Outcomes from a 12-Week, Open-Label, Multicenter Clinical Trial of Teduglutide in Pediatric Short Bowel Syndrome.

OBJECTIVE: To determine safety and pharmacodynamics/efficacy of teduglutide in children with intestinal failure associated with short bowel syndrome (SBS-IF). STUDY DESIGN: This 12-week, open-label study enrolled patients aged 1-17 years with SBS-IF who required parenteral nutrition (PN) and showed minimal or no advance in enteral nutrition (EN) feeds. Patients enrolled sequentially into 3 teduglutide cohorts (0.0125 mg/kg/d [n = 8], 0.025 mg/kg/d [n = 14], 0.05 mg/kg/d [n = 15]) or received standard of care (SOC, n = 5). Descriptive summary statistics were used. RESULTS: All patients experienced ≥1 treatment-emergent adverse event; most were mild or moderate. No serious teduglutide-related treatment-emergent adverse events occurred. Between baseline and week 12, prescribed PN volume and calories (kcal/kg/d) changed by a median of -41% and -45%, respectively, with 0.025 mg/kg/d teduglutide and by -25% and -52% with 0.05 mg/kg/d teduglutide. In contrast, PN volume and calories changed by 0% and -6%, respectively, with 0.0125 mg/kg/d teduglutide and by 0% and -1% with SOC. Per patient diary data, EN volume increased by a median of 22%, 32%, and 40% in the 0.0125, 0.025, and 0.05 mg/kg/d cohorts, respectively, and by 11% with SOC. Four patients achieved independence from PN, 3 in the 0.05 mg/kg/d cohort and 1 in the 0.025 mg/kg/d cohort. Study limitations included its short-term, open-label design, and small sample size. CONCLUSIONS: Teduglutide was well tolerated in pediatric patients with SBS-IF. Teduglutide 0.025 or 0.05 mg/kg/d was associated with trends toward reductions in PN requirements and advancements in EN feeding in children with SBS-IF. TRIAL REGISTRATION: ClinicalTrials.gov:NCT01952080; EudraCT: 2013-004588-30.

A integralidade do SUS e a Judicialização por transplantes intestinais e multiviscerais / The integrality in the SUS and the judicialization by intestinal and multiple visceral transplants / La integridad del SUS y la legalización por los trasplantes intestinales y multiviscerales

A discussão sobre a judicialização da saúde no Brasil tem como tema central a questão do Sistema Único de Saúde - SUS não disponibilizar insumos e serviços de forma integral à população. Há demandas para transplantes de intestino e múltiplos órgãos, que em virtude do seu custo elevado, são combatidos pelos órgãos gestores do SUS, pelo custo elevado do procedimento. Tratou-se de um estudo de 4 casos ocorrido em 2014 sobre o tema para pacientes portadores de Síndrome de Berdon, Síndrome do Intestino Curto e a Doença de Inclusão Microvilositária. Concluiu-se pela pertinência da judicialização em vista da falta de recursos para os pacientes.

The discussion about the Health Judicialization has a main topic that is the public system does not offer integrally services and inputs to the population. There are judicial demands for intestine and multiple organs transplants, but they are very expensive. There was a case study about 4 cases in Ministry of Health with intestine and multiple visceral transplant in patients who carry Berdon Syndrome, Short-Gut Syndrome and the Microvillous Inclusion Disease. It concluded for the relevancy of the judicialization for these poor patients.

El debate sobre la judicialización de la salud en Brasil se centró en la cuestión de que el Sistema Único de Salud - SUS no está disponible en su totalidad al público. Hace demandas dirigidas a los trasplantes de intestino y de múltiples órganos, que, en virtud de su alto costo, son rechazadas por los órganos de gestión del SUS. Hizo un estudio de 4 casos teniendo como demandada el Ministerio de la Salud, sobre trasplantes de intestino y trasplantes multiviscerales para pacientes con Síndrome de Berdon, el Síndrome de intestino corto y la llamada Enfermedad de Inclusión Microvellosa. En este trabajo se analiza la cuestión de la integridad del Sistema Único de Salud, tratando de resolver el asunto que implica la prestación de cualquier tratamiento necesario a los pacientes del SUS.