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Precision Medicine: Long-Term Treatment with Sulfonylureas in Patients with Neonatal Diabetes Due to KCNJ11 Mutations

  • Pediatric Type 2 and Monogenic Diabetes (O Pinhas-Hamiel, Section Editor)
  • Published:
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Purpose of Review

The goal of this review is to provide updates on the safety and efficacy of long-term sulfonylurea use in patients with KCNJ11-related diabetes. Publications from 2004 to the present were reviewed with an emphasis on literature since 2014.

Recent Findings

Sulfonylureas, often taken at high doses, have now been utilized effectively in KCNJ11 patients for over 10 years. Mild–moderate hypoglycemia can occur, but in two studies with a combined 975 patient-years on sulfonylureas, no severe hypoglycemic events were reported. Improvements in neurodevelopment and motor function after transition to sulfonylureas continue to be described.


Sulfonylureas continue to be an effective, sustainable, and safe treatment for KCNJ11-related diabetes. Ongoing follow-up of patients in research registries will allow for deeper understanding of the facilitators and barriers to long-term sustainability. Further understanding of the effect of sulfonylurea on long-term neurodevelopmental outcomes, and the potential for adjunctive therapies, is needed.

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KATP channel:

ATP-dependent potassium channel




Permanent neonatal diabetes


Transient neonatal diabetes


Diabetic ketoacidosis


Developmental delay, epilepsy, and neonatal diabetes


Central nervous system


Single-photon emission computed tomography


Cerebrospinal fluid


Papers of particular interest, published recently, have been highlighted as: • Of importance •• Of major importance

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We would like to acknowledge the international group of scientists and families who contribute to monogenic diabetes research. We would especially like to thank the families who participate in the Monogenic Diabetes Registry at the University of Chicago, and the healthcare teams providing care for them.


This work was supported by the National Institute of Diabetes and Digestive and Kidney Diseases of the National Institutes of Health [grant numbers R01 DK104942, P30 DK020595, and K23 DK094866], the CTSA [grant number UL1 TR002389], as well as by grants from the American Diabetes Association [grant numbers 1-11-CT-41 and 1-17-JDF-008], and gifts from the Kovler Family Foundation.

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Correspondence to Siri Atma W. Greeley.

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Letourneau, L.R., Greeley, S.A.W. Precision Medicine: Long-Term Treatment with Sulfonylureas in Patients with Neonatal Diabetes Due to KCNJ11 Mutations. Curr Diab Rep 19, 52 (2019).

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