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Original Research

Identifying a subpopulation with higher likelihoods of early response to treatment in a heterogeneous rare disease: a post hoc study of response to teduglutide for short bowel syndrome

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Pages 1267-1277 | Published online: 25 Jul 2018

Figures & data

Table 1 Baseline characteristics of SBS–IF patients in the STEPS trial

Table 2 Prediction models for response to teduglutide and placebo

Figure 1 Difference in response between teduglutide and placebo in potential subpopulations.

Figure 1 Difference in response between teduglutide and placebo in potential subpopulations.

Figure 2 Response in potential subpopulations by treatment.

Figure 2 Response in potential subpopulations by treatment.

Figure 3 Efficiency frontier.

Figure 3 Efficiency frontier.

Table 3 Comparison of baseline characteristics in the higher- and lower-response subpopulations

Table 4 Comparison of clinical outcomes between teduglutide and placebo in subpopulations

Figure S1 Illustration of subpopulation identification based on effect score.a

Notes: aPatients were ranked by their individual effect scores from highest to lowest and were sequentially grouped together as potential subpopulations by 10% increments, starting from the top 20% of patients with the highest individual effect score, until all patients (100%) were included.

Figure S1 Illustration of subpopulation identification based on effect score.aNotes: aPatients were ranked by their individual effect scores from highest to lowest and were sequentially grouped together as potential subpopulations by 10% increments, starting from the top 20% of patients with the highest individual effect score, until all patients (100%) were included.

Table S1 List of Independent Ethics Committees or Institutional Review Boards that approved the study