{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,10,11]],"date-time":"2025-10-11T13:33:52Z","timestamp":1760189632613,"version":"3.37.3"},"reference-count":68,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2018,4,20]],"date-time":"2018-04-20T00:00:00Z","timestamp":1524182400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100006021","name":"US Immunodeficiency Network","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100006021","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Vox Sanguinis"],"published-print":{"date-parts":[[2018,7]]},"abstract":"Background and Objectives<\/jats:title>Our research aim is to model latent therapeutic demand (LTD<\/jats:styled-content>) for the immunoglobulin replacement therapy (IgGRT<\/jats:styled-content>) of primary immune deficiency disorders (PIDD<\/jats:styled-content>s) in the USA<\/jats:styled-content>. Given the high level of variability of IgGRT<\/jats:styled-content> use and major differences among American and European practices in the management of patients with PIDD<\/jats:styled-content>s, we develop a USA<\/jats:styled-content>\u2010specific LTD<\/jats:styled-content> model for common variable immune deficiency (CVID<\/jats:styled-content>), hyper IGM<\/jats:styled-content> syndrome, severe combined immune deficiency, Wiskott\u2013Aldrich syndrome and X\u2010linked agammaglobulinemia (XLA<\/jats:styled-content>).<\/jats:p><\/jats:sec>Methods and Materials<\/jats:title>We use decision analysis methods to model the underlying IgGRT<\/jats:styled-content> demand for PIDD<\/jats:styled-content>s by assessing USA<\/jats:styled-content>\u2010specific epidemiology and treatment. Data for the epidemiology and treatment variables were obtained from the medical literature, USIDNET<\/jats:styled-content> and Immune Deficiency Foundation. The uncertainty surrounding the variables was modelled using probability distributions and evaluated using Monte Carlo simulation.<\/jats:p><\/jats:sec>Results<\/jats:title>The mean treatment dose from USIDNET<\/jats:styled-content> and European Society for Immunodeficiencies (ESID<\/jats:styled-content>) was significantly different for treating CVID<\/jats:styled-content>, and the number of annual infusions from USIDNET<\/jats:styled-content> and ESID<\/jats:styled-content> was significantly different for treating CVID<\/jats:styled-content> and XLA<\/jats:styled-content>. The mean and standard deviation of LTD<\/jats:styled-content> for all PIDD<\/jats:styled-content>s is 105\u00b71\u00a0\u00b1\u00a088\u00b75\u00a0g per 1000 population, with CVID<\/jats:styled-content> contributing the most to LTD<\/jats:styled-content>.<\/jats:p><\/jats:sec>Conclusion<\/jats:title>Estimating country\u2010specific LTD<\/jats:styled-content> is important to ensure an adequate supply of IgGRT<\/jats:styled-content> and an optimal treatment for patients with PIDD<\/jats:styled-content>s and for improving national healthcare policymaking and production planning.<\/jats:p><\/jats:sec>","DOI":"10.1111\/vox.12651","type":"journal-article","created":{"date-parts":[[2018,4,20]],"date-time":"2018-04-20T08:37:03Z","timestamp":1524213423000},"page":"430-440","source":"Crossref","is-referenced-by-count":5,"title":["Latent therapeutic demand model for the immunoglobulin replacement therapy of primary immune deficiency disorders in the USA<\/scp>"],"prefix":"10.1111","volume":"113","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-2856-329X","authenticated-orcid":false,"given":"J. 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