Achieving Enteral Autonomy in Children with Intestinal Failure Following Inpatient Admission: A Case Series.

We describe cases of intestinal failure wherein inpatient admission was critical toward enteral autonomy. We performed a retrospective chart review of 6 children with long-term parenteral nutrition dependence who were weaned from parenteral nutrition after admission. Admissions included feeding and medication titration, interdisciplinary care, and a home parenteral nutrition team consultation.

Baseline Characteristics of Adult Patients Treated and Never Treated with Teduglutide in a Multinational Short Bowel Syndrome and Intestinal Failure Registry.

The Short Bowel Syndrome (SBS) Registry (NCT01990040) is a multinational real-world study evaluating the long-term safety of teduglutide in patients with SBS and intestinal failure (SBS-IF) in routine clinical practice. This paper describes the study methodology and baseline characteristics of adult patients who have (ever-treated) or have never (never-treated) received teduglutide. A total of 1411 adult patients (679 ever-treated; 732 never-treated) were enrolled at 124 sites across 17 countries. The mean (standard deviation [SD]) age at enrollment was 55.4 (15.46) years, and 60.2% of patients were women. Crohn's disease was the most common cause of major intestinal resection in both ever-treated (34.1%) and never-treated patients (20.4%). A similar proportion of ever-treated and never-treated patients had a prior history of colorectal polyps (2.7% vs. 3.6%), whereas proportionally fewer ever-treated patients reported a history of colorectal cancer (1.8% vs. 6.2%) or any malignancy (17.7% vs. 30.0%) than never-treated patients. Never-treated patients received a numerically greater mean (SD) volume of parenteral nutrition and/or intravenous fluids than ever-treated patients (12.4 [8.02] vs. 10.1 [6.64] L/week). Ever-treated patients received a mean teduglutide dosage of 0.05 mg/kg/day. This is the first report of patient baseline characteristics from the SBS Registry, and the largest cohort of patients with SBS-IF to date. Overall, ever-treated and never-treated patients had similar baseline characteristics. Differences between treatment groups may reflect variations in patient selection and degree of monitoring.

Translation into Brazilian Portuguese and transcultural adaptation and validation of the treatment specific questionnaire Home Parenteral Nutrition- Quality of Life (HPN-QOL©).

BACKGROUND & AIMS: Home Parenteral Nutrition (HPN) is the main treatment for patients with chronic intestinal failure. It is commonly prescribed for nutritional recovery, survival increase and, whenever possible, improvement of quality of life. As there are no validated instruments in Brazilian Portuguese to be used in these patients, the objective of this study was to carry out the transcultural adaptation and validation of Home Parenteral Nutrition - Quality of life (HPN-QOL©) into Brazilian Portuguese. METHODS: This observational and cross-sectional study was conducted at the multidisciplinary short-bowel syndrome clinic (AMULSIC) of the Hospital das Clínicas of the University of São Paulo Medical School (HC-FMUSP). A five-stage protocol was adopted for the transcultural adaptation: initial translation; synthesis; reverse translation; experts committee and pre-test. The adapted questionnaire was applied to a convenience (representative) sample (n = 16) and Cronbach's Alpha Coefficient, Intraclass Correlation Coefficient (ICC), and Bland-Altman Test were submitted for the analysis of the internal consistency and intraobserver and interobserver reproducibility. RESULTS: The transcultural adaptation was considered excellent (Content Validity Index = 100%). The internal consistency was satisfactory for most of the scales (16/19), and α > 0.70 was 84.21%. ICC values revealed high intraobserver and interobserver reproducibility in most of the scales. No significant difference was observed between intraobservers and interobservers in any of the questions (p > 0.05). CONCLUSIONS: The questionnaire was shown as adapted and valid for use in Brazil. Future trials with a higher sample are yet to be developed to shed light on specific scales that were inconsistent. It's expected that this would contribute for the usual quality of life assessment for individuals treated with HPN in Brazil.

Bioelectrical impedance phase angle and nutritional status in children with intestinal failure on prolonged parenteral nutrition.

OBJECTIVE: To compare the phase angle (PhA) through bioelectrical impedance (BIA) of children with intestinal failure (IF) using prolonged parenteral nutrition (PN) followed by an Intestinal Rehabilitation Program, with a control group. METHODS: Children under 10 years of age with IF using prolonged PN for >60 days (study group) were included. The control group consisted of healthy children without chronic pathologies, matched by sex and age. Anthropometric parameters evaluated were: weight, height, weight/age z-score (W/A), height/age z-score (H/A), BMI, BMI/A z-score, arm circumference, triceps skinfold, subscapular skinfold, mid-arm muscle circumference. BIA parameters were resistance (R), reactance (Xc), and phase angle (PhA). RESULTS: Twenty-eight children were included in the study group, median (IQR) age was 11 (8-27) months, 53.6 % were male. In the control group, 28 children were included, median (IQR) age was 12.5 (8-24.7) months, 50 % were male. Children from the study group had W/A z-scores and H/A z-scores significantly lower than controls. There was no significant difference between PhA in the study group and controls, [median (IQR) 4.3° (3.8;4.6) vs 4.0° (3.8;5.4) respectively, p = 0.980]. Prematurity was significantly higher in the study group than in the controls, but there was no significant correlation between gestational age at birth and PhA of the children from the study group. CONCLUSION: Children with IF using prolonged PN showed lower W/A and H/A compared to the control group, but without significant difference between the PhA of children with IF compared to controls.

Factores asociados al desenlace en niños con mal rotación intestinal manejados en el Hospital Infantil de San Vicente Fundación, Medellín, Colombia / Factors associated with outcome in children with intestinal malrotation managed at the Hospital Infantil of San Vicente Fundación, Medellín, Colombia

Introducción. El intestino primitivo rota durante la vida embrionaria. Cuando ocurre de forma inadecuada aparece la malrotación intestinal que puede llevar a la obstrucción o al vólvulo del intestino medio. La incidencia disminuye con el aumento de la edad. La malrotación intestinal es una de las principales causas de complicaciones del tracto gastrointestinal en la edad pediátrica. Métodos. Estudio retrospectivo, observacional, transversal y analítico, de la experiencia durante 10 años en pacientes menores de 15 años con diagnóstico de malrotación intestinal, tratados en el Hospital Infantil de San Vicente Fundación, en Medellín, Colombia. Se buscó la asociación entre variables demográficas, clínicas e imagenológicas con los desenlaces. Resultados. Se obtuvieron 58 pacientes con malrotación intestinal, 65 % menores de 1 año. En 29,3 % de los pacientes se hizo el diagnóstico con la presentación clínica; los síntomas predominantes fueron distensión abdominal y emesis. En el 24,1 % se confirmó el diagnóstico con imágenes. Las variables con una diferencia estadísticamente significativa a favor de encontrar una malrotación complicada fueron choque séptico (OR=11,7), síndrome de respuesta inflamatoria sistémica (OR=8,4) y deshidratación (OR=5,18). Conclusiones. La malrotación intestinal tiene complicaciones como perforación, peritonitis, vólvulo e intestino corto. El vólvulo se acompaña de shock y sepsis, con mortalidad hasta del 50 %. Las imágenes diagnósticas son una ayuda, pero no se puede basar la conducta médica en estas porque ninguna imagen garantiza el diagnóstico definitivo. Los signos de alarma son poco específicos. En menores de un año con emesis, distensión y dolor abdominal se debe sospechar malrotación intestinal.

Introduction. The primitive intestine rotates during embryonic life. When it occurs inappropriately, intestinal malrotation appears, which can lead to obstruction or midgut volvulus. The incidence decreases when age increases. Intestinal malrotation is one of the main causes of complications of the gastrointestinal tract in pediatric age. Method. Retrospective, observational, cross-sectional and analytical study of the experience over 10 years in patients under 15 years of age with a diagnosis of intestinal malrotation, treated at Hospital Infantil of San Vicente Fundación, in Medellín, Colombia. The association between demographic, clinical and imaging variables with the outcomes was sought. Results. There were 58 patients with intestinal malrotation, 65% under one year of age. In 29.3% of patients, intestinal malrotation was diagnosed clinically. The predominant symptoms were abdominal distension and emesis. In 24.1% the diagnosis was confirmed with imaging. The variables with a statistically significant difference in favor of finding a complicated malrotation were septic shock (OR=11.7), systemic inflammatory response syndrome (OR-8.4), and dehydration (OR=5.18). Conclusions. Malrotation has complications such as perforation, peritonitis, volvulus, and short bowel. Volvulus is accompanied by shock and sepsis, with mortality of up to 50%. Diagnostic images are helpful, but medical conduct cannot be based on them because no image guarantees a definitive diagnosis. The warning signs are not very specific. In children under one year of age with emesis, distension and abdominal pain, intestinal malrotation should be suspected.

Usefulness and description of the intestinal bypass technique in children with short bowel syndrome: report of a Mexican cohort.

BACKGROUND: Short bowel syndrome (SBS) is one of the most frequent causes of intestinal failure, needing parenteral nutrition to maintain an energy-protein and water-electrolyte balance. At the Hospital Infantil de México Federico Gómez (HIMFG), the formation of two stomas is a technique used for intestinal rehabilitation, where the use of residue through the bypass technique (BT) helps to maintain gastrointestinal functionality, water-electrolyte, and nutritional stability. This study aimed to describe the technique of using intestinal residue through BT as a treatment strategy in intestinal rehabilitation and its effect on the biochemical and nutritional status of pediatric patients with SBS. METHODS: An analytical and retrospective cross-sectional study was performed in patients hospitalized at HIMFG with SBS who underwent BT during their hospital stay between 2019 and 2020 and then followed up for 8 weeks. RESULTS: A total of 10 patients were included in this study, with a mean age of 24 months; 50% were female. BT was able to reduce the inflammatory process in the liver caused by the continuous use of parenteral nutrition; enteral caloric intake increased from 25.32 kcal/kg/day to 72.94 kcal/kg/day, but it was insufficient to improve their nutritional status. CONCLUSIONS: BT is a safe and effective alternative in intestinal rehabilitation in patients with SBS to stimulate trophism and intestinal functionality, allowing a progression of enteral feeding and a decrease in the hepatic inflammatory process that occurs in these patients with prolonged parenteral nutrition.

INTRODUCCIÓN: El síndrome de intestino corto (SIC) es una de las causas más frecuentes de insuficiencia intestinal que requiere del uso de nutrición parenteral para mantener un balance energético-proteico e hidroelectrolítico. En el Hospital Infantil de México Federico Gómez (HIMFG) la formación de dos estomas es una técnica empleada para la rehabilitación intestinal, donde con el aprovechamiento de residuo mediante la técnica de puenteo (TP) se ayuda a mantener la funcionalidad gastrointestinal, equilibrio hidro-electrolítico y estabilidad nutricional. El objetivo de este estudio fue describir la técnica del aprovechamiento de residuo intestinal mediante TP como estrategia de tratamiento en la rehabilitación intestinal y su efecto en el estado bioquímico y nutricional de pacientes pediátricos con SIC. MÉTODOS: Se llevó a cabo un estudio transversal analítico y retrospectivo en pacientes hospitalizados en el HIMFG con SIC en quienes se realizó la TP durante su estancia intrahospitalaria entre 2019 y 2020. RESULTADOS: Se incluyeron 10 pacientes en este estudio, con una edad promedio de 24 meses, y el 50% de sexo femenino. La TP logró disminuir el proceso inflamatorio hepático ocasionado por el uso continuo de nutrición parenteral; la ingesta calórica por vía enteral incrementó de 25.32 kcal/kg/día a 72.94 kcal/kg/día, pero fue insuficiente para mejorar el estado nutricional. CONCLUSIONES: La TP es una alternativa segura y efectiva en la rehabilitación intestinal en pacientes con SIC para estimular el trofismo y funcionalidad intestinal, permitiendo una progresión de la alimentación enteral y disminución del proceso inflamatorio hepático que se presentan en estos pacientes con nutrición parenteral prolongada.

Iodine status in children with intestinal failure.

OBJECTIVES: Children on long-term parenteral nutrition (PN) are at high risk of iodine deficiency (ID). However, most available information comes from cross-sectional studies. We investigated the iodine status, associated factors, and prevalence of hypothyroidism in children with intestinal failure (IF) who were followed up longitudinally. METHODS: This was a cohort study of children with IF monitored for urine iodine concentration (UIC), iodine intake, serum selenium concentration, and thyroid function in an intestinal rehabilitation program. The outcome variable ID was defined as a UIC value < 100 µg/L. Adjusted generalized estimating equations were used to assess the effects of the exposure variables on the UIC. RESULTS: Twenty-four patients aged 62.7 (39.1; 79.7) months who received PN for 46.5 (21.5) months were included. The average energy supply was 81.2 kcal/kg/day, 77.6% of which was provided by PN. An average of 5.2 UIC measurements per patient were performed. ID prevalence decreased from baseline (83.3%) to the last assessment (45.8%). Three patients had hypothyroidism secondary to iodine and selenium combined severe deficiency. Iodine intake from enteral or oral nutritional formulas was positively associated with UIC (ß = 0.71 [0.35, 1.07]; p < 0.001). Meeting approximately 80% of the estimated average requirement for iodine from nutritional formulas resulted in a greater probability of normal UIC values. CONCLUSION: ID is highly prevalent in children with IF who receive long-term PN and its frequency decreases with iodine intake from nutritional formulas. Severe combined iodine and selenium deficiencies are associated with the development of hypothyroidism in these patients.

Usefulness and description of the intestinal bypass technique in children with short bowel syndrome: report of a Mexican cohort / Utilidad y descripción de la técnica de puenteo intestinal en niños con síndrome de intestino corto: reporte de una cohorte en México

Abstract Background: Short bowel syndrome (SBS) is one of the most frequent causes of intestinal failure, needing parenteral nutrition to maintain an energy-protein and water-electrolyte balance. At the Hospital Infantil de México Federico Gómez (HIMFG), the formation of two stomas is a technique used for intestinal rehabilitation, where the use of residue through the bypass technique (BT) helps to maintain gastrointestinal functionality, water-electrolyte, and nutritional stability. This study aimed to describe the technique of using intestinal residue through BT as a treatment strategy in intestinal rehabilitation and its effect on the biochemical and nutritional status of pediatric patients with SBS. Methods: An analytical and retrospective cross-sectional study was performed in patients hospitalized at HIMFG with SBS who underwent BT during their hospital stay between 2019 and 2020 and then followed up for 8 weeks. Results: A total of 10 patients were included in this study, with a mean age of 24 months; 50% were female. BT was able to reduce the inflammatory process in the liver caused by the continuous use of parenteral nutrition; enteral caloric intake increased from 25.32 kcal/kg/day to 72.94 kcal/kg/day, but it was insufficient to improve their nutritional status. Conclusions: BT is a safe and effective alternative in intestinal rehabilitation in patients with SBS to stimulate trophism and intestinal functionality, allowing a progression of enteral feeding and a decrease in the hepatic inflammatory process that occurs in these patients with prolonged parenteral nutrition.

Resumen Introducción: El síndrome de intestino corto (SIC) es una de las causas más frecuentes de insuficiencia intestinal que requiere del uso de nutrición parenteral para mantener un balance energético-proteico e hidroelectrolítico. En el Hospital Infantil de México Federico Gómez (HIMFG) la formación de dos estomas es una técnica empleada para la rehabilitación intestinal, donde con el aprovechamiento de residuo mediante la técnica de puenteo (TP) se ayuda a mantener la funcionalidad gastrointestinal, equilibrio hidro-electrolítico y estabilidad nutricional. El objetivo de este estudio fue describir la técnica del aprovechamiento de residuo intestinal mediante TP como estrategia de tratamiento en la rehabilitación intestinal y su efecto en el estado bioquímico y nutricional de pacientes pediátricos con SIC. Métodos: Se llevó a cabo un estudio transversal analítico y retrospectivo en pacientes hospitalizados en el HIMFG con SIC en quienes se realizó la TP durante su estancia intrahospitalaria entre 2019 y 2020. Resultados: Se incluyeron 10 pacientes en este estudio, con una edad promedio de 24 meses, y el 50% de sexo femenino. La TP logró disminuir el proceso inflamatorio hepático ocasionado por el uso continuo de nutrición parenteral; la ingesta calórica por vía enteral incrementó de 25.32 kcal/kg/día a 72.94 kcal/kg/día, pero fue insuficiente para mejorar el estado nutricional. Conclusiones: La TP es una alternativa segura y efectiva en la rehabilitación intestinal en pacientes con SIC para estimular el trofismo y funcionalidad intestinal, permitiendo una progresión de la alimentación enteral y disminución del proceso inflamatorio hepático que se presentan en estos pacientes con nutrición parenteral prolongada.

The Impact of Teduglutide on Real-Life Health Care Costs in Children with Short Bowel Syndrome.

OBJECTIVES: To analyze the real-life health care costs of home parenteral nutrition (HPN) in children with short bowel syndrome with intestinal failure (SBS-IF) before and after treatment with teduglutide, and to compare those with costs of children with SBS-IF not treated with teduglutide. STUDY DESIGN: All consecutive children with SBS-IF on HPN treated with subcutaneous teduglutide starting from 2018 through 2020 in a tertiary French referral center were retrospectively included. These patients were matched to children with SBS-IF on HPN followed during the same 3-year period who were eligible for the teduglutide but were not treated. HPN direct medical costs included home-care charges, HPN bags, hospital admissions, and teduglutide. A comparison of costs before/after treatment and between patients treated/not treated was performed. RESULTS: Sixty children were included: 30 (50%) were treated with teduglutide and 30 (50%) were untreated. In the treated group, the median total costs of HPN significantly decreased after 1 (P < .001) and 2 years of treatment (P < .001) from 59 454 euros/year/patient to 43 885 euros/year/patient and 34 973 euros/year/patient, respectively. When we compared patients treated and not treated, the total HPN costs/year/patient were similar at baseline (P = .6) but were significantly lower in the teduglutide-treated group after 1 (P = .006) and 2 years of treatment (P < .001). When we added the cost of teduglutide into the analysis, the total cost increased significantly in the treated group and remained much greater even after modeling a reduction in the cost of the drug to one-third the present cost and PN weaning (P < .001). CONCLUSIONS: Treatment with teduglutide is associated with a significant reduction in the annual costs of HPN but still remains expensive because of the drug itself. Finding cost-saving strategies is essential.

Fallo intestinal crónico secundario a síndrome de intestino corto: papel actual de la cirugía, la rehabilitación médica y el trasplante intestinal. Informe evolutivo a 16 años de manejo multidisciplinario / Chronic intestinal failure due to short bowel syndrome: current role of surgery, medical rehabilitation, and intestinal transplantation. A progress report after 16 years of multidisciplinary management

RESUMEN Antecedentes: la insuficiencia intestinal, en su grado más grave, se conoce como fallo intestinal crónico (FIC). Las últimas décadas han sido testigo de la incorporación, evolución y perfeccionamiento de tratamientos que, en su conjunto, se denominan rehabilitación intestinal y abarcan tanto procedimientos quirúrgicos como tratamientos médicos que, llevados a cabo por equipos multi e interdisciplinarios, alcanzan altas tasas de éxito. Objetivo: describir los resultados de 16 años en el tratamiento de pacientes con FIC secundario a síndrome de intestino corto (SIC), y la evolución del papel de la cirugía, la rehabilitación médica y el trasplante. Material y métodos: análisis retrospectivo de una base de datos prospectiva de pacientes con fallo intestinal crónico secundario a SIC entre febrero de 2006 y marzo de 2022. Resultados: se incluyeron 492 pacientes (368 adultos-A y 124 pediátricos-P). Grupo A: 111 pacientes recibieron cirugía de reconstrucción autóloga del tracto gastrointestinal (CRATGI), 16 péptido semisintético similar al glucagón de tipo 2 (sGLP-2); el 83% logró la rehabilitación, con un 77% de supervivencia a 10 años; el 6,8% (17 pacientes) requirió trasplante intestinal (TxI), con un 89% de independencia de la nutrición parenteral (NP) al año y supervivencia post-TxI del 29% a los 10 años. Grupo B: 18 recibieron CRATGI; 9, enteroplastia serial transversa (STEP); y 6, sGLP-2; el 52% se rehabilitó, con una supervivencia del 69% a los 10 años; 28 pacientes recibieron TxI, con 69% de independencia de nutrición parenteral (NP) al año y supervivencia del 39% a los 10 años. Conclusión: los resultados presentados resaltan el papel central de la cirugía y la rehabilitación médica para alcanzar la suficiencia intestinal.

ABSTRACT Background: Severe intestinal insufficiency is known as chronic intestinal failure (CIF). In recent decades, medical treatments and surgical procedures have been incorporated, developed and improved under the name intestinal rehabilitation. When performed by multiand interdisciplinary teams, these treatments have high success rates. Objective: The aim of present study is to describe the 16-year outcomes in the management of patients with CIF secondary to short bowel syndrome (SBS) and the role of surgery, medical rehabilitation, and transplantation. Material and methods: We conducted a retrospective analysis on a prospective database of patients treated with chronic intestinal failure due to SBS between February 2006 and March 2022. Results: A total of 492 patients (368 adults (Group A) and 124 pediatric patients (Group B)] were included. Group A: 111 patients underwent autologous gastrointestinal reconstruction surgery (AGIRS), 16 were treated with semisynthetic glucagon-like peptide-2 (sGLP2); 83% achieved rehabilitation, with 77% survival at 10 years; 6.8% (17 patients) required intestinal transplantation (ITx), with 89% independence from parenteral nutrition (PN) at 1 year and post-ITx survival of 29% at 10 years. Group B: 18 patients underwent AGIRS; 9 underwent serial transverse enteroplasty (STEP); and 6 received sGLP2; 52% were rehabilitated, with 69% survival at 10 years; 28 patients received ITx, with 69% independence from PN at 1 year and 39% survival at 10 years. Conclusion: These results highlight the central role of surgery and medical rehabilitation in the recovery of intestinal function.

Consenso colombiano de Falla Intestinal Crónica en Síndrome de Intestino Corto / Colombian Consensus on Chronic Intestinal Failure in Short Bowel Syndrome

Introducción. Los términos falla intestinal crónica, síndrome de intestino corto (SIC) y nutrición parenteral total son muy frecuentes en la práctica clínica cotidiana.El objetivo de esta guía fue establecer un marco de referencia de práctica clínica basado en el mejor de nivel de evidencia en pacientes con falla intestinal crónica secundaria a síndrome de intestino corto. Métodos. Se estableció un grupo de expertos interdisciplinarios en el manejo de la falla intestinal crónica quienes, previa revisión de la literatura escogida, se reunieron de manera virtual acogiendo el método Delphi para discutir una serie de preguntas seleccionadas, enfocadas en el contexto terapéutico de la falla intestinal crónica asociada al síndrome de intestino corto. Resultados. La recomendación del grupo de expertos colombianos es que se aconseje a los pacientes con SIC consumir dietas regulares de alimentos integrales que genere hiperfagia para compensar la malabsorción. Las necesidades proteicas y energéticas dependen de las características individuales de cada paciente; la adecuación del régimen debe ser evaluada a través de pruebas clínicas, antropométricas y parámetros bioquímicos. Se sugiere, especialmente a corto plazo después de la resección intestinal, el uso de análogos de somatostatina para pacientes con yeyunostomía de alto gasto en quienes el manejo de líquidos y electrolitos es problemático. En pacientes con SIC, que son candidatos a tratamiento con enterohormonas, Teduglutida es la primera opción. Conclusión. Existen recomendaciones en el manejo integral de la rehabilitación intestinal respaldadas ampliamente por este consenso y es importante el reconocimiento de alternativas terapéuticos enmarcadas en el principio de buenas prácticas clínicas.

Introduction. The terms chronic intestinal failure, short bowel syndrome (SBS), and total parenteral nutrition are very common in daily clinical practice. The objective of this guideline was to establish a reference framework for clinical practice based on the best level of evidence in patients with chronic intestinal failure secondary to short bowel syndrome. Methods. A group of interdisciplinary experts in the management of chronic intestinal failure was established who, after reviewing the selected literature, met virtually using the Delphi method to discuss a series of selected questions, focused on the therapeutic context of chronic intestinal failure associated with short bowel syndrome. Results. The recommendation of the Colombian expert group is that patients with SBS be advised to consume regular diets of whole foods that generate hyperphagia to compensate malabsorption. Protein and energy needs depend on the individual characteristics of each patient; the adequacy of the regimen must be evaluated through clinical, anthropometric tests and biochemical parameters. The use of somatostatin analogue is suggested, especially in the short term after bowel resection, for patients with high-output jejunostomy in whom fluid and electrolyte management is problematic. In SBS, who are candidates for enterohormonal therapy, Teduglutide is the first choice. Conclusion. There are recommendations on the comprehensive management of intestinal rehabilitation that are widely supported by this consensus and it is important to recognize therapeutic alternatives framed in the principle of good clinical practice.

Recomendaciones de expertos acerca del uso de teduglutide en pacientes pediátricos con sindrome de intestino corto / Expert recommendations on the use of teduglutide in pediatric patients with short bowel syndrome

Resumen El síndrome de intestino corto es una entidad de baja incidencia en los pacientes pediátricos, pero se asocia con elevadas tasas de morbimortalidad. El abordaje de estos pacientes por un equipo interdisciplinario de expertos enfocados en la rehabilitación intestinal mejora los resultados a corto y a largo plazo. Entre los recursos disponibles para el tratamiento se incluye el teduglutide, un análogo del péptido similar al glucagón tipo 2 (GLP-2) elaborado mediante técnicas recombinantes. Por medio de la aplicación del método Delphi, a partir de la evidencia disponible y de la experiencia de los autores, se proponen recomendaciones para el uso de teduglutide, dirigidas a los profesionales de la salud que tratan a los pacientes pediátricos con síndrome de intestino corto, así como a las autoridades sanitarias.

Abstract Short bowel syndrome is a low-incidence disorder among pediatric patients, but it is associated with high morbidity and mortality rates. Management of these patients by an interdisciplinary team of experts focused on intestinal rehabilitation improves short- and long-term outcomes. Available resources for treatment include teduglutide, a glucagon-like peptide type 2 (GLP-2) analog made by recombinant techniques. Considering the available evi dence and the authors' experience, Delphi-based recommendations for the use of teduglutide are suggested for healthcare professionals who treat pediatric patients with short bowel syndrome, as well as for health authorities.

[Expert recommendations on the use of teduglutide in pediatric patients with short bowel syndrome]. / Recomendaciones de expertos acerca del uso de teduglutide en pacientes pediátricos con síndrome de intestino corto.

Short bowel syndrome is a low-incidence disorder among pediatric patients, but it is associated with high morbidity and mortality rates. Management of these patients by an interdisciplinary team of experts focused on intestinal rehabilitation improves short- and long-term outcomes. Available resources for treatment include teduglutide, a glucagon-like peptide type 2 (GLP-2) analog made by recombinant techniques. Considering the available evidence and the authors' experience, Delphi-based recommendations for the use of teduglutide are suggested for healthcare professionals who treat pediatric patients with short bowel syndrome, as well as for health authorities.

El síndrome de intestino corto es una entidad de baja incidencia en los pacientes pediátricos, pero se asocia con elevadas tasas de morbimortalidad. El abordaje de estos pacientes por un equipo interdisciplinario de expertos enfocados en la rehabilitación intestinal mejora los resultados a corto y a largo plazo. Entre los recursos disponibles para el tratamiento se incluye el teduglutide, un análogo del péptido similar al glucagón tipo 2 (GLP-2) elaborado mediante técnicas recombinantes. Por medio de la aplicación del método Delphi, a partir de la evidencia disponible y de la experiencia de los autores, se proponen recomendaciones para el uso de teduglutide, dirigidas a los profesionales de la salud que tratan a los pacientes pediátricos con síndrome de intestino corto, así como a las autoridades sanitarias.

High Rate of Venous Thromboembolism in Severe Pediatric Intestinal Failure.

OBJECTIVE: To quantify the rate of venous thromboembolism (VTE) in patients with pediatric intestinal failure and identify associated risk factors. STUDY DESIGN: We performed a retrospective cohort study in pediatric patients (<21 years old) with severe pediatric intestinal failure (≥90 consecutive days of parenteral nutrition) secondary to short bowel syndrome who were treated from 2014 to 2021 at an interdisciplinary intestinal rehabilitation program. The primary outcome was the incidence of VTE. Multivariable regression was performed to identify independent clinical predictors of VTE. RESULTS: A total of 263 patients (59.7% male) met the criteria for inclusion. The cumulative incidence of VTE was 28.1%, with a rate of 0.32 VTEs per 1000 catheter-days. On univariate analysis, the number of catheter days, number of catheters, and history of central line-associated blood stream infection were associated with VTE. On multivariable logistic regression, a higher number of catheters was an independent risk factor for VTE (aOR, 1.17; 95% CI, 1.06-1.29). Additionally, earlier gestational age was a risk factor for VTE such that every week decrease in gestational age conferred a 9% increased risk of VTE (aOR, 1.09; 95% CI, 1.02-1.16). CONCLUSIONS: In this retrospective study, 28.1% of patients with severe pediatric intestinal failure developed VTE; the number of catheters and early gestational age were noted to be independent risk factors for VTE. This high incidence of VTE highlights the need to investigate VTE in pediatric intestinal failure prospectively, including the potential benefit of prophylactic anticoagulation.

Thyroid dysfunction in infants with severe intestinal insufficiency: a case series / Disfunção tireoidiana em lactentes com insuficiência intestinal grave: descrição de uma série de casos

Abstract Objective: The aim of this study was to describe the status of thyroid function in infants with severe intestinal dysfunction. Case description: A retrospective study was conducted in a tertiary neonatal intensive care center, including newborns and infants with severe intestinal dysfunction, hospitalized between 2015 and 2020. From the medical records, the following data were collected: gestational age, birth weight, underlying pathology that led to intestinal dysfunction, hospital stay, presence of thyroid dysfunction, age from the onset of thyroid dysfunction, initial and maximum dose of levothyroxine replacement, and levothyroxine administration route and outcome. Seven children (0.76% of 914 hospitalizations) developed severe intestinal insufficiency: vanishing gastroschisis (42.9%), Berdon syndrome (28.5%), apple peel (14.3%), and OIES syndrome (14.3%) - omphalocele, exstrophy of cloaca, imperforate anus, and spina bifida. The mean gestational age was 33.3±1.6 weeks, the mean birth weight was 2,113.9±370.9 g, the median hospitalization was 420 days, and mortality was 42.9%. Of these seven cases, four (57.1%) presented thyroid dysfunction, evaluated by blood hormone dosages and the dose of levothyroxine replacement ranged from 25 to 100 μg/day, administered by gastric or rectal route. Comments: This series of cases draws attention to thyroid dysfunction (hypothyroidism) in children with severe intestinal insufficiency receiving exclusive parenteral nutrition for a prolonged period, whose etiology is iodine deficiency, because, in Brazil, micronutrient solutions added to parenteral nutrition do not contain iodine.

RESUMO Objetivo: Descrever o status da função tireoidiana em lactentes com disfunção intestinal grave. Descrição do caso: Estudo retrospectivo, realizado em um centro de terapia intensiva neonatal de nível terciário, que incluiu recém-nascidos e lactentes com disfunção intestinal grave, internados entre 2015 e 2020. Dos prontuários foram obtidos: idade gestacional; peso de nascimento; patologia de base que levou à disfunção intestinal; tempo de internação; presença de disfunção tireoidiana; idade no início da disfunção tireoidiana; dose inicial e máxima da reposição de levotiroxina; via de administração da levotiroxina e desfecho. Sete crianças (0,76% do total de 914 internações) evoluíram com insuficiência intestinal grave: vanishing gastrosquise (42,9%), síndrome de Berdon (28,5), apple peel (14,3%) e síndrome onfalocele, ânus imperfurado, extrofia de cloaca e espinha bífida — OIES (14,3%). A média de idade gestacional foi de 33,3±1,6 semanas e de peso ao nascimento de 2113,9±370,9 gramas; a mediana de internação foi de 420 dias e a mortalidade foi de 42,9%. Desses sete casos, quatro (57,1%) apresentaram disfunção tireoidiana, avaliada por dosagens hormonais séricas, e a dose de reposição de levotiroxina variou de 25 a 100 mcg/dia, administrada por via gástrica ou retal. Comentários: Esta série de casos chama a atenção para a disfunção tireoidiana (hipotireoidismo) em crianças com insuficiência intestinal severa que receberam nutrição parenteral exclusiva por tempo prolongado, cuja etiologia é a deficiência de iodo, pois no Brasil as soluções de micronutrientes adicionadas à nutrição parenteral não contêm iodo.

Successful pregnancy in a patient with short bowel syndrome after surgical rehabilitation and sGLP-2 treatment: novel report on endogenous GLP-2 levels at delivery and during breastfeeding.

Pregnant patients with short bowel syndrome (SBS) and chronic intestinal failure (CIF) can successfully reach to term their pregnancies while on parenteral nutrition (PN) but with high rates of complications. The combination of rehabilitation surgery, combined with the use of novel treatment with enterohormones, especially semisynthetic glucagon-like peptide 2 (sGLP-2), has increased the chances to achieve intestinal sufficiency. Here, we report the case of a 33-year-old female with SBS/CIF (anatomy type 2), weaned off PN using sGLP-2 for 3.7 years, discontinued when she became pregnant. She was able to carry the pregnancy to term without any additional PN support. Considering that, we queried if the endogenous GLP-2 (eGLP-2) levels in this SBS patient, during the pregnancy and breastfeeding period, could be like those presented in healthy pregnant women and in non-pregnant SBS patients. Also, we inquired if there was any passage or increase in the plasmatic eGLP-2 from the fetus to the mother. Thus, we determined eGLP-2 levels in paired neonatal (cord blood) and maternal plasma samples from the SBS pregnant patient (n = 1), healthy pregnant women (controls, n = 2), and non-pregnant SBS patients (n = 12). The results indicated that the SBS pregnant patient showed higher eGLP-2 levels than non-SBS pregnant patients and healthy pregnant women along all the period studied. Furthermore, we found that the maternal sample had higher eGLP-2 levels than the neonatal sample, suggesting that fetal contribution to maternal eGLP2 levels would be minor. In conclusion, this study not only reports for the first time a case of a patient with SBS that was able to achieve intestinal adaptation after combining the use of autologous reconstructive surgery and sGLP-2, but also enlightens the possibility of carrying out an uneventful pregnancy and lactation without any nutritional support and remaining independent of sGLP-2.

Ethanol lock prophylaxis in long-stay central venous catheters in children with severe intestinal dysfunction: a report of six cases.

The aim of this study was to report on use of ethanol lock in long-term catheters in newborns with severe intestinal dysfunction, dependent on total and prolonged parenteral nutrition, in a Neonatal Intensive Care Center (tertiary level), between 2015 and 2020. Six infants (0.65%) out of the 914 admitted during the period met the inclusion criteria. The median age at catheter placement was 121.5 days. Two Powerpicc (PICC Power Sinergy™, São Paulo), one Groshong (Groshong™ Central Venous Catheter BD, São Paulo), and three silicone catheters were used, all tunneled, and the median dwell duration was 182.5 days. Four patients had at least one episode of infection related to the central venous catheter, and Gram-positive, Gram-negative, and fungal agents were isolated. The median length of hospital stay was 555 days and mortality was 33.3%. The ethanol lock did not cause any side effects and was relatively effective in preventing infections related to the central venous catheter.

Carrying the Burden: Informal Care Requirements by Caregivers of Children with Intestinal Failure Receiving Home Parenteral Nutrition.

OBJECTIVE: To measure the time that caregivers spend on tasks related to providing care to their child with intestinal failure receiving home parenteral nutrition (PN). STUDY DESIGN: We conducted an exploratory cross-sectional study of caregivers of children with intestinal failure receiving long-term PN followed by our intestinal rehabilitation program. Caregivers completed a daily diary of care-related tasks. Data were analyzed using descriptive statistics. Exploratory models were completed to evaluate factors that influenced the amount of time that caregivers spent providing care. SAS University Edition 2018 (SAS Institute, Cary, NC) was used for data analysis with a P value of less than .05 considered significant. RESULTS: Thirty-four caregivers of children with intestinal failure consented with response rates of 85%. The mean age of the primary caregiver was 37 ± 7.9 years of age with 97% being the child's mother. The median PN exposure was 1239 days (IQR, 432-3012). Caregivers reported a median of 29.2 hours per week (IQR, 20.8-45.7 hours per week) of direct medical care. The majority of time was spent on providing PN and care of the central venous catheter (6.1 hours; IQR, 5.2-8.8). CONCLUSIONS: Caregivers of children with intestinal failure receiving long-term PN provide a significant amount of care to ensure their child remains healthy at home. The most significant amounts of time were spent on the administration of the PN and care of the central venous catheter.

First registry of adult patients with chronic intestinal failure due to short bowel syndrome in Argentina: The RESTORE project.

BACKGROUND: Short bowel syndrome (SBS) is considered a low prevalence disease. In Argentina, no registries are available on chronic intestinal failure (CIF) and SBS. This project was designed as the first national registry to report adult patients with this disease. METHODS: A prospective multicenter observational registry was created including adult patients with CIF/SBS from approved centers. Demographics, clinical characteristics, nutrition assessment, home parenteral nutrition (HPN) management, surgeries performed, medical treatment, overall survival, and freedom from HPN survival were analyzed. RESULTS: Of the 61 enrolled patients, 56 with available follow-up data were analyzed. At enrollment, the mean intestinal length was 59.5 ± 47.3 cm; the anatomy was type 1 (n = 41), type 2 (n = 10), and type 3 (n = 5). At the end of the interim analysis, anatomy changed to type 1 in 31, type 2 in 17, and type 3 in 8 patients. The overall mean time on HPN before enrollment was 33.5 ± 56.2 months. Autologous gastrointestinal reconstruction surgery was performed before enrollment on 21 patients, and afterward on 11. Nine patients (16.1%) were weaned off HPN with standard medical nutrition treatment; 12 patients received enterohormones, and 2 of them suspended HPN; one patient was considered a transplant candidate. In 23.7 ± 14.5 months, 11 of 56 patients discontinued HPN; Kaplan-Meier freedom from HPN survival was 28.9%. The number of cases collected represented 19.6 new adult CIF/SBS patients per year. CONCLUSION: The RESTORE project allowed us to know the incidence, the current medical and surgical approach for this pathology, as well as its outcome and complications at dedicated centers.

Hospital-based delays to revascularization increase risk of postoperative mortality and short bowel syndrome in acute mesenteric ischemia.

OBJECTIVE: Acute mesenteric ischemia (AMI) is a surgical emergency for which delays in treatment have been closely associated with high morbidity and mortality. Although the duration of ischemia as a determinant of outcomes for AMI is well known, the objective of this study was to identify hospital-based determinants of delayed revascularization and their effects on postoperative morbidity and mortality in AMI. METHODS: All patients who underwent any surgery for AMI from a multi-center hospital system between 2010 and 2020 were divided into two groups based on timeliness of mesenteric revascularization after presentation. Early revascularization (ER) was defined as having both vascular consultation ≤12 hours of presentation and vascular surgery performed at the patient's initial operation. Delayed revascularization (DR) was defined as having either delays to vascular consultation or vascular surgery. A retrospective review of demographic and postoperative data was performed. The effect of DR on major postoperative outcomes, including 30-day and 2-year mortality, total length of bowel resection, and development of short bowel syndrome, were analyzed. Effects of delayed vascular consultation alone, delayed vascular surgery alone, no revascularization during admission, and admitting service on outcomes were also examined on subgroup analyses. RESULTS: A total of 212 patients were analyzed. Ninety-nine patients received ER, whereas the remaining 113 patients experienced a DR after hospital presentation. Among the DR group, 55 patients (25.9%) had delayed vascular consultation, whereas vascular surgery was deferred until after the initial operation in 37 patients (17.4%). Fifty-one patients (24.0%) were never revascularized during admission. DR was a significant predictor of 30-day (odds ratio [OR], 2.09; 95% confidence interval [CI], 1.4-4.9; P = .03) and 2-year mortality (hazard ratio, 1.55, 95% CI, 1.0-2.3; P = .04). DR was also independently associated with increased bowel resection length (OR, 7.47; P < .01) and postoperative short bowel syndrome (OR, 2.4; P = .03) on multivariate analyses. When examined separately on subgroup analysis, both delayed vascular consultation (OR, 3.38; P = .03) and vascular surgery (OR, 4.31; P < .01) independently increased risk of 30-day mortality. Hospital discharge after AMI without mesenteric revascularization was associated with increased risk of short bowel syndrome (OR, 2.94; P < .01) and late mortality (hazard ratio, 1.60; P = .04). CONCLUSIONS: Delayed vascular consultation and vascular surgery are both significant hospital-based determinants of postoperative mortality and short bowel syndrome in patients with AMI. Timing-based management protocols that emphasize routine evaluation by a vascular surgeon and early, definitive mesenteric revascularization should be established and widely adopted for all patients with clinically suspected AMI at presentation.