Introduction: Novel therapies, including disease-modifying and cell replacement therapies, may preserve or replace beta cells in people with type 1 diabetes. This study sought to understand how people living with type 1 diabetes or caring for someone with type 1 diabetes perceive the benefits and risks of novel therapies.
Methods: Semistructured qualitative interviews were conducted with 26 participants in the United States: four adolescents and 12 adults with type 1 diabetes, and 10 caregivers of children with type 1 diabetes.
The underrepresentation of women in senior positions persists in academic medicine. Data-driven strategies are needed to catalyze advancement. We designed a novel, National Institutes of Health-funded intervention-Engaging Peer Mentors for Opportunity, Well-Being, and Equity Realization (EMPOWER) to be evaluated in a randomized study.
View Article and Find Full Text PDFObjective: Continuous glucose monitoring (CGM) measures could be a surrogate for stimulated C-peptide outcomes in type 1 diabetes trials.
Research Design And Methods: CGM and mixed-meal tolerance test-derived C-peptide measures at time points out to 52 weeks after diagnosis were compared in 103 children.
Results: At 52 weeks, CGM metrics moderately correlated with C-peptide area under the curve.
Due to a high percentage of hemoglobin F, Hemoglobin A1C (HbA1C) measurements are inaccurate for assessing glycemia in infants. We aimed to determine when HbA1C might have utility and to assess the value of fructosamine. We measured HbA1C in healthy infants aged 3 weeks to 12 months.
View Article and Find Full Text PDFThe TrialNet Oral Insulin (OI) prevention trial showed no overall treatment effect, using the diagnosis of type 1 diabetes as an endpoint. A significant delay in onset was only found in a high-risk stratum (termed secondary stratum 1) of participants with low first-phase insulin release (FPIR). Since trials with an endpoint of type 1 diabetes take years to complete, in this post hoc analysis, we assessed whether a novel combination of glucose and C-peptide markers could identify a therapeutic benefit after 1 year of follow-up (trial participants followed for a median 2.
View Article and Find Full Text PDFAims/hypothesis: Accurate understanding of type 1 diabetes risk is critical for optimisation of counselling, monitoring and interventions, yet even within established staging classifications, individual time to clinical disease varies. Previous work has associated IA-2A positivity with increased type 1 diabetes progression but a comprehensive assessment of the impact of screening for IA-2A positivity across the natural history of autoantibody positivity has not been performed. We asked whether IA-2A would consistently be associated with higher risk of progression within and across established stages of type 1 diabetes in a large natural history study.
View Article and Find Full Text PDFDiabetologia
April 2025
The International Society for Pediatric and Adolescent Diabetes (ISPAD) guidelines represent a rich repository that serves as the only comprehensive set of clinical recommendations for children, adolescents, and young adults living with diabetes worldwide. This chapter builds on the 2022 ISPAD guidelines, and updates recommendations on the glycemic targets for children and adolescents living with diabetes. A new target for hemoglobin A1c (HbA1c) of ≤6.
View Article and Find Full Text PDFHorm Res Paediatr
December 2024
J Clin Endocrinol Metab
May 2025
Context: Low bone mineral density (BMD) has been reported in children and adolescents living with perinatally acquired HIV (PHIV). Little is known about their bone accrual through puberty compared to an uninfected healthy cohort.
Objective: To compare bone accrual in PHIV and healthy children.
The approval of teplizumab to delay the onset of type 1 diabetes is an important inflection point in the decades-long pursuit to treat the cause of the disease rather than its symptoms. The National Institute of Diabetes and Digestive and Kidney Diseases convened a workshop of the Diabetes Mellitus Interagency Coordinating Committee titled "Evolving Concepts in Pathophysiology, Screening, and Prevention of Type 1 Diabetes" to review this accomplishment and identify future goals. Speakers representing Type 1 Diabetes TrialNet (TrialNet) and the Immune Tolerance Network emphasized that the ability to robustly identify individuals destined to develop type 1 diabetes was essential for clinical trials.
View Article and Find Full Text PDFJ Clin Endocrinol Metab
January 2025
Context: Obesity is prevalent in type 1 diabetes (T1D) and is problematic with higher risk for diabetes complications. It is unknown to what extent gut microbiome changes are associated with obesity and T1D.
Objective: This work aimed to describe the gut microbiome and microbial metabolite changes associated with obesity in T1D.
Given the proven benefits of screening to reduce diabetic ketoacidosis (DKA) likelihood at the time of stage 3 type 1 diabetes diagnosis, and emerging availability of therapy to delay disease progression, type 1 diabetes screening programs are being increasingly emphasized. Once broadly implemented, screening initiatives will identify significant numbers of islet autoantibody-positive (IAb+) children and adults who are at risk for (confirmed single IAb+) or living with (multiple IAb+) early-stage (stage 1 and stage 2) type 1 diabetes. These individuals will need monitoring for disease progression; much of this care will happen in nonspecialized settings.
View Article and Find Full Text PDFGiven the proven benefits of screening to reduce diabetic ketoacidosis (DKA) likelihood at the time of stage 3 type 1 diabetes diagnosis, and emerging availability of therapy to delay disease progression, type 1 diabetes screening programmes are being increasingly emphasised. Once broadly implemented, screening initiatives will identify significant numbers of islet autoantibody-positive (IAb) children and adults who are at risk of (confirmed single IAb) or living with (multiple IAb) early-stage (stage 1 and stage 2) type 1 diabetes. These individuals will need monitoring for disease progression; much of this care will happen in non-specialised settings.
View Article and Find Full Text PDFObjectives: To report the safety and side effects associated with taking verapamil for beta-cell preservation in children with newly-diagnosed T1D.
Research Design And Methods: Eighty-eight participants aged 8.5 to 17.
J Pediatric Infect Dis Soc
August 2024
Background: Tenofovir disoproxil fumarate (TDF) is often used in treating pregnant women living with HIV. Third-trimester TDF exposure is associated with a 12% reduction in bone mineral content in HIV-exposed uninfected (HEU) neonates. The potential mechanisms underlying this observation are unknown.
View Article and Find Full Text PDFAcetylated and butyrylated high amylose starch (HAMS-AB) is a prebiotic shown to be effective in type 1 diabetes (T1D) prevention in mouse models and is safe in adults with established T1D. HAMS-AB alters the gut microbiome profile with increased bacterial fermenters that produce short chain fatty acids (SCFAs) with anti-inflammatory and immune-modulatory effects. We performed a pilot study using a cross-over design to assess the safety and efficacy of 4 weeks of oral HAMS-AB consumption by recently diagnosed (< 2 years of diagnosis) youths with T1D.
View Article and Find Full Text PDFTo evaluate the safety and explore the efficacy of use of ultra-rapid lispro (URLi, Lyumjev) insulin in the Tandem t:slim X2 insulin pump with Control-IQ 1.5 technology in children, teenagers, and adults living with type 1 diabetes (T1D). At 14 U.
View Article and Find Full Text PDFLancet Diabetes Endocrinol
May 2024
Teplizumab (TzieldTM, Provention Bio), a monoclonal antibody directed at T-cell marker CD3, is the first medication approved by the FDA to delay progression from stage 2 to stage 3 type 1 diabetes. To date, the overwhelming majority of pediatric endocrinologists do not have experience using immunotherapeutics and seek guidance on the use of teplizumab in clinical practice. To address this need, the Pediatric Endocrine Society (PES) Diabetes Special Interest Group (Diabetes SIG) and Drug and Therapeutics Committee assembled a task force to review clinical trial data and solicit expert recommendations on the approach to teplizumab infusions.
View Article and Find Full Text PDF