treatment goals

治疗目标
  • 文章类型: Journal Article
    背景:遗传性血管性水肿(HAE)是一种罕见的,以皮下或粘膜下血管性水肿反复发作为特征的危及生命的遗传性疾病。HAE治疗的最终目标仍然不明确。
    目的:本德尔菲过程的目的是确定HAE治疗的目标,并检查在评估疾病控制和患者生活正常化时应考虑哪些因素。
    方法:Delphi小组由23名参与者组成,他们是根据参与HAE科学研究或世界过敏组织/欧洲过敏与临床免疫学学会HAE指南的最新更新和修订而选择的。该过程包括3轮投票。最后一轮旨在汇总专家小组的意见并达成共识。
    结果:在第2轮中,根据第1轮收到的答复,提出了两个直接的共识问题,专家组一致认为治疗的目标是实现疾病的完全控制和患者生活的正常化。对于第三轮投票,审议了21项声明,与会者就18日达成共识。从广泛的共识声明中可以清楚地看出,在评估疾病控制和患者生活正常化时,应考虑疾病和治疗的负担。
    结论:HAE治疗的最终目标是不发生血管性水肿。在过去的十年中,改进的治疗和疾病管理的可用性现在使完全控制HAE成为大多数患者的现实可能性。
    BACKGROUND: Hereditary angioedema (HAE) is a rare, life-threatening genetic disorder characterized by recurrent episodes of subcutaneous or submucosal angioedema. The ultimate goals of treatment for HAE remain ill-defined.
    OBJECTIVE: The aim of this Delphi process was to define the goals of HAE treatment and to examine which factors should be considered when assessing disease control and normalization of the patient\'s life.
    METHODS: The Delphi panel comprised 23 participants who were selected based on involvement with scientific research on HAE or coauthorship of the most recent update and revision of the World Allergy Organization/European Academy of Allergy and Clinical Immunology guideline on HAE. The process comprised 3 rounds of voting. The final round aimed to aggregate the opinions of the expert panel and to achieve consensus.
    RESULTS: Two direct consensus questions were posed in round 2, based on the responses received in round 1, and the panel agreed that the goals of treatment are to achieve total control of the disease and to normalize the patient\'s life. For the third round of voting, 21 statements were considered, with the participants reaching consensus on 18. It is clear from the wide-ranging consensus statements that the burdens of disease and treatment should be considered when assessing disease control and normalization of patients\' lives.
    CONCLUSIONS: The ultimate goal for HAE treatment is to achieve no angioedema attacks. The availability of improved treatments and disease management over the last decade now makes complete control of HAE a realistic possibility for most patients.
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