Skip to main content
Top
Published in: Orphanet Journal of Rare Diseases 1/2018

Open Access 01-12-2018 | Research

Treatment patterns and healthcare resource utilization among patients with hereditary angioedema in the United States

Authors: Marc A Riedl, Aleena Banerji, Michael E Manning, Earl Burrell, Namita Joshi, Dipen Patel, Thomas Machnig, Ming-Hui Tai, Douglas J Watson

Published in: Orphanet Journal of Rare Diseases | Issue 1/2018

Login to get access

Abstract

Background

Real-world data on usage and associated outcomes with hereditary angioedema (HAE)-specific medications introduced to the United States (US) market since 2009 are very limited. The purpose of this retrospective study was to evaluate real-world treatment patterns of HAE-specific medications in the US and to assess their impact on healthcare resource utilization (HCRU). This analysis used IMS PharMetrics PlusTM database records (2006–2014) of patients with HAE, ≥1 insurance claim for an HAE-specific medication, and continuous insurance enrollment for ≥3 months following the first HAE prescription claim.

Results

Of 631 total patients, 434 (68.8%) reported C1-INH(IV) use; 396 (62.8%) reported using ecallantide and/or icatibant. There were 306 episodes of prophylactic use of C1-INH(IV) (defined by continuous refills averaging ≥1500 IU/week for ≥13 weeks) in 155 patients; use of ≥1 on-demand rescue medication was implicated during 53% (163/306) of those episodes. Sixty-eight (20.2%) of 336 C1-INH(IV) users eligible for the HCRU analysis were hospitalized at least once, and 191 (56.8%) visited the emergency department (ED). Eighteen patients (5.4%) had a central venous access device (CVAD); of these, 5 (27.7%) required hospitalization and 14 (77.7%) had an ED visit. The adjusted relative risk of hospitalization and/or ED visits for patients with a CVAD was 2.6 (95% CI: 0.17, 39.23) compared to C1-INH(IV) users without a CVAD.

Conclusions

Despite widespread availability of modern HAE medications in the US, we identified a subset of patients requiring long-term prophylaxis who continue to be burdened by frequent rescue medication usage and/or complications related to the use of CVADs for intravenous HAE medication.
Literature
1.
go back to reference Zuraw BL, Banerji A, Bernstein JA, Busse PJ, Christiansen SC, Davis-Lorton M, et al. US hereditary angioedema association medical advisory board 2013 recommendations for the management of hereditary angioedema due to C1 inhibitor deficiency. J Allergy Clin Immunol Pract. 2013;1:458–67.CrossRef Zuraw BL, Banerji A, Bernstein JA, Busse PJ, Christiansen SC, Davis-Lorton M, et al. US hereditary angioedema association medical advisory board 2013 recommendations for the management of hereditary angioedema due to C1 inhibitor deficiency. J Allergy Clin Immunol Pract. 2013;1:458–67.CrossRef
2.
go back to reference Aygören-Pürsün E, Magerl M, Maetzel A, Maurer M. Epidemiology of bradykinin-mediated angioedema: a systematic investigation of epidemiological studies. Orphanet J Rare Dis. 2018;13(1):73.CrossRefPubMedCentral Aygören-Pürsün E, Magerl M, Maetzel A, Maurer M. Epidemiology of bradykinin-mediated angioedema: a systematic investigation of epidemiological studies. Orphanet J Rare Dis. 2018;13(1):73.CrossRefPubMedCentral
3.
go back to reference Zanichelli A, Longhurst HJ, Maurer M, Bouillet L, Aberer W, Fabien V, IOS Study Group, et al. Misdiagnosis trends in patients with hereditary angioedema from the real-world clinical setting. Ann Allergy Asthma Immunol. 2016;117(4):394–8.CrossRefPubMedCentral Zanichelli A, Longhurst HJ, Maurer M, Bouillet L, Aberer W, Fabien V, IOS Study Group, et al. Misdiagnosis trends in patients with hereditary angioedema from the real-world clinical setting. Ann Allergy Asthma Immunol. 2016;117(4):394–8.CrossRefPubMedCentral
4.
go back to reference Jindal NL, et al. Hereditary angioedema: health related quality of life in Canadian patients as measured by the SF-36. Allergy Asthma Clin Immunol. 2017;13:4.CrossRefPubMedCentral Jindal NL, et al. Hereditary angioedema: health related quality of life in Canadian patients as measured by the SF-36. Allergy Asthma Clin Immunol. 2017;13:4.CrossRefPubMedCentral
5.
go back to reference Longhurst H, Bygum A. The humanistic, societal, and Pharmaco-economic burden of angioedema. Clinic Rev Allerg Immunol. 2016;51:230–9.CrossRef Longhurst H, Bygum A. The humanistic, societal, and Pharmaco-economic burden of angioedema. Clinic Rev Allerg Immunol. 2016;51:230–9.CrossRef
6.
go back to reference Lumry WR, Castaldo AJ, Vernon MK, Blaustein MB, Wilson DA, Horn PT. The humanistic burden of hereditary angioedema: impact on health-related quality of life, productivity, and depression. Allergy Asthma Proc. 2010;31(5):407–14.CrossRefPubMedCentral Lumry WR, Castaldo AJ, Vernon MK, Blaustein MB, Wilson DA, Horn PT. The humanistic burden of hereditary angioedema: impact on health-related quality of life, productivity, and depression. Allergy Asthma Proc. 2010;31(5):407–14.CrossRefPubMedCentral
7.
go back to reference Bygum A, Aygören-Pürsün E, Beusterien K, Hautamaki E, Sisic Z, Wait S, et al. Burden of illness in hereditary angioedema: a conceptual model. Acta Derm Venereol. 2015;95:706–10.CrossRefPubMedCentral Bygum A, Aygören-Pürsün E, Beusterien K, Hautamaki E, Sisic Z, Wait S, et al. Burden of illness in hereditary angioedema: a conceptual model. Acta Derm Venereol. 2015;95:706–10.CrossRefPubMedCentral
8.
go back to reference Aygören-Pürsün E, Bygum A, Beusterien K, Hautamaki E, Sisic Z, Wait S, et al. Socioeconomic burden of hereditary angioedema: results from the hereditary angioedema burden of illness study in Europe. Orphanet J Rare Dis. 2014;9:99.CrossRefPubMedCentral Aygören-Pürsün E, Bygum A, Beusterien K, Hautamaki E, Sisic Z, Wait S, et al. Socioeconomic burden of hereditary angioedema: results from the hereditary angioedema burden of illness study in Europe. Orphanet J Rare Dis. 2014;9:99.CrossRefPubMedCentral
9.
go back to reference Aygören-Pürsün E, Bygum A, Beusterien K, Hautamaki E, Sisic Z, Boysen HB, et al. Estimation of EuroQol 5-dimensions health status utility values in hereditary angioedema. Patient Prefer Adherence. 2016;10:1699–707.CrossRefPubMedCentral Aygören-Pürsün E, Bygum A, Beusterien K, Hautamaki E, Sisic Z, Boysen HB, et al. Estimation of EuroQol 5-dimensions health status utility values in hereditary angioedema. Patient Prefer Adherence. 2016;10:1699–707.CrossRefPubMedCentral
10.
go back to reference Farkas H, Martinez-Saguer I, Bork K, Bowen T, Craig T, Frank M, et al. International consensus on the diagnosis and management of pediatric patients with hereditary angioedema with C1 inhibitor deficiency. Allergy. 2017;72(2):300–13.CrossRef Farkas H, Martinez-Saguer I, Bork K, Bowen T, Craig T, Frank M, et al. International consensus on the diagnosis and management of pediatric patients with hereditary angioedema with C1 inhibitor deficiency. Allergy. 2017;72(2):300–13.CrossRef
11.
go back to reference Longhurst HJ, Tarzi MD, Ashworth F, et al. C1 inhibitor deficiency: 2014 United Kingdom consensus document. Clin Exp Immunol. 2015;180(3):475–83.CrossRefPubMedCentral Longhurst HJ, Tarzi MD, Ashworth F, et al. C1 inhibitor deficiency: 2014 United Kingdom consensus document. Clin Exp Immunol. 2015;180(3):475–83.CrossRefPubMedCentral
12.
go back to reference Tse KY, Zuraw BL, Chen Q, Christiansen SC. Anabolic androgen use in the management of hereditary angioedema: Not so cheap after all. Ann Allergy Asthma. 2017;118(4):456–60.CrossRef Tse KY, Zuraw BL, Chen Q, Christiansen SC. Anabolic androgen use in the management of hereditary angioedema: Not so cheap after all. Ann Allergy Asthma. 2017;118(4):456–60.CrossRef
13.
go back to reference Craig TJ, Aygören-Pürsün E, Bork K, Bowen T, Boysen H, Farkas H, et al. WAO guideline for the management of hereditary angioedema. World Allergy Organ J. 2012;5:182–99.CrossRefPubMedCentral Craig TJ, Aygören-Pürsün E, Bork K, Bowen T, Boysen H, Farkas H, et al. WAO guideline for the management of hereditary angioedema. World Allergy Organ J. 2012;5:182–99.CrossRefPubMedCentral
14.
go back to reference Zuraw B, Cicardi M, Levy RJ, Nuijens JH, Relan A, Visscher S, et al. Recombinant human C1-inhibitor for the treatment of acute angioedema attacks in patients with hereditary angioedema. J Allergy Clin Immunol. 2010;126(4):821–7.CrossRef Zuraw B, Cicardi M, Levy RJ, Nuijens JH, Relan A, Visscher S, et al. Recombinant human C1-inhibitor for the treatment of acute angioedema attacks in patients with hereditary angioedema. J Allergy Clin Immunol. 2010;126(4):821–7.CrossRef
15.
go back to reference Longhurst H, Cicardi M, Craig T, Bork K, Grattan C, Baker J, et al. Prevention of hereditary angioedema attacks with a subcutaneous C1 inhibitor. N Engl J Med. 2017;376:1131–40.CrossRefPubMedCentral Longhurst H, Cicardi M, Craig T, Bork K, Grattan C, Baker J, et al. Prevention of hereditary angioedema attacks with a subcutaneous C1 inhibitor. N Engl J Med. 2017;376:1131–40.CrossRefPubMedCentral
16.
go back to reference Riedl MA, Banerji A, Busse PJ, Johnston DT, Davis-Lorton MA, Patel S, et al. Patient satisfaction and experience with intravenously administered C1-inhibitor concentrates in the United States. Ann Allergy Asthma Immunol. 2017;119(1):59–64.CrossRef Riedl MA, Banerji A, Busse PJ, Johnston DT, Davis-Lorton MA, Patel S, et al. Patient satisfaction and experience with intravenously administered C1-inhibitor concentrates in the United States. Ann Allergy Asthma Immunol. 2017;119(1):59–64.CrossRef
17.
go back to reference Wang A, Fouche A, Craig TJ. Patients perception of self-administered medication in the treatment of hereditary angioedema. Ann Allergy Asthma Immunol. 2015;115:120–5.CrossRefPubMedCentral Wang A, Fouche A, Craig TJ. Patients perception of self-administered medication in the treatment of hereditary angioedema. Ann Allergy Asthma Immunol. 2015;115:120–5.CrossRefPubMedCentral
18.
go back to reference Zuraw BL, Kalfus I. Safety and efficacy of prophylactic nanofiltered C1-inhibitor in hereditary angioedema. Am J Med. 2012;125(9):938.CrossRef Zuraw BL, Kalfus I. Safety and efficacy of prophylactic nanofiltered C1-inhibitor in hereditary angioedema. Am J Med. 2012;125(9):938.CrossRef
19.
go back to reference Craig TJ, Bewtra AK, Bahna SL, Hurewitz D, Schneider LC, Levy RJ, et al. C1 esterase inhibitor concentrate in 1085 hereditary angioedema attacks—final results of the I.M.P.a.C.T.2 study. Allergy. 2011;66(12):1604–11.CrossRefPubMedCentral Craig TJ, Bewtra AK, Bahna SL, Hurewitz D, Schneider LC, Levy RJ, et al. C1 esterase inhibitor concentrate in 1085 hereditary angioedema attacks—final results of the I.M.P.a.C.T.2 study. Allergy. 2011;66(12):1604–11.CrossRefPubMedCentral
20.
go back to reference Bygum A, Martinez-Saguer I, Bas M, Rosch J, Edelman J, Rojavin M, et al. Use of a C1 inhibitor concentrate in adults ≥65 years of age with hereditary angioedema: findings from the international Berinert® (C1-INH) registry. Drugs Aging. 2016;33(11):819–27.CrossRefPubMedCentral Bygum A, Martinez-Saguer I, Bas M, Rosch J, Edelman J, Rojavin M, et al. Use of a C1 inhibitor concentrate in adults ≥65 years of age with hereditary angioedema: findings from the international Berinert® (C1-INH) registry. Drugs Aging. 2016;33(11):819–27.CrossRefPubMedCentral
21.
go back to reference Riedl MA, Bygum A, Lumry W, Magerl M, Bernstein JA, Busse P, et al. Safety and usage of C1-inhibitor in hereditary angeioedema: Berinert registry data. J Allergy Clin Immunol Pract. 2016;4(5):963–71.CrossRefPubMedCentral Riedl MA, Bygum A, Lumry W, Magerl M, Bernstein JA, Busse P, et al. Safety and usage of C1-inhibitor in hereditary angeioedema: Berinert registry data. J Allergy Clin Immunol Pract. 2016;4(5):963–71.CrossRefPubMedCentral
22.
go back to reference Zanichelli A, Maurer M, Aberer W, Caballero T, Longhurst HJ, Bouillet L, et al. Long-term safety of icatibant treatment of patients with angioedema in real-world clinical practice. Allergy. 2017;72:994–8.CrossRefPubMedCentral Zanichelli A, Maurer M, Aberer W, Caballero T, Longhurst HJ, Bouillet L, et al. Long-term safety of icatibant treatment of patients with angioedema in real-world clinical practice. Allergy. 2017;72:994–8.CrossRefPubMedCentral
23.
go back to reference Cinryze® (C1 Esterase Inhibitor [Human]) prescribing information. Shire ViroPharma Incorporated. Lexington, MA, USA; 2018. Cinryze® (C1 Esterase Inhibitor [Human]) prescribing information. Shire ViroPharma Incorporated. Lexington, MA, USA; 2018.
24.
go back to reference Deyo RA, Cherkin DC, Ciol MA. Adapting a clinical comorbidity index for use with ICD-9-CM administrative databases. J Clin Epidemiol. 1992;45(6):613–9.CrossRefPubMedCentral Deyo RA, Cherkin DC, Ciol MA. Adapting a clinical comorbidity index for use with ICD-9-CM administrative databases. J Clin Epidemiol. 1992;45(6):613–9.CrossRefPubMedCentral
25.
go back to reference Charlson ME, Pompei P, Ales KL, MacKenzie CR. A new method of classifying prognostic comorbidity in longitudinal studies: development and validation. J Chronic Dis. 1987;40(5):373–83.CrossRefPubMedCentral Charlson ME, Pompei P, Ales KL, MacKenzie CR. A new method of classifying prognostic comorbidity in longitudinal studies: development and validation. J Chronic Dis. 1987;40(5):373–83.CrossRefPubMedCentral
26.
go back to reference Maki DG, Kluger DM, Crnich CJ. The risk of bloodstream infection in adults with different intravascular devices: a systematic review of 200 published prospective studies. Mayo Clin Proc. 2006;81(9):1159–71.CrossRefPubMedCentral Maki DG, Kluger DM, Crnich CJ. The risk of bloodstream infection in adults with different intravascular devices: a systematic review of 200 published prospective studies. Mayo Clin Proc. 2006;81(9):1159–71.CrossRefPubMedCentral
27.
go back to reference Walser EM. Venous access ports: indications, implantation technique, follow-up, and complications. Cardiovasc Intervent Radiol. 2012;35(4):751–64.CrossRefPubMedCentral Walser EM. Venous access ports: indications, implantation technique, follow-up, and complications. Cardiovasc Intervent Radiol. 2012;35(4):751–64.CrossRefPubMedCentral
28.
go back to reference Shim J, Seo TS, Song MG, Cha IH, Kim JS, Choi CW, et al. Incidence and risk factors of infectious complications related to implantable venous-access ports. Korean J Radiol. 2014;15(4):494–500.CrossRefPubMedCentral Shim J, Seo TS, Song MG, Cha IH, Kim JS, Choi CW, et al. Incidence and risk factors of infectious complications related to implantable venous-access ports. Korean J Radiol. 2014;15(4):494–500.CrossRefPubMedCentral
29.
go back to reference Nakamura T, Sasaki J, Asari Y, Sato T, Torii S, Watanabe M. Complications after implantation of subcutaneous central venous ports (PowerPort). Ann Med Surg (Lond). 2017;17:1–6.CrossRef Nakamura T, Sasaki J, Asari Y, Sato T, Torii S, Watanabe M. Complications after implantation of subcutaneous central venous ports (PowerPort). Ann Med Surg (Lond). 2017;17:1–6.CrossRef
30.
go back to reference Yoo SM, Khan DA. Implantable venous access device associated complications in patients with hereditary angioedema. J Allergy Clin Immunol Pract. 2013;1(5):524–5.CrossRef Yoo SM, Khan DA. Implantable venous access device associated complications in patients with hereditary angioedema. J Allergy Clin Immunol Pract. 2013;1(5):524–5.CrossRef
Metadata
Title
Treatment patterns and healthcare resource utilization among patients with hereditary angioedema in the United States
Authors
Marc A Riedl
Aleena Banerji
Michael E Manning
Earl Burrell
Namita Joshi
Dipen Patel
Thomas Machnig
Ming-Hui Tai
Douglas J Watson
Publication date
01-12-2018
Publisher
BioMed Central
Published in
Orphanet Journal of Rare Diseases / Issue 1/2018
Electronic ISSN: 1750-1172
DOI
https://doi.org/10.1186/s13023-018-0922-3

Other articles of this Issue 1/2018

Orphanet Journal of Rare Diseases 1/2018 Go to the issue