| Outcome | CABLIVI
(n=72) |
Placebo
| p value |
| Primary Outcomes | |||
|
Time to normalization of platelet count | |||
|
25th Percentile (95% CI)—days | 1.75 (1.65-1.87) | 1.94 (1.70-2.64) | |
|
50th Percentile (95% CI)—days | 2.69 (1.89-2.83) | 2.88 (2.68-3.56) | |
|
75th Percentile (95% CI)—days | 2.95 (2.85-3.81) | 4.50 (3.78-7.79) | |
|
Rate ratio for normalization of platelet count, CABLIVI vs placebo (95% CI) | 1.55 (1.09-2.19) | 0.01 | |
|
Key secondary outcomes | |||
|
Composite of TTP-related death, recurrence of TTP, or major thromboembolic event during the double-blind treatment period—no. (%) | 9 (12) | 36 (49) | <0.001 |
|
TTP-related death | 0 | 3 (4) | |
|
Recurrence of TTP: exacerbation* | 3 (4) | 28 (38) | |
|
Major thromboembolic event | 6 (8) | 6 (8) | |
|
Recurrence of TTP at any time during the trial—no. (%)* | 9 (12) | 28 (38) | 0.001 |
|
Refractory TTP—no. (%)† | 0 | 3 (4) | 0.06 |
| Other secondary outcomes‡ | |||
|
Mean number of days of PEX (95% CI) | 5.8 (4.8-6.8) | 9.4 (7.8-11.0) | |
|
Mean volume of plasma exchanged (95% CI) | 21.3 L
(18.1-24.6) | 35.9 L
(27.6-44.2) | |
|
Mean number of days of hospitalization (95% CI) | 9.9 (8.5-11.3) | 14.4 (12.0-16.9) | |
|
Number of patients admitted to the ICU (%)§ | 28 (39) | 27 (37) | |
|
Mean number of days in the ICU (95% CI) | 3.4 (2.6-4.2) | 9.7 (5.3-14.1) | |
CABLIVI addresses the pathophysiological platelet adhesion that leads to the formation of microthrombi and the resultant mortality and morbidity seen in aTTP1,2
- Patients treated with CABLIVI were 1.55 times more likely to achieve platelet count normalization at any given time point compared with patients treated with placebo (P<0.01)2
- Clinically relevant reduction in the composite of aTTP-related death, recurrence of aTTP, or a major thromboembolic event2
- Prevention of aTTP relapses when treatment was extended until resolution of underlying disease2
- Potential to prevent refractory disease and accelerate normalization of markers of organ damage2
- Reduction in use of PEX and length of stay in the ICU and hospital2
- Safety profile in line with previous study results and mechanism of action1,3
* Recurrence was defined as a new decrease in the platelet count after initial normalization of the platelet count, necessitating reinitiation of PEX. According to the consensus terminology, a recurrence within 30 days after the end of daily PEX is considered to be an exacerbation, and a recurrence that occurs more than 30 days after the end of daily PEX is considered to be a relapse.
† Refractory TTP was defined as the lack of a doubling of the platelet count after 4 days of treatment and an LDH level that remained above the upper limit of the normal range.
‡These outcomes were assessed during the trial treatment period.
§ Admission to the ICU for administration of PEX is standard practice at some centers and is not necessarily indicative of more severe clinical presentation.
ICU=intensive care unit; LDH=lactate dehydrogenase; TTP=thrombotic thrombocytopenic purpura.