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The main goal is to perform a cost-utility analysis to compare islet cell transplantation versus best medical treatment (defined as Sensor augmented pump therapy) for patients with brittle type1 diabetes.
The main goal is to perform a cost-utility analysis to compare islet cell transplantation versus best medical treatment (defined as Sensor augmented insulin pump therapy) for patients with brittle type1 diabetes.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Islet graft | Experimental | Patients who receive islet graft Intervention : Procedure/Surgery |
|
| Best medical care | Active Comparator | Patients who continue their optimal medical treatment (insulin pump therapy coupled with real time continuous glucose monitoring) Intervention : insulin treatment |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Islet graft | Procedure | Patients will be transplanted with pancreatic islet cells |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incremental cost- utility ratio at 1 year | The primary endpoint will be the incremental cost-effectiveness ratio at one year for islet transplantation versus Best Medical Treatment of brittle type 1 diabetes.The effectiveness will be expressed as quality adjusted life years (QALYs) in a cost-utility analysis. QALYs are a composite measure of outcomes where utilities for health states (on 0-1 scale, where 0 corresponds to death and 1 to full health) act as qualitative weights to combine quantity and quality of life. The number of QALYs in each group will be assessed with the EuroQol 5 Dimensions questionnaire (EQ5D). The EQ-5D measures health status in terms of mobility, self-care, usual activities, pain/discomfort and anxiety/depression. | 1 year |
| Measure | Description | Time Frame |
|---|---|---|
| Cost-effectiveness ratio at 1 year | Assessment of the cost-effectiveness ratio at 1 year between islet cell transplantation versus best medical treatment (SAP therapy) for patients with brittle type 1 diabetes without impairment of vital prognosis. Two criteria of effectiveness will be used : the life years gained and the number of hypoglycemia | 1 year |
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Inclusion Criteria :
Exclusion Criteria :
Exclusion criteria related to islet infusion:
Exclusion criteria related to diabetic complications:
Exclusion criteria related to immunosuppressant use:
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| Name | Affiliation | Role |
|---|---|---|
| Pierre-Yves BENHAMOU, MD, PhD | University Hospital, Grenoble | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University Hospital of Besançon | Besançon | France | ||||
| university hospital of Clermont Ferrand |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 22723582 | Background | Barton FB, Rickels MR, Alejandro R, Hering BJ, Wease S, Naziruddin B, Oberholzer J, Odorico JS, Garfinkel MR, Levy M, Pattou F, Berney T, Secchi A, Messinger S, Senior PA, Maffi P, Posselt A, Stock PG, Kaufman DB, Luo X, Kandeel F, Cagliero E, Turgeon NA, Witkowski P, Naji A, O'Connell PJ, Greenbaum C, Kudva YC, Brayman KL, Aull MJ, Larsen C, Kay TW, Fernandez LA, Vantyghem MC, Bellin M, Shapiro AM. Improvement in outcomes of clinical islet transplantation: 1999-2010. Diabetes Care. 2012 Jul;35(7):1436-45. doi: 10.2337/dc12-0063. | |
| 21323736 |
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| best medical care | Drug | Patients will continue their insulin treatment |
|
|
| Assessment of individual medical benefit of quality of life | Evaluate with DQOL questionnaire | 6 months and 1year |
| Assessment of individual medical benefit in terms of metabolic efficacy | measured from the following criteria: severe hypoglycemia, HbA1c, stimulated C-peptide, fasting glucose, insulin dose or oral diabetes, glycemic variability | 6 months and 1 year |
| Assessment and comparison of individual medical benefit in terms of complications of islet cell transplantation between the two groups | measured from in insulin independence, hospitalizations | 6 months and 1 year |
| Assessment and comparison of clinical benefit for patients with brittle type 1 diabetes with impairment of vital prognosis before and after islet cell transplantation | measured from DQOL, insulin independence, complications of islet cell transplantation | 1 year |
| Assessment and comparison of costs for patients with brittle type 1 diabetes with impairment of vital prognosis before and after islet cell transplantation | measured from hospitalizations | 1 year |
| Assessment of total cost of islet cell transplantation | Assessment of total cost of islet cell transplantation for patients with type 1 diabetes without impairment of vital prognosis, from pre-transplant period until 1 year after the last injection. Two perspectives will be used: French health care system and hospital. | 1 year |
| Clermont-Ferrand |
| France |
| Grenoble University Hospital | Grenoble | 38000 | France |
| University hospital of Lille | Lille | 59000 | France |
| University Hospital of Lyon | Lyon | 69000 | France |
| University Hospital of Montpellier | Montpellier | 34000 | France |
| University hospital of Nancy | Nancy | 54511 | France |
| university hospital of Nantes | Nantes | 44093 | France |
| APHP | Paris | 75010 | France |
| University hospital of Strasbourg | Strasbourg | 67000 | France |
| University Hospital of Geneva | Geneva | CH-1211 | Switzerland |
| Background |
| Beckwith J, Nyman JA, Flanagan B, Schrover R, Schuurman HJ. A health economic analysis of clinical islet transplantation. Clin Transplant. 2012 Jan-Feb;26(1):23-33. doi: 10.1111/j.1399-0012.2011.01411.x. Epub 2011 Feb 16. |
| 18808408 | Background | Bellin MD, Kandaswamy R, Parkey J, Zhang HJ, Liu B, Ihm SH, Ansite JD, Witson J, Bansal-Pakala P, Balamurugan AN, Papas KK, Sutherland DE, Moran A, Hering BJ. Prolonged insulin independence after islet allotransplants in recipients with type 1 diabetes. Am J Transplant. 2008 Nov;8(11):2463-70. doi: 10.1111/j.1600-6143.2008.02404.x. Epub 2008 Sep 19. |
| 17627243 | Background | Badet L, Benhamou PY, Wojtusciszyn A, Baertschiger R, Milliat-Guittard L, Kessler L, Penfornis A, Thivolet C, Renard E, Bosco D, Morel P, Morelon E, Bayle F, Colin C, Berney T; GRAGIL Group. Expectations and strategies regarding islet transplantation: metabolic data from the GRAGIL 2 trial. Transplantation. 2007 Jul 15;84(1):89-96. doi: 10.1097/01.tp.0000268511.64428.d8. |
| 28219959 | Derived | Lablanche S, David-Tchouda S, Margier J, Schir E, Wojtusciszyn A, Borot S, Kessler L, Morelon E, Thivolet C, Pattou F, Vantyghem MC, Berney T, Benhamou PY. Randomised, prospective, medico-economic nationwide French study of islet transplantation in patients with severely unstable type 1 diabetes: the STABILOT study protocol. BMJ Open. 2017 Feb 20;7(2):e013434. doi: 10.1136/bmjopen-2016-013434. |
| ID | Term |
|---|---|
| D003922 | Diabetes Mellitus, Type 1 |
| ID | Term |
|---|---|
| D003920 | Diabetes Mellitus |
| D044882 | Glucose Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D004700 | Endocrine System Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
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| ID | Term |
|---|---|
| D016381 | Islets of Langerhans Transplantation |
| ID | Term |
|---|---|
| D017690 | Cell Transplantation |
| D064987 | Cell- and Tissue-Based Therapy |
| D001691 | Biological Therapy |
| D013812 | Therapeutics |
| D013507 | Endocrine Surgical Procedures |
| D013514 | Surgical Procedures, Operative |
| D014180 | Transplantation |
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