Efficacy and safety of apraglutide in short bowel syndrome with intestinal failure and colon-in-continuity: A multicenter, open-label, metabolic balance study.

BACKGROUND: Apraglutide is a novel long-acting GLP-2 analog in development for short bowel syndrome with intestinal failure (SBS-IF). This multicenter, open-label, phase 2 study in SBS-IF and colon-in-continuity (CiC) investigates the safety and efficacy of apraglutide. METHODS: This was a 52-week phase 2 metabolic balance study (MBS) in 9 adult patients with SBS-IF-CiC receiving once-weekly subcutaneous apraglutide injections. Safety was the primary endpoint. Secondary endpoints included changes in absorption parameters (MBS at baseline, after 4 weeks with stable parenteral support (PS), and 48 weeks), PS needs (48-week PS adjustment period based on monthly 48-h fluid balances) and intestinal morphology and motility (static and cine MRI at baseline and 4, 24 and 48 weeks). RESULTS: PS volume decreased by -4702 mL/week (-52 %; p < 0.001) at week 52. Seven patients (78 %) achieved ≥1 day off PS at week 52. At 4 weeks, fecal output was reduced by 253 g/day (p = 0.013). At 48 weeks, increases in wet weight absorption by 316 g/day (p = 0.039), energy absorption by 1134 kJ/day (p = 0.041) and carbohydrate absorption by 56.1 g/day (p = 0.024) were observed. Moreover, small bowel length increased from 29.7 to 40.7 cm (p = 0.012), duodenal wall thickness increased by 0.8 mm (p = 0.02) and motility in the proximal colon was reduced (p = 0.031). A total of 127 adverse events was reported, which were mostly mild to moderate. CONCLUSION: Apraglutide had an acceptable safety profile and was associated with significant reductions in PS needs and days off PS, improvements in intestinal absorption, and structural and functional intestinal changes in patients with SBS-IF-CiC. CLINICALTRIALS: gov, Number NCT04964986.

Operational recommendations for the implementation of home parenteral nutrition program of care according to the guidelines of the European society for clinical nutrition and metabolism.

Home parenteral nutrition (HPN) is a complex therapy, which requires dedicated facilities and expertise. However, the management and provision of HPN differs significantly between countries and between HPN centers within countries. These differences lead to heterogeneity in the quality of care received by patients, with variable impact on the appropriateness, safety, and effectiveness of HPN, and resultant variability in the quality of life that a patient may expect. The European Society for Clinical Nutrition and Metabolism (ESPEN) have published guidelines on the appropriate and safe provision of HPN, with an associated practical version describing a short and precise way to implement the guidelines' recommendations in clinical practice. This educational paper suggests means of implementation of evidence supported HPN guidelines, using "operational recommendations" applitngto healthcare professionals, administrators and stakeholders, with the ultimate aim of enhancing equity of patient access to an appropriate and safe HPN program of care.

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.

An innovative educational program for adolescents on home parenteral nutrition for the "transition" to adulthood.

Facing with an increasing demand for transition to adult care management, our home parenteral nutrition (HPN) team designed an adolescent therapeutic educational program (ATEP) specifically intended for adolescents on long-term HPN. The aim of this study was to report on the first sessions of this program. Methods: The ATEP is designed in three sessions of five consecutive days, during school holidays over the year. It includes group sessions on catheter handling, disconnecting and connecting the PN and catheter dressing, dealing with unforeseen events (e.g., fever or catheter injury), but also sessions with psychologist, social worker, sports teacher, fashion specialist, meeting with adults who received HPN since childhood. Specific course for the accompanying parents were also provided. Six months after the last session, a 3-day trip to the attraction park "le Futuroscope," Poitiers, France, was organized without any parental presence. Results: After 3 ATEP courses, a total of 16 adolescents have been enrolled. They were aged between 13 and 17 years (median 14 IQR: 14-16.25). All were on long term HPN started during the neonatal period except for four who started PN at a median age of 10 years old (IQR: 1-10). At the time of the ATEP, their median PNDI was 105% (IQR: 95.5-120.8) while receiving a median of six infusions per week (IQR: 5-7). Thirteen received Taurolidine lock procedure. After the ATEP, 11 adolescents could be considered as fully autonomous, 4 as partially autonomous and one failed to gain any autonomy. Course evaluation by adolescents or parents was good to excellent. Conclusion: Through the holistic and multiprofessional approach of this training and the group cohesion, the adolescents were not only able to handle catheter care and PN connections but were able to understand and accept better their illness and project themselves into their own future.

Patients' and caregivers' perspective on challenges and outcomes with tube feeding: Analysis of home enteral nutrition survey data.

BACKGROUND: Given the growing use of home enteral nutrition (HEN), assessing the experience of consumers and caregivers is crucial to understanding the real-world subjective and objective challenges of administering HEN. METHODS: After obtaining institutional review board approval, a survey was distributed to HEN consumers and caregivers between January 16, 2020, and July 16, 2021. Data collected included information regarding demographics, primary diagnosis, tube and connectors, HEN regimen, and overall HEN experience. RESULTS: A total of 884 individuals responded to the survey: 673 (76.1%) responses by caregivers and 211 (23.9%) responses by patients. The study cohort included 566 (64%) children and 318 (36%) adults. The leading primary diagnosis of participants was developmental delay and motility disorder for children and adults, respectively. Low-profile gastric tubes were the most used (75.7% of children and 30.3% of adults). Notably, legacy connectors were utilized for more patients (46.7% children, 52.6% adults) compared to ISO-80369-3 connectors (38.9% children, 29.7% adults). HEN complications were prevalent, including enteral tube site infections and other tube-related complications, including clogging and kinking. CONCLUSION: This real-world data reveals that HEN complications remain prevalent. Additionally, despite introducing ISO-80369-3 connectors many years ago, most patients continue to use legacy tubes with a significant lack of knowledge about ISO-80369-3 connectors. The survey results guide HEN providers to focus on several areas to reduce complications.

Emerging drugs for the treatment of short bowel syndrome.

INTRODUCTION: SBS is a rare and disabling condition. The standard management is based on diet optimization with parenteral supplementation. In addition, glucagon-like peptide-2 (GLP-2)analogs, have shown promising results as disease-modifying therapies for SBS. AREAS COVERED: Short bowel syndrome (SBS) is defined as a reduction in functional intestinal length to less than 200 cm, leading to intestinal failure (IF) leading to malnutrition and parenteral support dependency. This review discusses the current management of SBS-CIFpatients, the place of GLP-2 analog treatment in terms of efficacy, safety and availability, and the new perspectives opened by the use of enterohormones. EXPERT OPINION: Clinical trials and real-world experience demonstrated that Teduglutide   reduces dependence on parenteral support and has a place in the management of patients with SBS-CIF.  The use of Teduglutide should be discussed in patients stabilized after resection and its introduction requires the advice of an expert center capable of assessing the benefit-risk ratio. The complex, individualized management of SBS-C IF requires theexpertise of a specialized IF center which a multidisciplinary approach. The arrival of new treatments will call for new therapeutic strategies, and the question of how to introduce and monitor them will represent a new therapeutic challenge.

[Prehabilitation in chronic inflammatory bowel disease: the Proactive care pathway]. / Préhabilitation dans les maladies inflammatoires chroniques de l'intestin : le parcours de soins Proactive.

Inflammatory Bowel Diseases (IBD) cause diarrhea and abdominal pain that impair quality of life. Digestive damage frequently leads to undernutrition and sarcopenia, which worsen the prognosis of the disease. This led to the development of PROACTIVE, a multimodal prehabilitation program designed to improve the functional capacities, nutritional status and quality of life of IBD patients. 19 patients have been included in our pilot program, with an initial personalized multimodal assessment, 10 group sessions with 4 patients, and a final multimodal assessment proposing personalized care for home. Initial data are positive, showing an improvement in patients' physical capacity and quality of life after 6 weeks.

Important issues in proposing autonomy training in home parenteral nutrition for short bowel syndrome patients: a qualitative insight from the patients' perspectives.

OBJECTIVES: The standard treatment for short bowel syndrome is home parenteral nutrition. Patients' strict adherence to protocols is essential to decrease the risk of complications such as infection or catheter thrombosis. Patient training can even result in complete autonomy in daily care. However, some patients cannot or do not want too much responsibility. However, doctors often encourage them to acquire these skills. Based on qualitative investigations with patients, we wanted to document issues of importance concerning perceptions of autonomy in daily care. METHODS: Semistructured interviews were conducted with 13 adult patients treated by home parenteral nutrition using a maximum variation sampling strategy. We proceeded to a thematic analysis following an inductive approach. RESULTS: After achieving clinical management of symptoms, a good quality of life is within the realm of possibility for short bowel syndrome patients with home parenteral nutrition. In this context, achieving autonomy in home parenteral nutrition could be a lever to sustain patients' quality of life by providing better life control. However, counterintuitively, not all patients aim at reducing constraints by reaching autonomy in home parenteral nutrition. First, they appreciate the social contact with the nurses, which is particularly true among patients who live alone. Second, they can feel safer with the nurse's visits. Regaining freedom was the main motivation for patients in the training program and the main benefit for those who were already autonomous. CONCLUSIONS: Medical teams should consider patients' health locus of control (internal or external) for disease management to support them concerning the choice of autonomy in daily care for parenteral nutrition.

Use of teduglutide in adults with short bowel syndrome-associated intestinal failure.

Short bowel syndrome (SBS) is a rare gastrointestinal disorder associated with intestinal failure (SBS-IF) and poor health-related outcomes. Patients with SBS-IF are unable to absorb sufficient nutrients or fluids to maintain significantly metabolic homeostasis via oral or enteral intake alone and require long-term intravenous supplementation (IVS), consisting of partial or total parenteral nutrition, fluids, electrolytes, or a combination of these. The goal of medical and surgical treatment for patients with SBS-IF is to maximize intestinal remnant absorptive capacity so that the need for IVS support may eventually be reduced or eliminated. Daily subcutaneous administration of the glucagon-like peptide 2 analog, teduglutide, has been shown to be clinically effective in reducing IVS dependence and potentially improving the health-related quality of life of patients with SBS-IF. The management of patients with SBS-IF is complex and requires close monitoring. This narrative review discusses the use of teduglutide for patients with SBS-IF in clinical practice. The screening of patient eligibility for teduglutide treatment, initiation, monitoring of efficacy and safety of treatment, adapting or weaning off IVS, and the healthcare setting needed for SBS-IF management are described, taking into consideration data from clinical trials, observational studies, and clinical experience.

The science of micronutrients in clinical practice - Report on the ESPEN symposium.

BACKGROUND & AIMS: The European Society for Clinical Nutrition and Metabolism published its first clinical guidelines for use of micronutrients (MNs) in 2022. A two-day web symposium was organized in November 2022 discussing how to apply the guidelines in clinical practice. The present paper reports the main findings of this symposium. METHODS: Current evidence was discussed, the first day being devoted to clarifying the biology underlying the guidelines, especially regarding the definition of deficiency, the impact of inflammation, and the roles in antioxidant defences and immunity. The second day focused on clinical situations with high prevalence of MN depletion and deficiency. RESULTS: The importance of the determination of MN status in patients at risk and diagnosis of deficiencies is still insufficiently perceived, considering the essential role of MNs in immune and antioxidant defences. Epidemiological data show that deficiencies of several MNs (iron, iodine, vitamin D) are a global problem that affects human health and well-being including immune responses such as to vaccination. Clinical conditions frequently associated with MN deficiencies were discussed including cancer, obesity with impact of bariatric surgery, diseases of the gastrointestinal tract, critical illness, and aging. In all these conditions, MN deficiency is associated with worsening of outcomes. The recurrent problem of shortage of MN products, but also lack of individual MN-products is a worldwide problem. CONCLUSION: Despite important progress in epidemiology and clinical nutrition, numerous gaps in practice persist. MN depletion and deficiency are frequently insufficiently searched for in clinical conditions, leading to inadequate treatment. The symposium concluded that more research and continued education are required to improve patient outcome.

Pancytopenia related to acquired sea-blue histiocytosis during chronic parenteral nutrition.

Bone marrow smear showing histiocytes (black arrow) containing sea blue granules stained with May-Grünwald Giemsa.

Central venous catheter (CVC) salvage in case of central line-associated bloodstream infection (CLABSI): A monocentric prospective study in patients on long-term home parenteral nutrition (HPN).

BACKGROUND & AIMS: CLABSI is a major complication in HPN and frequently leads to central venous catheter (CVC) removal. We developed a salvaging attitude in long term HPN patients due to the necessity of venous preservation. The main objective of this study is to determine the prognosis of CLABSI. METHODS: We followed-up for three months, in an approved HPN centre, a cohort of 250 adult patients receiving HPN with CLABSI from 2018 to 2020. CLABSI was defined by a blood culture growth differential [peripheral blood] - [CVC blood] ≥ 2h. A therapeutic approach to conserve CVC was established according to the department's protocol. The primary endpoint was conservation of CVC with negative CVC and peripheral blood cultures at 3 months without complications. RESULTS: Data from 30 CLABSIs were collected for 22 HPN patients. The incidence rate of CLABSIs was 0.28 infections/1000 catheter days. Sixteen CVCs were removed immediately, with causes due to the type of germ (staphylococcus aureus: n = 6, candida parapsilosis: n = 4, klebsiella: n = 2), chronic colonization (n = 4) or initial complications (n = 4). Among the 14 non-removed CVC, 11 were maintained at 3 months with blood cultures on CVC and peripheral negative for 9 (80%) of them. 3 CVC were removed during the 3 months follow-up (non-CVC-related sepsis n = 2, and resistant pseudomonas aeruginosa n = 1). CONCLUSION: The incidence rate of CLABSIs in an expert HPN centre remains low. In case of CLABSIs, according to specific protocol, approximately 50% of CVC were removed immediately (essentially due to bacteriological characteristics). In case of CLABSIs and without initial complication, 80% of CVCs can be maintained at 3 months. These results justify a conservative attitude according to standardized protocol.

Hyperphagia is prominent in adult patients with short bowel syndrome: A role for the colon?

RATIONALE: Short Bowel Syndrome (SBS) is the major cause of chronic intestinal failure (IF) and requires parenteral nutrition (PN). After bowel resection, some patients develop spontaneous intestinal adaptations and hyperphagia. Since promoting oral energy intake contributes to PN weaning, this study aims to characterize hyperphagia in patients with SBS and identify its determinants. METHODS: This observational retrospective study included adult patients with SBS who were followed at an expert PN center between 2006 and 2019, with at least 2 separate nutritional assessments. Exclusion criteria were: active neoplasia, alternative treatment for IF or appetite-affecting medication. Resting energy expenditure (REE) was calculated for each patient using the Harris-Benedict equation. Food Intake Ratio (FIR) was calculated by dividing the highest caloric oral intake by REE and hyperphagia was defined as FIR >1.5. RESULTS: Among the 59 patients with SBS included in this study, 82.6% had a FIR >1.5, including 15.5% with a FIR >3. Protein supplied approximately 16% of total energy intake while fat and carbohydrates provided 36% and 48%, respectively. The FIR was independent of gender and whether patients received oral nutrition alone (n = 28) or combined with PN (n = 31). The FIR was also not associated with residual small bowel length, nor the proportion of preserved colon. However, it was negatively correlated with the body mass index (BMI) of these patients (r = -0.533, p < 0.001), whether they had PN support or not. Patients with either a jejuno-colonic (n = 31) or a jejuno-ileal anastomosis (n = 9), had a significantly higher FIR compared to those with an end-jejunostomy (n = 18) (p < 0.05). However, no difference was found in the proportion of calories provided by protein, fat and carbohydrate between the 3 patients groups divided according to the SBS anatomical type. CONCLUSION: A large majority of patients with SBS exhibited a hyperphagia regardless of PN dependence or bowel length, which was inversely correlated with BMI. The presence of the colon in continuity, thus in contact with the nutritional flow, seems to favor a higher oral intake which is beneficial for the nutritional autonomy of patients. This raises the question of a role of colonic microbiota and hormones in this behavior. Finally, this study also revealed an unexpected discrepancy between recommended energy intakes from protein, fat and carbohydrate and the actual intake of patients with SBS.

A multi-national survey of experience and attitudes towards managing catheter related blood stream infections for home parenteral nutrition.

BACKGROUND AND AIMS: Catheter-related bloodstream infection (CRBSI) is the most common complication of home parenteral nutrition (HPN) in patients with chronic intestinal failure (CIF). The aim of this study was to assess the broad range of practices of international multi-disciplinary teams involved in the care of this complication occurring in CIF patients. DESIGN: An online questionnaire was designed and distributed to members of the European Society for Clinical Nutrition and Metabolism (ESPEN) and distributed to colleagues involved in managing patients with CIF. RESULTS: A total of 47 responses were included from centers across 21 countries. The centers had been delivering HPN for a median 21 years (IQR 11-35) and were actively following a median 58 patients (27-120) per center for benign CIF in 80% of cases (67-95). Tunneled catheters were the most common type of central venous catheters (CVC), representing 70% (47-86) of all CVC in use. For the management of CRBSI, written procedures were provided in 87% of centers. First measures included simultaneous central and peripheral blood cultures (90%), stopping HPN infusion (74%), and administrating an antibiotic lock and systemic antibiotics (44%). Immediate removal of the CVC was more likely in case of fungal infection (78%), Staphylococcus aureus (53%), or in case of PICC catheter (52%) (all p < 0.01). After the first CRBSI, 80% of centers used preventive CVC locks (taurolidine in 84% of cases, p < 0.001). We observed a large heterogeneity in practices regarding preparation, duration, reaspiration, and volume of CVC locks, and monitoring of CRBSI (timing of blood cultures, radiological work-up). CONCLUSION: In this international survey of HPN expert centers, we observed a significant consensus regarding the initial management of CRBSI and the use of secondary preventive CVC locks, while areas of variation exist. Management of CRBSI may be improved with clearer recommendations based on the micro-organism and the type of CVC, including PICC lines which are increasingly used yet insufficiently studied in HPN patients.

Circulating Apolipoprotein B-48 as a Biomarker of Parenteral Nutrition Dependence in Adult Patients with Short Bowel Syndrome.

Short bowel syndrome (SBS) is a rare but serious condition that may lead to chronic intestinal failure. Citrulline concentrations are currently used to reflect the residual intestinal mass in patients with SBS, although this method has several limitations. In a cohort of patients with SBS, we quantified apolipoprotein B-48 (ApoB-48), which is exclusively synthesized by enterocytes and secreted associated with dietary lipids and investigated the relationship between ApoB-48 and clinical and biological data as well as PN dependence. A total of 51 adult patients were included, 36 of whom were PN-dependent. We found a robust positive correlation between circulating ApoB-48 and residual small bowel length, which was also found in the subgroup of patients with jejunocolic anastomosis. Fasting ApoB-48 levels were significantly lower in PN-dependent patients than in PN-weaned patients and negatively correlated with parenteral nutrition dependence. Our results suggest that ApoB-48 could be proposed as a marker of intestinal absorptive function and could be an interesting follow-up marker in patients with SBS.

Experience and opinions relating to pregnancy in patients with chronic intestinal failure: an international survey.

Introduction: Pregnancy in patients with chronic intestinal failure (CIF) is a relatively rare occurrence but is an important contemporary topic given both the increasing use of home parenteral nutrition (HPN) and the demographics of patients with CIF. Method: An opinion-based survey was produced in a multidisciplinary manner, which was then distributed internationally, via the European Society for Clinical Nutrition and Metabolism network, using a web-based survey tool for healthcare professionals with a specialist interest in the management of CIF. Results: Seventy specialists from 11 countries completed the survey. Fifty-four per cent of the respondents reported some experience of managing pregnancy in patients with CIF. However, 60% stated that they did not feel that it was their role to discuss the topic of pregnancy with their patients, with fewer than 10% stating that they routinely did so. Respondents felt that an individualised approach was required when considering alterations to parenteral support prior to conception, during pregnancy and in the postnatal period. Most respondents also felt there was no increased risk of catheter-related blood stream infections, while catheter-related thrombosis was deemed to be the most significant HPN-related complication for pregnant women. Conclusion: This study reports a variable experience, knowledge and confidence of healthcare professionals when considering pregnancy in patients with CIF. The risk of HPN-related complication was felt to be greater during pregnancy, with an individualised approach being the preferred route for most aspects of care. The findings support the need for an international registry and subsequent consensus guidelines for the management of pregnancy in CIF.

ESPEN guideline on chronic intestinal failure in adults - Update 2023.

BACKGROUND & AIMS: In 2016, ESPEN published the guideline for Chronic Intestinal Failure (CIF) in adults. An updated version of ESPEN guidelines on CIF due to benign disease in adults was devised in order to incorporate new evidence since the publication of the previous ESPEN guidelines. METHODS: The grading system of the Scottish Intercollegiate Guidelines Network (SIGN) was used to grade the literature. Recommendations were graded according to the levels of evidence available as A (strong), B (conditional), 0 (weak) and Good practice points (GPP). The recommendations of the 2016 guideline (graded using the GRADE system) which were still valid, because no studies supporting an update were retrieved, were reworded and re-graded accordingly. RESULTS: The recommendations of the 2016 guideline were reviewed, particularly focusing on definitions, and new chapters were included to devise recommendations on IF centers, chronic enterocutaneous fistulas, costs of IF, caring for CIF patients during pregnancy, transition of patients from pediatric to adult centers. The new guideline consist of 149 recommendations and 16 statements which were voted for consensus by ESPEN members, online in July 2022 and at conference during the annual Congress in September 2022. The Grade of recommendation is GPP for 96 (64.4%) of the recommendations, 0 for 29 (19.5%), B for 19 (12.7%), and A for only five (3.4%). The grade of consensus is "strong consensus" for 148 (99.3%) and "consensus" for one (0.7%) recommendation. The grade of consensus for the statements is "strong consensus" for 14 (87.5%) and "consensus" for two (12.5%). CONCLUSIONS: It is confirmed that CIF management requires complex technologies, multidisciplinary and multiprofessional activity, and expertise to care for the underlying gastrointestinal disease and to provide HPN support. Most of the recommendations were graded as GPP, but almost all received a strong consensus.

The indications and results of the use of teduglutide in patients with short bowel.

Short bowel syndrome (SBS) is a rare condition defined as a reduced residual functional small intestinal length to less than 200 cm often resulting from extensive intestinal resection, and can lead to chronic intestinal failure (CIF). Patients with SBS-CIF are unable to absorb sufficient nutrients or fluids to maintain metabolic homeostasis through oral or enteral intake and require long-term parenteral nutrition and/or fluids and electrolytes. However, complications may arise from both SBS-IF and life-sustaining intravenous support, such as intestinal failure-associated liver disease (IFALD), chronic renal failure, metabolic bone disease and catheter-related complications. An interdisciplinary approach is required to optimize intestinal adaptation and decrease complications. In the last two decades, glucagon-like peptide 2 (GLP-2) analogs have sparked pharmacological interest as a potential disease-modifying therapy for SBS-IF. Teduglutide (TED) is the first developed and marketed GLP-2 analog for SBS-IF. It is approved in the United States, Europe, and Japan for use in adults and children with SBS-IF who are intravenous supplementation dependent. This article discusses the indications, candidacy criteria and results of the use of TED in patients with SBS.

Post-Marketing Use of Teduglutide in a Large Cohort of Adults with Short Bowel Syndrome-Associated Chronic Intestinal Failure: Evolution and Outcomes.

Teduglutide, a GLP-2 analogue, has been available in France since 2015 to treat short-bowel-syndrome (SBS)-associated chronic intestinal failure (CIF) but it remains very expensive. No real-life data on the number of potential candidates are available. The aim of this real-life study was to assess teduglutide initiation and outcomes in SBS-CIF patients. All SBS-CIF patients cared for in an expert home parenteral support (PS) center between 2015 and 2020 were retrospectively included. Patients were divided into two subpopulations: prevalent patients, already cared for in the center before 2015, and incident patients, whose follow-up started between 2015 and 2020. A total of 331 SBS-CIF patients were included in the study (156 prevalent and 175 incident patients). Teduglutide was initiated in 56 patients (16.9% of the cohort); in 27.9% of prevalent patients and in 8.0% of incident patients, with a mean annual rate of 4.3% and 2.5%, respectively. Teduglutide allowed a reduction in the PS volume by 60% (IQR: 40-100), with a significantly higher reduction in incident versus prevalent patients (p = 0.02). The two- and five-year treatment retention rates were 82% and 64%. Among untreated patients, 50 (18.2%) were considered ineligible for teduglutide for non-medical reasons. More than 25% of prevalent SBS patients were treated with teduglutide compared to 8% of incident patients. The treatment retention rate was >80% at 2 years, which could be explained by a careful selection of patients. Furthermore, this real-life study confirmed the long-term efficacy of teduglutide and showed a better response to teduglutide in incident patients, suggesting a benefit in early treatment.