Assessment of Motor Unit Potentials Duration as the Biomarker of DT-DEC01 Cell Therapy Efficacy in Duchenne Muscular Dystrophy Patients up to 12 Months After Systemic-Intraosseous Administration.

Duchenne muscular dystrophy (DMD) is a lethal X-linked disease caused by mutations in the dystrophin gene, leading to muscle degeneration and wasting. Electromyography (EMG) is an objective electrophysiological biomarker of muscle fiber function in muscular dystrophies. A novel, DT-DEC01 therapy, consisting of Dystrophin Expressing Chimeric (DEC) cells created by fusing allogeneic myoblasts from normal donors with autologous myoblasts from DMD-affected patients, was assessed for safety and preliminary efficacy in boys of age 6-15 years old (n = 3). Assessments included EMG testing of selected muscles of upper (deltoideus, biceps brachii) and lower (rectus femoris and gastrocnemius) extremities at the screening visit and at 3, 6, and 12 months following systemic-intraosseous administration of a single low dose of DT-DEC01 therapy (Bioethics Committee approval no. 46/2019). No immunosuppression was administered. Safety of DT-DEC01 was confirmed by the lack of therapy-related Adverse Events or Serious Adverse Events up to 22 months following DT-DEC01 administration. EMG of selected muscles of both, ambulatory and non-ambulatory patients confirmed preliminary efficacy of DT-DEC01 therapy by an increase in motor unit potentials (MUP) duration, amplitudes, and polyphasic MUPs at 12 months. This study confirmed EMG as a reliable and objective biomarker of functional assessment in DMD patients after intraosseous administration of the novel DT-DEC01 therapy.

Assessment of changes in blood lactate levels in children and adolescents with type 1 diabetes during a football tournament (GoalDiab Study).

INTRODUCTION: Monitoring physical activity is a very important issue, especially in type 1 diabetes. One of the parameters assessing the intensity of exercise is the concentration of lactate in the blood. Aim of the study We assessed the intensity of PE and changes in lactate levels in children and adolescents with type 1 diabetes (T1D) during a football tournament. MATERIAL AND METHODS: We enrolled 141 participants, the results of 70 of whom were analyzed, playing in two age categories: 10-13 and 14-17 years. Lactate levels were measured in the capillary blood before and after matches. Blood lactate of 4 mmol/l (Onset Blood Lactate Accumulation OBLA) was used as parameter indicating the prevalence of anaerobic metabolic changes. RESULTS: The median lactate level was 1.8 mmol/l before and 4.4 mmol/l after matches (p < 0.001). The increase in lactate levels was higher in the older age category (4.3 vs. 1.8, p = 0.001) and was independent on gender (3.2 vs. 2.1, p = 0.597), personal insulin pump vs insulin pen use (3.0 vs. 1.5, p = 0.145) or training in a sports club (1.4 vs. 3.0, p = 0.084). A positive correlation was noted between increased lactate levels and age (Rs = 0.253, p = 0.034). 61% of the participants exceeded lactate levels ≥ 4 mmol/l. In univariate logistic regression analysis age was a significantly associated with lactate level ≥ 4 mmol/l [OR = 1.45 (1.08-1.95)] independent of HbA1c, gender, treatment method and training in a sports club. CONCLUSIONS: PE intensity levels during football matches were found to be mixed aerobic-anaerobic. Increases in lactate levels were greater in the older subjects independently on the assessed factors.

Assessment of Safety and Glycemic Control During Football Tournament in Children and Adolescents With Type 1 Diabetes-Results of GoalDiab Study.

PURPOSE: To assess glycemic control and safety of children and adolescents with type 1 diabetes participating in a 2-day football tournament. METHODS: In total, 189 children with type 1 diabetes from 11 diabetes care centers, in Poland, participated in a football tournament in 3 age categories: 7-9 (21.2%), 10-13 (42.9%), and 14-17 (36%) years. Participants were qualified and organized in 23 football teams, played 4 to 6 matches of 30 minutes, and were supervised by a medical team. Data on insulin dose and glycemia were downloaded from personal pumps, glucose meters, continuous glucose monitoring, and flash glucose monitoring systems. RESULTS: The median level of blood glucose before the matches was 6.78 (4.89-9.39) mmol/L, and after the matches, it was 7.39 (5.5-9.87) mmol/L (P = .001). There were no episodes of severe hypoglycemia or ketoacidosis. The number of episodes of low glucose value (blood glucose ≤3.9 mmol/L) was higher during the tournament versus 30 days before: 1.2 (0-1.5) versus 0.7 (0.3-1.1) event/person/day, P < .001. Lactate levels increased during the matches (2.2 [1.6-4.0] mmol/L to 4.4 [2.6-8.5] mmol/L after the matches, P < .001). CONCLUSIONS: Large football tournaments can be organized safely for children with type 1 diabetes. For the majority of children, moderate mixed aerobic-anaerobic effort did not adversely affect glycemic results and metabolic safety.