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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
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172