PIN71 QUALITY OF LIFE (QOL) AND OTHER ENDPOINTS COMPARISON IN THE TREATMENT OF FACIAL LIPOATROPHY WITH INJECTION OF POLY-L-LACTIC ACID

Context: Longitudinal data on bone mineral density(BMD) in children and adolescents with Prader-Willi Syndrome (PWS) during long-term GH treatment are not available. Objective: This study aimed to determine effects of long-term GH treatment and puberty on BMD of total body (BMDTB), lumbar spine (BMDLS), and bone mineral apparent density of the lumbar spine (BMAD(LS)) in children with PWS. Design and Setting: This was a prospective longitudinal study of a Dutch PWS cohort. Participants: Seventy-seven children with PWS who remained prepubertal during GH treatment for 4 years and 64 children with PWS who received GH treatment for 9 years participated in the study. Intervention: The children received GH treatment, 1 mg/m(2)/day (congruent to 0.035 mg/kg/d). Main Outcome Measures: BMDTB, BMDLS, and BMAD(LS) was measured by using the same dual-energy x-ray absorptiometry machine for all annual measurements. Results: In the prepubertal group, BMDTB standard deviation score (SDS) and BMDLSSDS significantly increased during 4 years of GH treatment whereas BMAD(LS)SDS remained stable. During adolescence, BMDTBSDS and BMAD(LS)SDS decreased significantly, in girls from the age of 11 years and in boys from the ages of 14 and 16 years, respectively, but all BMD parameters remained within the normal range. Higher Tanner stages tended to be associated with lower BMDTBSDS (P = .083) and a significantly lowerBMAD(LS)SDS (P = .016). After 9 years of GH treatment, lean body mass SDS was the most powerful predictor of BMDTBSDS and BMDLSSDS in adolescents with PWS. Conclusions: This long-term GH study demonstrates that BMDTB, BMDLS, and BMAD(LS) remain stable in prepubertal children with PWS but decreases during adolescence, parallel to incomplete pubertal development. Based on our findings, clinicians should start sex hormone therapy from the age of 11 years in girls and 14 years in boys unless there is a normal progression of puberty

Social Anxiety Modulates Subliminal Affective Priming

BACKGROUND: It is well established that there is anxiety-related variation between observers in the very earliest, pre-attentive stage of visual processing of images such as emotionally expressive faces, often leading to enhanced attention to threat in a variety of disorders and traits. Whether there is also variation in early-stage affective (i.e. valenced) responses resulting from such images, however, is not yet known. The present study used the subliminal affective priming paradigm to investigate whether people varying in trait social anxiety also differ in their affective responses to very briefly presented, emotionally expressive face images. METHODOLOGY/PRINCIPAL FINDINGS: Participants (n = 67) completed a subliminal affective priming task, in which briefly presented and smiling, neutral and angry faces were shown for 10 ms durations (below objective and subjective thresholds for visual discrimination), and immediately followed by a randomly selected Chinese character mask (2000 ms). Ratings of participants' liking for each Chinese character indicated the degree of valenced affective response made to the unseen emotive images. Participants' ratings of their liking for the Chinese characters were significantly influenced by the type of face image preceding them, with smiling faces generating more positive ratings than neutral and angry ones (F(2,128) = 3.107, p<0.05). Self-reported social anxiety was positively correlated with ratings of smiling relative to neutral-face primed characters (Pearson's r = .323, p<0.01). Individual variation in self-reported mood awareness was not associated with ratings. CONCLUSIONS: Trait social anxiety is associated with individual variation in affective responding, even in response to the earliest, pre-attentive stage of visual image processing. However, the fact that these priming effects are limited to smiling and not angry (i.e. threatening) images leads us to propose that the pre-attentive processes involved in generating the subliminal affective priming effect may be different from those that generate attentional biases in anxious individuals

The impact of inflammation on bone mass in children

Bone is a dynamic tissue. Skeletal bone integrity is maintained through bone modeling and remodeling. The mechanisms underlying this bone mass regulation are complex and interrelated. An imbalance in the regulation of bone remodeling through bone resorption and bone formation results in bone loss. Chronic inflammation influences bone mass regulation. Inflammation-related bone disorders share many common mechanisms of bone loss. These mechanisms are ultimately mediated through the uncoupling of bone remodeling. Cachexia, physical inactivity, pro-inflammatory cytokines, as well as iatrogenic factors related to effects of immunosuppression are some of the common mechanisms. Recently, cytokine signaling through the central nervous system has been investigated for its potential role in bone mass dysregulation in inflammatory conditions. Growing research on the molecular mechanisms involved in inflammation-induced bone loss may lead to more selective therapeutic targeting of these pathological signaling pathways

No association between glucocorticoid receptor polymorphisms and long-term respiratory outcome after very preterm birth

Developmen

Endocrine and Growth Abnormalities in 4H Leukodystrophy Caused by Variants in POLR3A, POLR3B, and POLR1C.

CONTEXT: 4H or POLR3-related leukodystrophy is an autosomal recessive disorder typically characterized by hypomyelination, hypodontia, and hypogonadotropic hypogonadism, caused by biallelic pathogenic variants in POLR3A, POLR3B, POLR1C, and POLR3K. The endocrine and growth abnormalities associated with this disorder have not been thoroughly investigated to date. OBJECTIVE: To systematically characterize endocrine abnormalities of patients with 4H leukodystrophy. DESIGN: An international cross-sectional study was performed on 150 patients with genetically confirmed 4H leukodystrophy between 2015 and 2016. Endocrine and growth abnormalities were evaluated, and neurological and other non-neurological features were reviewed. Potential genotype/phenotype associations were also investigated. SETTING: This was a multicenter retrospective study using information collected from 3 predominant centers. PATIENTS: A total of 150 patients with 4H leukodystrophy and pathogenic variants in POLR3A, POLR3B, or POLR1C were included. MAIN OUTCOME MEASURES: Variables used to evaluate endocrine and growth abnormalities included pubertal history, hormone levels (estradiol, testosterone, stimulated LH and FSH, stimulated GH, IGF-I, prolactin, ACTH, cortisol, TSH, and T4), and height and head circumference charts. RESULTS: The most common endocrine abnormalities were delayed puberty (57/74; 77% overall, 64% in males, 89% in females) and short stature (57/93; 61%), when evaluated according to physician assessment. Abnormal thyroid function was reported in 22% (13/59) of patients. CONCLUSIONS: Our results confirm pubertal abnormalities and short stature are the most common endocrine features seen in 4H leukodystrophy. However, we noted that endocrine abnormalities are typically underinvestigated in this patient population. A prospective study is required to formulate evidence-based recommendations for management of the endocrine manifestations of this disorder

If you were a policymaker, which treatment would you disinvest? A participatory value evaluation on public preferences for active disinvestment of health care interventions in the Netherlands.

IntroductionCurrently, it is not known what attributes of health care interventions citizens consider important in disinvestment decision-making (i.e. decisions to discontinue reimbursement). Therefore, this study aims to investigate the preferences of citizens of the Netherlands toward the relative importance of attributes of health care interventions in the context of disinvestment.MethodsA participatory value evaluation (PVE) was conducted in April and May 2020. In this PVE, 1143 Dutch citizens were asked to save at least €100 million by selecting health care interventions for disinvestment from a list of eight unlabeled health care interventions, described solely with attributes. A portfolio choice model was used to analyze participants' choices.ResultsParticipants preferred to disinvest health care interventions resulting in smaller gains in quality of life and life expectancy that are provided to older patient groups. Portfolios (i.e. combinations of health care interventions) resulting in smaller savings were preferred for disinvestment over portfolios with larger savings.ConclusionThe disinvestment of health care interventions resulting in smaller health gains and that are targeted at older patient groups is likely to receive most public support. By incorporating this information in the selection of candidate interventions for disinvestment and the communication on disinvestment decisions, policymakers may increase public support for disinvestment.Transport and Logistic