Adult Clinical Study Data

Post-Hoc: Time to Response & Residual Anatomy –
Early and Late Responders1

A Takeda-sponsored post-hoc analysis of STEPS and STEPS-2 identified factors
that may be associated with mean time to sustain PS reduction.1*

Early Responders1

3.6 Months

(n=27) (1.1 SD)

Adult patients who completed STEPS with a PS volume reduction of ≥20% from baseline at both Week-20 and Week-24 visits during STEPS.

Late Responders1

10.0 Months

(n=7) (6.1 SD)

Adult patients who completed STEPS-2 and had a PS volume reduction of ≥20% from baseline at any 2 consecutive visits during the extension study or at both the Week-24 visit in STEPS and the Month-1 visit in STEPS-2.

  • PS volume reduction was defined as 2 consecutive visits with a PS volume reduction of ≥20%1
  • Time to sustained PS volume reduction was defined as the period between the baseline visit for STEPS and the second consecutive visit at which PS volume reduction was ≥20%1
  • This is a post-hoc analysis of patients who completed STEPS and STEPS-2. Please note that efficacy in STEPS was based on the intent-to-treat population, while efficacy in STEPS-2 was based on study completers. Further randomized, controlled clinical studies are necessary to corroborate these findings1

SD, standard deviation.

* To evaluate factors associated with sustained PS volume reduction and early vs late response, a Takeda-sponsored post-hoc analysis was conducted for patients with SBS who were treated with GATTEX in the STEPS study (n=43) and who then continued treatment with GATTEX for up to 24 months in the extension study, STEPS-2 (n=37).1

Time to Response for Adults on GATTEX Varies1

Residual bowel anatomy influenced time to response1

Early Responders1

  • Lower percentage had
    colon-in-continuity (51.9%)
  • Patients had a lower
    mean percentage     of
    colon remaining (24.6%)
  • Fewer patients had an
    ileocecal valve (0%)

Late Responders1

  • Higher percentage had
    colon-in-continuity (100%)
  • Patients had a higher
    mean percentage     of
    colon remaining (57.1%)
  • More patients had an
    ileocecal valve (28.6%)

Discuss treatment expectations

Time to response may vary for each patient. Ongoing discussion on response time with your patient is important.1

Study limitations: This study was limited by the relatively small sample size, which may not have sufficient statistical power to identify factors associated with response. This study was also limited by the heterogeneity within the group of patients who had SBS with intestinal failure. The population consisted of patients who were subject to specific inclusion and exclusion criteria. Thus, the results may have limited generalizability.1*

A Kaplan-Meier analysis was conducted for time to sustained PS volume reduction; a multivariable Cox proportional hazards model was used for predictors of sustained PS volume reduction. Time to sustained PS volume reduction was described for early and late responders. Patient characteristics were described and compared between early vs late responders using chi-square tests for categorical variables and Wilcoxon rank sum tests for continuous variables.1

Characteristics of early vs late responders1

Early responders
(n=27)		 Late responders
(n=7)		 P value
Baseline demographics
Age (years), mean (SD) 51.6 (13.3) 52.4 (10.2) 0.966
Male, n (%) 14 (51.9) 3 (42.9) 0.672
White, n (%) 26 (96.3) 7 (100.0) 0.605
BMI (kg/m2), mean (SD) 22.3 (3.3) 22.3 (3.9) 0.881
Baseline PS characteristics, mean (SD)
Composite fluid balance (L/week) 17.3 (11.7) 18.3 (15.0) 0.966
Actual baseline PS volume (L/week) 13.5 (7.7) 12.7 (9.8) 0.639
Time since start on PS dependence (years) 7.4 (6.4) 3.5 (3.0) 0.287

This is a post-hoc analysis of patients who completed STEPS and STEPS-2. Please note that efficacy in STEPS was based on the intent-to-treat population, while efficacy in STEPS-2 was based on study completers. Further randomized, controlled clinical studies are necessary to corroborate these findings.1