6/30/23, 2:21 AM TRIUMPH Pathway Feedback Search Clinical Topics Home Studies TRIUMPH TRIUMPH Trial question What is the effect of eculizumab in patients with paroxysmal nocturnal Hgburia? Study design Multi-center Double blinded RCT Population Characteristics of study participants 60.0% female N = 87 40.0% male 87 patients (52 female, 35 male) Inclusion criteria: patients with paroxysmal nocturnal Hgburia Key exclusion criteria: complement deficiency, an active bacterial infection, or a history of meningococcal disease and receipt of BMT 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 Significant increase in stabilization of Hgb levels in the absence of transfusions (ARD 49, 95% CI 19.93 to 78.07) Secondary outcomes Significant decrease in units of packed red cells transfusion (MD -10, 95% CI -15.93 to -4.07) Significant increase in change in the quality of life during treatment , global health status scale (10.9 vs. -8.5; MD 19.4, 95% CI 7.89 to 30.91) https://web.pathway.md/studies/recQFOqolL7vWjhyu 1/2 6/30/23, 2:21 AM TRIUMPH Pathway Safety outcomes Significant difference in serious adverse events (9% vs. 20%). Conclusion In patients with paroxysmal nocturnal Hgburia, eculizumab was superior to placebo with respect to stabilization of Hgb 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. Open reference URL https://web.pathway.md/studies/recQFOqolL7vWjhyu 2/2