Modeling primary immunodeficiency disease epidemiology and its treatment to estimate latent therapeutic demand for immunoglobulin

Journal of Clinical Immunology

Volumn 34, Issue 2, 2014, Pages 233-244

  Stonebraker, Jeffrey S ^a   Farrugia, Albert ^b,c   Gathmann, Benjamin ^d,e   Orange, Jordan S ^f,g  

^a NORTH CAROLINA STATE UNIVERSITY   (United States)

^b Plasma Protein Therapeutics Association   (United States)

^c UNIVERSITY OF WESTERN AUSTRALIA   (Australia)

^d UNIVERSITY OF FREIBURG   (Germany)

^e UNIVERSITY OF FREIBURG   (Germany)

^f TEXAS CHILDREN S HOSPITAL   (United States)

^g BAYLOR COLLEGE OF MEDICINE   (United States)

Author keywords

decision analysis; epidemiology; latent therapeutic demand; Primary immune deficiency; treatment

Indexed keywords

IMMUNOGLOBULIN;

ADOLESCENT; ADULT; AGED; ARTICLE; AUSTRALIA; BODY WEIGHT; CANADA; CHILD; COMMON VARIABLE IMMUNODEFICIENCY; CONTROLLED STUDY; DRUG USE; FEMALE; FRANCE; GERMANY; HEALTH CARE NEED; HUMAN; IMMUNOTHERAPY; INTRAVENOUS IMMUNOTHERAPY; IRELAND; ISRAEL; JAPAN; LATENT THERAPEUTIC DEMAND; MAJOR CLINICAL STUDY; MALE; MODEL; NEW ZEALAND; NORWAY; PATIENT COMPLIANCE; PREVALENCE; PRIORITY JOURNAL; SEX DIFFERENCE; SPAIN; SUBCUTANEOUS IMMUNOTHERAPY; UNITED KINGDOM; UNITED STATES; X LINKED AGAMMAGLOBULINEMIA;

ADOLESCENT; ADULT; AGAMMAGLOBULINEMIA; AGED; AGED, 80 AND OVER; CHILD; CHILD, PRESCHOOL; COMMON VARIABLE IMMUNODEFICIENCY; FEMALE; GENETIC DISEASES, X-LINKED; HUMANS; IMMUNOGLOBULINS; IMMUNOLOGIC DEFICIENCY SYNDROMES; MALE; MIDDLE AGED; MODELS, STATISTICAL; MONTE CARLO METHOD; PREVALENCE; YOUNG ADULT;

EID: 84898640926     PISSN: 02719142     EISSN: 15732592     Source Type: Journal    
DOI: 10.1007/s10875-013-9975-1     Document Type: Article

References (68)

1. Shehata N, Palda V, Bowen T, Haddad E, Issekutz TB, Mazer B, et al. The use of immunoglobulin therapy for patients with primary immune deficiency: an evidence-based practice guideline. Transfus Med Rev. 2010;24 Suppl 1:S28-50.
2. Wood P. Immunotherapy for primary immunodeficiency diseases. Med Clin N Am. 2012;96(3):433-54.
3. Eibl MM. History of immunoglobulin replacement. Immunol Allergy Clin N Am. 2008;28(4):737-64.
4. Hooper JA. Intravenous immunoglobulins: evolution of commercial IVIG preparations. Immunol Allergy Clin N Am. 2008;28(4):765-78.
5. Buchacher A, Iberer G. Purification of intravenous immunoglobulin G from human plasma - aspects of yield and virus safety. Biotechnol J. 2006;1(2):148-63.
6. Jolles S, Sewell W, Misbah S. Clinical uses of intravenous immunoglobulin. Clin Exp Immunol. 2005;142(1):1-11.
7. Robert P. IVIG/SCIG: GLOBAL USAGE TRENDS. 2011. http://www.ipopi.org/ uploads/Patrick%20Robert.pdf. Accessed 14 May 2013.
8. Orange JS, Hossny EM, Weiler CR, Ballow M, Berger M, Bonilla FA, et al. Use of intravenous immunoglobulin in human disease: a review of evidence by members of the primary immunodeficiency committee of the American academy of allergy, asthma and immunology. J Allergy Clin Immunol. 2006;117(4 Suppl):S525-53.
9. Australian National Blood Authority. Criteria for the clinical use of intravenous immunoglobulin in Australia, 2nd ed. http://www.blood.gov.au/pubs/ ivig/index.html#/ivig/pdf/criteria.pdf. Accessed 1 August 2013.
10. Australian National Blood Authority. Issues of IVIg (grams) per 1000 head of population, 2007-08 to 2011-12. 2011. http://www.nba.gov.au/publications/ 1112report/chapter03/3.2.html#figure3-17. Accessed 1 August 2013.
11. Farrugia A, Cassar J. Is self-sufficiency in haemotherapies a practical or necessary goal? Blood Transfus. 2012;13:1-12.
12. Rossi F, Perry R, de Wit J, Evers T, Folléa G. How expanding voluntary non-remunerated blood donations would benefit patients, donors and healthcare systems? Vox Sang. 2011;101(2):176-7.
13. Joshi AY, Iyer VN, Hagan JB, St Sauver JL, Boyce TG. Incidence and temporal trends of primary immunodeficiency: a population-based cohort study. Mayo Clin Proc. 2009;84(1):16-22.
14. Orange JS, Grossman WJ, Navickis RJ, Wilkes MM. Impact of trough IgG on pneumonia incidence in primary immunodeficiency: a meta-analysis of clinical studies. Clin Immunol. 2010;137(1):21-30.
15. Goel V. Decision analysis: applications and limitations. The Health Services Research Group. CMAJ. 1992;147(4):413-7.
16. Stonebraker JS, Amand RE, Bauman MV, Nagle AJ, Larson PJ. Modelling haemophilia epidemiology and treatment modalities to estimate the unconstrained factor VIII demand. Haemophilia. 2004;10(1):18-26.
17. Stonebraker JS, Keefer DL. Modeling potential demand for supply-constrained drugs: a new hemophilia drug at Bayer Biological Products. Oper Res. 2009;57(1):19-31.
18. European Society for Immunodeficiencies Registry (ESID). 2011. http://www.esid.org. Accessed September 14, 2011.
19. Gathmann B, Grimbacher B, Beauté J, Dudoit Y, Mahlauoui N, Fischer A, et al. The European internet-based patient and research database for primary immunodeficiencies: results 2006-2008. Clin Exp Immunol. 2009;157 Suppl 1:3-11.
20. Immune Deficiency Foundation: Primary immune deficiency diseases in America. Prepared by Schulman, Ronca and Bucuvalas, Inc., 1995.
21. Immune Deficiency Foundation: Primary immune deficiency diseases in America: 2002. The second national survey of patients. Prepared by Schulman, Ronca and Bucuvalas, Inc., April 28, 2003
22. Immune Deficiency Foundation: Treatment experiences and preferences of patients with primary immune deficiency diseases: First national survey. Prepared by Schulman, Ronca and Bucuvalas, Inc., June 20, 2003.
23. Immune Deficiency Foundation: Primary immunodeficiency diseases in America: 2007. The third national survey of patients. Prepared by Abt SRBI, Inc., May 1, 2009.
24. Immune Deficiency Foundation: Treatment experiences and preferences among patients with primary immunodeficiency diseases. National survey of patients: 2008. Prepared by Abt, SRBI, Inc., May 6, 2009.
25. Briggs AH, Weinstein MC, Fenwick EAL, Karnon J, Sculpher MJ, Paltiel AD, et al. Model parameter estimation and uncertainty analysis: a report of the ISPOR-SMDM modeling good research practices task force working group - 6. Med Decis Mak. 2012;32:722-32.
26. Keefer DL, Bodily SE. Three-point approximation for continuous random variables. Manag Sci. 1983;29:595-609.
27. Park MA, Li JT, Hagan JB, Maddox DE, Abraham RS. Common variable immunodeficiency: a new look at an old disease. Lancet. 2008;372(9637):489-502.
28. Yong PFK, Tarzi M, Chua I, Grimbacher B, Chee R. Common variable immunodeficiency: an update on etiology and management. Immunol Allergy Clin N Am. 2008;28(2):367-86.
29. World Population Prospects: The 2012 Revision. In: United Nations, Department of Economic and Social Affairs, Population Division. 2013. http://esa.un.org/wpp/unpp/panel-indicators.htm. Accessed July 10, 2013.
30. Abuzakouk M, Feighery C. Primary immunodeficiency disorders in the Republic of Ireland: first report of the National Registry in children and adults. J Clin Immunol. 2005;25(1):73-7.
31. Beauté J et al. The French national registry of primary immunodeficiency diseases. Clin Immunol. 2010;135:264-72.
32. Boyle JM, Buckley RH. Population prevalence of diagnosed primary immunodeficiency diseases in the United States. J Clin Immunol. 2007;27:497-502.
33. Edgar JDM, Buckland M, Guzman D, Conlon NP, Knerr V, et al. The United Kingdom Primary Immune Deficiency (UKPID) Registry: report of the first 4 years' activity 2008-2012. Clin Exp Immunol doi: 10.1111/cei.12172.
34. Gathmann B, Goldacker S, Klima M, et al. The German national registry for primary immunodeficiencies (PID). Clin Exp Immunol. 2013;173(2):372-80.
35. Golan H et al. The incidence of primary immunodeficiency syndromes in Israel. IMAJ. 2002;4(Suppl):868-71.
36. Ishimura M et al. Nationwide survey of patients with primary immunodeficiency diseases in Japan. J Clin Immunol. 2011;31:968-76.
37. Kirkpatrick P, Riminton S. Primary immunodeficiency diseases in Australia and New Zealand. J Clin Immunol. 2007;27(5):517-24.
38. Flori NM, Llambi JM, Boren TE, Borja SR, Casariego GF. Primary immunodeficiency syndrome in Spain: first report of the National Registry in children and adults. J Clin Immunol. 1997;17(4):333-9.
39. Modell V, Gee B, Lewis DB, Orange JS, Rolfman CM, Routes JM, et al. 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.
40. Resnick ES, Bhatt P, Sidi P, Cunningham-Rundles C. Examining the use of ICD-9 Diagnosis Codes for primary immune deficiency diseases in New York State. J Clin Immunol. 2013;33:40-8.
41. Ryser O, Morell A, Hitzig WH. Primary immunodeficiencies in Switzerland: first report of the National Registry in adults and children. J Clin Immunol. 1988;8(6):479-85.
42. Stray-Pedersen A, Abrahamsen TG, Frøland SS. Primary immunodeficiency diseases in Norway. J Clin Immunol. 2000;20(6):477-85.
43. Furst DE. Serum immunoglobulins and risk of infection: how long can you go? Semin Arthritis Rheum. 2009;39(1):18-29.
44. McDowell MA, Fryar CD, Ogden CL, Flegal KM. Anthropometric reference data for children and adults: United States, 2003-2006. National Health Statistics Reports: no. 10. Hyattsville: National Center for Health Statistics; 2008.
45. Klein RJ, Schoenborn CA. Age adjustment using the 2000 projected U.S. population. Healthy People Statistics notes, no. 20. Hyattsville: National Center for Health Statistics; 2001.
46. Ballow M, Notarangelo L, Grimbacher B, Cunningham-Rundles C, Stein M, Helber M, et al. Immunodeficiencies. Clin Exp Immunol. 2009;158 Suppl 1:14-22.
47. Yong PL, Boyle J, Ballow M, Boyle M, Berger M, Bleesing J, et al. Use of intravenous immunoglobulin and adjunctive therapies in the treatment of primary immunodeficiencies: a working group report of and study by the Primary Immunodeficiency Committee of the American Academy of Allergy Asthma and Immunology. Clin Immunol. 2010;135(2):255-63.
48. Hernandez-Trujillo HS, Chapel H, Lo Re 3rd V, Notarangelo LD, Gathmann B, Grimbacher B, et al. Comparison of American and European practices in the management of patients with primary immunodeficiencies. Clin Exp Immunol. 2012;169(1):57-69.
49. Lucas M, Lee M, Lortan J, Lopez-Granados E, Misbah S, Chapel H. Infection outcomes in patients with common variable immunodeficiency disorders: relationship to immunoglobulin therapy over 22 years. J Allergy Clin Immunol. 2010;125(6):1354-60.
50. Farrugia A, Cassar J. Plasma-derived medicines: access and usage issues. Blood Transfus. 2012;10(3):273-8.
51. National Health Service. Demand Management Programme for Immunoglobulin. 2009. http://www.ivig.nhs.uk. Accessed 14 May 2013.
52. Elovaara I, Apostolski S, van Doorn P, Gilhus NE, Hietaharju A, Honkaniemi J, et al. EFNS guidelines for the use of intravenous immunoglobulin in treatment of neurological diseases: EFNS task force on the use of intravenous immunoglobulin in treatment of neurological diseases. Eur J Neurol. 2008;15(9):893-908.
53. Patwa HS, Chaudhry V, Katzberg H, Rae-Grant AD, So YT. Evidence-based guideline: intravenous immunoglobulin in the treatment of neuromuscular disorders: report of the Therapeutics and Technology Assessment Subcommittee of the American Academy of Neurology. Neurology. 2012;78(13):1009-15.
54. Bonilla FA, Bernstein IL, Khan DA, Ballas ZK, Chinen J, Frank MM, et al. Practice parameter for the diagnosis and management of primary immunodeficiency. Ann Allergy Asthma Immunol. 2005;94(5 Suppl 1):S1-S63.
55. Roifman CM, Levison H, Gelfand EW. High-dose versus low-dose intravenous immunoglobulin in hypogammaglobulinaemia and chronic lung disease. Lancet. 1987;1(8541):1075-7.
56. Liese JG, Wintergerst U, Tympner KD, Belohradsky BH. High- vs low-dose immunoglobulin therapy in the long-term treatment of X-linked agammaglobulinemia. Am J Dis Child. 1992;146(3):335-9.
57. Eijkhout HW, van Der Meer JW, Kallenberg CG, Weening RS, van Dissel JT, Sanders LA, et al. The effect of two different dosages of intravenous immunoglobulin on the incidence of recurrent infections in patients with primary hypogammaglobulinemia. A randomized, double-blind, multicenter crossover trial. Ann Intern Med. 2001;135(3):165-74.
58. Berger M. Incidence of infection is inversely related to steady-state (Trough) serum IgG level in studies of subcutaneous IgG in PIDD. J Clin Immunol. 2011;31(5):924-6.
59. Quinti I, Soresina A, Guerra A, Rondelli R, Spadaro G, Agostini C, et al. Effectiveness of immunoglobulin replacement therapy on clinical outcome in patients with primary antibody deficiencies: results from a multicenter prospective cohort study. J Clin Immunol. 2011;31(3):315-22.
60. Carsetti R, Rosado MM, Donnanno S, Guazzi V, Soresina A, Meini A, et al. The loss of IgM memory B cells correlates with clinical disease in common variable immunodeficiency. J Allergy Clin Immunol. 2005;115(2):412-7.
61. Chapel H, Lucas M, Lee M, Bjorkander J, Webster D, Grimbacher B, et al. Common variable immunodeficiency disorders: division into distinct clinical phenotypes. Blood. 2008;112(2):277-86.
62. Chapel H, Cunningham-Rundles C. Update in understanding common variable immunodeficiency disorders (CVIDs) and the management of patients with these conditions. Br J Haematol. 2009;145(6):709-27.
63. Chapel H, Lucas M, Patel S, Lee M, Cunningham-Rundles C, Resnick E. Confirmation and improvement of criteria for clinical phenotyping in common variable immunodeficiency disorders in replicate cohorts. J Allergy Clin Immunol. 2012;130(5):1197-1198e9.
64. Orange JS, Ballow M, Stiehm ER, Ballas ZK, Chinen J, De La Morena M, et al. Use and interpretation of diagnostic vaccination in primary immunodeficiency: a working group report of the Basic and Clinical Immunology Interest Section of the American Academy of Allergy, Asthma & Immunology Genome-wide association identifies diverse causes of common variable immunodeficiency. J Allergy Clin Immunol. 2012;130:S1-S24.
65. Wehr C, Kivioja T, Schmitt C, Ferry B, Witte T, Eren E, et al. The EUROclass trial: defining subgroups in common variable immunodeficiency. Blood. 2008;111(1):77-85.
66. Orange JS, Glessner JT, Resnick E, Sullivan KE, Lucas M, Ferry B, et al. Genome-wide association identifies diverse causes of common variable immunodeficiency. J Allergy Clin Immunol. 2011;127:1360-7.
67. Stonebraker JS, Farrugia A, Gathmann B. A decision analysis model to estimate latent therapeutic demand for immunoglobulin therapy in primary immunodeficiencies. Proceedings of the 15th Meeting of the European Society of Immunodeficiencies - ESID (October 3-6, 2012, Florence, Italy). 2013;29-34.
68. Schöndorf I. Resolving barriers to the treatment of primary immunodeficiencies. The Source. 2011;19-20.