ADA Workshop Report

  • Palmer J
  • Fleming G
  • Greenbaum C
 et al. 
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11 The underlying cause of type 1 diabetes, loss of ␤-cell function, has become the therapeutic target for a num-ber of interventions in patients with type 1 diabetes. Even though insulin therapies continue to improve, it remains difficult to achieve normal glycemic control in type 1 diabetes, especially long term. The associated risks of hypoglycemia and end-organ diabetic complica-tions remain. Retention of ␤-cell function in patients with type 1 diabetes is known to result in improved glycemic control and reduced hypoglycemia, retinopa-thy, and nephropathy. To facilitate the development of therapies aimed at altering the type 1 diabetes disease process, an American Diabetes Association workshop was convened to identify appropriate efficacy outcome measures in type 1 diabetes clinical trials. The following consensus emerged: While measurements of immune responses to islet cells are important in elucidating pathogenesis, none of these measures have directly correlated with the decline in endogenous insulin secre-tion. HbA 1c is a highly valuable clinical measure of glycemic control, but it is an insensitive measure of ␤-cell function, particularly with the currently accepted standard of near-normal glycemic control. Rates of se-vere hypoglycemia and diabetic complications ulti-mately will be improved by therapies that are effective at preserving ␤-cell function but as primary outcomes require inordinately large and protracted trials. Endog-enous insulin secretion is assessed best by measure-ment of C-peptide, which is cosecreted with insulin in a one-to-one molar ratio but unlike insulin experiences little first pass clearance by the liver. Measurement of C-peptide under standardized conditions provides a sensitive, well accepted, and clinically validated assess-ment of ␤-cell function. C-peptide measurement is the most suitable primary outcome for clinical trials of therapies aimed at preserving or improving endogenous insulin secretion in type 1 diabetes patients. Available data demonstrate that even relatively modest treatment effects on C-peptide will result in clinically meaningful benefits. The development of therapies for addressing this important unmet clinical need will be facilitated by trials that are carefully designed with ␤-cell function as determined by C-peptide measurement as the primary efficacy outcome. Diabetes 53:250 –264, 2004 R

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  • Jerry P Palmer

  • G Alexander Fleming

  • Carla J Greenbaum

  • Kevan C Herold

  • Lisa D Jansa

  • Hubert Kolb

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