Outcomes according to inhibitor history and ITI treatment among people with severe hemophilia A
| . | Participants with, and without, an inhibitor . | ITI treatment among participants with inhibitor . | |||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|
| Total . | No inhibitor . | Inhibitor . | Inhibitor vs no inhibitor . | No ITI . | ITI . | No ITI vs ITI . | Successful ITI . | Partially successful ITI . | Failed ITI . | Successful ITI vs partially successful/failed ITI . | |
| N = 3659 . | n = 3086 . | n = 576 . | P value . | n = 70 . | n = 485 . | P value . | n = 91 . | n = 95 . | n = 299 . | P value . | |
| Clinical outcomes | |||||||||||
| Median number of treated bleeds (IQR) | 3 (1-10) | 3 (1-10) | 3, (1-9) | .688 | 6 (2-19) | 3 (1-8) | .081 | 3 (1-8) | 2 (0-7) | 6 (4-15) | <.001 |
| Joint procedures, % (any) | 28.5 | 28.6 | 28.0 | .781 | 39.1 | 25.7 | .020 | 23.9 | 22.1 | 38.5 | .132 |
| Health care use | |||||||||||
| ED visits, % (past 12 mo) | 21.8 | 20.4 | 29.2 | <.001 | 31.9 | 29.1 | .639 | 21.7 | 30.5 | 48.9 | <.001 |
| Hospitalization, % (past 12 mo) | 12.0 | 10.1 | 21.3 | <.001 | 33.8 | 19.6 | .007 | 13.4 | 20.4 | 38.9 | <.001 |
| Quality of life | |||||||||||
| Chronic pain, % (past 12 mo) | 39.8 | 39.6 | 41.0 | .494 | 67.7 | 36.6 | <.001 | 37.8 | 56.8 | 65.9 | .002 |
| Limited function, % (current) | 24.7 | 24.1 | 27.7 | .064 | 55.2 | 23.0 | <.001 | 17.9 | 19.3 | 43.8 | <.001 |
| Median number of missed days, past 12 mo (IQR)∗ | 0 (0-2) | 0 (0-2) | 0 (0-4) | <.001 | 5 (0-20) | 0 (0-2) | .182 | 0 (0-2) | 1 (0-2) | 2 (0- 10) | <.001 |
| Socioeconomic status | |||||||||||
| Unemployed, %† | 37.6 | 35.9 | 48.3 | .001 | 51.3 | 41.7 | .108 | 39.8 | 32.1 | 58.1 | .099 |
| Disabled, % | 13.5 | 13.3 | 15.1 | .233 | 18.0 | 10.1 | .002 | 12.3 | 11.4 | 8.9 | .410 |
| Low education, %‡ | 37.0 | 36.6 | 39.1 | .469 | 37.2 | 34.8 | .531 | 30.3 | 39.3 | 38.2 | .529 |
| Mortality | |||||||||||
| Deaths, n | 61 | 48 | 13 | 6 | 7 | 6 | -- | -- | -- | ||
| PYs | 9748 | 8152 | 1596 | 242.5 | 1304 | ||||||
| Crude rate (per 1000 PYs) | 6.3 | 5.8 | 8.2 | .550 | 24.7 | 5.4 | .012 | ||||
| Age-adjusted rate (per 1000 PYs) | 25.1 | 21.9 | 46.2 | .231 | 46.8 | 26.9 | .477 | ||||
| . | Participants with, and without, an inhibitor . | ITI treatment among participants with inhibitor . | |||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|
| Total . | No inhibitor . | Inhibitor . | Inhibitor vs no inhibitor . | No ITI . | ITI . | No ITI vs ITI . | Successful ITI . | Partially successful ITI . | Failed ITI . | Successful ITI vs partially successful/failed ITI . | |
| N = 3659 . | n = 3086 . | n = 576 . | P value . | n = 70 . | n = 485 . | P value . | n = 91 . | n = 95 . | n = 299 . | P value . | |
| Clinical outcomes | |||||||||||
| Median number of treated bleeds (IQR) | 3 (1-10) | 3 (1-10) | 3, (1-9) | .688 | 6 (2-19) | 3 (1-8) | .081 | 3 (1-8) | 2 (0-7) | 6 (4-15) | <.001 |
| Joint procedures, % (any) | 28.5 | 28.6 | 28.0 | .781 | 39.1 | 25.7 | .020 | 23.9 | 22.1 | 38.5 | .132 |
| Health care use | |||||||||||
| ED visits, % (past 12 mo) | 21.8 | 20.4 | 29.2 | <.001 | 31.9 | 29.1 | .639 | 21.7 | 30.5 | 48.9 | <.001 |
| Hospitalization, % (past 12 mo) | 12.0 | 10.1 | 21.3 | <.001 | 33.8 | 19.6 | .007 | 13.4 | 20.4 | 38.9 | <.001 |
| Quality of life | |||||||||||
| Chronic pain, % (past 12 mo) | 39.8 | 39.6 | 41.0 | .494 | 67.7 | 36.6 | <.001 | 37.8 | 56.8 | 65.9 | .002 |
| Limited function, % (current) | 24.7 | 24.1 | 27.7 | .064 | 55.2 | 23.0 | <.001 | 17.9 | 19.3 | 43.8 | <.001 |
| Median number of missed days, past 12 mo (IQR)∗ | 0 (0-2) | 0 (0-2) | 0 (0-4) | <.001 | 5 (0-20) | 0 (0-2) | .182 | 0 (0-2) | 1 (0-2) | 2 (0- 10) | <.001 |
| Socioeconomic status | |||||||||||
| Unemployed, %† | 37.6 | 35.9 | 48.3 | .001 | 51.3 | 41.7 | .108 | 39.8 | 32.1 | 58.1 | .099 |
| Disabled, % | 13.5 | 13.3 | 15.1 | .233 | 18.0 | 10.1 | .002 | 12.3 | 11.4 | 8.9 | .410 |
| Low education, %‡ | 37.0 | 36.6 | 39.1 | .469 | 37.2 | 34.8 | .531 | 30.3 | 39.3 | 38.2 | .529 |
| Mortality | |||||||||||
| Deaths, n | 61 | 48 | 13 | 6 | 7 | 6 | -- | -- | -- | ||
| PYs | 9748 | 8152 | 1596 | 242.5 | 1304 | ||||||
| Crude rate (per 1000 PYs) | 6.3 | 5.8 | 8.2 | .550 | 24.7 | 5.4 | .012 | ||||
| Age-adjusted rate (per 1000 PYs) | 25.1 | 21.9 | 46.2 | .231 | 46.8 | 26.9 | .477 | ||||
Data were collected from the CC registry between 2013 to 2017; the following number of participants had missing data on ED visits (n = 55); hospitalization (n = 47), bleeding events (n = 754), joint procedures (n = 66); CVAD (n = 149); ICH (n = 126); chronic pain (n = 96); limited function (n = 107), employment (n = 267); and education (n = 114). Bold values are statistically significant.
CVAD, central venous access device inserted; HS, high school; ICH, intracerebral hemorrhage.
Missed days of school and work among students and employed adults, respectively.
Among adults aged 22 to 64 years, unemployed includes those who are unemployed and able, disabled/not able to work, retired, or are still in school.
Defined as high school degree or less, among adults aged ≥22 years.