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Latent therapeutic demand model for the immunoglobulin replacement therapy of primary immune deficiency disorders in the USA
Author(s) -
Stonebraker J. S.,
Hajjar J.,
Orange J. S.
Publication year - 2018
Publication title -
vox sanguinis
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.68
H-Index - 83
eISSN - 1423-0410
pISSN - 0042-9007
DOI - 10.1111/vox.12651
Subject(s) - common variable immunodeficiency , medicine , epidemiology , immunology , population , immune system , primary immunodeficiency , antibody , pediatrics , environmental health
Background and Objectives Our research aim is to model latent therapeutic demand ( LTD ) for the immunoglobulin replacement therapy (Ig GRT ) of primary immune deficiency disorders ( PIDD s) in the USA . Given the high level of variability of Ig GRT use and major differences among American and European practices in the management of patients with PIDD s, we develop a USA ‐specific LTD model for common variable immune deficiency ( CVID ), hyper IGM syndrome, severe combined immune deficiency, Wiskott–Aldrich syndrome and X‐linked agammaglobulinemia ( XLA ). Methods and Materials We use decision analysis methods to model the underlying Ig GRT demand for PIDD s by assessing USA ‐specific epidemiology and treatment. Data for the epidemiology and treatment variables were obtained from the medical literature, USIDNET and Immune Deficiency Foundation. The uncertainty surrounding the variables was modelled using probability distributions and evaluated using Monte Carlo simulation. Results The mean treatment dose from USIDNET and European Society for Immunodeficiencies ( ESID ) was significantly different for treating CVID , and the number of annual infusions from USIDNET and ESID was significantly different for treating CVID and XLA . The mean and standard deviation of LTD for all PIDD s is 105·1 ± 88·5 g per 1000 population, with CVID contributing the most to LTD . Conclusion Estimating country‐specific LTD is important to ensure an adequate supply of Ig GRT and an optimal treatment for patients with PIDD s and for improving national healthcare policymaking and production planning.