Background
Methods
Study participants
Assessment of HRQoL
Definitions
Statistical methods
Results
Study population
eGFR ≥60 ml/min/1.73 m2n = 77 | eGFR <60 ml/min/1.73 m2n = 10 | RRT (dialysis or KTx) n = 9 | p-value | |
---|---|---|---|---|
Age, yrs | 38.8 ± 13.1 | 53.0 ± 11.3 | 37.0 ± 8.6 | 0.004 |
Male gender | 39.1 % | 60.0 % | 100.0 % | 0.002 |
Diagnosis Fabry disease, months | 9 (6; 14) | 12 (8; 22) | 14 (7; 94) | 0.31 |
Enzyme replacement therapya
| 37.7 % | 60.0 % | 88.9 % | 0.01 |
BMI, kg/m2
| 22.4 (20.8; 24.9) | 23.6 (20.9; 30.8) | 21.9 (19.9; 24.4) | 0.36 |
Systolic blood pressure, mmHg | 118 ± 15 | 133 ± 21 | 131 ± 25 | 0.01 |
Diastolic blood pressure, mmHg | 75 ± 10 | 81 ± 14 | 81 ± 9 | 0.07 |
eGFR, ml/min/1.73 m2
| 96 (83; 110) | 37 (25; 45) | dialysis 7 (5; 13) KTx 37 (19; 54) | <0.001 |
Vascular event | 0.05 | |||
Minorb
| 13.2 % | 40.0 % | 22.2 % | |
Majorc
| 4.0 % | 10.0 % | 0 % | |
Pain | 44.3 % | 77.8 % | 71.4 % | 0.08 |
Pain therapy | 18.8 % | 60.0 % | 62.5 % | 0.002 |
VASd
| 2 (1; 3) | 2 (0; 3) | 2 (2; 3) | 0.46 |
Health related quality of life
eGFR >60 ml/min/1.73 m2n = 66 | eGFR <60 ml/min/1.73 m2n = 10 | RRT (dialysis or KTx) n = 9 | p-value | |
---|---|---|---|---|
Physical functioning | 85 (65; 100) | 52.5 (25; 75) | 50 (30; 55) | <0.001 |
Role physical | 100 (25; 100) | 37.5 (0; 100) | 0 (0; 37.5) | 0.004 |
Bodily pain | 68 (51; 100) | 41 (41; 64) | 22 (12; 51) | 0.004 |
General health | 57 (46; 79.5) | 45 (35; 52) | 25 (17; 40) | <0.001 |
Vitality | 52.5 (32.5; 65) | 40 (30; 50) | 30 (5; 30) | 0.01 |
Social functioning | 87.5 (62.5; 100) | 75 (50; 100) | 25 (25; 50) | <0.001 |
Role emotional | 100 (66.7; 100) | 100 (0; 100) | 33.3 (0; 33.3) | 0.008 |
Mental health | 66 (52; 80) | 64 (52; 76) | 44 (28; 60) | 0.08 |
Physical component summary | 49.8 (37.7; 54.7) | 35.9 (24.8; 44.4) | 29.4 (25.0; 35.5) | <0.001 |
Mental component summary | 47.1 (41.5; 54.5) | 49.8 (35.4; 56.3) | 30.1 (24.5; 34.9) | 0.01 |
Determinants of health related quality of life
Physical functioning | Role physical | Bodily pain | General health | |||||
---|---|---|---|---|---|---|---|---|
Univariate | Multivariate | Univariate | Multivariate | Univariate | Multivariate | Univariate | Multivariate | |
Age (year)
| −0.1 (−0.5; 0.4) | -- | −0.2 (−1.0; 0.6) | -- | 0.3 (−0.3; 0.8) | -- | −0.03 (−0.4; 0.4) | -- |
Gender (male)
|
−14.8 (−25.6; −4.0)
| −1.7 (−12.1; 8.6) |
−33.2 (−51.6; −14.9)
| −17.1 (−37.1; 2.9) |
−18.1 (−31.9; −4.3)
| 0.1 (−13.1; 13.2) | −21.6 (−30.8; −12.4) |
−12.4 (−22.7; −2.1)
|
Diagnosis (month)
a
| −2.4 (−6.8; 1.9) | -- | 0.1 (−7.6; 7.8) | -- | 0.3 (−5.3; 5.9) | -- | −3.3 (−7.2; 0.73) | -- |
Kidney function
b
| ||||||||
<60 vs. >60 ml/min/1.73 m2
|
−25.6 (−39.8; −11.4)
|
−16.3 (−29.9; −2.7)
| −25.1 (−52.6; 2.5) | −14.8 (−41.1; 11.4) | −14.0 (−33.7; 5.7) | −0.9 (−18.1; 16.3) | −15.8 (−29.4; −2.1) | −9.3 (−22.7; 4.1) |
RRT vs. >60 ml/min/1.73 m2
|
−31.9 (−46.7; −17.0)
|
−22.7 (−39.6; −5.9)
|
−48.2 (−78.6; −17.8)
|
−34.1 (−66.6; −1.5)
|
−36.3 (−57.0; −15.6)
|
−32.5 (−53.8; −11.2)
| −32.3 (−46.7; −18.0) |
−28.8 (−45.4; −12.2)
|
Vascular event
| ||||||||
Minorc
| −4.4 (−17.7; 8.9) | 4.3 (−7.2; 15.8) | −9,5 (−33.7; 14.6) | −10.5 (−32.7; 11.7) | −9.1 (−26.3; 8.0) | −2.6 (−17.1; 11.9) | −10.3 (−23.0; 2.5) | −6.6 (−18.4; 5.1) |
Majord
| 10.2 (−18.0; 38.3) | 18.4 (−3.7; 40.6) | 2.4 (−48.6; 53.4) | 19.1 (−23.6; 61.8) | 18.3 (−18.0; 54.7) | 24.6 (−3.4; 52.5) | −12.8 (−39.1; 13.5) | −5.0 (−26.7; 16.8) |
Pain
|
−23.3 (−23.3; −13.3)
|
−14.4 (−24.4; −4.4)
|
−37.7 (−56.3; −19.1)
|
−19.9 (−39.2; −0.6)
|
−37.2 (−49.6; −24.8)
|
−28.9 (−41.6; −16.3)
| −22.2 (−32.4; −12.2) |
−12.5 (−22.6; −2.5)
|
Pain-therapy
|
−20.1 (−32.1; −8.1)
| −10.4 (−22.0; 1.1) |
−38.8 (−58.6; −18.0)
| −16.4 (−38.6; 5.8) |
−26.0 (−40.9; −11.1)
| −10.3 (−24.8; 4.3) | −19.2 (−30.3; −8.1) | −2.8 (−14.2; 8.7) |
Vitality
|
Social functioning
|
Role emotional
|
Mental health
| |||||
Univariate
|
Multivariate
|
Univariate
|
Multivariate
|
Univariate
|
Multivariate
|
Univariate
|
Multivariate
| |
Age (year)
| −0.1 (−0.5; 0.3) | -- | −0.1 (−0.6; 0.4) | -- | −0.5 (−1.2; 0.2) | -- | −0.1 (−0.4; 0.3) | -- |
Gender (male)
|
−12.1 (−22.3; −1.9)
| 0.5 (−10.6; 11.6) |
−18.2 (−29.8; −6.5)
| −4.2 (−15.6; 7.2) |
−22.8 (−40.1; −5.5)
| −8.6 (−26.9; 9.8) | −7.7 (−16.1; 0.8) | −0.9 (−10.3; 8.5) |
Diagnosis (month)
a
| −1.1 (−5.2; 3.1) | -- | 0.3 (−4.5; 5.1) | -- | −4.6 (−11.6; 2.3) | -- | 0.7 (−2.7; 4.0 ) | -- |
Kidney function
b
| ||||||||
<60 vs. >60 ml/min/1.73 m2
| −6.7 (−21.1; 7.7) | −1.5 (−15.9; 13.0) | −3.9 (−19.6; 12.1) | 3.0 (−11.9; 18.0) | −15.6 (−40.1; 8.8) | −6.6 (−30.4; 17.2) | −2.0 (−14.0; 10.1) | 1.9 (−10.4; 14.2) |
RRT vs. >60 ml/min/1.73 m2
|
−25.3 (−40.4; −10.2)
|
−24.5 (−42.4; −6.6)
|
−41.5 (−58.3; −24.7)
|
−46.2 (−64.7; −27.6)
|
−45.7 (−71.3; −20.0)
|
−45.0 (−74.6; −15.5)
|
−17.4 (−30.0; −4.8)
|
−15.8 (−31.0; −0.6)
|
Vascular event
| ||||||||
Minorc
| −1.0 (−13.8; 11.9) | −0.7 (−13.4; 12.0) | −0.66 (−15.5; 14.2) | 0.5 (−12.1; 13.2) | −4.1 (−25.6; 17.5) | 0.4 (−19.7; 20.5) | −3.0 (−13.6; 7.6) | −1.5 (−12.3; 9.3) |
Majord
| −13.0 (−39.5; 13.5) | −14.1 (−37.7; 9.4) | 10.8 (−20.7; 42.3) | 11.2 (−13.1; 35.6) | 5.0 (−40.7; 50.6) | 7.2 (−31.6; 46.0) | −5.1 (−26.9; 16.7) | −5.4 (−25.4; 14.6) |
Pain
|
−19.8 (−29.7; −10.0)
|
−14.8 (−25.7; −3.9)
|
−20.0 (−31.8; −8.2)
| −9.3 (−20.3; 1.8) |
−20.8 (−38.1; −3.5)
| −5.1 (−22.8; 12.6) | −8.1 (−16.7; 0.5) | −1.6 (−10.9; 7.6) |
Pain-therapy
| −10.2 (−21.9; 1.4) | −0.6 (−13.0; 11.7) |
−14.6 (−28.1; −1.1)
| −7.6 (−20.3; 5.1) | −14.6 (−34.6; 5.3) | −8.1 (−28.4; 12.2) | −6.6 (−16.0; 2.8) | −4.8 (−15.3; 5.6) |
Physical component summary
|
Mental component summary
| |||||||
Univariate
|
Multivariate
|
Univariate
|
Multivariate
| |||||
Age (year)
| 0.03 (−0.2; 0.2) | -- | −0.1 (−0.39; 0.1) | -- | ||||
Male gender
|
−8.9 (−13.8; −4.0)
| −2.4 (−7.3; 2.5) |
−4.9 (−9.8; −0.02)
| −0.2 (−6.2; 4.5) | ||||
Diagnosis (month)
a
| −0.6 (−2.7; 1.4) | -- | −0.3 (−2.2; 1.7) | -- | ||||
Kidney function
b
| ||||||||
<60 vs. >60 ml/min/1.73 m2
|
−10.4 (−17.3; −3.4)
| −6.2 (−12.5; 0.1) | 1.1 (−5.7; 7.9) | 2.8 (3.5; −4.1) | ||||
RRT vs. >60 ml/min/1.73 m2
|
−16.0 (−23.7; −8,4)
|
−11.8 (−19.6; −4.0)
|
−14.3 (−21.8; −6.8)
|
−13.3 (−21.8; −4.7)
| ||||
Vascular event
| ||||||||
Minorc
| −2.9 (−9.5; 3.7) | −0.1 (−5.5; 5.4) | −0.6 (−6.8; 5.6) | −1.1 (−7.1; 4.9) | ||||
Majord
| 3.3 (−10.3; 16.8) | 8.2 (−2.0; 18.4) | −1.1 (−13.9; 11.7) | −3.4 (−14.6; 7.8) | ||||
Pain
|
−14.3 (−18.9; −9.7)
|
−9.1 (−13.8; −4.3)
| −4.1 (−9.0; 0.9) | −1.1 (−6.3; 4.1) | ||||
Pain-therapy
| −12.2 (−17.7; 6.8) |
−5.5 (−10.9; −0.1)
| −1.8 (−7.4; 3.7) | −0.4 (−6.3; 5.6) |