title: Randomised, prospective, medico-economic nationwide French study of islet transplantation in patients with severely unstable type 1 diabetes: the STABILOT study protocol.
abstract: Islet transplantation may be an appropriate treatment option for patients with severely unstable type 1 diabetes experiencing major glucose variability with severe hypoglycaemia despite intensive insulin therapy. Few data are available on the costs associated with islet transplantation in relation to its benefits. The STABILOT study proposes to assess the economic impact of islet transplantation in comparison with the current best medical treatment defined as sensor-augmented pump (SAP) therapy.
  
  
  
  The trial will adopt an open-label, randomised, multicentred design. The study will include 30 patients with severely unstable type 1 diabetes. Eligible participants will be 18-65 years old, with type 1 diabetes duration >5 years, a negative basal or stimulated C-peptide, and severe instability defined by persistent, recurrent and disabling severe hypoglycaemia, despite optimised medical treatment. Participants will be randomised into two groups: one group with immediate registration for islet transplantation, and one group with delayed registration for 1 year while patients receive SAP therapy. The primary endpoint will be the incremental cost-utility ratio at 1 year between islet transplantation and SAP therapy. Perspectives of both the French Health Insurance System and the hospitals will be retained.
  
  
  
  Ethical approval has been obtained at all sites. The trial has been approved by ClinicalTrials.gov (Trial registration ID NCT02854696). All participants will sign a free and informed consent form before randomisation. Results of the study will be communicated during national and international meetings in the field of diabetes and transplantation. A publication will be sought in journals usually read by physicians involved in diabetes care, transplantation and internal medicine.
  
  
  
  NCT02854696; Pre-results.
authors.affiliation: Department of Diabetology, Pôle DigiDune, Grenoble University Hospital, Grenoble Alpes University, Grenoble, France.|Grenoble Alpes University, INSERM, U1055Laboratory of Fundamental and Applied Bioenergetics, Grenoble, France.
authors.forename: Sandrine
authors.identifier: 0000-0001-7044-8474
authors.initials: S
authors.lastname: Lablanche
authors.affiliation: Cellule d'évaluation médico-économique des innovations, CHU Grenoble Alpes, CIC 1406 Grenoble, TIMC-Imag UMR 5525 Univ. Grenoble Alpes, Grenoble, France.
authors.forename: Sandra
authors.initials: S
authors.lastname: David-Tchouda
authors.affiliation: Cellule d'évaluation médico-économique des innovations, CHU Grenoble Alpes, CIC 1406 Grenoble, TIMC-Imag UMR 5525 Univ. Grenoble Alpes, Grenoble, France.
authors.forename: Jennifer
authors.initials: J
authors.lastname: Margier
authors.affiliation: Centre régional de Pharmacovigilance, Grenoble, France.
authors.forename: Edith
authors.initials: E
authors.lastname: Schir
authors.affiliation: Centre Hospitalier de Montpellier, Pôle Rein Hypertension Métabolisme, Service d'Endocrinologie, Montpellier, France.
authors.forename: Anne
authors.initials: A
authors.lastname: Wojtusciszyn
authors.affiliation: Centre Hospitalier Universitaire Jean Minjoz, Service d'Endocrinologie-Métabolisme et Diabétologie-Nutrition, Besançon, France.
authors.forename: Sophie
authors.initials: S
authors.lastname: Borot
authors.affiliation: Hôpitaux Universitaires de Strasbourg, Service d'Endocrinologie Diabète et Maladies Métaboliques, Pôle NUDE, Strasbourg, France.
authors.forename: Laurence
authors.initials: L
authors.lastname: Kessler
authors.affiliation: Hospices Civils de Lyon, Service d'Urologie et de Chirurgie de la Transplantation, Pôle Chirurgie, Lyon, France.
authors.forename: Emmanuel
authors.initials: E
authors.lastname: Morelon
authors.affiliation: Hospices Civils de Lyon, Service de Diabétologie-Endocrinologie-Maladies Métaboliques et de Chirurgie de la Transplantation, Pôle Chirurgie, Lyon, France.
authors.forename: Charles
authors.initials: C
authors.lastname: Thivolet
authors.affiliation: University Lille, CHRU Lille, Inserm U1190 Translational research for diabetes, Lille, France.|European Genomic Institute for Diabetes, EGID, Lille, France.|Department of Endocrine Surgery, Hôpital Huriez Lille University Hospital, Lille Cedex, France.
authors.forename: François
authors.initials: F
authors.lastname: Pattou
authors.affiliation: Department of Endocrine Surgery, Hôpital Huriez Lille University Hospital, Lille Cedex, France.|Department of Endocrinology and Metabolism, Hôpital Huriez, Lille University Hospital, Lille Cedex, France.
authors.forename: Marie Christine
authors.initials: MC
authors.lastname: Vantyghem
authors.affiliation: Departement of Surgery, Islet Isolation, and Transplantation Center, Geneva University Hospitals, Geneva, Switzerland.
authors.forename: Thierry
authors.initials: T
authors.lastname: Berney
authors.affiliation: Department of Diabetology, Pôle DigiDune, Grenoble University Hospital, Grenoble Alpes University, Grenoble, France.|Grenoble Alpes University, INSERM, U1055Laboratory of Fundamental and Applied Bioenergetics, Grenoble, France.
authors.forename: Pierre-Yves
authors.initials: PY
authors.lastname: Benhamou
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D015374:Biosensing Techniques; D003362:Cost-Benefit Analysis; D003922:Diabetes Mellitus, Type 1 / Q000150:complications / Q000628:therapy*; D005260:Female; D005602:France; D006442:Glycated Hemoglobin / Q000378:metabolism; D006801:Humans; D007003:Hypoglycemia / Q000139:chemically induced; D007004:Hypoglycemic Agents / Q000008:administration & dosage*; D007332:Insulin Infusion Systems; D016381:Islets of Langerhans Transplantation / Q000191:economics* / Q000379:methods*; D008297:Male; D008875:Middle Aged; D011446:Prospective Studies; D012044:Regression Analysis; D012107:Research Design; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D006442:Glycated Hemoglobin; D007004:Hypoglycemic Agents
keywords: HEALTH ECONOMICS; Islet Transplantation; Sensor augmented pump therapy; Unstable type 1 Diabetes