PDF(Pubmed)
Abstract：
BACKGROUND: Hereditary angioedema (HAE) is a rare and potentially life-threatening disease that manifests clinically as recurrent episodes of swelling affecting multiple anatomical locations. Long-term prophylaxis (LTP) aims to control the disease by preventing HAE attacks. Previously, treatments such as attenuated androgens have been used for LTP, but they have an unfavorable adverse effect profile. Today, these limitations may be overcome by patients transitioning to newer, targeted therapies including oral berotralstat and subcutaneous lanadelumab. This case series reports the transition process between different prophylactic therapies in a family with HAE in a real-world setting.
RESULTS: Four adult patient cases from the same family who underwent transitions in HAE prophylaxis are presented. Three were female and one male. Two patients who transitioned to berotralstat were initially prescribed attenuated androgens. Two patients were not taking LTP at the time of initiating targeted treatment but had previously been prescribed tranexamic acid. The length of transition varied between the patients, with the longest time taken to stabilize on new therapy being 26 months. All patients received regular follow-up in person or by telephone and all four required an adjustment from their initial treatment plan.
CONCLUSIONS: Transitioning between LTP in HAE may help improve control of attacks, avoid unwanted adverse effects, or better cater to individual patient preferences. Newer targeted therapies have been shown to be effective and should be discussed with patients. Shared decision-making is a tool that can aid these discussions. The transition journey between LTP therapies in HAE may not be straightforward and is specific to each patient. Physicians should consider complicating factors such as patient anxieties around changing treatment, adverse effects, preferred routes of administration, and speed of transition. Following patients closely during the transition period helps identify any issues, including difficulties with treatment adherence, and may allow the transition plan to be adapted when necessary.
RESULTS: Four adult patient cases from the same family who underwent transitions in HAE prophylaxis are presented. Three were female and one male. Two patients who transitioned to berotralstat were initially prescribed attenuated androgens. Two patients were not taking LTP at the time of initiating targeted treatment but had previously been prescribed tranexamic acid. The length of transition varied between the patients, with the longest time taken to stabilize on new therapy being 26 months. All patients received regular follow-up in person or by telephone and all four required an adjustment from their initial treatment plan.
CONCLUSIONS: Transitioning between LTP in HAE may help improve control of attacks, avoid unwanted adverse effects, or better cater to individual patient preferences. Newer targeted therapies have been shown to be effective and should be discussed with patients. Shared decision-making is a tool that can aid these discussions. The transition journey between LTP therapies in HAE may not be straightforward and is specific to each patient. Physicians should consider complicating factors such as patient anxieties around changing treatment, adverse effects, preferred routes of administration, and speed of transition. Following patients closely during the transition period helps identify any issues, including difficulties with treatment adherence, and may allow the transition plan to be adapted when necessary.
摘要：
背景：遗传性血管性水肿（HAE）是一种罕见且可能危及生命的疾病，在临床上表现为反复发作的肿胀影响多个解剖部位。长期预防（LTP）旨在通过预防HAE发作来控制疾病。以前,减毒雄激素等治疗已用于LTP，但是它们有不利的不利影响。今天,这些限制可以通过患者过渡到较新的，靶向治疗包括口服berotralstat和皮下lanadelumab。本病例系列报告了在现实世界中患有HAE的家庭中不同预防疗法之间的过渡过程。
结果：介绍了来自同一家庭的4例经历了HAE预防过渡的成年患者病例。三个是女性，一个是男性。两名过渡到berotralstat的患者最初服用了减毒雄激素。两名患者在开始靶向治疗时没有服用LTP，但之前已经服用了氨甲环酸。患者之间的过渡长度不同，新疗法稳定所需的最长时间为26个月。所有患者都亲自或通过电话接受了定期随访，所有四名患者都需要对其初始治疗计划进行调整。
结论：HAE中LTP之间的转换可能有助于改善对发作的控制，避免不必要的不良影响，或更好地满足个人患者的喜好。较新的靶向治疗已被证明是有效的，应与患者讨论。共享决策是可以帮助这些讨论的工具。HAE中LTP疗法之间的过渡过程可能并不简单，并且对每个患者都是特定的。医生应该考虑复杂的因素，如患者对改变治疗的焦虑，不利影响，首选的给药途径，过渡的速度。在过渡期密切关注患者有助于识别任何问题，包括治疗依从性困难，并可能允许在必要时调整过渡计划。
结果：介绍了来自同一家庭的4例经历了HAE预防过渡的成年患者病例。三个是女性，一个是男性。两名过渡到berotralstat的患者最初服用了减毒雄激素。两名患者在开始靶向治疗时没有服用LTP，但之前已经服用了氨甲环酸。患者之间的过渡长度不同，新疗法稳定所需的最长时间为26个月。所有患者都亲自或通过电话接受了定期随访，所有四名患者都需要对其初始治疗计划进行调整。
结论：HAE中LTP之间的转换可能有助于改善对发作的控制，避免不必要的不良影响，或更好地满足个人患者的喜好。较新的靶向治疗已被证明是有效的，应与患者讨论。共享决策是可以帮助这些讨论的工具。HAE中LTP疗法之间的过渡过程可能并不简单，并且对每个患者都是特定的。医生应该考虑复杂的因素，如患者对改变治疗的焦虑，不利影响，首选的给药途径，过渡的速度。在过渡期密切关注患者有助于识别任何问题，包括治疗依从性困难，并可能允许在必要时调整过渡计划。
