Table 5, Inclusion Criteria for the Systematic Review - Ravulizumab (Ultomiris)

Criteria	Description
Population	Patients aged ≥ 18 years with PNH Subgroups: disease severity (e.g., PNH clone size, serum LDH, history of thrombotic event)
Intervention	Ravulizumab IV infusion with the following dosage schedule: single loading dose of 2,400 mg (for body weight of ≥ 40 kg to < 60 kg), 2,700 mg (≥ 60 kg to < 100 kg), or 3,000 mg (≥ 100 kg) followed 2 weeks later by the first maintenance dose maintenance dose of 3,000 mg (for body weight of ≥ 40 kg to < 60 kg), 3,300 mg (≥ 60 kg to < 100 kg), or 3,600 mg (≥ 100 kg) every 8 weeks
Comparator	Eculizumab
Outcomes	Efficacy outcomes: survival thrombotic events HRQoL transfusions symptoms of PNH (e.g., fatigue, dyspnea, abdominal pain, dysphagia, erectile dysfunction) breakthrough hemolysis events complications of PNH other than thrombotic events (e.g., renal insufficiency, pulmonary hypertension) intravascular hemolysis (e.g., serum LDH) hemoglobin stabilization health care resource utilization (e.g., hospitalization) Harms outcomes: AEs, SAEs, deaths, WDAEs notable harms (e.g., meningococcal infection, serious infection, immunogenicity, infusion reaction)
Study designs	Published and unpublished phase III and IV RCTs

Criteria

Description

Population

Patients aged ≥ 18 years with PNH

Subgroups: disease severity (e.g., PNH clone size, serum LDH, history of thrombotic event)

Intervention

Ravulizumab IV infusion with the following dosage schedule:

single loading dose of 2,400 mg (for body weight of ≥ 40 kg to < 60 kg), 2,700 mg (≥ 60 kg to < 100 kg), or 3,000 mg (≥ 100 kg) followed 2 weeks later by the first maintenance dose
maintenance dose of 3,000 mg (for body weight of ≥ 40 kg to < 60 kg), 3,300 mg (≥ 60 kg to < 100 kg), or 3,600 mg (≥ 100 kg) every 8 weeks

Comparator

Eculizumab

Outcomes

Efficacy outcomes:

survival
thrombotic events
HRQoL
transfusions
symptoms of PNH (e.g., fatigue, dyspnea, abdominal pain, dysphagia, erectile dysfunction)
breakthrough hemolysis events
complications of PNH other than thrombotic events (e.g., renal insufficiency, pulmonary hypertension)
intravascular hemolysis (e.g., serum LDH)
hemoglobin stabilization
health care resource utilization (e.g., hospitalization)

Harms outcomes:

AEs, SAEs, deaths, WDAEs
notable harms (e.g., meningococcal infection, serious infection, immunogenicity, infusion reaction)

Study designs

Published and unpublished phase III and IV RCTs

From: Clinical Review

Ravulizumab (Ultomiris): CADTH Reimbursement Reviews and Recommendations: Therapeutic area: Paroxysmal nocturnal hemoglobinuria [Internet].

Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2022 Apr.

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Table 5Inclusion Criteria for the Systematic Review