecimiento de13C se midió usando un espectrómetro de infrarrojos, calculando el delta 13C antes y después de la ingesta de urea marcada con 13C. Los datos clínicos de los pacientes se obtuvieron al momento del examen. Resultados: Incluimos 241 niños y 900 adultos con valores delta de UBT-13C de 16,1 ± 8,7 frente a 37 ± 52,9, respectivamente. Las tasas de infección fueron mayores en los hombres reclutados para el diagnóstico. Se obtuvieron diferencias significativas entre la positividad para H. pylori en niños con sobrepeso y obesidad, pero no en adultos. Los títulos de UBT-13C se asociaron significativamente con el índice de masa corporal (IMC) solo en adultos. Conclusiones: Las tasas de infección por H. pylori son similares entre los sexos y aumentan en los niños probablemente debido al sesgo de selección. En niños, la positividad para H. pylori se asocia con un IMC más alto y malnutrición por exceso, aunque con valores similares de UBT-13C. En los adultos, la infección por H. pylori no se relaciona con el IMC ni con la obesidad, pero el aumento del IMC afecta los títulos de UBT-13C.

Comparison of efficacy and safety of vildagliptin 50 mg tablet twice daily and vildagliptin 100 mg sustained release once daily tablet on top of metformin in Indian patients with Type 2 diabetes mellitus: A randomized, open label, Phase IV parallel group, clinical trial

Background: Type 2 diabetes mellitus (DM) is mainly due to multifactorial of which insulin resistance and deficiency in the incretion are two important pathophysiological factors. Vildagliptin, an oral hypoglycemic agent, acts by inhibiting dipeptidyl peptidase-4 enzyme, often uses as a first line drug along with metformin to enhance outcome. Aim and Objective: The aim of this study was to compare the effectiveness and safety of vildagliptin 50 mg twice daily dose with vildagliptin 100 mg sustained release tablet (SR) once daily in Type 2 DM patients and uncontrolled with metformin monotherapy. Materials and Methods: Adult patients with Type 2 DM fulfilling the inclusion criteria were randomized in two groups. Group 1 patient received metformin 1000 mg/day in two divided dose and tablet vildagliptin 50 mg 2 times daily, while Group 2 patients received metformin 1000 mg/day in two divided dose along with vildagliptin 100 mg SR once daily. Fasting plasma glucose (FPG), postprandial plasma glucose (PPPG), and glycated hemoglobin (HbA1C), were measured at baseline, on week 4, week 8, and week 12 visits. Liver function test (SGOT), SGPT, Serum Bilirubin), kidney function test electrolytes, serum urea, serum creatinine), and body weight also measured in first visit and in 12th week. Results: HbA1C, FPG, and PPPG all three decreased equally at 12 week from their respective baseline values (P < 0.05) in both groups. There is no statistically significant alteration of liver enzymes and in serum bilirubin level from baseline to 12th week in both groups. Conclusion: Vildagliptin 100 mg SR once daily dose is equally effective and safe as 50 mg twice daily dose in terms of reducing HbA1C, FPG, and PPPG when it is used along with metformin 1000 mg.

Clinical and laboratory characteristics of Brazilian versus non-Brazilian primary antiphospholipid syndrome patients in AntiPhospholipid Syndrome Alliance for Clinical Trials and InternatiOnal Networking (APS ACTION) clinical database and repository

Abstract Background: Antiphospholipid syndrome (APS) is characterized by episodes of thrombosis, obstetric morbidity or both, associated with persistently positive antiphospholipid antibodies (aPL). Studying the profile of a rare disease in an admixed population is important as it can provide new insights for understanding an autoimmune disease. In this sense of miscegenation, Brazil is characterized by one of the most heterogeneous populations in the world, which is the result of five centuries of interethnic crosses of people from three continents. The objective of this study was to compare the clinical and laboratory characteristics of Brazilian vs. non-Brazilian primary antiphospholipid syndrome (PAPS) patients. Methods: We classified PAPS patients into 2 groups: Brazilian PAPS patients (BPAPS) and PAPS patients from other countries (non-BPAPS). They were compared regarding demographic characteristics, criteria and non-criteria APS manifestations, antiphospholipid antibody (aPL) profile, and the adjusted Global Antiphospholipid Syndrome Score (aGAPSS). Results: We included 415 PAPS patients (88 [21%] BPAPS and 327 [79%] non-BPAPS). Brazilian patients were significantly younger, more frequently female, sedentary, obese, non-white, and had a higher frequency of livedo (25% vs. 10%, p < 0.001), cognitive dysfunction (21% vs. 8%, p = 0.001) and seizures (16% vs. 7%, p = 0.007), and a lower frequency of thrombocytopenia (9% vs. 18%, p = 0.037). Additionally, they were more frequently positive for lupus anticoagulant (87.5% vs. 74.6%, p = 0.01), and less frequently positive to anticardiolipin (46.6% vs. 73.7%, p < 0.001) and anti-ß2-glycoprotein-I (13.6% vs. 62.7%, p < 0.001) antibodies. Triple aPL positivity was also less frequent (8% vs. 41.6%, p < 0.001) in Brazilian patients. Median aGAPSS was lower in the Brazilian group (8 vs. 10, p < 0.0001). In the multivariate analysis, BPAPS patients still presented more frequently with livedo, cognitive dysfunction and sedentary lifestyle, and less frequently with thrombocytopenia and triple positivity to aPL. They were also less often white. Conclusions: Our study suggests a specific profile of PAPS in Brazil with higher frequency of selected non-criteria manifestations and lupus anticoagulant positivity. Lupus anticoagulant (not triple positivity) was the major aPL predictor of a classification criteria event.

Adaptación a la realidad de Latinoamérica de la Guía Clínica NASPGHAN/ESPGHAN 2016 sobre Diagnóstico, Prevención y Tratamiento de Infección por Helicobacter pylori en Pediatría / Adaptation to the reality of Latin America of the NASPGHAN/ESPGHAN 2016 Guidelines on the Diagnosis, Prevention and Treatment of Helicobacter pylori Infection in Pediatrics

Resumen: Introducción: Las últimas guías clínicas conjuntas de NASPGHAN y ESPGHAN en relación a la infección por H. pylori publicadas el año 2016, contienen 20 afirmaciones que han sido cuestionadas en la práctica respecto a su aplicabilidad en Latinoamérica (LA); en particular en relación a la preven ción del cáncer gástrico. Métodos: Se realizó un análisis crítico de la literatura, con especial énfasis en datos de LA y se estableció el nivel de evidencia y nivel de recomendación de las afirmaciones mas controversiales de las Guías Conjuntas. Se realizaron 2 rondas de votación de acuerdo a la técnica Delfi de consenso y se utilizó escala de Likert (de 0 a 4) para establecer el "grado de acuerdo" entre un grupo de expertos de SLAGHNP. Resultados: Existen pocos estudios en relación a diagnóstico, efectividad de tratamiento y susceptibilidad a antibióticos de H. pylori en pacientes pediátricos de LA. En base a estos estudios, extrapolaciones de estudios de adultos y la experiencia clínica del panel de expertos participantes, se realizan las siguientes recomendaciones. Recomendamos la toma de biopsias para test rápido de ureasa e histología (y muestras para cultivo o técnicas moleculares, cuando estén disponibles) durante la endoscopia digestiva alta sólo si en caso de confirmar la infección por H. pylori, se indicará tratamiento de erradicación. Recomendamos que centros regionales seleccio nados realicen estudios de sensibilidad/resistencia antimicrobiana para H. pylori y así actúen como centros de referencia para toda LA. En caso de falla de erradicación de H. pylori con tratamiento de primera línea, recomendamos tratamiento empírico con terapia cuádruple con inhibidor de bomba de protones, amoxicilina, metronidazol y bismuto por 14 días. En caso de falla de erradicación con el esquema de segunda línea, se recomienda indicar un tratamiento individualizado considerando la edad del paciente, el esquema indicado previamente y la sensibilidad antibiótica de la cepa, lo que implica realizar una nueva endoscopía con extracción de muestra para cultivo y antibiograma o es tudio molecular de resistencia. En niños sintomáticos referidos a endoscopía que tengan antecedente de familiar de primer o segundo grado con cáncer gástrico, se recomienda considerar la búsqueda de H. pylori mediante técnica directa durante la endoscopia (y erradicarlo cuando es detectado). Con clusiones: La evidencia apoya mayoritariamente los conceptos generales de las Guías NASPGHAN/ ESPGHAN 2016, pero es necesario adaptarlas a la realidad de LA, con énfasis en el desarrollo de centros regionales para el estudio de sensibilidad a antibióticos y mejorar la correcta selección del tratamiento de erradicación. En niños sintomáticos con antecedente familiar de primer o segundo grado de cáncer gástrico, se debe considerar la búsqueda y erradicación de H. pylori.

Abstract: Introduction: The latest joint H. pylori NASPGHAN and ESPGHAN clinical guidelines published in 2016, contain 20 statements that have been questioned in practice regarding their applicability in Latin America (LA); in particular in relation to gastric cancer prevention. Methods: We conduc ted a critical analysis of the literature, with special emphasis on LA data and established the level of evidence and level of recommendation of the most controversial claims in the Joint Guidelines. Two rounds of voting were conducted according to the Delphi consensus technique and a Likert scale (from 0 to 4) was used to establish the "degree of agreement" among a panel of SLAGHNP ex perts. Results: There are few studies regarding diagnosis, treatment effectiveness and susceptibility to antibiotics of H. pylori in pediatric patients of LA. Based on these studies, extrapolations from adult studies, and the clinical experience of the participating expert panel, the following recom mendations are made. We recommend taking biopsies for rapid urease and histology testing (and samples for culture or molecular techniques, when available) during upper endoscopy only if in case of confirmed H. pylori infection, eradication treatment will be indicated. We recommend that selected regional centers conduct antimicrobial sensitivity/resistance studies for H. pylori and thus act as reference centers for all LA. In case of failure to eradicate H. pylori with first-line treatment, we recommend empirical treatment with quadruple therapy with proton pump inhibitor, amoxi cillin, metronidazole, and bismuth for 14 days. In case of eradication failure with the second line scheme, it is recommended to indicate an individualized treatment considering the age of the pa tient, the previously indicated scheme and the antibiotic sensitivity of the strain, which implies performing a new endoscopy with sample extraction for culture and antibiogram or molecular resistance study. In symptomatic children referred to endoscopy who have a history of first or se cond degree family members with gastric cancer, it is recommended to consider the search for H. pylori by direct technique during endoscopy (and eradicate it when detected). Conclusions: The evidence supports most of the general concepts of the NASPGHAN/ESPGHAN 2016 Guidelines, but it is necessary to adapt them to the reality of LA, with emphasis on the development of regional centers for the study of antibiotic sensitivity and to improve the correct selection of the eradication treatment. In symptomatic children with a family history of first or second degree gastric cancer, the search for and eradication of H. pylori should be considered.

Healthcare workers in Saudi Arabia perceive stress differently according to gender but not in cortisol levels: an immunoassay study

Background: Working in the healthcare sector is generally regarded as stress inductive, which hampers performance, yet one demanding constant accuracy. This dichotomy has led to numerous investigations on the impact from perceived stress on hospital workers but focused primarily on employing psychological methods to determine perceived stress. This study sought to employ an arguably more objective measure of chronic stress on female healthcare professionals in Saudi Arabia, by assaying the concentration of hair cortisol (HCC) in parallel with stress questionnaires.Methods: Pharmacists, nurses and lab workers participated in providing hair samples. Cortisol levels were subsequently quantified using immunoassay methods. Investigations considered the variables of age, gender, and smoking, hair coloring or bleaching or working in shifts on both stress perception and HCC.Results: On average chronic stress was perceived comparably between the different healthcare professions and not differ significantly against the female control group. However, chronic stress differed significantly between genders within the healthcare profession. In contrast, HCC levels showed no direct relation to stress perception with respect to either gender or profession. HCC did, however, show steady decreases with respect to age, as an indirect measure of experience, that contrasted against the identical scores for stress perception. Finally, night shifts, smoking or hair colouring did not produce a significant change on HCC in the healthcare cohorts.Conclusions: Women in the healthcare profession perceive stress higher irrespective of profession compared to men. Also show a pattern of decreasing levels of cortisol with increasing age despite reporting similar stress perception against younger participants.

¿Qué sabemos de la importancia de la microbiota intestinal a lo largo de la vida? / What do we know about intestinal microbiota through the life cycle?

Over the last decades, modern lifestyle and environment have contributed to a shift in gut microbial colonization patterns and composition. Not only intestinal but also extraintestinal disorders have been proposed to be linked to changes in the gut microbiome. There is increasing evidence from clinical, epidemiologic and animal studies exploring associations between the dysbiotic microbiome and an increased risk of allergic, inflammatory, autoimmune, and metabolic diseases. The fetus is essentially sterile until the amniotic sac ruptures. After that, the maternal microbiota of the mouth, intestine, vagina and urinary tract contribute to the initial seeding of neonatal microbiota. Newborns are mainly inoculated at birth in the passage through the birth canal (vertical transmission). A number of exposure events occur afterwards (horizontal transmission), and by age 2, an infant's microbiota composition becomes indistinguishable from that of an adult. In mammalian evolution, the potential loss or change in vertical transmission of microbiota from mother to offspring could be compensated through horizontally transmitted microbiota (fecally contaminated drinking and bathing water, frequent physical contact, social crowding, and large families). However, the progressive loss of vertically transmitted microbiota without horizontal replacement represents a cumulative birth cohort phenomenon. Events that decrease microbiota diversity have been classically associated with risk of disease

En las últimas décadas, el estilo de vida y el ambiente moderno, han contribuido a un cambio en los patrones y la composición de colonización microbiana intestinal. No sólo se ha propuesto que los trastornos intestinales, sino también los extraintestinales, estarían relacionados con cambios en la microbioma intestinal. Cada vez hay más evidencias de estudios clínicos, epidemiológicos y en animales que exploran las asociaciones entre el microbioma disbiótico y un mayor riesgo de enfermedades alérgicas, inflamatorias, autoinmunes y metabólicas. El feto es esencialmente estéril hasta que el saco amniótico se rompe. Después de eso, la microbiota materna de la boca, el intestino, la vagina y el tracto urinario contribuye a la siembra inicial de la microbiota neonatal. Los recién nacidos se inoculan principalmente al nacer en el pasaje a través del canal de parto (transmisión vertical). Después de una serie de eventos de exposición (transmisión horizontal), a los 2 años, la composición de microbiota de un bebé se vuelve indistinguible de la de un adulto. En la evolución de los mamíferos, la pérdida o cambio potencial en la transmisión vertical de la microbiota de la madre a la descendencia podría compensarse mediante la microbiota transmitida horizontalmente (agua potable y de baño contaminada con heces, contacto físico frecuente, aglomeración social y familias numerosas). Sin embargo, la pérdida progresiva de microbiota transmitida verticalmente sin reemplazo horizontal representa un fenómeno de cohorte de nacimiento acumulativo. Los eventos que disminuyen la diversidad de la microbiota se han asociado clásicamente con el riesgo de enfermedad.

Estudio de validez diagnóstica de la prueba de hemorragia oculta fecal en lactantes con proctocolitis alérgica inducida por proteína alimentaria / Diagnostic validity of fecal occult blood test in infants with food protein-induced allergic proctocolitis

Resumen: Introducción: La proctocolitis alérgica inducida por proteína alimentaria (PCA) es la forma más fre cuente de alergia alimentaria no mediada por IgE. El diagnóstico se realiza por prueba de provocación oral, sin embargo, no existe una prueba diagnóstica no invasiva para su diagnóstico. Frecuentemente en Chile se utiliza la prueba de hemorragia oculta fecal (PHOF) para confirmar PCA, pero no hay estudios que respalden su indicación. Objetivo: Determinar la validez diagnóstica de la PHOF en la evaluación de lactantes con PCA. Pacientes y Método: Estudio de casos y controles con recluta miento prospectivo de lactantes con rectorragia y sospecha de PCA y lactantes sanos, en quienes se realizó una PHOF. Se indicó dieta de exclusión a los casos y luego se confirmó diagnóstico de PCA mediante contraprueba. Resultados: Se incluyó a 25 casos y 29 controles sin diferencias signi ficativas en edad, sexo, tipo de parto, alimentación o edad materna. Los casos presentaron con mayor frecuencia comorbilidades alérgicas, uso de medicamentos y antecedentes familiares de alergia. La PHOF fue positiva en 84% de casos y en 34% de controles (p<0,001). La sensibilidad de la PHOF para diagnosticar PCA fue 84%, especificidad 66%, valor predictivo positivo 68% y valor predictivo nega tivo 83%. El área bajo la curva ROC fue de 0,75 (IC 95% 0,61-0,88). Conclusiones: Si bien la PHOF tiene sensibilidad adecuada para detectar PCA en lactantes con rectorragia, resulta alterada en más de un tercio de lactantes sanos por lo que no se recomienda su uso habitual para el diagnóstico de PCA.

Abstract: Introduction: Food protein-induced allergic proctocolitis (FPIAP) is the most frequent presenta tion of non-IgE mediated food allergy (FA). The diagnosis is made by oral food challenge, however, non-invasive diagnostic tests are not available. In Chile, the fecal occult blood test (FOBT) is fre quently used to confirm FPIAP, however, there are no studies that support this practice. Objective: To establish the diagnostic validity of FOBT in the evaluation of infants with FPIAP. Patients and Method: Case-control study with prospective recruitment of infants with rectal bleeding and suspicion of FPIAP, and controls were healthy infants, in whom the FOBT was conducted. All cases underwent an elimination diet, after which the diagnosis of FPIAP was confirmed by oral food cha llenge. Results: 25 cases and 29 controls were included without significant differences in age, gen der, type of delivery, feeding, and maternal age. The cases had higher rates of allergic comorbidities, medication use, and family history of allergy. The FOBT was positive in 84% of cases and in 34% of controls (p < 0.001). The sensitivity of the FOBT for the diagnosis of FPIAP was 84%, specificity was 66%, positive predictive value 68%, and the negative predictive value 83%. The area under the ROC curve was 0.75 (CI 95% 0.61-0.88). Conclusions: Although the FOBT has an adequate sensitivity to diagnose FPIAP in infants with rectal bleeding, this test had abnormal results in more than a third of healthy infants. Therefore, the routine use of FOBT is not recommended for the diagnosis of FPIAP.

Outcomes of a Physician Survey on the Type, Progression, Assessment, and Treatment of Neurological Disease in Mucopolysaccharidoses

Abstract The mucopolysaccharidosis (MPS) disorders are a group of rare, inherited lysosomal storage disorders. In each of the 11 MPS (sub)types, deficiency in a specific lysosomal enzyme (1 of 11 identified enzymes) leads to accumulation of glycosaminoglycans, resulting in cell, tissue, and multi-organ dysfunction. There is great heterogeneity in the clinical manifestations both between and within each MPS type. Somatic signs and symptoms include short stature, coarse facial features, skeletal and joint abnormalities, cardiorespiratory dysfunction, hepatosplenomegaly, and vision and hearing problems. In addition, patients with MPS I, II, III, and VII can have significant neurological manifestations, including impaired cognitive, language, and speech abilities, behavioral abnormalities, sleep problems, and/or epileptic seizures. Hydrocephalus is a frequent finding in patients with MPS I, II, and VI. Spinal cord compression can develop in almost all MPS disorders. Effective management and development of therapies that target these neurological manifestations warrant a profound understanding of their pathophysiology and progression in the different MPS types and best practices for evaluation and treatment. In order to obtain expert opinion addressing these topics we performed an online survey among an international group of experts with extensive experience in managing and treating MPS disorders. The results of this survey provide important insights into the management of neurological manifestations of MPS in clinical practice and are a valuable addition to current evidence.

Long-Term Galsulfase Treatment Associated With Improved Survival of Patients With Mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome): 15-Year Follow-Up From the Survey Study

Abstract Mucopolysaccharidosis VI (MPS VI) is a progressive lysosomal storage disorder with multiorgan and multisystemic pathology. Currently, galsulfase enzyme replacement therapy (ERT) is the only approved treatment for MPS VI. A crosssectional survey study of 121 patients with MPS VI conducted in 2001 to 2002 and a 10-year follow-up study of the same patients (resurvey study; ClinicalTrials.gov NCT01387854) found that those receiving galsulfase at any time showed physical improvements and a lower mortality rate (16.5%) versus treatment-naive patients (50%). After *15 years, galsulfasetreated patients (n » 104) continue to have a survival advantage over treatment-naive patients (n » 14), as demonstrated by a 24% versus 57% mortality rate. This survival advantage is further supported by data from the commercial use of galsulfase (2005-2016), which show a 5-year mortality rate for galsulfase-treated patients of 12.5%. Together, these findings suggest that galsulfase ERT can increase life expectancies for patients with MPS VI over a period of at least 15 years.

Neonatal Sepsis-Culture Positive Sepsis Vs Clinical Sepsis

The incidence of sepsis is increasing globally, with high morbidity and mortality. Diagnosis of neonatal sepsis is still a clinical and laboratory challenge. Though blood culture is gold standard, it sometimes gives false negative result. So, judgement of clinical condition along with various investigations is important

Impact of Elosulfase Alfa on Pain in Patients with Morquio A Syndrome over 52 Weeks: MOR-008: A Randomized, Double-Blind, Pilot Study

Abstract Patients with mucopolysaccharidosis (MPS), and Morquio A syndrome (MPS IVA) in particular, often report substantial pain burden. MOR-008 was a randomized, double-blind, pilot study assessing the safety and efficacy, including impact on patient-reported pain, of 52 weeks of treatment with elosulfase alfa (at a dose of 2.0 or 4.0 mg/kg/week) in patients with Morquio A syndrome (?7 years old). Assessment of pain at baseline revealed that patients (N = 25) had a mean number of pain locations of 5.7, mean pain intensity score of 4.6 (indicative of medium pain), and a mean number of selected pain descriptors of 7.4 words. Treatment with elosulfase alfa improved subjective pain score (reduced to 3.2), pain locations (reduced by a mean of 1 location), and pain descriptor words (reduced to 4.9 words) over 1 year (52 weeks), suggesting that elosulfase alfa can reduce pain in some patients with Morquio A.

Endoscopic retrograde cholangiopancreatography complications: Techniques to reduce risk and management strategies

Adverse events after endoscopic retrograde cholangiopancreatography (ERCP) are not uncommon and can be associated with tragic outcomes. Bleeding, perforation, and post-ERCP pancreatitis are the most common complications. Some events are unavoidable; others are associated with well described risk factors so that they can be either anticipated and/or measures can be taken for prevention or at least risk reduction. This review will focus on the more common complications after ERCP, their risk factors, and potential strategies for risk reduction. Additionally, recommendations for management of ERCP complications will be presented.

La infección materna por Helicobacter pylori no aumenta el riesgo de contraer la bacteria en el primer trimestre de vida de sus lactantes / Maternal infection due to Helicobacter pylori does not increase the risk of the infection in the first trimester of the life of their infants

Introducción: La infección por H. pylori se adquiere tempranamente en la infancia. Sin embargo, existe escasa información acerca del rol de la lactancia materna y la adquisición de la bacteria en la etapa neonatal/lactante. Objetivo: Evaluar algunos factores que afectan la adquisición de H. pylori en recién nacidos y lactantes hijos de madres infectadas. Pacientes y método: Reclutamiento consecutivo de binomios madre-hijo en maternidad, inmediatamente posparto. Luego de la firma de consentimiento informado, se obtuvo una muestra de deposición de la madre, previo al alta. Posteriormente se obtuvieron 3 muestras de deposición de los recién nacidos/lactantes a los 15, 60 y 90 días de vida, para la detección de antígeno en deposición de H. pylori (HpSAg monoclonal, sensibilidad 94% y especificidad 97%). Además se registraron variables socio-epidemiológicas y biomédicas. Resultados: Se reclutaron 32 binomios madre-hijo, 64 sujetos. Promedio de edad materna de 30,1 ± 5,1 años, 53% parto eutócico, 85% con lactancia materna exclusiva al final del seguimiento. Se encontró 13 madres (40%) infectadas por H. pylori. No hubo infección por H. pylori en los recién nacidos y lactantes a los 3 meses de seguimiento. No hubo diferencia significativa en el nivel socioeconómico entre madres infectadas versus no infectadas (ambos grupos en nivel socioeconómico muy alto: 28% y 32% respectivamente, p = 0,15), ni en el número de habitantes por domicilio entre madres infectadas y no infectadas (3,8 ± 0,8 vs 4,2 ± 1,8 personas, p = 0,18). Conclusión: A pesar de tener un alto porcentaje de madres infectadas por H. pylori, no hubo recién nacidos/lactantes infectados al tercer mes de vida. El rol protector de la lactancia maternal no se puede descartar.

Introduction: H. pylori infection is acquired early in childhood. However, there is little information available regarding the role of breastfeeding and neonatal acquisition of the infection. Objective: To evaluate factors affecting the acquisition of H. pylori in newborns and infants from infected mothers. Patients and method: Consecutive mothers and their newborns were recruited into the study from the maternity unit, immediately after delivery. After signing informed consent, one stool sample from the mother was obtained before hospital discharge. Three stool samples of the newborns were then collected at home at 15, 60, and 90 days of life, for the detection of H. pylori antigen (Monoclonal HpSAg, sensitivity 94% and specificity 97%). The socio-epidemiological and biomedical variables were also analysed using a questionnaire. Results: A total of 32 mother-child pairs (64 subjects) were enrolled. The mean maternal age was 30.1 ± 5.1 years, with 53% vaginal delivery, and 85% exclusively breastfed. There were 13 (40%) infected mothers. No H. pylori infection was detected in newborns and infants up to 3 months of follow-up. No significant differences were found in socioeconomic level between infected versus non-infected mothers (both groups mostly in the very high socioeconomic category: 28% and 32%, respectively, P = .15) and in the number of family members between infected versus non-infected mothers (3.8 ± 0.8 vs 4.2 ± 1.8 persons, P = .18). Conclusion: Despite having a significant percentage of H. pylori-infected mothers, no newborn was infected at the third month of life. The protective role of breastfeeding cannot be ruled out.