Skip to main content

Table 4 Long-term complications in tyrosinemia patients treated primarily either with liver transplantation or with nitisinone medication

From: Type 1 tyrosinemia in Finland: a nationwide study

  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
  1. aOne patient needed kidney transplant
  2. bThe patient had received liver transplant before the development of kidney dysfunction
  3. cAll patients with hypertension had received liver transplant and two had secondary cardiac hypertrophy
  4. dTwo patients had seizures, one had seizure in childhood and porphyrin crises at the age of 13 years, and one had facial paresis
  5. 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