The hypothesis that insufficient body weight is partly responsible for idiopathic short stature was tested by evaluating 79 prepubertal children having idiopathic short stature, classified according to their body mass index (BMI) zs: group 1 BMI zs ≤0 (m ± SE, -0.9 ± 0.1, n = 53) and group 2 BMI zs > 0 (0.6 ± 0.1, n = 26). Their ages were similar (8.1 ± 0.3 and 8.4 ± 0.4 years). The following parameters were significantly lower in group 1 than in group 2: height (-2.5 ± 0.1 vs.-2.2 ± 0.1 SDp < 0.05), height velocity (-1.5 ± 0.2 vs. -0.9 ± 0.4 SD, p < 0.05), bone age (bone age retardation 2.3 ± 0.2 vs. 1.3 ± 0.4 years, p < 0.005) and plasma insulin-like growth factor 1 (IGF-1, 0.8 ± 0.1 vs. 1.2 ± 0.1 U/ml, p < 0.05). The BMI was significantly lower in children with idiopathic short stature (-0.4 ± 0.1) than in the normal population (zs, p < 0.0002). BMI zs was positively correlated with height (p < 0.005), height velocity (p < 0.05) and plasma IGF-1 (p < 0.01). We conclude that children with idiopathic short stature are leaner than the normal population. An inadequate or insufficient nutritional intake may be partly responsible for idiopathic short stature. The effects of improved intake on the height change are under evaluation.

Summary: A recent United Kingdom cost minimization analysis (CMA) of four antiepileptic drugs (AEDs) used to treat newly diagnosed adult epilepsy demonstrated that a new drug, lamotrigine (LTG), incurred higher costs than carbamazepine (CBZ), phenytoin (PHT), and valproate (VPA), whose costs were similar. This analysis took account of each drug's sideeffect and tolerability profile. The present analysis investigated the costs of treatment with LTG, CBZ, PHT, and VPA in 12 European countries. Data were derived from published sources and from a panel of locally based experts. When no published data were available, estimates were obtained using expert opinion by a consensus method. These data were incorporated into a treatment pathway model, which considered the treatment of patients during the first 12 months after diagnosis. The primary outcome considered was seizure freedom, Randomized controlled trials demonstrate that the drugs considered are equally effective in terms of their ability to achieve seizure freedom, and thus the most appropriate form of economic evaluation is a CMA. These trials provided data on the incidence of side effects, dosages, and retention rates. The economic perspective taken was that of society as a whole and the analysis was calculated on an "intent-to-treat'' basis. Only direct medical costs were considered. In each country considered, LTG was twofold to threefold more expensive than the other drugs considered. A sensitivity analysis demonstrated that varying each of the assumptions (range defined by expert panels) did not significantly alter the results obtained.

Utility of ruxolitinib in a child with chronic mucocutaneous candidiasis caused by a novel STAT1 gain-of-function mutation.

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96.5% (Nϭ193), with Nϭ180 indicating continuous use. Nϭ141 patients (71%) had a hospitalization with average 3.5 hospitalizations per patient (medianϭ2); an average patient spent 48 days in hospital during the study period. On average, for those with a procedure, 1.8 (SDϭ1.4) surgical (Nϭ55) or 7.9 (SDϭ5.8) diagnostic (Nϭ200) procedures were recorded. No differences were observed between G551D and DF508 genotypes in terms of severity of disease as gauged by health care use. CONCLUSIONS: This study, linking both clinical and resources use information indicates that high frequency and intensity of health care resource use contributes substantially to the overall burden of CF in UK. G551D and ⌬F508 genotypes appear to have similar patterns of resource utilization.