Specialist Advice Support for Management of Severe Hereditary Angioedema Attacks: A Multicenter Cluster-Randomized Controlled Trial

Javaud, Nicolas; Fain, Olivier; Durand‐Zaleski, Isabelle; Launay, David; Bouillet, Laurence; Gompel, Anne; Sobel, Alain; Woimant, Maguy; Rabetrano, Hasina; Pétrovic, Tomislav; Lapostolle, Frédéric; Boccon‐Gibod, Isabelle; Bertrand, Philìppe; Mezaour, M.; Coppéré, B.; Floccard, Bernard; Kanny, G.; Baker, Edward; Martin, Ludovic; Vicaut, Éric; Adnet, Frédéric

doi:10.1016/j.annemergmed.2018.01.053

Cited by 7 publications

(4 citation statements)

References 18 publications

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“…Surprisingly, other studies have shown that most patients with idiopathic acquired angioedema have more than 12 episodes annually, whereas our study group had a mean of 1.4 episodes per year 14,16 . This discordance provides further justification that presentations of recurrent angioedema in our study population are significantly different from other studies.…”

Section: Discussioncontrasting

confidence: 83%

Recurrent angioedema: Experience at a tertiary care urban medical center

Kedarisetty

Tint

Michael

et al. 2021

Laryngoscope Investig Oto

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Objective To determine the demographics, presentation, management, and outcomes of patients with recurrent angioedema. To compare the findings to patients with ACE inhibitor related angioedema. Methods Retrospective case series with chart review of patients who presented to a tertiary‐care hospital between January 2010 and December 2017 with two or more episodes of angioedema. Excluded were patients with anaphylactic reaction, medication induced angioedema, or angioedema secondary to an infectious etiology. A group of 88 patients who presented during the same time period with ACE inhibitor related angioedema was used as a control. Statistical analysis was conducted using a two‐tailed Fisher exact test and a multivariate logistical regression model to determine significant associations. Results Ninety‐one patients were identified; 61 met the selection criteria and had 217 total episodes of angioedema episodes presenting to the emergency department. Fifty percent were Caucasian or Hispanic. The average number of episodes was 3.5 (range: 2‐23). The lips and tongue were the most commonly affected sites (37% and 39%). The larynx and floor of mouth were least likely to be involved (7% and 6%). Only 1 patient was found to have C1 esterase inhibitor deficiency. Twenty‐eight percent of patients had asthma, allergic rhinitis, food allergies, or atopic dermatitis. Only 11% of episodes required airway intervention. No patients required airway intervention after admission. Conclusion Recurrent angioedema was primarily idiopathic, was less severe than ACE inhibitor angioedema, and was associated with an atopic history. There was less frequent worsening of symptoms after admission, and recurrences occurred more frequently are at the same anatomic subsite. Level of Evidence IV.

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Section: Discussioncontrasting

confidence: 83%

Recurrent angioedema: Experience at a tertiary care urban medical center

Kedarisetty

Tint

Michael

et al. 2021

Laryngoscope Investig Oto

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“…It introduced the SOS-HAE intervention, which is a call center offering 24/7 access to specialized advice for patients. 24 The call center was staffed by emergency physicians trained in hereditary angioedema management. The ending result was a decrease in hospital admissions for hereditary angioedema attacks.…”

Section: Resultsmentioning

confidence: 99%

Health Services Interventions to Improve the Quality of Care in Rare Disease: A Scoping Review

Chou,

Wiredu,

Imhof

et al. 2024

Preprint

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BackgroundRare diseases are often complex, multi-system disorders requiring specialized, lifelong care. These diseases share significant challenges in healthcare delivery, including diagnostic delays, limited access to specialists, and lack of effective treatments.ObjectivesTo aggregate and critically examine innovative health services interventions for rare diseases, with the goal of identifying effective, scalable strategies to improve careDesignScoping reviewData SourcesComprehensive searches were conducted in April 2022 in Ovid MEDLINE,Embase.com, Web of Science Core Collection, Cochrane CENTRAL, andClinicalTrials.gov.MethodsWe sought to identify peer-reviewed original research published in English reporting results of interventions to improve guideline-concordant care, care coordination, and care transitions in rare disease populations. Using Covidence software, two researchers independently screened abstracts using pre-established inclusion and exclusion criteria, with conflicts resolved through consensus discussion with a third researcher. The same process was used to screen full-text research for eligibility and to extract study characteristics and results from eligible research.ResultsOur search identified 2899 articles. After screening for eligibility criteria, 12 articles describing health services interventions in rare diseases were identified. Most studies were conducted in Europe and involved adult participants. Three main intervention targets were identified: 1) increasing patients’ access to multidisciplinary expertise (e.g. using expert panels/tumor boards, integrating additional disciplines into care teams, and creating a hotline for specialist advice); 2) using technology to give point-of-care physicians access to information (e.g., electronic medical record templates/decision-support), and 3) standardizing care through clinical care pathways.ConclusionsThis review identified several efficacious interventions to improve healthcare delivery for individuals with a single rare disease. Testing these strategies across broader groups of rare disease patients could more efficiently improve healthcare delivery for the rare disease community, facilitating patients’ timely access to treatments, improving their health outcomes, and potentially reducing healthcare costs with economies of scale.Plain Language SummaryWhy was this study done?While each rare disease is unique and affects only a small number of individuals, the rare disease community as a whole faces similar challenges seeking healthcare, such as delayed diagnosis, limited access to specialists, and insufficient treatment options. This study seeks to find innovative strategies to enhance healthcare delivery that have been tested in at least one rare disease that might be applicable across a broad spectrum of these conditions.What did the researchers do?We reviewed public reports of research that aimed to improve guideline-concordant care, care coordination, or care transitions for people with rare diseases. At least two researchers screened each paper to assess whether it met inclusion and exclusion criteria, and all conflicts were resolved by consensus discussion.What did we find?After searching 2899, we found 12 describing successful interventions for people with rare diseases. These interventions either 1) increased patients’ access to multidisciplinary expertise (through things like expert review panels, integrating pharmacists into the care team, or creating a specialist hotline); 2) used technology to facilitate physicians access to rare disease information (through things like electronic medical record templates and alerts); or 3) standardize care delivery through creating set clinical pathways.What do these findings mean?These results highlight how efforts to improve access to multidisciplinary experts, implement new technology, and standardize care for rare disease could be used to develop impactful healthcare interventions that are transferable across multiple rare diseases.

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“…There is a paucity of studies exploring attack frequency and treatment costs based on prospectively collected data at the attack level. A recent multicentre cluster-randomised controlled study 40 investigated the impact on resource use and quality-adjusted life years (QALYs) of a dedicated telephone care-management service for patients with C1-INH-HAE. Results showed that the intervention significantly reduced the number of hospital admission in comparison with usual practice, thus leading to a reduction in overall hospital costs.…”

Section: Discussionmentioning

confidence: 99%

Costs and effects of on-demand treatment of hereditary angioedema in Italy: a prospective cohort study of 167 patients

et al. 2018

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ObjectivesTo explore treatment behaviours in a cohort of Italian patients with hereditary angioedema due to complement C1-inhibitor deficiency (C1-INH-HAE), and to estimate how effects and costs of treating attacks in routine practice differed across available on-demand treatments.DesignCost analyses and survival analyses using attack-level data collected prospectively for 1 year.SettingNational reference centre for C1-INH-HAE.Participants167 patients with proved diagnosis of C1-INH-HAE, who reported data on angioedema attacks, including severity, localisation and duration, treatment received, and use of other healthcare services.InterventionsAttacks were treated with either icatibant, plasma-derived C1-INH (pdC1-INH) or just supportive care.Main outcome measuresTreatment efficacy in reducing attack duration and the direct costs of acute attacks.ResultsOverall, 133 of 167 patients (79.6%) reported 1508 attacks during the study period, with mean incidence of 11 attacks per patient per year. Only 78.9% of attacks were treated in contrast to current guidelines. Both icatibant and pdC1-INH significantly reduced attack duration compared with no treatment (median times from onset 7, 10 and 47 hours, respectively), but remission rates with icatibant were 31% faster compared with pdC1-INH (HR 1.31, 95% CI 1.14 to 1.51). However, observed treatment behaviours suggest patterns of suboptimal dosing for pdC1-INH. The average cost per attack was €1183 (SD €789) resulting in €1.58 million healthcare costs during the observation period (€11 912 per patient per year). Icatibant was 54% more expensive than pdC1-INH, whereas age, sex and prophylactic treatment were not associated to higher or lower costs.ConclusionsBoth icatibant and pdC1-INH significantly reduced attack duration compared with no treatment, however, icatibant was more effective but also more expensive. Treatment behaviours and suboptimal dosing of pdC1-INH may account for the differences, but further research is needed to define their role.

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Specialist Advice Support for Management of Severe Hereditary Angioedema Attacks: A Multicenter Cluster-Randomized Controlled Trial

Cited by 7 publications

References 18 publications

Recurrent angioedema: Experience at a tertiary care urban medical center

Recurrent angioedema: Experience at a tertiary care urban medical center

Health Services Interventions to Improve the Quality of Care in Rare Disease: A Scoping Review

Costs and effects of on-demand treatment of hereditary angioedema in Italy: a prospective cohort study of 167 patients

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