Adrenal insufficiency (AI) remains a significant cause of morbidity and mortality in children with 1 in 200 episodes of adrenal crisis resulting in death. The goal of this working group of the Pediatric Endocrine Society Drug and Therapeutics Committee was to raise awareness on the importance of early recognition of AI, to advocate for the availability of hydrocortisone sodium succinate (HSS) on emergency medical service (EMS) ambulances or allow EMS personnel to administer patient’s HSS home supply to avoid delay in administration of life-saving stress dosing, and to provide guidance on the emergency management of children in adrenal crisis. Currently, hydrocortisone, or an equivalent synthetic glucocorticoid, is not available on most ambulances for emergency stress dose administration by EMS personnel to a child in adrenal crisis. At the same time, many States have regulations preventing the use of patient’s home HSS supply to be used to treat acute adrenal crisis. In children with known AI, parents and care providers must be made familiar with the administration of maintenance and stress dose glucocorticoid therapy to prevent adrenal crises. Patients with known AI and their families should be provided an Adrenal Insufficiency Action Plan, including stress hydrocortisone dose (both oral and intramuscular/intravenous) to be provided immediately to EMS providers and triage personnel in urgent care and emergency departments. Advocacy efforts to increase the availability of stress dose HSS during EMS transport care and add HSS to weight-based dosing tapes are highly encouraged.
Objective
Diamond Blackfan anemia (DBA) is a rare inherited bone marrow failure syndrome. The mainstays of treatment involve chronic red cell transfusions, long-term glucocorticoid therapy, and stem cell transplantation. Systematic data concerning endocrine function in DBA are limited. We studied patients in the DBA Registry (DBAR) of North America to assess the prevalence of various endocrinopathies.
Study design
In a pilot study, retrospective data were collected for 12 patients with DBA. Subsequently, patients with DBA aged 1–39 years were recruited prospectively. Combined, 57 patients were studied; 38 chronically transfused, 12 glucocorticoid-dependent and 7 in remission. Data were collected on anthropometric measurements, systematic screening tests of pituitary, thyroid, parathyroid, adrenal, pancreatic and gonadal function and ferritin levels. Descriptive statistics were tabulated and group differences assessed.
Results
Fifty-three percent of patients had ≥ 1 endocrine disorder, including adrenal insufficiency (32%), hypogonadism (29%), hypothyroidism (14%), growth hormone dysfunction (7%), diabetes mellitus (2%), and/or diabetes insipidus (2%). Ten of 33 patients with available heights had height SD <−2. Low 25(OH)D levels were present in 50%. A small proportion also had osteopenia, osteoporosis or hypercalciuria. Most with adrenal insufficiency were glucocorticoid-dependent; other endocrinopathies were more common in chronically transfused patients.
Conclusions
Endocrine dysfunction is common in DBA, as early as the teenage years. While prevalence is highest in transfused patients, patients taking glucocorticoids or in remission also have endocrine dysfunction. Longitudinal studies are needed to better understand the etiology and true prevalence of these disorders.
<b><i>Background:</i></b> Pediatric endocrine practices had to rapidly transition to telemedicine care at the onset of the novel coronavirus disease 2019 (COVID-19) pandemic. For many, it was an abrupt introduction to providing virtual healthcare, with concerns related to quality of patient care, patient privacy, productivity, and compensation, as workflows had to change. <b><i>Summary:</i></b> The review summarizes the common adaptations for telemedicine during the pandemic with respect to the practice of pediatric endocrinology and discusses the benefits and potential barriers to telemedicine. <b><i>Key Messages:</i></b> With adjustments to practice, telemedicine has allowed providers to deliver care to their patients during the COVID-19 pandemic. The broader implementation of telemedicine in pediatric endocrinology practice has the potential for expanding patient access. Research assessing the impact of telemedicine on patient care outcomes in those with pediatric endocrinology conditions will be necessary to justify its continued use beyond the COVID-19 pandemic.
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