Soluble urokinase plasminogen activator receptor in type 1 diabetic children, relation to vascular complications.

BACKGROUND: Endothelial dysfunction caused by chronic inflammation is the cornerstone of vascular complications in type 1 Diabetes-Mellitus (T1DM). Soluble Urokinase Plasminogen Activator Receptor (SuPAR) is a novel marker of inflammation and endothelial dysfunction. AIM: To evaluate SuPAR in T1DM children and correlate it to diabetic vascular complications. METHODS: Seventy T1DM children and 40 matched healthy controls were studied focusing on disease duration, insulin therapy and symptoms of diabetic complications. Blood-pressure, fundus and screening for peripheral-neuropathy were done. Fasting lipid profile, fraction-C of glycosylated hemoglobin (HbA1c%), Urinary albumin excretion (UAE), estimated-glomerular filtration rate (eGFR) and SuPAR were measured. Internal aortic diameter was measured with calculation of aortic distensibility and stiffness index. RESULTS: Sixteen T1DM patients(22.9%) had peripheral neuropathy, 12(17%) had nephropathy and none had retinopathy. SuPAR was significantly elevated in diabetic nephropathy (p < 0.01) and neuropathy (p < 0.01). Aortic stiffness index was significantly higher (p < 0.01) whereas, aortic strain and distensibility were significantly lower (p < 0.01) in T1DM than controls. SuPAR was significantly correlated to disease duration (p < 0.01), systolic blood pressure (p < 0.01), total cholesterol (p < 0.01), triglycerides (p < 0.01), UAER (p < 0.01) and aortic strain (0.013). CONCLUSION: Increased SuPAR early in diabetes might become a useful indicator of developing vascular complications. Further prospective studies are needed to determine the cut-off level of SuPAR for detection of T1DM and its complications.

Evoked potentials and neurocognitive functions in pediatric Egyptian Gaucher patients on enzyme replacement therapy: a single center experience.

BACKGROUND: Effectiveness of enzyme replacement therapy (ERT) in reverting hematologic, skeletal, and visceral symptoms in Gaucher disease (GD) has been demonstrated, although, its efficacy in neurologic involvement is still debated. AIM: We evaluated the extent of neuro-cognitive dysfunction using brain stem evoked potential in GD3 patients, age-matched controls, and GD1 patients without neurological manifestations served as disease control group. METHODS: Study included 56 GD (36 had type 1, 20 had type 3) under ERT. Investigations included complete blood count, beta glucosidase assay in peripheral leucocytes, plasma chitotriosidase and bone marrow examination, electroencephalography, brain stem auditory (AEP), somatosensory (SSEP) and visual evoked potentials (VEP) as well as IQ testing. RESULTS: Both types of GD showed significantly higher mean latency at 75 on left eye, lower PP amplitude ratio, higher latency at 75, 100, 145, lower amplitude, and higher Lat Diff LT-RT ms and Lt-Rt % compared to controls (p < 0.05) with no difference between both groups in other values of VEP. Both groups showed significantly prolonged latency of N 13-19 compared to controls (p < 0.05) with positive correlation between age and duration of therapy with parameters of SSEP (p < 0.01). Both groups of GD had significantly prolonged latency of the mean waves of AEP compared to controls (p < 0.05) with no significant difference between both groups. There was a negative correlation between age and waves II, III, I-III, I-V and threshold values of AEP. IQ level was positively correlated with AEP values. Severity scoring tool was positively correlated with AEP and SSEP values. CONCLUSIONS: Electrophysiological abnormalities were present in both types of GD and have been correlated to cognitive function and disease characteristics.