Table 4.
Overview of HAVEN 2 adverse events
| Emicizumab | ||||
|---|---|---|---|---|
| Group A: once weekly, n = 68 | Group B: every 2 wk, n = 10 | Group C: every 4 wk, n = 10 | Total, N = 88 | |
| Participants with ≥1 adverse event, n (%) | 63 (96.2) | 9 (90.0) | 10 (100) | 82 (93.2) |
| Total adverse events, n | 615 | 43 | 54 | 712 |
| Participants with ≥1 adverse event, n (%) | ||||
| Adverse event with fatal outcome | 0 | 0 | 0 | 0 |
| Serious adverse event | 14 (20.6) | 1 (10.0) | 2 (20.0) | 17 (19.3)* |
| Adverse event leading to treatment withdrawal | 0 | 0 | 1 (10.0) | 1 (1.1)† |
| Adverse event leading to dose modification/interruption | 0 | 0 | 0 | 0 |
| Grade ≥3 adverse event | 11 (16.2) | 1 (10.0) | 3 (30.0) | 15 (17.0)‡ |
| Related adverse event | 22 (32.4) | 2 (20.0) | 6 (60.0) | 30 (34.1)§ |
| Local injection-site reaction | 19 (27.9) | 2 (20.0) | 6 (60.0) | 27 (30.7)‖ |
| Adverse events of special interest, n (%) | ||||
| Systemic hypersensitivity/anaphylactic/anaphylactoid reaction¶ | 1 (1.5) | 0 | 0 | 1 (1.1) |
| Thromboembolic event | 0 | 0 | 0 | 0 |
| TMA | 0 | 0 | 0 | 0 |
Multiple occurrences of the same adverse event in 1 individual are counted only once except for in the “Total adverse events” row, in which multiple occurrences of the same adverse event are counted separately.
Seventeen participants experienced 21 events: device-related infection (n = 2), muscle hemorrhage (n = 2), hemorrhage (n = 2), asthma (n = 2), mouth hemorrhage (n = 1), appendicitis (n = 1), bronchiolitis (n = 1), catheter site infection (n = 1), epididymitis (n = 1), clavicle fracture (n = 1), fall (n = 1), head injury (n = 1), ligament sprain (n = 1), mouth injury (n = 1), positive for ADAs with neutralizing potential (n = 1), ketoacidosis (n = 1), and headache (n = 1).
One participant withdrew from emicizumab treatment due to ADAs with neutralizing potential and subsequent lack of efficacy.
Fifteen participants experienced 19 events: asthma (n = 3), hemorrhage (n = 2), appendicitis (n = 1), catheter site infection (n = 1), device-related infection (n = 1), epididymitis (n = 1), sinusitis (n = 1), sleep apnea syndrome (n = 1), mouth hemorrhage (n = 1), chest pain (n = 1), clavicle fracture (n = 1), positivity for ADAs with neutralizing potential (n = 1), diabetes mellitus (n = 1), ketoacidosis (n = 1), pain in extremity (n = 1), and headache (n = 1).
Thirty participants experienced 67 events: injection-site reaction (n = 57), indeterminable ABO blood type (n = 3), eosinophil count increased (n = 1), positive for ADAs with neutralizing potential (n = 1), ecchymosis (n = 1), erythema (n = 1), urticaria (n = 1), nausea (n = 1), and cough (n = 1).
All but 2 of the injection-site reactions were grade 1 in intensity and resolved within 1 to 5 days without treatment. No participant discontinued due to injection-site reactions.
The numbers for systemic hypersensitivity/anaphylactic/anaphylactoid reaction using the Sampson criteria include all participants who experienced indicative symptoms. One participant experienced symptoms of abdominal pain and cough that were identified as a potential systemic hypersensitivity/anaphylactic/anaphylactoid reaction using the protocol-defined search criteria; however, medical review of the case confirmed that this was not indicative of a systemic hypersensitivity, anaphylactic, or anaphylactoid reaction.