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OBJECTIVES: To compare the recently proposed Capsule Endoscopy-Crohn's Disease index (CE-CD) to pre-existing capsule endoscopy (CE) scores, to measure its precision and accuracy to predict adverse clinical outcomes in children with Crohn's disease (CD). METHODS: Children with CD who underwent CE at diagnosis and had, at least, 1-year follow-up postprocedure were selected. Capsule study was viewed and the different indices were independently scored by two trained paediatric gastroenterologists. The relationship between pre-existing scores and CE-CD was assessed by linear regression analysis. Clinical outcomes prediction assessment was based on receiver operating characteristics curves, survival analysis and Cox regression. Finally, interobserver agreement was measured. RESULTS: Fifty-nine patients were finally included. CE-CD showed a strong positive correlation with the Lewis score (ρ = 0.947) and the Capsule Endoscopy Crohn's Disease Activity Index (CECDAI) (ρ = 0.982). Both CE-CD and CECDAI were significant predictors of treatment escalation (hazard ratio 1.07 and 1.09, respectively, with both p-values < 0.01). However, no score predicted risk of hospital admission, surgery or clinical/endoscopic relapse. The presence of moderate-to-severe small bowel (SB) inflammation, defined as a score of ≥9 on CE-CD, provided a hazard ratio of treatment escalation of 2.6 (95% confidence interval: 1.3-5.3). This cut-off provided the optimal sensitivity/specificity pair: 48.4%/89.3%. No interobserver misclassification among inflammation categories given by CE-CD were observed (kappa 100%). CONCLUSION: CE-CD is a useful tool to document SB inflammation in children with CD. It correlates strongly with classical scores, can better predict need for treatment escalation and shows good interobserver agreement.
ABSTRACT
OBJECTIVE: There is sufficient evidence on the feasibility of a vaccine to prevent Helicobacter pylori infection. Modeling studies in low prevalence environments report a very probable long-term cost-effectiveness. The objective of this study was to quantify its efficiency in a local context. METHODS: The evolution of a cohort of newborns was simulated through a compartmental model representing a series of clinical situations regarding H. pylori infection and related diseases. The model was run under the assumption of both vaccination in the first year of life and no intervention. The time horizon was set as equivalent to the life expectancy and the perspective of the health system was taken into account. RESULTS: Vaccination against H. pylori would cost an average of 2,168/person more than no intervention. This would yield an average additional 0.32 quality-adjusted life years gained (QALY), which would entail an incremental cost-effectiveness ratio (ICER) of 7,196/QALY. For a willingness to pay of 24,506/QALY, 99.96% of the simulations were cost-effective at eighty-four years old. This threshold was crossed thirty years after vaccination. The variables that carried the most weight in explaining the variability of the ICER were, in this order, vaccine effectiveness, the incidence of infection in young children, and the price of the vaccine. Vaccination would cease to be cost-effective with a price greater than 3,634/dose or with effective population coverage less than 11%. CONCLUSIONS: When implemented in an environment with the epidemiological and economic characteristics of Southern Europe, a prophylactic vaccination against H. pylori would be cost-effective in the long run.
OBJECTIVE: Existen pruebas de la factibilidad de una vacuna para prevenir la infección por Helicobacter pylori. Modelizaciones en entornos de baja prevalencia informan de una muy probable coste-efectividad a largo plazo. El objetivo de este estudio fue cuantificar su eficiencia en un contexto local. METHODS: Se simuló la evolución de una cohorte de nacidos a través de un modelo compartimental representativo de varios estados clínicos en relación a la infección por H. pylori. Se ejecutó dicho modelo bajo las premisas de vacunación en el periodo de lactante y de no intervención. El horizonte temporal fue equivalente a la esperanza de vida y se tuvo en cuenta la perspectiva del sistema de salud. RESULTS: La vacunación frente a H. pylori costaría de media 2.168 /persona más que la no intervención. Con ello se obtendrían 0,32 años de vida ganados ajustados por calidad (AVAC), lo que implicaría una razón de coste-efectividad incremental (RCEI) media de 7.196 /AVAC. Para una disposición a pagar de 24.506 /AVAC, el 99,96% de las simulaciones resultaron coste-efectivas al alcanzar el horizonte temporal y se cruzó dicho umbral a partir de los treinta años de la vacunación. Las variables que más peso tuvieron para explicar la variabilidad de la RCEI fueron, en este orden, la efectividad vacunal, la incidencia de la infección en la primera infancia y el precio de la vacuna. La vacunación dejaría de ser coste-efectiva con un precio mayor de 3.634/vial o con una cobertura poblacional efectiva menor del 11%. CONCLUSIONS: Una vacunación frente a la infección por H. pylori administrada en la infancia sería coste-efectiva a largo plazo en un entorno con las características epidemiológicas y económicas del sur de Europa.
Subject(s)
Helicobacter Infections , Helicobacter pylori , Vaccines , Child , Humans , Infant, Newborn , Child, Preschool , Aged, 80 and over , Cost-Effectiveness Analysis , Cost-Benefit Analysis , Helicobacter Infections/epidemiology , Helicobacter Infections/prevention & control , Spain , Europe , Quality-Adjusted Life YearsABSTRACT
Fundamentos: existen pruebas de la factibilidad de una vacuna para prevenir la infección por helicobacter pylori. Modelizacio-nes en entornos de baja prevalencia informan de una muy probable coste-efectividad a largo plazo. El objetivo de este estudio fue cuantificar su eficiencia en un contexto local.métodos: se simuló la evolución de una cohorte de nacidos a través de un modelo compartimental representativo de varios estados clínicos en relación a la infección por h. Pylori. Se ejecutó dicho modelo bajo las premisas de vacunación en el periodo de lactante y de no intervención. El horizonte temporal fue equivalente a la esperanza de vida y se tuvo en cuenta la perspectiva del sistema de salud.resultados: la vacunación frente a h. Pylori costaría de media 2.168 /persona más que la no intervención. Con ello se obten-drían 0,32 años de vida ganados ajustados por calidad (avac), lo que implicaría una razón de coste-efectividad incremental (rcei) media de 7.196 /avac. Para una disposición a pagar de 24.506 /avac, el 99,96% de las simulaciones resultaron coste-efectivas al alcanzar el horizonte temporal y se cruzó dicho umbral a partir de los treinta años de la vacunación. Las variables que más peso tuvieron para explicar la variabilidad de la rcei fueron, en este orden, la efectividad vacunal, la incidencia de la infección en la primera infancia y el precio de la vacuna. La vacunación dejaría de ser coste-efectiva con un precio mayor de 3.634/vial o con una cobertura poblacional efectiva menor del 11%.conclusiones: una vacunación frente a la infección por h. Pylori administrada en la infancia sería coste-efectiva a largo plazo en un entorno con las características epidemiológicas y económicas del sur de europa.(AU)
Background: there is sufficient evidence on the feasibility of a vaccine to prevent helicobacter pylori infection. Modeling studies in low prevalence environments report a very probable long-term cost-effectiveness. The objective of this study was to quantify its efficiency in a local context.methods: the evolution of a cohort of newborns was simulated through a compartmental model representing a series of clinical situations regarding h. Pylori infection and related diseases. The model was run under the assumption of both vaccination in the first year of life and no intervention. The time horizon was set as equivalent to the life expectancy and the perspective of the health system was taken into account.results: vaccination against h. Pylori would cost an average of 2,168/person more than no intervention. This would yield an average additional 0.32 quality-adjusted life years gained (qaly), which would entail an incremental cost-effectiveness ratio (icer) of 7,196/qaly. For a willingness to pay of 24,506/qaly, 99.96% of the simulations were cost-effective at eighty-four years old. This threshold was crossed thirty years after vaccination. The variables that carried the most weight in explaining the variability of the icer were, in this order, vaccine effectiveness, the incidence of infection in young children, and the price of the vaccine. Vaccination would cease to be cost-effective with a price greater than 3,634/dose or with effective population coverage less than 11%.(AU)
Subject(s)
Humans , Male , Female , Stomach Neoplasms/economics , Stomach Neoplasms/immunology , Vaccines , Helicobacter , VaccinationABSTRACT
INTRODUCTION: European guidelines for the diagnosis of celiac disease (CD) have been updated in 2020. The primary objective was to review the compliance with the diagnostic criteria for CD, according to ESPGHAN 2012. Secondarily, to describe the clinical characteristics of the patients and to assess the changes that would be implied by the application of the new 2020 criteria. PATIENTS AND METHODS: Retrospective multicenter study in which 10 centers participated. Patients from 0 to 16 years old with a new diagnosis of CD in 2018-2019 were included. Clinical, serological variables and the performance of intestinal biopsy (IB) were collected. RESULTS: 163 patients were included (57% female) with a median age of 7.6 years (SD 4.4). The form of presentation was: 47.8% classical, 30.7% no classical and 21.5% asymptomatic, with differences depending on age. Total IgA and anti-transglutaminase IgA antibodies were performed in all centers as the first diagnostic step. IgA anti-endomysial antibodies (EMA) were performed in 80%, and HLA haplotype in 95%. Of the total, 78 cases (47.9%) met criteria for not performing intestinal biopsy (IB). IB was indicated in the remaining 85 patients, but was not performed in 29 cases (17.8%). The performance of IB was lower in the secondary hospitals than in the tertiary ones (p < 0.05). If we applied the ESPGHAN 2020 criteria, we would disregard the HLA study, and 21 more patients would not have required IB (going from 47.9% to 60.7% of the total). CONCLUSIONS: Discrepancies are observed in the application of the ESPGHAN 2012 diagnostic criteria due to the different accessibility to EMA and endoscopic IB in secondary centers. With the ESPGHAN-2020 criteria, around 60% of patients will be able to be diagnosed without IB, provided that the determination of EMA is ensured.
Subject(s)
Celiac Disease , Adolescent , Autoantibodies/analysis , Biopsy , Celiac Disease/diagnosis , Child , Child, Preschool , Female , Humans , Infant , Infant, Newborn , Male , Retrospective Studies , TransglutaminasesABSTRACT
BACKGROUND: Children with autoimmune hepatitis (AIH) often exhibit particular features. Accordingly, seven pediatric-specific criteria have been proposed. AIM: To develop a prediction model based on them, transform it into a scoring system and study its accuracy. METHODS: A cohort of children under study for liver disease was consecutively selected. AIH diagnosis was based on classical criteria. Already proposed pediatric criteria were recorded. The best possible regression model was selected, and the beta coefficient of each criterion was translated into a whole number (points). Total scores were obtained following the points system and the best cut-off was calculated. Subsequently, accuracy of the diagnostic score was studied in the validation set. RESULTS: Among 212 included patients, 100 had AIH. The score included 5 criteria: autoantibodies (0-2 points), hypergammaglobulinemia, exclusion of viral hepatitis, exclusion of Wilson's disease (1 point each) and liver histology (3 points). In addition, a normal cholangiogram is mandatory. The validation set was formed of 70 patients (24 with AIH). In this subsample, a score of ≥6 renders a sensitivity/specificity of 95.8%/100%. The area under the receiver operating characteristic curve was 97.1%. CONCLUSION: Pediatric-specific criteria for the diagnosis of AIH can be reliably used as a scoring system.
Subject(s)
Hepatitis, Autoimmune/diagnosis , Autoantibodies/blood , Child , Child, Preschool , Cross-Sectional Studies , Female , Humans , Hypergammaglobulinemia/blood , Male , Prospective Studies , Sensitivity and SpecificityABSTRACT
PURPOSE: Classical criteria for diagnosis of autoimmune hepatitis (AIH) are intended as research tool and are difficult to apply at patient's bedside. We aimed to study the accuracy of simplified criteria and the concordance with the expert diagnosis based on the original criteria. METHODS: A cohort of children under study for liver disorder was selected through consecutive sampling to obtain the prevalence of AIH within the group of differential diagnoses. AIH was defined, based on classical criteria, through committee review of medical reports. Validity indicators of the simplified criteria were obtained in an intention to diagnose approach. Optimal cut-off and the area under the receiver operating characteristic (ROC) curve were calculated. RESULTS: Out of 212 cases reviewed, 47.2% were AIH. For the optimal cut-off (6 points), the simplified criteria showed a sensitivity of 72.0% and a specificity of 96.4%, with a 94.7% positive and a 79.4% negative predictive value. The area under the ROC curve was 94.3%. There was a good agreement in the pre-treatment concordance between the classical and the simplified criteria (kappa index, 0.775). CONCLUSION: Simplified criteria provide a moderate sensitivity for the diagnosis of AIH, but may help in indicating treatment in cases under suspicion with 6 or more points.
Subject(s)
Cyclosporine/adverse effects , Headache/diagnosis , Hypertension/diagnosis , Immunosuppressive Agents/adverse effects , Posterior Leukoencephalopathy Syndrome/chemically induced , Seizures/diagnosis , Central Nervous System Neoplasms/therapy , Child , Diagnosis, Differential , Female , Graft vs Host Disease/prevention & control , Headache/etiology , Hematopoietic Stem Cell Transplantation , Humans , Hypertension/etiology , Magnetic Resonance Imaging , Posterior Leukoencephalopathy Syndrome/diagnosis , Precursor Cell Lymphoblastic Leukemia-Lymphoma/therapy , Seizures/etiologySubject(s)
Epigenesis, Genetic , Insemination, Artificial , Spastic Paraplegia, Hereditary/genetics , Spermatozoa/physiology , Tissue Donors , Adult , Child , Female , Humans , Infant , Infant, Newborn , Male , PregnancyABSTRACT
BACKGROUND: The presence of a single umbilical artery (SUA) is a fetal soft marker of congenital abnormalities. Among the most common related malformations, there are cardiological, nephrourological and digestive anomalies, most of which are considered to have a vascular etiology. There is an association between increased incidence of intrauterine growth retardation and adverse perinatal indicators, but whether this association is due to related anomalies or isolated SUA (iSUA) is controvisal. METHODS: We reviewed 96 cases of iSUA and non-isolated SUA (niSUA), diagnosed in a period of two years in a referral hospital for high-risk pregnancies. Data on prenatal explorations, including fetal ultrasonography and karyotyping, were obtained. niSUA was diagnosed when no malformations were found prenatally or in postnatal evaluation. RESULTS: Sixty-six newborns (68.8%) had no other anomalies and 30 (31.3%) presented with a variety of malformations including heart diseases, urophaties, digestive, nervous and musculoskeletal disorders, genetic abnormalities and complex malformations. Cardiological and nephrourological abnormalities were found to be the most frequent association with a SUA (both in 23.8% of malformed SUA newborns). Intrauterine growth restriction was not higher in iSUA newborns than in a normal population. Ultrasound allowed optimal prenatal diagnosis in most cases. CONCLUSIONS: The prognosis of the fetus with a SUA is determined by the presence of other malformations observed by an expert sonographer. If no other findings are made, only a routine physical examination should be performed in newborns, but no other complementary examinations are required.
Subject(s)
Fetus/abnormalities , Heart Defects, Congenital/epidemiology , Urinary Tract/abnormalities , Abnormalities, Multiple/epidemiology , Adult , Birth Weight , Body Height , Female , Humans , Infant, Newborn , Kidney/abnormalities , Pregnancy , Pregnancy, High-Risk , Prognosis , Retrospective Studies , Single Umbilical Artery/diagnostic imaging , Ultrasonography, PrenatalABSTRACT
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