Giménez-Arnau Ana Maria, Balp Maria-Magdalena, Danyliv Andrii, Winders Tonya, O'Donoghue James, Kleebach Jörn, Morrison Samantha, Walsh Shaun, Mueller Maike, Lopez-Ortiz Daniela, Maurer Marcus, Bernstein Jonathan A
Dermatology Department, Hospital del Mar Research Institute, IMIM, Universitat Pompeu Fabra, Barcelona, Spain.
Novartis Pharma AG, Basel, Switzerland.
Patient. 2025 Mar;18(2):173-185. doi: 10.1007/s40271-024-00725-3. Epub 2025 Jan 28.
In the context of injectable biologic products approved or in development for chronic spontaneous urticaria (CSU), it is important to capture which treatment attributes matter most to patient and what trade-offs patients are willing to make.
The CHOICE-CSU study aimed to quantify patient preferences toward injectable treatment attributes among patients with CSU, inadequately controlled by H1-antihistamines.
This was a two-phase cross-sectional patient preference study in adult patients with a diagnosis of CSU, inadequately controlled by H1-antihistamines. A qualitative phase collected patients' insights and relevant treatment attributes that mattered to them, and the outputs were used for the quantitative phase to create the actual injectable treatment profiles with attributes and levels such as: efficacy, safety, and mode of administration. The quantitative phase used discrete choice experiment (DCE) methodology. Eligible patients were asked to make hypothetical choices between 12 treatment profile pairs, created by Sawtooth Software. The DCE data were analyzed using hierarchical Bayesian logistic regression models, enabling the quantification of the relative importance of each attribute/level during the decision-making process.
A total of 450 respondents participated in the DCE. The key attributes driving respondent preference amongst injectable treatment options were type of administration device (relative importance 18.5%), complete control of urticaria (relative importance 17.4%), and resolution of angioedema (relative importance 16.4%). Keeping all other attributes and levels equal, the predicted choice share was higher for a profile with an auto-injector versus one with a pre-filled syringe (72.9% versus 27.1%).
The CHOICE-CSU study is the first study to provide a quantitative assessment of preferences that patients with CSU, inadequately controlled by H1-antihistamines, have for injectable treatment attributes. Symptom-free periods are the most important overriding therapy goal for patients, and patients will accept some inconveniences, such as administration mode, to achieve this. Additionally, when efficacy is equivalent, administration ease of injectable therapies is valued by patients. As new CSU oral treatment options emerge, additional testing of patient preference toward oral treatments will be required.
在已获批或正在研发用于慢性自发性荨麻疹（CSU）的注射用生物制品背景下，了解哪些治疗属性对患者最为重要以及患者愿意做出哪些权衡至关重要。
CHOICE-CSU研究旨在量化CSU患者对注射治疗属性的偏好，这些患者使用H1抗组胺药控制不佳。
这是一项针对诊断为CSU且使用H1抗组胺药控制不佳的成年患者的两阶段横断面患者偏好研究。定性阶段收集了患者的见解以及对他们重要的相关治疗属性，其结果用于定量阶段，以创建具有诸如疗效、安全性和给药方式等属性及水平的实际注射治疗概况。定量阶段使用离散选择实验（DCE）方法。符合条件的患者被要求在由Sawtooth软件创建的12对治疗概况之间做出假设选择。使用分层贝叶斯逻辑回归模型分析DCE数据，从而能够量化决策过程中每个属性/水平的相对重要性。
共有450名受访者参与了DCE。在注射治疗选项中驱动受访者偏好的关键属性是给药装置类型（相对重要性18.5%）、荨麻疹的完全控制（相对重要性17.4%）和血管性水肿的消退（相对重要性16.4%）。在所有其他属性和水平相同的情况下，与预填充注射器相比，自动注射器治疗概况的预测选择份额更高（72.9%对27.1%）。
CHOICE-CSU研究是第一项对使用H1抗组胺药控制不佳的CSU患者对注射治疗属性的偏好进行定量评估的研究。无症状期是患者最重要的首要治疗目标，患者愿意接受一些不便之处，如给药方式，以实现这一目标。此外，当疗效相当时，患者重视注射疗法的给药便利性。随着新的CSU口服治疗选项出现，将需要对患者对口服治疗的偏好进行额外测试。
