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Table 5 Treatment initiation, dosage and treatment duration in the efficacy population

From: Carglumic acid enhances rapid ammonia detoxification in classical organic acidurias with a favourable risk-benefit profile: a retrospective observational study

 

Isovaleric aciduria (n = 4)

Methylmalonic aciduria (n = 25)

Propionic aciduria (n = 19)

Neonates(n = 29)

Non-neonates(n = 19)

Total(n = 48)

Time to treatment initiation

 Mean, days (SD)

0.8 (1.0)

2.5 (5.5)

4.2 (9.5)

1.5 (2.8)

5.3 (10.6)

3.0 (7.2)

 Median (range)

0.5 (0–2)

0.0 (0–24)

2.0 (0–41)

1.0 (0–14)

1.0 (0–41)

1.0 (0–41)

First dose

 Mean, mg/kg (SD)

99.0 (63.3)

78.2 (64.9)

119.5 (83.2)

106.7 (75.3)

80.4 (70.5)

96.3 (73.8)

 Median, mg/kg (range)

81.6 (50.0–182.7)

51.0 (13.7–236.2)

106.0 (13.3–303.0)

100.0 (13.7–303.0)

50.0 (13.3–256.4)

75.5 (13.3–303.0)

Initial 24-h dose

 Mean, mg/kg (SD)

216.0 (217.9)

160.6 (108.3)

209.3 (186.9)

214.1 (179.1)

139.2 (81.7)

184.5 (151.8)

 Median, mg/kg (range)

131.6 (62.5–538.5)

129.0 (41.1–470.8)

190.2 (53.1–909.1)

170.2 (41.1–909.1)

100 (45.3–333.3)

156.4 (41.0–909.1)

Treatment duration

 Mean, days (SD)

3.5 (1.7)

6.1 (4.6)

5.2 (5.0)

4.9 (3.5)

6.5 (5.9)

5.5 (4.6)

 Median, days (range)

3.5 (2–5)

5.0 (1–15)

3.0 (1–15)

4.0 (1–15)

3.0 (1–15)

4.0 (1–15)

  1. SD, standard deviation