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Table 1 Episode characteristics by patient diagnosis 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

Methylmalonic aciduria

Propionic aciduria

Neonates

Non-neonates

Total

N, patients

4

21

16

28

13

41

Gender

      

 Male

2

9

11

14

8

22

 Female

2

12

5

14

5

19

N, episodes

4

25

19

29a

19

48

Median age at episode, days

7 (neonate)

5 (neonate)

6.5 (neonate)

6

387

6.0 (neonate)

387.0 (non-neonate)

Median age at episode, months

44.84 (non-neonate)

2.53 (non-neonate)

12.73 (non-neonate)

0.20

12.73

0.2 (neonate)

12.73 (non-neonate)

Duration of episode,

 Mean, days (SD)

5.0 (1.8)

7.8 (6.0)

9.8 (10.5)

6.6 (4.8)

11.0 (10.8)

8.4 (7.9)

 Median, days

5.0

7.0

6.0

6.0

7.0

6.0

 Range, days

3–7

2–26

2–43

2–20

3–43

2–43

Concomitant treatmentb (%)

0 (0.0)

12 (48.0)

9 (47.4)

18 (62.1)

3 (15.8)

21 (43.8)

Scavengers

0

9

6

13

2

15

Haemodialysis/haemofiltration

0

3

3

5

1

6

Baseline NH3, μmol/L

 Mean (SD)

666.8 (692.2)

296.9 (206.2)

355.0 (326.8)

468.3 (365.3)

171.3 (75.7)

350.7 (321.3)

 Median

435.0

247.8

213.0

328.0

164.0

215.0

 Range

164.0–1633.0

76.1–868.0

76.0–1200.0

96.0–1633.0

76.0–385.0

76.0–1633.0

  1. aOne patient was treated for two episodes of hyperammonaemia within 28 days of birth
  2. bConcomitant treatment includes ammonia scavengers and haemodialysis/haemofiltration
  3. SD, standard deviation