Cholesterol supplementation does not improve developmental progress in Smith-Lemli-Opitz syndrome
DM Sikora, M Ruggiero, K Petit-Kekel, LS Merkens… - The Journal of …, 2004 - Elsevier
DM Sikora, M Ruggiero, K Petit-Kekel, LS Merkens, WE Connor, RD Steiner
The Journal of pediatrics, 2004•ElsevierOBJECTIVE: Smith-Lemli-Opitz syndrome (SLOS) results in multiple malformations, growth
deficiency, and mental retardation. Cholesterol supplementation has been used for several
years to treat symptoms of SLOS. We assessed the developmental progress of children and
adolescents with SLOS over a 6-year period. STUDY DESIGN: Patients with SLOS (n= 14)
received continuous cholesterol supplementation as part of a longitudinal study.
Assessment of their developmental progress in the areas of cognitive, motor, and adaptive …
deficiency, and mental retardation. Cholesterol supplementation has been used for several
years to treat symptoms of SLOS. We assessed the developmental progress of children and
adolescents with SLOS over a 6-year period. STUDY DESIGN: Patients with SLOS (n= 14)
received continuous cholesterol supplementation as part of a longitudinal study.
Assessment of their developmental progress in the areas of cognitive, motor, and adaptive …
OBJECTIVE
Smith-Lemli-Opitz syndrome (SLOS) results in multiple malformations, growth deficiency, and mental retardation. Cholesterol supplementation has been used for several years to treat symptoms of SLOS. We assessed the developmental progress of children and adolescents with SLOS over a 6-year period.
STUDY DESIGN
Patients with SLOS (n=14) received continuous cholesterol supplementation as part of a longitudinal study. Assessment of their developmental progress in the areas of cognitive, motor, and adaptive skills occurred every 6 to 12 months. The progress of each subject over time and the progress of the group as a whole were analyzed by using a repeated-measures design and multiple t tests.
RESULTS
Developmental quotients did not improve over time for children with SLOS receiving cholesterol. In addition, baseline cholesterol levels, rather than age when supplementation began or increase in cholesterol levels, best predicted developmental outcome.
CONCLUSIONS
These results suggest that cholesterol supplementation in its current form does not improve the developmental progress of children and adolescents with SLOS.
Elsevier