TY - JOUR
T1 - Long-term prophylaxis therapy in patients with hereditary angioedema with C1 inhibitor deficiency
AU - Craig, Timothy
AU - Busse, Paula
AU - Gower, Richard G.
AU - Johnston, Douglas T.
AU - Kashkin, Jay M.
AU - Li, Huamin H.
AU - Lumry, William R.
AU - Riedl, Marc A.
AU - Soteres, Daniel
N1 - Funding Information:
Funding Sources: Medical writing support was funded by CSL Behring. The funding agency was not involved in the writing of the manuscript and did not review the manuscript before submission.
Publisher Copyright:
© 2018 Elsevier Ltd
PY - 2018/12
Y1 - 2018/12
N2 - Objective: To review the criteria for long-term prophylaxis therapy in patients with hereditary angioedema due to C1 inhibitor deficiency (C1-INH-HAE), describe how these criteria have evolved over time, and anticipate how criteria may change in the future with the availability of new C1-INH-HAE treatment options. Data Sources: Treatment guidelines, consensus statements, and expert reviews. Study Selections: Manuscripts that described long-term prophylaxis therapy in patients with C1-INH-HAE were selected. Results: Historically, patients with C1-INH-HAE were considered to be candidates for long-term prophylaxis therapy if they had at least 1 attack per month, had at least 5 days of disability per month because of C1-INH-HAE, or did not sufficiently respond to on-demand treatment. More recently, guidelines and reviews state that thresholds of number of attacks or days of disability are arbitrary and that treatment plans should be individualized to the patient's needs. Furthermore, all patients should have a comprehensive management plan that is reviewed periodically and should have at least 2 doses of on-demand treatment available. Prophylaxis therapy should be discussed as a potential treatment option for each patient; however, the decision for its use will depend on the patient's individual needs and the course of their symptoms. Conclusion: The criteria for long-term prophylaxis therapy in C1-INH-HAE have changed with the recognition that treatments should be individualized to the patient's needs and with the availability of new medications that have more favorable benefit-risk profiles, are easier to use, and improve patients’ quality of life.
AB - Objective: To review the criteria for long-term prophylaxis therapy in patients with hereditary angioedema due to C1 inhibitor deficiency (C1-INH-HAE), describe how these criteria have evolved over time, and anticipate how criteria may change in the future with the availability of new C1-INH-HAE treatment options. Data Sources: Treatment guidelines, consensus statements, and expert reviews. Study Selections: Manuscripts that described long-term prophylaxis therapy in patients with C1-INH-HAE were selected. Results: Historically, patients with C1-INH-HAE were considered to be candidates for long-term prophylaxis therapy if they had at least 1 attack per month, had at least 5 days of disability per month because of C1-INH-HAE, or did not sufficiently respond to on-demand treatment. More recently, guidelines and reviews state that thresholds of number of attacks or days of disability are arbitrary and that treatment plans should be individualized to the patient's needs. Furthermore, all patients should have a comprehensive management plan that is reviewed periodically and should have at least 2 doses of on-demand treatment available. Prophylaxis therapy should be discussed as a potential treatment option for each patient; however, the decision for its use will depend on the patient's individual needs and the course of their symptoms. Conclusion: The criteria for long-term prophylaxis therapy in C1-INH-HAE have changed with the recognition that treatments should be individualized to the patient's needs and with the availability of new medications that have more favorable benefit-risk profiles, are easier to use, and improve patients’ quality of life.
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U2 - 10.1016/j.anai.2018.07.025
DO - 10.1016/j.anai.2018.07.025
M3 - Review article
C2 - 30056152
AN - SCOPUS:85054727065
SN - 1081-1206
VL - 121
SP - 673
EP - 679
JO - Annals of Allergy, Asthma and Immunology
JF - Annals of Allergy, Asthma and Immunology
IS - 6
ER -