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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

From: Development of a patient-reported outcome questionnaire for aplastic anemia and paroxysmal nocturnal hemoglobinuria (PRO-AA/PNH)

PRO-AA/PNH Questionnaire items Mean rating
AA
Patients; experts
PNH
Patients; experts
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
  1. N/A not applicable