Real-World Experience

GATTEX Real-World Data

A Single, Retrospective Analysis at Mount Sinai Medical Center

The first real-world study for GATTEX was a single, retrospective analysis at Mount Sinai Medical Center that included 18 adult patients with SBS who were dependent on PS and who were treated with GATTEX between 2009 and 20151

The goal of this analysis was to better characterize patients who achieved reductions in daily PS volume, reductions in the number of days on PS, and enteral autonomy.1

Enrolled in study1

Continued from STEPS-2 with GATTEX (0.05 mg/kg/day) n=5

Initiated GATTEX following FDA approval (0.05 mg/kg/day) n=13

Methods1

  • Patients were only considered for GATTEX after:
    • Management of diet, antidiarrheals, and antisecretory agents was optimized and stabilized
    • Attempts were made to achieve enteral autonomy without GATTEX
  • Patients expressed desire and motivation for further PS weaning
  • During the weaning process, patients were monitored weekly (weight, urine output, and lab values)
  • PS volume was reduced in 10% to 30% increments based on urine output and lab values
    • Once PS requirement was reduced to 2 days weekly, the next reduction was discontinuation of PS

Baseline demographics1

  • Patients were between the ages of 20 and 81 years (median: 47)—11 were female and 7 were male
  • Underlying etiologies of SBS were surgical resections due to Crohn’s disease (n=7), mesenteric ischemia (n=7), volvulus (n=2), recurrent bowel obstructions (n=1), radiation enteritis (n=1)
  • 3 patients had a stoma
  • Small bowel length ranged from 6 cm to 180 cm (median: 55 cm)
  • Patients were on PS for a median of 36 months (range: 4 to 96 months) before initiating GATTEX
  • Baseline PS volume ranged from 2.7 to 30 L/week

GATTEX Long-term Clinical Outcomes for Adults1

%

(16/18)

Achieved any reduction
in PS volume1 The median weekly PS volume reduction of all 18 patients was 5.4 L/week (range, 0-16.8 L/week).

%

(11/18)

Achieved enteral autonomy1 The median time to enteral autonomy from baseline was 10 months (range, 3-36 months).

Of the 11 patients who achieved enteral autonomy from PS1:

10

patients had presence of colon

2

patients required resumption of PS during periods of intercurrent illness

3

patients required supplemental tube feedings

3

patients participated in STEPS and STEPS-2—of those, 1 patient achieved complete enteral autonomy from PS during STEPS-2

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One patient who fully weaned off PS 3 months after starting GATTEX resumed PS 5 months later during several infectious episodes (at the time of publication of the study results in a peer-reviewed journal, the patient was dependent on PS 2 days/week).

Colonoscopy results1

Follow-up colonoscopy results 1-2 years after starting GATTEX were available for 12 of the 15 patients who had some or all their colons present. One colonoscopy revealed cecal polyps, which were removed and consistent with tubular adenomas. Eight of the 12 available colonoscopies were normal.* Five years after initiating GATTEX, 1 patient had a second colonoscopy that was positive for cecal polyps (tubular adenomas), which were removed. His prior colonoscopy at 1 year after GATTEX initiation was normal.

* In the remaining 3 patients, 1 showed nonspecific enteritis, 1 showed mild Crohn’s ileitis, and 1 showed dilated bowel with nonspecific colitis.1

Safety profile

Adverse events (AEs)1

Common AEs included stoma enlargement (n=3, 100% of patients with stomas), abdominal pain (n=6, 33%), nausea (n=4, 22%), abdominal pain and flatulence (n=2, 11%), and injection-site tenderness (n=1, 5%). One patient developed gastrointestinal AEs requiring suspension of GATTEX for 2 days until symptoms resolved. In another patient, GATTEX was temporarily discontinued for 1 month during evaluation for lower-extremity swelling, which was determined to be unrelated to GATTEX. No patient permanently discontinued GATTEX due to AEs.

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