Type 1 Diabetes
Conditions
Keywords
Islet cell Transplantation, cost-effectiveness analysis, Cost-utility analysis, Type 1 Diabetes
Brief summary
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.
Detailed description
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.
Interventions
PROCEDUREIslet graft
Patients will be transplanted with pancreatic islet cells
Patients will continue their insulin treatment
Sponsors
University Hospital, Grenoble
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
: * Suffering from diabetes since more than 5 years * Patient with brittle type 1 diabetes despite an optimized insulin treatment and educational training will be included. A patient will be considered as experiencing a brittle type 1 diabetes if at least two criteria are present among: persistence of severe hypoglycemia, occurrence of ketoacidosis events without obvious etiology, diagnosis of unaware hypoglycemic episodes \< 3 mmol/l based on CGM or self-monitoring blood glucose data, a mean blood glucose standard deviation\>50%, MAGE index (Mean amplitude of glucose excursions)\>60 mg/dl, LBGI index (low blood glucose index)\>5, Clarke score≥4 or HYPOSCORE\>800. * Insulin needs \< 0,85 U/kg/day * HbA1c \< 12% ; * No residual insulin secretion (plasmatic basal and stimulated C-peptide \< 0.3 ng/ml) * Social Security membership or benefit from Social Security * Patients who signed the consent form
Exclusion criteria
:
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incremental cost- utility ratio at 1 year | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Assessment of individual medical benefit of quality of life | 6 months and 1year | Evaluate with DQOL questionnaire |
| Assessment of individual medical benefit in terms of metabolic efficacy | 6 months and 1 year | measured from the following criteria: severe hypoglycemia, HbA1c, stimulated C-peptide, fasting glucose, insulin dose or oral diabetes, glycemic variability |
| Assessment and comparison of individual medical benefit in terms of complications of islet cell transplantation between the two groups | 6 months and 1 year | measured from in insulin independence, hospitalizations |
| Cost-effectiveness ratio at 1 year | 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 |
| Assessment and comparison of costs for patients with brittle type 1 diabetes with impairment of vital prognosis before and after islet cell transplantation | 1 year | measured from hospitalizations |
| Assessment of total cost of islet cell transplantation | 1 year | 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. |
| Assessment and comparison of clinical benefit for patients with brittle type 1 diabetes with impairment of vital prognosis before and after islet cell transplantation | 1 year | measured from DQOL, insulin independence, complications of islet cell transplantation |
Countries
France, Switzerland
Outcome results
None listed