Clinical, Economic, and Humanistic Impact of Short-Bowel Syndrome/Chronic Intestinal Failure in Portugal (PARENTERAL Study)

Abstract

<b><i>Introduction:</i></b> This study aimed to assess the clinical, economic, and humanistic impact of short-bowel syndrome/chronic intestinal failure (SBS/CIF) in Portugal. <b><i>Methods:</i></b> This is a retrospective multicenter cohort chart review study, with a cross-sectional component for quality-of-life (QoL) evaluation. Inclusion criteria comprised patients with SBS/CIF, aged ≥1 year, with stable parenteral nutrition (PN). Data collection included patient chart review over a 12-month period and patient/caregiver self-report and SF-36/PedsQL™ questionnaires. Main endpoints comprised clinical and PN characterization, healthcare resource use (HRU), direct costs, and patient QoL. <b><i>Results:</i></b> Thirty-one patients were included (11 adults and 20 children). Patients’ mean age (standard deviation [SD]) was 57.9 (14.3) years in adults and 7.5 (5.0) years in children, with a mean time since diagnosis of 10.2 (5.9) and 6.6 (4.2) years, respectively. PN was administered for a mean of 5.2 and 6.6 days/week in adults and children, respectively; home PN occurred in 81.8% of adults and 90.0% of children for a mean of 9.6 and 10.8 months/year, respectively. The mean annual number of hospitalizations was 1.9 and 2.0 which lasted for a mean of 34.0 and 29.4 days in adults and children, respectively. Twenty-one and forty hospitalization episodes were reported in adults and children, respectively, of which 71.4% and 85.0% were due to catheter-related complications. Mean annual direct costs per patient amounted to 47,857.53 EUR in adults and 74,734.50 EUR in children, with PN and hospitalizations as the main cost-drivers. QoL assessment showed a clinically significant impaired physical component in adults and a notable deterioration in the school functioning domain in children. <b><i>Conclusion:</i></b> In Portugal, SBS/CIF patient management is characterized by a substantial therapeutic burden and HRU, translating into high direct costs and a substantial impairment of the adults’ physical function and children’s school functioning.

A Narrative Review of Patient-Reported Outcome Measures and Their Application in Recent Pediatric Surgical Research: Advancing Knowledge and Offering New Perspectives to the Field

“…Out of 345 articles identified by the database searches, 49 articles met the inclusion criteria. 28 29 30 31 32 33 34 35 36 37 38 39 40 41 42 43 44 45 46 47 48 49 50 51 52 53 54 55 56 57 58 59 60 61 62 63 64 65 66 67 68 69 70 71 72 73 74 75 76 Most concerned esophageal atresia ( n = 17) and Hirschsprung's disease ( n = 14). None of the articles focused on duodenal atresia or omphalocele.…”

A Systematic Review of Quality of Life in Patients with Short Bowel Syndrome and Their Caregivers

“…for SBS patients who were actively undergoing PS treatment during the research period. 9 , 10 , 13 , 21 , 25 , 26 , 28 , 30 Two studies addressed the utilization of Teduglutide (Ted). 24 , 25 One study explored the impact on caregiver QoL after children underwent autologous gastrointestinal reconstruction surgery (AGIR).…”

“… 10 , 13 , 24 , 25 , 29 , 30 Two studies compared the patients receiving Ted against those receiving placebo, 9 , 25 one study compared the patients with SBS with patients with dysmotility, both dysmotility and SBS are the underlying diseases that lead to intestinal failure (IF), 10 and three studies compared the patients with SBS with normal healthy population. 13 , 29 , 30 Two studies 21 , 26 compared the SBS population with two different populations such as one study involving patients with inflammatory bowel disease (IBD) and the normal healthy population. 26 Another one compared the patients with SBS with the normal healthy population and made subgroup comparisons.…”

The Anxiety Burden in Patients with Chronic Intestinal Failure on Long-Term Parenteral Nutrition and in Their Caregivers

“…A retrospective multicentre cohort study by Silva R et al [ 26 ] assessed the burden of CIF in Portuguese patients on parenteral nutrition (PN). Twenty of the thirty-one evaluated patients were paediatric.…”