1,305 research outputs found
Complex Latent Variable Modeling in Educational Assessment
Bayesian item response theory models have been widely used in different research fields. They support measuring constructs and modeling relationships between constructs, while accounting for complex test situations (e.g., complex sampling designs, missing data, heterogenous population). Advantages of this flexible modeling framework together with powerful simulation-based estimation techniques are discussed. Furthermore, it is shown how the Bayes factor can be used to test relevant hypotheses in assessment using the College Basic Academic Subjects Examination (CBASE) data
Effect of discontinuation of growth hormone treatment on risk factors for cardiovascular disease in adolescents born small for gestational age
Hyperlipidemia, diabetes mellitus type 2, and coronary heart disease have
been associated with being born small for gestational age (SGA). It has
been reported that GH treatment induced higher insulin levels, which has
led to concern regarding the long-term effect of GH treatment in
predisposed individuals such as children born SGA. In this study, we
assessed the effect of discontinuation of long-term GH treatment in 47
adolescents born SGA on oral glucose tolerance tests, blood pressure (BP),
and serum lipid levels for two GH dosage groups (3 vs. 6 IU/m2 x d). At 6
months after discontinuation of GH treatment mean (SD) age was 16.0 (2.1)
yr. Mean duration of GH treatment had been 6.9 (1.5) yr. Fasting glucose
levels and 120-min area under the curve for glucose 6 months after
discontinuation of GH treatment showed no difference from pretreatment
levels for both GH dosage groups. After discontinuation of GH treatment,
fasting insulin levels returned to pretreatment levels (8.4 mU/liter),
whereas the 120-min area under the curve for insulin decreased, compared
with 6-yr levels (P < 0.01), regardless of GH dosage group. No significant
difference was found when levels were compared with a control group. In
addition, for both GH dosage groups, no significant changes in systolic
and diastolic BP SD score, total cholesterol, and atherogenic index (total
cholesterol/high-density lipoprotein cholesterol) were seen from 6 yr of
GH until 6 months after discontinuation of GH treatment. In conclusion, in
children born SGA, the GH-induced insulin insensitivity disappeared after
discontinuation of GH, even after long-term GH treatment. Furthermore, the
beneficial effect of GH on BP was not changed after discontinuation of GH,
and most children had normal lipid levels
Adult height after long-term, continuous growth hormone (GH) treatment in short children born small for gestational age: results of a randomized, double-blind, dose-response GH trial
The GH dose-response effect of long-term continuous GH treatment on adult
height (AH) was evaluated in 54 short children born small for gestational
age (SGA) who were participating in a randomized, double-blind,
dose-response trial. Patients were randomly and blindly assigned to
treatment with either 3 IU (group A) or 6 IU (group B) GH/m(2).d (
approximately 0.033 or 0.067 mg/kg.d, respectively). The mean (+/-SD)
birth length was -3.6 (1.4), the age at the start of the study was 8.1
(1.9) yr, and the height SD score (SDS) at the start of the study -3.0
(0.7). Seventeen of the 54 children were partially GH deficient
(stimulated GH peak, 10-20 mU/liter). Fifteen non-GH-treated,
non-GH-deficient, short children born SGA, with similar inclusion
criteria, served as controls [mean (+/-SD) birth length, -3.3 (1.2); age
at start, 7.8 (1.7) yr; height SDS at start, -2.6 (0.5)]. GH treatment
resulted in an AH above -2 SDS in 85% of the children after a mean (+/-SD)
GH treatment period of 7.8 (1.7) yr. The mean (SD) AH SDS was -1.1 (0.7)
for group A and -0.9 (0.8) for group B, resulting from a mean (+/-SD) gain
in height SDS of 1.8 (0.7) for group A and 2.1 (0.8) for group B. No
significant differences between groups A and B were found for AH SDS (mean
difference, 0.3 SDS; 95% confidence interval, -0.2, 0.6; P > 0.2) and gain
in height SDS (mean difference, 0.3 SDS; 95% confidence interval, -0.1,
0.7; P > 0.1). When corrected for target height, the mean corrected AH SDS
was -0.2 (0.8) for group A and -0.4 (0.9) for group B. The mean (+/-SD) AH
SDS of the control group [-2.3 (0.7)] was significantly lower than that of
the GH-treated group (P < 0.001). Multiple regression analysis indicated
the following predictive variables for AH SDS: target height SDS, height
SDS, and chronological age minus bone age (years) at the start of the
study. GH dose had no significant effect. In conclusion, long-term
continuous GH treatment in short children born SGA without signs of
persistent catch-up growth leads to a normalization of AH, even with a GH
dose of 3 IU/m(2).d ( approximately 0.033 mg/kg.d)
Bioenergetic Impairment in Congenital Muscular Dystrophy Type 1A and Leigh Syndrome Muscle Cells
Skeletal muscle has high energy requirement and alterations in metabolism are associated with pathological conditions causing muscle wasting and impaired regeneration. Congenital muscular dystrophy type 1A (MDC1A) is a severe muscle disorder caused by mutations in the LAMA2 gene. Leigh syndrome (LS) is a neurometabolic disease caused by mutations in genes related to mitochondrial function. Skeletal muscle is severely affected in both diseases and a common feature is muscle weakness that leads to hypotonia and respiratory problems. Here, we have investigated the bioenergetic profile in myogenic cells from MDC1A and LS patients. We found dysregulated expression of genes related to energy production, apoptosis and proteasome in myoblasts and myotubes. Moreover, impaired mitochondrial function and a compensatory upregulation of glycolysis were observed when monitored in real-time. Also, alterations in cell cycle populations in myoblasts and enhanced caspase-3 activity in myotubes were observed. Thus, we have for the first time demonstrated an impairment of the bioenergetic status in human MDC1A and LS muscle cells, which could contribute to cell cycle disturbance and increased apoptosis. Our findings suggest that skeletal muscle metabolism might be a promising pharmacological target in order to improve muscle function, energy efficiency and tissue maintenance of MDC1A and LS patients
Growth hormone treatment in children with short stature born small for gestational age: 5-year results of a randomized, double-blind, dose-response trial
The growth-promoting effect of continuous GH treatment was evaluated over
5 yr in 79 children with short stature (height SD score, less than -1.88)
born small for gestational age (SGA; birth length SD score, less than
-1.88). Patients were randomly and blindly assigned to 1 of 2 GH dosage
groups (3 vs. 6 IU/m2 body surface-day). GH deficiency was not an
exclusion criterium. After 5 yr of GH treatment almost every child had
reached a height well within the normal range for healthy Dutch children
and in the range of their target height SD score. Only in children who
remained prepubertal during the study period was the 5-yr increase in
height SD score (HSDS) for chronological age significantly higher in the
study group receiving 6 compared to 3 IU GH/m2 x day. Remarkably, the 5-yr
increment in HSDS for chronological age was not related to spontaneous GH
secretion, maximum GH levels after provocation, or baseline insulin-like
growth factor I levels. GH treatment was associated with an acceleration
of bone maturation regardless of the GH dose given. The HSDS for bone age
and predicted adult height increased significantly. GH treatment was well
tolerated. In conclusion, our 5-yr data show that long term continuous GH
treatment at a dose of 3 or 6 IU/m2 x day in short children born SGA
results in a normalization of height during childhood followed by growth
along the target height percentile
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