ESPE Abstracts (2023) 97 P1-228

1Aretaieion University Hospital, Athens, Greece. 2Pediatric Endocrine Clinics, Marousi, Greece. 3University of Thessaly, Trikala, Greece. 4Attikon University Hospital, Haidari, Greece

Type 1 diabetes (T1D) hits about 1:300 with rising incidence affecting increasingly younger children. Population screening at ages 2-6yrs with T1D associated autoantibodies (T1Ab) has been recently proven sensitive. While potential treatments to prevent or delay T1D are currently in development, a population based cost-effective preventive strategy is still lacking. Hence, 2000IU cholecalciferol daily in a large birth cohort study published in 2001 reduced by 80% the risk of T1D in 1yr. A pilot clinical trial in 2013, demonstrated negativation of T1Ab within 0.6(0.4-2.1)yrs under oral calcitriol. In a prospective interventional non-randomized clinical trial, the PRECAL study (ISRCTN17354692). Between 2010-2022, 50 children (26 boys, 24 girls) aged 0.65-16.37yrs identified as at high risk for T1D were included: 44 with +T1Abs (Insulin auto-antibodies, IAA; anti-tyrosinase abs, IA2, anti-glutamic decarboxylase abs, GAD; islet auto-abs, ICA; anti-Zn8 if available) and 6 with negative T1Abs but predisposing HLA A DQ DR haplotypes/genotypes. Nine had varied impaired glucose tolerance (IGT) (T1Ab+), 4 pre-T1D (3 T1Ab+ and 1 HLA+), and 9 new-onset T1D (T1Ab+). Serum T1Ab levels and fasting plasma glucose, HbA1c, c-peptide, Ca (tolerated ≤11.5 mg/dl), P, ALP, Ca/Cr 2-hr urine morning sample, 25(OH)D, 1-25(OH)2D with renal ultrasound in cases with hypercalcemia/hypercalciuria (tolerated ≤50%, or at the upper normal for age) were determined before and q3-6 months after initiation of calcitriol (0.05 mcg/Kg/day) 0.25-0.5 x 1-3/day or its synthetic analogue, paricalcitol (thrice the calcitriol dose) 1-4 mg x 1-3/day p.o. under cholecalciferol repletion. Measurements are given as median and range. Available data on 42 (7 dropouts, 1 follow-up <3m). All 26 without pre-T1D/T1D followed 3.06(0.5-10)yrs negativized T1Abs (15IAA, 3IA2, 4ICA, 2GAD, 1IAA/GAD, 1ICA/GAD) within 0.57(0.32-1.3)yrs or did not progress to T1D [5 +HLA, follow-up 3(1-4)yrs]. From 4 pre-T1D, 1 negativized T1Abs (follow-up 1yr), 1 with +HLA did not progress to T1D (follow-up 3.3yrs) and 2 with +T1Ab developed T1D in 6m/3yrs. Three out of 8 children with T1D progressed immediately to overt disease, 5 showed complete remission for 1yr (1m-2yrs). Five with +T1Ab relapsed and negativized again after resuming therapy. Four (<3yrs) negativized anti-TPO/TG, and two anti-transglutaminase-IgA. Eight presented mild hypercalciuria/hypercalcemia, resolving with dose titration/discontinuation. Calcitriol, and paricalcitol were 100% effective and reasonably safe in negativizing T1Abs in healthy children, possibly preventing T1D, at least if started soon enough after seroconversion.

Volume 97

61st Annual ESPE (ESPE 2023)

The Hague, Netherlands
21 Sep 2023 - 23 Sep 2023

European Society for Paediatric Endocrinology 

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