REVOLADE, in combination with IST, has shown to be effective in treatment-naive SAA patients 1

REVOLADE improved Complete Response across all cohorts,
most effectively when started on Day 1 & continued for 6 months

  • Based on the results in Cohort 3, an additional 61 patients were recruited for
    an Extension Phase1

    • The median age was 28.0 years (range 5 to 82 years), 45.7% of patients were
      male, and the majority of patients were white (62.0%)1

Study Design

  • Single-arm, open-label, sequential cohort trial in patients 2 years of age or older (N=153)1

  • Primary end point: complete hematologic response at 6 months, defined by meeting the following 3 criteria: an absolute neutrophil count of >1000/microL, a hemoglobin level of >10 g/dL, and a platelet count of >100,000/microL1,2

  • REVOLADE was initiated at 150 mg in combination with h-ATG, 40 mg/kg/day,
    based on actual body weight, administered intravenously on Days 1 to 4 of the 6-month treatment period, and cyclosporine, 3 mg/kg, based on actual body weight, orally every 12 hours (total daily dose of 6 mg/kg/day) for 6 months starting on Day 11

  • The cohorts differed by REVOLADE regimen and the initiation of low-dose cyclosporine (maintenance dose) for patients who achieved a hematologic response at 6 months1 

    • Some patients in Cohort 2 and all patients in Cohort 3 and Cohort 3 + Extension cohort received low-dose CsA for an additional 18 months1

IST, immunosuppressive therapy; SAA, severe aplastic anemia.

*The P value is for testing the null hypothesis that the rate of complete response at 6 months would be 30% or greater.2