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Allergy/immunology specialists in the United States prescribing allergy immunotherapy (AIT) have placed a heavy value on practical experience and anecdotal evidence rather than research-based evidence. With the extensive research on AIT conducted in the last few decades, the time has come to better implement evidence-based medicine (EBM) for AIT. The goal of this review was to critically assess EBM for debated concepts in US AIT practice for respiratory allergies in the context and quality of today's regulatory standards. Debated topics reviewed were the efficacy and safety of AIT in various subgroups (eg, polyallergic patients, older patients, patients with asthma, and pregnant women), diagnosis topics (eg, skin prick test vs allergen-specific serum IgE, factors affecting skin prick tests, use of nasal or conjunctival allergen challenges, and telemedicine for diagnosis), and dosing topics (eg, optimal dosing for subcutaneous immunotherapy and sublingual immunotherapy tablets, US liquid allergen extract history, duration of treatment, and biomarkers of efficacy). In addition, EBM for patient-centered AIT issues (eg, adherence, use of practice guidelines, and pharmacoeconomics) and the approach to implementation of AIT EBM in future clinical practice were addressed. The EBM for each concept was briefly summarized, and when possible, a practical, concise recommendation was given., (Copyright © 2022 The Authors. Published by Elsevier Inc. All rights reserved.)

Background: Hereditary angioedema (HAE) is often caused by low serum levels or functional deficiency in C1 inhibitor (C1-INH); however, in some cases, C1-INH serum level and function are measured as normal (HAE-nl-C1INH). Management of HAE-nl-C1INH is similar to management of HAE with C1-INH deficiency, including on-demand therapy for angioedema attacks and, potentially, prophylaxis. Recombinant human C1 esterase inhibitor (rhC1-INH) is indicated for treatment of acute HAE attacks. This study assessed the clinical profile and treatment outcomes in an HAE-nl-C1INH population with a history of rhC1-INH treatment., Methods: Medical records containing patient-reported outcomes from ten US treatment centers were analyzed retrospectively for medical history, angioedema attack characteristics, attack treatments, and clinical outcomes., Results: Twenty-three patients were included, with wide US geographic representation. Most patients (87.0%) were female; median age was 36.0 years (range, 19-67 years). Of 20 patients with available data, 4 had their first angioedema attack during childhood (aged <12 years), 3 during adolescence (aged 12-17 years), and 13 during adulthood (aged 18-29 years, n = 7; aged ≥30 years, n = 6). Median age at HAE-nl-C1INH diagnosis was 31.5 years (range, 9-59 years). Previous failed treatments included high-dose antihistamines (n = 20) and corticosteroids (n = 20). Use of US Food and Drug Administration (FDA)-approved HAE therapy positively impacted patient-reported assessments of angioedema attacks. Most patients were taking rhC1-INH or lanadelumab as prophylaxis and icatibant or rhC1-INH for acute management. Most patients reported improved disease control with these therapies, including reductions in angioedema attack frequency and severity. Although most patients were receiving prophylactic therapy, availability of treatment for breakthrough attacks was important., Conclusion: Findings from this retrospective study support use of FDA-approved HAE medications for prophylaxis and acute treatment of HAE attacks in patients with HAE-nl-C1INH. Individualized HAE treatment regimens were needed to optimize therapeutic outcomes., Competing Interests: Douglas H. Jones reports being a consultant for and receiving consulting fees from BioCryst, Pharming Technologies BV, and Takeda Pharmaceutical Company Ltd. Priya Bansal reports serving as a speaker and consultant for and receiving speaker honoraria from AstraZeneca and ALK; serving as a speaker for and receiving speaker honoraria from Regeneron and Teva Pharmaceutical Industries Ltd; serving as a consultant for and receiving consulting fees from Pfizer Inc.; and serving as a consultant and speaker for and receiving consulting fees and speaker honoraria from CSL Behring, Pharming Technologies BV, and Takeda Pharmaceutical Company Ltd. Jonathan A. Bernstein reports serving as a consultant for, receiving consulting fees from, and performing research for BioCryst, KalVista Pharmaceuticals, and Ionis Pharmaceuticals; and serving as a consultant and speaker for, receiving consulting fees and speaker honoraria from, and performing research for CSL Behring, Pharming Technologies BV, and Takeda; and serving as a board member for AAAAI Foundation (vice chair), World Allergy Organization, Joint Task Force, Allergists for Israel (chair), and Interasma. Shahnaz Fatteh reports serving as a consultant, speaker, and advisory board member for Pharming Technologies BV; and serving on the Broward County Medical Association. Joseph Harper and Nami Park report being employees of Pharming Healthcare Inc. F. Ida Hsu reports serving as a consultant for and receiving consulting fees from BioCryst and Takeda Pharmaceutical Company Ltd; serving as a speaker for and receiving speaker honoraria from CSL Behring and Pharming Technologies BV; and performing contracted research for Takeda Pharmaceutical Company Ltd. Maeve O'Connor reports serving as a consultant and speaker for and receiving consulting fees and speaker honoraria from AstraZeneca, BioCryst, CSL Behring, GlaxoSmithKline, Pharming Technologies BV, Sanofi, and Teva Pharmaceuticals Industries Ltd. Daniel Suez reports serving as a consultant for and receiving consulting fees from Pharming Technologies BV; and performing contracted research for BioCryst., (© 2021 The Authors.)