Primary and secondary efficacy end points (full analysis set)
| End point . | Ticagrelor group (n = 101) . | Placebo group (n = 92) . | Rate ratio (95% CI) . | P . |
|---|---|---|---|---|
| Vaso-occlusive crises (primary end point)*† | ||||
| Patients with events, n (%) | 70 (69) | 58 (63) | ||
| Total no. of events | 249 | 202 | ||
| Total follow-up, patient-years | 89.0 | 80.0 | ||
| Incidence rate (per year) | 2.74 | 2.60 | 1.06 (0.75-1.50) | .7597 |
| Painful crises†‡ | ||||
| Patients with events, n (%) | 69 (68) | 58 (63) | ||
| Total no. of events | 248 | 209 | ||
| Total follow-up, patient-years | 89.0 | 80.0 | ||
| Incidence rate (per year) | 2.73 | 2.67 | 1.02 (0.72-1.45) | .9037 |
| ACS†‡ | ||||
| Patients with events, n (%) | 5 (5) | 4 (4) | ||
| Total no. of events | 6 | 6 | ||
| Total follow-up, patient-years | 89.0 | 80.0 | ||
| Incidence rate (per year) | 0.05 | 0.06 | 0.76 (0.17-3.30) | .7136 |
| Duration of painful crises (days)§ | ||||
| Patients with events, n (%) | 69 (68) | 58 (63) | ||
| Total no. of days | 1476 | 1441 | ||
| Total follow-up, patient-years | 89.0 | 80.0 | ||
| Incidence rate (number of days per year) | 16.09 | 19.20 | 0.84 (0.50-1.40) | .4970 |
| Hospitalization or emergency department visit for vaso-occlusive crises†‡ | ||||
| Patients with events, n (%) | 42 (42) | 27 (29) | ||
| Total no. of events | 87 | 51 | ||
| Total follow-up, patient-years | 89.0 | 80.0 | ||
| Incidence rate (per year) | 0.87 | 0.61 | 1.43 (0.87-2.36) | .1636 |
| Days hospitalized for vaso-occlusive crises§ | ||||
| Patients with events, n (%) | 39 (39) | 23 (25) | ||
| Total no. of days | 526‖ | 256‖ | ||
| Total follow-up, patient-years | 89.0 | 80.0 | ||
| Incidence rate (number of days per year) | 5.07 | 3.01 | 1.68 (0.76-3.75) | .2011 |
| Acute SCD complications‡ | ||||
| Patients with events, n (%) | 3 (3) | 2 (2) | ||
| Total no. of events | 6 | 3 | ||
| Total follow-up, patient-years | 89.0 | 80.0 | ||
| Incidence rate (per year) | NP¶ | NP¶ | ||
| Days hospitalized for acute SCD complications§ | ||||
| Patients with events, n (%) | 0 (0) | 3 (3) | ||
| Total no. of days | 0 | 6 | ||
| Total follow-up, patient-years | 89.0 | 80.0 | ||
| Incidence rate (number of days per year) | NP¶ | NP¶ | ||
| Red blood cell transfusions due to SCD‡# | ||||
| Patients with events, n (%) | 21 (21) | 19 (21) | ||
| Total no. of events | 39 | 49 | ||
| Total follow-up, patient-years | 89.0 | 80.0 | ||
| Incidence rate (per year) | 0.41 | 0.53 | 0.77 (0.38-1.58) | .4822 |
| End point . | Ticagrelor group (n = 101) . | Placebo group (n = 92) . | Rate ratio (95% CI) . | P . |
|---|---|---|---|---|
| Vaso-occlusive crises (primary end point)*† | ||||
| Patients with events, n (%) | 70 (69) | 58 (63) | ||
| Total no. of events | 249 | 202 | ||
| Total follow-up, patient-years | 89.0 | 80.0 | ||
| Incidence rate (per year) | 2.74 | 2.60 | 1.06 (0.75-1.50) | .7597 |
| Painful crises†‡ | ||||
| Patients with events, n (%) | 69 (68) | 58 (63) | ||
| Total no. of events | 248 | 209 | ||
| Total follow-up, patient-years | 89.0 | 80.0 | ||
| Incidence rate (per year) | 2.73 | 2.67 | 1.02 (0.72-1.45) | .9037 |
| ACS†‡ | ||||
| Patients with events, n (%) | 5 (5) | 4 (4) | ||
| Total no. of events | 6 | 6 | ||
| Total follow-up, patient-years | 89.0 | 80.0 | ||
| Incidence rate (per year) | 0.05 | 0.06 | 0.76 (0.17-3.30) | .7136 |
| Duration of painful crises (days)§ | ||||
| Patients with events, n (%) | 69 (68) | 58 (63) | ||
| Total no. of days | 1476 | 1441 | ||
| Total follow-up, patient-years | 89.0 | 80.0 | ||
| Incidence rate (number of days per year) | 16.09 | 19.20 | 0.84 (0.50-1.40) | .4970 |
| Hospitalization or emergency department visit for vaso-occlusive crises†‡ | ||||
| Patients with events, n (%) | 42 (42) | 27 (29) | ||
| Total no. of events | 87 | 51 | ||
| Total follow-up, patient-years | 89.0 | 80.0 | ||
| Incidence rate (per year) | 0.87 | 0.61 | 1.43 (0.87-2.36) | .1636 |
| Days hospitalized for vaso-occlusive crises§ | ||||
| Patients with events, n (%) | 39 (39) | 23 (25) | ||
| Total no. of days | 526‖ | 256‖ | ||
| Total follow-up, patient-years | 89.0 | 80.0 | ||
| Incidence rate (number of days per year) | 5.07 | 3.01 | 1.68 (0.76-3.75) | .2011 |
| Acute SCD complications‡ | ||||
| Patients with events, n (%) | 3 (3) | 2 (2) | ||
| Total no. of events | 6 | 3 | ||
| Total follow-up, patient-years | 89.0 | 80.0 | ||
| Incidence rate (per year) | NP¶ | NP¶ | ||
| Days hospitalized for acute SCD complications§ | ||||
| Patients with events, n (%) | 0 (0) | 3 (3) | ||
| Total no. of days | 0 | 6 | ||
| Total follow-up, patient-years | 89.0 | 80.0 | ||
| Incidence rate (number of days per year) | NP¶ | NP¶ | ||
| Red blood cell transfusions due to SCD‡# | ||||
| Patients with events, n (%) | 21 (21) | 19 (21) | ||
| Total no. of events | 39 | 49 | ||
| Total follow-up, patient-years | 89.0 | 80.0 | ||
| Incidence rate (per year) | 0.41 | 0.53 | 0.77 (0.38-1.58) | .4822 |
Vaso-occlusive crisis was defined as a composite of painful crisis and/or ACS. Number of vaso-occlusive crises is defined as the count of events of vaso-occlusive crisis assessed throughout the treatment period from randomization to end-of-study visit or date of premature study discontinuation.
Events with an onset date within 7 days of the previous event onset date are not counted as new events.
Number of secondary end point events as assessed throughout the treatment period from randomization to end-of-study visit or date of premature study discontinuation (observed follow-up).
For event-free patients, the duration is set to 0.
The difference in number of days hospitalized for vaso-occlusive crises was due to a few patients with many days of hospitalization.
Incidence rate not presented (NP) owing to the low number of events.
Sickle cell–related red blood cell transfusions as identified by study physician review.