Health-Related Quality of Life with Subcutaneous C1-Inhibitor for Prevention of Attacks of Hereditary Angioedema

Lumry, William R.; Craig, Timothy J.; Zuraw, Bruce L.; Longhurst, Hilary; Baker, James; Li, H. Henry; Bernstein, Jonathan A.; Anderson, J.; Riedl, Marc A.; Manning, M. A.; Keith, Paul K.; Levy, Donald S.; Caballero, Teresa; Banerji, Aleena; Gower, Richard; Farkas, Henriette; Lawo, John Philip; Pragst, Ingo; Machnig, Thomas; Watson, Douglas J.

doi:10.1016/j.jaip.2017.12.039

Cited by 74 publications

(80 citation statements)

References 47 publications

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“…It was hard for her to take care of the family, and she had difficulty attending school. After the disease was controlled, she was able to live her life fully, in accordance to the study of Lumry et al 7. The Lumry et al study showed that administrating subcutaneous C1-INH can improve many aspects of the burden of HAE such as anxiety, work productivity and activity impairment.…”

Section: Discussionsupporting

confidence: 72%

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New approach in prophylactic treatment of a challenged HAE patient

Jordal¹,

Bygum

2019

BMJ Case Rep

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Hereditary angioedema (HAE) is a relapsing swelling disorder which can cause severe pain, affect quality of life and potentially be life threatening with involvement of the airways. We present a 34-year-old immigrant who suffered from very frequent and severe HAE attacks. The attacks often involved the face, mouth and the airways. She often went to the hospital for treatment, where the language barrier made the situation complicated. The traditional therapy for HAE was not successful treating this patient. In June 2017, off-label treatment with prophylactic subcutaneous complement C1-inhibitor concentrate was initiated. The treatment was very successful and the patient has not been hospitalised since. Treatment for HAE is nowadays under investigation, and many drugs are under development. Especially, medication which works prophylactically and is administered orally or subcutaneously is in the horizon.

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Section: Discussionsupporting

confidence: 72%

“…Low values should be repeated to confirm the diagnosis 6. HAE can have severe impact on various aspects of health-related quality of life, because the disease is lifelong, unpredictable and debilitating 7. Untreated angioedema of the larynx is associated with a high mortality rate where fatal events may occur within 10 min to 14.3 hours 8.…”

Section: Introductionmentioning

confidence: 99%

New approach in prophylactic treatment of a challenged HAE patient

Jordal¹,

Bygum

2019

BMJ Case Rep

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“… 11 , 15 – 21 Study findings based on the European Quality of Life-5 Dimensions Questionnaire (EQ-5D) reveal that HAE attacks have a marked impact on perceived health status. As shown in Figure 2 , 16 , 20 , 22 – 28 EQ-5D scores reflecting HRQoL during an HAE attack, as reported in a study conducted in Denmark, Germany, and Sweden, ranged from 0.08 during a severely painful attack to 0.61 during an attack with mild pain (1 = full health). Even between HAE attacks, the mean EQ-5D scores reflected impairment (0.72).…”

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confidence: 72%

Advances in Hereditary Angioedema: The Prevention of Angioedema Attacks With Subcutaneous C1-Inhibitor Replacement Therapy

Lumry¹,

Templeton

Omert

et al. 2020

Journal of Infusion Nursing

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“…18 In a post hoc analysis of all patients randomized in the OLE (n = 90), Lumry et al reported on the benefit of C1INH (SC) of various patient-reported outcome QoL measures related to the burden of HAE such as clinically meaningful improvements in anxiety and depression scores, improved work productivity, and less nonwork activity impairment, as well as high treatment satisfaction and perceived effectiveness compared with on-demand treatment alone. 19 The frequency, severity, and location of attacks are major factors associated with negative QoL in children with HAE. 7 The inability to attend school and decreased involvement in social activities, as well as physical impairment result in reduced QoL of pediatric patients.…”

Section: Discussionmentioning

confidence: 99%

Long-Term Efficacy of Subcutaneous C1 Inhibitor in Pediatric Patients with Hereditary Angioedema

Levy

Caballero

Hussaın

et al. 2020

Pediatric Allergy, Immunology, and Pulmonology

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Background: Hereditary angioedema (HAE) due to C1 inhibitor (C1INH) deficiency is characterized by recurrent attacks of edema of the skin and mucosal tissues. Symptoms usually present during childhood (mean age at first attack, 10 years). Earlier symptom onset may predict a more severe disease course. Subcutaneous (SC) C1INH is indicated for routine prophylaxis to prevent HAE attacks in adolescents and adults. We analyzed the long-term efficacy of C1INH (SC) in subjects £17 years old treated in an open-label extension (OLE) of the pivotal phase III Clinical Study for Optimal Management of Preventing Angioedema with Low-Volume Subcutaneous C1 Inhibitor Replacement Therapy (COMPACT) trial. Methods: Eligible subjects (age ‡6 years, with ‡4 attacks over 2 consecutive months before entry into the OLE or placebo-controlled COMPACT trial) were treated with C1INH (SC) 40 or 60 IU/kg twice weekly for 52-140 weeks. Subgroup analyses by age (£17 vs. >17 years) were performed for key efficacy endpoints. Results: Ten subjects were £17 years old [mean (range) age, 13.3 (8-16) years, 3 subjects <12 years old; exposure range, 51-133 weeks]. All 10 pediatric subjects experienced ‡50% reduction (mean, 93%) in number of attacks versus the prestudy period, with a 97% reduction in the median number of attacks/month (0.11). All subjects had <1 attack/4-week period and 4 had <1 attack/year (1 subject was attack free). No subject discontinued treatment due to a treatment-related adverse event. Conclusions: Data from pediatric subjects treated with C1INH (SC) for up to 2.55 years and adult subjects revealed similar efficacy. C1INH (SC) is effective and well tolerated as long-term prophylaxis in children, adolescents, and adults with HAE.

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Health-Related Quality of Life with Subcutaneous C1-Inhibitor for Prevention of Attacks of Hereditary Angioedema

Cited by 74 publications

References 47 publications

New approach in prophylactic treatment of a challenged HAE patient

New approach in prophylactic treatment of a challenged HAE patient

Advances in Hereditary Angioedema: The Prevention of Angioedema Attacks With Subcutaneous C1-Inhibitor Replacement Therapy

Long-Term Efficacy of Subcutaneous C1 Inhibitor in Pediatric Patients with Hereditary Angioedema

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