Literature DB >> 31250216

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

Lisa R Letourneau1, Siri Atma W Greeley2.   

Abstract

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.

Entities:  

Keywords:  DEND syndrome; Glyburide; KCNJ11; Monogenic diabetes; Neonatal diabetes; Sulfonylurea

Mesh:

Substances:

Year:  2019        PMID: 31250216      PMCID: PMC6894166          DOI: 10.1007/s11892-019-1175-9

Source DB:  PubMed          Journal:  Curr Diab Rep        ISSN: 1534-4827            Impact factor:   4.810


  59 in total

1.  Diagnosis and treatment of neonatal diabetes: a United States experience.

Authors:  Julie Støy; Siri Atma W Greeley; Veronica P Paz; Honggang Ye; Ashley N Pastore; Kinga B Skowron; Rebecca B Lipton; Fran R Cogen; Graeme I Bell; Louis H Philipson
Journal:  Pediatr Diabetes       Date:  2008-07-25       Impact factor: 4.866

2.  Variation of interstitial glucose measurements assessed by continuous glucose monitors in healthy, nondiabetic individuals.

Authors:  Larry A Fox; Roy W Beck; Dongyuan Xing
Journal:  Diabetes Care       Date:  2010-03-09       Impact factor: 19.112

3.  Activating mutations in the gene encoding the ATP-sensitive potassium-channel subunit Kir6.2 and permanent neonatal diabetes.

Authors:  Anna L Gloyn; Ewan R Pearson; Jennifer F Antcliff; Peter Proks; G Jan Bruining; Annabelle S Slingerland; Neville Howard; Shubha Srinivasan; José M C L Silva; Janne Molnes; Emma L Edghill; Timothy M Frayling; I Karen Temple; Deborah Mackay; Julian P H Shield; Zdenek Sumnik; Adrian van Rhijn; Jerry K H Wales; Penelope Clark; Shaun Gorman; Javier Aisenberg; Sian Ellard; Pål R Njølstad; Frances M Ashcroft; Andrew T Hattersley
Journal:  N Engl J Med       Date:  2004-04-29       Impact factor: 91.245

4.  The G53D mutation in Kir6.2 (KCNJ11) is associated with neonatal diabetes and motor dysfunction in adulthood that is improved with sulfonylurea therapy.

Authors:  Joseph C Koster; Francesco Cadario; Cinzia Peruzzi; Carlo Colombo; Colin G Nichols; Fabrizio Barbetti
Journal:  J Clin Endocrinol Metab       Date:  2007-12-11       Impact factor: 5.958

5.  Neuropsychological dysfunction and developmental defects associated with genetic changes in infants with neonatal diabetes mellitus: a prospective cohort study [corrected].

Authors:  Kanetee Busiah; Séverine Drunat; Laurence Vaivre-Douret; Amélie Bonnefond; Albane Simon; Isabelle Flechtner; Bénédicte Gérard; Nathalie Pouvreau; Caroline Elie; Revital Nimri; Liat De Vries; Nadia Tubiana-Rufi; Chantal Metz; Anne-Marie Bertrand; Sylvie Nivot-Adamiak; Marc de Kerdanet; Chantal Stuckens; Farida Jennane; Pierre-François Souchon; Claire Le Tallec; Christelle Désirée; Sabrina Pereira; Aurélie Dechaume; Jean-Jacques Robert; Moshe Phillip; Raphaël Scharfmann; Paul Czernichow; Philippe Froguel; Martine Vaxillaire; Michel Polak; Hélène Cavé
Journal:  Lancet Diabetes Endocrinol       Date:  2013-09-06       Impact factor: 32.069

6.  Effectiveness and safety of long-term treatment with sulfonylureas in patients with neonatal diabetes due to KCNJ11 mutations: an international cohort study.

Authors:  Pamela Bowman; Åsta Sulen; Fabrizio Barbetti; Jacques Beltrand; Pernille Svalastoga; Ethel Codner; Ellen H Tessmann; Petur B Juliusson; Torild Skrivarhaug; Ewan R Pearson; Sarah E Flanagan; Tarig Babiker; Nicholas J Thomas; Maggie H Shepherd; Sian Ellard; Iwar Klimes; Magdalena Szopa; Michel Polak; Dario Iafusco; Andrew T Hattersley; Pål R Njølstad
Journal:  Lancet Diabetes Endocrinol       Date:  2018-06-04       Impact factor: 32.069

7.  Sulfonylureas and their use in clinical practice.

Authors:  Daniele Sola; Luca Rossi; Gian Piero Carnevale Schianca; Pamela Maffioli; Marcello Bigliocca; Roberto Mella; Francesca Corlianò; Gian Paolo Fra; Ettore Bartoli; Giuseppe Derosa
Journal:  Arch Med Sci       Date:  2015-08-11       Impact factor: 3.318

8.  Systemic Administration of Glibenclamide Fails to Achieve Therapeutic Levels in the Brain and Cerebrospinal Fluid of Rodents.

Authors:  Carolina Lahmann; Holger B Kramer; Frances M Ashcroft
Journal:  PLoS One       Date:  2015-07-30       Impact factor: 3.240

9.  Switching to sulphonylureas in children with iDEND syndrome caused by KCNJ11 mutations results in improved cerebellar perfusion.

Authors:  Wojciech Fendler; Iwona Pietrzak; Melissa F Brereton; Carolina Lahmann; Mariusz Gadzicki; Malgorzata Bienkiewicz; Izabela Drozdz; Maciej Borowiec; Maciej T Malecki; Frances M Ashcroft; Wojciech M Mlynarski
Journal:  Diabetes Care       Date:  2013-03-05       Impact factor: 19.112

10.  Successful transfer to sulfonylureas in KCNJ11 neonatal diabetes is determined by the mutation and duration of diabetes.

Authors:  Tarig Babiker; Natascia Vedovato; Kashyap Patel; Nicholas Thomas; Roisin Finn; Roope Männikkö; Ali J Chakera; Sarah E Flanagan; Maggie H Shepherd; Sian Ellard; Frances M Ashcroft; Andrew T Hattersley
Journal:  Diabetologia       Date:  2016-03-31       Impact factor: 10.122

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  3 in total

1.  Genetic Spectrum of Neonatal Diabetes.

Authors:  M Kocova
Journal:  Balkan J Med Genet       Date:  2021-03-23       Impact factor: 0.519

2.  Developmental defects and impaired network excitability in a cerebral organoid model of KCNJ11 p.V59M-related neonatal diabetes.

Authors:  Gokhan Dalgin; Andrew K Tryba; Ashley P Cohen; Soo-Young Park; Louis H Philipson; Siri Atma W Greeley; Alfredo J Garcia
Journal:  Sci Rep       Date:  2021-11-03       Impact factor: 4.379

Review 3.  Treatable inherited metabolic epilepsies.

Authors:  Khalid Hundallah; Brahim Tabarki
Journal:  Neurosciences (Riyadh)       Date:  2021-07       Impact factor: 0.906

  3 in total

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