Table 2.
Patient demographics and baseline characteristics
| Patient | Sex | Age (years) | BMI (kg/m2) | Smoker (N = never, F = former) | HHT gene mutated | Baseline ESS | Baseline Hgb (g/dL) | HHT GI bleeding (Y/N) |
|---|---|---|---|---|---|---|---|---|
| 1 | M | 58 | 32 | F | ENG | 4.17 | 12.2 | Y |
| 2 | F | 43 | 31 | N | SMAD4 | 5.25 | 10.4 | Y |
| 3 | F | 65 | 21 | F | ENG | 6.59 | 7.3 | Y |
| 4 | M | 69 | 36 | N | ACVRL1 | 4.17 | 9.6 | Y |
| 5 | M | 52 | 25 | N | ENG | 2.75 | 11.2 | Y |
| 6 | F | 51 | 43 | N | ACVRL1 | 4.35 | 11.6 | N |
| 7 | F | 63 | 25 | N | ACVRL1 | 4.06 | 8.7 | Y |
| Summary/mean | 4F/3M | 57 (SD = 9) | 30 (SD = 7) | 5N/2F | 3ACVRL1/3ENG/1SMAD4 | 4.47 (SD = 1.18) | 10.1 (SD = 1.7) | 6Y/1N |
HHT genes mutated are endoglin (ENG), activin receptor-like kinase 1 (ACVRL1) or SMAD4. Baseline ESS is epistaxis severity score (ESS) on Day 1 of dosing. Baseline Hgb is the mean of last two Hgb measures (includes Day1) of the run-in period. HHT GI bleeding is reported Y if patient diagnosed with chronic HHT-related GI bleeding; N if not. Race was white/Caucasian/European heritage for all seven, and non-hispanic ethnicity for all seven