A decision analysis model to estimate latent therapeutic demand for immunoglobulin therapy in Primary Immunodeficiencies

摘要

Therapeutic human polyclonal immunoglobulin (Ig) has resulted in a significant enhancement in survival and quality of life in patients with primary immunodeficiencies (PID). An estimate of the potential demand for Ig in PID may contribute to adequate planning and access to therapy. In this study, the latent therapeutic demand (LTD) for Ig was studied for two PIDs – Common Variable Immunodeficiency (CVID) and X-Linked Agammaglobulinaemia (XLA). The epidemiological and treatment variables impacting LTD were identified and defined through the medical literature and their uncertainty was modeled using decision analysis. In addition, treatment related data were obtained from the Registry maintained by the European Society for Immunodeficiences (ESID). Estimates for LTD for CVID and XLA derived from the model were 44 and 48 grams of Ig per 1000 inhabitants, respectively. These results indicate that evidence-based latent demand for Ig in PID exceeds the current total Ig consumption of many countries, and emphasize the need for continuing efforts in identifying and treating PID.