Randomised, prospective, medico-economic nationwide French study of islet transplantation in patients with severely unstable type 1 diabetes: the STABILOT study protocol. Issue 2 (20th February 2017)

• Record Type: Journal Article
• Authors: Lablanche, Sandrine; David-Tchouda, Sandra; Margier, Jennifer; Schir, Edith; Wojtusciszyn, Anne; Borot, Sophie; Kessler, Laurence; Morelon, Emmanuel; Thivolet, Charles; Pattou, François; Vantyghem, Marie Christine; Berney, Thierry; Benhamou, Pierre-Yves
• Other Names: Benhamou Pierre Yves author non-byline.; Lablanche Sandrine author non-byline.; Tetaz Rachel author non-byline.; Borot Sophie author non-byline.; Tauveron Igor author non-byline.; Roche Béatrice author non-byline.; Berney Thierry author non-byline.; Pattou François author non-byline.; Vantyghem Marie-Christine author non-byline.; Benomar Kanza author non-byline.; Noel Christian author non-byline.; Thivolet Charles author non-byline.; Morelon Emmanuel author non-byline.; Badet Lionel author non-byline.; Buron Fanny author non-byline.; Wojtusciszyn Anne author non-byline.; Renard Eric author non-byline.; Frimat Luc author non-byline.; Girerd Sophie author non-byline.; Cantarovich Diego author non-byline.; Chaillous Lucy author non-byline.; Cattan Pierre author non-byline.; Riveline Jean-Pierre author non-byline.; Peraldi Marie-Noelle author non-byline.; Bourron Olivier author non-byline.; Kessler Laurence author non-byline.; Moreau François author non-byline.; Baltzinger Philippe author non-byline.; Bahougne Thibault author non-byline.; Bosco Domenico author non-byline.; Pernin Nadine author non-byline.; Egelhofer Harald author non-byline.; Moisan Anaick author non-byline.; Persoons Virginie author non-byline.; Kerr-Conte Julie author non-byline.; Gmyr Valery author non-byline.; Ezzouaoui Rimed author non-byline.; Armanet Mathieu author non-byline.;
• Is Part Of: BMJ open. Volume 7:Issue 2(2017)
• Publication Date: 2017-02-20
• Subjects: HEALTH ECONOMICS -- Sensor augmented pump therapy -- Islet Transplantation -- Unstable type 1 Diabetes; Medicine -- Research -- Periodicals; 610.72
• DOI: 10.1136/bmjopen-2016-013434
• Languages: English
• ISSNs: 2044-6055

Abstract

Abstract : Introduction: 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. Methods: 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. Ethics and dissemination: 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. Trial registration number: NCT02854696 ; Pre-results.