Burden of illness seen in hereditary angioedema in Japanese patients: Results from a patient reported outcome survey.

IF 1.1 Q2 MEDICINE, GENERAL & INTERNAL
Beverley Yamamoto, Daisuke Honda, Isao Ohsawa, Kazumasa Iwamoto, Takahiko Horiuchi, Atsushi Fukunaga, Junichi Maehara, Kouhei Yamashita, Michihiro Hide
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引用次数: 1

Abstract

Hereditary angioedema (HAE) is a potentially life-threatening rare disease, which is mainly caused by the deficiency or dysfunction of C1-esterase inhibitor, and characterized by spontaneous, recurrent episodes of edema in various parts of the body including internal organs and the laryngeal area. Delayed diagnosis and treatment increase the burdens and risks of this condition. The current study aimed to understand the burden of illness for HAE patients in Japan before and after diagnosis through a patient reported outcome survey. A survey instrument was distributed to 121 adult patients with HAE by a patient organization via HAE treating physicians between July and November in 2016. Seventy patients (57.9%) returned the questionnaire. Patients reported high levels of medical resource utilization, including emergency procedures and services. Episodes of receiving laparotomy were somewhat less after diagnosis with HAE than before, but no apparent difference in episodes of tracheotomy between before and after the diagnosis. The economic burden, including direct and indirect medical costs, was highest before diagnosis, but still perceived as substantial after diagnosis. Patients reported disruption of work and school life, with 40% reporting that they miss 10 or more days from work or education per year. Sixty percent of patients reported that HAE affected their daily activities. We concluded that HAE is associated with considerable physical, social, economic and psycho-social burdens even after diagnosis, and that higher attack frequency is associated with a heavy disease burden for patients in Japan.

日本遗传性血管性水肿患者的疾病负担:一项患者报告结果调查的结果。
遗传性血管性水肿(遗传性血管性水肿,HAE)是一种潜在危及生命的罕见疾病,主要由c1酯酶抑制剂缺乏或功能障碍引起,其特征是包括内脏和喉部在内的身体各部位自发性、反复发作的水肿。延误的诊断和治疗增加了这种疾病的负担和风险。目前的研究旨在通过一项患者报告的结果调查,了解日本HAE患者诊断前后的疾病负担。2016年7月至11月,由患者组织通过HAE治疗医师向121名成年HAE患者分发了一份调查工具。70例患者(57.9%)回复问卷。患者报告医疗资源利用率很高,包括急诊程序和服务。诊断为HAE后接受剖腹手术的次数比诊断前有所减少,但诊断前后气管切开术的次数无明显差异。经济负担,包括直接和间接医疗费用,在诊断前是最高的,但在诊断后仍然被认为是很大的。患者报告工作和学校生活受到干扰,40%的人报告他们每年有10天或更多的时间没有工作或上学。60%的患者报告HAE影响了他们的日常活动。我们的结论是,即使在诊断后,HAE也与相当大的身体、社会、经济和心理社会负担相关,并且在日本,较高的发作频率与患者沉重的疾病负担相关。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
Intractable & rare diseases research
Intractable & rare diseases research MEDICINE, GENERAL & INTERNAL-
CiteScore
2.10
自引率
0.00%
发文量
29
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