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Table 1 Effect of low dose nitisinone on alkaptonuria-related variables

From: Preventive use of nitisinone in alkaptonuria

 

N patients

Duration

Dose

Δ uHGA (%)

Mean pTyrosine on treatment (µmol/l)

Suwannarat et al. [15]

9

3–4 mo

1.05 mg bid

− 94

755 ± 167

603 ± 114*

Introne et al. [8]

40

4 mo–3 yrs

2 mg od

− 95

670–826

Sloboda et al. [16]

3

3–5 yrs

0.2 mg od

− 90

305–416

Ranganath et al. [14]

8

4 wks

1 mg od

− 89.4

653 (450–806)

8

2 mg od

− 94.9

715 (506–865)

8

4 mg od

− 98.3

803 (657–1115)

8

8 mg od

− 99

813 (523–927)

  1. *After initiation of protein restriction
  2. Bid, twice daily; mo, months; N, number; od, once daily; uHGA, urinary homogentisic acid excretion