Revisión sobre la experiencia en vida real de teduglutida / Review of real-life teduglutide experience

Introducción: la teduglutida es un agonista del péptido relacionado con glucagón (aGLP2) eficaz como tratamiento de pacientes con síndrome de intestino corto (SIC) una entidad que afecta a la calidad de vida, suele precisar de nutrición parenteral domiciliaria (NPD) y genera importantes costes sanitarios. El objetivo de la presente revisión narrativa fue evaluar la experiencia en vida real reportada con teduglutida. Métodos y resultados: en vida real un metaanálisis y estudios publicados con 440 pacientes, indican que teduglutida es efectivo pasado el periodo de adaptación intestinal posterior a la cirugía, reduciendo las necesidades de NPD y en algunos casos permite incluso suspenderla. La respuesta es heterogénea, aumenta progresivamente hasta 2 años después del inicio del tratamiento y alcanza el 82 % en algunas series. La presencia de colon en continuidad es factor predictivo negativo de respuesta precoz, pero un factor predictivo positivo para la retirada de NPD. Los efectos adversos más frecuentes son de origen gastrointestinal en las primeras etapas del tratamiento. Hay complicaciones tardías relacionadas con el estoma o con la aparición de pólipos de colon, aunque la frecuencia de estas últimas es muy baja. En adultos son escasos los datos en mejoría de calidad de vida y en coste eficacia. Conclusiones: teduglutida es efectivo y seguro confirmándose en vida real los datos de los ensayos pivotales para tratamiento de pacientes con SIC y permite reducir o incluso suspender en algunos casos la NPD. Aunque parece coste efectivo son necesarios más estudios para identificar aquellos pacientes con mayor beneficio.(AU)

Background: teduglutide is an agonist of glucagon-related peptide (aGLP2) effective as a treatment for patients with short bowel syndrome (SBS), an entity that affects quality of life, usually requires home parenteral nutrition (HPN) and generates significant health costs. The objective of the present narrative review was to assess the real-life experience reported with teduglutide.Methods and results: in real life, one meta-analysis and studies published with 440 patients indicate that Teduglutide is effective after the period of intestinal adaptation after surgery, reducing the need for HPN and in some cases even allowing it to be suspended. The response is heterogeneous, increasing progressively up to 2 years after the start of treatment and reaching 82 % in some series. The presence of colon in continuity is a negative predictor of early response, but a positive predictive factor for the withdrawal of HPN. The most common side effects are gastrointestinal in the early stages of treatment. There are late complications related to the stoma or the occurrence of colon polyps, although the frequency of the latter is very low. In adults, data on improved quality of life and cost-effectiveness are scarce. Conclusions: teduglutide is effective and safe and data from pivotal trials for the treatment of patients with SBS are confirmed in real life and can reduce or even stop HPN in some cases. Although it seems cost-effective, more studies are needed to identify those patients with the greatest benefit.(AU)

Rehabilitación del intestino utilizando un análogo semisintético del péptido 2 similar al glucagón. Primera experiencia con teduglutide en Uruguay / Intestinal rehabilitation using semisynthetic glucagon-like peptide-2 analogue. First experience with teduglutide in Uruguay / Reabilitação do intestino usando um análogo semi-sintético do peptídeo 2 semelhante ao glucagon. Primeira experiência com teduglutide no Uruguai

Resumen: La falla intestinal crónica (FIC) o tipo III es una condición invalidante, y la nutrición parenteral crónica (NPC) domiciliaria es el tratamiento que permite a estos pacientes mantenerse con vida. Sin embargo, solamente uno de cada tres países latinoamericanos cuentan con ese recurso, y sus complicaciones no son infrecuentes. Estas complicaciones son las principales indicaciones para trasplante intestinal, un procedimiento que en la mayoría de los países de ingresos medios no se ha desarrollado y no ha presentado los resultados esperados. En los últimos años, la rehabilitación intestinal a nivel mundial ha mejorado sustancialmente con el uso de análogos semisintéticos del péptido 2 similares al glucagón, existiendo cada vez mayor evidencia que demuestra la posibilidad de rehabilitación intestinal e independencia de la NPC con este fármaco, incluso en pacientes con anatomía desfavorable. Estos resultados han permitido mejorar la supervivencia y la calidad de vida de pacientes con FIC y, en muchas ocasiones, prescindir del trasplante. El paciente del caso que presentamos es el primero en recibir esta terapéutica en nuestro país. En este artículo analizamos la respuesta precoz favorable al tratamiento y sus perspectivas a futuro.

Abstract: Long-term home parenteral nutrition (HPN) is a life-saving treatment for patients with chronic intestinal failure, an invalidating condition. However, only 1 out of 3 countries can rely on this treatment and complications associated to chronic parenteral nutrition are rather frequent. The latter constitute the main indication for intestinal transplantion, a procedure that in most middle-income countries has not yet developed and has not shown the expected outcome. In recent years, intestinal rehabilitation has significantly improved at the global level with the use of GLP2, based on the growing evidence that proves the possibility of intestinal rehabilitation and independence from parenteral nutrition with Teduglutide, even in the case of patients with unfavorable anatomy. These results have caused a positive impact on survival and the quality of life of patients with chronic renal failure, and they can often abstain from transplant. The patient of the case study is the first one who received this therapy in our country and this article analyses his favorable early response to treatment and future perspectives.

Resumo: A insuficiência intestinal crônica (CIF) ou tipo III é uma condição incapacitante e a nutrição parenteral crônica (NPC) domiciliar é o tratamento que permite a sobrevida desses pacientes. No entanto, apenas 1 em cada 3 países latino-americanos dispõe desse recurso e as complicações da NPC não são raras. Essas complicações são as principais indicações para o transplante intestinal, procedimento que na maioria dos países de renda média não foi desenvolvido ou não apresentou os resultados esperados. Nos últimos anos, a reabilitação intestinal em todo o mundo tem melhorado substancialmente com o uso de sGLP2, com um número cada vez maior de evidências que mostram a possibilidade de reabilitação intestinal e independência da NPC, mesmo em pacientes com anatomia desfavorável. Esses resultados têm possibilitado prolongar a sobrevida e melhorar a qualidade de vida dos pacientes com CIF e, em muitos casos, dispensar o transplante. O paciente do caso que apresentamos é o primeiro a receber essa terapia em nosso país. Neste artigo, analisamos a resposta favorável ao tratamento precoce e suas perspectivas futuras.

Inquérito Brasileiro Sobre Terapia de Nutrição Domiciliar: panorama atual / Brazilian Survey on Home Nutrition Therapy: current overview / Encuesta brasileña sobre terapia nutricional domiciliaria: panorama actual

Objetivo: descrever como a terapêutica nutricional domiciliar é realizada no Programa Melhor em casa do Ministério da Saúde e na Saúde suplementar. Método: Estudo transversal, com dados secundários, onde foram selecionados os perfis de profissionais atuantes em atenção domiciliaria no Brasil. A coleta de dados ocorreu de março a junho de 2018, depois de submetido e aprovado pelo Comitê de Ética e Pesquisa. Resultados: Dos 289 brasileiros, 74% eram profissionais atuantes na Assistência domiciliaria. O tipo de Terapia Nutricional realizada foi 67% enteral exclusiva seguida de 33% terapia mista. A prescrição realizada de dieta exclusiva artesanal foi de 9% e de dieta enteral mista 55%. A maioria recebe dieta por gravidade intermitente, seguida de administração em "bolus", gravitacional contínua e controle do gotejamento através da bomba de infusão. Conclusão: Os achados nos dão uma visão panorâmica da terapia nutricional enteral domiciliar no Brasil. A aumento da terapia nutricional domiciliaria se faz necessária especialmente pelo aumento da população idosa e consequentemente de maior presença das doenças crônicas que podem levar a incapacidade, dependência, maior tempo de hospitalização e custos para o sistema de saúde. É fundamental a presença da equipe interdisciplinar, de boas práticas e do acompanhamento das famílias nos domicílios.

Objective: To learn how home nutritional therapy is carried out in the Ministry of Health's Better Home Program and in Supplementary Health. Method: Cross-sectional study, with secondary data, in which the profiles of professionals working in home care in Brazil were selected. Data collection took place from March to June 2018, after being submitted and approved by the Ethics and Research Committee. Results: Of the 289 Brazilians, 74% were professionals working in home care. The type of nutritional therapy performed was 67% exclusive enteral followed by 33% mixed therapy. The prescription of an exclusive handmade diet was 9% and a mixed enteral diet 55%. Most receive intermittent gravity diet, followed by bolus administration, continuous gravitational and drip control through the infusion pump. Conclusion: The findings give us a panoramic view of home enteral nutritional therapy in Brazil. The increase in home nutritional therapy is necessary especially because of the increase in the elderly population and, consequently, the greater presence of chronic diseases that can lead to disability, dependence, longer hospitalization and costs for the health system. The presence of an interdisciplinary team, good practices and monitoring of families at home is essential.

Objetivo: Conocer cómo se lleva a cabo la terapia nutricional domiciliaria en el Programa Mejor Hogar del Ministerio de Salud y en Salud Complementaria. Método: Estudio transversal, con datos secundarios, en el que se seleccionaron los perfiles de los profesionales que trabajan en la atención domiciliaria en Brasil. La recolección de datos tuvo lugar de marzo a junio de 2018, luego de ser presentados y aprobados por el Comité de Ética e Investigación. Resultados: De los 289 brasileños, el 74% eran profesionales que trabajaban en la atención domiciliaria. El tipo de terapia nutricional realizada fue 67% enteral exclusiva seguida de 33% terapia mixta. La prescripción de una dieta exclusiva artesanal fue del 9% y una dieta enteral mixta del 55%. La mayoría recibe una dieta de gravedad intermitente, seguida de administración de bolo, control gravitacional continuo y de goteo a través de la bomba de infusión. Conclusión: Los hallazgos nos brindan una visión panorámica de la terapia nutricional enteral domiciliaria en Brasil. El aumento de la terapia nutricional domiciliaria es necesario sobre todo por el aumento de la población anciana y, en consecuencia, la mayor presencia de enfermedades crónicas que pueden derivar en discapacidad, dependencia, mayor internación y costos para el sistema de salud. La presencia de un equipo interdisciplinario, buenas prácticas y seguimiento de las familias en el hogar es fundamental.

Small intestinal bacterial overgrowth in children with intestinal failure on home parenteral nutrition.

BACKGROUND AND AIM: Children with intestinal failure (IF) have abnormal intestinal anatomy, secretion, or motility, which impairs homeostatic mechanisms and can lead to small intestinal bacterial overgrowth (SIBO). We sought to describe clinical features at the time of clinically suspected SIBO by experienced clinicians in children with IF on home parenteral nutrition (PN), review specific challenges of diagnostic testing in this population, and describe potential new diagnostic surrogate markers. METHODS: A descriptive single-center retrospective chart review was performed during all episodes of clinically suspected SIBO over 33 months. Information was recorded on clinical symptoms, and diagnostic tests performed. RESULTS: Of all patients on home PN, 71% (12/17) had at least one episode of clinically suspected SIBO (mean 1 episode/year, range 1-7); 50% of patients had short bowel syndrome (SBS), and 50% had non-SBS IF. The average reported symptoms per episode were 1.9 (range 1-5). Children with SBS reported fewer symptoms per episode (1.5) than children with non-SBS IF (2.3). Diarrhea was the most commonly reported symptom, particularly in children with SBS. CONCLUSIONS: Children with IF on home PN are a high-risk group for SIBO. Clinical features of SIBO vary depending on the cause of IF and may mimic symptoms of the underlying condition. Diagnostic tests have innate challenges in this group, and a strong index of clinical suspicion is paramount. Further research is recommended into potential new surrogate markers (urinary metabolite screen, gastric aspirate) for this diagnostically challenging population.

Training of children's and adolescents' family members in home parenteral nutrition care / Capacitação dos familiares de crianças e adolescentes para os cuidados com nutrição parenteral domiciliar

ABSTRACT Objective: To report the experience of the training in home parenteral nutrition (PN) directed to family members of children and adolescents participating in a multidisciplinary intestinal rehabilitation program of a tertiary public hospital. Methods: Cross-sectional descriptive study with family caregivers of patients from the Intestinal Rehabilitation Program of Hospital de Clínicas de Porto Alegre, RS, Brazil, from July/2014 to January/2017. Inclusion criteria: family members of children aged 30 days to 17 years and estimated PN use ≥8 weeks; and family members motivated to care for the child. The training covered: hand washing and disinfection; infusion pump handling; and central venous catheter (CVC) and PN care. Outcomes assessed: catheter-related bloodstream infection (CRBSI) rate, accidental CVC exit, end of PN infusion with more than 60minutes of delay or advance compared to the time predicted, mechanical obstruction, bleeding in the CVC insertion site, and death. Results: Twenty-seven family members of 17 children were trained. Their median age was 28 (18-60) years, and 63% were mothers. The mean CRBSI rate was 1.7/1,000 days of CVC use, and 29.4% of patients had at least one episode of accidental CVC exit. There were no complications related to PN infusion, bleeding, or death. Conclusions: The training of family caregivers allowed the safe implementation of home PN, with the active participation of families, making the procedure feasible in the public health system in Brazil.

RESUMO Objetivo: Apresentar a experiência da capacitação de familiares de crianças e adolescentes participantes de um programa multiprofissional de reabilitação intestinal de um hospital público terciário para uso de nutrição parenteral (NP) no domicílio. Métodos: Estudo descritivo transversal com familiares cuidadores de pacientes do Programa de Reabilitação Intestinal do Hospital de Clínicas de Porto Alegre, RS, Brasil, entre julho/2014 e janeiro/2017. Critérios de inclusão: familiares das crianças com idades entre 30dias e 17 anos e previsão de uso de NP≥8 semanas; e familiares que demonstrassem motivação para os cuidados da criança. Acapacitação contemplou: lavagem e higienização das mãos; manuseio da bomba de infusão; cuidados com cateter venoso central (CVC) e com solução de NP. Desfechos avaliados foram: taxa de infecção de corrente sanguínea relacionada ao CVC (ICSRC), saída acidental do CVC, término da infusão da NP com atraso ou adiantamento maior que 60minutos em comparação ao previsto, obstrução mecânica, sangramento do sítio de inserção do CVC e óbito. Resultados: Foram capacitados 27 familiares de 17 crianças, com mediana de idade de 28 (18-60) anos, dos quais 63% eram mães. A taxa média de ICSRC observada foi 1,7/1.000 dias de uso de CVC, e a saída acidental do CVC ocorreu em 29,4% dos pacientes. Não foram observadas complicações referentes à infusão da NP, a sangramento ou a óbito. Conclusões: A capacitação de familiares cuidadores possibilitou a execução de maneira segura da NP no domicílio com participação ativa das famílias, tornando o procedimento viável no sistema público de saúde no Brasil.

Wide variation in organisation and clinical practice of paediatric intestinal failure teams: an international survey.

BACKGROUND & AIMS: We aimed to assess the current organisation and clinical practice of teams treating children with intestinal failure (IF) across Europe and compare the results with the current guideline. METHODS: A two-part online survey was sent to all the major European specialist IF services. The first part concerned general information about the team and patients monitored. The second part concerned important care topics such as vascular access and monitoring of complications. No patient identifiers were collected. RESULTS: Seventy-three respondents completed the first part, representing 61 teams in 20 countries. The median number of children on parenteral nutrition (PN) at home per team was 15 (range 1-125). Teams consisted of the following members: paediatric gastroenterologist (present in 100% of the teams), dietitian (95%), specialist nurse (92%), paediatric surgeon (89%), pharmacist (82%), psychologist (66%), social worker (62%), speech therapist (48%), physiotherapist (38%), general paediatrician (33%). The second part was completed by 67/73 respondents (59/61 teams). Vascular access care was comparable with the guideline. Somatostatin analogues were prescribed by 14% of the IF teams and probiotics by 44% of the teams. Prophylactic anticoagulation was used by 46% of the teams. In 81% of the teams a multicomponent lipid emulsion containing fish oil was routinely used. Bone densitometry was regularly performed in 75% of teams, but never performed in 19%. CONCLUSIONS: In conclusion, there is a wide diversity of composition of IF teams and their number of patients treated. Overall, there is good compliance to the current guideline. Clinical practice that varied most was the standard use of medication such as probiotics and somatostatin analogues, and standard monitoring of long-term complications. Experience regarding specific treatment options should be shared. Moreover, international agreement on standards of care with focus on implementation of the guideline is needed to optimise care and improve outcomes of children with IF.

Sixteen years′ home parenteral nutrition for a patient with total small bowel resection / 中国普通外科杂志

ObjectiveTo study the metabolic changes and complications of a patient on long term home parenteral nutrition (HPN). MethodsClinical data were summarized on a female patient with total small bowel plus right colon resection receiving home parenteral nutrition support for 16 years. ResultsThe patient became pregnant on the 5th year of HPN, and gave birth to a healthy baby. Her liver function remains almost normal during the 16 years. Each central vein catheter averagely lasted for 240 days, with the longest one more than 26 months. Catheter related sepsis rate was very low. Several complications occurred during the 16 years, including anaemia, zinc deficiency and increased serum iron level. Lipid peroxidation increased significantly. ConclusionsLong-term HPN support patient could suffer many metabolic abnormalities complications. Catheter related sepsis and occlusion should be prevented. The HPN formula should be adjusted according to the patient′s condition.