Table 3.
Change in Number of Duodenal Polyps From Baseline in the Intention-to-Treat Analysis
| No. of Participants |
No. of Duodenal Polyps | |||||
|---|---|---|---|---|---|---|
| Baseline Median (IQR) |
6-mo Follow-up Median (IQR) |
Change (6-mo Follow-up - Baseline) Median (IQR)a,b |
Net Between-Group Difference (95% CI)b,c |
PValue | ||
| All participants | ||||||
| Sulindac-erlotinib | 46 | 13.5 (8.0 to 28.5) | 10.0 (9.0 to 11.0) | −2.8 (−4.0 to −1.5) | −8.0 (−12.2 to −4.7) | <.001 |
| Placebo | 46 | 10.5 (7.0 to 26.8) | 17.0 (13.1 to 20.0) | 4.3 (3.1 to 5.5) | ||
| Classic FAPd | ||||||
| Sulindac-erlotinib | 32 | 19.0 (9.5 to 32.2) | 14.5 (12.5 to 17.0) | −2.1 (−4.0 to −0.5) | −7.1 (−13.2 to −3.0) | <.001 |
| Placebo | 32 | 16.0 (7.8 to 32.2) | 20.5 (16.7 to 27.9) | 4.0 (2.5 to 5.6) | ||
| Attenuated FAPe | ||||||
| Sulindac-erlotinib | 14 | 7.5 (4.2 to 11.0) | 4.0 (4.0 to 4.0) | −4.3 (−6.0 to −2.5) | −9.7 (−15.2 to −5.4) | <.001 |
| Placebo | 14 | 6.5 (5.2 to 13.0) | 11.0 (9.0 to 13.0) | 4.9 (3.6 to 6.0) | ||
| Genetic Diagnosisf | ||||||
| Sulindac-erlotinib | 39 | 11.0 (7.0 to 20.0) | 8.0 (6.0 to 10.0) | −3 (−4.8 to −2.0) | −9 (−13.1 to −5.7) | <.001 |
| Placebo | 42 | 10.0 (6.2 to 30.8) | 17.2 (14.5 to 20.0) | 5.0 (4.0 to 6.0) | ||
Abbreviations: FAP, familial adenomatous polyposis; IQR, interquartile range.
Change is calculated individually for each participant (6-month polyp burden minus baseline polyp burden). The median change within treatment and placebo groups represents the 50th percentile of these calculated changes.
The median of the differences does not necessarily equal the difference between the medians.
Hodges-Lehmann estimates of net difference reflect the difference in the group change from 6 months to baseline. These estimates were calculated for each sample. Percentile bootstrap confidence intervals were calculated for the Hodges-Lehmann estimator.
Classic FAP was defined as presentation with more than 100 colonic adenomas and either (1) multiple family members with a classic FAP phenotype or (2) an adenomatous polyposis coli (APC) mutation in a region of the gene known to correlate with classic FAP, or (3) both.
Attenuated FAP was defined as presence of a mutation in a portion of the APC gene known to correlate with attenuated FAP and presentation of a milder phenotype in terms polyp density in the participant and the family. All participants with attenuated FAP had a confirmed mutation in the APC gene.
Genetic diagnosis was defined as identification of a pathologic mutation of the APC gene.