Feasibility, Acceptability, and Preliminary Efficacy of an Intensive Clinic-Based Intervention for Children With Poorly Controlled Type 1 Diabetes.

OBJECTIVE: To evaluate the feasibility, acceptability, and preliminary efficacy of a team-based intervention for youth with type 1 diabetes (T1D) with suboptimal glycemia, as detected based on the measurement of hemoglobin A1C (HbA1C). METHODS: Forty participants with T1D for >1 year and an HbA1C level of ≥9.5% (80 mmol/mol) enrolled for a multidisciplinary intervention that included pediatric endocrinologists, pediatric psychologists, and a certified diabetes care and education specialist (CDCES). The CDCES-integrated medical management, while reinforcing physical, emotional, and behavioral health, connected with families to set and monitor goals and reviewed medication adjustments. The feasibility was assessed based on enrollment targets; acceptability based on retention rates; and preliminary efficacy based on changes in HbA1C levels, quality of life, diabetes-related strengths and resilience, hospital admissions, emergency room visits, and missed school days. RESULTS: Of 43 patients and families approached, 40 agreed to participate, 36 completed the 4-month intervention, and 31 completed full 8 months of follow-up data collection. The CDCES coach averaged 6.8 contacts per participant during the 8-month study period. The HbA1C level reduced significantly from baseline to 4 months (12.1% ± 1.6% to 11.0% ± 1.9%, P = .001) and was sustained at 8 months (10.7% ± 1.9%, P < .001). The participants reported significant increases in diabetes-specific quality of life (P < .05) and diabetes-related strength and resilience (P = .003). The missed school days reduced from 7.23 ± 7.5 days to 1.55 ± 1.9 days (P < .001), and the diabetes-related hospitalizations decreased from 0.4 ± 0.6 to 0.1 ± 0.3 (P = .009). CONCLUSION: Preliminary data suggest that a multidisciplinary intervention leveraging a team-based approach with a physician, psychologist, and CDCES can support improvements in glycemic control and psychosocial outcomes among youth with T1D with an HbA1C level above the target.

Endocrine and pubertal disturbances in optic nerve hypoplasia, from infancy to adolescence.

BACKGROUND: Endocrinologic abnormalities are a common co-morbidity in patients with optic nerve hypoplasia (ONH), however the impact on puberty is unknown. The purpose of this study was to examine rates of endocrine dysfunction and pubertal disturbances in a pediatric population of ONH. METHODS: A retrospective chart review was conducted on a cohort of children with ONH between January 2005 and March 2013. Endocrine dysfunction was determined based on laboratory evidence of hormone deficiency or hormone replacement. Pubertal disturbances were characterized based on presence of micropenis, tanner staging, menarche and hormone replacement. Pituitary abnormalities were classified using MRI findings. Descriptive statistics were used, and comparisons between groups were performed using the chi-square test. RESULTS: During the study period, 101 patients underwent an endocrine evaluation (median age: 2.3 years [0.76 - 6.5]). Hypopituitarism was present in 73% of patients with growth hormone deficiency (56%) and hypothyroidism (54%) being the most common. Pubertal disturbances (n = 19) were common; micropenis in 31% (13/42) of males and 2% with precocious puberty. Half of adolescents (n = 4/8) were diagnosed with gonadotropin deficiency. Patients with MRI pituitary abnormalities were more likely to have endocrine dysfunction than those without (p = 0.004). The sensitivity and specificity of MRI pituitary abnormalities for hypopituitarism was 54% and 92%, respectively. CONCLUSIONS: A significant proportion of children with ONH have endocrine dysfunction. The high frequency of pubertal disturbances in this study emphasizes the need for long-term monitoring of developing endocrinopathy. While pituitary gland abnormalities are a good predictor of endocrine dysfunction, a normal pituitary gland does not rule out endocrinopathy.

Soluble receptor for advanced glycation end products independently predicts cardiometabolic syndrome in Latino youth.

OBJECTIVE: The soluble receptor for advanced glycation end products (sRAGE) has anti-atherogenic properties in adults, however, little is known about the relationship between sRAGE and cardiometabolic disease risk in pediatric populations. Therefore, the purpose of this investigation is to examine the relationship between sRAGE and cardiometabolic risk factors in Latino youth. RESEARCH DESIGN AND METHODS: Data from 133 Latino youth (58% female; age 16.3 ± 2.8 yr) enrolled in the Arizona Insulin Resistance Registry were analyzed. Cardiometabolic syndrome was determined using a continuous risk score normalized to the population. Components included waist circumference, high-density lipoprotein cholesterol (HDL-c), triglycerides, mean arterial pressure (MAP), and homeostatic assessment of insulin resistance (HOMA-IR). sRAGE levels were determined from fasting serum using an enzyme-linked immunosorbent assay. RESULTS: In univariate analysis, sRAGE was inversely associated with waist circumference (r = -0.22, p = 0.01), MAP (r = -0.15, p = 0.09), and HOMA-IR (r = -0.29, p < 0.01) and positively associated with HDL-c (r = 0.19, p < 0.05). In multiple regression analysis with age, sex, and BMI, sRAGE remained an independent predictor of cardiometabolic syndrome risk score (R(2) = 0.55, p < 0.001). CONCLUSIONS: These data suggest that sRAGE may be an early independent biomarker of cardiometabolic disease risk in youth. Prospective studies are needed to establish the predictive utility of sRAGE for long-term disease outcomes.

Tumor size and extent of disease at diagnosis predict the response to initial therapy for papillary thyroid carcinoma in children and adolescents.

Treatment of papillary thyroid carcinoma (PTC) in children and adolescents is controversial. We previously showed that large tumor size, multifocal disease, and extensive disease at diagnosis predict recurrence. We examined 47 patients with PTC to determine whether these features predict response to treatment. Overall, 70% of the patients (33/47) remitted with initial treatment. 79% (15/19), of Class I, 86% (12/14) of Class II, and 100% (6/6) of Class III, but none of Class IV patients (n = 8) (p < 0.001) achieved remission. Tumor size for patients who entered remission (2.0 +/- 0.2 cm) was less than for patients with persistent disease (4.2 +/- 0.4) (p < 0.0005). Extent of disease at diagnosis correlated with the number of radioactive iodine (RAI) treatments (p = 0.022) and dose (p = 0.002) required to achieve first remission. We conclude that extensive disease at diagnosis and larger tumor size predict failure to remit after initial treatment of PTC in children and adolescents.