Introduction
Materials and methods
Patient inclusion
Instrument description and scoring
During the last 14 days: |
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1. Have you felt tired? |
2. Have you had to rest? |
3. Have you been exhausted for days after you exerted yourself? |
4. Have you had difficulties getting oud of bed in the morning? |
5. Has your body felt heavy? |
6. Have you been unable to bring yourself to do things or have you been apathic? |
7. Has it been a problem for you to ration your strength? |
8. Have you been short of breath? |
9. Have you had a tendency to bleed? |
10. Have you had problems with susceptibility to infections? |
11. Have you had problems with swelling or inflammation of the mouth? |
12. Have you had problems sleeping? |
13. Has your everyday life been affected by pain? |
14. Have you had difficulty standing for an extended period? |
15. Has going for a long walk caused you difficulties? |
16. Have you had difficulty climbing stairs? |
17. Has your normal routine been disrupted? |
18. Have your work or other daily activities been restricted? |
19. Have you had problems coping with the household chores? |
20. Have you had no energy left for your personal life and hobbies? |
21. Have you found it a problem to give op sporting activities? |
22. Has it bothered you that you were unable to make plans? |
23. Has it annoyed you that you had to explain yourself, e.g. why you have been unable to do this or that? |
24. Has it bothered you that you were unable to be spontaneous? |
25. Has it bothered you that you had to be careful? |
26. Have you had to take care all the time to avoid picking up infections? |
27. Have you had difficulty concentrating? |
28. Has everything revolved around your illness? |
29. Has it bothered you repeatedly having to face up to your illness? |
30. Have you had signs (e.g. pallor, bruises, dark urine, yellow skin) that repeatedly reminded you of your illness? |
31. Has it bothered you to be classified as ill? |
32. Has it bothered you that your relatives were upset by your illness? |
33. Have you felt supported by friends and family? |
34. Have you worried a lot? |
35. Has it bothered you that you had to look out for minor symptoms because they could mean something bad? |
36. Have you felt good about your body? |
37. Have you been bothered by your blood count results? |
38. Have you been afraid of a deterioration in your blood count |
39. Have you been afraid that therapies might not work? |
40. Have you been afraid of a relapse or deterioration? |
41. Have you been concerned that there might not be any more therapy for you? |
42. Have you felt vulnerable? |
43. Have you felt at the mercy of your illness? |
44. Have you felt depressed? |
45. Did you feel irritable? |
46. Has the illness made you feel less attractive? |
47. Have you been less interested in sex? |
48. Have you been less able to enjoy sex? |
49. Have you felt that you were missing out on something in life? |
50. Have you been proud of what you achieved despite the illness? |
51. Have you been able to do what you wanted? |
52. Have you been troubled by thoughts of an uncertain future? |
During the last six months: |
53. Have you still been able to go on holiday as you wished? |
54. Have you missed the interaction with other patients? |
Do you have any further comments? |
Statistical analysis and presentation of results
Feasibility and validity of questionnaire
QoL analysis
Results
Patient characteristics
Whole cohort (n = 36) | PR (n = 21) | CR (n = 15) | P-score | |
---|---|---|---|---|
Median age in years (range) | 54 (21 -71) | 48 (30–71) | 55 (21–66) | P = 0,500 |
Female | 17 (46%) | 9 (43%) | 8 (53%) | P = 0,535 |
Median age at diagnose in years (range) | 30 (14–65) | 30 (17–65) | 30 (14–64) | P = 0,999 |
Diagnosis classification | ||||
Non-severe AA Severe AA Very severe AA | 8 (22%) 20 (56%) 8 (22%) | 5 (24%) 10 (48%) 6 (29%) | 3 (20%) 10 (67%) 2 (13%) | P = 0,462 |
Current presence of GPI deficient clone | ||||
> 1% > 40% (with overt hemolysis) | 10 (28%) 5 (14%) | 7 (33%) 5 (24%) | 3 (20%) 0 | P = 0,468 P = 0,042 |
Past IST treatment | ||||
Ciclosporin ATG ATG and ciclosporin ATG, ciclosporin and eltrombopag | 2 (6%) 9 (25%) 22 (61%) 3 (8%) | 1 (5%) 4 (19%) 15 (71%) 1 (5%) | 1 (7%) 5 (33%) 7 (47%) 2 (13%) | P = 0,178 |
Still on active treatment | 2 (6%) | 1 (5%) | 1 (7%) | P = 0.806 |
Median amount of years after IST (range) | 5 (0 – 41) | 5 (1–41) | 5 (0–37) | P = 0,499 |
Current median level of hemoglobin (g/dL) (range) | 15 (10 – 16) | 13 (10 – 16) | 15 (11 – 16) | P = 0,176 |
Current median thrombocyte count (× 103/ mm3) (range) | 141 (27–415) | 103 (27 – 226) | 174 (147 – 415) | P = 0,000 |
Questionnaire feasibility and internal consistency
Higher age is correlated with lower QoL scores
Coefficient | R2 | P-score | |
---|---|---|---|
Age (y) | -0.63x | 0.112 | 0.05 |
Years after last IST (y) | -0.19x | 0.009 | 0.52 |
Hemoglobin level (g/dL) | -1.50x | 0.004 | 0.71 |
Thrombocyte count (× 103/ mm3) | -0.10x | 0.065 | 0.13 |
Psychological wellbeing is affected, even after successful IST
Domain | Whole cohort (n = 36) | PR (n = 21) | CR (n = 15) | p-score |
---|---|---|---|---|
Role functioning | 2.08 (SD 0.57) | 2.22 (SD 0.57) | 1.88 (SD 0.53) | 0.07 |
Body image | 1.99 (SD 0.78) | 2.17 (SD 0.86) | 1.73 (SD 0.59) | 0.10 |
Emotional functioning | 1.97 (SD 0.70) | 2.11 (SD 0.75) | 1.76 (SD 0.58) | 0.14 |
Fear of progression | 1.96 (SD 0.69) | 2.12 (SD 0.78) | 1.73 (SD 0.46) | 0.08 |
Fatigue | 1.93 (SD 0.68) | 2.02 (SD 0.72) | 1.81 (SD 0.63) | 0.38 |
Cognitive functioning | 1.83 (SD 0.61) | 1.86 (SD 0.48) | 1.80 (SD 0.77) | 0.80 |
Illness intrusiveness | 1.79 (SD 0.59) | 1.92 (SD 0.63) | 1.61 (SD 0.50) | 0.14 |
Stigmatization | 1.78 (SD 0.75) | 1.95 (SD 0.81) | 1.53 (SD 0.61) | 0.10 |
Physical functioning | 1.76 (SD 0.82) | 1.71 (SD 0.70) | 1.82 (SD 0.98) | 0.70 |
Social support | 1.69 (SD 0.50) | 1.64 (SD 0.43) | 1.77 (SD 0.59) | 0.47 |
Other symptoms | 1.61 (SD 0.45) | 1.61 (SD 0.39) | 1.62 (SD 0.54) | 0.93 |
Infections | 1.40 (SD 0.43) | 1.38 (SD 0.47) | 1.43 (SD 0.37) | 0.72 |