Cost-effectiveness of teduglutide in adult patients with short bowel syndrome - a European socioeconomic perspective.

BACKGROUND: Short bowel syndrome with intestinal failure (SBS-IF) is a rare but devastating medical condition. An absolute loss of bowel length forces the patients into parenteral support dependency and a variety of medical sequelae, resulting in increased morbidity and mortality. Interdisciplinary treatment may include therapy with the effective but expensive intestinotrophic peptide teduglutide. OBJECTIVES: A time-discrete Markov model was developed to simulate the treatment effect [lifetime costs, quality-adjusted life years (QALYs), and life years (LYs)] of teduglutide plus best supportive care compared with best supportive care alone in patients with SBS-IF. METHODS: The health status of the model was structured around the number of days on PS. Clinical data from 3 data sets were used: 1) an Austrian observational study (base case), 2) pooled observational cohort studies, and 3) a prospective study of teduglutide effectiveness in parenteral nutrition-dependent short bowel syndrome subjects. Direct and indirect costs were derived from published sources. QALYs, LYs, and costs were discounted (3% per annum). RESULTS: Under the base case assumption, teduglutide is associated with costs of 2,296,311 € per patient and 10.78 QALYs (13.74 LYs) over a lifetime horizon. No teduglutide is associated with 1,236,816 € and 2.24 QALYs (8.57 LYs). The incremental cost-utility ratio (ICUR) amounts to 123,945 €. In case of the pooled clinical data set, the ICUR increases to 184,961 €. If clinical data based on the study of teduglutide effectiveness in parenteral nutrition-dependent short bowel syndrome subjects were used, the ICUR increased to 235,612 €. CONCLUSIONS: Teduglutide in treating patients with SBS-IF meets the traditional cost-effectiveness criteria from a European societal perspective. Nevertheless, the varying concentrations of teduglutide efficacy leave a degree of uncertainty in the calculations.

Teduglutide in amyloidosis-associated intestinal failure.

Amyloidosis is a heterogeneous disease characterized by tissue deposition of abnormally folded fibrillary proteins that can manifest itself by a wide variety of symptoms depending on the affected organs. GI involvement among amyloidosis patients is common. Its clinical manifestation often presents with nonspecific symptoms such as weight loss, diarrhea, and malabsorption. With no specific treatment existing for GI amyloidosis, therapy focuses on impeding amyloid deposition and managing the patients' symptoms with supportive measures. Here, we present an AL-amyloidosis patient with GI involvement and intestinal failure (IF) who was successfully treated with the glucagon-like peptide-2 (GLP-2) analogue teduglutide. Over the course of treatment with teduglutide, the patient was able to achieve independence from parenteral nutrition and experienced a significant improvement in quality of life (QoL) as stool frequency and consistency improved, urinary output was stabilized and body weight as well as body composition improved over the course of teduglutide therapy. With no longer being exposed to the burden and associated risks of parenteral nutrition, we were able to reduce the potential morbidity and mortality rate as well as to improve the patient's overall QoL. Intestinal tissue biopsy workup revealed a histopathological correlate for the clinical response; Congo-Red-positive intestinal depositions almost completely disappeared within 6 months of teduglutide therapy. Implementing intestinotrophic GLP-2 analogue teduglutide may enrich the spectrum of treatment options for amyloidosis patients with IF who are dependent on parenteral support.

Teduglutide in short bowel syndrome patients: A way back to normal life?

BACKGROUND: The glucagon-like peptide 2 analogue teduglutide is an effective drug for the treatment of short bowel syndrome patients with intestinal failure (SBS-IF). This intestinotrophic peptide improves intestinal capacity for fluid and nutrient absorption through induction of mucosal growth and reduction of gastrointestinal motility. Clinical trials demonstrated the efficacy of teduglutide in reducing the need for parenteral support (PS). This study describes an SBS-IF patient population receiving teduglutide therapy in a specialized medical care setting. METHOD: A retrospective analysis was performed using data of patients experiencing nonmalignant SBS-IF. They were treated with teduglutide in a multidisciplinary SBS-IF program at a single university medical center between June 2016 and June 2020. RESULTS: Thirteen patients under teduglutide treatment were included in the final analysis. Mean small bowel length was 82 ± 31 cm, with 77% of patients having their colon in continuity. Over a median follow-up of 107 weeks, all patients (13 of 13, 100%) responded to the therapy with a clinically significant reduction of PS volume. Mean PS reduction increased with therapy duration and ranged from -82.5% at week 24 (n = 13) to -100% in patients (n = 5) who were treated for 144 weeks. Enteral autonomy was achieved in 12 of 13 (92%) patients. Teduglutide therapy improved stool frequency and consistency, changed dietary habits, and reduced disease-associated sleep disruptions. CONCLUSION: Integrating SBS-IF patients treated with teduglutide in a proactive and tight-meshed patient care program significantly improves the clinical outcome, leading to an increased proportion of patients reaching enteral autonomy.

Bridging intestinal failure with Teduglutide - A case report.

INTRODUCTION AND IMPORTANCE: Intestinal failure (IF) describes the state of a person's gastrointestinal absorption capabilities becoming unable to absorb fluids and nutrients needed to sustain normal physiology, leading to severe comorbidities and if left untreated, to death. IF is most commonly seen as a result of short bowel syndrome (SBS). Teduglutide is a glucagon-like peptide 2 (GLP-2) analogue used in the treatment of patients with SBS and intestinal failure (IF) as a way to reduce the need for parenteral support. Teduglutide leads to the growth of intestinal mucosa by stimulating intestinal crypt cell growth and inhibiting enterocyte apoptosis. It is usually prescribed as a final treatment step after the diagnosis of SBS-IF is made. CASE PRESENTATION: In this case report we present a novel strategy for using teduglutide as a bridging therapy to intestinal reconstruction. The patient achieved enteral autonomy preoperatively, underwent surgery, and remained in enteral autonomy after intestinal reconstruction. CLINICAL DISCUSSION: Teduglutide has been previously exclusively used as continuous therapy in SBS-IF, this is the first reported case of using teduglutide as bridging to intestinal reconstruction. The hypothesis of this approach was to achieve an adequate nutritional status for reconstruction without the disadvantages of parenteral support. CONCLUSION: The controlled application of teduglutide can provide the benefits of preoperative nutritional optimization without the disadvantages of parenteral support and at the same time facilitate an earlier and easier intestinal reconstruction.

ESPEN practical guideline: Clinical Nutrition in cancer.

BACKGROUND: This practical guideline is based on the current scientific ESPEN guidelines on nutrition in cancer patients. METHODS: ESPEN guidelines have been shortened and transformed into flow charts for easier use in clinical practice. The practical guideline is dedicated to all professionals including physicians, dieticians, nutritionists and nurses working with patients with cancer. RESULTS: A total of 43 recommendations are presented with short commentaries for the nutritional and metabolic management of patients with neoplastic diseases. The disease-related recommendations are preceded by general recommendations on the diagnostics of nutritional status in cancer patients. CONCLUSION: This practical guideline gives guidance to health care providers involved in the management of cancer patients to offer optimal nutritional care.

Assessing the feasibility of a mobile health-supported clinical decision support system for nutritional triage in oncology outpatients using Arden Syntax.

BACKGROUND: Nutritional screening procedures followed by regular nutrition monitoring for oncological outpatients are no standard practice in many European hospital wards and outpatient settings. As a result, early signs of malnutrition are missed and nutritional treatment is initiated when patients have already experienced severe weight loss. OBJECTIVE: We report on a novel clinical decision support system (CDSS) for the global assessment and nutritional triage of the nutritional condition of oncology outpatients. The system combines clinical and laboratory data collected in the clinical setting with patient-generated data from a smartphone application for monitoring the patients' nutritional status. Our objective is to assess the feasibility of a CDSS that combines the aforementioned data sources and describe its integration into a hospital information system. Furthermore, we collected patients' opinions on the value of the system, and whether they would regard the system as a useful aid in coping with their condition. MATERIALS AND METHODS: The system implements the Patient-Generated Subjective Global Assessment (PG-SGA) to monitor nutritional status in the outpatient setting. A smartphone application is used to collect patient-generated data by performing weekly mini-surveys on patients concerning their eating habits, weight, and overall well-being. Data are uploaded on completion of each mini-survey and stored on a secure server at the Medical University of Vienna (MUV). The data are then combined with relevant clinical information from the Vienna General Hospital (VGH) information system. The knowledge base for the CDSS is implemented in medical logic modules (MLMs) using Arden Syntax. A three-month pilot clinical trial was performed to test the feasibility of the system. Qualitative questionnaires were used to obtain the patients' opinions on the usability and personal value of the system during the four-week test period. RESULTS: We used the existing separation between the scientific and clinical data domains in the secured network environment (SNE) at the MUV and VGH to our advantage by importing, storing, and processing both patient-generated and routine data in the scientific data domain. To limit exposure to the SNE, patient-generated data stored outside the SNE were imported to the scientific domain once a day. The CDSS created for nutritional assessment and triage comprised ten MLMs, each including either a sub-assessment or the final results of the PG-SGA. Finally, an interface created for the hospital information system showed the results directly in clinical routine. In all 22 patients completed the clinical study. The results of the questionnaires showed that 91% of the patients were generally happy with the usability of the system, 91% believed that the application was of additional value in detecting cancer-related malnutrition, and 82% found it helpful as a long-term monitoring tool. DISCUSSION AND CONCLUSION: Despite strict protection of the clinical data domain, a CDSS employing patient-generated data can be integrated into clinical routine. The CDSS discussed in this report combined the information entered into a smartphone application with clinical data in order to inform the physician of a patient's nutritional status and thus permit suitable and timely intervention. The initial results show that the smartphone application was well accepted by patients, who considered it useful, but not many oncological outpatients were willing to participate in the clinical study because they did not possess an Android phone or lacked smartphone expertise. Furthermore, the results indicate that patient-generated data could be employed to augment clinical data and calculate metrics such as the PG-SGA without excessive effort by using a secure intermediate location as the locus of data storage and processing.

ESPEN guidelines on nutrition in cancer patients.

Cancers are among the leading causes of morbidity and mortality worldwide, and the number of new cases is expected to rise significantly over the next decades. At the same time, all types of cancer treatment, such as surgery, radiation therapy, and pharmacological therapies are improving in sophistication, precision and in the power to target specific characteristics of individual cancers. Thus, while many cancers may still not be cured they may be converted to chronic diseases. All of these treatments, however, are impeded or precluded by the frequent development of malnutrition and metabolic derangements in cancer patients, induced by the tumor or by its treatment. These evidence-based guidelines were developed to translate current best evidence and expert opinion into recommendations for multi-disciplinary teams responsible for identification, prevention, and treatment of reversible elements of malnutrition in adult cancer patients. The guidelines were commissioned and financially supported by ESPEN and by the European Partnership for Action Against Cancer (EPAAC), an EU level initiative. Members of the guideline group were selected by ESPEN to include a range of professions and fields of expertise. We searched for meta-analyses, systematic reviews and comparative studies based on clinical questions according to the PICO format. The evidence was evaluated and merged to develop clinical recommendations using the GRADE method. Due to the deficits in the available evidence, relevant still open questions were listed and should be addressed by future studies. Malnutrition and a loss of muscle mass are frequent in cancer patients and have a negative effect on clinical outcome. They may be driven by inadequate food intake, decreased physical activity and catabolic metabolic derangements. To screen for, prevent, assess in detail, monitor and treat malnutrition standard operating procedures, responsibilities and a quality control process should be established at each institution involved in treating cancer patients. All cancer patients should be screened regularly for the risk or the presence of malnutrition. In all patients - with the exception of end of life care - energy and substrate requirements should be met by offering in a step-wise manner nutritional interventions from counseling to parenteral nutrition. However, benefits and risks of nutritional interventions have to be balanced with special consideration in patients with advanced disease. Nutritional care should always be accompanied by exercise training. To counter malnutrition in patients with advanced cancer there are few pharmacological agents and pharmaconutrients with only limited effects. Cancer survivors should engage in regular physical activity and adopt a prudent diet.