| |
Transplantation, n = 7
|
Nitisinone, n = 14
|
P value
|
|---|
|
n
|
%
|
n
|
%
|
|---|
|
Kidney dysfunctiona
|
6
|
85.7
|
1b
|
7.1
|
0.001e
|
|
Hypertensionc
|
4
|
57.1
|
1
|
7.1
|
0.025f
|
|
Osteopenia/osteoporosis
|
5
|
71.4
|
2
|
14.3
|
0.017 g
|
|
Osteoporotic fractures
|
2
|
28.6
|
0
|
0.0
|
0.100
|
|
Growth failure
|
2
|
28.6
|
3
|
21.4
|
0.999
|
|
Learning difficulties
|
4
|
57.1
|
4
|
28.6
|
0.346
|
|
Neurological symptomsd
|
2
|
28.6
|
2
|
14.3
|
0.440
|
|
Developmental delay
|
1
|
14.3
|
2
|
14.3
|
0.999
|
|
Any complication
|
7
|
100
|
8
|
57.1
|
0.061 h
|
- aOne patient needed kidney transplant
- bThe patient had received liver transplant before the development of kidney dysfunction
- cAll patients with hypertension had received liver transplant and two had secondary cardiac hypertrophy
- dTwo patients had seizures, one had seizure in childhood and porphyrin crises at the age of 13 years, and one had facial paresis
- e−hP = 0.999 for each if adjusted for current age. Kidney dysfunction and hypertension appeared at the age of 14–25 years, osteoporosis/osteopenia and fractures at the age of 6–20 years, neurological symptoms/developmental delay at the age of 3–17 years and growth failure at the age of 1–3 years
