Abstract

The study objective was to evaluate the cost-effectiveness of allogenic pancreatic islet transplantation (PIT) compared to whole pancreas transplantation (WPT) in adult patients with brittle type 1 diabetes (T1DM) and eligible for β-cell replacement in UK. A life-time horizon Markov model was developed to reflect the progression of patients who undergo β-cell replacement. The cohort moves across 8 health states: transplant, insulin independent, insulin dependent without and with complete graft function loss, macrovascular complications, microvascular complications, macro- and microvascular complications, death. Acute adverse events were also considered. Parametric extrapolations of insulin independence and graft survival were performed based on data from the Collaborative Islet Transplantation Registry (CITR) and the International Pancreas Transplant Registry (IPTR). All other clinical and quality of life data were derived from literature. Costs from the 3rd party payer perspective were obtained from official sources and from the literature. Both costs and health outcomes, life years (LYs) and quality adjusted LYs (QALYs), were annually discounted at 3.5%. As expected, better overall survival and lower complication rates increased both LYs and QALYs gained in PIT vs WPT, resulting in incremental values of 1.15 and 0.62 respectively. PIT was also associated with an incremental cost of £18,132 vs WPT. The resulting cost per LY and QALY gained with PIT vs WPT were £15,784 and £29,281 respectively. Deterministic and probabilistic sensitivity analysis showed overall robustness of the basecase results, with 74% of simulations located below the threshold of £100,000/QALY, usually considered for the assessment of orphan drugs in UK. To our knowledge, this is the first model to assess PIT against WPT for the treatment of brittle T1DM. Recognizing limitations, mainly due to the lack of direct comparative evidence, PIT seems to represent a cost-effective option to WPT in UK.

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