Table 1. Summary of Paroxysmal Nocturnal Hemoglobinuria Patient Preference Questionnaire^© content^* [15].

Question (Q) Item	Response/Score
Q1. Overall preference question, which asked patients to indicate which of the 2 medications they prefer based on their experience with the 2 treatments	0 = eculizumab
	1 = ravulizumab
	2 = I do not have a preference
Q2a-2j. Questions evaluating preference for eculizumab or ravulizumab based on controlling fatigue (a), controlling symptoms other than fatigue (b), frequency of infusions (c), side effects of treatment (d), convenience of receiving treatment (e), being able to plan activities (f), effectiveness of the medication until the next infusion (g), anxiety related to the infusion (h), and your overall quality (i)	A 5-point ordered response scale ranging from 0 to 4
	0 = strongly prefer eculizumab
	1 = somewhat prefer eculizumab
	2 = I do not have a preference
	3 = somewhat prefer ravulizumab
And one write-in option (j)	4 = strongly prefer ravulizumab
Q3. One question asking patients to indicate which treatment factor was most important for their overall medication preference	Choice of a through j from Q2
Q4 through Q7. Four questions evaluating aspects of treatment with eculizumab	A 5-point scale ranging from 0 to 4
	0 = Not at all
	1 = A little bit
	2 = Some-what
	3 = Quite a bit
	4 = Very much
Q8 through Q11. Four questions evaluating those same aspects of treatment with ravulizumab	A 5-point scale ranging from 0 to 4
	0 = Not at all
	1 = A little bit
	2 = Some-what
	3 = Quite a bit
	4 = Very much

*The questions listed in this table are rephrased summaries adapted from the actual PNH-PPQ [15]. Critical elements needed for a valid assessment of patient preference are missing from this summary; for more information on the full PNH-PPQ, please review the publication by Kaiser K et al. Patient Prefer Adherence. 2020;14:705–715. For any questions or copyright information, please contact Alexion Pharmaceuticals, Inc.