Considerations for transition from subcutaneous to oral prophylaxis in the treatment of hereditary angioedema
Open Access
- 9 October 2021
- journal article
- research article
- Published by Springer Science and Business Media LLC in Allergy, Asthma & Clinical Immunology
- Vol. 17 (1), 1-7
- https://doi.org/10.1186/s13223-021-00603-9
Abstract
Hereditary angioedema (HAE) is a rare genetic disorder characterized by unpredictable localized episodes of edema, which is frequently managed with long-term prophylactic medications. Until recently, long-term prophylaxis has predominantly required regular intravenous or subcutaneous administration, however the recent approval of berotralstat (Orladeyo™) offers an orally administered prophylactic which may be associated with a lower burden of treatment compared to injectable options for some patients. This report describes four participants in the APeX-S trial who transitioned from subcutaneously administered lanadelumab (Takhzyro®) to daily oral berotralstat for long-term HAE prophylaxis. Lanadelumab dosing continued after berotralstat commencement in all patients and was tapered before discontinuation in three of the four patients. No substantial increases in HAE attack rates were observed after the transition to berotralstat monotherapy. One patient experienced a treatment-related adverse event (dyspepsia), which was mild and self-resolving. All four patients described in this case series successfully transitioned from lanadelumab to berotralstat monotherapy for long-term prophylaxis without significant complications and without the use of a complex transition protocol. The decision to transition to berotralstat monotherapy and how the transition should be achieved was discussed between patient and physician, ensuring that the comfort and perspectives of the patients were considered during the treatment transition. This report highlights the importance of individualization of HAE management plans to address both the disease and treatment burdens of HAE, and thus to provide the best possible quality of life for each patient.Keywords
Funding Information
- BioCryst Pharmaceuticals, Inc.
This publication has 27 references indexed in Scilit:
- Systematic Literature Review and Meta-analysis of Medication Adherence With Once-weekly Versus Once-daily TherapyClinical Therapeutics, 2015
- Characterization of Anaphylaxis After Ecallantide Treatment of Hereditary Angioedema AttacksThe Journal of Allergy and Clinical Immunology: In Practice, 2014
- Abdominal attacks and treatment in hereditary angioedema with C1-inhibitor deficiencyBMC Gastroenterology, 2014
- Current Status of Implementation of Self-Administration Training in Various Regions of Europe, Canada and the USA in the Management of Hereditary AngioedemaInternational Archives of Allergy and Immunology, 2013
- Fatal laryngeal attacks and mortality in hereditary angioedema due to C1-INH deficiencyJournal of Allergy and Clinical Immunology, 2012
- The humanistic burden of hereditary angioedema: Impact on health-related quality of life, productivity, and depressionAllergy and Asthma Proceedings, 2010
- Fear of needles Nature and prevalence in general practice2009
- Factors affecting therapeutic compliance: A review from the patient’s perspectiveTherapeutics and Clinical Risk Management, 2008
- Clinical Studies of Sudden Upper Airway Obstruction in Patients With Hereditary Angioedema Due to C1 Esterase Inhibitor DeficiencyArchives of Internal Medicine, 2003
- Patient adherence to treatment: three decades of research. A comprehensive reviewJournal of Clinical Pharmacy & Therapeutics, 2001