Cost-Effectiveness of Prophylactic Medications for the Treatment of Hereditary Angioedema Due to C1 Inhibitor Deficiency: A Real-World U.S. Perspective

Zuraw, Bruce L.

doi:10.18553/jmcp.2019.25.2.148

Cited by 6 publications

(6 citation statements)

References 14 publications

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“…In 2013, Zanichelli et al reported that the median delay in diagnosis of HAE type I and II was 8.5 years in Europe [ 2 ]. The median age at first symptoms was 12.0 years, and the corresponding median age at diagnosis was 24.3 years [ 5 ]. The disease is frequently misdiagnosed and mistreated, leading to several unnecessary procedures that may potentially mask severe life-threatening complications such as laryngeal edema.…”

Section: Discussionmentioning

confidence: 99%

Extremely Late Diagnosis of Hereditary Angioedema Type I in an Elderly Female

Estaris,

Ostroukhova

2024

Cureus

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This case presents an instance of an extremely delayed diagnosis of hereditary angioedema (HAE) type I in an elderly female with no significant past medical history. The patient had a prolonged history of recurrent lip swelling and itchiness dating back to her teenage years, leading to multiple visits to the emergency room (ER). These recurrent episodes were characterized by random onset and accompanied by generalized pruritus and urticaria. During these ER visits, the patient would be inappropriately treated for presumed hypersensitivity reaction due to her confounding environmental allergies presenting with urticaria, complicating and significantly delaying her diagnosis. The patient was adopted, and the family history was unknown. There was no history of medication use suggestive of acquired angioedema. At the time of the visit, she had signs of chronic lip changes and atopy. After an extensive workup, it showed severely low levels of C1 esterase inhibitor and borderline low to normal C4 and C1q, consistent with the diagnosis of HAE type I. Initial treatment with an on-demand C1 esterase inhibitor reduced the recurrence of lip swelling and transitioned to long-term prophylaxis use. Overall, the treatment outcome was generally successful, with less recurrence of lip swelling and ER visits.

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Section: Discussionmentioning

confidence: 99%

Extremely Late Diagnosis of Hereditary Angioedema Type I in an Elderly Female

Estaris,

Ostroukhova

2024

Cureus

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“…Recurrent AE disease activity can vary strongly over time, and insufficiently controlled disease goes along with an increased risk for life‐threatening AE episodes and major impairment of patients' quality of life . On the other hand, an adjustment of treatment may increase treatment costs, for example when a CSU patient's therapy is changed from an antihistamine to omalizumab or when a prophylaxis with a C1 inhibitor or lanadelumab is initiated in HAE patients . Accordingly, the assessment of disease control in recurrent AE should be performed by using a valid and reliable approach.…”

Section: Discussionmentioning

confidence: 99%

“…6,[16][17][18][19][20][21] On the other hand, an adjustment of treatment may increase treatment costs, for example when a CSU patient's therapy is changed from an antihistamine to omalizumab or when a prophylaxis with a C1 inhibitor or lanadelumab is initiated in HAE patients. 22,23 Accordingly, the assessment of disease control in recurrent AE should be performed by using a valid and reliable approach. This ensures that changes in treatment are done in the right (undertreated) patients and that the reasons for keeping or changing a therapy are welldocumented.…”

Section: Discussionmentioning

confidence: 99%

Development of the Angioedema Control Test—A patient‐reported outcome measure that assesses disease control in patients with recurrent angioedema

Weller

Donoso

Magerl

et al. 2020

Allergy

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Background: Recurrent angioedema (AE) is an important clinical problem in the context of chronic urticaria (mast cell mediator-induced), ACE-inhibitor intake and hereditary angioedema (both bradykinin-mediated). To help patients obtain control of their recurrent AE is a major treatment goal. However, a tool to assess control of recurrent AE is not yet available. This prompted us to develop such a tool, the Angioedema Control Test (AECT).Methods: After a conceptional framework was developed for the AECT, a list of potential AECT items was generated by a combined approach of patient interviews, literature review and expert input. Subsequent item reduction was based on impact analysis, inter-item correlation, additional predefined criteria for item performance, and a review of the item selection process for content validity. Finally, an instruction section was generated, and an US-American-English version was developed by a structured translation process. Results:A 4-item AECT with recall periods of 4 weeks and 3 months was developed based on 106 potential items tested in 97 patients with mast cell mediator-induced (n = 49) or bradykinin-mediated recurrent AE (n = 48). Eighty-four items were excluded based on impact analysis. The remaining 22 items could be further reduced by a method-mix of inter-item correlation, additional predefined criteria for item performance and review for content validity. Conclusions:The AECT is the first tool to assess disease control in recurrent AE patients. Its retrospective approach, its brevity and its simple scoring make the AECT ideally suited for clinical practice and trials. Its validity and reliability need to be determined in future independent studies. K E Y W O R D SAECT, angioedema, development, disease control, impact analysis, item selection

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“…156,159,160 Self-administration of on-demand treatments decreases cost by reducing hospital visits. 153,154 A recent study examining the economics of HAE treatments found that the newest prophylactic agents lead to clinically significant improvements in QoL and avoid the high direct (medical) and indirect (socioeconomic) costs associated with the on-demand only treatment model 161 (Castaldo et al, manuscript submitted, 2020). The treating physician may need to write letters of medical necessity to support HAE patient access to effective medications.…”

Section: Economic Costsmentioning

confidence: 99%