Background Limited data exist on the impact of prophylaxis on adults with severe hemophilia A and pre-existing joint disease. Objectives To describe 3-year bleeding, joint health and structure, health-related quality-of-life (HRQoL) and other outcomes from the open-label, randomized, multinational SPINART study. Patients/Methods Males aged 12-50 years with severe hemophilia A, ≥ 150 factor VIII exposure days, no inhibitors and no prophylaxis for > 12 consecutive months in the past 5 years were randomized to sucrose-formulated recombinant FVIII prophylaxis or on-demand therapy (OD). Data collected included total and joint bleeding events (BEs), joint structure (magnetic resonance imaging [MRI]), joint health (Colorado Adult Joint Assessment Scale [CAJAS]), HRQoL, pain, healthcare resource utilization (HRU), activity, and treatment satisfaction. Results Following 3 years of prophylaxis, adults maintained excellent adherence, with a 94% reduction in BEs despite severe pre-existing arthropathy; 35.7% and 76.2% of prophylaxis participants were bleed-free or had fewer than two BEs per year, respectively. As compared with OD, prophylaxis was associated with improved CAJAS scores (least squares [LS] mean, - 0.31 [n = 42] versus + 0.63 [n = 42]) and HAEMO-QoL-A scores (LS mean, + 3.98 [n = 41] versus - 6.00 [n = 42]), less chronic pain (50% decrease), and approximately two-fold less HRU; activity, Euro QoL-5D-3L (EQ-5D-3L) scores and satisfaction scores also favored prophylaxis. However, MRI score changes were not different for prophylaxis versus OD (LS mean, + 0.79 [n = 41] versus + 0.96 [n = 38]). Conclusions Over a period of 3 years, prophylaxis versus OD in adults with severe hemophilia A and arthropathy led to decreased bleeding, pain, and HRU, better joint health, activity, satisfaction, and HRQoL, but no reduction in structural arthropathy progression, suggesting that pre-existing joint arthropathy may be irreversible.

Risk of type 1 diabetes at 3 years is high for initially multiple and single Ab+ IT and multiple Ab+ NT. Genetic predisposition, age, and male sex are significant risk factors for development of Ab+ in twins.

Background: People with type 1 diabetes (T1D) aged <21 years are eligible for subsidised continuous glucose monitoring (CGM) products under the Australian National Diabetes Services Scheme. There are few real-world published studies to evaluate the benefits of CGM in young adults.Aims: To perform a real-world observation study among youth with T1D to evaluate CGM use and benefits of CGM.Methods: Patients at the Westmead Hospital Young Adult Diabetes Clinic aged 15-21 years who commenced CGM before July 2018 were followed for 6 months post commencement of CGM. Differences in HbA1c and glucose metrics at baseline and follow up are compared between those commencing CGM and those that did not.Results: Forty-four (38%) of 115 eligible patients commenced CGM. Demographic characteristics and baseline HbA1c did not differ significantly between those started on CGM and those that did not. At 6 months, 18 (41%) of 44 patients still used CGM, with discomfort and inconvenience the most common reasons for dropout. In CGM continuers, at 6 months compared with baseline, there was no change in HbA1c (8.2% vs 8.0%; P = 0.8), coefficient of variation of glucose (38% vs 39%; P = 0.5) or percentage time in range (52% vs 58%; P = 0.3). Six-month follow-up HbA1c in CGM non-users deteriorated significantly compared with users. Mean hypoglycaemia fear scores (worry scale) were significantly decreased from baseline at 6 months (33 vs 18; P < 0.01). Conclusion:There are high rates of discontinuation in CGM use among youth with T1D. At 6 months of CGM use, there was no significant change in glycaemic control, although HbA1c in non-users deteriorated significantly. Worry of hypoglycaemia was significantly decreased among those who continued CGM.