TRIUMPH
Trial question
What is the effect of eculizumab in patients with paroxysmal nocturnal hemoglobinuria?
Study design
Multi-center
Double blinded
RCT
Population
Characteristics of study participants
60.0% female
40.0% male
N = 87
87 patients (52 female, 35 male).
Inclusion criteria: patients with paroxysmal nocturnal hemoglobinuria.
Key exclusion criteria: complement deficiency, an active bacterial infection, or a history of meningococcal disease and receipt of bone marrow transplantation.
Interventions
N=43 eculizumab (IV at a dose of 600 mg weekly for 4 weeks, followed 1 week later by a 900-mg dose and then 900 mg every other week through week 26).
N=44 placebo (matching placebo).
Primary outcome
Stabilization of hemoglobin levels in absence of transfusions
49%
0%
49.0 %
36.8 %
24.5 %
12.3 %
0.0 %
Eculizumab
Placebo
Significant
increase ▲
NNH = 2
Significant increase in stabilization of hemoglobin levels in the absence of transfusions (49% vs. 0%; ARD 49, 95% CI 19.93 to 78.07).
Secondary outcomes
Significant decrease in units of packed red cells transfusion (0 vs. 10; MD -10, 95% CI -15.93 to -4.07).
Significant increase in change in the QoL during treatment , global health status scale (10.9 points vs. -8.5 points; MD 19.4, 95% CI 7.89 to 30.91).
Safety outcomes
Significant difference in serious adverse events (9% vs. 20%).
Conclusion
In patients with paroxysmal nocturnal hemoglobinuria, eculizumab was superior to placebo with respect to stabilization of hemoglobin levels in the absence of transfusions.
Reference
Hillmen P, Young NS, Schubert J et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2006 Sep 21;355(12):1233-43.
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