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Special Populations (SPO)

Advancing the Utilization of Real-World Data (RWD) and Real-World Evidence (RWE) in Clinical Pharmacology and Translational Research

RWE-011 - THE IMPACT OF PARENT-OF-ORIGIN ON CHILDHOOD GROWTH TRAJECTORIES AND SULFONYLUREAS USE IN CHILDREN WITH HNF1A-MODY IN THE U.S. MONOGENIC DIABETES REGISTRY.

Tuesday, March 21, 2023
4:35 PM – 5:30 PM EDT
M. Salguero, A. Ravisankar, L. Philipson, R. Naylor; The University of Chicago, Chicago, IL, USA.


Background: MODY (autosomal dominant diabetes) accounts for ~3% of pediatric diabetes. Precision therapy for HNF1A-MODY is sulfonylureas (SU). There are gaps in understanding impact of in utero maternal hyperglycemia on childhood weight and SU efficacy.

Objective: Assess impact of parent-of-origin on childhood growth trajectories and SU use in pediatric HNF1A-MODY in the U.S. Monogenic Diabetes Registry.

Methods: Cross-sectional analysis of Registry data on 23 pediatric participants with HNF1A-MODY. We compared BMI percentiles at birth, diabetes diagnosis, and most recent Registry survey as well as rates of precision therapy using non-parametric methods.

Results: There were 7 males (30%), 16 females (70%). 12 inherited HNF1A-MODY maternally (MAT), 10 paternally (PAT). One mutation was spontaneous. Birth weight percentile was 72 MAT vs 56 PAT, p=0.16. BMI percentile at diabetes diagnosis was 76 MAT vs 64 PAT, p=0.20. Most recent BMI percentile was 84 MAT vs 66 PAT, p=0.44).

33% of MAT participants vs 44% of PAT participants had ever taken SU. On most recent Registry survey, 33% of MAT participants were taking SU compared to 60% of PAT. Specific data for SU failure was absent in most participants. One male patient with maternally-inherited HNF1A-MODY and weight and BMI percentiles >99th, failed treatment after 1 year.

Conclusion: There was higher weight/BMI percentiles in MAT group compared to PAT, but this did not reach statistical significance. We note a trend toward less SU use in MAT group. Conclusions cannot be drawn based on small numbers and limitations of missing data. Future studies will collect detailed information to test the hypothesis that negative fetal programming from in utero maternal hyperglycemia decreases SU efficacy of in pediatric HNF1A-MODY.