Novel Treatment Options in Childhood Bone Diseases.

BACKGROUND: Several novel treatment options have recently become available in childhood bone diseases. The purpose of this article is to provide an update on some of the therapeutic agents used in the treatment of pediatric osteoporosis, X-linked hypophosphatemic rickets, and achondroplasia (ACH). SUMMARY: Vitamin D3 and Ca supplementation remains the basis of childhood osteoporosis treatment. Bisphosphonate (BP) therapy is the main antiresorptive therapeutic option, while denosumab, a human monoclonal IgG2 antibody with high affinity and specificity for a primary regulator of bone resorption - RANKL, represents a possible alternative. Its potent inhibition of bone resorption and turnover process leads to continuous increase of bone mineral density throughout the treatment also in the pediatric population. With a half-life much shorter than BPs, its effects are rapidly reversible upon discontinuation. Safety and dosing concerns in children remain. Novel treatment options have recently become available in two rare bone diseases. Burosumab, a monoclonal antibody against FGF-23, has been approved for the treatment of children with X-linked hypophosphatemic rickets older than 1 year. It presents an effective, more etiology-based treatment for rickets compared to conventional therapy, without the need for multiple daily oral phosphate supplementation. Its long-term efficacy and safety are currently being investigated. After years of anticipation, a novel treatment option for ACH has become available. C-type natriuretic peptide analog vosoritide effectively increases proportional growth and has a reasonable safety profile in children >2 years. Its effect on other features of the disease and the final height is yet to be determined. Several other treatment options for ACH exploring different therapeutic approaches are currently being investigated. KEY MESSAGES: Denosumab is effective in the treatment of childhood-onset osteoporosis; however, further studies are necessary to determine the optimal treatment protocol. Burosumab is more etiology-based and convenient in comparison to conventional treatment of X-linked hypophospha--temic rickets in children and adults. Vosoritide importantly changes the natural course of achondroplasia, at least in the short term.

Advances in the management of craniopharyngioma in children and adults.

Background Childhood and adult-onset craniopharyngioma is a rare embryogenic tumor of the sellar, suprasellar, and parasellar region. Survival rates are high; however, tumor location and treatment sequalae including endocrine deficits, visual impairment, metabolic complications, cognitive and psychosocial deficits can significantly impair patient's quality of life. There is considerable controversy regarding the optimal management of craniopharyngiomas. Subtotal resection of the tumor followed by targeted irradiation to avoid further hypothalamic damage is currently indicated. Novel insights in the tumor's molecular pathology present the possibility for targeted therapy possibly decreasing the rate and severity of treatment-associated morbidity. Conclusions Craniopharyngioma should be seen as a chronic disease. To achieve optimal outcomes a multidisciplinary team of specialized neurosurgeons, neuro-radiologists, neuro-oncologists, pathologists and endocrinologists should be involved in the diagnosis, planning of the surgery, irradiation and long-term follow-up.

Adequate iodine intake of Slovenian adolescents is primarily attributed to excessive salt intake.

In Slovenia, table salt iodization has been applied to combat iodine deficiency. Recently, we found that Slovenian adolescents attained iodine sufficiency (median urinary iodine concentration was 140 microg/L; prevalence of goiter was <1%). National data indicate that salt intake of Slovenian population is too high (150% above the recommended limit); therefore, we hypothesized that sufficient iodine intake in adolescents can be primarily attributed to excessive salt intake. In a cross-sectional study, we investigated iodine and salt intake in Slovenian adolescents as well as the contributions of different foods to their intake. We determined the iodine and salt intake of a national representative sample of 2581 adolescents, aged 14 to 17 years, using the Food Frequency Questionnaire (FFQ). The FFQ covered habitual diets over the past year, and 2485 (96%) adolescents completed a valid FFQ (1370 girls, 1115 boys). The iodine intake was 189.7 +/- 2.6 microg/d (mean +/- standard error of mean), well above the recommended 150 microg/d (P < .001). Table salt was by far the biggest dietary source of iodine and sodium for both sexes. Total salt intake (mean +/- standard error of mean, 10.4 +/- 0.2 g/d) significantly exceeded the upper World Health Organization limit (<5 g/d, P < .001), especially in boys (11.5 +/- 0.3 vs 9.4 +/- 0.2 g/d in girls, P < .001). The main food sources of salt were table salt (33%), bread (24%), salty snack products (10%), meat products (8%), fish products (6%), and milk (4%). Salt intake from foods, excluding table salt, was 6.9 g/d (67% of total salt intake). We conclude that although Slovenian adolescents are iodine sufficient, their salt intake, especially among boys, is too high. Several nutritional interventions are proposed to reduce total salt intake while ensuring adequate iodine intake.

Iodine intake of Slovenian adolescents.

BACKGROUND: Slovenia is classified as being iodine-deficient. We recently found that Slovenian adolescents are iodine-sufficient (median urinary iodine concentration of the population 140 microg/l) and the prevalence of goiter is low (0.9%). The objective of this study was to evaluate iodine intake, the prevalence of marginal, low and excessive intake (<50, 50-100 and >or=300 microg/day), as well as the main sources of iodine in the diet of Slovenian adolescents. METHODS: A cross-sectional study included 2,581 adolescents (1,415 girls, 1,166 boys, mean age +/- SD 15.6 +/- 0.5 years) representing 10% of 15-year-old Slovenian adolescents. Iodine intake was determined using a food frequency questionnaire (FFQ) in the whole population studied (n = 2,485) and weighted 3-day dietary protocols (3DPs) in a subgroup of participants (n = 191). RESULTS: Median iodine intake determined from FFQ was 155.8 microg/day. There was no significant difference between genders. Marginal, low and excessive iodine intake was observed in 3.3, 20.3 and 11.3% of the adolescents, respectively. The major food sources of dietary iodine included table salt (39 % of the mean daily iodine intake), beverages (22%) and milk/milk products (19%). CONCLUSIONS: Dietary iodine intake in Slovenian adolescents is adequate, illustrating the effective salt iodization program.