Safety and Usage of C1-Inhibitor in Hereditary Angioedema: Berinert Registry Data

Riedl, Marc A.; Bygum, Anette; Lumry, William R.; Magerl, Markus; Bernstein, Jonathan A.; Busse, Paula J.; Craig, Timothy J.; Frank, Michael M.; Edelman, Jonathan M.; Williams-Herman, Debora; Feuersenger, Henrike; Rojavin, Mikhail; Offenberger, J.; Levy, Robyn J.; Hurewitz, David; Li, H. Henry; Shapiro, Ralph; Davis, Aaron; Rosch, Jeffrey; Fox, J A; Janss, Gerti; Baker, James; Packer, Flint; Vegh, Art; Sher, Ellen; Wedner, James; Amrol, David J.; Gower, Richard; Silber, Glenn; Portnoy, Jay M.; Paris, Kenneth; Darter, Amy; Mumneh, Nayla; Petrov, Andrej A.; Schneider, Lynda C.; Martinez‐Saguer, Inmaculada; Staubach‐Renz, Petra; Maurer, Marcus; Baş, Murat; Aygören‐Pürsün, Emel; Wuillemin, Walter A.

doi:10.1016/j.jaip.2016.04.018

Cited by 53 publications

(38 citation statements)

References 33 publications

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“…Among older adult subjects in the registry, mean (8.8 IU/kg) and median (6.4 IU/kg) weight-based pnfC1-INH doses were notably lower than the recommended dose and also lower than the mean and median weight-based doses reported for 252 younger adults in the registry (12.9 and 12.5 IU/kg, respectively). Dosing patterns by geographic location identified a trend of lower mean weight-based pnfC1-INH doses for subjects in Europe relative to those in the USA, a pattern that was also demonstrated for the entire registry population, regardless of subject age [18]. Given that the older adult subgroup was skewed heavily toward European subjects, this presents another contributing factor to the relatively low mean pnfC1-INH doses observed in this subgroup as a whole.…”

Section: Discussionmentioning

confidence: 87%

“…The number of pnfC1-INH infusions per subject exhibited substantial variability, ranging from 1 to 612. Among the older adults, mean [SD] (8.8 [4.1] IU/kg) and median (6.4 IU/kg) pnfC1-INH doses were lower than those reported for the younger adults (12.9 [6.2] and 12.5 IU/kg, respectively) (Table 4) and were also the lowest of any age group in the registry [18]. …”

Section: Resultsmentioning

confidence: 97%

“…To evaluate the continued safety of pnfC1-INH administration within the real-world setting, the international Berinert Patient Registry (hereafter, ‘registry’) was designed to gather data on pnfC1-IHN treatment across all age groups [18]. The final registry dataset included a sufficient number of subjects aged ≥65 years (hereafter, ‘older adults’) to allow for analysis of usage patterns and safety in this population.…”

Section: Introductionmentioning

confidence: 99%

See 2 more Smart Citations

Use of a C1 Inhibitor Concentrate in Adults ≥65 Years of Age with Hereditary Angioedema: Findings from the International Berinert® (C1-INH) Registry

et al. 2016

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BackgroundTreatment of hereditary angioedema (HAE) in ‘older adults’ (those aged ≥65 years) has not been well studied. The international Berinert Patient Registry collected data on the use of intravenous plasma-derived, pasteurized, nanofiltered C1-inhibitor concentrate (pnfC1-INH; Berinert®/CSL Behring) in patients of any age, including many older adults.MethodsThis observational registry, conducted from 2010 to 2014 at 30 US and seven European sites, gathered prospective (post-enrollment) and retrospective (pre-enrollment) usage and adverse event (AE) data on subjects treated with pnfC1-INH.ResultsThe registry documented 1701 pnfC1-INH infusions in 27 older adults. A total of 1511 HAE attacks treated with pnfC1-INH administration were reported among 25 of the 27 (92.6 %) older adults. Among the older adults, mean (standard deviation [SD]) (8.8 [4.1] IU/kg) and median (6.4 IU/kg) pnfC1-INH doses were lower than those reported for 252 ‘younger adults’ (those aged <65 years: 12.9 [6.2], 12.5 IU/kg, respectively). A total of 19 AEs occurred in 8 of 23 (34.8 %) older adults with prospective data, for rates of 0.83 events per subject and 0.02 events per infusion, similar to corresponding rates in younger adults (0.91 and 0.03, respectively). None of the AEs were considered related to pnfC1-INH, and all but two events (prostatectomy, gastrointestinal bleeding) were mild or moderate in severity. Administration of pnfC1-INH outside of a healthcare setting was reported for 1609 infusions in 16 older adults, representing 94.6 % of all pnfC1-INH infusions in this age group. There were no recorded instances of difficulty with self-administration of intravenous pnfC1-INH.ConclusionsThese findings suggest a high degree of safety with intravenous pnfC1-INH use in older adults with HAE, regardless of administration setting. Trial Registration Clinicaltrials.gov NCT01108848.

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Section: Discussionmentioning

confidence: 87%

Section: Resultsmentioning

confidence: 97%

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

Use of a C1 Inhibitor Concentrate in Adults ≥65 Years of Age with Hereditary Angioedema: Findings from the International Berinert® (C1-INH) Registry

et al. 2016

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“…It is presented in 500 U vials of a lyophilized product for intravenous administration, which has to be stored at 2-25 C [8], and when reconstituted, a 50 IU/mL solution is formed. It has an excellent safety post-launch record after more than 35 years of its availability [54][55][56].…”

Section: Us Consensus For the Management Of Children With C1-inh-haementioning

confidence: 99%

“…® is currently approved by the FDA for the treatment of AE attacks in adults and adolescents, 12 years and older, with C1-INH-HAE, and by the European Medical Agencies (EMA) for the treatment of AE attacks and short-term prophylaxis in adults and children with C1-INH-HAE [56].…”

Section: Berinertmentioning

confidence: 99%

Short‐Term Prophylaxis in Odontostomatological, Maxillofacial and ENT Procedures in Patients with Hereditary Angioedema Due to C1‐Inhibitor Deficiency

Palomo¹,

Caballero²

2017

A Comprehensive Review of Urticaria and Angioedema

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Oestrogens, trauma, infections or stress has been described as triggers for angioedema (AE) attacks in patients with hereditary angioedema due to C1-inhibitor deficiency (C1-INH-HAE). Microtrauma can precipitate the onset of acute AE attacks, and thus, dental-oral procedures carry a high risk of triggering them and also an increased risk of death from asphyxiation due to the AE location. In the past, without proper specific treatment, the overall mortality after dental surgery in patients with C1-INH-HAE was up to 30-40%. Some dental-oral, medical and/or surgical procedures are susceptible to receive "short-term prophylaxis" (STP) in order to reduce the risk of AE. We describe the published case reports of dental-oral, maxillofacial and ear, nose and throat (ENT) procedures in patients with C1-INH-HAE. Different consensus algorithms and clinical guidelines have been published for managing dental-oral, maxillofacial and otolaryngological procedures (DOMFOPs) and will be reviewed below. Based on the clinical experience of the Department of Allergology of the University Hospital La Paz (Madrid) and the University General Hospital Nuestra Señora del Prado (Talavera de la Reina), these algorithms have been updated and modified. We advise to classify procedures according to the risk of producing AE as minor, intermediate and major risks.

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Immunodeficiency Syndromes

Schaffer¹,

Makhija²,

Paller³

2019

Harper's Textbook of Pediatric Dermatology

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Safety and Usage of C1-Inhibitor in Hereditary Angioedema: Berinert Registry Data

Cited by 53 publications

References 33 publications

Use of a C1 Inhibitor Concentrate in Adults ≥65 Years of Age with Hereditary Angioedema: Findings from the International Berinert® (C1-INH) Registry

Use of a C1 Inhibitor Concentrate in Adults ≥65 Years of Age with Hereditary Angioedema: Findings from the International Berinert® (C1-INH) Registry

Short‐Term Prophylaxis in Odontostomatological, Maxillofacial and ENT Procedures in Patients with Hereditary Angioedema Due to C1‐Inhibitor Deficiency

Immunodeficiency Syndromes

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