scholarly journals P431 Teduglutide use and nutritional outcomes in short bowel syndrome with intestinal failure: a real-world claims database analysis

2021 ◽  
Vol 15 (Supplement_1) ◽  
pp. S433-S434
Author(s):  
D Micic ◽  
J Jiang ◽  
L Chen ◽  
T Fan ◽  
F Mu ◽  
...  
Keyword(s):  
Sensitivity Analysis ◽  
Clinical Trials ◽  
Short Bowel Syndrome ◽  
Real World ◽  
Index Date ◽  
Intestinal Failure ◽  
Sensitivity Analyses ◽  
Future Research ◽  
Short Bowel

Abstract Background Teduglutide (TED) is a glucagon-like peptide 2 analogue approved for the treatment of patients with short bowel syndrome (SBS) requiring parenteral support (PS). SBS is a rare condition resulting from a reduced absorptive surface area of the small intestine, most commonly due to inflammatory bowel disease (IBD). Patients with SBS with intestinal failure (SBS-IF) remain dependent on PS to maintain adequate calorie, fluid, electrolyte and micronutrient stability. In phase 3 clinical trials, TED reduced PS requirements in patients with SBS-IF. This study aimed to assess PS use and discontinuation rates among patients with SBS on TED using real-world data. Methods This retrospective cohort study of adults with SBS-IF (≥18 years) with ≥1 TED pharmacy claim(s) used the US-based administrative healthcare claims IBM MarketScan database (2009–2019). The first TED claim was defined as the index date. Patients required ≥6 months of continuous enrolment prior to index date (baseline period) and no history of malignancy. Primary analysis was conducted during the follow-up period (index date to earliest of continuous enrolment end or 2 years post-index). A sensitivity analysis was also conducted among the cohort during the TED utilization period (index date to the earliest of continuous enrolment end or TED discontinuation). Patients required PS use during both baseline and follow-up/TED utilization periods (primary and sensitivity analyses). PS discontinuation was defined as a PS utilization gap of ≥30 days. A generalized estimating equation linear regression model evaluated if PS use (days/week) changed significantly from baseline to selected time points post-index. Results Of 110 identified patients with SBS-IF, mean age was 53.4 (SD 13.2) years and 77 (70%) were women. Included were 51 (46%) patients with Crohn’s disease and 20 (18%) with ulcerative colitis. The main comorbidities were renal disease (23%) and liver disease (15%). PS frequency was 4.6 (2.5), 3.3 (2.9), 2.9 (3.0) and 3.6 (3.0) days/week at baseline and months 6 (p<0.0001), 12 (p<0.0001), and 24 (p=0.0267), respectively. PS discontinuation increased over time to 34.4%, 46.7% and 65.2% at 3, 6, and 12 months, respectively. The sensitivity analysis demonstrated similar rates of PS use and discontinuation. Conclusion In this real-world study of adults with SBS-IF, including >50% with IBD, TED was associated with PS reductions comparable to those achieved in clinical trials and higher PS discontinuation rates even when using a conservative analysis approach. Future research will be required to determine individual predictive factors of PS discontinuation.

PTU-110 Long-Term Safety of Teduglutide Treatment for Patients with Intestinal Failure Associated with Short Bowel Syndrome: Pooled Data from 4 Clinical Trials

Gut ◽  
2016 ◽  
Vol 65 (Suppl 1) ◽  
pp. A109-A110
Author(s):  
SM Gabe ◽  
U-F Pape ◽  
E Delmaestro ◽  
B Li ◽  
NN Youssef ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Short Bowel Syndrome ◽  
Intestinal Failure ◽  
Short Bowel ◽  
Pooled Data

scholarly journals Teduglutide for the treatment of adults with intestinal failure associated with short bowel syndrome: pooled safety data from four clinical trials

2020 ◽  
Vol 13 ◽  
pp. 175628482090576 ◽  
Author(s):  
Ulrich-Frank Pape ◽  
Kishore R. Iyer ◽  
Palle B. Jeppesen ◽  
Marek Kunecki ◽  
Loris Pironi ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Short Bowel Syndrome ◽  
Safety Profile ◽  
Safety Data ◽  
Intestinal Failure ◽  
Abdominal Distension ◽  
Clinical Trial Registry ◽  
Data Set ◽  
Short Bowel ◽  
Patient Groups

Background: In multiple clinical studies, teduglutide reduced parenteral support (PS) with a consistent safety profile in adults with short bowel syndrome–associated intestinal failure (SBS–IF). The objective of this study was to assess adverse events (AEs) from a pooled data set. Methods: Safety data from four prospective clinical trials of teduglutide in patients with SBS–IF were assimilated. AEs were evaluated in patient groups based on treatment received in each study and in populations stratified to create distinct subgroups based on aetiology, bowel anatomy and baseline PS volume requirements. Results: Safety data are reported for up to 2.5 years, totalling 222 person-years exposure to teduglutide. In most patients, AEs were reported as mild or moderate in severity in all patient groups and occurred at comparable rates between patients who received teduglutide or placebo. Several common gastrointestinal AEs, including abdominal pain, nausea and abdominal distension, were reported more frequently earlier in the course of treatment, with their frequency declining over time. Fewer gastrointestinal AEs were reported in patients with vascular causes of SBS–IF and patients with most of their colon-in-continuity than in other patient subgroups. Across the patient stratification subgroups, the predominant treatment-emergent AEs for which patients receiving teduglutide had a significantly increased relative risk were abdominal distension and gastrointestinal stoma complication compared with patients receiving placebo. Conclusions: Teduglutide had a safety profile consistent with prior adult data and no new safety concerns were identified. The most frequently reported AEs were gastrointestinal in origin, consistent with the underlying disease condition and intestinotrophic actions of teduglutide. Clinical Trial Registry information: NCT00081458/EudraCT, 2004-000438-35; NCT00798967/EudraCT, 2008-006193-15; NCT00172185/EudraCT, 2004-000439-27; NCT00930644/EudraCT, 2009-011679-65

scholarly journals P462 Impact of teduglutide, a GLP-2 agonist, on inflammatory bowel disease-associated short bowel syndrome: a tertiary single-center study

2021 ◽  
Vol 15 (Supplement_1) ◽  
pp. S454-S455
Author(s):  
M de Seze ◽  
L Billiauws ◽  
J Bettolo ◽  
V Damas ◽  
C Hutinet ◽  
...  
Keyword(s):  
Short Bowel Syndrome ◽  
Controlled Trial ◽  
Intestinal Failure ◽  
Specific Effect ◽  
Positive Trend ◽  
Biologic Treatment ◽  
Short Bowel ◽  
Bowel Involvement ◽  
Active Disease

Abstract Background Teduglutide (TED), a GLP2 agonist, have been shown to exert anti-inflammatory effects on the intestinal mucosa in murine preclinical models and evaluated with a positive trend in a phase 2a randomized controlled trial in non-short bowel syndrome (SBS) Crohn’s disease (CD) patients. TED showed efficacy in reducing parenteral nutrition and or intravenous (PN/IV) dependence in SBS with intestinal failure (SBS-IF). However, limited data exists on its specific effect on the natural history of IBD in SBS patients treated with TED. Therefore, we aimed to assess the efficacy and safety of TED in patients with IBD-associated SBS as well as the effect of TED on the course of IBD. Methods We conducted an observational retrospective study. Adult patients SBS-IF due to IBD and who were treated with TED were enrolled. Response was defined as a decrease of at least 20% in total volume of PN. Active IBD was defined based on morphologic and histologic data (CT-scan, colonoscopy and/or enteroMRI). Patients were followed until last news or TED discontinuation. Results We enrolled 14 IBD patients, including 2 patients with ulcerative colitis, with a median follow-up of 4.2 ± 4 years. Of the 12 CD patients, 11 had ileocolonic involvement and 1 had only small bowel involvement. 11 patients had type-1 SBS and 3 had type-2 SBS. 7 patients were only receiving intravenous fluid and 7 were on PN/IV. Patients were in average receiving PN/IV for 9.1 years at TED initiation. Regarding IBD, 4 patients had active disease at inclusion, 3 with a structuring behavior and 1 with a fistulizing behavior. 5 patients were on biologic treatment (antiTNF or ustekinumab) at inclusion. At month 3 (M3), 9 of 14 patients were responders. 3 were weaned from any iv supplementation. 3 of 7 patients initially on TPN were weaned from caloric input, including one still receiving iv fluids. Caloric input was in average at 7330 kcal/week at baseline versus 4615 at M3. Mean total parenteral volume went from 11849 ml/week at M0 to 8223 at M3. 2 of 4 patients with active disease at baseline showed improvement in disease activity while on TED. The latter 2 patients were totally weaned off PN/IV.Six patients withdrew TED during follow-up. 2 patients stopped after a diagnosis of cancer (1 melanoma and 1 chronic myeloid leukemia). Survival without TED discontinuation was estimated at 85.7 % IC95(69.2–100) at 1 year, 78.6% IC95(59.8–100) at 2 years and at 64.3% IC95(43.5–95) at 5 years Conclusion TED appears effective and safe in IBD-associated SBS-IF patients. In specific cases, it may impact positively the inflammatory course of the disease. Further prospective studies are warranted to specifically evaluate TED in IBD-associated SBS, including patients with active IBD.

scholarly journals Real-World Analysis of the Clinical and Economic Burden of Later Line in Chronic Myeloid Leukemia Patients in Italy

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 1943-1943
Author(s):  
Massimo Breccia ◽  
Francesca Chiodi ◽  
Diletta Valsecchi ◽  
Valentina Perrone ◽  
Diego Sangiorgi ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Chronic Myeloid Leukemia ◽  
Drug Utilization ◽  
Real World ◽  
Economic Burden ◽  
Myeloid Leukemia ◽  
Blood Count ◽  
Index Date ◽  
The Impact

Abstract Background The use of real-world data in oncology is gaining increasing interest, as it can provide valuable insights into treatments and related outcomes in routine daily oncology practice, thus integrating the evidence coming from clinical trials. The study aims to describe the characteristics of patients (pts) with chronic myeloid leukemia (CML) in 2 nd or ≥3 rd tyrosine kinase inhibitors (TKI) lines of therapy, analyze their drug utilization and evaluate healthcare direct costs for the Italian National Health Service in order to estimate the impact of disease burden. Methods This retrospective observational analysis was based on administrative databases covering around 15.3 million subjects in Italy. All adult pts with prescription of the TKIs bosutinib (BOS), dasatinib (DAS), imatinib (IMA), nilotinib (NIL), ponatinib (PON) in 2 nd or ≥3 rd line of therapy during 01/2015-12/2018 were included. Specifically, those with IMA, DAS or PON prescription were enrolled if they also presented ≥1 CML hospitalization or previous prescription for NIL or BOS or ≥1 BCR-ABL1 RQ-PCR test without hospitalization for acute lymphoid leukemia. Pts enrolled in clinical trials were not captured. Index date was 1 st prescription date for a TKI in 2 nd or ≥3 rd line. Comorbidities were assessed by hospitalization diagnosis codes and/or presence of specific drugs as proxy of diagnosis. Mean annual healthcare resource costs were evaluated during follow-up (from index date to end of study) in terms of drugs other than TKI (excluded), visits, tests, hospitalizations (overall and comorbidity-related during treatment). Results Overall, 491 pts in 2 nd and 144 in ≥3 rd line were included. In each calendar year from 2015 to 2018, the incidence of pts who entered a 2 nd line changed from 22.6% to 28.7%, whereas the ≥3 rd line fluctuated from 37% to 46.7%. An increment of 97.6% was observed in the number of pts treated in ≥3 rd line from 2015 to 2018 (figure 1). As 2 nd line, 40.9% was represented by DAS, 28.9% NIL, 12.2% BOS, 10.2% PON and 7.8% IMA. Mean age ranged from 56.1 (PON) to 68.2 (BOS) years. At baseline, hypertension was the most reported comorbidity (from 64.1% for NIL to 91.7% for BOS), followed by metabolic (from 27.9% in DAS pts to 63.2% for IMA) and blood count (from 28.4% for DAS to 58.0% for PON) alterations. Among pts in ≥3 rd line, 26.3% received imatinib, 22.2% PON, 18.8% NIL, 16.7% BOS, 16% DAS. Mean age was 57 years for NIL, 64.8 PON and 69.5 BOS pts. Same trend of comorbidities was reported in the ≥3 rd line cohort, with hypertension detected in all pts starting BOS to a minimum of 65.2% pts in DAS group, followed by blood count alteration (34.8% for DAS to 59.4% for PON) and metabolic alterations (37.0% for NIL to 50.0% for BOS). Baseline cardiovascular comorbidities were present in 22.8% of pts starting a 2 nd line and in 35.4% of pts starting ≥3 rd line. After a median follow up of 3.0 and 2.6 years, 13% and 19% of pts died in 2 nd and ≥3 rd lines, respectively, around 40% in both lines discontinued their treatment. Median time to discontinuation was 5.5 (95%CI: 4.7-6.2) (2 nd line) and 4.3 (95%CI: 3.2-5.2) (≥3 rd line) years. Mean number of annual hospitalizations was 0.6 (from 0.4 for NIL to 1.1 for PON) in 2 nd line, and 0.5 (from 0.4 for IMA and NIL to 0.7 for PON) in ≥3 rd line. Total mean annual costs/pts (TKI excluded) were of €10,168 (PON), €6,106 (BOS), €5,086 (DAS), €4,779 (NIL) and €3,905 (IMA), with hospitalizations comorbidity-related accounting for €3,245 among PON, €2,820 BOS, €1,739 NIL, €1,573 DAS and €1,448 IMA pts. Mean annual costs per lines are shown in figure 2. Conclusions This real-world study provided a demographic and clinical profile of CML pts in 2 nd and ≥3 rd TKI lines and described drug utilization and resource consumption in the Italian clinical practice setting. An increase of pts treated with TKIs in ≥3 rd line was reported, reflecting the availability of multiple TKIs over the years; moreover, a highly comorbid population suggests an increasingly complex CML management. Our results underlined a heavy clinical and economic burden for pts in 2 nd or ≥3 rd lines, especially in terms of comorbidities, treatment discontinuation and hospitalizations suggesting the need of novel therapeutic options for management of later lines CML. Figure 1 Figure 1. Disclosures Breccia: Novartis: Consultancy; Pfizer: Consultancy; BMS: Consultancy; INCYTE: Consultancy; Abbvie: Consultancy. Chiodi: Novartis: Current Employment. Valsecchi: Novartis Farma SpA: Current Employment. Rendace: Novartis Farma S.p.A.: Current Employment. Coco: Novartis Farma S.p.A.: Current Employment. Premoli: Novartis Farma SpA: Current Employment.

scholarly journals Energy absorption as a measure of intestinal failure in the short bowel syndrome.

Gut ◽  
1989 ◽  
Vol 30 (2) ◽  
pp. 176-183 ◽  
Author(s):  
C A Rodrigues ◽  
J E Lennard-Jones ◽  
D G Thompson ◽  
M J Farthing
Keyword(s):  
Energy Absorption ◽  
Short Bowel Syndrome ◽  
Intestinal Failure ◽  
Short Bowel

scholarly journals Real-World Experience with Bezlotoxumab for Prevention of Recurrence of Clostridioides difficile Infection

2020 ◽  
Vol 10 (1) ◽  
pp. 2
Author(s):  
Rosa Escudero-Sánchez ◽  
María Ruíz-Ruizgómez ◽  
Jorge Fernández-Fradejas ◽  
Sergio García Fernández ◽  
María Olmedo Samperio ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Real World ◽  
Recurrence Rates ◽  
Factors Associated ◽  
Clostridioides Difficile ◽  
Multicentre Cohort Study ◽  
Clostridioides Difficile Infection ◽  
Prevention Of Recurrence ◽  
Multicentre Cohort

Bezlotoxumab is marketed for the prevention of recurrent Clostridioides difficile infection (rCDI). Its high cost could be determining its prescription to a different population than that represented in clinical trials. The objective of the study was to verify the effectiveness and safety of bezlotoxumab in preventing rCDI and to investigate factors related to bezlotoxumab failure in the real world. A retrospective, multicentre cohort study of patients treated with bezlotoxumab in Spain was conducted. We compared the characteristics of cohort patients with those of patients treated with bezlotoxumab in the pivotal MODIFY trials. We assessed recurrence rates 12 weeks after completion of treatment against C. difficile, and we analysed the factors associated with bezlotoxumab failure. Ninety-one patients were included in the study. The cohort presented with more risk factors for rCDI than the patients included in the MODIFY trials. Thirteen (14.2%) developed rCDI at 12 weeks of follow-up, and rCDI rates were numerically higher in patients with two or more previous episodes (25%) than in those who had fewer than two previous episodes of C. difficile infection (CDI) (10.4%); p = 0.09. There were no adverse effects attributable to bezlotoxumab. Despite being used in a more compromised population than that represented in clinical trials, we confirm the effectiveness of bezlotoxumab for the prevention of rCDI.

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