TABLE 1.

Workshop session objectives

Indications for and Approaches to β-cell Replacement Therapy

Patient candidates and available forms of β-cell replacement vary. The goal of this session was to define those patient characteristics that directly influence the type of β-cell graft and measures of glycemic control and graft function, and so lay the framework for how definitions of successful outcomes may be tailored by indication.

Outcome Measures of Glucose Homeostasis

Regulation of glucose homeostasis involves the maintenance and return of glucose excursions to a nondiabetic range of glycemia. Various measures of glycemic control capture average glycemia, glycemic variability, and exposure to hyper- and hypoglycemia, as well as hypoglycemia awareness and severity. The goal of this session was to define successful outcomes for glycemic control, and align definitions with those used in the field of artificial insulin delivery/artificial pancreas development.

Outcome Measures of β-cell Graft Function and Demand

Measures of β-cell graft function may vary by the stimulus for secretion, differences in metabolic clearance, demands for secretion imposed by differences in insulin sensitivity or the use of insulin, as well as any possible residual native β-cell function. The goal of this session was to define a meaningful reduction in insulin requirements attributable to β-cell graft function, necessary confirmatory testing, relationship to standardized measures of glucose tolerance, and differences between type 1 and type 2 diabetic recipients.

Outcome Measures of Immunologic Mechanisms

Distinguishing immunologic from metabolic mechanisms for β-cell graft dysfunction and/or failure is paramount to understanding the mechanisms underlying current graft status and implications of functional β-cell graft monitoring. The goal of this session was to define useful assays of allo- and autoimmune reactivity and when they should be employed to complement the metabolic evaluation of β-cell replacement therapies.

Defining Successful Outcomes

Clear definitions for success or failure of available β-cell replacement therapies require incorporation of both metrics for glycemic control and β-cell graft function. The goal of this session was to establish a practical consensus definition for β-cell graft functional and efficacy outcomes for β-cell replacement therapies.