Table 35Summary of Outcome Measures and Their Measurement Properties

Outcome measureTypeConclusions about measurement propertiesMID
EORTC QLQ-C3030-item, patient-reported, cancer-specific, quality of life questionnaire using 4- and 7- point Likert scales.26 It consists of 5 multi-item functional scales (physical, role, emotional, cognitive, and social), 3 multi-item symptom scales (fatigue, nausea-vomiting, and pain), 6 single-item symptom scales (dyspnea, insomnia, appetite loss, constipation, diarrhea, and financial impact), and a 2-item GHS/QoL scale.Reliability of the EORTC QLQ-C30 in HL and DLBCL patients undergoing chemotherapy measured by Cronbach alpha was 0.79 for GHS/QoL, 0.51 to 0.85 for functional scales, and 0.82 to 0.86 for symptom scales/items.27

No evidence of validity, reliability, or responsiveness in patients with PNH.

Patients with cancer28:

• 5 to 10 points: small change

• 10 to 20 points: moderate change

• > 20 points: large change

No MID identified in patients with PNH.

FACIT-Fatigue13-item, patient-reported, fatigue-specific, quality of life questionnaire using a 5-point Likert scale.29Internal consistency by Cronbach alpha of 0.9529 and test-retest by intraclass correlation coefficient of 0.95.30

No evidence of validity, reliability, or responsiveness in patients with PNH.

No MID identified in patients with PNH.
LDH ≥ 1.5 x ULNLaboratory test used in diagnosis and monitoring of disease activity.31Associated with 4.8-fold increase in risk of mortality32 and increased risk of thromboembolism (OR 7.0; P = 0.013) in patients with PNH.33NA

DLBCL = diffuse large B-cell lymphoma; EORTC QLQ-C30 = European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30; GHS = global health status; HL = Hodgkin’s lymphoma; HRQoL = health-related quality of life; LDH = lactate dehydrogenase; MID = minimal important difference; OR = odds ratio; PNH = paroxysmal nocturnal hemoglobinuria; QoL = quality of life; ULN = upper limit of normal.

From: Clinical Review

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