Effectiveness and safety of long-term treatment with sulfonylureas in patients with neonatal diabetes due to KCNJ11 mutations: an international cohort study.
| Autor: | Bowman P; University of Exeter Medical School, Exeter, UK., Sulen Å; KG Jebsen Centre for Diabetes Research, Department of Clinical Science, University of Bergen, Bergen, Norway; Department of Paediatrics and Adolescents, Haukeland University Hospital, Bergen, Norway., Barbetti F; Bambino Gesù Children's Hospital, Rome, Italy., Beltrand J; Service Endocrinologie, Gynécologie et Diabétologie Pédiatrique, Hôpital Universitaire Necker Enfants Malades Paris, Assistance Publique-Hôpitaux de Paris, Faculté de Médecine Paris Descartes-Université Sorbonne Paris Cité, Inserm U1016, Institut Imagine, Paris, France., Svalastoga P; KG Jebsen Centre for Diabetes Research, Department of Clinical Science, University of Bergen, Bergen, Norway; Department of Paediatrics and Adolescents, Haukeland University Hospital, Bergen, Norway., Codner E; Institute for Maternal and Child Research, School of Medicine, University of Chile, Santiago, Chile., Tessmann EH; Children's Hospital Erlanger, Chattanooga, TN, USA., Juliusson PB; KG Jebsen Centre for Diabetes Research, Department of Clinical Science, University of Bergen, Bergen, Norway; Department of Paediatrics and Adolescents, Haukeland University Hospital, Bergen, Norway., Skrivarhaug T; Department of Paediatrics, Oslo University Hospital, Oslo, Norway., Pearson ER; Division of Molecular and Clinical Medicine, School of Medicine, University of Dundee, Dundee, UK., Flanagan SE; University of Exeter Medical School, Exeter, UK., Babiker T; University of Exeter Medical School, Exeter, UK., Thomas NJ; University of Exeter Medical School, Exeter, UK., Shepherd MH; University of Exeter Medical School, Exeter, UK; Exeter NIHR Clinical Research Facility, Royal Devon and Exeter NHS Foundation Trust, Exeter, UK., Ellard S; University of Exeter Medical School, Exeter, UK., Klimes I; Biomedical Research Centre, Slovak Academy of Sciences, Bratislava, Slovakia., Szopa M; Department of Metabolic Diseases, Jagiellonian University Medical College, Krakow, Poland., Polak M; Service Endocrinologie, Gynécologie et Diabétologie Pédiatrique, Hôpital Universitaire Necker Enfants Malades Paris, Assistance Publique-Hôpitaux de Paris, Faculté de Médecine Paris Descartes-Université Sorbonne Paris Cité, Inserm U1016, Institut Imagine, Paris, France., Iafusco D; Department of Paediatrics, University of Campania Luigi Vanvitelli, Naples, Italy., Hattersley AT; University of Exeter Medical School, Exeter, UK. Electronic address: a.t.hattersley@exeter.ac.uk., Njølstad PR; KG Jebsen Centre for Diabetes Research, Department of Clinical Science, University of Bergen, Bergen, Norway; Department of Paediatrics and Adolescents, Haukeland University Hospital, Bergen, Norway. |
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| Jazyk: | angličtina |
| Zdroj: | The lancet. Diabetes & endocrinology [Lancet Diabetes Endocrinol] 2018 Aug; Vol. 6 (8), pp. 637-646. Date of Electronic Publication: 2018 Jun 04. |
| DOI: | 10.1016/S2213-8587(18)30106-2 |
| Abstrakt: | Background: KCNJ11 mutations cause permanent neonatal diabetes through pancreatic ATP-sensitive potassium channel activation. 90% of patients successfully transfer from insulin to oral sulfonylureas with excellent initial glycaemic control; however, whether this control is maintained in the long term is unclear. Sulfonylurea failure is seen in about 44% of people with type 2 diabetes after 5 years of treatment. Therefore, we did a 10-year multicentre follow-up study of a large international cohort of patients with KCNJ11 permanent neonatal diabetes to address the key questions relating to long-term efficacy and safety of sulfonylureas in these patients. Methods: In this multicentre, international cohort study, all patients diagnosed with KCNJ11 permanent neonatal diabetes at five laboratories in Exeter (UK), Rome (Italy), Bergen (Norway), Paris (France), and Krakow (Poland), who transferred from insulin to oral sulfonylureas before Nov 30, 2006, were eligible for inclusion. Clinicians collected clinical characteristics and annual data relating to glycaemic control, sulfonylurea dose, severe hypoglycaemia, side-effects, diabetes complications, and growth. The main outcomes of interest were sulfonylurea failure, defined as permanent reintroduction of daily insulin, and metabolic control, specifically HbA Findings: 90 patients were identified as being eligible for inclusion and 81 were enrolled in the study and provided long-term (>5·5 years cut-off) outcome data. Median follow-up duration for the whole cohort was 10·2 years (IQR 9·3-10·8). At most recent follow-up (between Dec 1, 2012, and Oct 4, 2016), 75 (93%) of 81 participants remained on sulfonylurea therapy alone. Excellent glycaemic control was maintained for patients for whom we had paired data on HbA Interpretation: High-dose sulfonylurea therapy is an appropriate treatment for patients with KCNJ11 permanent neonatal diabetes from diagnosis. This therapy is safe and highly effective, maintaining excellent glycaemic control for at least 10 years. Funding: Wellcome Trust, Diabetes UK, Royal Society, European Research Council, Norwegian Research Council, Kristian Gerhard Jebsen Foundation, Western Norway Regional Health Authority, Southern and Eastern Norway Regional Health Authority, Italian Ministry of Health, Aide aux Jeunes Diabetiques, Societe Francophone du Diabete, Ipsen, Slovak Research and Development Agency, and Research and Development Operational Programme funded by the European Regional Development Fund. (Copyright © 2018 The Author(s). Published by Elsevier Ltd. This is an Open Access article under the CC BY 4.0 license. Published by Elsevier Ltd.. All rights reserved.) |
| Databáze: | MEDLINE |
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