--- a +++ b/processing/MACCROBAT/25293719.txt @@ -0,0 +1,26 @@ +Our patient (ID 73387) is a female child, born by an emergency caesarean section at 34 weeks of gestation to consanguineous, first-cousin British Pakistani parents. +Antenatal scans showed that she was small for her gestation, weighing 1.19 kg at birth with a head circumference of 26.7 cm, considerably below the 0.4th centile. +Induction of labour had been attempted because of the growth retardation but had failed, leading to the emergency caesarean section. +The Apgar scores were 4 at 1 min, 7 at 5 min and 9 at 10 min. +She was admitted to the neonatal intensive care unit for continuous positive airway pressure ventilation. +At a few hours of age, she developed a severe lactic acidosis. +The initial lactic acid concentration was 22 mmol/l and subsequently increased to 63 mmol/l (normal range, 0.7–2.1 mmol/l). +She was treated with intravenous infusions of sodium bicarbonate and Tris-hydroxymethyl aminomethane (THAM), but it was never possible to correct the metabolic acidosis. +She also developed hypoglycaemia within hours of birth that was corrected with an intravenous infusion of 15% glucose (7.8 mg/kg/min). +The ammonia concentration was normal. +Urine organic acid profile showed massive excretion of lactic acid and increased phenolic acids, especially hydroxyphenyllactate. +Plasma amino acids showed raised concentrations of alanine and glutamine (1567 and 1369 μmol/l, respectively), consistent with the lactic acidosis; several other amino acids were also mildly increased. +There was gross generalised aminoaciduria. +Blood acylcarnitine analysis was normal. +Echocardiography showed a structurally normal heart and good ventricular function. +Cranial ultrasound showed bilateral intraventricular cysts within the frontal horns and anterior portions of the lateral ventricles. +The left-sided cysts were larger, up to 15 mm in diameter, whereas the largest cyst on the right was 8 mm in diameter. +The choroid plexuses were hyperechoic and irregular, suggesting previous intraventricular haemorrhage. +Abdominal ultrasound showed a distended urinary bladder but was otherwise unremarkable. +There was severe coagulopathy with an extended prothrombin time of 47.7 s (normal 12.3–16.6 s), a very low plasma albumin of 7 g/l (normal 35–50 g/l), otherwise normal liver function tests but a raised creatine kinase of 2700 U/l (normal <300 U/l). +She was transferred to a tertiary centre because of her worsening metabolic acidosis. +She started having seizures at ∼48 h of age. +Despite infusions of bicarbonate and THAM, her acidosis continued to worsen. +Muscle and skin biopsies were performed and the family agreed to the withdrawal of intensive care treatment. +She died aged 55 h. +All documented studies were approved and performed under the ethical guidelines issued by each of our Institutions for clinical studies, with written informed consent obtained from the family.