OBJECTIVETo identify and define clinically meaningful type 1 diabetes outcomes beyond hemoglobin A1c (HbA1c) based upon a review of the evidence, consensus from clinical experts, and input from researchers, people with type 1 diabetes, and industry. Priority outcomes include hypoglycemia, hyperglycemia, time in range, diabetic ketoacidosis (DKA), and patient-reported outcomes (PROs). While priority outcomes for type 1 and type 2 diabetes may overlap, type 1 diabetes was the focus of this work.RESEARCH AND METHODSA Steering Committee—comprising representatives from the American Association of Clinical Endocrinologists, the American Association of Diabetes Educators, the American Diabetes Association, the Endocrine Society, JDRF International, The Leona M. and Harry B. Helmsley Charitable Trust, the Pediatric Endocrine Society, and the T1D Exchange—was the decision-making body for the Type 1 Diabetes Outcomes Program. Their work was informed by input from researchers, industry, and people with diabetes through Advisory Committees representing each stakeholder group. Stakeholder surveys were used to identify priority outcomes. The outcomes prioritized in the surveys were hypoglycemia, hyperglycemia, time in range, DKA, and PROs. To develop consensus on the definitions of these outcomes, the Steering Committee relied on published evidence, their clinical expertise, and feedback from the Advisory Committees.RESULTSThe Steering Committee developed definitions for hypoglycemia, hyperglycemia, time in range, and DKA in type 1 diabetes. The definitions reflect their assessment of the outcome’s short- and long-term clinical impact on people with type 1 diabetes. Knowledge gaps to be addressed by future research were identified. The Steering Committee discussed PROs and concluded that further type 1 diabetes–specific development is needed.CONCLUSIONSThe Steering Committee recommends use of the defined clinically meaningful outcomes beyond HbA1c in the research, development, and evaluation of type 1 diabetes therapies.
Traumatic brain injuries (TBI) are common occurrences in childhood, often resulting in long term, life altering consequences. Research into endocrine sequelae following injury has gained attention; however, there are few studies in children. This paper reviews the pathophysiology and current literature documenting risk for endocrine dysfunction in children suffering from TBI. Primary injury following TBI often results in disruption of the hypothalamic-pituitary-adrenal axis and antidiuretic hormone production and release, with implications for both acute management and survival. Secondary injuries, occurring hours to weeks after TBI, result in both temporary and permanent alterations in pituitary function. At five years after moderate to severe TBI, nearly 30% of children suffer from hypopituitarism. Growth hormone deficiency and disturbances in puberty are the most common; however, any part of the hypothalamic-pituitary axis can be affected. In addition, endocrine abnormalities can improve or worsen with time, having a significant impact on children’s quality of life both acutely and chronically. Since primary and secondary injuries from TBI commonly result in transient or permanent hypopituitarism, we conclude that survivors should undergo serial screening for possible endocrine disturbances. High indices of suspicion for life threatening endocrine deficiencies should be maintained during acute care. Additionally, survivors of TBI should undergo endocrine surveillance by 6–12 months after injury, and then yearly, to ensure early detection of deficiencies in hormonal production that can substantially influence growth, puberty and quality of life.
Context Pituitary dysfunction with abnormal GH secretion and neurocognitive deficits are common consequences of traumatic brain injury (TBI). Recognizing the comorbidity of these symptoms is of clinical importance; however, efficacious treatment is currently lacking. Evidence Acquisition A review of studies in PubMed published between January 1980 to March 2020 and ongoing clinical trials was conducted using the search terms “growth hormone,” “traumatic brain injury,” and “gut microbiome.” Evidence Synthesis Increasing evidence have implicated the effects of TBI in promoting an interplay of ischemia, cytotoxicity and inflammation that renders a subset of patients to develop post-injury hypopituitarism, severe fatigue, impaired cognition and behavioral processes. Recent data have suggested an association between abnormal GH secretion and altered gut microbiome in TBI patients, thus prompting the description of a hypothesized new clinical syndrome called “Brain Injury Associated Fatigue and Altered Cognition” (BIAFAC). Notably, these patients demonstrate distinct characteristics to those with GH deficiency from other non-TBI causes in that their symptom complex improves significantly with recombinant human GH treatment, but does not reverse the underlying mechanistic cause as symptoms typically recur upon treatment cessation. Conclusion The reviewed data describes the importance of alterations of the GH/IGF-I axis and gut microbiome after brain injury and its influence in promoting neurocognitive and behavioral deficits in a bi-directional relationship, and highlight a new clinical syndrome that may exist in a subset of TBI patients in whom recombinant human GH therapy could significantly improve symptomatology. More studies are needed to further characterize this clinical syndrome.
Setting: Hemangioma and vascular malformation center at a tertiary care children's hospital. Patients: Sixteen infants with hemangiomas had an adrenal axis evaluation as soon as possible following the completion of GC therapy. Ten healthy control infants were also evaluated for comparison. Interventions: Prednisolone at a starting dose of 2 to 3 mg/kg/d for 4 weeks, followed by a tapering period. The mean duration of GC treatment was 7.2 months. Main Outcome Measure: Prevalence of adrenal insufficiency in GC-treated subjects as assessed by a combination low-dose/high-dose corticotropin stimulation test. Results: Subjects underwent corticotropin testing at a mean of 13 days after the completion of therapy. Only 1 of the 16 GC-treated infants (6%) had adrenal insufficiency. This subject was tested 1 day after GC treatment was stopped, and results from retesting 3 months later were normal. All control subjects had normal adrenal function. Conclusion: Infants with hemangiomas are at low risk of adrenal insufficiency following the completion of GC therapy, as used in our hemangioma center.
Our survey reveals wide variation in all aspects of pediatric diabetes care delivery in the United States. Pediatric Endocrinologists responding to the survey identified a lack of resources and the current fee for service payment model as a major impediment to practice and the lack of integrated BH staff as a key gap in service. The respondents strongly support its organizations' involvement in the dissemination of standards for care delivery and advocacy for a national payment model aligned with chronic diabetes care in the context of our emerging value-based healthcare system.
Youth with diabetes frequently have limited access to traditional camps because of the need for accessible medical staff. With organized camping becoming more specialized with regard to meeting the needs of youth, there is an increased interest in developing and examining the efficacy of programs that serve individuals in specific illness groups, such as youth with type 1 diabetes. In a collaborative effort between a local university, a diabetes center of a local hospital, and the Lions Club, a diabetes camp was created to assist youth in the management of their diabetes. Data were collected over the 3-day family diabetes camp through three approaches: a pre- and post-program resiliency-based questionnaire, the 14-item Camper Learning Scale, and open-ended questions for parents of children with diabetes who were involved in camp. Wilcoxon t tests were used to analyze any differences between pre- and post-program scores on resiliency. The results indicated a positive increase of parents’ perceptions of their child’s resiliency ( Z = –1.833, P = 0.67). Findings from the Camper Learner Scale indicated that 77.14% of campers felt they “learned a little” or “learned a lot” about crucial youth development outcomes (e.g., independence). Finally, direct content analysis of the qualitative measures indicated several themes among parent respondents, which were generalized into three categories: motivation, community, and challenges. Diabetes camps and family diabetes camps have a great opportunity to address some of the challenges young people face while living with the second most common chronic illness facing youth.
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