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斑秃成人和青少年患者的治疗偏好:离散选择实验。

Treatment preferences of adults and adolescents with alopecia areata: A discrete choice experiment.

机构信息

Kielo Research, Zug, Switzerland.

Evidera Inc., London, UK.

出版信息

J Dermatol. 2024 Feb;51(2):243-252. doi: 10.1111/1346-8138.17056. Epub 2023 Dec 12.

Abstract

PRODUCTS with janus kinase (JAK) inhibition have been shown to promote hair regrowth in patients with alopecia areata (AA). To guide drug-approval and treatment decisions, it is important to understand patients' willingness to accept the potential risks of JAK inhibition in exchange for potential benefits. We quantified the treatment preferences of adult (≥18 years) and adolescent patients (12-17 years) with AA in the US and Europe to determine the trade-offs they are willing to make between benefits and risks. Preferences for oral AA treatment attributes were elicited using a discrete choice experiment consisting of 12 tasks in which patients chose between two hypothetical treatment alternatives and no treatment. Benefits included the probability of 80%-100% scalp hair regrowth (Severity of Alopecia Tool score ≤ 20) and achieving moderate-to-normal eyebrow and eyelash hair. Treatment-related risks included 3-year probabilities of serious infection, cancer, and blood clots. Preference estimates were used to calculate the maximum level of each risk that patients were willing to accept for increases in treatment benefits. The most important attribute to both adults (n = 201) and adolescents (n = 120) was a 50% probability of achieving hair regrowth on most or all the scalp; however, adolescents placed greater relative importance on this attribute than did adults. Adults were averse to the risks of serious infection, cancer, and blood clots, whereas adolescents were averse to the risk of cancer. For a 20% increase in the probability of 80%-100% scalp hair regrowth, adults were willing to accept a mean (95% confidence interval) 3-year risk of serious infection, cancer, and blood clots of 7.4% (5.5-9.3), 2.5% (1.9-3.1), and 9.3% (6.4-12.2). Adolescents were willing to accept a 3-year risk of cancer of 3.3% (2.4-4.2). Patients with AA in the US and Europe are willing to accept substantial risks to obtain an effective treatment.

摘要

已证实,具有 Janus 激酶(JAK)抑制作用的产品可促进斑秃(AA)患者的头发生长。为了指导药物批准和治疗决策,了解患者对 JAK 抑制的潜在风险的接受意愿,以换取潜在益处非常重要。我们量化了美国和欧洲 AA 成年(≥18 岁)和青少年(12-17 岁)患者的治疗偏好,以确定他们在收益和风险之间愿意做出的权衡。使用包含 12 项任务的离散选择实验来评估 AA 口服治疗属性的偏好,在这些任务中,患者在两种假设的治疗选择和无治疗之间进行选择。益处包括 80%-100%头皮毛发再生(脱发严重程度工具评分≤20)和实现中度至正常眉毛和睫毛毛发的概率。治疗相关风险包括 3 年内发生严重感染、癌症和血栓的概率。偏好估计用于计算患者愿意为治疗获益增加而接受的每种风险的最大水平。对成年人(n=201)和青少年(n=120)而言,最重要的属性都是获得大部分或全部头皮毛发再生的概率为 50%;然而,青少年比成年人更看重这一属性。成年人对严重感染、癌症和血栓的风险感到厌恶,而青少年对癌症的风险感到厌恶。为了将 80%-100%头皮毛发再生的概率提高 20%,成年人愿意接受的平均(95%置信区间)3 年内严重感染、癌症和血栓的风险为 7.4%(5.5-9.3)、2.5%(1.9-3.1)和 9.3%(6.4-12.2)。青少年愿意接受 3 年内癌症风险为 3.3%(2.4-4.2)。美国和欧洲的 AA 患者愿意接受重大风险以获得有效的治疗。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/97a7/11483896/d7a32d14efb2/JDE-51--g002.jpg

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