Table 3.
Health outcomes that are important for patients with autosomal inherited bleeding disorders.
| Consensus achieved by panel(s) |
||||
|---|---|---|---|---|
| # | Health outcomes | Definition | Patients and caregivers | Healthcare professionals |
| Bleeding episodes | ||||
| 10 | Frequency of bleeding episodes | The number of bleeding episodes within a year | ✓ | |
| 11 | Number of bleeding episodes per year that require treatment | The number of bleeding episodes that require treatment per year | ✓ | ✓ |
| 5 | Total number of bleeding episodes | The total number of bleeding episodes including severe, life-threatening, and intracranial bleeds | ✓ | |
| 6 | Total number of severe bleeding episodes | The total number of severe bleeding episodes a person has experienced | ✓ | |
| 7 | Total number of life-threatening bleeding episodes | The total number of life-threatening bleeding episodes the person has experienced | ✓ | ✓ |
| 8 | Total number of intracranial bleeds | The total number of intracranial bleeds a person has experienced | ✓ | |
| 9 | Severity of the bleeding episode | The severity of the bleeding episode | ✓ | ✓ |
| Treatment | ||||
| 12 | Response to treatment | How well does a person respond to treatment | ✓ | |
| 152 | Use of on-demand medication | The use of on-demand medication for a person’s bleeding disorder | ✓ | |
| 48 | Treatment side effects | The occurrence of treatment side effects including inhibitor status, allergic reactions | ✓ | |
| 50 | Allergic/hypersensitivity reactions | Allergic reactions to treatment | ✓ | |
| 51 | Inhibitor development | The presence of antibodies against factor concentrates and/or platelets | ✓ | |
| 52 | Inhibitor recurrence | The repeated development of antibodies against factor concentrates and/or platelets | ✓ | |
| Joint health | ||||
| 22 | Joint damage | The presence of joint damage due to joint bleeding episodes | ✓ | |
| 25 | Presence of target joints | The presence and number of target joints | ✓ | |
| 26 | Alteration in joint functional status | The alteration (improvement/deterioration) in joint functional capacity to perform functions of daily living | ✓ | |
| 151 | Increase in joint bleeds | Has the number of joint bleeds increased in a short period? | ✓ | |
| Complications | ||||
| 57 | Life-threatening complications | The occurrence of complications that threaten a person’s life | ✓ | ✓ |
| Menstruation | ||||
| 123 | Intensity of menstrual bleeding | Number of times a person needs to change their menstrual products per day | ✓ | ✓ |
| 122 | Duration and frequency of menstrual bleeding | The duration and frequency of menstrual bleeding | ✓ | |
| 125 | Impact of menstruation on daily life | The impact of menstrual bleeding on a person’s ability to engage in activities of daily living (including social relations, work/school) | ✓ | ✓ |
| 127 | Impact of menstruation on work/school absentee-ism | The impact of menstrual bleeding on a person’s ability to go to work/school | ✓ | |
| 128 | Impact of menstruation on maintaining social relationships | The impact of menstrual bleeding on a person’s ability to maintain social relationships | ✓ | |
| 129 | Impact of menstruation on family life | The impact of menstrual bleeding on a person’s family life | ✓ | |
| 130 | Impact of menstruation on the ability to enjoy life | The impact of menstrual bleeding on a person’s ability to enjoy life | ✓ | |
| 131 | Impact of menstruation on sleep | The impact of menstrual bleeding on a person’s sleep pattern | ✓ | |
| Pain | ||||
| 38 | Pain interference | The presence of pain and the interference with daily life | ✓ | |
| Knowledge, concerns, and impact of having a bleeding disorder | ||||
| 137 | Knowledge about the bleeding disorder | A person’s level of knowledge about the possible consequences and risks associated with a bleeding disorder | ✓ | |
| 140 | Concerns about the bleeding disorder | A person’s concern about his/her bleeding disorder | ✓ | |
| 147 | Concerns about inheritance of the bleeding disorder | The extent to which a person is concerned about the inheritance of his/her bleeding disorder | ✓ | |
| 148 | Concerns about pregnancy/miscarriage/giving birth | The extent to which a person is concerned about a possible pregnancy/miscarriage or giving birth | ✓ | |
| 136 | Ability to maintain basic self-care | The extent to which a person is able to continue to care for himself/herself as he/she gets older | ✓ | |
| 110 | Impact on emotions | The impact of the bleeding disorder on a person’s emotions | ✓ | |
| 27 | Age at diagnosis | The age at which the bleeding disorder was diagnosed by a hematologist | ✓ | |