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Table 4 Summary of normalisation of leucine concentrations (efficacy set)

From: Intravenous administration of a branched-chain amino-acid-free solution in children and adults with acute decompensation of maple syrup urine disease: a prospective multicentre observational study

  

Children (N = 22)

Adults (N = 80)

All (N = 102)

Leucine level at admission (µmol/L)

N

22

80

102

Mean (SD)

900 (267)

1008 (279)

985 (279)

Median [min; max]

964 [396; 1304]

972 [518; 1723]

972 [396; 1723]

Episodes with normalisation of leucine during hospitalisation

Number of episodes

 < 381 μmol/L (< 5 mg/100 mL)

18 (81.8%)

67 (83.8%)

85 (83.3%)

Leucine level at time of normalisation (µmol/L)

N

18

67

85

Mean (SD)

216 (116)

217 (105)

217 (107)

Median [min; max]

248 [30; 374]

213 [23; 381]

221 [23; 381]

% Change from admission

N

18

67

85

Mean (SD)

− 71 (20.9)

− 76 (14.8)

− 75 (16.2)

Median [min; max]

− 75 [− 97; − 22]

− 78 [− 97; − 31]

− 77 [− 97; − 22]

Rate of decrease (µmol/L/day)

N

18

67

85

Mean (SD)

− 267 (146)

− 276 (117)

− 274 (123)

Median [min; max]

− 264 [− 526; − 53]

− 267 [− 724; − 50]

− 267 [− 724; − 50]

Episodes without any normalisation of leucine during/at end of hospitalisation

Number of episodes by leucine category (plasma leucine concentration)

381–762 µmol/L (5–10 mg/100 mL)

4 (18.2%)

12 (15.0%)

16 (15.7%)

 > 762 µmol/L (10 mg/100 mL)

0 (0.0%)

1 (1.3%)

1 (1.0%)

  1. SD standard deviation