Am J Med Genet A. 2008 Feb 6. [Epub ahead of print]
Developmental profiles in young children with Prader-Labhart-Willi syndrome: Effects of weight and therapy with growth hormone or coenzyme Q(10).
Eiholzer U, Meinhardt U, Rousson V, Petrovic N, Schlumpf M, L'allemand D.
Center for Pediatric Endocrinology Zurich (PEZZ), Zurich, Switzerland.

Muscle hypotonia and failure to thrive are key symptoms of Prader-Willi syndrome (PWS) allowing diagnosis during infancy already. Improved general care as well as Coenzyme Q(10) (CoQ(10)) and growth hormone (GH) are administered to improve PWS children's outcome. This study aims to investigate psychomotor development of young PWS children in relation to body weight and body composition at baseline as well as to the effects of GH or CoQ(10) therapy. Twenty-six young children (age 1.0 +/- 0.1 years, mean +/- SEM) with PWS genetically proven at age 0.1 +/- 0.1 years (17 deletions, 8 maternal disomy) were divided into three groups: Group 1 on GH therapy (started in 1994-1996, 6 mg/kg/week) tolerating low body weight (<50th centile), group 2 on GH (1997-2000) and group 3 on CoQ(10) (2001-2002, 2.5 mg/kg/day orally), both combined with active early weight management to achieve weight >50th centile. Anthropometry, body composition and Griffith's developmental scores (DQs) were assessed before therapy and after 12 months. DQs were not related to infants' weight, lean mass or genetic background. DQs improved significantly with chronological age and were best in the most recently diagnosed group. Improved psychomotor development, mainly due to progress in locomotor development, did not differ between GH and CoQ(10) treated groups. In conclusion, while only GH has significant effects on growth and body composition, GH and CoQ(10) therapy act equally on psychomotor development of PWS infants. However, improving psychomotor development may merely reflect an age-related phenomenon additionally depending on early diagnosis and introduction of appropriate care.

[Note the relatively small dose of CoQ10, especially for very young children, compared to what most parents report is required for a significant effect. For example, the dose used in this study would only be 25 mg/day for a 10 kg (22 lb) infant. Despite that, the study still found no difference between the GH and CoQ10 groups in terms of improved psychomotor development.]

Graphs from this article:

Figure 1

Twelve-months period of height (a) and weight (b) in young children with PWS with growth hormone (GH), group 1 (n=6, broken line with black rhombus) and group 2 (n=12, dotted line with open triangle) or coenzyme Q10 treatment group (group 3, n=8, full line with black square), means +- SEM.

Figure 2

Developmental Quotients (DQ) in young children with PWS during 12 months of therapy with GH (group 1 = broken line with black rhombus, group 2 = dotted line with open triangle) or CoQ₁₀ (full line with black square), means +- SEM.

Categories: 2008, PWS, CoQ10, Failure to thrive, Growth, Motor development, Developmental delays, Body composition, Growth hormone treatment