Table 1 Comparison of the mean rating (Likert scale) of questionnaire items from the second consensus round between patients and medical experts. Importance rating: 1 = not at all, 2 = a little, 3 = quite a bit, 4 = very. Number of participants evaluating the questionnaire: AA patients n = 13, PNH patients n = 5, AA-PNH patients n = 2, experts n = 6

1. In general, do or did you feel tired?

3.3; 3.7

3.4; 3.8

2. Did you experience shortness of breath?

2.9; 3.7

2.6; 3.8

3. Do you have an increased bleeding tendency?

2.7; 3.8

1.9; 3.3

4. Were you limited in doing either your work or other daily as well as leisure time activities?

3.1; 3.3

2.7; 3.0

5. Did you have difficulties in concentrating on things?

2.4; 2.7

2.9; 2.5

6. Do you have any trouble doing strenuous and/or long-lasting activities?

(e.g. carrying a heavy bag, taking long walks)

3.1; 3.2

2.6; 2.7

7. Was your mood impaired?

(feeling depressed, being worried, feeling tense and others)

2.5; 2.7

3.1; 2.5

8. Did you have fever?

(from 38.1 °C at least 2 times or once ≥38.3 °C)

1.9; 3.8

2.1; 3.7

9. Did you record a high blood pressure?

(upper value > 140 mmHg, lower value > 90 mmHg)

1.9; 2.8

1.4; 2.7

10. Was your sleep impaired?

(difficulties in falling asleep, staying asleep or waking up)

2.7; 2.2

2.9; 2.2

11. Have you been in pain?

2.2; 3.2

2.7; 3.8

12. Have you noticed any changes in hair, skin and/or mucous membranes?

2.7; 2.7

2.1; 2.0

13. Did you feel dizzy/lightheaded/unsteady?

2.4; 2.8

2.4; 2.5

14. Did you record a too low or to high pulse?

(< 60 beats/minute or > 90 beats/minute)

2.5; 2.8

1.6; 2.2

15. Did you experience one or more changes in your sensory perception?

2.0; 2.3

1.6; 2.5

16. Did you suffer from muscle cramps/spasms?

2.8; 2.5

N/A

17. Did you experience tremor ^a and/or ataxia ^b?

(^a uncontrolled shaking movements of the whole or parts of the body; ^b lack of coordination of muscle movements)

1.7; 2.5

N/A

18. Was the time with your family and/or your availability to your children impaired?

2.5; 2.7

N/A

19. Do you need to stay in bed or a chair during the day?

1.9; 2.3

1.7; 2.2

20. Did you have digestive/gastrointestinal problems?

2.2; 2.3

2.1; 3.0

21. Did you have a cough?

1.7; 2.5

2.0; 2.5

22. Did you have swelling/edema of your limbs?

1.9; 2.3

1.9; 2.3

23. Did you lose or gain weight unintentionally?

2.0; 2.2

2.4; 2.3

24. Did you experience palpitations ^c?

(^c unpleasant sensation of irregular and/or forceful beating of the heart)

1.7; 1.8

2.3; 2.3

25. Did your skin feel itchy?

1.5; 2.0

1.6; 1.8

26. Have you noticed a dark discoloration of the urine?

N/A

2.9; 3.8

27. Have you noticed a yellowish discoloration of your ‘white of the eye’?

N/A

1.6; 3.2

28. Men only: Do you suffer from erectile dysfunction ^d? (^d inability to achieve or to maintain an erection during sexual activity)

N/A

1.4; 3.0

29. Did you have difficulties in swallowing things?

N/A

1.6; 2.7

30. When was your last IV infusion of eculizumab?

N/A

1.9; 3.3