Endocrine Abstracts

Introduction: Wolfram syndrome (WS) is an ultra-rare form of progressive neurodegeneration with diabetes mellitus and optic atrophy as the key clinical manifestations in childhood. Birmingham Children’s Hospital (BCH) is the UK lead centre for management of paediatric WS. In recent years, monitoring of glucose in diabetes management increasingly involves continuous glucose monitoring sensors (rtCGM) rather than capillary blood glucose meters (CBG). Using rtCGM in type 1 diabetes mellitus (T1DM) has been shown to improve mean glycated haemoglobin levels (HbA1c), but there is yet to be a study evaluating rtCGM in WS.

Aim: Does rtCGM improve glycaemic control in paediatric patients with WS?

Method: Retrospective case note review of all patients with WS and insulin-dependent diabetes seen between 2018 and 2024 and switched to rtCGM. For each year within this period, data was collected on glucose monitoring device (rtCGM vs CBG), insulin regimen (pump vs multiple dose insulin), and demographic information.

Results: For all patients in the BCH Wolfram Clinic, median HbA1c reduced from 2018 to 2024 (median HbA1c in 2018 was 64 mmol/mol, range 40-122 mmol/mol, vs median HbA1c in 2024 was 53 mmol/mol, range 48-71 mmol/mol). 15 patients from this cohort met the inclusion criteria (median age at study onset was 11 years, range 6-18 years), 9 females: 6 males. rtCGM methods included Dexcom (n = 6) and Libre (n = 7). At baseline, median HbA1c was 65 mmol/mol (range 47-122 mmol/mol) vs median first recorded HbA1c post-change to rtCGM of 61 mmol/mol (range 48-84 mmol/mol). Following change to rtCGM, 9 patients showed an overall decrease in HbA1c (5 females: 4 males) vs 6 patients who showed an overall increase in HbA1c (4 females: 2 males).

Conclusion: In summary, the mean HbA1c of children with WS has improved over the last 6 years in line with national data for T1DM. Patients who commenced rtCGM demonstrated a modest improvement in HbA1c, perhaps because of the additional burden of complications in WS. Patient feedback suggested acceptability and improved quality of life with rtCGM. Hybrid closed loop treatment in this cohort of patients has the potential to further impact on glycaemic control and quality of life outcomes.