Optic pathway glioma and endocrine disorders in patients with and without NF1.

BACKGROUND: Optic pathway gliomas (OPGs) are classified by anatomic location and the association with neurofibromatosis type 1 (NF1). Children with OPGs face sequelae related to tumor location and treatment modalities. We assessed the prevalence of endocrine dysfunction in children with OPGs and compared outcomes between those with and without NF1. METHODS: We performed a retrospective medical record review of medical history, and clinical and laboratory data, of children diagnosed with OPGs (n = 59, 61% with NF1) during 1990-2020, followed at a tertiary endocrine clinic. Growth and puberty parameters and occurrence of endocrine dysfunction were evaluated. RESULTS: Isolated optic nerve involvement was higher among patients with than without NF1. Patients without NF1 were younger at OPG diagnosis and more often treated with debulking surgery or chemotherapy. At the last endocrine evaluation, patients without NF1 had comparable height SDS, higher BMI SDS, and a higher rate of endocrine complications (78.3% vs. 41.7%, p = 0.006). Younger age at diagnosis, older age at last evaluation, and certain OPG locations were associated with increased endocrine disorder incidence. CONCLUSIONS: Endocrine dysfunction was more common in patients without NF1; this may be related to younger age at presentation, tumor locations, a greater progressive rate, and more aggressive treatments. IMPACT: The literature is sparse regarding sporadic OPGs, and the mean duration of follow-up is shorter than at our study. Our data show a higher rate of endocrine dysfunction in patients with OPGs than previously described. We also found a higher prevalence of endocrine dysfunctions among patients without compared to those with NF-1. A better understanding of the true prevalence of endocrine disabilities that may evolve along time can help in guiding physicians in the surveillance needed in patients with OPG.

Which predictors differentiate between obese children and adolescents with cardiometabolic complications and those with metabolically healthy obesity?

BACKGROUND/AIMS: Childhood obesity and associated metabolic comorbidities is a major global health concern. Metabolically healthy obesity (MHO) may represent a subgroup of individuals in which excessive body fat accumulation does not lead to adverse metabolic effects. We aimed to determine the prevalence of MHO among obese Israeli children and adolescents and to find predictors for metabolically unhealthy obesity (MUO). METHODS: In a retrospective study, demographic, anthropometric, lifestyle, and cardiometabolic data were retrieved from medical records of patients with a body mass index (BMI) >95th percentile aged 6 to 17.6 years, attending a tertiary pediatric obesity clinic between 2008 and 2015, with at least 1 year of follow-up. Participants were dichotomized as either MHO or MUO based on cardiometabolic risk factor clustering (blood pressure, serum lipids, and glucose). Multivariable logistic regression was used to determine predictors of MUO. RESULTS: Of the 230 children (median age 9.9 years) fulfilling study criteria, 48 (20.9%) were classified as MHO. Occurrence of MUO was associated with male gender, Arabic ethnicity, higher BMI-SD score, higher tri-ponderal mass index (TMI), and higher insulin resistance (IR) (presence of acanthosis nigricans and a higher level of homeostasis model assessment-IR [HOMA-IR]). Male gender (odds ratio [OR] 2.27, P = .033), presence of acanthosis nigricans at baseline (OR 2.35, P = .035), and a greater increase in BMI-SDS during follow-up (OR 2.82, P = .05) were the best predictors of MUO. CONCLUSIONS: The MHO phenotype was present in only 20.9% of obese Israeli children. MUO was significantly associated with male gender, with presence of acanthosis nigricans, and with a greater increase in BMI-SDS during follow-up.

Growth and pubertal patterns in young survivors of childhood acute lymphoblastic leukemia.

BACKGROUND: Survivors of acute lymphoblastic leukemia (ALL) may experience endocrine dysfunction. This study evaluated growth and pubertal patterns in survivors of childhood ALL. METHODS: Longitudinal assessment of anthropometric measurements and pubertal status was performed in a retrospective cohort of survivors (n=183). Median age at last endocrine visit was 16.1 years (range 8.2-27.6); median follow-up time was 8.7 years (range 3-21.4). RESULTS: Treatment with chemotherapy+prophylactic cranial radiation (pCRT, n=29) was associated with lower mean height standard deviation score (SDS) than chemotherapy alone (n=154) (p=0.001) and higher prevalence of adult short stature (13% vs. 2.2%). Mean age at pubertal onset was normal (girls: 10.3±1.3 years; boys: 12.0±1.3 years). Precocious puberty, diagnosed in 8.7% of patients, was more prevalent in pCRT-treated girls. Rates of overweight and obesity were 22.9% and 9.3%, respectively. Predictors of endocrine disorders were pCRT (p=0.031) and female gender (p=0.041); of obesity, higher body mass index (BMI)-SDS at diagnosis (p=0.001); and of short stature, lower height-SDS at diagnosis (p=0.038). CONCLUSIONS: Most childhood ALL survivors given chemotherapy alone attain normal adult height and puberty. Childhood ALL survivors are at increased risk of overweight, especially those with increased BMI at diagnosis. Clinicians should screen for overweight early in survivorship and introduce early interventions.