HPE 101 – July 2022 | Page 13

There have been great strides made in treatment options in the last 30 years . At the 2022 European Academy of Allergy & Clinical Immunology ( EAACI ) Congress , we saw data from both clinical trials and real-worldevidence including data from the open-label randomised Phase III APeX-2 trial 8 showing that patients over 12 years of age with HAE receiving the oral prophylactic berotralstat experienced fewer attacks that required treatment with injectable on-demand medication and used less on-demand medication ( doses / month ) to treat attacks from baseline . 8 This effect was observed and continued to improve throughout the 96-week trial . 8
Q What are the factors that need to be considered in the decision-making process for each patient and optimising adherence and outcomes ?
A When choosing a treatment regimen for patients , it is vital that healthcare professionals consider the patient ’ s lifestyle , preferences and requirements . The shared decision-making process between a healthcare professional and their patient is an opportunity to discuss the factual information on available treatments , treatment goals and suitability to the patient ’ s lifestyle .
Q What are the mainstays of treatment ?
A International consensus guidelines 9 recommend that patients are assessed at every visit for their eligibility for long-term prophylaxis to prevent attacks , alongside a choice of acute / on-demand treatment for use when a HAE attack occurs . The mainstays of an effective acute or prophylactic treatment includes factors such as ease of administration , speed of resolution of swellings or the degree to which attacks are prevented with minimal side effects .
Q Please tell us about some of the novel developments in management ( and how you see their impact and place in therapy )
A In the last 10 – 20 years , novel therapeutics targeting the vascular permeability pathway ( kinin-kallikrein system ) have provided efficient treatment options to prevent HAE attacks and to treat acute attacks . Of note , international consensus guidelines recommend three first-line treatments for the prevention of HAE attacks , providing patients with a greater variety of treatment options . These prophylactic treatments can allow patients greater time between attacks , and in some cases reducing severity of their attacks .
With greater time between attacks , patients are allowed to live a more normal life which does not always revolve around their attacks . They feel more empowered to attend important events and milestones such as family weddings , to work full-time , and travel abroad safely , and generally enjoy their lives without constant fear of an HAE attack . If pharmaceutical companies remain committed to the HAE community , and continue supporting studies prioritising real-world outcomes , there is an even greater opportunity on the horizon to improve the lives of people living with HAE .
Many clinical trials have also prioritised the evaluation of ‘ real-world outcomes ’ to measure the impact of HAE treatments , ensuring that novel treatments are tailored to patient preferences . In the UK , we have the Early Access to Medicines Scheme ( EAMS ). This gave the UK HAE network an opportunity to perform a survey and evaluate real-world outcomes for 54 patients across 12 UK centres who were initiated on the treatment Orladeyo ® ( berotralstat ) before its commercialisation . The data from this independent real-world study was presented at EAACI in July 2022 . 10 Through participation in the EAMS , HAE specialist centres such as Barts Health NHS Trust have been able to generate valuable real-world evidence , and allow patients with significant unmet needs to gain early access to innovative treatments , 10 which is another great step towards standardising care across the global HAE community .
References 1 NHS Commissioning Board . Clinical Commissioning Policy : Treatment of Acute Attacks in Hereditary Angiodema ( Adult ). 2013 . Reference : NHSCB / B09 / P / b 2 Bork K , Hardt J , Witzke G . Fatal laryngeal attacks and mortality in hereditary angioedema due to C1-INH deficiency . J Allergy Clin Immunol 2012 ; 130 ( 3 ): 692 – 7 . 3 Longhurst H , Bork K . Hereditary angioedema : an update on causes , manifestations and treatment . Br J Hosp Med 2019 ; 80 ( 7 ): 391 – 8 . 4 Henao M , Kraschnewski J . Diagnosis
and screening of patients with hereditary angioedema in primary care . Ther Clin Risk Manag 2016 ; 12:701 – 11 . 5 Bygum A et al . Disease Severity , Activity , Impact , and Control and How to Assess Them in Patients with Hereditary Angioedema . Proc 2014 ; 35:47 – 53 . 6 Caballero T et al . The humanistic burden of hereditary angioedema : results from the Burden of Illness Study in Europe . Allergy Asthma Proc 2014 ; 35 ( 1 ): 47 – 53 . 7 Devercelli G et al . Patient-Reported Burden of Hereditary Angioedema : Findings from a US Patient Survey .
Presented at the 2018 American Academy of Allergy , Asthma and Immunology ( AAAAI )/ World Allergy Organization ( WAO ) Joint Congress . 2 – 5 March 2018 , Orlando , FL . 8 Geng B et al . Improvement in Quality of Life and Hereditary Angioedema ( HAE ) Attack Rates Observed in Patients Treated with Long-term Berotralstat in the APeX-2 Study . Abstract presented at the European Academy of Allergy and Clinical Immunology ( EAACI ) Hybrid Congress in Prague , Czech Republic , 1 – 3 July 2022 . 9 Maurer M et al . The international WAO /
EAACI guideline for the management of hereditary angioedema – The 2021 revision and update . World Allergy Organization Journal 2022 ; 15.3 : 100627 . 10 Ahuja M et al . Berotralstat for the prophylaxis of hereditary angioedema – a national survey of patient outcomes in the UK . Poster presented at the European Academy of Allergy and Clinical Immunology ( EAACI ) Hybrid Congress in Prague , Czech Republic , 1 – 3 July 2022 .
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