ESPE Abstracts

Purpose: Modified ketogenic diet (MKD) is one treatment option for intractable epilepsy and metabolic conditions such as glucose transporter type 1 deficiency syndrome (GLUT1-DS) and pyruvate dehydrogenase complex (PDC) deficiency. MKD is a less restrictive diet than the classical ketogenic diet (KD) and thus more tolerable. Some studies indicate that prolonged KD treatment can negatively affect linear growth in children. Long-term data is missing regarding the effects of MKD treatment in children. This study was designed to prospectively assess growth in children treated with MKD for 24 months.

Methods: The included patients (n=38; 21 girls, 17 boys) had a mean±SD age of 6.1±4.8 years at MKD initiation. Underlying etiologies were genetic epilepsy (n=6), GLUT1-DS (n=7), PDC deficiency (n=9), cortical malformation (n=3), mitochondriopathy (n=2), tuberous sclerosis complex (n=2), encephalitis (n=2), stroke (n=2), Aicardi syndrome (n=1) and of unknown etiology (n=4). Thirty patients had seizures prior to MKD. Body weight, height and laboratory tests were assessed at baseline, 6, 12 and 24 months.

Results: After 24 months, 29 patients remained on MKD and 57% responded to the diet with >50% seizure reduction. Weight SDS and height SDS were stable over 24 months (P=0.054 and 0.10 respectively), i.e., weight SDS median (min-max) −0.4 (−2.5 to 3.5) at baseline and 0.2 (−1.8 to 2.1) after 24 months; and corresponding values for height SDS −0.4 (−4.0 to 2.5) to −0.3 (−2.9 to 1.4). BMI SDS increased from 0.2 (−3.3 to 4.5) to 0.7 (−0.9 to 2.6) after 24 months, P<0.005. The median plasma 3-hydroxybutyric acid levels increased from 0.05 mmol/l to 2.35 mmol/l (0.42–5.20 mmol/l) after 6 months, P<0.0001, but remained stable thereafter, i.e., 2.30 mmol/l (0.18–4.90 mmol/l) after 12 months and 2.30 mmol/l (0.16–4.90 mmol/l) at 24 months. Median pH was 7.38 (7.23–7.51) at baseline and 7.39 (7.34–7.45) after 24 months, P=0.45. At baseline, median IGF-I SDS was −0.15, which decreased to −0.85 after 6 months and to −1.0 at 12 months. From 12 to 24 months IGF-I SDS increased to 0.05. Median IGFBP3 SDS at baseline was 1.0 and was stable after 6 months, then decreased at 12 months to 0.1. From 12 to 24 months IGFBP3 SDS increased to 0.6.

Conclusions: MKD is as effective as KD with respect to seizure reduction. This first prospective longitudinal study demonstrates no negative impact on growth for MKD treatment in children.