Skip to main content

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