Quality of Life in Patients With Short Bowel Syndrome Treated Without and With Teduglutide - a Prospective Nested Matched Pair Analysis

• Conditions: Intestinal Failure; Short Bowel Syndrome
• Interventions: Drug: Teduglutide

• Registration Number: NCT04733066
• Lead Sponsor: Charite University, Berlin, Germany

Brief Summary

The aim of this prospective longitudinal study is to compare the quality of life of short bowel patients prior to and on teduglutide treatment with a non-treated patient group in a matched-pair design.

Detailed Description

Short bowel syndrome (SBS) is a malabsorptive disorder mostly caused by surgical interventions, which may result in chronic intestinal failure (cIF). The incidence is progressively increasing due to more aggressive surgical and medical approaches. Parenteral nutrition is still the mainstay of treatment for patients with irreversible cIF. New hormonal therapies hold promise by romoting mucosal growth and intestinal absorption and thereby leading to a consequent reduction in parenteral support and symptoms related to large stomal or faecal losses. Outside the typical primary endpoints of cost data, quality of life (QoL) is an important and yet underappreciated consideration of the effectiveness of targeted therapies. In the clinical STEPS trial, Jeppesen et al. have previously shown a significant improvement of the SBS-QoL total score after 24 week treatment with teduglutide longitudinally but could not identify a statistical significance to the placebo group. Accordingly, Chen and colleagues carried out a post hoc analysis of the afore mentioned clinical trial data based on 86 patients and could confirm the results auf Jeppesen et al. Taken together, these clinical trial data did not show a direct benefit of teduglutide treatment on QoL in these patients, while real life data on QoL changes are not available yet. During the release of teduglutide in Germany, the PNLiver trial (DRKS00010993) recruited chronic intestinal failure patients with parenteral nutrition from 2014 till 2019 to evaluate the capability of non-invasive liver function tests in cIF patients in a cross-sectional (n=90) and longitudinal study (n=20. All participants underwent study visits including clinical examination, dynamic liver function assessment, comprehensive blood tests, nutritional status assessment and quality of life assessment (Short Form 36 \[SF-36\] and SBS-QoL). Therefore, we have a comprehensive data set of a large monocentric cIF cohort prior to teduglutide treatment. Consequently, our unique QoL data from the PNLiver trail enables a followup assessment for an observation period above 24 weeks of patients who were in the meantime exposed to teduglutide in a real life setting. Nevertheless, QoL also tends to improve with longer duration on HPN. Therefore, we are aiming to extend the results with a pairwise matched control group from non teduglutide-treated PNLiver trail patients and collect follow-up data from this group accordingly.

Study Timeline

• Study First Submitted: 2020-08-29
• Study Start: 2020-09-01
• Status Verified: 2021-01
• Study First Submitted QC: 2021-01-26
• Last Update Submitted: 2021-01-26
• Study First Posted: 2021-02-01
• Last Update Posted: 2021-02-01
• Primary Completion: 2021-09
• Study Completion: 2022-09

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

• SBS-associated adult chronic IF with parenteral nutritional support
• previous inclusion in the PNLiver trial with existing SF-36 and SBSQoL data
• signed informed consent

Exclusion Criteria

• teduglutide stop before study initiation
• refusal or withdrawal of consent

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Teduglutide-treated Group	Teduglutide	1. Quality of life assessment (SBS-QoL, SF- 36) 2. Nutrition status assessment (BIA, BMI) 3. Assessment of functional absorptive small bowel length (citrulline) 4. Clinical data (nutritional program, stool characteristics)

Primary Outcome Measures

Name	Time	Method
changes in QoL data of patients without teduglutide therapy	through study completion, an average of 1 year	SBS-QoL, SF-36
changes in QoL data of teduglutide treated patients compared to changes in pairwise matched controls	through study completion, an average of 1 year	SBS-QoL, SF-36
changes in QoL data during teduglutide therapy	through study completion, an average of 1 year	SBS-QoL, SF-36

Secondary Outcome Measures

Name	Time	Method
Body cell mass in kg	through study completion, an average of 1 year	measured by bioelectrical impedance analysis
stool characteristics by bristol stool scale	through study completion, an average of 1 year	seven types of stool from 1 (hard lumps) till 7 (watery)
BMI in kg/m^2	through study completion, an average of 1 year	weight and height will be combined to report BMI in kg/m\^2
parenteral support volume	through study completion, an average of 1 year
parenteral support calories	through study completion, an average of 1 year
citrulline levels	through study completion, an average of 1 year
parenteral support frequency	through study completion, an average of 1 year

Trial Locations

Locations (1)

Charite University, Berlin, Germany; 🇩🇪 Berlin, Germany