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Erschienen in: Immunologic Research 3/2017

21.02.2017 | Original Article

Modeling strategy to identify patients with primary immunodeficiency utilizing risk management and outcome measurement

verfasst von: Vicki Modell, Jessica Quinn, Grant Ginsberg, Ron Gladue, Jordan Orange, Fred Modell

Erschienen in: Immunologic Research | Ausgabe 3/2017

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Abstract

This study seeks to generate analytic insights into risk management and probability of an identifiable primary immunodeficiency defect. The Jeffrey Modell Centers Network database, Jeffrey Modell Foundation’s 10 Warning Signs, the 4 Stages of Testing Algorithm, physician-reported clinical outcomes, programs of physician education and public awareness, the SPIRIT® Analyzer, and newborn screening, taken together, generates P values of less than 0.05%. This indicates that the data results do not occur by chance, and that there is a better than 95% probability that the data are valid. The objectives are to improve patients’ quality of life, while generating significant reduction of costs. The advances of the world’s experts aligned with these JMF programs can generate analytic insights as to risk management and probability of an identifiable primary immunodeficiency defect. This strategy reduces the uncertainties related to primary immunodeficiency risks, as we can screen, test, identify, and treat undiagnosed patients. We can also address regional differences and prevalence, age, gender, treatment modalities, and sites of care, as well as economic benefits. These tools support high net benefits, substantial financial savings, and significant reduction of costs. All stakeholders, including patients, clinicians, pharmaceutical companies, third party payers, and government healthcare agencies, must address the earliest possible precise diagnosis, appropriate intervention and treatment, as well as stringent control of healthcare costs through risk assessment and outcome measurement. An affected patient is entitled to nothing less, and stakeholders are responsible to utilize tools currently available. Implementation offers a significant challenge to the entire primary immunodeficiency community.
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Literatur
1.
Zurück zum Zitat National Academies of Sciences, Engineering, and Medicine. How modeling can inform strategies to improve population health: workshop summary. Washington, DC: The National Academies Press; 2016. doi:10.17226/21807. National Academies of Sciences, Engineering, and Medicine. How modeling can inform strategies to improve population health: workshop summary. Washington, DC: The National Academies Press; 2016. doi:10.​17226/​21807.
2.
Zurück zum Zitat Gass SI, Fu MC. Encyclopedia of Operations Research and Management Science. 3rd ed. US: Springer; 2013. Gass SI, Fu MC. Encyclopedia of Operations Research and Management Science. 3rd ed. US: Springer; 2013.
3.
Zurück zum Zitat Modell V. The impact of physician education and public awareness on early diagnosis of primary immunodeficiencies. Immunol Res. 2007;38:43–7.CrossRefPubMed Modell V. The impact of physician education and public awareness on early diagnosis of primary immunodeficiencies. Immunol Res. 2007;38:43–7.CrossRefPubMed
4.
Zurück zum Zitat Modell F. Immunology today and new discoveries: building upon legacies of Dr. Robert A. Good. Immunol Res. 2007;38:48–50.CrossRefPubMed Modell F. Immunology today and new discoveries: building upon legacies of Dr. Robert A. Good. Immunol Res. 2007;38:48–50.CrossRefPubMed
5.
Zurück zum Zitat Cunningham-Rundles C, Ponda PP. Molecular defects in T- and B-cell primary immunodeficiency diseases. Nat Rev Immunol. 2005;5:880–92.CrossRefPubMed Cunningham-Rundles C, Ponda PP. Molecular defects in T- and B-cell primary immunodeficiency diseases. Nat Rev Immunol. 2005;5:880–92.CrossRefPubMed
6.
Zurück zum Zitat Cooper MA, Pommering TL, Koranyi K. Primary immunodeficiencies. Am Fam Physician. 2003;68:2001–8.PubMed Cooper MA, Pommering TL, Koranyi K. Primary immunodeficiencies. Am Fam Physician. 2003;68:2001–8.PubMed
7.
Zurück zum Zitat Bousfiha A, Jeddane L, Al-Herz W, Ailal F, Casanova JL, Chatila T, Conley ME, Cunningham-Rundles C, Etzioni A, Franco JL, Gaspar HB, Holland SM, Klein C, Nonoyam S, Ochs HD, Oksenhendler E, Picard C, Puck JM, Sullivan K, Tang MLK. The 2015 IUIS phenotypic classification for primary immunodeficiencies. J Clin Immunol. 2015; doi:10.1007/s10875-015-0198-5. (Updated) Bousfiha A, Jeddane L, Al-Herz W, Ailal F, Casanova JL, Chatila T, Conley ME, Cunningham-Rundles C, Etzioni A, Franco JL, Gaspar HB, Holland SM, Klein C, Nonoyam S, Ochs HD, Oksenhendler E, Picard C, Puck JM, Sullivan K, Tang MLK. The 2015 IUIS phenotypic classification for primary immunodeficiencies. J Clin Immunol. 2015; doi:10.​1007/​s10875-015-0198-5. (Updated)
8.
Zurück zum Zitat Picard C, Al-Herz W, Bousfiha A, Casanova JL, Chatila T, Conley ME, Cunningham-Rundles C, Etzioni A, Holland SM, Klein C, Nonoyama S, Ochs HD, Oksenhendler E, Puck J, Sullivan KE, Tang MLK, Franco JL, Gaspar HB. Primary immunodeficiency diseases: an update on the classification from the International Union of Immunological Societies Expert Committee for primary immunodeficiency 2015. J Clin Immunol. 2015; doi:10.1007/s10875-015-0201-1. Picard C, Al-Herz W, Bousfiha A, Casanova JL, Chatila T, Conley ME, Cunningham-Rundles C, Etzioni A, Holland SM, Klein C, Nonoyama S, Ochs HD, Oksenhendler E, Puck J, Sullivan KE, Tang MLK, Franco JL, Gaspar HB. Primary immunodeficiency diseases: an update on the classification from the International Union of Immunological Societies Expert Committee for primary immunodeficiency 2015. J Clin Immunol. 2015; doi:10.​1007/​s10875-015-0201-1.
9.
Zurück zum Zitat Hammarström L. Professor and Director, Division of Clinical Immunology, Karolinska Institute, Karolinska University Hospital, Huddinge, Sweden. Presentation to European Union (EU) Parliament. 2004. Hammarström L. Professor and Director, Division of Clinical Immunology, Karolinska Institute, Karolinska University Hospital, Huddinge, Sweden. Presentation to European Union (EU) Parliament. 2004.
10.
Zurück zum Zitat U.S. Cancer Statistics Working Group. United States Cancer Statistics: 1999–2013 Incidence and Mortality Web-based Report. Atlanta: U.S. Department of Health and Human Services, Centers for Disease Control and Prevention and National Cancer Institute. 2016. Available at: www.cdc.gov/uscs. U.S. Cancer Statistics Working Group. United States Cancer Statistics: 1999–2013 Incidence and Mortality Web-based Report. Atlanta: U.S. Department of Health and Human Services, Centers for Disease Control and Prevention and National Cancer Institute. 2016. Available at: www.​cdc.​gov/​uscs.
12.
Zurück zum Zitat Bharat ST, Alizadehfar R, Desrosiers M, Shuster J, Pant N, Tsoukasa CM. Adult primary immune deficiency: what are We missing? Am J Med. 2012;125(8):779–86.CrossRef Bharat ST, Alizadehfar R, Desrosiers M, Shuster J, Pant N, Tsoukasa CM. Adult primary immune deficiency: what are We missing? Am J Med. 2012;125(8):779–86.CrossRef
13.
Zurück zum Zitat Ochs H, Smith E, Puck J. PID diseases, genetic approach to CVID and IgA deficiency. New York, NY: Oxford Press, 1999. Ochs H, Smith E, Puck J. PID diseases, genetic approach to CVID and IgA deficiency. New York, NY: Oxford Press, 1999.
14.
Zurück zum Zitat Stiehm R, Ochs H, Winkelstein J. Immunologic disorders in infants and children. Philadelphia, PA: Elsevier Saunders Press. 2004. Stiehm R, Ochs H, Winkelstein J. Immunologic disorders in infants and children. Philadelphia, PA: Elsevier Saunders Press. 2004.
16.
Zurück zum Zitat U.S. Centers for Disease Control and Prevention. Office of Genomics and Disease Prevention. Applying Genetics and Public Health Strategies to Primary Immunodeficiency Diseases. 2001. Published proceedings. U.S. Centers for Disease Control and Prevention. Office of Genomics and Disease Prevention. Applying Genetics and Public Health Strategies to Primary Immunodeficiency Diseases. 2001. Published proceedings.
17.
18.
Zurück zum Zitat Modell V, Gee B, Lewis DB, Orange JS, Roifman CM, Routes JM, Sorensen RU, Notarangelo LD, Modell F. Global study of primary immunodeficiency diseases (PI)—diagnosis, treatment, and economic impact: an updated report from the Jeffrey Modell Foundation. Immunol Res. 2011;51:61–70.CrossRefPubMed Modell V, Gee B, Lewis DB, Orange JS, Roifman CM, Routes JM, Sorensen RU, Notarangelo LD, Modell F. Global study of primary immunodeficiency diseases (PI)—diagnosis, treatment, and economic impact: an updated report from the Jeffrey Modell Foundation. Immunol Res. 2011;51:61–70.CrossRefPubMed
19.
Zurück zum Zitat Modell F, Puente D, Modell V. From genotype to phenotype. Further studies measuring the impact of a physician education and public awareness campaign on early diagnosis and management of primary immunodeficiencies. Immunol Res. 2009;44(1–3):132–49.CrossRefPubMed Modell F, Puente D, Modell V. From genotype to phenotype. Further studies measuring the impact of a physician education and public awareness campaign on early diagnosis and management of primary immunodeficiencies. Immunol Res. 2009;44(1–3):132–49.CrossRefPubMed
24.
Zurück zum Zitat Bundorf KM, Royalty A, Baker LC. Health care cost growth among the privately insured. Health Aff. 2009;28(5):1294–304.CrossRef Bundorf KM, Royalty A, Baker LC. Health care cost growth among the privately insured. Health Aff. 2009;28(5):1294–304.CrossRef
29.
Zurück zum Zitat Fronstin P. Sources of health insurance and characteristics of the uninsured: analysis of the March 2013 current population survey, vol 390. EBRI (Employee Benefit Research Institute) Issue Brief No. 390, September 2013, Washington, DC; 2013. p. 1–36. Fronstin P. Sources of health insurance and characteristics of the uninsured: analysis of the March 2013 current population survey, vol 390. EBRI (Employee Benefit Research Institute) Issue Brief No. 390, September 2013, Washington, DC; 2013. p. 1–36.
30.
Zurück zum Zitat Chan K, Davis J, Pai SY, Bonilla FA, Puck JM, Apkon M. A Markov model to analyze cost-effectiveness of screening for severe combined immunodeficiency (SCID). Mol Genet Metab. 2011;104(3):383–9.CrossRefPubMedPubMedCentral Chan K, Davis J, Pai SY, Bonilla FA, Puck JM, Apkon M. A Markov model to analyze cost-effectiveness of screening for severe combined immunodeficiency (SCID). Mol Genet Metab. 2011;104(3):383–9.CrossRefPubMedPubMedCentral
32.
Zurück zum Zitat Menzin J, Sussman M, Munsell M, Zbrozek A. Economic impact of infections among patients with primary immunodeficiency disease receiving IVIG therapy. Clinicoecon Outcomes Res. 2014;6:297–302.CrossRefPubMedPubMedCentral Menzin J, Sussman M, Munsell M, Zbrozek A. Economic impact of infections among patients with primary immunodeficiency disease receiving IVIG therapy. Clinicoecon Outcomes Res. 2014;6:297–302.CrossRefPubMedPubMedCentral
Metadaten
Titel
Modeling strategy to identify patients with primary immunodeficiency utilizing risk management and outcome measurement
verfasst von
Vicki Modell
Jessica Quinn
Grant Ginsberg
Ron Gladue
Jordan Orange
Fred Modell
Publikationsdatum
21.02.2017
Verlag
Springer US
Erschienen in
Immunologic Research / Ausgabe 3/2017
Print ISSN: 0257-277X
Elektronische ISSN: 1559-0755
DOI
https://doi.org/10.1007/s12026-017-8907-1

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