Table 2.
Primary and Secondary Outcomes
| Outcome | TA | rVWF | Adjusted Mean Diff* (95% CI) | P-Value (Trt Diff) |
|---|---|---|---|---|
| Primary Outcome | ||||
| Pictorial Blood Assessment Chart (PBAC) across 2 cycles, mean (SD) | 225 (207) | 272 (223) | 46 (2, 90) | 0.039 |
| Cycle 1 | 217 (207) | 311 (263) | ||
| Cycle 2 | 233 (209) | 234 (168) | ||
| ln(PBAC), mean (SD) | 5.1 (0.9) | 5.3 (0.8) | 0.3 (0.1, 0.4) | 0.004 |
| Secondary Outcomes & Quality of Life | ||||
| Continuous, mean (SD) | ||||
| Cycle Length | 7.1 (4.5) | 7.0 (3.9) | 0.0 (-0.7, 0.7) | 0.998 |
| Ruta Menorrhagia | 32.4 (13.5) | 34.8 (13.0) | 2.4 (-1.0, 5.8) | 0.168 |
| CES-D | 13.5 (10.6) | 11.0 (10.2) | -2.5 (-4.9, 0.1) | 0.055 |
| SF-36 | ||||
| Physical functioning | 84.4 (19.6) | 83.6 (20.0) | -0.8 (-4.8, 3.2) | 0.681 |
| Role limitations of physical health | 70.1 (39.1) | 67.4 (37.7) | -2.4 (-12.4,7.6) | 0.633 |
| Role limitations of emotional health | 63.9 (41.7) | 68.5 (39.0) | 4.8 (-5.7, 15.3) | 0.362 |
| Energy/fatigue | 50.3 (20.6) | 47.9 (22.8) | -2.3 (-7.4, 2.8) | 0.375 |
| Emotional well-being | 69.2 (17.7) | 72.9 (20.0) | 3.6 (-0.3, 7.4) | 0.068 |
| Social functioning | 76.4 (21.9) | 80.9 (20.2) | 4.5 (-2.3, 11.3) | 0.189 |
| Pain | 71.2 (23.5) | 68.8 (24.2) | -2.4 (-9.2, 4.5) | 0.489 |
| General health | 58.8 (19.9) | 59.3 (21.2) | 0.5 (-3.2, 4.3) | 0.786 |
| CDC HRQoL | ||||
| Physically Unhealthy Days | 3.1 (4.0) | 1.9 (3.2) | -1.0 (-2.3, 0.2) | 0.098 |
| Mentally Unhealthy Days | 4.7 (5.1) | 5.7 (7.0) | 0.7 (-1.6, 3.0) | 0.557 |
| Categorical, frequency (%) | ||||
| Cycle Severity Rating | 1.4 (0.8, 2.7)* | 0.266 | ||
| 0: Mild bleeding, much less than usually experienced | 25 (34.7%) | 17 (23.6%) | ||
| 1: Moderate bleeding, less than usually experienced | 29 (40.3%) | 33 (45.8%) | ||
| 2: Moderately severe bleeding, but not as bad as the worst menstrual bleeding experienced | 14 (19.4%) | 22 (30.6%) | ||
| 3: Severe bleeding, as bad as the worst menstrual bleeding ever experienced | 4 (5.6%) | 0 (0%) | ||
| No. with Clots | 69 (95.8%) | 67 (93.1%) | 0.2 (0,1174) * | 0.724 |
| No. with Flooding | 32 (44.4%) | 43 (59.7%) | 3.8 (1.3, 10.7)* | 0.014 |
| No. Cycle Length > 5 days | 34 (47.2%) | 39 (54.2%) | 1.8 (0.7, 5.0)* | 0.221 |
| Healthcare Utilization | ||||
| Days lost from school/work in last 2 cycles | NA | NA | ||
| 0 | 28 (77.8%) | 29 (80.6%) | ||
| 1 | 3 (8.3%) | 3 (8.3%) | ||
| 2 | 3 (8.3%) | 2 (5.6%) | ||
| 3 | 2 (5.6%) | 0 (0.0%) | ||
| 4 or more | 0 (0.0%) | 2 (5.6%) | ||
| Received iron infusion(s) in last 2 cycles | NA | NA | ||
| 0 | 34 (94.4%) | 33 (91.7%) | ||
| 1 | 1 (2.8%) | 3 (8.3%) | ||
| 2 | 1 (2.8%) | 0 (0.0%) | ||
| Received RBC transfusion(s) in last 2 cycles | ||||
| 0 | 36 (100%) | 36 (100%) | ||
| ER visits for heavy periods in last 2 cycles | NA | NA | ||
| 0 | 34 (94.4%) | 36 (100%) | ||
| 1 | 1 (2.8%) | 0 (0.0%) | ||
| 2 | 1 (2.8%) | 0 (0.0%) | ||
| Hospitalizations in last 2 cycles | ||||
| 0 | 36 (100%) | 36 (100%) | ||
| Satisfaction Survey | ||||
| Rating rVWF vs past treatment | ||||
| Much more satisfied | NA | 18 (50.0%) | NA | NA |
| Somewhat more satisfied | NA | 8 (22.2%) | NA | NA |
| About as satisfied | NA | 5 (13.9%) | NA | NA |
| Somewhat less satisfied | NA | 4 (11.1%) | NA | NA |
| Much less satisfied | NA | 1 (2.8%) | NA | NA |
| Difficulty of rVWF administration | ||||
| Not difficult at all | NA | 13 (36.1%) | NA | NA |
| Somewhat difficult | NA | 14 (38.9%) | NA | NA |
| No more difficult than past treatment | NA | 4 (11.1%) | NA | NA |
| Somewhat more difficult than past treatment | NA | 3 (8.3%) | NA | NA |
| Much more difficult than past treatment | NA | 2 (5.6%) | NA | NA |
| Likelihood to use rVWF in the future | ||||
| Very likely | NA | 14 (38.9%) | NA | NA |
| Somewhat likely | NA | 8 (22.2%) | NA | NA |
| About as likely | NA | 3 (8.3%) | NA | NA |
| Somewhat unlikely | NA | 6 (16.7%) | NA | NA |
| Very unlikely | NA | 5 (13.9%) | NA | NA |